Study to Find a Safe Dose and Show Early Clinical Activit... | NCT01962103 | Trialant
NCT01962103
Sponsor
Celgene
Status
Completed
Last Update Posted
Dec 27, 2019Actual
Enrollment
107Actual
Phase
Phase 1Phase 2
Conditions
Neuroblastoma
Rhabdomyosarcoma
Ewing's Sarcoma
Ewing's Tumor
Sarcoma, Ewing's
Sarcomas, Epitheliod
Sarcoma, Soft Tissue
Sarcoma, Spindle Cell
Melanoma
Malignant Melanoma
Clinical Oncology
Oncology, Medical
Pediatrics, Osteosarcoma
Osteogenic Sarcoma
Osteosarcoma Tumor
Sarcoma, Osteogenic
Tumors
Cancer
Neoplasia
Neoplasm
Histiocytoma
Fibrosarcoma
Dermatofibrosarcoma
Interventions
nab-paclitaxel
nab-paclitaxel
Countries
United States
Canada
France
Italy
Spain
Switzerland
United Kingdom
Protocol Section
Identification Module
NCT ID
NCT01962103
Obsolete or Duplicate NCT IDs
Not provided
Organization Study
ABI-007-PST-001
Secondary IDs
Not provided
Brief Title
Study to Find a Safe Dose and Show Early Clinical Activity of Weekly Nab-paclitaxel in Pediatric Patients With Recurrent/ Refractory Solid Tumors
Official Title
A Phase 1/2, Multicenter, Open-label, Dose-finding Study to Assess the Safety, Tolerability, and Preliminary Efficacy of Weekly Nab-paclitaxel in Pediatric Patients With Recurrent or Refractory Solid Tumors.
Acronym
Not provided
Organization
CelgeneINDUSTRY
Status Module
Record Verification Date
Dec 2019
Overall Recruitment Status or Expanded Access Status
Completed
Last Known Status
Not provided
Delayed Posting
Not provided
Why Stopped
Not provided
Expanded Access Info
No
Start Date
Dec 4, 2013Actual
Primary Completion Date
Dec 5, 2017Actual
Completion Date
Nov 6, 2018Actual
First Submitted Date
Sep 27, 2013
First Submission Date that Met QC Criteria
Oct 10, 2013
First Posted Date
Oct 14, 2013Estimated
Results Waived
Not provided
Results First Submitted Date
Dec 5, 2018
Results First Submitted that Met QC Criteria
Dec 5, 2018
Results First Posted Date
Dec 27, 2018Actual
Certification/Extension (aka Delayed Results) First Submitted Date
Not provided
Certification/Extension First Submitted that Passed QC Review
Not provided
Certification/Extension First Posted Date
Not provided
Last Update Submitted Date
Dec 19, 2019
Last Update Posted Date
Dec 27, 2019Actual
Sponsor/Collaborators Module
Responsible Party, by Official Title
Sponsor
Lead Sponsor
CelgeneINDUSTRY
Collaborators
Not provided
Oversight Module
Has Data Monitoring Committee (DMC)
No
Is FDA Regulated Drug
Not provided
Is FDA Regulated Device
Not provided
Is Unapproved Device
Not provided
Pediatric Postmarket Surveillance of a Device Product
Not provided
Product Exported from US
Not provided
FDAAA801 Violation
Not provided
Description Module
Brief Summary
The purpose of this study is to find the safe dose of nab-paclitaxel in children with solid tumors, and to see if it works to treat these solid tumors in children and young adults (in Phase 1 ≤ 18 years old and in Phase 2 ≤ 24 years old). After the final dose has been chosen, patients will be enrolled according to the specific solid tumor type, (neuroblastoma, rhabdomyosarcoma, or Ewing's sarcoma), to see how nab-paclitaxel works in treating these tumors.
Detailed Description
ABI-007-PST-001 is a Phase 1/2, multicenter, open-label, dose-finding study to assess the safety , tolerability, and preliminary efficacy of weekly nab-paclitaxel in pediatric patients with recurrent or refractory solid tumors (excluding brain tumors). The Phase 1 portion of the study, with a dose escalation design, ended and the recommended Phase 2 dose (RP2D) was determined as 240 mg/m^2 intravenously (IV) in patients weighing > 10 kg and 11.5 mg/kg in patients weighing ≤ 10 kg, on Days 1, 8 and 15 of a 28-day cycle. The Phase 2 portion of the study will enroll additional patients at the RP2D into 1 of 3 solid tumor groups [neuroblastomas, rhabdomyosarcomas, Ewing's sarcomas]. Both phases of the study are open-label and conducted at multiple centers.
The Phase 2 is using a Simon 2-stage design to monitor patient enrollment for each group separately. The rhabdomyosarcoma group, neuroblastoma or Ewing's sarcoma groups did not reach the expected number of 2 responders out of 14 efficacy eligible patients. Consequently, the groups were stopped.
Conditions Module
Conditions
Neuroblastoma
Rhabdomyosarcoma
Ewing's Sarcoma
Ewing's Tumor
Sarcoma, Ewing's
Sarcomas, Epitheliod
Sarcoma, Soft Tissue
Sarcoma, Spindle Cell
Melanoma
Malignant Melanoma
Clinical Oncology
Oncology, Medical
Pediatrics, Osteosarcoma
Osteogenic Sarcoma
Osteosarcoma Tumor
Sarcoma, Osteogenic
Tumors
Cancer
Neoplasia
Neoplasm
Histiocytoma
Fibrosarcoma
Dermatofibrosarcoma
Keywords
Neuroblastoma
Rhabdomyosarcoma
Soft Tissue
Pediatric Oncology
Abraxane
albumin-bound paclitaxel
nab-paclitaxel
ABI-007
taxane
solid tumors
Design Module
Study Type
Interventional
Number of References to an Expanded Access Study
Not provided
Expanded Access Types
Not provided
Patient Registry
Not provided
Target Follow-Up Duration
Not provided
Phases
Phase 1Phase 2
Interventional Study Design
Allocation
Biospecimen
No data available
No data is available for this block.
Enrollment
107Actual
Arms/Interventions Module
Arm Groups
Label
Type
Description
Intervention Names
nab-paclitaxel
Experimental
nab-paclitaxel 100-240 mg/m2 IV on Days 1, 8 and 15 of a 28-day cycle.
Drug: nab-paclitaxel
Phase 1: Nab-Paclitaxel 120 mg/m^2
Experimental
nab-paclitaxel 120 mg/m^2 IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity to establish the RP2D.
Drug: nab-paclitaxel
Phase 1: Nab-Paclitaxel 150 mg/m^2
Experimental
nab-paclitaxel 150 mg/m^2 IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity to establish the RP2D.
Drug: nab-paclitaxel
Phase 1: Nab-Paclitaxel 180 mg/m^2
Experimental
nab-paclitaxel 180 mg/m^2 IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity to establish the RP2D.
Drug: nab-paclitaxel
Phase 1: Nab-Paclitaxel 210 mg/m^2
Experimental
nab-paclitaxel 210 mg/m^2 IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity to establish the RP2D.
Interventions
Name
Type
Description
Arm Group Labels
Other Names
nab-paclitaxel
Drug
nab-paclitaxel 120-270 mg/m2 IV on Days 1, 8 and 15 of a 28-day cycle
nab-paclitaxel
Outcomes Module
Primary Outcomes
Measure
Description
Time Frame
Phase 1: Number of Participants With Dose Limiting Toxicities (DLTs)
A DLT was defined as investigational product (IP)-related adverse events occurring during the DLT assessment period that led to treatment discontinuation or met one of the following criteria: - Common Terminology Criteria for Adverse Events (CTCAE) Grade (Gr) 3 or 4 nonhematologic toxicity (excluding transient transaminitis) - CTCAE Gr 3 or 4 nausea or vomiting that persisted > 5 days despite maximal anti-emetic treatment - CTCAE Gr 4 thrombocytopenia or anemia that persisted > 7 days or required transfusion > 7 days - CTCAE Gr 3 thrombocytopenia with bleeding - CTCAE Gr 4 uncomplicated neutropenia lasting > 7 days - Febrile neutropenia with confirmed bacterial infection - CTCAE Gr 3 hematologic toxicity requiring treatment (tx) delay > 21 days. Use of "..." in the table rows signifies the continuation of row title per the above list.
DLT assessment period: For participants > 10 kg: the first 28-day cycle including Cycle 2 Day 1 predose evaluations; for participants ≤ 10 kg: the first two 28-day cycles including Cycle 3 Day 1 predose evaluations
Phase 1: Number of Participants With Treatment-Emergent Adverse Events (TEAEs)
An adverse event (AE) was defined as any noxious, unintended, or untoward medical occurrence that may appear or worsen in a participant during the course of a study. A serious AE (SAE) is any AE occurring at any dose that: results in death; is life-threatening; requires inpatient hospitalization or prolongation of existing hospitalization; results in persistent or significant disability/incapacity; is a congenital anomaly/birth defect; constitutes an important medical event. TEAEs were defined as AEs that began or worsened in severity on or after the date of the first dose of study drug and within 28 days of the date of the last dose of study drug. The severity of an AE was graded according to the CTCAE, Version 4.0.
Median treatment duration in Phase 1 was 7.0 weeks, with minimum and maximum duration of 1 and 49 weeks, respectively. Participants were followed for 28 days after discontinuing treatment for safety and monitoring of AEs.
Phase 2: Overall Response Rate (ORR)
Overall response rate was defined as the percentage of participants who achieved a complete response (CR; disappearance of all target lesions) or partial response (PR; at least a 30% decrease in the sum of diameters of target lesions) confirmed no less than 4 weeks after the criteria for response were first met using Response Evaluation Criteria in Solid Tumors (RECIST) version 1.1 guidelines. (For Phase 2 neuroblastoma participants who had both RECIST and Curie Score tumor evaluations, both tumor response results were considered and an overall response was derived.) Confidence interval was obtained using the Clopper-Pearson method.
Secondary Outcomes
Measure
Description
Time Frame
Phase 1: ORR
Overall response rate was defined as the percentage of participants who achieved a complete response (CR; disappearance of all target lesions) or partial response (PR: at least a 30% decrease in the sum of diameters of target lesions) confirmed no less than 4 weeks after the criteria for response were first met) using RECIST version 1.1 guidelines over the total number of participants available for the analysis. Confidence interval was obtained using the Clopper-Pearson method.
Other Outcomes
Not provided
Eligibility Module
Eligibility Criteria
Inclusion Criteria:
Patients must meet all of the following criteria to be enrolled in the study:
Patient has a confirmed solid tumor diagnosis according to the
following:
Phase 1: patient has a recurrent or refractory solid tumor that has
progressed or did not respond to standard therapy, or for which no
standard anticancer therapy exists
Phase 2: patient has radiologically documented measurable disease by Response Evaluation Criteria in Solid Tumors (RECIST) 1.1 (for neuroblastoma, evaluable disease by 123^I-metaiodobenzylguanidine [MIBG]/Curie score is also acceptable) in 1 of the following tumor types and has failed up to 3 lines of treatment: Group 1: neuroblastoma, Group 2: rhabdomyosarcoma; Group 3: Ewing's sarcoma.
The patient has a Lansky/Karnofsky performance status score of ≥ 70%.
The patient has adequate serum chemistry levels, evidenced by the
pyruvate transaminase (SGPT) ≤ 2.5 × upper limit of normal range (ULN)
Total bilirubin ≤ 1.5 × ULN
Creatinine ≤ 1.5 × ULN
The patient has adequate bone marrow function, evidenced by the
following:
Absolute neutrophil count ≥ 1.0 × 10^9 cells/L
Platelets ≥ 80 × 10^9 cells/L (transfusion independent, defined as not
receiving platelet transfusions within 7 days prior to laboratory sample). In the phase 2 portion, for patients with known bone marrow involvement, platelets ≥ 50 × 10^9 cells/L
Hemoglobin ≥ 8 g/dL (transfusion is permitted to fulfill this criterion).
The patient (when applicable) or patient's parent(s) or legal guardian(s)
understand(s) and voluntarily signed an informed consent document prior
to any study-related assessments/procedures being conducted. Where
locally applicable, the patient also understands and voluntarily provides
his/her assent prior to any study-related assessments/procedures being
conducted.
Male patients of childbearing potential must use a condom during
sexual intercourse and shall not father a child during the study and for 6 months after the last dose of study medication.
Female patients of childbearing potential [defined as all female
patients ≥ 12 years old or who have reached menarche, whichever occurs
first] must have both of the following:
a. Agree to the use of two physician-approved contraceptive methods
simultaneously or practice complete abstinence while on study medication or for a longer period if required by regulations.
i. True abstinence: When this is in line with the preferred and usual
lifestyle of the patient. Periodic abstinence (e.g., calendar, ovulation,
symptothermal, postovulation methods) and withdrawal are not
acceptable methods of contraception.
ii. Acceptable contraceptive methods include: oral, injectable, or
barrier contraceptive with spermicide; or vasectomized partner) including
at least one barrier method.
b. Have negative serum pregnancy test result at screening confirmed by
negative urine pregnancy dipstick within 72 hours prior to first dose of
investigational product (if serum test occurred > 72 hours from first
dose); pregnancy test with sensitivity of at least 25 mIU/mL.
Exclusion Criteria:
The presence of any of the following will exclude a patient from enrollment:
The patient has a primary brain tumor(s) or brain metastasis (unless metastasis is treated and stable for > 28 days). In patients who are symptomatic, a brain scan is required to exclude metastasis.
The patient has received therapeutic dose chemotherapy or radiotherapy ≤ 21 days prior to start of investigational product.
The patient has received maintenance dose chemotherapy (e.g., low dose cyclophosphamide) ≤ 7 days from the first dose of investigational product.
The patient has received any investigational therapy ≤ 28 days prior to start of investigational product. Investigational therapy is defined as any medicinal product that is not approved in the country of treatment for any indication, adult or pediatric.
The patient has received any biological therapy ≤ 7 days prior to the start of investigational product, or monoclonal antibody ≤ 3 half-lives or 28 days, whichever is shorter, prior to the first dose of investigational product.
The patient has received any hematopoietic stem cell transplantation (HSCT) ≤ 3 months prior to start of investigational product.
The patient has received allogeneic hematopoietic stem cell transplantation (HSCT) ≤ 3 months or autologous HSCT ≤ 21 days prior to start of investigational product.
The patient has not recovered from the acute toxic effects of prior chemotherapy, radiation, or major surgery/significant trauma.
The patient has had minor surgery ≤ 7 days from the start of study treatment (excluding the placement of central/peripheral lines, skin biopsy).
The patient has a known history of stroke, myocardial infarction, peripheral vascular disease, or recent (within 3 months) uncontrolled deep venous thrombosis.
The patient has a known history or current diagnosis of human immunodeficiency virus (HIV) infection, regardless of treatment status.
The patient has an uncontrolled intercurrent illness including but not limited to ongoing or active infection requiring antibiotic, antifungal, or antiviral therapy, symptomatic heart failure, cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements.
The patient has any significant medical condition, laboratory abnormality, or psychiatric illness that would prevent the patient from participating in the study.
The patient has any condition, including the presence of laboratory abnormalities, that places the patient at unacceptable risk if he/she were to participate in the study.
The patient has any condition that confounds the ability to interpret data from the study.
The patient or parent(s)/guardian(s) is/are unable to comply with the study visit schedule and other protocol requirements, in the opinion of the investigator.
The patient has ≥ Grade 2 peripheral neuropathy by National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) at screening.
Moreno L, Casanova M, Chisholm JC, Berlanga P, Chastagner PB, Baruchel S, Amoroso L, Gallego Melcon S, Gerber NU, Bisogno G, Fagioli F, Geoerger B, Glade Bender JL, Aerts I, Bergeron C, Hingorani P, Elias I, Simcock M, Ferrara S, Le Bruchec Y, Slepetis R, Chen N, Vassal G. Phase I results of a phase I/II study of weekly nab-paclitaxel in paediatric patients with recurrent/refractory solid tumours: A collaboration with innovative therapies for children with cancer. Eur J Cancer. 2018 Sep;100:27-34. doi: 10.1016/j.ejca.2018.05.002. Epub 2018 Jun 21.
Sixty-five participants were included in the Phase 1 enrolled population, and 64 enrolled partcipants received at least 1 dose of study drug and were included in the safety population, noted below. Forty-two participants were included in the Phase 2 enrolled and safety populations.
Recruitment Details
Not provided
Type of Units Analyzed
Not provided
Arm/Group Information
ID
Title
Description
FG000
Phase 1: Nab-Paclitaxel 120 mg/m^2
nab-paclitaxel 120 mg/m^2 intravenously (IV) on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity to establish the recommended phase 2 dose (RP2D).
FG001
Phase 1: Nab-Paclitaxel 150 mg/m^2
Periods
Title
Milestones
Reasons Not Completed
Phase 1
Type
Comment
Milestone Data
STARTED
Baseline Characteristics Module
Baseline Analysis Population Description
Not provided
Outcome Measures Module
Outcome Measures
Adverse Events Module
Frequency Threshold
5
More Info Module
Limitations and Caveats
Not provided
Annotation Section
No data available
No data is available for this block.
Document Section
Large Document Module
Document Has No Statistical Analysis Plan (SAP)
Not provided
Uploaded Document Information
Type
Includes Protocol
Includes SAP
Includes ICF
Document Label
Document Date
Document Uploaded Date
Document File Name
Prot
Yes
No
No
Study Protocol
Jul 13, 2016
Dec 5, 2018
Derived Section
Miscellaneous Info Module
Version Holder
Jul 10, 2026
Removed Countries
Not provided
Submission Tracking
No data available
No data is available for this block.
Condition Browse Module
MeSH Terms
Intervention Browse Module
MeSH Terms
Non-Randomized
Intervention Model
Single Group Assignment
Intervention Model Description
Not provided
Primary Purpose
Treatment
Observational Model
Not provided
Time Perspective
Not provided
Masking Info
Masking
None (Open Label)
Masking Description
Not provided
Who Masked
Not provided
Drug: nab-paclitaxel
Phase 1: Nab-Paclitaxel 240 mg/m^2
Experimental
nab-paclitaxel 240 mg/m^2 IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity to establish the RP2D.
Drug: nab-paclitaxel
Phase 1: Nab-Paclitaxel 270 mg/m^2
Experimental
nab-paclitaxel 270 mg/m^2 IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity to establish the RP2D.
Drug: nab-paclitaxel
Phase 2: Ewing's Sarcoma
Experimental
Participants with Ewing's sarcoma: nab-paclitaxel at the RP2D (240 mg/m^2 in participants weighing > 10 kg and 11.5 mg/kg in participants weighing ≤ 10 kg) IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity.
Drug: nab-paclitaxel
Phase 2: Neuroblastoma
Experimental
Participants with neuroblastoma: nab-paclitaxel at the RP2D (240 mg/m^2 in participants weighing > 10 kg and 11.5 mg/kg in participants weighing ≤ 10 kg) IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity.
Drug: nab-paclitaxel
Phase 2: Rhabdomyosarcoma
Experimental
Participants with rhabdomyosarcoma: nab-paclitaxel at the RP2D (240 mg/m^2 in participants weighing > 10 kg and 11.5 mg/kg in participants weighing ≤ 10 kg) IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity.
Drug: nab-paclitaxel
Abraxane
nab-paclitaxel
Drug
IV infusion
Phase 1: Nab-Paclitaxel 120 mg/m^2
Phase 1: Nab-Paclitaxel 150 mg/m^2
Phase 1: Nab-Paclitaxel 180 mg/m^2
Phase 1: Nab-Paclitaxel 210 mg/m^2
Phase 1: Nab-Paclitaxel 240 mg/m^2
Phase 1: Nab-Paclitaxel 270 mg/m^2
Phase 2: Ewing's Sarcoma
Phase 2: Neuroblastoma
Phase 2: Rhabdomyosarcoma
Abraxane
Median treatment duration in Phase 2 per group: Ewings Sarcoma = 14 weeks (3-31), Neuroblastoma = 7 weeks (3-23), Rhabdomyosarcoma = 5 weeks (1-13).
Median treatment duration in Phase 1 was 7.0 weeks, with minimum and maximum duration of 1 and 49 weeks, respectively.
Phase 1: Maximum Observed Concentration of Paclitaxel in Blood Plasma (Cmax)
Cycle 1 Day 1 (Participants ≥ 6 years: 1-2 minutes prior to the end of infusion [EOI], and 15 minutes, 1, 3, 5, 8, 24, 48, and 72 hours after the EOI. Participants <6 years: 1-2 minutes prior to the EOI, and 15 minutes, 3, 5, and 24 hours after the EOI.)
Phase 1: Cmax - Dose-Normalized
Cycle 1 Day 1 (Participants ≥ 6 years: 1-2 minutes prior to the end of infusion [EOI], and 15 minutes, 1, 3, 5, 8, 24, 48, and 72 hours after the EOI. Participants <6 years: 1-2 minutes prior to the EOI, and 15 minutes, 3, 5, and 24 hours after the EOI.)
Phase 1: Area Under the Plasma Concentration-Time Curve (AUC)
Measurements include: AUC from time zero to the last measurable concentration (AUCt), AUC from time zero to 24 hours (AUC24), and AUC from time zero to infinity (AUCinf).
Cycle 1 Day 1 (Participants ≥ 6 years: 1-2 minutes prior to the end of infusion [EOI], and 15 minutes, 1, 3, 5, 8, 24, 48, and 72 hours after the EOI. Participants <6 years: 1-2 minutes prior to the EOI, and 15 minutes, 3, 5, and 24 hours after the EOI.)
Phase 1: AUC - Dose-Normalized
Measurements include: AUC24 and AUCinf.
Cycle 1 Day 1 (Participants ≥ 6 years: 1-2 minutes prior to the end of infusion [EOI], and 15 minutes, 1, 3, 5, 8, 24, 48, and 72 hours after the EOI. Participants <6 years: 1-2 minutes prior to the EOI, and 15 minutes, 3, 5, and 24 hours after the EOI.)
Phase 1: Clearance (CL)
Measurement of renal clearance from the body.
Cycle 1 Day 1 (Participants ≥ 6 years: 1-2 minutes prior to the end of infusion [EOI], and 15 minutes, 1, 3, 5, 8, 24, 48, and 72 hours after the EOI. Participants <6 years: 1-2 minutes prior to the EOI, and 15 minutes, 3, 5, and 24 hours after the EOI.)
Phase 1: CL - Body Surface Area (BSA)-Normalized
Measurement of renal clearance from the body.
Cycle 1 Day 1 (Participants ≥ 6 years: 1-2 minutes prior to the end of infusion [EOI], and 15 minutes, 1, 3, 5, 8, 24, 48, and 72 hours after the EOI. Participants <6 years: 1-2 minutes prior to the EOI, and 15 minutes, 3, 5, and 24 hours after the EOI.)
Phase 1: Volume of Distribution (Vss)
Cycle 1 Day 1 (Participants ≥ 6 years: 1-2 minutes prior to the end of infusion [EOI], and 15 minutes, 1, 3, 5, 8, 24, 48, and 72 hours after the EOI. Participants <6 years: 1-2 minutes prior to the EOI, and 15 minutes, 3, 5, and 24 hours after the EOI.)
Phase 1: Vss - BSA-Normalized
Cycle 1 Day 1 (Participants ≥ 6 years: 1-2 minutes prior to the end of infusion [EOI], and 15 minutes, 1, 3, 5, 8, 24, 48, and 72 hours after the EOI. Participants <6 years: 1-2 minutes prior to the EOI, and 15 minutes, 3, 5, and 24 hours after the EOI.)
Phase 1 and 2 Population PK: Volume of Distribution of the Central Compartment (V1)
Population PK analysis was performed using nonlinear mixed effect modeling. The estimated allometric function for V1 was 0.888.
Cycle 1 Day 1 (Participants ≥ 6 years: 1-2 minutes prior to the end of infusion [EOI], and 15 minutes, 1, 3, 5, 8, 24, 48, and 72 hours after the EOI. Participants < 6 years: 1-2 minutes prior to the EOI, and 15 minutes, 3, 5, and 24 hours after the EOI.)
Phase 1 and 2 Population PK: Maximum Elimination Rate From the Central Compartment (VMEL)
Population PK analysis was performed using nonlinear mixed effect modeling. The estimated allometric function for VMEL was 1.12.
Cycle 1 Day 1 (Participants ≥ 6 years: 1-2 minutes prior to the end of infusion [EOI], and 15 minutes, 1, 3, 5, 8, 24, 48, and 72 hours after the EOI. Participants < 6 years: 1-2 minutes prior to the EOI, and 15 minutes, 3, 5, and 24 hours after the EOI.)
Phase 1 and 2 Population PK: Concentration in the Central Compartment at 50% of VMEL (KMEL)
Population PK analysis was performed using nonlinear mixed effect modeling.
Cycle 1 Day 1 (Participants ≥ 6 years: 1-2 minutes prior to the end of infusion [EOI], and 15 minutes, 1, 3, 5, 8, 24, 48, and 72 hours after the EOI. Participants < 6 years: 1-2 minutes prior to the EOI, and 15 minutes, 3, 5, and 24 hours after the EOI.)
Phase 1 and 2 Population PK: Intercompartmental CL Between the Central Compartment and the First Peripheral Compartment (Q2)
Population PK analysis was performed using nonlinear mixed effect modeling. The estimated allometric function for Q2 was 1.12.
Cycle 1 Day 1 (Participants ≥ 6 years: 1-2 minutes prior to the end of infusion [EOI], and 15 minutes, 1, 3, 5, 8, 24, 48, and 72 hours after the EOI. Participants < 6 years: 1-2 minutes prior to the EOI, and 15 minutes, 3, 5, and 24 hours after the EOI.)
Phase 1 and 2 Population PK: Intercompartmental CL Between the Central Compartment and the Second Peripheral Compartment (Q3)
Population PK analysis was performed using nonlinear mixed effect modeling. The estimated allometric function for Q3 was 1.12.
Cycle 1 Day 1 (Participants ≥ 6 years: 1-2 minutes prior to the end of infusion [EOI], and 15 minutes, 1, 3, 5, 8, 24, 48, and 72 hours after the EOI. Participants < 6 years: 1-2 minutes prior to the EOI, and 15 minutes, 3, 5, and 24 hours after the EOI.)
Phase 1 and 2 Population PK: Volume of Distribution of the First Peripheral Compartment (V2)
Population PK analysis was performed using nonlinear mixed effect modeling. The estimated allometric function for V2 was 0.888.
Cycle 1 Day 1 (Participants ≥ 6 years: 1-2 minutes prior to the end of infusion [EOI], and 15 minutes, 1, 3, 5, 8, 24, 48, and 72 hours after the EOI. Participants < 6 years: 1-2 minutes prior to the EOI, and 15 minutes, 3, 5, and 24 hours after the EOI.)
Phase 1 and 2 Population PK: Volume of Distribution of the Second Peripheral Compartment (V3)
Population PK analysis was performed using nonlinear mixed effect modeling. The estimated allometric function for V3 was 0.888.
Cycle 1 Day 1 (Participants ≥ 6 years: 1-2 minutes prior to the end of infusion [EOI], and 15 minutes, 1, 3, 5, 8, 24, 48, and 72 hours after the EOI. Participants < 6 years: 1-2 minutes prior to the EOI, and 15 minutes, 3, 5, and 24 hours after the EOI.)
Phase 2: Duration of Response (DOR)
Duration of response was defined as the time from the date of the first response (CR/PR, using RECIST version 1.1 guidelines) to disease progression for participants with a confirmed CR or PR. Participants who did not have disease progression or had not died were censored at the time of their last disease assessment or at time of start of new anticancer therapy, whichever occurred first. (For Phase 2 neuroblastoma patients who had both RECIST version 1.1 and Curie Score tumor evaluations, both tumor responses results were considered and an overall response was derived.)
Median treatment duration in Phase 2 per group: Ewings Sarcoma = 14 weeks (3-31), Neuroblastoma = 7 weeks (3-23), Rhabdomyosarcoma = 5 weeks (1-13). Participants were followed until disease progression (if applicable) up to a maximum of 100.3 weeks.
Phase 2: Disease Control Rate (DCR)
Disease control rate was defined as the percentage of participants who achieved either a stable disease maintained for ≥ 16 weeks or confirmed CR (confirmed no less than 4 weeks after criteria for response were first met) or confirmed PR (confirmed no less than 4 weeks after criteria for response were first met) over the total number of participants available for the analysis. Confidence interval was obtained using the Clopper-Pearson method.
Median treatment duration in Phase 2 per group: Ewings Sarcoma = 14 weeks (3-31), Neuroblastoma = 7 weeks (3-23), Rhabdomyosarcoma = 5 weeks (1-13).
Phase 2: Progression-Free Survival (PFS)
PFS was defined as the time from the first dose date to the start of disease progression or participant death (any cause), whichever occurred first. Disease progression was classed as either a disease progression observed as a response assessment, or a disease progression or symptomatic deterioration at treatment/study discontinuation. Participants who did not have disease progression or had not died were censored at the last known time that the participant was progression free. Disease progression was considered according to RECIST version 1.1 for Phase 2 Ewing's sarcoma and rhabdomyosarcoma participants. (For Phase 2 neuroblastoma participants who had both RECIST 1.1 and Curie score tumor evaluations, both tumor responses results were considered and an overall response was derived.) Median PFS time was estimated through Kaplan-Meier methods. 95% confidence interval about the median time to PFS event was obtained using Greenwood's method.
Median treatment duration in Phase 2 per group: Ewings Sarcoma = 14 weeks (3-31), Neuroblastoma = 7 weeks (3-23), Rhabdomyosarcoma = 5 weeks (1-13). Participants were followed until disease progression (if applicable) up to a maximum of 100.3 weeks.
Phase 2: Kaplan-Meier Estimate of Overall Survival Rate at 1 Year
Overall survival was defined as the time from the first dose date to date of death (any cause). Participants who were alive were censored at the last known time that the participant was alive.
1 year
Phase 2: Number of Participants With Treatment-Emergent Adverse Events (TEAEs)
An AE was defined as any noxious, unintended, or untoward medical occurrence that may appear or worsen in a participant during the course of a study. A SAE is any AE occurring at any dose that: results in death; is life-threatening; requires inpatient hospitalization or prolongation of existing hospitalization; results in persistent or significant disability/incapacity; is a congenital anomaly/birth defect; constitutes an important medical event. TEAEs were defined as AEs that began or worsened in severity on or after the date of the first dose of study drug and within 28 days of the date of the last dose of study drug. The severity of the AEs was graded according to the Common Terminology Criteria for Adverse Events, Version 4.0. Participants were followed for 28 days after discontinuing treatment for safety and monitoring of AEs.
Median treatment duration in Phase 2 per group: Ewings Sarcoma = 14 weeks (3-31), Neuroblastoma = 7 weeks (3-23), Rhabdomyosarcoma = 5 weeks (1-13). Participants were followed for 28 days after discontinuing treatment for safety and monitoring of AEs.
New York
New York
10032
United States
The Hospital for Sick Children
Toronto
Ontario
M5G 1X8
Canada
Institute for Pediatric Hematology - Oncology, Leon Berard Cancer Center
Lyon
69008
France
Hopital d'Enfants, CHU Nancy
Nancy
54000
France
Institut Curie
Paris
75005
France
Institut Gustave Roussy
Villejuif
94805
France
Azienda Ospedaliera Universitaria Meyer
Florence
50139
Italy
Children's Hospital Largo
Genova
16147
Italy
Istituto Nazionale Tumori
Milan
Italy
Clinica di Oncoematologia
Padova
35128
Italy
Policlinico Agostino Gemelli
Rome
00168
Italy
l'Azienda Ospedaliera Regina Margherita - Sant Anna
Torino
10126
Italy
Hospital Universitario Vall D Hebron
Barcelona
8035
Spain
Hospital Sant Joan de Deu
Barcelona
8950
Spain
Spanish National Cancer Research Centre
Madrid
28029
Spain
Hospital Universitario Virgen Del Rocio
Seville
41013
Spain
Unidad de Oncologia Pediatrica, Hospital Universitario la Fe
Valencia
46026
Spain
Universitäts-Kinderklinik
Zurich
8032
Switzerland
Royal Marsden Hospital
Sutton
SM2 5PT
United Kingdom
Derived
Amoroso L, Castel V, Bisogno G, Casanova M, Marquez-Vega C, Chisholm JC, Doz F, Moreno L, Ruggiero A, Gerber NU, Fagioli F, Hingorani P, Melcon SG, Slepetis R, Chen N, le Bruchec Y, Simcock M, Vassal G. Phase II results from a phase I/II study to assess the safety and efficacy of weekly nab-paclitaxel in paediatric patients with recurrent or refractory solid tumours: A collaboration with the European Innovative Therapies for Children with Cancer Network. Eur J Cancer. 2020 Aug;135:89-97. doi: 10.1016/j.ejca.2020.04.031. Epub 2020 Jun 15.
Hattinger CM, Patrizio MP, Magagnoli F, Luppi S, Serra M. An update on emerging drugs in osteosarcoma: towards tailored therapies? Expert Opin Emerg Drugs. 2019 Sep;24(3):153-171. doi: 10.1080/14728214.2019.1654455. Epub 2019 Aug 14.
nab-paclitaxel 150 mg/m^2 IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity to establish the RP2D.
FG002
Phase 1: Nab-Paclitaxel 180 mg/m^2
nab-paclitaxel 180 mg/m^2 IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity to establish the RP2D.
FG003
Phase 1: Nab-Paclitaxel 210 mg/m^2
nab-paclitaxel 210 mg/m^2 IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity to establish the RP2D.
FG004
Phase 1: Nab-Paclitaxel 240 mg/m^2
nab-paclitaxel 240 mg/m^2 IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity to establish the RP2D.
FG005
Phase 1: Nab-Paclitaxel 270 mg/m^2
nab-paclitaxel 270 mg/m^2 IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity to establish the RP2D.
FG006
Phase 2: Ewing's Sarcoma
Participants with Ewing's sarcoma: nab-paclitaxel at the RP2D (240 mg/m^2 in participants weighing > 10 kg and 11.5 mg/kg in participants weighing ≤ 10 kg) IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity.
FG007
Phase 2: Neuroblastoma
Participants with neuroblastoma: nab-paclitaxel at the RP2D (240 mg/m^2 in participants weighing > 10 kg and 11.5 mg/kg in participants weighing ≤ 10 kg) IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity.
FG008
Phase 2: Rhabdomyosarcoma
Participants with rhabdomyosarcoma: nab-paclitaxel at the RP2D (240 mg/m^2 in participants weighing > 10 kg and 11.5 mg/kg in participants weighing ≤ 10 kg) IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity.
FG00016 subjects
FG0018 subjects
FG00214 subjects
FG00311 subjects
FG0048 subjects
FG0057 subjects
FG0060 subjects
FG0070 subjects
FG0080 subjects
COMPLETED
FG0000 subjects
FG0010 subjects
FG0020 subjects
FG0030 subjects
FG0040 subjects
FG0050 subjects
FG0060 subjects
FG0070 subjects
FG0080 subjects
NOT COMPLETED
FG00016 subjects
FG0018 subjects
FG00214 subjects
FG00311 subjects
FG0048 subjects
FG0057 subjects
FG0060 subjects
FG0070 subjects
FG0080 subjects
Type
Comment
Reasons
Adverse Event
FG0004 subjects
FG0010 subjects
FG0022 subjects
FG0031 subjects
FG0042 subjects
FG0052 subjects
FG0060 subjects
FG0070 subjects
FG0080 subjects
Progressive Disease
FG0008 subjects
FG0016 subjects
FG0028 subjects
FG0034 subjects
FG004
Withdrawal by Subject
FG0000 subjects
FG0010 subjects
FG0020 subjects
FG0032 subjects
FG004
Withdrawal by Parent/Guardian
FG0000 subjects
FG0010 subjects
FG0022 subjects
FG0031 subjects
FG004
Physician Decision
FG0000 subjects
FG0010 subjects
FG0020 subjects
FG0031 subjects
FG004
Symptomatic Deterioration
FG0003 subjects
FG0012 subjects
FG0022 subjects
FG0032 subjects
FG004
Miscellaneous
FG0001 subjects
FG0010 subjects
FG0020 subjects
FG0030 subjects
FG004
Phase 2
Type
Comment
Milestone Data
STARTED
FG0000 subjects
FG0010 subjects
FG0020 subjects
FG0030 subjects
FG0040 subjects
FG0050 subjects
FG00614 subjects
FG00714 subjects
FG00814 subjects
COMPLETED
FG0000 subjects
FG0010 subjects
FG0020 subjects
FG0030 subjects
FG004
NOT COMPLETED
FG0000 subjects
FG0010 subjects
FG0020 subjects
FG0030 subjects
FG004
Type
Comment
Reasons
Adverse Event
FG0000 subjects
FG0010 subjects
FG0020 subjects
FG003
Type of Units Analyzed
Not provided
Arm/Group Information
ID
Title
Description
BG000
Phase 1: Nab-Paclitaxel 120 mg/m^2
nab-paclitaxel 120 mg/m^2 IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity to establish the RP2D.
BG001
Phase 1: Nab-Paclitaxel 150 mg/m^2
nab-paclitaxel 150 mg/m^2 IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity to establish the RP2D.
BG002
Phase 1: Nab-Paclitaxel 180 mg/m^2
nab-paclitaxel 180 mg/m^2 IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity to establish the RP2D.
BG003
Phase 1: Nab-Paclitaxel 210 mg/m^2
nab-paclitaxel 210 mg/m^2 IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity to establish the RP2D.
BG004
Phase 1: Nab-Paclitaxel 240 mg/m^2
nab-paclitaxel 240 mg/m^2 IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity to establish the RP2D.
BG005
Phase 1: Nab-Paclitaxel 270 mg/m^2
nab-paclitaxel 270 mg/m^2 IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity to establish the RP2D.
BG006
Phase 2: Ewing's Sarcoma
Participants with Ewing's sarcoma: nab-paclitaxel at the RP2D (240 mg/m^2 in participants weighing > 10 kg and 11.5 mg/kg in participants weighing ≤ 10 kg) IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity.
BG007
Phase 2: Neuroblastoma
Participants with neuroblastoma: nab-paclitaxel at the RP2D (240 mg/m^2 in participants weighing > 10 kg and 11.5 mg/kg in participants weighing ≤ 10 kg) IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity.
BG008
Phase 2: Rhabdomyosarcoma
Participants with rhabdomyosarcoma: nab-paclitaxel at the RP2D (240 mg/m^2 in participants weighing > 10 kg and 11.5 mg/kg in participants weighing ≤ 10 kg) IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity.
BG009
Total
Total of all reporting groups
Denominators
Units
Counts
Participants
BG00016
BG0018
BG00214
BG00311
BG0048
BG0057
BG00614
BG00714
BG00814
BG009106
Baseline Measures
Title
Description
Population Description
Parameter Type
Dispersion Type
Unit of Measure
Calculate Percentage
Denominator Units Selected
Denominators
Classes
Age, Continuous
Mean
Standard Deviation
years
Title
Denominators
Categories
Title
Measurements
BG00011.7± 3.32
BG00112.1± 5.84
BG00210.2± 4.64
BG003
Sex: Female, Male
Count of Participants
Participants
Title
Denominators
Categories
Title
Measurements
Female
BG0009
BG0014
BG002
Ethnicity (NIH/OMB)
Count of Participants
Participants
Title
Denominators
Categories
Title
Measurements
Hispanic or Latino
BG0002
BG0013
BG002
Race/Ethnicity, Customized
Count of Participants
Participants
Title
Denominators
Categories
White
Title
Measurements
BG00011
BG0018
BG002
Participants With Any Prior Cancer Treatment
Count of Participants
Participants
Title
Denominators
Categories
Any Prior Cancer Treatment
Title
Measurements
BG00016
BG0018
BG002
Number of Prior Systemic Anticancer Regimens Received
Mean
Standard Deviation
systemic anticancer regimens
Title
Denominators
Categories
Title
Measurements
BG0003.69± 1.815
BG0013.25± 1.832
BG002
Type
Title
Description
Population Description
Reporting Status
Anticipated Posting Date
Parameter Type
Dispersion Type
Unit of Measure
Calculate Percentage
Time Frame
Units Analyzed
Denominator Units Selected
Arm/Group Information
Denominators
Classes
Analyses
Primary
Phase 1: Number of Participants With Dose Limiting Toxicities (DLTs)
A DLT was defined as investigational product (IP)-related adverse events occurring during the DLT assessment period that led to treatment discontinuation or met one of the following criteria: - Common Terminology Criteria for Adverse Events (CTCAE) Grade (Gr) 3 or 4 nonhematologic toxicity (excluding transient transaminitis) - CTCAE Gr 3 or 4 nausea or vomiting that persisted > 5 days despite maximal anti-emetic treatment - CTCAE Gr 4 thrombocytopenia or anemia that persisted > 7 days or required transfusion > 7 days - CTCAE Gr 3 thrombocytopenia with bleeding - CTCAE Gr 4 uncomplicated neutropenia lasting > 7 days - Febrile neutropenia with confirmed bacterial infection - CTCAE Gr 3 hematologic toxicity requiring treatment (tx) delay > 21 days. Use of "..." in the table rows signifies the continuation of row title per the above list.
Dose Determining Set (DDS): all Phase 1 participants who received all 3 weekly doses of nab-paclitaxel at the cohort planned dose during Cycle 1 and had adequate safety assessments during the DLT assessment period or experienced a DLT. The DDS did not include participants who were enrolled at each dose once the dose had been determined to be safe.
Posted
Count of Participants
Participants
DLT assessment period: For participants > 10 kg: the first 28-day cycle including Cycle 2 Day 1 predose evaluations; for participants ≤ 10 kg: the first two 28-day cycles including Cycle 3 Day 1 predose evaluations
ID
Title
Description
OG000
Phase 1: Nab-Paclitaxel 120 mg/m^2
nab-paclitaxel 120 mg/m^2 intravenously (IV) on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity to establish the recommended phase 2 dose (RP2D).
OG001
Phase 1: Nab-Paclitaxel 150 mg/m^2
nab-paclitaxel 150 mg/m^2 IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity to establish the RP2D.
OG002
Phase 1: Nab-Paclitaxel 180 mg/m^2
nab-paclitaxel 180 mg/m^2 IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity to establish the RP2D.
OG003
Phase 1: Nab-Paclitaxel 210 mg/m^2
nab-paclitaxel 210 mg/m^2 IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity to establish the RP2D.
OG004
Phase 1: Nab-Paclitaxel 240 mg/m^2
nab-paclitaxel 240 mg/m^2 IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity to establish the RP2D.
OG005
Phase 1: Nab-Paclitaxel 270 mg/m^2
nab-paclitaxel 270 mg/m^2 IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity to establish the RP2D.
Units
Counts
Participants
OG0006
OG0016
OG0026
OG003
Title
Denominators
Categories
At least 1 DLT
Title
Measurements
OG0001
OG0010
OG0020
OG003
Primary
Phase 1: Number of Participants With Treatment-Emergent Adverse Events (TEAEs)
An adverse event (AE) was defined as any noxious, unintended, or untoward medical occurrence that may appear or worsen in a participant during the course of a study. A serious AE (SAE) is any AE occurring at any dose that: results in death; is life-threatening; requires inpatient hospitalization or prolongation of existing hospitalization; results in persistent or significant disability/incapacity; is a congenital anomaly/birth defect; constitutes an important medical event. TEAEs were defined as AEs that began or worsened in severity on or after the date of the first dose of study drug and within 28 days of the date of the last dose of study drug. The severity of an AE was graded according to the CTCAE, Version 4.0.
Safety Population: all participants who took at least 1 dose of study drug.
Posted
Count of Participants
Participants
Median treatment duration in Phase 1 was 7.0 weeks, with minimum and maximum duration of 1 and 49 weeks, respectively. Participants were followed for 28 days after discontinuing treatment for safety and monitoring of AEs.
ID
Title
Description
OG000
Phase 1: Nab-Paclitaxel 120 mg/m^2
nab-paclitaxel 120 mg/m^2 intravenously (IV) on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity to establish the recommended phase 2 dose (RP2D).
OG001
Phase 1: Nab-Paclitaxel 150 mg/m^2
Primary
Phase 2: Overall Response Rate (ORR)
Overall response rate was defined as the percentage of participants who achieved a complete response (CR; disappearance of all target lesions) or partial response (PR; at least a 30% decrease in the sum of diameters of target lesions) confirmed no less than 4 weeks after the criteria for response were first met using Response Evaluation Criteria in Solid Tumors (RECIST) version 1.1 guidelines. (For Phase 2 neuroblastoma participants who had both RECIST and Curie Score tumor evaluations, both tumor response results were considered and an overall response was derived.) Confidence interval was obtained using the Clopper-Pearson method.
Efficacy Evaluable Population: participants who met eligibility criteria for Phase 2, completed at least 1 dose of study drug, and had baseline and at least 1 postbaseline efficacy assessment if having not discontinued the investigational product prior to postbaseline efficacy assessment due to disease progression or systematic deterioration.
Posted
Number
95% Confidence Interval
percentage of participants
Median treatment duration in Phase 2 per group: Ewings Sarcoma = 14 weeks (3-31), Neuroblastoma = 7 weeks (3-23), Rhabdomyosarcoma = 5 weeks (1-13).
ID
Title
Description
OG000
Phase 2: Ewing's Sarcoma
Participants with Ewing's sarcoma: nab-paclitaxel at the RP2D (240 mg/m^2 in participants weighing > 10 kg and 11.5 mg/kg in participants weighing ≤ 10 kg) IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity.
OG001
Secondary
Phase 1: ORR
Overall response rate was defined as the percentage of participants who achieved a complete response (CR; disappearance of all target lesions) or partial response (PR: at least a 30% decrease in the sum of diameters of target lesions) confirmed no less than 4 weeks after the criteria for response were first met) using RECIST version 1.1 guidelines over the total number of participants available for the analysis. Confidence interval was obtained using the Clopper-Pearson method.
Efficacy Evaluable Population: participants who met eligibility criteria for Phase 1, completed at least 1 dose of study drug, and had baseline and at least 1 postbaseline efficacy assessment if having not discontinued the investigational product prior to postbaseline efficacy assessment due to disease progression or systematic deterioration.
Posted
Number
95% Confidence Interval
percentage of participants
Median treatment duration in Phase 1 was 7.0 weeks, with minimum and maximum duration of 1 and 49 weeks, respectively.
ID
Title
Description
OG000
Phase 1: Nab-Paclitaxel 120 mg/m^2
nab-paclitaxel 120 mg/m^2 intravenously (IV) on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity to establish the recommended phase 2 dose (RP2D).
OG001
Phase 1: Nab-Paclitaxel 150 mg/m^2
nab-paclitaxel 150 mg/m^2 IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity to establish the RP2D.
Secondary
Phase 1: Maximum Observed Concentration of Paclitaxel in Blood Plasma (Cmax)
Pharmacokinetic (PK) population: all participannts who received at least one dose of nab-paclitaxel and had evaluable concentration data.
Posted
Geometric Mean
Geometric Coefficient of Variation
ng/mL
Cycle 1 Day 1 (Participants ≥ 6 years: 1-2 minutes prior to the end of infusion [EOI], and 15 minutes, 1, 3, 5, 8, 24, 48, and 72 hours after the EOI. Participants <6 years: 1-2 minutes prior to the EOI, and 15 minutes, 3, 5, and 24 hours after the EOI.)
ID
Title
Description
OG000
Phase 1: Nab-Paclitaxel 120 mg/m^2
nab-paclitaxel 120 mg/m^2 intravenously (IV) on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity to establish the recommended phase 2 dose (RP2D).
OG001
Phase 1: Nab-Paclitaxel 150 mg/m^2
nab-paclitaxel 150 mg/m^2 IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity to establish the RP2D.
OG002
Phase 1: Nab-Paclitaxel 180 mg/m^2
nab-paclitaxel 180 mg/m^2 IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity to establish the RP2D.
Secondary
Phase 1: Cmax - Dose-Normalized
PK population: all participants who received at least one dose of nab-paclitaxel and had evaluable concentration data.
Posted
Geometric Mean
Geometric Coefficient of Variation
ng/mL/[mg]
Cycle 1 Day 1 (Participants ≥ 6 years: 1-2 minutes prior to the end of infusion [EOI], and 15 minutes, 1, 3, 5, 8, 24, 48, and 72 hours after the EOI. Participants <6 years: 1-2 minutes prior to the EOI, and 15 minutes, 3, 5, and 24 hours after the EOI.)
ID
Title
Description
OG000
Phase 1: Nab-Paclitaxel 120 mg/m^2
nab-paclitaxel 120 mg/m^2 intravenously (IV) on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity to establish the recommended phase 2 dose (RP2D).
OG001
Phase 1: Nab-Paclitaxel 150 mg/m^2
nab-paclitaxel 150 mg/m^2 IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity to establish the RP2D.
OG002
Phase 1: Nab-Paclitaxel 180 mg/m^2
nab-paclitaxel 180 mg/m^2 IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity to establish the RP2D.
Secondary
Phase 1: Area Under the Plasma Concentration-Time Curve (AUC)
Measurements include: AUC from time zero to the last measurable concentration (AUCt), AUC from time zero to 24 hours (AUC24), and AUC from time zero to infinity (AUCinf).
PK population: all participants who received at least one dose of nab-paclitaxel and had evaluable concentration data for given measure.
Posted
Geometric Mean
Geometric Coefficient of Variation
ng*h/mL
Cycle 1 Day 1 (Participants ≥ 6 years: 1-2 minutes prior to the end of infusion [EOI], and 15 minutes, 1, 3, 5, 8, 24, 48, and 72 hours after the EOI. Participants <6 years: 1-2 minutes prior to the EOI, and 15 minutes, 3, 5, and 24 hours after the EOI.)
ID
Title
Description
OG000
Phase 1: Nab-Paclitaxel 120 mg/m^2
nab-paclitaxel 120 mg/m^2 intravenously (IV) on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity to establish the recommended phase 2 dose (RP2D).
OG001
Phase 1: Nab-Paclitaxel 150 mg/m^2
nab-paclitaxel 150 mg/m^2 IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity to establish the RP2D.
OG002
Phase 1: Nab-Paclitaxel 180 mg/m^2
Secondary
Phase 1: AUC - Dose-Normalized
Measurements include: AUC24 and AUCinf.
PK population: all participants who received at least one dose of nab-paclitaxel and had evaluable concentration data for given measure.
Posted
Geometric Mean
Geometric Coefficient of Variation
ng*h/mL/[mg]
Cycle 1 Day 1 (Participants ≥ 6 years: 1-2 minutes prior to the end of infusion [EOI], and 15 minutes, 1, 3, 5, 8, 24, 48, and 72 hours after the EOI. Participants <6 years: 1-2 minutes prior to the EOI, and 15 minutes, 3, 5, and 24 hours after the EOI.)
ID
Title
Description
OG000
Phase 1: Nab-Paclitaxel 120 mg/m^2
nab-paclitaxel 120 mg/m^2 intravenously (IV) on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity to establish the recommended phase 2 dose (RP2D).
OG001
Phase 1: Nab-Paclitaxel 150 mg/m^2
nab-paclitaxel 150 mg/m^2 IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity to establish the RP2D.
OG002
Phase 1: Nab-Paclitaxel 180 mg/m^2
nab-paclitaxel 180 mg/m^2 IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity to establish the RP2D.
Secondary
Phase 1: Clearance (CL)
Measurement of renal clearance from the body.
PK population: all participants who received at least one dose of nab-paclitaxel and had evaluable concentration data.
Posted
Geometric Mean
Geometric Coefficient of Variation
L/h
Cycle 1 Day 1 (Participants ≥ 6 years: 1-2 minutes prior to the end of infusion [EOI], and 15 minutes, 1, 3, 5, 8, 24, 48, and 72 hours after the EOI. Participants <6 years: 1-2 minutes prior to the EOI, and 15 minutes, 3, 5, and 24 hours after the EOI.)
ID
Title
Description
OG000
Phase 1: Nab-Paclitaxel 120 mg/m^2
nab-paclitaxel 120 mg/m^2 intravenously (IV) on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity to establish the recommended phase 2 dose (RP2D).
OG001
Phase 1: Nab-Paclitaxel 150 mg/m^2
nab-paclitaxel 150 mg/m^2 IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity to establish the RP2D.
OG002
Phase 1: Nab-Paclitaxel 180 mg/m^2
nab-paclitaxel 180 mg/m^2 IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity to establish the RP2D.
Secondary
Phase 1: CL - Body Surface Area (BSA)-Normalized
Measurement of renal clearance from the body.
PK population: all participants who received at least one dose of nab-paclitaxel and had evaluable concentration data.
Posted
Geometric Mean
Geometric Coefficient of Variation
L/h/m^2
Cycle 1 Day 1 (Participants ≥ 6 years: 1-2 minutes prior to the end of infusion [EOI], and 15 minutes, 1, 3, 5, 8, 24, 48, and 72 hours after the EOI. Participants <6 years: 1-2 minutes prior to the EOI, and 15 minutes, 3, 5, and 24 hours after the EOI.)
ID
Title
Description
OG000
Phase 1: Nab-Paclitaxel 120 mg/m^2
nab-paclitaxel 120 mg/m^2 intravenously (IV) on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity to establish the recommended phase 2 dose (RP2D).
OG001
Phase 1: Nab-Paclitaxel 150 mg/m^2
nab-paclitaxel 150 mg/m^2 IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity to establish the RP2D.
OG002
Phase 1: Nab-Paclitaxel 180 mg/m^2
nab-paclitaxel 180 mg/m^2 IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity to establish the RP2D.
Secondary
Phase 1: Volume of Distribution (Vss)
PK population: all participants who received at least one dose of nab-paclitaxel and had evaluable concentration data.
Posted
Geometric Mean
Geometric Coefficient of Variation
liters
Cycle 1 Day 1 (Participants ≥ 6 years: 1-2 minutes prior to the end of infusion [EOI], and 15 minutes, 1, 3, 5, 8, 24, 48, and 72 hours after the EOI. Participants <6 years: 1-2 minutes prior to the EOI, and 15 minutes, 3, 5, and 24 hours after the EOI.)
ID
Title
Description
OG000
Phase 1: Nab-Paclitaxel 120 mg/m^2
nab-paclitaxel 120 mg/m^2 intravenously (IV) on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity to establish the recommended phase 2 dose (RP2D).
OG001
Phase 1: Nab-Paclitaxel 150 mg/m^2
nab-paclitaxel 150 mg/m^2 IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity to establish the RP2D.
OG002
Phase 1: Nab-Paclitaxel 180 mg/m^2
nab-paclitaxel 180 mg/m^2 IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity to establish the RP2D.
Secondary
Phase 1: Vss - BSA-Normalized
PK population: all participants who received at least one dose of nab-paclitaxel and had evaluable concentration data.
Posted
Geometric Mean
Geometric Coefficient of Variation
L/m^2
Cycle 1 Day 1 (Participants ≥ 6 years: 1-2 minutes prior to the end of infusion [EOI], and 15 minutes, 1, 3, 5, 8, 24, 48, and 72 hours after the EOI. Participants <6 years: 1-2 minutes prior to the EOI, and 15 minutes, 3, 5, and 24 hours after the EOI.)
ID
Title
Description
OG000
Phase 1: Nab-Paclitaxel 120 mg/m^2
nab-paclitaxel 120 mg/m^2 intravenously (IV) on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity to establish the recommended phase 2 dose (RP2D).
OG001
Phase 1: Nab-Paclitaxel 150 mg/m^2
nab-paclitaxel 150 mg/m^2 IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity to establish the RP2D.
OG002
Phase 1: Nab-Paclitaxel 180 mg/m^2
nab-paclitaxel 180 mg/m^2 IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity to establish the RP2D.
Secondary
Phase 1 and 2 Population PK: Volume of Distribution of the Central Compartment (V1)
Population PK analysis was performed using nonlinear mixed effect modeling. The estimated allometric function for V1 was 0.888.
PK population: all participants who received at least one dose of nab-paclitaxel and had evaluable concentration data.
Posted
Number
liters
Cycle 1 Day 1 (Participants ≥ 6 years: 1-2 minutes prior to the end of infusion [EOI], and 15 minutes, 1, 3, 5, 8, 24, 48, and 72 hours after the EOI. Participants < 6 years: 1-2 minutes prior to the EOI, and 15 minutes, 3, 5, and 24 hours after the EOI.)
ID
Title
Description
OG000
PK Population
Phase 1: nab-paclitaxel 120-270 mg/m^2 IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity to establish the RP2D. Phase 2: nab-paclitaxel at the RP2D (240 mg/m^2 in participants weighing > 10 kg and 11.5 mg/kg in participants weighing ≤ 10 kg) IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity.
Units
Counts
Participants
OG000
Secondary
Phase 1 and 2 Population PK: Maximum Elimination Rate From the Central Compartment (VMEL)
Population PK analysis was performed using nonlinear mixed effect modeling. The estimated allometric function for VMEL was 1.12.
PK population: all participants who received at least one dose of nab-paclitaxel and had evaluable concentration data
Posted
Number
μg/h
Cycle 1 Day 1 (Participants ≥ 6 years: 1-2 minutes prior to the end of infusion [EOI], and 15 minutes, 1, 3, 5, 8, 24, 48, and 72 hours after the EOI. Participants < 6 years: 1-2 minutes prior to the EOI, and 15 minutes, 3, 5, and 24 hours after the EOI.)
ID
Title
Description
OG000
PK Population
The PK population included all participants who received at least one dose of nab-paclitaxel and had evaluable concentration data.
Units
Counts
Participants
OG000
Secondary
Phase 1 and 2 Population PK: Concentration in the Central Compartment at 50% of VMEL (KMEL)
Population PK analysis was performed using nonlinear mixed effect modeling.
PK population: all participants who received at least one dose of nab-paclitaxel and had evaluable concentration data
Posted
Number
μg/L
Cycle 1 Day 1 (Participants ≥ 6 years: 1-2 minutes prior to the end of infusion [EOI], and 15 minutes, 1, 3, 5, 8, 24, 48, and 72 hours after the EOI. Participants < 6 years: 1-2 minutes prior to the EOI, and 15 minutes, 3, 5, and 24 hours after the EOI.)
ID
Title
Description
OG000
PK Population
Phase 1: nab-paclitaxel 120-270 mg/m^2 IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity to establish the RP2D. Phase 2: nab-paclitaxel at the RP2D (240 mg/m^2 in participants weighing > 10 kg and 11.5 mg/kg in participants weighing ≤ 10 kg) IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity.
Units
Counts
Participants
OG000
Secondary
Phase 1 and 2 Population PK: Intercompartmental CL Between the Central Compartment and the First Peripheral Compartment (Q2)
Population PK analysis was performed using nonlinear mixed effect modeling. The estimated allometric function for Q2 was 1.12.
PK population: all participants who received at least one dose of nab-paclitaxel and had evaluable concentration data.
Posted
Number
L/h
Cycle 1 Day 1 (Participants ≥ 6 years: 1-2 minutes prior to the end of infusion [EOI], and 15 minutes, 1, 3, 5, 8, 24, 48, and 72 hours after the EOI. Participants < 6 years: 1-2 minutes prior to the EOI, and 15 minutes, 3, 5, and 24 hours after the EOI.)
ID
Title
Description
OG000
PK Population
Phase 1: nab-paclitaxel 120-270 mg/m^2 IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity to establish the RP2D. Phase 2: nab-paclitaxel at the RP2D (240 mg/m^2 in participants weighing > 10 kg and 11.5 mg/kg in participants weighing ≤ 10 kg) IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity.
Units
Counts
Participants
OG000
Secondary
Phase 1 and 2 Population PK: Intercompartmental CL Between the Central Compartment and the Second Peripheral Compartment (Q3)
Population PK analysis was performed using nonlinear mixed effect modeling. The estimated allometric function for Q3 was 1.12.
PK population: all participants who received at least one dose of nab-paclitaxel and had evaluable concentration data.
Posted
Number
L/h
Cycle 1 Day 1 (Participants ≥ 6 years: 1-2 minutes prior to the end of infusion [EOI], and 15 minutes, 1, 3, 5, 8, 24, 48, and 72 hours after the EOI. Participants < 6 years: 1-2 minutes prior to the EOI, and 15 minutes, 3, 5, and 24 hours after the EOI.)
ID
Title
Description
OG000
PK Population
Phase 1: nab-paclitaxel 120-270 mg/m^2 IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity to establish the RP2D. Phase 2: nab-paclitaxel at the RP2D (240 mg/m^2 in participants weighing > 10 kg and 11.5 mg/kg in participants weighing ≤ 10 kg) IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity.
Units
Counts
Participants
OG000
Secondary
Phase 1 and 2 Population PK: Volume of Distribution of the First Peripheral Compartment (V2)
Population PK analysis was performed using nonlinear mixed effect modeling. The estimated allometric function for V2 was 0.888.
PK population: all participants who received at least one dose of nab-paclitaxel and had evaluable concentration data.
Posted
Number
liters
Cycle 1 Day 1 (Participants ≥ 6 years: 1-2 minutes prior to the end of infusion [EOI], and 15 minutes, 1, 3, 5, 8, 24, 48, and 72 hours after the EOI. Participants < 6 years: 1-2 minutes prior to the EOI, and 15 minutes, 3, 5, and 24 hours after the EOI.)
ID
Title
Description
OG000
PK Population
Phase 1: nab-paclitaxel 120-270 mg/m^2 IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity to establish the RP2D. Phase 2: nab-paclitaxel at the RP2D (240 mg/m^2 in participants weighing > 10 kg and 11.5 mg/kg in participants weighing ≤ 10 kg) IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity.
Units
Counts
Participants
OG000
Secondary
Phase 1 and 2 Population PK: Volume of Distribution of the Second Peripheral Compartment (V3)
Population PK analysis was performed using nonlinear mixed effect modeling. The estimated allometric function for V3 was 0.888.
PK population: all participants who received at least one dose of nab-paclitaxel and had evaluable concentration data.
Posted
Number
liters
Cycle 1 Day 1 (Participants ≥ 6 years: 1-2 minutes prior to the end of infusion [EOI], and 15 minutes, 1, 3, 5, 8, 24, 48, and 72 hours after the EOI. Participants < 6 years: 1-2 minutes prior to the EOI, and 15 minutes, 3, 5, and 24 hours after the EOI.)
ID
Title
Description
OG000
PK Population
Phase 1: nab-paclitaxel 120-270 mg/m^2 IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity to establish the RP2D. Phase 2: nab-paclitaxel at the RP2D (240 mg/m^2 in participants weighing > 10 kg and 11.5 mg/kg in participants weighing ≤ 10 kg) IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity.
Units
Counts
Participants
OG000
Secondary
Phase 2: Duration of Response (DOR)
Duration of response was defined as the time from the date of the first response (CR/PR, using RECIST version 1.1 guidelines) to disease progression for participants with a confirmed CR or PR. Participants who did not have disease progression or had not died were censored at the time of their last disease assessment or at time of start of new anticancer therapy, whichever occurred first. (For Phase 2 neuroblastoma patients who had both RECIST version 1.1 and Curie Score tumor evaluations, both tumor responses results were considered and an overall response was derived.)
Efficacy Evaluable Population: participants (with response) who met eligibility criteria for Phase 2, completed ≥ 1 dose of study drug, and had baseline and ≥ 1 postbaseline efficacy assessment if having not discontinued the investigational product prior to postbaseline efficacy assessment due to disease progression or systematic deterioration.
Posted
Median
Full Range
weeks
Median treatment duration in Phase 2 per group: Ewings Sarcoma = 14 weeks (3-31), Neuroblastoma = 7 weeks (3-23), Rhabdomyosarcoma = 5 weeks (1-13). Participants were followed until disease progression (if applicable) up to a maximum of 100.3 weeks.
ID
Title
Description
OG000
Phase 2: Ewing's Sarcoma
Participants with Ewing's sarcoma: nab-paclitaxel at the RP2D (240 mg/m^2 in participants weighing > 10 kg and 11.5 mg/kg in participants weighing ≤ 10 kg) IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity.
OG001
Secondary
Phase 2: Disease Control Rate (DCR)
Disease control rate was defined as the percentage of participants who achieved either a stable disease maintained for ≥ 16 weeks or confirmed CR (confirmed no less than 4 weeks after criteria for response were first met) or confirmed PR (confirmed no less than 4 weeks after criteria for response were first met) over the total number of participants available for the analysis. Confidence interval was obtained using the Clopper-Pearson method.
Efficacy Evaluable Population: participants who met eligibility criteria for Phase 2, completed at least 1 dose of study drug, and had baseline and at least 1 postbaseline efficacy assessment if having not discontinued the investigational product prior to postbaseline efficacy assessment due to disease progression or systematic deterioration.
Posted
Number
95% Confidence Interval
percentage of participants
Median treatment duration in Phase 2 per group: Ewings Sarcoma = 14 weeks (3-31), Neuroblastoma = 7 weeks (3-23), Rhabdomyosarcoma = 5 weeks (1-13).
ID
Title
Description
OG000
Phase 2: Ewing's Sarcoma
Participants with Ewing's sarcoma: nab-paclitaxel at the RP2D (240 mg/m^2 in participants weighing > 10 kg and 11.5 mg/kg in participants weighing ≤ 10 kg) IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity.
OG001
Phase 2: Neuroblastoma
Participants with neuroblastoma: nab-paclitaxel at the RP2D (240 mg/m^2 in participants weighing > 10 kg and 11.5 mg/kg in participants weighing ≤ 10 kg) IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity.
Secondary
Phase 2: Progression-Free Survival (PFS)
PFS was defined as the time from the first dose date to the start of disease progression or participant death (any cause), whichever occurred first. Disease progression was classed as either a disease progression observed as a response assessment, or a disease progression or symptomatic deterioration at treatment/study discontinuation. Participants who did not have disease progression or had not died were censored at the last known time that the participant was progression free. Disease progression was considered according to RECIST version 1.1 for Phase 2 Ewing's sarcoma and rhabdomyosarcoma participants. (For Phase 2 neuroblastoma participants who had both RECIST 1.1 and Curie score tumor evaluations, both tumor responses results were considered and an overall response was derived.) Median PFS time was estimated through Kaplan-Meier methods. 95% confidence interval about the median time to PFS event was obtained using Greenwood's method.
Efficacy Evaluable Population: participants who met eligibility criteria for Phase 2, completed at least 1 dose of study drug, and had baseline and at least 1 postbaseline efficacy assessment if having not discontinued the investigational product prior to postbaseline efficacy assessment due to disease progression or systematic deterioration.
Posted
Median
95% Confidence Interval
weeks
Median treatment duration in Phase 2 per group: Ewings Sarcoma = 14 weeks (3-31), Neuroblastoma = 7 weeks (3-23), Rhabdomyosarcoma = 5 weeks (1-13). Participants were followed until disease progression (if applicable) up to a maximum of 100.3 weeks.
ID
Title
Description
OG000
Phase 2: Ewing's Sarcoma
Participants with Ewing's sarcoma: nab-paclitaxel at the RP2D (240 mg/m^2 in participants weighing > 10 kg and 11.5 mg/kg in participants weighing ≤ 10 kg) IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity.
Secondary
Phase 2: Kaplan-Meier Estimate of Overall Survival Rate at 1 Year
Overall survival was defined as the time from the first dose date to date of death (any cause). Participants who were alive were censored at the last known time that the participant was alive.
Efficacy Evaluable Population: participants who met eligibility criteria for Phase 2, completed ≥1 dose of study drug, and had baseline and ≥1 postbaseline efficacy assessment if having not discontinued the investigational product prior to postbaseline efficacy assessment due to disease progression or systematic deterioration.
Posted
Number
95% Confidence Interval
percentage of participants
1 year
ID
Title
Description
OG000
Phase 2: Ewing's Sarcoma
Participants with Ewing's sarcoma: nab-paclitaxel at the RP2D (240 mg/m^2 in participants weighing > 10 kg and 11.5 mg/kg in participants weighing ≤ 10 kg) IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity.
OG001
Phase 2: Neuroblastoma
Participants with neuroblastoma: nab-paclitaxel at the RP2D (240 mg/m^2 in participants weighing > 10 kg and 11.5 mg/kg in participants weighing ≤ 10 kg) IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity.
OG002
Secondary
Phase 2: Number of Participants With Treatment-Emergent Adverse Events (TEAEs)
An AE was defined as any noxious, unintended, or untoward medical occurrence that may appear or worsen in a participant during the course of a study. A SAE is any AE occurring at any dose that: results in death; is life-threatening; requires inpatient hospitalization or prolongation of existing hospitalization; results in persistent or significant disability/incapacity; is a congenital anomaly/birth defect; constitutes an important medical event. TEAEs were defined as AEs that began or worsened in severity on or after the date of the first dose of study drug and within 28 days of the date of the last dose of study drug. The severity of the AEs was graded according to the Common Terminology Criteria for Adverse Events, Version 4.0. Participants were followed for 28 days after discontinuing treatment for safety and monitoring of AEs.
Safety Population: all participants who took at least 1 dose of study drug.
Posted
Count of Participants
Participants
Median treatment duration in Phase 2 per group: Ewings Sarcoma = 14 weeks (3-31), Neuroblastoma = 7 weeks (3-23), Rhabdomyosarcoma = 5 weeks (1-13). Participants were followed for 28 days after discontinuing treatment for safety and monitoring of AEs.
ID
Title
Description
OG000
Phase 2: Ewing's Sarcoma
Participants with Ewing's sarcoma: nab-paclitaxel at the RP2D (240 mg/m^2 in participants weighing > 10 kg and 11.5 mg/kg in participants weighing ≤ 10 kg) IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity.
Time Frame
Median treatment duration in Phase 1 was 7.0 weeks, with minimum and maximum duration of 1 and 49 weeks, respectively. Median treatment duration in Phase 2 per group: Ewings Sarcoma = 14 weeks (3-31), Neuroblastoma = 7 weeks (3-23), rhabdomyosarcoma = 5 weeks (1-13).
Description
All participants in both portions of the study were followed for 28 days after discontinuing treatment for safety and monitoring of AEs. AE's were analyzed in terms of TEAEs, which were defined as any AEs that began or worsened in severity on or after the start of study drug through 28 days after the last dose of study drug.
All-Cause Mortality Comment
Not provided
Arm/Groups
ID
Title
Description
Deaths (Affected)
Deaths (At Risk)
Serious Events (Affected)
Serious Events (At Risk)
Other Events (Affected)
Other Events (At Risk)
EG000
Phase 1: Nab-Paclitaxel 120 mg/m^2
nab-paclitaxel 120 mg/m^2 intravenously (IV) on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity to establish the recommended phase 2 dose (RP2D).
14
16
10
16
16
16
EG001
Phase 1: Nab-Paclitaxel 150 mg/m^2
nab-paclitaxel 150 mg/m^2 IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity to establish the RP2D.
7
8
7
8
8
8
EG002
Phase 1: Nab-Paclitaxel 180 mg/m^2
nab-paclitaxel 180 mg/m^2 IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity to establish the RP2D.
11
14
6
14
14
14
EG003
Phase 1: Nab-Paclitaxel 210 mg/m^2
nab-paclitaxel 210 mg/m^2 IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity to establish the RP2D.
5
11
5
11
11
11
EG004
Phase 1: Nab-Paclitaxel 240 mg/m^2
nab-paclitaxel 240 mg/m^2 IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity to establish the RP2D.
8
8
3
8
8
8
EG005
Phase 1: Nab-Paclitaxel 270 mg/m^2
nab-paclitaxel 270 mg/m^2 IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity to establish the RP2D.
5
7
4
7
7
7
EG006
Phase 2: Nab-Paclitaxel 240 mg/m^2
Participants with Ewing's sarcoma, neuroblastoma, or rhabdomyosarcoma: nab-paclitaxel at the RP2D (240 mg/m^2 in participants weighing > 10 kg and 11.5 mg/kg in participants weighing ≤ 10 kg) IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity.
25
42
23
42
42
42
Serious Adverse Events
Term
Organ System
Source Vocabulary
Assessment Type
Notes
Statistical Information
Febrile neutropenia
Blood and lymphatic system disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0010 affected8 at risk
EG0021 affected14 at risk
EG0030 affected11 at risk
EG0040 affected8 at risk
EG0051 affected7 at risk
EG0064 affected42 at risk
Leukopenia
Blood and lymphatic system disorders
MedDRA 20.0
Systematic Assessment
EG0001 affected16 at risk
EG0010 affected8 at risk
EG0020 affected14 at risk
EG003
Neutropenia
Blood and lymphatic system disorders
MedDRA 20.0
Systematic Assessment
EG0001 affected16 at risk
EG0010 affected8 at risk
EG0021 affected14 at risk
EG003
Thrombocytopenia
Blood and lymphatic system disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0011 affected8 at risk
EG0020 affected14 at risk
EG003
Tachycardia
Cardiac disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0011 affected8 at risk
EG0020 affected14 at risk
EG003
Abdominal pain
Gastrointestinal disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0011 affected8 at risk
EG0020 affected14 at risk
EG003
Diarrhoea
Gastrointestinal disorders
MedDRA 20.0
Systematic Assessment
EG0001 affected16 at risk
EG0010 affected8 at risk
EG0021 affected14 at risk
EG003
Nausea
Gastrointestinal disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0010 affected8 at risk
EG0020 affected14 at risk
EG003
Vomiting
Gastrointestinal disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0011 affected8 at risk
EG0021 affected14 at risk
EG003
Chills
General disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0010 affected8 at risk
EG0020 affected14 at risk
EG003
General physical health deterioration
General disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0011 affected8 at risk
EG0020 affected14 at risk
EG003
Generalised oedema
General disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0010 affected8 at risk
EG0021 affected14 at risk
EG003
Oedema peripheral
General disorders
MedDRA 20.0
Systematic Assessment
EG0001 affected16 at risk
EG0011 affected8 at risk
EG0021 affected14 at risk
EG003
Pyrexia
General disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0013 affected8 at risk
EG0022 affected14 at risk
EG003
Cellulitis
Infections and infestations
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0011 affected8 at risk
EG0020 affected14 at risk
EG003
Device related infection
Infections and infestations
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0010 affected8 at risk
EG0020 affected14 at risk
EG003
Gastroenteritis
Infections and infestations
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0010 affected8 at risk
EG0020 affected14 at risk
EG003
Lower respiratory tract infection bacterial
Infections and infestations
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0010 affected8 at risk
EG0020 affected14 at risk
EG003
Pneumonia
Infections and infestations
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0010 affected8 at risk
EG0020 affected14 at risk
EG003
Soft tissue infection
Infections and infestations
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0010 affected8 at risk
EG0020 affected14 at risk
EG003
Staphylococcal bacteraemia
Infections and infestations
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0010 affected8 at risk
EG0020 affected14 at risk
EG003
Varicella
Infections and infestations
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0010 affected8 at risk
EG0020 affected14 at risk
EG003
Anaphylactic transfusion reaction
Injury, poisoning and procedural complications
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0010 affected8 at risk
EG0020 affected14 at risk
EG003
Dehydration
Metabolism and nutrition disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0011 affected8 at risk
EG0020 affected14 at risk
EG003
Hypercreatininaemia
Metabolism and nutrition disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0011 affected8 at risk
EG0020 affected14 at risk
EG003
Hyponatraemia
Metabolism and nutrition disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0012 affected8 at risk
EG0020 affected14 at risk
EG003
Back pain
Musculoskeletal and connective tissue disorders
MedDRA 20.0
Systematic Assessment
EG0002 affected16 at risk
EG0011 affected8 at risk
EG0020 affected14 at risk
EG003
Bone pain
Musculoskeletal and connective tissue disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0010 affected8 at risk
EG0020 affected14 at risk
EG003
Pain in extremity
Musculoskeletal and connective tissue disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0011 affected8 at risk
EG0020 affected14 at risk
EG003
Cancer pain
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0010 affected8 at risk
EG0021 affected14 at risk
EG003
Osteosarcoma
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
MedDRA 20.0
Systematic Assessment
EG0001 affected16 at risk
EG0010 affected8 at risk
EG0020 affected14 at risk
EG003
Osteosarcoma metastatic
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0010 affected8 at risk
EG0020 affected14 at risk
EG003
Refractory anaemia with an excess of blasts
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0010 affected8 at risk
EG0021 affected14 at risk
EG003
Tumour pain
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
MedDRA 20.0
Systematic Assessment
EG0001 affected16 at risk
EG0010 affected8 at risk
EG0020 affected14 at risk
EG003
Dizziness
Nervous system disorders
MedDRA 20.0
Systematic Assessment
EG0001 affected16 at risk
EG0010 affected8 at risk
EG0020 affected14 at risk
EG003
Headache
Nervous system disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0010 affected8 at risk
EG0020 affected14 at risk
EG003
Intracranial pressure increased
Nervous system disorders
MedDRA 20.0
Systematic Assessment
EG0001 affected16 at risk
EG0010 affected8 at risk
EG0020 affected14 at risk
EG003
Seizure
Nervous system disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0010 affected8 at risk
EG0020 affected14 at risk
EG003
Slow speech
Nervous system disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0010 affected8 at risk
EG0020 affected14 at risk
EG003
Somnolence
Nervous system disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0011 affected8 at risk
EG0020 affected14 at risk
EG003
Tremor
Nervous system disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0010 affected8 at risk
EG0020 affected14 at risk
EG003
Restlessness
Psychiatric disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0011 affected8 at risk
EG0020 affected14 at risk
EG003
Acute kidney injury
Renal and urinary disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0011 affected8 at risk
EG0020 affected14 at risk
EG003
Anuria
Renal and urinary disorders
MedDRA 20.0
Systematic Assessment
EG0001 affected16 at risk
EG0010 affected8 at risk
EG0020 affected14 at risk
EG003
Urinary retention
Renal and urinary disorders
MedDRA 20.0
Systematic Assessment
EG0001 affected16 at risk
EG0010 affected8 at risk
EG0020 affected14 at risk
EG003
Urinary tract obstruction
Renal and urinary disorders
MedDRA 20.0
Systematic Assessment
EG0001 affected16 at risk
EG0010 affected8 at risk
EG0020 affected14 at risk
EG003
Dyspnoea
Respiratory, thoracic and mediastinal disorders
MedDRA 20.0
Systematic Assessment
EG0001 affected16 at risk
EG0010 affected8 at risk
EG0020 affected14 at risk
EG003
Hypoxia
Respiratory, thoracic and mediastinal disorders
MedDRA 20.0
Systematic Assessment
EG0001 affected16 at risk
EG0010 affected8 at risk
EG0020 affected14 at risk
EG003
Pleural effusion
Respiratory, thoracic and mediastinal disorders
MedDRA 20.0
Systematic Assessment
EG0002 affected16 at risk
EG0010 affected8 at risk
EG0020 affected14 at risk
EG003
Pneumonitis
Respiratory, thoracic and mediastinal disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0010 affected8 at risk
EG0020 affected14 at risk
EG003
Pneumothorax
Respiratory, thoracic and mediastinal disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0010 affected8 at risk
EG0020 affected14 at risk
EG003
Pulmonary embolism
Respiratory, thoracic and mediastinal disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0010 affected8 at risk
EG0021 affected14 at risk
EG003
Respiratory failure
Respiratory, thoracic and mediastinal disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0010 affected8 at risk
EG0020 affected14 at risk
EG003
Dermatitis acneiform
Skin and subcutaneous tissue disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0010 affected8 at risk
EG0020 affected14 at risk
EG003
Dermatitis bullous
Skin and subcutaneous tissue disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0010 affected8 at risk
EG0020 affected14 at risk
EG003
Erythema
Skin and subcutaneous tissue disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0010 affected8 at risk
EG0020 affected14 at risk
EG003
Skin exfoliation
Skin and subcutaneous tissue disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0010 affected8 at risk
EG0020 affected14 at risk
EG003
Hypertension
Vascular disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0011 affected8 at risk
EG0020 affected14 at risk
EG003
Hypotension
Vascular disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0011 affected8 at risk
EG0021 affected14 at risk
EG003
Other Adverse Events
Term
Organ System
Source Vocabulary
Assessment Type
Notes
Statistical Information
Anaemia
Blood and lymphatic system disorders
MedDRA 20.0
Systematic Assessment
EG0008 affected16 at risk
EG0016 affected8 at risk
EG0026 affected14 at risk
EG0035 affected11 at risk
EG0046 affected8 at risk
EG0056 affected7 at risk
EG00627 affected42 at risk
Febrile neutropenia
Blood and lymphatic system disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0010 affected8 at risk
EG0020 affected14 at risk
EG003
Leukopenia
Blood and lymphatic system disorders
MedDRA 20.0
Systematic Assessment
EG0006 affected16 at risk
EG0011 affected8 at risk
EG0022 affected14 at risk
EG003
Lymphadenitis
Blood and lymphatic system disorders
MedDRA 20.0
Systematic Assessment
EG0001 affected16 at risk
EG0010 affected8 at risk
EG0020 affected14 at risk
EG003
Lymphopenia
Blood and lymphatic system disorders
MedDRA 20.0
Systematic Assessment
EG0006 affected16 at risk
EG0012 affected8 at risk
EG0021 affected14 at risk
EG003
Neutropenia
Blood and lymphatic system disorders
MedDRA 20.0
Systematic Assessment
EG0007 affected16 at risk
EG0014 affected8 at risk
EG0026 affected14 at risk
EG003
Neutrophilia
Blood and lymphatic system disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0010 affected8 at risk
EG0020 affected14 at risk
EG003
Thrombocytopenia
Blood and lymphatic system disorders
MedDRA 20.0
Systematic Assessment
EG0001 affected16 at risk
EG0012 affected8 at risk
EG0022 affected14 at risk
EG003
Thrombocytosis
Blood and lymphatic system disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0010 affected8 at risk
EG0020 affected14 at risk
EG003
Aortic valve disease
Cardiac disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0010 affected8 at risk
EG0020 affected14 at risk
EG003
Palpitations
Cardiac disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0010 affected8 at risk
EG0021 affected14 at risk
EG003
Pericardial effusion
Cardiac disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0011 affected8 at risk
EG0020 affected14 at risk
EG003
Sinus bradycardia
Cardiac disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0011 affected8 at risk
EG0020 affected14 at risk
EG003
Sinus tachycardia
Cardiac disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0012 affected8 at risk
EG0020 affected14 at risk
EG003
Hydrocele
Congenital, familial and genetic disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0010 affected8 at risk
EG0020 affected14 at risk
EG003
Ear pain
Ear and labyrinth disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0010 affected8 at risk
EG0020 affected14 at risk
EG003
Ear swelling
Ear and labyrinth disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0010 affected8 at risk
EG0021 affected14 at risk
EG003
Vertigo
Ear and labyrinth disorders
MedDRA 20.0
Systematic Assessment
EG0001 affected16 at risk
EG0010 affected8 at risk
EG0020 affected14 at risk
EG003
Cushingoid
Endocrine disorders
MedDRA 20.0
Systematic Assessment
EG0001 affected16 at risk
EG0010 affected8 at risk
EG0020 affected14 at risk
EG003
Diplopia
Eye disorders
MedDRA 20.0
Systematic Assessment
EG0001 affected16 at risk
EG0010 affected8 at risk
EG0020 affected14 at risk
EG003
Dry eye
Eye disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0010 affected8 at risk
EG0020 affected14 at risk
EG003
Eye irritation
Eye disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0010 affected8 at risk
EG0020 affected14 at risk
EG003
Eye pain
Eye disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0011 affected8 at risk
EG0020 affected14 at risk
EG003
Eye pruritus
Eye disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0011 affected8 at risk
EG0020 affected14 at risk
EG003
Keratitis
Eye disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0010 affected8 at risk
EG0020 affected14 at risk
EG003
Lacrimation increased
Eye disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0011 affected8 at risk
EG0020 affected14 at risk
EG003
Periorbital oedema
Eye disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0010 affected8 at risk
EG0021 affected14 at risk
EG003
Photophobia
Eye disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0011 affected8 at risk
EG0020 affected14 at risk
EG003
Vision blurred
Eye disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0011 affected8 at risk
EG0020 affected14 at risk
EG003
Abdominal distension
Gastrointestinal disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0011 affected8 at risk
EG0020 affected14 at risk
EG003
Abdominal pain
Gastrointestinal disorders
MedDRA 20.0
Systematic Assessment
EG0002 affected16 at risk
EG0012 affected8 at risk
EG0023 affected14 at risk
EG003
Abdominal pain upper
Gastrointestinal disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0011 affected8 at risk
EG0022 affected14 at risk
EG003
Anal fissure
Gastrointestinal disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0011 affected8 at risk
EG0020 affected14 at risk
EG003
Anal incontinence
Gastrointestinal disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0010 affected8 at risk
EG0021 affected14 at risk
EG003
Anal inflammation
Gastrointestinal disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0010 affected8 at risk
EG0020 affected14 at risk
EG003
Anorectal discomfort
Gastrointestinal disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0010 affected8 at risk
EG0021 affected14 at risk
EG003
Ascites
Gastrointestinal disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0011 affected8 at risk
EG0020 affected14 at risk
EG003
Constipation
Gastrointestinal disorders
MedDRA 20.0
Systematic Assessment
EG0004 affected16 at risk
EG0013 affected8 at risk
EG0023 affected14 at risk
EG003
Diarrhoea
Gastrointestinal disorders
MedDRA 20.0
Systematic Assessment
EG0002 affected16 at risk
EG0014 affected8 at risk
EG0024 affected14 at risk
EG003
Diarrhoea haemorrhagic
Gastrointestinal disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0010 affected8 at risk
EG0020 affected14 at risk
EG003
Dyspepsia
Gastrointestinal disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0012 affected8 at risk
EG0021 affected14 at risk
EG003
Eructation
Gastrointestinal disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0011 affected8 at risk
EG0020 affected14 at risk
EG003
Flatulence
Gastrointestinal disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0010 affected8 at risk
EG0022 affected14 at risk
EG003
Gastrointestinal pain
Gastrointestinal disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0010 affected8 at risk
EG0020 affected14 at risk
EG003
Gastrooesophageal reflux disease
Gastrointestinal disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0010 affected8 at risk
EG0021 affected14 at risk
EG003
Haematochezia
Gastrointestinal disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0010 affected8 at risk
EG0021 affected14 at risk
EG003
Haemorrhoids
Gastrointestinal disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0010 affected8 at risk
EG0020 affected14 at risk
EG003
Nausea
Gastrointestinal disorders
MedDRA 20.0
Systematic Assessment
EG0006 affected16 at risk
EG0013 affected8 at risk
EG0022 affected14 at risk
EG003
Odynophagia
Gastrointestinal disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0010 affected8 at risk
EG0020 affected14 at risk
EG003
Salivary hypersecretion
Gastrointestinal disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0011 affected8 at risk
EG0020 affected14 at risk
EG003
Stomatitis
Gastrointestinal disorders
MedDRA 20.0
Systematic Assessment
EG0001 affected16 at risk
EG0011 affected8 at risk
EG0020 affected14 at risk
EG003
Toothache
Gastrointestinal disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0011 affected8 at risk
EG0021 affected14 at risk
EG003
Vomiting
Gastrointestinal disorders
MedDRA 20.0
Systematic Assessment
EG0008 affected16 at risk
EG0013 affected8 at risk
EG0022 affected14 at risk
EG003
Asthenia
General disorders
MedDRA 20.0
Systematic Assessment
EG0001 affected16 at risk
EG0011 affected8 at risk
EG0021 affected14 at risk
EG003
Catheter site pain
General disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0010 affected8 at risk
EG0020 affected14 at risk
EG003
Face oedema
General disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0011 affected8 at risk
EG0020 affected14 at risk
EG003
Fatigue
General disorders
MedDRA 20.0
Systematic Assessment
EG0003 affected16 at risk
EG0013 affected8 at risk
EG0024 affected14 at risk
EG003
General physical health deterioration
General disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0011 affected8 at risk
EG0021 affected14 at risk
EG003
Generalised oedema
General disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0011 affected8 at risk
EG0021 affected14 at risk
EG003
Influenza like illness
General disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0010 affected8 at risk
EG0020 affected14 at risk
EG003
Malaise
General disorders
MedDRA 20.0
Systematic Assessment
EG0001 affected16 at risk
EG0012 affected8 at risk
EG0020 affected14 at risk
EG003
Non-cardiac chest pain
General disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0012 affected8 at risk
EG0020 affected14 at risk
EG003
Oedema peripheral
General disorders
MedDRA 20.0
Systematic Assessment
EG0003 affected16 at risk
EG0013 affected8 at risk
EG0022 affected14 at risk
EG003
Pain
General disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0010 affected8 at risk
EG0020 affected14 at risk
EG003
Pyrexia
General disorders
MedDRA 20.0
Systematic Assessment
EG0006 affected16 at risk
EG0013 affected8 at risk
EG0025 affected14 at risk
EG003
Xerosis
General disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0010 affected8 at risk
EG0020 affected14 at risk
EG003
Hyperbilirubinaemia
Hepatobiliary disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0011 affected8 at risk
EG0020 affected14 at risk
EG003
Bacillus bacteraemia
Infections and infestations
MedDRA 20.0
Systematic Assessment
EG0001 affected16 at risk
EG0010 affected8 at risk
EG0020 affected14 at risk
EG003
Cellulitis
Infections and infestations
MedDRA 20.0
Systematic Assessment
EG0001 affected16 at risk
EG0011 affected8 at risk
EG0020 affected14 at risk
EG003
Clostridium difficile infection
Infections and infestations
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0011 affected8 at risk
EG0020 affected14 at risk
EG003
Conjunctivitis
Infections and infestations
MedDRA 20.0
Systematic Assessment
EG0001 affected16 at risk
EG0010 affected8 at risk
EG0020 affected14 at risk
EG003
Cystitis
Infections and infestations
MedDRA 20.0
Systematic Assessment
EG0001 affected16 at risk
EG0010 affected8 at risk
EG0020 affected14 at risk
EG003
Device related infection
Infections and infestations
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0010 affected8 at risk
EG0022 affected14 at risk
EG003
Ear infection
Infections and infestations
MedDRA 20.0
Systematic Assessment
EG0001 affected16 at risk
EG0010 affected8 at risk
EG0020 affected14 at risk
EG003
Herpes zoster
Infections and infestations
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0011 affected8 at risk
EG0021 affected14 at risk
EG003
Infection
Infections and infestations
MedDRA 20.0
Systematic Assessment
EG0001 affected16 at risk
EG0010 affected8 at risk
EG0020 affected14 at risk
EG003
Lung infection
Infections and infestations
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0011 affected8 at risk
EG0020 affected14 at risk
EG003
Mucosal infection
Infections and infestations
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0010 affected8 at risk
EG0020 affected14 at risk
EG003
Oral candidiasis
Infections and infestations
MedDRA 20.0
Systematic Assessment
EG0001 affected16 at risk
EG0010 affected8 at risk
EG0020 affected14 at risk
EG003
Oropharyngeal candidiasis
Infections and infestations
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0011 affected8 at risk
EG0020 affected14 at risk
EG003
Otitis externa
Infections and infestations
MedDRA 20.0
Systematic Assessment
EG0001 affected16 at risk
EG0010 affected8 at risk
EG0020 affected14 at risk
EG003
Otitis media
Infections and infestations
MedDRA 20.0
Systematic Assessment
EG0001 affected16 at risk
EG0010 affected8 at risk
EG0020 affected14 at risk
EG003
Paronychia
Infections and infestations
MedDRA 20.0
Systematic Assessment
EG0001 affected16 at risk
EG0010 affected8 at risk
EG0020 affected14 at risk
EG003
Rash pustular
Infections and infestations
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0011 affected8 at risk
EG0021 affected14 at risk
EG003
Rhinitis
Infections and infestations
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0011 affected8 at risk
EG0022 affected14 at risk
EG003
Skin infection
Infections and infestations
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0010 affected8 at risk
EG0020 affected14 at risk
EG003
Upper respiratory tract infection
Infections and infestations
MedDRA 20.0
Systematic Assessment
EG0002 affected16 at risk
EG0010 affected8 at risk
EG0021 affected14 at risk
EG003
Urinary tract infection
Infections and infestations
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0010 affected8 at risk
EG0020 affected14 at risk
EG003
Vulvitis
Infections and infestations
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0010 affected8 at risk
EG0020 affected14 at risk
EG003
Arthropod bite
Injury, poisoning and procedural complications
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0010 affected8 at risk
EG0021 affected14 at risk
EG003
Contusion
Injury, poisoning and procedural complications
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0010 affected8 at risk
EG0020 affected14 at risk
EG003
Excoriation
Injury, poisoning and procedural complications
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0011 affected8 at risk
EG0020 affected14 at risk
EG003
Fall
Injury, poisoning and procedural complications
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0010 affected8 at risk
EG0020 affected14 at risk
EG003
Humerus fracture
Injury, poisoning and procedural complications
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0010 affected8 at risk
EG0020 affected14 at risk
EG003
Muscle injury
Injury, poisoning and procedural complications
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0010 affected8 at risk
EG0021 affected14 at risk
EG003
Recall phenomenon
Injury, poisoning and procedural complications
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0010 affected8 at risk
EG0020 affected14 at risk
EG003
Scar
Injury, poisoning and procedural complications
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0010 affected8 at risk
EG0020 affected14 at risk
EG003
Vascular access complication
Injury, poisoning and procedural complications
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0010 affected8 at risk
EG0020 affected14 at risk
EG003
Wound secretion
Injury, poisoning and procedural complications
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0010 affected8 at risk
EG0021 affected14 at risk
EG003
Activated partial thromboplastin time prolonged
Investigations
MedDRA 20.0
Systematic Assessment
EG0001 affected16 at risk
EG0011 affected8 at risk
EG0020 affected14 at risk
EG003
Alanine aminotransferase increased
Investigations
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0013 affected8 at risk
EG0021 affected14 at risk
EG003
Aspartate aminotransferase increased
Investigations
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0012 affected8 at risk
EG0021 affected14 at risk
EG003
Blood alkaline phosphatase increased
Investigations
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0014 affected8 at risk
EG0020 affected14 at risk
EG003
Blood bicarbonate decreased
Investigations
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0011 affected8 at risk
EG0020 affected14 at risk
EG003
Blood bilirubin increased
Investigations
MedDRA 20.0
Systematic Assessment
EG0001 affected16 at risk
EG0011 affected8 at risk
EG0020 affected14 at risk
EG003
Blood creatinine increased
Investigations
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0011 affected8 at risk
EG0020 affected14 at risk
EG003
Blood lactate dehydrogenase increased
Investigations
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0010 affected8 at risk
EG0021 affected14 at risk
EG003
Blood urea increased
Investigations
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0010 affected8 at risk
EG0020 affected14 at risk
EG003
C-reactive protein increased
Investigations
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0010 affected8 at risk
EG0020 affected14 at risk
EG003
Candida test positive
Investigations
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0010 affected8 at risk
EG0020 affected14 at risk
EG003
Electrocardiogram QT prolonged
Investigations
MedDRA 20.0
Systematic Assessment
EG0001 affected16 at risk
EG0010 affected8 at risk
EG0020 affected14 at risk
EG003
Electroencephalogram abnormal
Investigations
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0010 affected8 at risk
EG0020 affected14 at risk
EG003
Gamma-glutamyltransferase increased
Investigations
MedDRA 20.0
Systematic Assessment
EG0001 affected16 at risk
EG0011 affected8 at risk
EG0020 affected14 at risk
EG003
Glucose urine present
Investigations
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0011 affected8 at risk
EG0020 affected14 at risk
EG003
International normalised ratio increased
Investigations
MedDRA 20.0
Systematic Assessment
EG0001 affected16 at risk
EG0011 affected8 at risk
EG0020 affected14 at risk
EG003
Neutrophil count decreased
Investigations
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0010 affected8 at risk
EG0020 affected14 at risk
EG003
Platelet count decreased
Investigations
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0010 affected8 at risk
EG0020 affected14 at risk
EG003
Urine output decreased
Investigations
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0011 affected8 at risk
EG0020 affected14 at risk
EG003
Weight decreased
Investigations
MedDRA 20.0
Systematic Assessment
EG0002 affected16 at risk
EG0010 affected8 at risk
EG0020 affected14 at risk
EG003
Weight increased
Investigations
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0010 affected8 at risk
EG0021 affected14 at risk
EG003
Acidosis
Metabolism and nutrition disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0011 affected8 at risk
EG0020 affected14 at risk
EG003
Cachexia
Metabolism and nutrition disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0011 affected8 at risk
EG0020 affected14 at risk
EG003
Decreased appetite
Metabolism and nutrition disorders
MedDRA 20.0
Systematic Assessment
EG0003 affected16 at risk
EG0013 affected8 at risk
EG0025 affected14 at risk
EG003
Dehydration
Metabolism and nutrition disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0011 affected8 at risk
EG0020 affected14 at risk
EG003
Hyperchloraemia
Metabolism and nutrition disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0011 affected8 at risk
EG0020 affected14 at risk
EG003
Hypercreatininaemia
Metabolism and nutrition disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0011 affected8 at risk
EG0020 affected14 at risk
EG003
Hyperglycaemia
Metabolism and nutrition disorders
MedDRA 20.0
Systematic Assessment
EG0002 affected16 at risk
EG0011 affected8 at risk
EG0020 affected14 at risk
EG003
Hyperkalaemia
Metabolism and nutrition disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0011 affected8 at risk
EG0020 affected14 at risk
EG003
Hypermagnesaemia
Metabolism and nutrition disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0011 affected8 at risk
EG0020 affected14 at risk
EG003
Hypernatraemia
Metabolism and nutrition disorders
MedDRA 20.0
Systematic Assessment
EG0001 affected16 at risk
EG0010 affected8 at risk
EG0020 affected14 at risk
EG003
Hyperphosphataemia
Metabolism and nutrition disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0011 affected8 at risk
EG0020 affected14 at risk
EG003
Hypertriglyceridaemia
Metabolism and nutrition disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0011 affected8 at risk
EG0020 affected14 at risk
EG003
Hypoalbuminaemia
Metabolism and nutrition disorders
MedDRA 20.0
Systematic Assessment
EG0002 affected16 at risk
EG0013 affected8 at risk
EG0020 affected14 at risk
EG003
Hypocalcaemia
Metabolism and nutrition disorders
MedDRA 20.0
Systematic Assessment
EG0002 affected16 at risk
EG0012 affected8 at risk
EG0021 affected14 at risk
EG003
Hypochloraemia
Metabolism and nutrition disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0011 affected8 at risk
EG0020 affected14 at risk
EG003
Hypoglycaemia
Metabolism and nutrition disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0011 affected8 at risk
EG0020 affected14 at risk
EG003
Hypokalaemia
Metabolism and nutrition disorders
MedDRA 20.0
Systematic Assessment
EG0001 affected16 at risk
EG0013 affected8 at risk
EG0022 affected14 at risk
EG003
Hypomagnesaemia
Metabolism and nutrition disorders
MedDRA 20.0
Systematic Assessment
EG0001 affected16 at risk
EG0011 affected8 at risk
EG0021 affected14 at risk
EG003
Hyponatraemia
Metabolism and nutrition disorders
MedDRA 20.0
Systematic Assessment
EG0001 affected16 at risk
EG0014 affected8 at risk
EG0021 affected14 at risk
EG003
Hypophosphataemia
Metabolism and nutrition disorders
MedDRA 20.0
Systematic Assessment
EG0001 affected16 at risk
EG0010 affected8 at risk
EG0020 affected14 at risk
EG003
Polydipsia
Metabolism and nutrition disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0010 affected8 at risk
EG0021 affected14 at risk
EG003
Arthralgia
Musculoskeletal and connective tissue disorders
MedDRA 20.0
Systematic Assessment
EG0001 affected16 at risk
EG0012 affected8 at risk
EG0020 affected14 at risk
EG003
Back pain
Musculoskeletal and connective tissue disorders
MedDRA 20.0
Systematic Assessment
EG0002 affected16 at risk
EG0012 affected8 at risk
EG0023 affected14 at risk
EG003
Bone pain
Musculoskeletal and connective tissue disorders
MedDRA 20.0
Systematic Assessment
EG0001 affected16 at risk
EG0011 affected8 at risk
EG0020 affected14 at risk
EG003
Coccydynia
Musculoskeletal and connective tissue disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0011 affected8 at risk
EG0020 affected14 at risk
EG003
Epiphysiolysis
Musculoskeletal and connective tissue disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0010 affected8 at risk
EG0020 affected14 at risk
EG003
Flank pain
Musculoskeletal and connective tissue disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0011 affected8 at risk
EG0020 affected14 at risk
EG003
Groin pain
Musculoskeletal and connective tissue disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0010 affected8 at risk
EG0020 affected14 at risk
EG003
Joint swelling
Musculoskeletal and connective tissue disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0010 affected8 at risk
EG0020 affected14 at risk
EG003
Muscular weakness
Musculoskeletal and connective tissue disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0010 affected8 at risk
EG0020 affected14 at risk
EG003
Musculoskeletal chest pain
Musculoskeletal and connective tissue disorders
MedDRA 20.0
Systematic Assessment
EG0001 affected16 at risk
EG0010 affected8 at risk
EG0021 affected14 at risk
EG003
Musculoskeletal pain
Musculoskeletal and connective tissue disorders
MedDRA 20.0
Systematic Assessment
EG0002 affected16 at risk
EG0011 affected8 at risk
EG0021 affected14 at risk
EG003
Myalgia
Musculoskeletal and connective tissue disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0014 affected8 at risk
EG0020 affected14 at risk
EG003
Neck pain
Musculoskeletal and connective tissue disorders
MedDRA 20.0
Systematic Assessment
EG0001 affected16 at risk
EG0010 affected8 at risk
EG0021 affected14 at risk
EG003
Pain in extremity
Musculoskeletal and connective tissue disorders
MedDRA 20.0
Systematic Assessment
EG0002 affected16 at risk
EG0013 affected8 at risk
EG0022 affected14 at risk
EG003
Pain in jaw
Musculoskeletal and connective tissue disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0010 affected8 at risk
EG0020 affected14 at risk
EG003
Spinal pain
Musculoskeletal and connective tissue disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0010 affected8 at risk
EG0020 affected14 at risk
EG003
Cancer pain
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
MedDRA 20.0
Systematic Assessment
EG0001 affected16 at risk
EG0010 affected8 at risk
EG0022 affected14 at risk
EG003
Skin papilloma
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0010 affected8 at risk
EG0020 affected14 at risk
EG003
Tumour haemorrhage
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0010 affected8 at risk
EG0021 affected14 at risk
EG003
Tumour pain
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
MedDRA 20.0
Systematic Assessment
EG0002 affected16 at risk
EG0011 affected8 at risk
EG0020 affected14 at risk
EG003
Depressed level of consciousness
Nervous system disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0010 affected8 at risk
EG0021 affected14 at risk
EG003
Dizziness
Nervous system disorders
MedDRA 20.0
Systematic Assessment
EG0001 affected16 at risk
EG0012 affected8 at risk
EG0021 affected14 at risk
EG003
Dysaesthesia
Nervous system disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0010 affected8 at risk
EG0020 affected14 at risk
EG003
Dysgeusia
Nervous system disorders
MedDRA 20.0
Systematic Assessment
EG0001 affected16 at risk
EG0011 affected8 at risk
EG0020 affected14 at risk
EG003
Headache
Nervous system disorders
MedDRA 20.0
Systematic Assessment
EG0003 affected16 at risk
EG0012 affected8 at risk
EG0023 affected14 at risk
EG003
Lethargy
Nervous system disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0010 affected8 at risk
EG0021 affected14 at risk
EG003
Neuralgia
Nervous system disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0010 affected8 at risk
EG0020 affected14 at risk
EG003
Neuropathy peripheral
Nervous system disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0010 affected8 at risk
EG0021 affected14 at risk
EG003
Paraesthesia
Nervous system disorders
MedDRA 20.0
Systematic Assessment
EG0001 affected16 at risk
EG0010 affected8 at risk
EG0020 affected14 at risk
EG003
Peripheral motor neuropathy
Nervous system disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0010 affected8 at risk
EG0021 affected14 at risk
EG003
Peripheral sensory neuropathy
Nervous system disorders
MedDRA 20.0
Systematic Assessment
EG0001 affected16 at risk
EG0010 affected8 at risk
EG0022 affected14 at risk
EG003
Posterior reversible encephalopathy syndrome
Nervous system disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0010 affected8 at risk
EG0020 affected14 at risk
EG003
Sciatica
Nervous system disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0010 affected8 at risk
EG0021 affected14 at risk
EG003
Somnolence
Nervous system disorders
MedDRA 20.0
Systematic Assessment
EG0001 affected16 at risk
EG0011 affected8 at risk
EG0021 affected14 at risk
EG003
Spinal cord compression
Nervous system disorders
MedDRA 20.0
Systematic Assessment
EG0001 affected16 at risk
EG0010 affected8 at risk
EG0020 affected14 at risk
EG003
Visual field defect
Nervous system disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0011 affected8 at risk
EG0020 affected14 at risk
EG003
Anxiety
Psychiatric disorders
MedDRA 20.0
Systematic Assessment
EG0002 affected16 at risk
EG0013 affected8 at risk
EG0021 affected14 at risk
EG003
Confusional state
Psychiatric disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0010 affected8 at risk
EG0021 affected14 at risk
EG003
Depression
Psychiatric disorders
MedDRA 20.0
Systematic Assessment
EG0001 affected16 at risk
EG0012 affected8 at risk
EG0020 affected14 at risk
EG003
Enuresis
Psychiatric disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0011 affected8 at risk
EG0020 affected14 at risk
EG003
Insomnia
Psychiatric disorders
MedDRA 20.0
Systematic Assessment
EG0001 affected16 at risk
EG0010 affected8 at risk
EG0020 affected14 at risk
EG003
Mood altered
Psychiatric disorders
MedDRA 20.0
Systematic Assessment
EG0001 affected16 at risk
EG0010 affected8 at risk
EG0020 affected14 at risk
EG003
Restlessness
Psychiatric disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0011 affected8 at risk
EG0020 affected14 at risk
EG003
Acute kidney injury
Renal and urinary disorders
MedDRA 20.0
Systematic Assessment
EG0001 affected16 at risk
EG0011 affected8 at risk
EG0020 affected14 at risk
EG003
Dysuria
Renal and urinary disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0011 affected8 at risk
EG0020 affected14 at risk
EG003
Haematuria
Renal and urinary disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0011 affected8 at risk
EG0020 affected14 at risk
EG003
Ketonuria
Renal and urinary disorders
MedDRA 20.0
Systematic Assessment
EG0001 affected16 at risk
EG0010 affected8 at risk
EG0020 affected14 at risk
EG003
Pollakiuria
Renal and urinary disorders
MedDRA 20.0
Systematic Assessment
EG0001 affected16 at risk
EG0010 affected8 at risk
EG0020 affected14 at risk
EG003
Polyuria
Renal and urinary disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0010 affected8 at risk
EG0021 affected14 at risk
EG003
Renal pain
Renal and urinary disorders
MedDRA 20.0
Systematic Assessment
EG0001 affected16 at risk
EG0010 affected8 at risk
EG0020 affected14 at risk
EG003
Urinary incontinence
Renal and urinary disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0010 affected8 at risk
EG0020 affected14 at risk
EG003
Urinary retention
Renal and urinary disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0011 affected8 at risk
EG0021 affected14 at risk
EG003
Urinary tract obstruction
Renal and urinary disorders
MedDRA 20.0
Systematic Assessment
EG0001 affected16 at risk
EG0011 affected8 at risk
EG0020 affected14 at risk
EG003
Urinary tract pain
Renal and urinary disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0010 affected8 at risk
EG0020 affected14 at risk
EG003
Genital pain
Reproductive system and breast disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0011 affected8 at risk
EG0020 affected14 at risk
EG003
Menstruation irregular
Reproductive system and breast disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0010 affected8 at risk
EG0020 affected14 at risk
EG003
Oedema genital
Reproductive system and breast disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0012 affected8 at risk
EG0020 affected14 at risk
EG003
Scrotal oedema
Reproductive system and breast disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0010 affected8 at risk
EG0021 affected14 at risk
EG003
Vaginal haemorrhage
Reproductive system and breast disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0010 affected8 at risk
EG0020 affected14 at risk
EG003
Vulvovaginal discomfort
Reproductive system and breast disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0010 affected8 at risk
EG0020 affected14 at risk
EG003
Atelectasis
Respiratory, thoracic and mediastinal disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0010 affected8 at risk
EG0020 affected14 at risk
EG003
Cough
Respiratory, thoracic and mediastinal disorders
MedDRA 20.0
Systematic Assessment
EG0002 affected16 at risk
EG0013 affected8 at risk
EG0022 affected14 at risk
EG003
Dysaesthesia pharynx
Respiratory, thoracic and mediastinal disorders
MedDRA 20.0
Systematic Assessment
EG0001 affected16 at risk
EG0010 affected8 at risk
EG0020 affected14 at risk
EG003
Dysphonia
Respiratory, thoracic and mediastinal disorders
MedDRA 20.0
Systematic Assessment
EG0001 affected16 at risk
EG0010 affected8 at risk
EG0020 affected14 at risk
EG003
Dyspnoea
Respiratory, thoracic and mediastinal disorders
MedDRA 20.0
Systematic Assessment
EG0003 affected16 at risk
EG0010 affected8 at risk
EG0022 affected14 at risk
EG003
Epistaxis
Respiratory, thoracic and mediastinal disorders
MedDRA 20.0
Systematic Assessment
EG0001 affected16 at risk
EG0012 affected8 at risk
EG0021 affected14 at risk
EG003
Haemoptysis
Respiratory, thoracic and mediastinal disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0011 affected8 at risk
EG0021 affected14 at risk
EG003
Hypoxia
Respiratory, thoracic and mediastinal disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0010 affected8 at risk
EG0020 affected14 at risk
EG003
Lung consolidation
Respiratory, thoracic and mediastinal disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0011 affected8 at risk
EG0020 affected14 at risk
EG003
Nasal congestion
Respiratory, thoracic and mediastinal disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0011 affected8 at risk
EG0020 affected14 at risk
EG003
Oropharyngeal pain
Respiratory, thoracic and mediastinal disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0012 affected8 at risk
EG0020 affected14 at risk
EG003
Pharyngeal erythema
Respiratory, thoracic and mediastinal disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0012 affected8 at risk
EG0021 affected14 at risk
EG003
Pharyngeal inflammation
Respiratory, thoracic and mediastinal disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0010 affected8 at risk
EG0021 affected14 at risk
EG003
Pleural effusion
Respiratory, thoracic and mediastinal disorders
MedDRA 20.0
Systematic Assessment
EG0002 affected16 at risk
EG0011 affected8 at risk
EG0020 affected14 at risk
EG003
Productive cough
Respiratory, thoracic and mediastinal disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0010 affected8 at risk
EG0021 affected14 at risk
EG003
Pulmonary haemorrhage
Respiratory, thoracic and mediastinal disorders
MedDRA 20.0
Systematic Assessment
EG0001 affected16 at risk
EG0010 affected8 at risk
EG0020 affected14 at risk
EG003
Rhinitis allergic
Respiratory, thoracic and mediastinal disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0011 affected8 at risk
EG0020 affected14 at risk
EG003
Tachypnoea
Respiratory, thoracic and mediastinal disorders
MedDRA 20.0
Systematic Assessment
EG0001 affected16 at risk
EG0010 affected8 at risk
EG0020 affected14 at risk
EG003
Wheezing
Respiratory, thoracic and mediastinal disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0010 affected8 at risk
EG0020 affected14 at risk
EG003
Alopecia
Skin and subcutaneous tissue disorders
MedDRA 20.0
Systematic Assessment
EG0002 affected16 at risk
EG0014 affected8 at risk
EG0024 affected14 at risk
EG003
Dermatitis
Skin and subcutaneous tissue disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0010 affected8 at risk
EG0020 affected14 at risk
EG003
Dermatitis acneiform
Skin and subcutaneous tissue disorders
MedDRA 20.0
Systematic Assessment
EG0001 affected16 at risk
EG0010 affected8 at risk
EG0020 affected14 at risk
EG003
Dermatitis atopic
Skin and subcutaneous tissue disorders
MedDRA 20.0
Systematic Assessment
EG0001 affected16 at risk
EG0010 affected8 at risk
EG0020 affected14 at risk
EG003
Dry skin
Skin and subcutaneous tissue disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0010 affected8 at risk
EG0021 affected14 at risk
EG003
Ecchymosis
Skin and subcutaneous tissue disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0010 affected8 at risk
EG0020 affected14 at risk
EG003
Erythema
Skin and subcutaneous tissue disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0011 affected8 at risk
EG0020 affected14 at risk
EG003
Haemorrhage subcutaneous
Skin and subcutaneous tissue disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0010 affected8 at risk
EG0020 affected14 at risk
EG003
Intertrigo
Skin and subcutaneous tissue disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0010 affected8 at risk
EG0020 affected14 at risk
EG003
Nail bed inflammation
Skin and subcutaneous tissue disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0010 affected8 at risk
EG0020 affected14 at risk
EG003
Nail discolouration
Skin and subcutaneous tissue disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0010 affected8 at risk
EG0020 affected14 at risk
EG003
Onychoclasis
Skin and subcutaneous tissue disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0010 affected8 at risk
EG0020 affected14 at risk
EG003
Onycholysis
Skin and subcutaneous tissue disorders
MedDRA 20.0
Systematic Assessment
EG0001 affected16 at risk
EG0010 affected8 at risk
EG0020 affected14 at risk
EG003
Pain of skin
Skin and subcutaneous tissue disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0010 affected8 at risk
EG0020 affected14 at risk
EG003
Palmar-plantar erythrodysaesthesia syndrome
Skin and subcutaneous tissue disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0011 affected8 at risk
EG0020 affected14 at risk
EG003
Photosensitivity reaction
Skin and subcutaneous tissue disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0010 affected8 at risk
EG0020 affected14 at risk
EG003
Pruritus
Skin and subcutaneous tissue disorders
MedDRA 20.0
Systematic Assessment
EG0001 affected16 at risk
EG0012 affected8 at risk
EG0020 affected14 at risk
EG003
Pruritus generalised
Skin and subcutaneous tissue disorders
MedDRA 20.0
Systematic Assessment
EG0003 affected16 at risk
EG0010 affected8 at risk
EG0021 affected14 at risk
EG003
Rash
Skin and subcutaneous tissue disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0010 affected8 at risk
EG0020 affected14 at risk
EG003
Rash erythematous
Skin and subcutaneous tissue disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0010 affected8 at risk
EG0021 affected14 at risk
EG003
Rash macular
Skin and subcutaneous tissue disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0010 affected8 at risk
EG0020 affected14 at risk
EG003
Rash maculo-papular
Skin and subcutaneous tissue disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0011 affected8 at risk
EG0020 affected14 at risk
EG003
Rash papular
Skin and subcutaneous tissue disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0010 affected8 at risk
EG0020 affected14 at risk
EG003
Skin exfoliation
Skin and subcutaneous tissue disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0010 affected8 at risk
EG0021 affected14 at risk
EG003
Skin hyperpigmentation
Skin and subcutaneous tissue disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0011 affected8 at risk
EG0020 affected14 at risk
EG003
Toxic erythema of chemotherapy
Skin and subcutaneous tissue disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0010 affected8 at risk
EG0020 affected14 at risk
EG003
Hyperaemia
Vascular disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0010 affected8 at risk
EG0020 affected14 at risk
EG003
Hypertension
Vascular disorders
MedDRA 20.0
Systematic Assessment
EG0001 affected16 at risk
EG0011 affected8 at risk
EG0020 affected14 at risk
EG003
Hypotension
Vascular disorders
MedDRA 20.0
Systematic Assessment
EG0000 affected16 at risk
EG0011 affected8 at risk
EG0022 affected14 at risk
EG003
Lymphoedema
Vascular disorders
MedDRA 20.0
Systematic Assessment
EG0001 affected16 at risk
EG0010 affected8 at risk
EG0020 affected14 at risk
EG003
Certain Agreements
Are all PI(s) employees of the sponsor?
No
Restriction Type
OTHER
Results Disclosure Restriction on PI(s)?
Yes
Other Details
Results from a center cannot be submitted for publication before results of multicenter study are published unless it is more than one year since study completion. Then, Investigator can publish if manuscript is submitted to Celgene 60 days prior to submission. If Celgene decides publication would hinder drug development, Investigator must delay submission for up to ninety days. Investigator must delete confidential information before submission or defer publication to permit patent applications
Gr 4 Thrombocytopenia/Anemia Persisting >7 days...
Title
Measurements
OG0000
OG0010
OG0020
OG0030
OG0040
OG0050
Gr 3 Thrombocytopenia with Bleeding
Title
Measurements
OG0000
OG0010
OG0020
OG0030
OG0040
OG0050
Gr 4 Uncomplicated Neutropenia Lasting >7 days
Title
Measurements
OG0000
OG0010
OG0020
OG0030
OG0040
OG0051
Febrile Neutropenia+Confirmed Bacterial Infection
Title
Measurements
OG0000
OG0010
OG0020
OG0030
OG0040
OG0050
Gr3 Hematologic Toxicity Requiring Tx Delay...
Title
Measurements
OG0000
OG0010
OG0020
OG0030
OG0040
OG0050
nab-paclitaxel 150 mg/m^2 IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity to establish the RP2D.
OG002
Phase 1: Nab-Paclitaxel 180 mg/m^2
nab-paclitaxel 180 mg/m^2 IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity to establish the RP2D.
OG003
Phase 1: Nab-Paclitaxel 210 mg/m^2
nab-paclitaxel 210 mg/m^2 IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity to establish the RP2D.
OG004
Phase 1: Nab-Paclitaxel 240 mg/m^2
nab-paclitaxel 240 mg/m^2 IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity to establish the RP2D.
OG005
Phase 1: Nab-Paclitaxel 270 mg/m^2
nab-paclitaxel 270 mg/m^2 IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity to establish the RP2D.
Units
Counts
Participants
OG00016
OG0018
OG00214
OG00311
OG0048
OG0057
Title
Denominators
Categories
TEAE
Title
Measurements
OG00016
OG0018
OG00214
OG00311
OG0048
OG0057
Treatment-related (TR) TEAE
Title
Measurements
OG00014
OG0018
OG00212
OG003
Grade 3 or 4 TEAE
Title
Measurements
OG00013
OG0018
OG00210
OG003
TR Grade 3 or 4 TEAE
Title
Measurements
OG0009
OG0017
OG0027
OG003
Serious TEAE
Title
Measurements
OG00010
OG0017
OG0026
OG003
TR Serious TEAE
Title
Measurements
OG0001
OG0014
OG0024
OG003
TEAE Leading to Drug Discontinuation
Title
Measurements
OG0004
OG0010
OG0022
OG003
TR TEAE Leading to Drug Discontinuation
Title
Measurements
OG0001
OG0010
OG0020
OG003
TEAE Leading to Dose Reduction
Title
Measurements
OG0000
OG0011
OG0022
OG003
TR TEAE Leading to Dose Reduction
Title
Measurements
OG0000
OG0011
OG0022
OG003
TEAE Leading to Drug Interruption
Title
Measurements
OG0002
OG0012
OG0023
OG003
TR TEAE Leading to Drug Interruption
Title
Measurements
OG0000
OG0012
OG0021
OG003
TEAE Leading to Death
Title
Measurements
OG0002
OG0011
OG0020
OG003
TR TEAE Leading to Death
Title
Measurements
OG0000
OG0010
OG0020
OG003
Phase 2: Neuroblastoma
Participants with neuroblastoma: nab-paclitaxel at the RP2D (240 mg/m^2 in participants weighing > 10 kg and 11.5 mg/kg in participants weighing ≤ 10 kg) IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity.
OG002
Phase 2: Rhabdomyosarcoma
Participants with rhabdomyosarcoma: nab-paclitaxel at the RP2D (240 mg/m^2 in participants weighing > 10 kg and 11.5 mg/kg in participants weighing ≤ 10 kg) IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity.
Units
Counts
Participants
OG00013
OG00114
OG00214
Title
Denominators
Categories
Title
Measurements
OG0000(0.0 to 24.7)
OG0010(0.0 to 23.2)
OG0027.1(0.2 to 33.9)
OG002
Phase 1: Nab-Paclitaxel 180 mg/m^2
nab-paclitaxel 180 mg/m^2 IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity to establish the RP2D.
OG003
Phase 1: Nab-Paclitaxel 210 mg/m^2
nab-paclitaxel 210 mg/m^2 IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity to establish the RP2D.
OG004
Phase 1: Nab-Paclitaxel 240 mg/m^2
nab-paclitaxel 240 mg/m^2 IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity to establish the RP2D.
OG005
Phase 1: Nab-Paclitaxel 270 mg/m^2
nab-paclitaxel 270 mg/m^2 IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity to establish the RP2D.
Units
Counts
Participants
OG00014
OG0018
OG00212
OG00310
OG0048
OG0057
Title
Denominators
Categories
Title
Measurements
OG0000(0.0 to 23.2)
OG0010(0.0 to 36.9)
OG0020(0.0 to 26.5)
OG0030(0.0 to 30.8)
OG00412.5(0.3 to 52.7)
OG00514.3(0.4 to 57.9)
OG003
Phase 1: Nab-Paclitaxel 210 mg/m^2
nab-paclitaxel 210 mg/m^2 IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity to establish the RP2D.
OG004
Phase 1: Nab-Paclitaxel 240 mg/m^2
nab-paclitaxel 240 mg/m^2 IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity to establish the RP2D.
OG005
Phase 1: Nab-Paclitaxel 270 mg/m^2
nab-paclitaxel 270 mg/m^2 IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity to establish the RP2D.
Units
Counts
Participants
OG00013
OG0017
OG00212
OG0039
OG0047
OG0056
Title
Denominators
Categories
Title
Measurements
OG0003488± 73.7
OG0015468± 38.0
OG0025597± 33.4
OG0035616± 63.9
OG0047831± 23.1
OG0058078± 41.5
OG003
Phase 1: Nab-Paclitaxel 210 mg/m^2
nab-paclitaxel 210 mg/m^2 IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity to establish the RP2D.
OG004
Phase 1: Nab-Paclitaxel 240 mg/m^2
nab-paclitaxel 240 mg/m^2 IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity to establish the RP2D.
OG005
Phase 1: Nab-Paclitaxel 270 mg/m^2
nab-paclitaxel 270 mg/m^2 IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity to establish the RP2D.
Units
Counts
Participants
OG00013
OG0017
OG00212
OG0039
OG0047
OG0056
Title
Denominators
Categories
Title
Measurements
OG00023.3± 87.5
OG00125.4± 46.6
OG00227.3± 47.3
OG00323.2± 80.3
OG00428.2± 48.7
OG00521.0± 46.6
nab-paclitaxel 180 mg/m^2 IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity to establish the RP2D.
OG003
Phase 1: Nab-Paclitaxel 210 mg/m^2
nab-paclitaxel 210 mg/m^2 IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity to establish the RP2D.
OG004
Phase 1: Nab-Paclitaxel 240 mg/m^2
nab-paclitaxel 240 mg/m^2 IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity to establish the RP2D.
OG005
Phase 1: Nab-Paclitaxel 270 mg/m^2
nab-paclitaxel 270 mg/m^2 IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity to establish the RP2D.
Units
Counts
Participants
OG00013
OG0017
OG00212
OG0039
OG0047
OG0056
Title
Denominators
Categories
AUCt
ParticipantsOG00013
ParticipantsOG0017
ParticipantsOG00212
ParticipantsOG0039
ParticipantsOG0047
ParticipantsOG0056
Title
Measurements
OG0007844± 73.4
OG00110374± 91.8
OG0029690± 37.1
OG003
AUC24
ParticipantsOG00013
ParticipantsOG0017
ParticipantsOG00212
ParticipantsOG0038
AUCinf
ParticipantsOG0009
ParticipantsOG0016
ParticipantsOG00210
ParticipantsOG0036
OG003
Phase 1: Nab-Paclitaxel 210 mg/m^2
nab-paclitaxel 210 mg/m^2 IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity to establish the RP2D.
OG004
Phase 1: Nab-Paclitaxel 240 mg/m^2
nab-paclitaxel 240 mg/m^2 IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity to establish the RP2D.
OG005
Phase 1: Nab-Paclitaxel 270 mg/m^2
nab-paclitaxel 270 mg/m^2 IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity to establish the RP2D.
Units
Counts
Participants
OG00013
OG0017
OG00212
OG0038
OG0047
OG0056
Title
Denominators
Categories
AUC24
ParticipantsOG00013
ParticipantsOG0017
ParticipantsOG00212
ParticipantsOG0038
ParticipantsOG0047
ParticipantsOG0056
Title
Measurements
OG00042.7± 77.4
OG00141.6± 87.0
OG00240.8± 39.7
OG003
AUCinf
ParticipantsOG0009
ParticipantsOG0016
ParticipantsOG00210
ParticipantsOG0036
OG003
Phase 1: Nab-Paclitaxel 210 mg/m^2
nab-paclitaxel 210 mg/m^2 IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity to establish the RP2D.
OG004
Phase 1: Nab-Paclitaxel 240 mg/m^2
nab-paclitaxel 240 mg/m^2 IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity to establish the RP2D.
OG005
Phase 1: Nab-Paclitaxel 270 mg/m^2
nab-paclitaxel 270 mg/m^2 IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity to establish the RP2D.
Units
Counts
Participants
OG0009
OG0016
OG00210
OG0036
OG0046
OG0055
Title
Denominators
Categories
Title
Measurements
OG00016.1± 75.6
OG00120.3± 101
OG00220.9± 39.9
OG00315.4± 25.4
OG00419.1± 67.4
OG00531.9± 35.0
OG003
Phase 1: Nab-Paclitaxel 210 mg/m^2
nab-paclitaxel 210 mg/m^2 IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity to establish the RP2D.
OG004
Phase 1: Nab-Paclitaxel 240 mg/m^2
nab-paclitaxel 240 mg/m^2 IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity to establish the RP2D.
OG005
Phase 1: Nab-Paclitaxel 270 mg/m^2
nab-paclitaxel 270 mg/m^2 IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity to establish the RP2D.
Units
Counts
Participants
OG0009
OG0016
OG00210
OG0036
OG0046
OG0055
Title
Denominators
Categories
Title
Measurements
OG00013.5± 85.1
OG00112.5± 99.3
OG00217.8± 38.3
OG00314.6± 72.3
OG00416.7± 29.2
OG00521.8± 28.4
OG003
Phase 1: Nab-Paclitaxel 210 mg/m^2
nab-paclitaxel 210 mg/m^2 IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity to establish the RP2D.
OG004
Phase 1: Nab-Paclitaxel 240 mg/m^2
nab-paclitaxel 240 mg/m^2 IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity to establish the RP2D.
OG005
Phase 1: Nab-Paclitaxel 270 mg/m^2
nab-paclitaxel 270 mg/m^2 IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity to establish the RP2D.
Units
Counts
Participants
OG0009
OG0016
OG00210
OG0036
OG0046
OG0055
Title
Denominators
Categories
Title
Measurements
OG000127± 145
OG001266± 78.3
OG002146± 106
OG00389.8± 45.1
OG004175± 117
OG005446± 17.6
OG003
Phase 1: Nab-Paclitaxel 210 mg/m^2
nab-paclitaxel 210 mg/m^2 IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity to establish the RP2D.
OG004
Phase 1: Nab-Paclitaxel 240 mg/m^2
nab-paclitaxel 240 mg/m^2 IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity to establish the RP2D.
OG005
Phase 1: Nab-Paclitaxel 270 mg/m^2
nab-paclitaxel 270 mg/m^2 IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity to establish the RP2D.
Units
Counts
Participants
OG0009
OG0016
OG00210
OG0036
OG0046
OG0055
Title
Denominators
Categories
Title
Measurements
OG000106± 95.3
OG001164± 78.4
OG002124± 82.8
OG00384.9± 49.7
OG004154± 56.5
OG005304± 29.0
106
Title
Denominators
Categories
Title
Measurements
OG00011.8
106
Title
Denominators
Categories
Title
Measurements
OG00031983
106
Title
Denominators
Categories
Title
Measurements
OG000951
106
Title
Denominators
Categories
Title
Measurements
OG00022.4
106
Title
Denominators
Categories
Title
Measurements
OG00034.8
106
Title
Denominators
Categories
Title
Measurements
OG000545
106
Title
Denominators
Categories
Title
Measurements
OG00045.3
Phase 2: Neuroblastoma
Participants with neuroblastoma: nab-paclitaxel at the RP2D (240 mg/m^2 in participants weighing > 10 kg and 11.5 mg/kg in participants weighing ≤ 10 kg) IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity.
OG002
Phase 2: Rhabdomyosarcoma
Participants with rhabdomyosarcoma: nab-paclitaxel at the RP2D (240 mg/m^2 in participants weighing > 10 kg and 11.5 mg/kg in participants weighing ≤ 10 kg) IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity.
Units
Counts
Participants
OG0000
OG0010
OG0021
Title
Denominators
Categories
Title
Measurements
OG0026.14(6.14 to 6.14)
OG002
Phase 2: Rhabdomyosarcoma
Participants with rhabdomyosarcoma: nab-paclitaxel at the RP2D (240 mg/m^2 in participants weighing > 10 kg and 11.5 mg/kg in participants weighing ≤ 10 kg) IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity.
Units
Counts
Participants
OG00013
OG00114
OG00214
Title
Denominators
Categories
Title
Measurements
OG00030.8(9.1 to 61.4)
OG0017.1(0.2 to 33.9)
OG0027.1(0.2 to 33.9)
OG001
Phase 2: Neuroblastoma
Participants with neuroblastoma: nab-paclitaxel at the RP2D (240 mg/m^2 in participants weighing > 10 kg and 11.5 mg/kg in participants weighing ≤ 10 kg) IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity.
OG002
Phase 2: Rhabdomyosarcoma
Participants with rhabdomyosarcoma: nab-paclitaxel at the RP2D (240 mg/m^2 in participants weighing > 10 kg and 11.5 mg/kg in participants weighing ≤ 10 kg) IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity.
Units
Counts
Participants
OG00013
OG00114
OG00214
Title
Denominators
Categories
Title
Measurements
OG00013(7.4 to 16.1)
OG0017.4(4.6 to 8.1)
OG0025.1(2.1 to 7.9)
Phase 2: Rhabdomyosarcoma
Participants with rhabdomyosarcoma: nab-paclitaxel at the RP2D (240 mg/m^2 in participants weighing > 10 kg and 11.5 mg/kg in participants weighing ≤ 10 kg) IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity.
Units
Counts
Participants
OG00013
OG00114
OG00214
Title
Denominators
Categories
Title
Measurements
OG00048(14 to 76)
OG00125(4 to 54)
OG00215(2 to 39)
OG001
Phase 2: Neuroblastoma
Participants with neuroblastoma: nab-paclitaxel at the RP2D (240 mg/m^2 in participants weighing > 10 kg and 11.5 mg/kg in participants weighing ≤ 10 kg) IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity.
OG002
Phase 2: Rhabdomyosarcoma
Participants with rhabdomyosarcoma: nab-paclitaxel at the RP2D (240 mg/m^2 in participants weighing > 10 kg and 11.5 mg/kg in participants weighing ≤ 10 kg) IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity.