Phase 3 Study of Pexidartinib for Pigmented Villonodular... | NCT02371369 | Trialant
NCT02371369
Sponsor
Daiichi Sankyo
Status
Completed
Last Update Posted
May 11, 2022Actual
Enrollment
120Actual
Phase
Phase 3
Conditions
Pigmented Villonodular Synovitis
Giant Cell Tumors of the Tendon Sheath
Tenosynovial Giant Cell Tumor
Interventions
Pexidartinib
Placebo
Countries
United States
Australia
Canada
Denmark
France
Germany
Hungary
Italy
Netherlands
Poland
Spain
United Kingdom
Protocol Section
Identification Module
NCT ID
NCT02371369
Obsolete or Duplicate NCT IDs
Not provided
Organization Study
PLX108-10
Secondary IDs
ID
Type
Description
Link
2014-000148-14
EudraCT Number
Brief Title
Phase 3 Study of Pexidartinib for Pigmented Villonodular Synovitis (PVNS) or Giant Cell Tumor of the Tendon Sheath (GCT-TS)
Official Title
A Double-blind, Randomized, Placebo-controlled Phase 3 Study of Orally Administered PLX3397 in Subjects With Pigmented Villonodular Synovitis or Giant Cell Tumor of the Tendon Sheath
Acronym
ENLIVEN
Organization
Daiichi SankyoINDUSTRY
Status Module
Record Verification Date
Apr 2022
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
May 11, 2015Actual
Primary Completion Date
Mar 27, 2017Actual
Completion Date
Apr 30, 2021Actual
First Submitted Date
Feb 19, 2015
First Submission Date that Met QC Criteria
Feb 19, 2015
First Posted Date
Feb 25, 2015Estimated
Results Waived
Not provided
Results First Submitted Date
Aug 26, 2019
Results First Submitted that Met QC Criteria
Jan 6, 2020
Results First Posted Date
Jan 10, 2020Actual
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
Apr 11, 2022
Last Update Posted Date
May 11, 2022Actual
Sponsor/Collaborators Module
Responsible Party, by Official Title
Sponsor
Lead Sponsor
Daiichi SankyoINDUSTRY
Collaborators
Not provided
Oversight Module
Has Data Monitoring Committee (DMC)
Yes
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
This is a Phase 3 clinical study, which aims to evaluate the effectiveness of an investigational drug called pexidartinib for the treatment of certain tumors for which surgical removal could cause more harm than good.
The main purpose of this study is to gather information about the investigational drug pexidartinib, which may help to treat tumors of pigmented villonodular synovitis (PVNS) or giant cell tumor of the tendon sheath (GCT-TS).
The study consists of two parts with a follow-up period. In Part 1, eligible study participants will be assigned to receive either pexidartinib or matching placebo for 24 weeks. A number of assessments will be carried out during the course of the study, including physical examinations, blood tests, imaging studies, electrocardiograms, and questionnaires. MRI scans will be used to evaluate the response of the tumors to the treatment. Some subjects, assigned to placebo in Part 1 transitioned to pexidartinib for Part 2.
Then a protocol amendment was written to allow only pexidartinib patients to continue into Part 2. Part 2 is a long-term treatment phase in which all participants receive open-label pexidartinib. There was also a follow-up period added to Part 2.
Participants received blinded treatment of pexidartinib,1000 mg (5 capsules per day ) for 2 weeks, then 800 mg (4 capsules per day) for 22 weeks
Drug: Pexidartinib
Part 1 - Placebo
Placebo Comparator
Participants received blinded treatment of matching placebo (5 capsules per day) for 2 weeks, then matching placebo (4 capsules per day) for 22 weeks
Drug: Placebo
Part 2 - All Pexidartinib
Experimental
Participants received pexidartinib in Part 1 and in Part 2 at their prescribed dose
Drug: Pexidartinib
Part 2 - Placebo-Pexidartinib
Experimental
Participants received placebo in Part 1 and pexidartinib in Part 2 at their prescribed dose
Drug: Pexidartinib
Drug: Placebo
Interventions
Name
Type
Description
Arm Group Labels
Other Names
Pexidartinib
Drug
Each capsule contains 200 mg of pexidartinib for oral administration
Part 1 - Pexidartinib
Part 2 - All Pexidartinib
Part 2 - Placebo-Pexidartinib
Outcomes Module
Primary Outcomes
Measure
Description
Time Frame
Percentage of Participants With Symptomatic, Locally Advanced Tenosynovial Giant Cell Tumor (TGCT) Achieving Complete or Partial Response to Pexidartinib Compared With That of Placebo Per Response Evaluation Criteria in Solid Tumors Version 1.1 at Week 25
Complete response (CR) and partial responses (PR) were assessed based on centrally-read magnetic resonance imaging (MRI) scans and Response Evaluation Criteria in Solid Tumors, version 1.1 (RECIST 1.1). A CR was defined as disappearance of all tumors and a PR was defined as at least a 30% decrease in the sum of diameters of target tumors using the baseline sum diameters as the reference.
Week 25
Secondary Outcomes
Measure
Description
Time Frame
Mean Change From Baseline For Range of Motion (ROM) Score in Participants Receiving Pexidartinib Compared With Those on Placebo Up to Week 25
Range of motion (ROM) of the joint was assessed by a qualified, independent, and blinded or third-party assessors at the clinical site. Measurements were recorded in degrees. At baseline, the plane of movement with the smallest relative value (worst) was identified and this plane was used for evaluating the relative change of motion subsequently. Only the plane with the worst impaired ROM at baseline was selected for subsequent analyses.
Other Outcomes
Measure
Description
Time Frame
Percentage of Participants With Symptomatic, Locally Advanced Tenosynovial Giant Cell Tumor (TGCT) Achieving Complete or Partial Response to Pexidartinib Compared With That of Placebo Per Response Evaluation Criteria in Solid Tumors Version 1.1 by Week 49
Complete (CR) and partial responses (PR) were assessed based on centrally-read magnetic resonance imaging (MRI) scans and Response Evaluation Criteria in Solid Tumors, version 1.1 (RECIST 1.1). A CR was defined as disappearance of all tumors and a PR was defined as at least a 30% decrease in the sum of diameters of target tumors using the baseline sum diameters as the reference.
Eligibility Module
Eligibility Criteria
Inclusion Criteria
Age ≥ 18 years.
A diagnosis of PVNS or GCT-TS (i) that has been histologically confirmed either by a pathologist at the treating institution or a central pathologist, and (ii) where surgical resection would be associated with potentially worsening functional limitation or severe morbidity (locally advanced disease), with morbidity determined consensually by qualified personnel (eg, two surgeons or a multi-disciplinary tumor board).
Measurable disease of at least 2 cm and otherwise based on RECIST 1.1, assessed from MRI scans by a central radiologist.
Symptomatic disease because of active PVNS or GCT-TS, defined as one or more of the following:
a worst pain of at least 4 at any time during the week preceding the Screening Visit (based on scale of 0 to 10, with 10 representing "pain as bad as you can imagine").
a worst stiffness of at least 4 at any time during the week preceding the Screening Visit (based on a scale of 0 to 10, with 10 representing "stiffness as bad as you can imagine").
Stable prescription of analgesic regimen during the 2 weeks prior to randomization.
During the 2 weeks prior to randomization, at least 4 of 7 consecutive days of Brief Pain Inventory (BPI) Worst Pain Numeric Rating Scale (NRS) items and Worst Stiffness NRS items completed correctly.
Women of childbearing potential must have a negative serum pregnancy test within the 14-day period prior to randomization. (Where demanded by local regulations, this test may be required within 72 hours of randomization.)
Males and females of childbearing potential are permitted in the study so long as they consent to avoid getting their partner pregnant or becoming pregnant, respectively, by using a highly effective contraception method, as described below, throughout the study and for up to 90 days after completion. Highly effective methods of contraception include: intra-uterine device (non-hormonal or hormonal), bilateral tubal occlusion, vasectomy, sexual abstinence, or barrier methods (eg, condom, diaphragm) used in combination with hormonal methods associated with inhibition of ovulation. Women of non-childbearing potential may be included if they are either surgically sterile or have been postmenopausal for ≥ 1 year. Women who have documentation of at least 12 months of spontaneous amenorrhea and have a follicle stimulating hormone (FSH) level > 40 milli-International units (mIU/mL) will be considered postmenopausal.
Adequate hematologic, hepatic, and renal function, defined by:
Absolute neutrophil count ≥ 1.5 × 10^9/L
aspartate aminotransferase/alanine (AST/ALT) ≤ 1.5 × upper limit of normal (ULN)
Hemoglobin > 10 g/dL
Total bilirubin ≤ 1.5 × ULN
Platelet count ≥ 100 × 10^9/L
Serum creatinine ≤ 1.5 × ULN
Willingness and ability to complete the Worst Pain NRS item, Worst Stiffness NRS item, Patient-reported Outcomes Measurement Information System (PROMIS) Physical Function Scale, and other self-assessment instruments throughout the study.
Willingness and ability to use an electronic diary.
Willingness and ability to provide written informed consent prior to any study-related procedures and to comply with all study requirements.
Exclusion Criteria
Investigational drug use within 28 days of randomization.
Previous use of pexidartinib or any biologic treatment targeting CSF-1 or the CSF-1R; previous use of oral tyrosine kinase inhibitors, eg, imatinib or nilotinib, are allowed.
Active cancer (either concurrent or within the last year of starting study treatment) that requires therapy (eg, surgical, chemotherapy, or radiation therapy), with the exception of adequately treated basal or squamous cell carcinoma of the skin, melanoma in-situ, carcinoma in-situ of the cervix or breast, or prostate carcinoma with a prostate-specific antigen value <0.2 ng/mL.
Known metastatic PVNS/GCT-TS.
Active or chronic infection with hepatitis C virus (HCV) or hepatitis B virus or known active or chronic infection with human immunodeficiency virus.
Known active tuberculosis.
Significant concomitant arthropathy in the affected joint, serious illness, uncontrolled infection, or a medical or psychiatric history that, in the Investigator's opinion, would likely interfere with the person's study participation or the interpretation of his or her results.
Women who are breastfeeding.
A screening Fridericia corrected QT interval (QTcF) ≥ 450 ms (men) or ≥ 470 ms (women).
MRI contraindications.
History of hypersensitivity to any excipients in the investigational product.
Tap W. Pexidartinib in patients with tenosynovial giant cell tumor: a plain language summary of the final results from ENLIVEN. Future Oncol. 2026 May;22(12):1347-1357. doi: 10.1080/14796694.2026.2663959. Epub 2026 May 14.
Healey JH, Tap WD, Gelhorn HL, Ye X, Speck RM, Palmerini E, Stacchiotti S, Desai J, Wagner AJ, Alcindor T, Ganjoo K, Martin-Broto J, Wang Q, Shuster D, Gelderblom H, van de Sande M. Pexidartinib Provides Modest Pain Relief in Patients With Tenosynovial Giant Cell Tumor: Results From ENLIVEN. Clin Orthop Relat Res. 2023 Jan 1;481(1):107-116. doi: 10.1097/CORR.0000000000002335. Epub 2022 Aug 24.
See Also Links
Not provided
Available IPD Information
Not provided
IPD Sharing Statement Module
Plan to Share IPD
Yes
Description
De-identified individual participant data (IPD) and applicable supporting clinical trial documents may be available upon request at https://vivli.org/. In cases where clinical trial data and supporting documents are provided pursuant to our company policies and procedures, Daiichi Sankyo will continue to protect the privacy of our clinical trial participants. Details on data sharing criteria and the procedure for requesting access can be found at this web address: https://vivli.org/ourmember/daiichi-sankyo/
Types
Study Protocol
Statistical Analysis Plan (SAP)
Clinical Study Report (CSR)
Time Frame
Studies for which the medicine and indication have received European Union (EU) and United States (US), and/or Japan (JP) marketing approval on or after 01 January 2014 or by the US or EU or JP Health Authorities when regulatory submissions in all regions are not planned and after the primary study results have been accepted for publication.
Access Criteria
Formal request from qualified scientific and medical researchers on IPD and clinical study documents from clinical trials supporting products submitted and licensed in the United States, the European Union and/or Japan from 01 January 2014 and beyond for the purpose of conducting legitimate research. This must be consistent with the principle of safeguarding study participants' privacy and consistent with provision of informed consent.
Participants were screened for inclusion and exclusion criteria. Screening procedures were performed after consent was obtained and within the 42 days before the first dose of study drug, unless otherwise noted.
Recruitment Details
Part 1 was a double-blind, randomized, Pexidartinib or placebo in participants with symptomatic TGCT for whom surgical resection would be associated with potentially worsening functional limitation or severe morbidity. Part 2 is a long-term treatment phase in which all eligible participants received open-label Pexidartinib.
Type of Units Analyzed
Not provided
Arm/Group Information
ID
Title
Description
FG000
Pexidartinib Part 1, Then Pexidartinib Part 2
Participants received Pexidartinib,1000 mg (5 capsules per day ) for 2 weeks, then 800 mg (4 capsules per day) for 22 weeks in Part 1 of study. Eligible participants from this group also received Pexidartinib in Part 2 at their prescribed dose.
Pexidartinib: Each capsule contains 200 mg of pexidartinib for oral administration.
Periods
Title
Milestones
Reasons Not Completed
Part 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
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_SAP
Yes
Yes
No
Study Protocol and Statistical Analysis Plan
Jan 22, 2020
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
Randomized
Intervention Model
Crossover Assignment
Intervention Model Description
Not provided
Primary Purpose
Treatment
Observational Model
Not provided
Time Perspective
Not provided
Masking Info
Masking
Quadruple
Masking Description
Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor) in Part 1, Open-label (no masking) in Part 2
Placebo capsule matching pexidartinib capsule for oral administration
Part 1 - Placebo
Part 2 - Placebo-Pexidartinib
Placebo Capsule
Baseline, Week 13, and Week 25
Percentage of Participants With Symptomatic, Locally Advanced Tenosynovial Giant Cell Tumor (TGCT) Achieving Complete or Partial Response Based on Tumor Volume Score (TVS) After Receiving Pexidartinib Compared With Those on Placebo at Week 25
Complete response (CR) and partial response (PR) were assessed using tumor volume score (TVS). A CR was defined as disappearance of all tumors and a PR was defined as at least a 30% decrease in the sum of diameters of target tumors using the baseline sum diameters as the reference. TVS is a semi-quantitative MRI scoring system that describes tumor mass and is based on 10% increments of the estimated volume of the maximally distended synovial cavity or tendon sheath involved. A tumor that is equal in volume to that of a maximally distended synovial cavity or tendon sheath was scored 10; a score of 0 indicated no evidence of tumor.
Week 25
Mean Change From Baseline in the Patient-reported Outcomes Measurement Information System (PROMIS) Physical Function Score in Participants Receiving Pexidartinib Compared With Those on Placebo Up to Week 25
The Patient-reported Outcomes Measurement Information System (PROMIS) physical function scale was used to assess physical function of the upper and lower limbs. The scale ranged from 1 defined as 'unable to do' or 'cannot do' to 5 defined as 'without any difficulty' or 'not at all', where higher scores represent better outcomes.
at Week 9 , Week 17, and Week 25
Mean Change From Baseline for Worst Stiffness Numeric Rating Scale Score (NRS) in Participants Receiving Pexidartinib Compared With Those on Placebo Up to Week 25
The Worst Stiffness Numeric Rating Scale (NRS) was a 1-item, self-administered questionnaire assessing the "worst" stiffness in the last 24 hours. The NRS for this item ranged from 0 (no stiffness) to 10 (stiffness as bad as you can imagine).
Baseline, Week 9, Week 17, and Week 25
Percentage of Participants Who Responded With a Decrease of at Least 30% in the Mean Brief Pain Inventory Worst Pain Numeric Rating Scale Score Among Participants Receiving Pexidartinib Compared With Those on Placebo at Week 25
The Brief Pain Inventory (BPI) Worst Pain Numeric Rating Scale Score (NRS) was a 1-item, self-administered questionnaire assessing the "worst" pain in the last 24 hours. The NRS for this item ranged from 0 (no pain) to 10 (pain as bad as you can imagine).
Week 25
Number of Responders to Pexidartinib With and Without Disease Progression
Duration of response (DOR) based on RECIST 1.1 is defined as the date of the first recorded response to the first date of documented disease progression. The overall number of responses and the number of participants with and without disease progression was assessed.
By Week 96
Number of Responders to Pexidartinib With and Without Disease Progression Based on Tumor Volume Score
Tumor Volume Score (TVS) is a semi-quantitative MRI scoring system that describes tumor mass and is based on 10% increments of the estimated volume of the maximally distended synovial cavity or tendon sheath involved. A tumor that is equal in volume to that of a maximally distended synovial cavity or tendon sheath was scored 10; a score of 0 indicated no evidence of tumor. The overall number of responses and the number of participants with and without disease progression was assessed.
By Week 120
Duration of Response (DOR) Based on RECIST 1.1
Duration of response (DOR) based on RECIST 1.1 is defined from the date of the first recorded evidence of response to the first date of documented disease progression.
Date of first documentation of objective response up to date of first documentation of progressive disease, assessed up to end of study (approximately 71 months)
Duration of Response (DOR) Based on Tumor Volume Score (TVS)
Duration of response (DOR) based on TVS is defined from the date of the first recorded evidence of response to the first date of documented disease progression.
Date of first documentation of objective response up to date of first documentation of progressive disease, assessed up to end of study (approximately 71 months)
Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term
Treatment-emergent adverse events (TEAEs) were defined as adverse events that started or worsened after the first dose of treatment and within 28 days after the last dose. The National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) version 4.0 was used to grade adverse events. Any Grade and Grade ≥3 (severe) TEAEs are reported. TEAEs were coded using MedDRA version 17.1.
After the first dose of treatment up to 28 days after the last dose
By Week 49
Mean Change From Baseline For Range of Motion (ROM) Score in Participants Receiving Pexidartinib Compared With Those on Placebo by Week 49
Range of motion (ROM) of the joint was assessed by a qualified, independent, and blinded or third-party assessors at the clinical site. Measurements were recorded in degrees. At baseline, the plane of movement with the smallest relative value (worst) was identified and this plane was used for evaluating the relative change of motion subsequently. Only the plane with the worst impaired ROM at baseline was selected for subsequent analyses.
By Week 49
Mean Change From Baseline in the Patient-reported Outcomes Measurement Information System (PROMIS) Physical Function Score in Participants Receiving Pexidartinib Compared With Those on Placebo by Week 49
The Patient-reported Outcomes Measurement Information System (PROMIS) physical function scale was used to assess physical function of the upper and lower limbs. The scale ranged from 1 defined as 'unable to do' or 'cannot do' to 5 defined as 'without any difficulty' or 'not at all', where higher scores represent better outcomes.
By Week 49
Mean Change From Baseline for Worst Stiffness Numeric Rating Scale Score (NRS) in Participants Receiving Pexidartinib Compared With Those on Placebo by Week 49
The Worst Stiffness Numeric Rating Scale (NRS) was a 1-item, self-administered questionnaire assessing the "worst" stiffness in the last 24 hours. The NRS for this item ranged from 0 (no stiffness) to 10 (stiffness as bad as you can imagine).
Baseline, Week 25, and Week 49
Mean Change From Baseline for Worst Pain Numeric Rating Scale Score (NRS) in Participants Receiving Pexidartinib Compared With Those on Placebo by Week 49
The Brief Pain Inventory (BPI) Worst Pain NRS was a 1-item, self-administered questionnaire assessing the "worst" pain in the last 24 hours. The NRS for this item ranged from 0 (no pain) to 10 (pain as bad as you can imagine).
By Week 49
Percentage of Participants With Symptomatic, Locally Advanced Tenosynovial Giant Cell Tumor (TGCT) Achieving Complete or Partial Response Based on Tumor Volume Score (TVS) After Receiving Pexidartinib Compared With Those on Placebo by Week 49
Best overall response (CR or PR) was assessed using tumor volume score (TVS) in the ITT population. Tumor Volume Score is a semi-quantitative MRI scoring system that describes tumor mass and is based on 10% increments of the estimated volume of the maximally distended synovial cavity or tendon sheath involved. A tumor that is equal in volume to that of a maximally distended synovial cavity or tendon sheath was scored 10; a score of 0 indicated no evidence of tumor.
By Week 49
Los Angeles
California
90033
United States
Stanford Cancer Center
Palo Alto
California
94305
United States
UCLA Medical Center
Santa Monica
California
90404
United States
Mayo Clinic Cancer Center
Jacksonville
Florida
32224
United States
Sylvester Comprehensive Cancer Center
Miami
Florida
33136
United States
Moffitt Cancer Center
Tampa
Florida
33612
United States
Massachusetts General Hospital
Boston
Massachusetts
02114
United States
: Dana Farber Cancer Institute
Boston
Massachusetts
02115
United States
Michigan Comprehensive Cancer Center
Ann Arbor
Michigan
48109
United States
Mayo Clinic Cancer Center
Rochester
Minnesota
55905
United States
Washington University School of Medicine
St Louis
Missouri
63110
United States
MD Anderson Cancer Center at Cooper
Camden
New Jersey
08103
United States
Memorial Sloan Kettering Cancer Center
New York
New York
10065
United States
Duke Cancer Center
Durham
North Carolina
27710
United States
OHSU Knight Cancer Institute
Portland
Oregon
97239
United States
Vanderbilt-Ingram Cancer Center
Nashville
Tennessee
37232
United States
Huntsman Cancer Institute
Salt Lake City
Utah
84112
United States
Seattle Cancer Care Alliance
Seattle
Washington
98109
United States
Chris O'Brien Lifehouse
Sydney
New South Wales
2050
Australia
Princess Alexandra Hospital
Woolloongabba
Queensland
4102
Australia
Peter MacCallum Cancer Centre
East Melbourne
Victoria
3000
Australia
Princess Margaret Hospital
Toronto
Ontario
M5G2M9
Canada
McGill University Health Centre
Montreal
Quebec
H4A3J1
Canada
Herlev Hospital
Herlev
2730
Denmark
Centre Leon Bérard
Lyon
69373
France
Institut Gustave Roussy
Villejuif
94800
France
HELIOS Klinikum Berlin-Buch
Berlin
13125
Germany
Universitätsklinikum Essen
Essen
45147
Germany
Military Hospital-State Health Center
Budapest
H1134
Hungary
Istituto Ortopedico Rizzoli
Bologna
BO
40136
Italy
Istituto Nazionale Tumori-Fondazione IRCCS
Milan
MI
20133
Italy
Leiden University Medical Center
Leiden
2333 ZA
Netherlands
Radboud Univ. Medical Center
Nijmegen
6525 GA
Netherlands
Centrum Onkologii-Instytut im. Marii Skłodowskiej-Curie
Lewis JH, Gelderblom H, van de Sande M, Stacchiotti S, Healey JH, Tap WD, Wagner AJ, Pousa AL, Druta M, Lin CC, Baba HA, Choi Y, Wang Q, Shuster DE, Bauer S. Pexidartinib Long-Term Hepatic Safety Profile in Patients with Tenosynovial Giant Cell Tumors. Oncologist. 2021 May;26(5):e863-e873. doi: 10.1002/onco.13629. Epub 2020 Dec 24.
Tap WD, Gelderblom H, Palmerini E, Desai J, Bauer S, Blay JY, Alcindor T, Ganjoo K, Martin-Broto J, Ryan CW, Thomas DM, Peterfy C, Healey JH, van de Sande M, Gelhorn HL, Shuster DE, Wang Q, Yver A, Hsu HH, Lin PS, Tong-Starksen S, Stacchiotti S, Wagner AJ; ENLIVEN investigators. Pexidartinib versus placebo for advanced tenosynovial giant cell tumour (ENLIVEN): a randomised phase 3 trial. Lancet. 2019 Aug 10;394(10197):478-487. doi: 10.1016/S0140-6736(19)30764-0. Epub 2019 Jun 19.
Gelhorn HL, Tong S, McQuarrie K, Vernon C, Hanlon J, Maclaine G, Lenderking W, Ye X, Speck RM, Lackman RD, Bukata SV, Healey JH, Keedy VL, Anthony SP, Wagner AJ, Von Hoff DD, Singh AS, Becerra CR, Hsu HH, Lin PS, Tap WD. Patient-reported Symptoms of Tenosynovial Giant Cell Tumors. Clin Ther. 2016 Apr;38(4):778-93. doi: 10.1016/j.clinthera.2016.03.008. Epub 2016 Apr 1.
FG001
Placebo Part 1, Then Pexidartinib Part 2
Participants received treatment of matching placebo (5 capsules per day) for 2 weeks, then matching placebo (4 capsules per day) for 22 weeks in Part 1 of study. Eligible participants from this group also received Pexidartinib in Part 2 at their prescribed dose.
Placebo: Placebo capsule matching Pexidartinib capsule for oral administration. Pexidartinib: Each capsule contains 200 mg of Pexidartinib for oral administration (Part 2).
FG00061 subjects
FG00159 subjects
COMPLETED
FG00052 subjects
FG00148 subjects
NOT COMPLETED
FG0009 subjects
FG00111 subjects
Type
Comment
Reasons
Physician Decision
FG0000 subjects
FG0013 subjects
Adverse Event
FG0008 subjects
FG0010 subjects
Withdrawal by Subject
FG0001 subjects
FG0016 subjects
Disease progression
FG0000 subjects
FG0011 subjects
Protocol Violation
FG0000 subjects
FG0011 subjects
Part 2
Type
Comment
Milestone Data
STARTED
FG00048 subjectsN=48; Only participants who were eligible to receive treatment in Part 2.
FG00130 subjectsN=30; Only participants who were eligible to receive treatment in Part 2.
COMPLETED
FG0000 subjects
FG0010 subjects
NOT COMPLETED
FG00048 subjects
FG00130 subjects
Type
Comment
Reasons
Adverse Event
FG0006 subjects
FG0015 subjects
Withdrawal by Subject
FG00016 subjects
FG001
Type of Units Analyzed
Not provided
Arm/Group Information
ID
Title
Description
BG000
Pexidartinib Part 1, Then Pexidartinib Part 2
Participants received Pexidartinib,1000 mg (5 capsules per day ) for 2 weeks, then 800 mg (4 capsules per day) for 22 weeks in Part 1 of study. Eligible participants from this group also received Pexidartinib in Part 2 at their prescribed dose.
Pexidartinib: Each capsule contains 200 mg of pexidartinib for oral administration
BG001
Placebo Part 1, Then Pexidartinib Part 2
Participants received treatment of matching placebo (5 capsules per day) for 2 weeks, then matching placebo (4 capsules per day) for 22 weeks in Part 1 of study. Eligible participants from this group also received Pexidartinib in Part 2 at their prescribed dose.
Placebo: Placebo capsule matching pexidartinib capsule for oral administration. Pexidartinib: Each capsule contains 200 mg of pexidartinib for oral administration (Part 2).
BG002
Total
Total of all reporting groups
Denominators
Units
Counts
Participants
BG00061
BG00159
BG002120
Baseline Measures
Title
Description
Population Description
Parameter Type
Dispersion Type
Unit of Measure
Calculate Percentage
Denominator Units Selected
Denominators
Classes
Age, Categorical
Count of Participants
Participants
Title
Denominators
Categories
Title
Measurements
<=18 years
BG0000
BG0010
BG0020
Between 18 and 65 years
Age, Continuous
Mean
Standard Deviation
years
Title
Denominators
Categories
Title
Measurements
BG00044.6± 13.2
BG00144.3± 13.6
BG002
Sex: Female, Male
Count of Participants
Participants
Title
Denominators
Categories
Title
Measurements
Female
BG00035
BG00136
BG002
Race (NIH/OMB)
Count of Participants
Participants
Title
Denominators
Categories
Title
Measurements
American Indian or Alaska Native
BG0002
BG0010
BG002
Region of Enrollment
Number
participants
Title
Denominators
Categories
Canada
Title
Measurements
BG0002
BG0013
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
Percentage of Participants With Symptomatic, Locally Advanced Tenosynovial Giant Cell Tumor (TGCT) Achieving Complete or Partial Response to Pexidartinib Compared With That of Placebo Per Response Evaluation Criteria in Solid Tumors Version 1.1 at Week 25
Complete response (CR) and partial responses (PR) were assessed based on centrally-read magnetic resonance imaging (MRI) scans and Response Evaluation Criteria in Solid Tumors, version 1.1 (RECIST 1.1). A CR was defined as disappearance of all tumors and a PR was defined as at least a 30% decrease in the sum of diameters of target tumors using the baseline sum diameters as the reference.
Best overall response was assessed in the Intent-to-Treat (ITT) population.
Posted
Number
Percentage of participants
Week 25
ID
Title
Description
OG000
Pexidartinib Part 1
Participants received treatment of Pexidartinib,1000 mg (5 capsules per day ) for 2 weeks, then 800 mg (4 capsules per day) for 22 weeks Pexidartinib: Each capsule contains 200 mg of Pexidartinib for oral administration.
OG001
Placebo Part 1
Participants received treatment of matching placebo (5 capsules per day) for 2 weeks, then matching placebo (4 capsules per day) for 22 weeks Placebo: Placebo capsule matching Pexidartinib capsule for oral administration.
Units
Counts
Participants
OG00061
OG00159
Title
Denominators
Categories
Complete Response (CR)
Title
Measurements
OG00014.8
OG0010
Partial Response (PR)
Title
Measurements
OG000
Group IDs
Group Description
Statistical Method
Statistical Comment
P-Value
P-Value Comment
Parameter Type
Parameter Value
Dispersion Type
Dispersion Value
Confidence Interval Sides
Confidence Interval %
CI Lower Limit
CI Upper Limit
CI Lower Limit Comment
CI Upper Limit Comment
Estimate Comment
Tested Non-Inferiority
Non-Inferiority Type
Non-Inferiority Comment
Other Analysis Description
OG000
OG001
Treatment comparison between the pexidartinib and placebo groups at Week 25
Fisher's Exact Test
<0.0001
Other
Treatment comparison analysis
Secondary
Mean Change From Baseline For Range of Motion (ROM) Score in Participants Receiving Pexidartinib Compared With Those on Placebo Up to Week 25
Range of motion (ROM) of the joint was assessed by a qualified, independent, and blinded or third-party assessors at the clinical site. Measurements were recorded in degrees. At baseline, the plane of movement with the smallest relative value (worst) was identified and this plane was used for evaluating the relative change of motion subsequently. Only the plane with the worst impaired ROM at baseline was selected for subsequent analyses.
Range of motion was assessed in the ITT population in participants where data were available.
Posted
Least Squares Mean
Standard Error
degrees
Baseline, Week 13, and Week 25
ID
Title
Description
OG000
Pexidartinib Part 1
Participants received treatment of Pexidartinib,1000 mg (5 capsules per day ) for 2 weeks, then 800 mg (4 capsules per day) for 22 weeks Pexidartinib: Each capsule contains 200 mg of Pexidartinib for oral administration.
OG001
Placebo Part 1
Participants received treatment of matching placebo (5 capsules per day) for 2 weeks, then matching placebo (4 capsules per day) for 22 weeks Placebo: Placebo capsule matching Pexidartinib capsule for oral administration.
Secondary
Percentage of Participants With Symptomatic, Locally Advanced Tenosynovial Giant Cell Tumor (TGCT) Achieving Complete or Partial Response Based on Tumor Volume Score (TVS) After Receiving Pexidartinib Compared With Those on Placebo at Week 25
Complete response (CR) and partial response (PR) were assessed using tumor volume score (TVS). A CR was defined as disappearance of all tumors and a PR was defined as at least a 30% decrease in the sum of diameters of target tumors using the baseline sum diameters as the reference. TVS is a semi-quantitative MRI scoring system that describes tumor mass and is based on 10% increments of the estimated volume of the maximally distended synovial cavity or tendon sheath involved. A tumor that is equal in volume to that of a maximally distended synovial cavity or tendon sheath was scored 10; a score of 0 indicated no evidence of tumor.
Best overall response was assessed in the ITT population.
Posted
Number
Percentage of participants
Week 25
ID
Title
Description
OG000
Pexidartinib Part 1
Participants received treatment of Pexidartinib,1000 mg (5 capsules per day ) for 2 weeks, then 800 mg (4 capsules per day) for 22 weeks Pexidartinib: Each capsule contains 200 mg of Pexidartinib for oral administration.
OG001
Placebo Part 1
Participants received treatment of matching placebo (5 capsules per day) for 2 weeks, then matching placebo (4 capsules per day) for 22 weeks Placebo: Placebo capsule matching Pexidartinib capsule for oral administration.
Secondary
Mean Change From Baseline in the Patient-reported Outcomes Measurement Information System (PROMIS) Physical Function Score in Participants Receiving Pexidartinib Compared With Those on Placebo Up to Week 25
The Patient-reported Outcomes Measurement Information System (PROMIS) physical function scale was used to assess physical function of the upper and lower limbs. The scale ranged from 1 defined as 'unable to do' or 'cannot do' to 5 defined as 'without any difficulty' or 'not at all', where higher scores represent better outcomes.
Physical function was assessed in the ITT population in participants where data were available.
Posted
Least Squares Mean
Standard Error
units on a scale
at Week 9 , Week 17, and Week 25
ID
Title
Description
OG000
Pexidartinib Part 1
Participants received treatment of Pexidartinib,1000 mg (5 capsules per day ) for 2 weeks, then 800 mg (4 capsules per day) for 22 weeks Pexidartinib: Each capsule contains 200 mg of Pexidartinib for oral administration.
OG001
Placebo Part 1
Participants received treatment of matching placebo (5 capsules per day) for 2 weeks, then matching placebo (4 capsules per day) for 22 weeks Placebo: Placebo capsule matching Pexidartinib capsule for oral administration.
Secondary
Mean Change From Baseline for Worst Stiffness Numeric Rating Scale Score (NRS) in Participants Receiving Pexidartinib Compared With Those on Placebo Up to Week 25
The Worst Stiffness Numeric Rating Scale (NRS) was a 1-item, self-administered questionnaire assessing the "worst" stiffness in the last 24 hours. The NRS for this item ranged from 0 (no stiffness) to 10 (stiffness as bad as you can imagine).
Worst stiffness was assessed in the ITT population.
Posted
Least Squares Mean
Standard Error
units on a scale
Baseline, Week 9, Week 17, and Week 25
ID
Title
Description
OG000
Pexidartinib Part 1
Participants received treatment of Pexidartinib,1000 mg (5 capsules per day ) for 2 weeks, then 800 mg (4 capsules per day) for 22 weeks Pexidartinib: Each capsule contains 200 mg of Pexidartinib for oral administration.
OG001
Placebo Part 1
Participants received treatment of matching placebo (5 capsules per day) for 2 weeks, then matching placebo (4 capsules per day) for 22 weeks Placebo: Placebo capsule matching Pexidartinib capsule for oral administration.
Units
Counts
Participants
Secondary
Percentage of Participants Who Responded With a Decrease of at Least 30% in the Mean Brief Pain Inventory Worst Pain Numeric Rating Scale Score Among Participants Receiving Pexidartinib Compared With Those on Placebo at Week 25
The Brief Pain Inventory (BPI) Worst Pain Numeric Rating Scale Score (NRS) was a 1-item, self-administered questionnaire assessing the "worst" pain in the last 24 hours. The NRS for this item ranged from 0 (no pain) to 10 (pain as bad as you can imagine).
Worst pain was assessed in the ITT population.
Posted
Number
percentage of participants
Week 25
ID
Title
Description
OG000
Pexidartinib Part 1
Participants received treatment of Pexidartinib,1000 mg (5 capsules per day ) for 2 weeks, then 800 mg (4 capsules per day) for 22 weeks
Pexidartinib: Each capsule contains 200 mg of Pexidartinib for oral administration.
OG001
Placebo Part 1
Participants received treatment of matching placebo (5 capsules per day) for 2 weeks, then matching placebo (4 capsules per day) for 22 weeks
Placebo: Placebo capsule matching Pexidartinib capsule for oral administration.
Units
Counts
Participants
Secondary
Number of Responders to Pexidartinib With and Without Disease Progression
Duration of response (DOR) based on RECIST 1.1 is defined as the date of the first recorded response to the first date of documented disease progression. The overall number of responses and the number of participants with and without disease progression was assessed.
The overall number of responses and the number of participants with and without disease progression were assessed in the ITT population, specifically among the Pexidartinib Part 1, Pexidartinib Part 2; Pexidartinib Part 2 only, All Pexidartinib Treated participants, . Participants randomized to Placebo Part 1 only were not analyzed.
Posted
Number
participants
By Week 96
ID
Title
Description
OG000
Pexidartinib Part 1 and Part 2
Part 1: Participants received treatment of pexidartinib,1000 mg (5 capsules per day ) for 2 weeks, then 800 mg (4 capsules per day) for 22 weeks
Part 2: Participants also received pexidartinib at their prescribed dose.
Pexidartinib: Each capsule contains 200 mg of pexidartinib for oral administration
OG001
Placebo Part 1, Pexidartinib Part 2
Participants that received placebo in part 1 and received Pexidartinib in Part 2 at their prescribed dose.
Pexidartinib: Each capsule contains 200 mg of pexidartinib for oral administration
OG002
Secondary
Number of Responders to Pexidartinib With and Without Disease Progression Based on Tumor Volume Score
Tumor Volume Score (TVS) is a semi-quantitative MRI scoring system that describes tumor mass and is based on 10% increments of the estimated volume of the maximally distended synovial cavity or tendon sheath involved. A tumor that is equal in volume to that of a maximally distended synovial cavity or tendon sheath was scored 10; a score of 0 indicated no evidence of tumor. The overall number of responses and the number of participants with and without disease progression was assessed.
The overall number of responses and the number of participants with and without disease progression was assessed in the ITT population, specifically among the Pexidartinib Part 1, Pexidartinib Part 2; Pexidartinib Part 2 only; and All Pexidartinib Treated participants. Participants who received Placebo Part 1 only were not analyzed.
Posted
Number
participants
By Week 120
ID
Title
Description
OG000
Pexidartinib in Part 1 and Part 2
Participants received treatment of Pexidartinib,1000 mg (5 capsules per day ) for 2 weeks, then 800 mg (4 capsules per day) for 22 weeks in Part 1 and also received Pexidartinib in Part 2 at their prescribed dose.
Pexidartinib: Each capsule contains 200 mg of pexidartinib for oral administration
OG001
Placebo Part 1, Pexidartinib Part 2
Participants received treatment of matching placebo (5 capsules per day) for 2 weeks, then matching placebo (4 capsules per day) for 22 weeks in Part 1 and also received Pexidartinib in Part 2 at their prescribed dose.
Placebo: Placebo capsule matching Pexidartinib capsule for oral administration Pexidartinib: Each capsule contains 200 mg of Pexidartinib for oral administration.
Secondary
Duration of Response (DOR) Based on RECIST 1.1
Duration of response (DOR) based on RECIST 1.1 is defined from the date of the first recorded evidence of response to the first date of documented disease progression.
DOR was assessed in the ITT population, specifically among the Pexidartinib Part 1, Pexidartinib Part 2; Pexidartinib Part 2 only. Participants randomized to Placebo Part 1 only were not analyzed. DOR was based on numbers of responders only.
Posted
Median
95% Confidence Interval
months
Date of first documentation of objective response up to date of first documentation of progressive disease, assessed up to end of study (approximately 71 months)
ID
Title
Description
OG000
Pexidartinib Part 1 and Part 2
Part 1: Participants received treatment of pexidartinib,1000 mg (5 capsules per day ) for 2 weeks, then 800 mg (4 capsules per day) for 22 weeks Part 2: Participants also received pexidartinib at their prescribed dose.
Pexidartinib: Each capsule contains 200 mg of pexidartinib for oral administration
OG001
Placebo Part 1, Pexidartinib Part 2
Participants that received placebo in part 1 and received Pexidartinib in Part 2 at their prescribed dose.
Pexidartinib: Each capsule contains 200 mg of pexidartinib for oral administration
Secondary
Duration of Response (DOR) Based on Tumor Volume Score (TVS)
Duration of response (DOR) based on TVS is defined from the date of the first recorded evidence of response to the first date of documented disease progression.
DOR was assessed in the ITT population, specifically among the Pexidartinib Part 1, Pexidartinib Part 2; Pexidartinib Part 2 only. Participants randomized to Placebo Part 1 only were not analyzed. DOR was based on numbers of responders only.
Posted
Median
95% Confidence Interval
months
Date of first documentation of objective response up to date of first documentation of progressive disease, assessed up to end of study (approximately 71 months)
ID
Title
Description
OG000
Pexidartinib Part 1 and Part 2
Part 1: Participants received treatment of pexidartinib,1000 mg (5 capsules per day ) for 2 weeks, then 800 mg (4 capsules per day) for 22 weeks Part 2: Participants also received pexidartinib at their prescribed dose.
Pexidartinib: Each capsule contains 200 mg of pexidartinib for oral administration
OG001
Placebo Part 1, Pexidartinib Part 2
Participants that received placebo in part 1 and received Pexidartinib in Part 2 at their prescribed dose.
Pexidartinib: Each capsule contains 200 mg of pexidartinib for oral administration
Secondary
Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term
Treatment-emergent adverse events (TEAEs) were defined as adverse events that started or worsened after the first dose of treatment and within 28 days after the last dose. The National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) version 4.0 was used to grade adverse events. Any Grade and Grade ≥3 (severe) TEAEs are reported. TEAEs were coded using MedDRA version 17.1.
All safety events were assessed in the Safety Analysis Set.
Posted
Number
Percentage of participants
After the first dose of treatment up to 28 days after the last dose
ID
Title
Description
OG000
Pexidartinib Part 1
Participants received treatment of Pexidartinib,1000 mg (5 capsules per day ) for 2 weeks, then 800 mg (4 capsules per day) for 22 weeks Pexidartinib: Each capsule contains 200 mg of Pexidartinib for oral administration.
OG001
Placebo Part 1
Participants received treatment of matching placebo (5 capsules per day) for 2 weeks, then matching placebo (4 capsules per day) for 22 weeks Placebo: Placebo capsule matching Pexidartinib capsule for oral administration.
OG002
Pexidartinib Part 1 and Part 2
Other Pre-specified
Percentage of Participants With Symptomatic, Locally Advanced Tenosynovial Giant Cell Tumor (TGCT) Achieving Complete or Partial Response to Pexidartinib Compared With That of Placebo Per Response Evaluation Criteria in Solid Tumors Version 1.1 by Week 49
Complete (CR) and partial responses (PR) were assessed based on centrally-read magnetic resonance imaging (MRI) scans and Response Evaluation Criteria in Solid Tumors, version 1.1 (RECIST 1.1). A CR was defined as disappearance of all tumors and a PR was defined as at least a 30% decrease in the sum of diameters of target tumors using the baseline sum diameters as the reference.
Best overall response was assessed in the ITT population.
Posted
Number
Percentage of participants
By Week 49
ID
Title
Description
OG000
Pexidartinib Part 1 and Part 2
Participants received treatment of Pexidartinib,1000 mg (5 capsules per day) for 2 weeks, then 800 mg (4 capsules per day) for 22 weeks and also received Pexidartinib in Part 2 at their prescribed dose.
Pexidartinib: Each capsule contains 200 mg of Pexidartinib for oral administration.
OG001
Placebo Part 1, Pexidartinib Part 2
Participants received treatment of matching placebo (5 capsules per day) for 2 weeks, then matching placebo (4 capsules per day) for 22 weeks in Part 1 and also received Pexidartinib in Part 2 at their prescribed dose.
Placebo: Placebo capsule matching Pexidartinib capsule for oral administration Pexidartinib: Each capsule contains 200 mg of Pexidartinib for oral administration.
Other Pre-specified
Mean Change From Baseline For Range of Motion (ROM) Score in Participants Receiving Pexidartinib Compared With Those on Placebo by Week 49
Range of motion (ROM) of the joint was assessed by a qualified, independent, and blinded or third-party assessors at the clinical site. Measurements were recorded in degrees. At baseline, the plane of movement with the smallest relative value (worst) was identified and this plane was used for evaluating the relative change of motion subsequently. Only the plane with the worst impaired ROM at baseline was selected for subsequent analyses.
Range of Motion (ROM) was assessed in the ITT population.
Posted
Mean
Standard Deviation
degrees
By Week 49
ID
Title
Description
OG000
Pexidartinib Part 1 and Part 2
Participants received treatment of Pexidartinib,1000 mg (5 capsules per day) for 2 weeks, then 800 mg (4 capsules per day) for 22 weeks and also received Pexidartinib in Part 2 at their prescribed dose.
Pexidartinib: Each capsule contains 200 mg of Pexidartinib for oral administration.
OG001
Placebo Part 1, Pexidartinib Part 2
Participants received treatment of matching placebo (5 capsules per day) for 2 weeks, then matching placebo (4 capsules per day) for 22 weeks in Part 1 and also received Pexidartinib in Part 2 at their prescribed dose.
Placebo: Placebo capsule matching Pexidartinib capsule for oral administration Pexidartinib: Each capsule contains 200 mg of Pexidartinib for oral administration.
Other Pre-specified
Mean Change From Baseline in the Patient-reported Outcomes Measurement Information System (PROMIS) Physical Function Score in Participants Receiving Pexidartinib Compared With Those on Placebo by Week 49
The Patient-reported Outcomes Measurement Information System (PROMIS) physical function scale was used to assess physical function of the upper and lower limbs. The scale ranged from 1 defined as 'unable to do' or 'cannot do' to 5 defined as 'without any difficulty' or 'not at all', where higher scores represent better outcomes.
Physical function was assessed in the ITT population.
Posted
Mean
Standard Deviation
units on a scale
By Week 49
ID
Title
Description
OG000
Pexidartinib Part 1 and Part 2
Participants received treatment of Pexidartinib,1000 mg (5 capsules per day) for 2 weeks, then 800 mg (4 capsules per day) for 22 weeks and also received Pexidartinib in Part 2 at their prescribed dose.
Pexidartinib: Each capsule contains 200 mg of Pexidartinib for oral administration.
OG001
Placebo Part 1, Pexidartinib Part 2
Participants received treatment of matching placebo (5 capsules per day) for 2 weeks, then matching placebo (4 capsules per day) for 22 weeks in Part 1 and also received Pexidartinib in Part 2 at their prescribed dose.
Placebo: Placebo capsule matching Pexidartinib capsule for oral administration Pexidartinib: Each capsule contains 200 mg of Pexidartinib for oral administration.
Other Pre-specified
Mean Change From Baseline for Worst Stiffness Numeric Rating Scale Score (NRS) in Participants Receiving Pexidartinib Compared With Those on Placebo by Week 49
The Worst Stiffness Numeric Rating Scale (NRS) was a 1-item, self-administered questionnaire assessing the "worst" stiffness in the last 24 hours. The NRS for this item ranged from 0 (no stiffness) to 10 (stiffness as bad as you can imagine).
Worst stiffness was assessed in the ITT population, specifically among the Pexidartinib Part 1, Pexidartinib Part 2; Placebo Part 1, Pexidartinib Part 2; and All Pexidartinib Treated participants. Participants who received Placebo Part 1 only or Pexidartinib Part 2 only were not analyzed.
Posted
Mean
Standard Deviation
units on a scale
Baseline, Week 25, and Week 49
ID
Title
Description
OG000
Pexidartinib Part 1 and Part 2
Participants received treatment of Pexidartinib,1000 mg (5 capsules per day) for 2 weeks, then 800 mg (4 capsules per day) for 22 weeks and also received Pexidartinib in Part 2 at their prescribed dose.
Pexidartinib: Each capsule contains 200 mg of Pexidartinib for oral administration.
OG001
Placebo Part 1, Pexidartinib Part 2
Participants received treatment of matching placebo (5 capsules per day) for 2 weeks, then matching placebo (4 capsules per day) for 22 weeks in Part 1 and also received Pexidartinib in Part 2 at their prescribed dose.
Placebo: Placebo capsule matching Pexidartinib capsule for oral administration Pexidartinib: Each capsule contains 200 mg of Pexidartinib for oral administration.
Other Pre-specified
Mean Change From Baseline for Worst Pain Numeric Rating Scale Score (NRS) in Participants Receiving Pexidartinib Compared With Those on Placebo by Week 49
The Brief Pain Inventory (BPI) Worst Pain NRS was a 1-item, self-administered questionnaire assessing the "worst" pain in the last 24 hours. The NRS for this item ranged from 0 (no pain) to 10 (pain as bad as you can imagine).
Worst pain was assessed in the ITT population, specifically among the Pexidartinib Part 1, Pexidartinib Part 2; Placebo Part 1, Pexidartinib Part 2; and All Pexidartinib Treated participants. Participants who received Placebo Part 1 only or Pexidartinib Part 2 only were not analyzed.
Posted
Mean
Standard Deviation
units on a scale
By Week 49
ID
Title
Description
OG000
Pexidartinib Part 1 and Part 2
Participants received treatment of Pexidartinib,1000 mg (5 capsules per day) for 2 weeks, then 800 mg (4 capsules per day) for 22 weeks and also received Pexidartinib in Part 2 at their prescribed dose.
Pexidartinib: Each capsule contains 200 mg of Pexidartinib for oral administration.
OG001
Placebo Part 1, Pexidartinib Part 2
Participants received treatment of matching placebo (5 capsules per day) for 2 weeks, then matching placebo (4 capsules per day) for 22 weeks in Part 1 and also received Pexidartinib in Part 2 at their prescribed dose.
Placebo: Placebo capsule matching Pexidartinib capsule for oral administration Pexidartinib: Each capsule contains 200 mg of Pexidartinib for oral administration.
Other Pre-specified
Percentage of Participants With Symptomatic, Locally Advanced Tenosynovial Giant Cell Tumor (TGCT) Achieving Complete or Partial Response Based on Tumor Volume Score (TVS) After Receiving Pexidartinib Compared With Those on Placebo by Week 49
Best overall response (CR or PR) was assessed using tumor volume score (TVS) in the ITT population. Tumor Volume Score is a semi-quantitative MRI scoring system that describes tumor mass and is based on 10% increments of the estimated volume of the maximally distended synovial cavity or tendon sheath involved. A tumor that is equal in volume to that of a maximally distended synovial cavity or tendon sheath was scored 10; a score of 0 indicated no evidence of tumor.
Best overall response on Tumor Volume Score was assessed in the ITT population, specifically among the Pexidartinib Part 1, Pexidartinib Part 2; Placebo Part 1, Pexidartinib Part 2; and All Pexidartinib Treated participants. Participants who received Placebo Part 1 only or Pexidartinib Part 2 only were not analyzed..
Posted
Number
Percentage of participants
By Week 49
ID
Title
Description
OG000
Pexidartinib Part 1 and Part 2
Participants received treatment of Pexidartinib,1000 mg (5 capsules per day) for 2 weeks, then 800 mg (4 capsules per day) for 22 weeks and also received Pexidartinib in Part 2 at their prescribed dose.
Pexidartinib: Each capsule contains 200 mg of Pexidartinib for oral administration.
OG001
Placebo Part 1, Pexidartinib Part 2
Time Frame
Adverse events were monitored throughout the study from the time the participant signed the informed consent form to 28 days after the final treatment dose, up to 71 months.
Description
Not provided
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
Pexidartinib (Part 1)
Participants received blinded treatment of pexidartinib,1000 mg (5 capsules per day ) for 2 weeks, then 800 mg (4 capsules per day) for 22 weeks
Pexidartinib: Each capsule contains 200 mg of pexidartinib for oral administration
0
61
8
61
60
61
EG001
Placebo (Part 1)
Participants received blinded treatment of matching placebo (5 capsules per day) for 2 weeks, then matching placebo (4 capsules per day) for 22 weeks
Placebo: Placebo capsule matching pexidartinib capsule for oral administration
0
59
1
59
55
59
EG002
Pexidartinib (Parts 1 and 2)
Participants received pexidartinib in Part 1 and in Part 2 at their prescribed dose
Pexidartinib: Each capsule contains 200 mg of pexidartinib for oral administration
0
61
12
61
61
61
EG003
Placebo (Part 1), Crossover Pexidartinib (Part 2)
Participants received placebo in Part 1 and pexidartinib in Part 2 at their prescribed dose
Pexidartinib: Each capsule contains 200 mg of pexidartinib for oral administration
Placebo: Placebo capsule matching pexidartinib capsule for oral administration
1
30
9
30
30
30
EG004
All Pexidartinib Treated
All participants who received pexidartinib in Part 1 and Part 2 (placebo crossed over to pexidartinib)
1
91
21
91
91
91
Serious Adverse Events
Term
Organ System
Source Vocabulary
Assessment Type
Notes
Statistical Information
Transaminases increased
Investigations
MedDRA (17.1)
Systematic Assessment
EG0001 affected61 at risk
EG0010 affected59 at risk
EG0021 affected61 at risk
EG0030 affected30 at risk
EG0041 affected91 at risk
Liver function test abnormal
Investigations
MedDRA (17.1)
Systematic Assessment
EG0001 affected61 at risk
EG0010 affected59 at risk
EG0021 affected61 at risk
EG003
Hepatic enzyme abnormal
Investigations
MedDRA (17.1)
Systematic Assessment
EG0001 affected61 at risk
EG0010 affected59 at risk
EG0021 affected61 at risk
EG003
Blood bilirubin increased
Investigations
MedDRA (17.1)
Systematic Assessment
EG0001 affected61 at risk
EG0010 affected59 at risk
EG0021 affected61 at risk
EG003
Adenosquamous carcinoma of the cervix
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
MedDRA (17.1)
Systematic Assessment
EG0000 affected61 at risk
EG0010 affected59 at risk
EG0020 affected61 at risk
EG003
Rectal adenocarcinoma
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
MedDRA (17.1)
Systematic Assessment
EG0000 affected61 at risk
EG0010 affected59 at risk
EG0020 affected61 at risk
EG003
Endometrial cancer
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
MedDRA (17.1)
Systematic Assessment
EG0000 affected61 at risk
EG0011 affected59 at risk
EG0020 affected61 at risk
EG003
Squamous cell carcinoma of skin
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
MedDRA (17.1)
Systematic Assessment
EG0000 affected61 at risk
EG0010 affected59 at risk
EG0020 affected61 at risk
EG003
Cardiac arrest
Cardiac disorders
MedDRA (17.1)
Systematic Assessment
EG0000 affected61 at risk
EG0010 affected59 at risk
EG0020 affected61 at risk
EG003
Asthma
Respiratory, thoracic and mediastinal disorders
MedDRA (17.1)
Systematic Assessment
EG0000 affected61 at risk
EG0010 affected59 at risk
EG0021 affected61 at risk
EG003
Migraine
Nervous system disorders
MedDRA (17.1)
Systematic Assessment
EG0001 affected61 at risk
EG0010 affected59 at risk
EG0021 affected61 at risk
EG003
Local swelling
General disorders
MedDRA (17.1)
Systematic Assessment
EG0000 affected61 at risk
EG0010 affected59 at risk
EG0020 affected61 at risk
EG003
Non-cardiac chest pain
General disorders
MedDRA (17.1)
Systematic Assessment
EG0000 affected61 at risk
EG0010 affected59 at risk
EG0020 affected61 at risk
EG003
Hepatoxicity
Hepatobiliary disorders
MedDRA (17.1)
Systematic Assessment
EG0001 affected61 at risk
EG0010 affected59 at risk
EG0021 affected61 at risk
EG003
Liver disorder
Hepatobiliary disorders
MedDRA (17.1)
Systematic Assessment
EG0001 affected61 at risk
EG0010 affected59 at risk
EG0021 affected61 at risk
EG003
Rash papular
Skin and subcutaneous tissue disorders
MedDRA (17.1)
Systematic Assessment
EG0000 affected61 at risk
EG0010 affected59 at risk
EG0021 affected61 at risk
EG003
Rash
Skin and subcutaneous tissue disorders
MedDRA (17.1)
Systematic Assessment
EG0001 affected61 at risk
EG0010 affected59 at risk
EG0021 affected61 at risk
EG003
Neck pain
Musculoskeletal and connective tissue disorders
MedDRA (17.1)
Systematic Assessment
EG0000 affected61 at risk
EG0010 affected59 at risk
EG0020 affected61 at risk
EG003
Hepatitis A
Infections and infestations
MedDRA (17.1)
Systematic Assessment
EG0001 affected61 at risk
EG0010 affected59 at risk
EG0021 affected61 at risk
EG003
Hepatitis E
Infections and infestations
MedDRA (17.1)
Systematic Assessment
EG0001 affected61 at risk
EG0010 affected59 at risk
EG0021 affected61 at risk
EG003
Lymphangitis
Infections and infestations
MedDRA (17.1)
Systematic Assessment
EG0000 affected61 at risk
EG0010 affected59 at risk
EG0020 affected61 at risk
EG003
Lipoma
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
MedDRA (17.1)
Systematic Assessment
EG0000 affected61 at risk
EG0010 affected59 at risk
EG0021 affected61 at risk
EG003
Rectal cancer stage II
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
MedDRA (17.1)
Systematic Assessment
EG0000 affected61 at risk
EG0010 affected59 at risk
EG0020 affected61 at risk
EG003
Squamous cell carcinoma
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
MedDRA (17.1)
Systematic Assessment
EG0000 affected61 at risk
EG0010 affected59 at risk
EG0021 affected61 at risk
EG003
Joint injury
Injury, poisoning and procedural complications
MedDRA (17.1)
Systematic Assessment
EG0000 affected61 at risk
EG0010 affected59 at risk
EG0020 affected61 at risk
EG003
Post procedural complication
Injury, poisoning and procedural complications
MedDRA (17.1)
Systematic Assessment
EG0000 affected61 at risk
EG0010 affected59 at risk
EG0021 affected61 at risk
EG003
Rib fracture
Injury, poisoning and procedural complications
MedDRA (17.1)
Systematic Assessment
EG0000 affected61 at risk
EG0010 affected59 at risk
EG0020 affected61 at risk
EG003
Spinal fracture
Injury, poisoning and procedural complications
MedDRA (17.1)
Systematic Assessment
EG0000 affected61 at risk
EG0010 affected59 at risk
EG0020 affected61 at risk
EG003
Wound dehiscence
Injury, poisoning and procedural complications
MedDRA (17.1)
Systematic Assessment
EG0000 affected61 at risk
EG0010 affected59 at risk
EG0020 affected61 at risk
EG003
Osteoarthritis
Musculoskeletal and connective tissue disorders
MedDRA (17.1)
Systematic Assessment
EG0000 affected61 at risk
EG0010 affected59 at risk
EG0021 affected61 at risk
EG003
Spondylolisthesis
Musculoskeletal and connective tissue disorders
MedDRA (17.1)
Systematic Assessment
EG0000 affected61 at risk
EG0010 affected59 at risk
EG0020 affected61 at risk
EG003
Hepatotoxicity
Hepatobiliary disorders
MedDRA (17.1)
Systematic Assessment
EG0001 affected61 at risk
EG0010 affected59 at risk
EG0021 affected61 at risk
EG003
Bronchopneumonia
Infections and infestations
MedDRA (17.1)
Systematic Assessment
EG0000 affected61 at risk
EG0010 affected59 at risk
EG0021 affected61 at risk
EG003
Paronychia
Infections and infestations
MedDRA (17.1)
Systematic Assessment
EG0000 affected61 at risk
EG0010 affected59 at risk
EG0021 affected61 at risk
EG003
Abdominal pain
Gastrointestinal disorders
MedDRA (17.1)
Systematic Assessment
EG0000 affected61 at risk
EG0010 affected59 at risk
EG0021 affected61 at risk
EG003
Abortion spontaneous
Pregnancy, puerperium and perinatal conditions
MedDRA (17.1)
Systematic Assessment
EG0000 affected61 at risk
EG0010 affected59 at risk
EG0020 affected61 at risk
EG003
Other Adverse Events
Term
Organ System
Source Vocabulary
Assessment Type
Notes
Statistical Information
Hypertension
Vascular disorders
MedDRA (17.1)
Systematic Assessment
EG00015 events9 affected61 at risk
EG0016 events6 affected59 at risk
EG00224 events14 affected61 at risk
EG00321 events12 affected30 at risk
EG00445 events26 affected91 at risk
Hypotension
Vascular disorders
MedDRA (17.1)
Systematic Assessment
EG0000 events0 affected61 at risk
EG0010 events0 affected59 at risk
EG0020 events0 affected61 at risk
EG003
Tumor pain
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
MedDRA (17.1)
Systematic Assessment
EG0007 events4 affected61 at risk
EG0016 events4 affected59 at risk
EG0027 events4 affected61 at risk
EG003
Fatigue
General disorders
MedDRA (17.1)
Systematic Assessment
EG00049 events33 affected61 at risk
EG00126 events21 affected59 at risk
EG00257 events35 affected61 at risk
EG003
Face oedema
General disorders
MedDRA (17.1)
Systematic Assessment
EG0008 events8 affected61 at risk
EG0011 events1 affected59 at risk
EG00211 events9 affected61 at risk
EG003
Oedema peripheral
General disorders
MedDRA (17.1)
Systematic Assessment
EG0009 events8 affected61 at risk
EG0012 events2 affected59 at risk
EG00218 events15 affected61 at risk
EG003
Asthenia
General disorders
MedDRA (17.1)
Systematic Assessment
EG0007 events6 affected61 at risk
EG0015 events3 affected59 at risk
EG00216 events9 affected61 at risk
EG003
Pyrexia
General disorders
MedDRA (17.1)
Systematic Assessment
EG0004 events4 affected61 at risk
EG0011 events1 affected59 at risk
EG0028 events8 affected61 at risk
EG003
Influenza like illness
General disorders
MedDRA (17.1)
Systematic Assessment
EG0000 events0 affected61 at risk
EG0010 events0 affected59 at risk
EG0026 events5 affected61 at risk
EG003
Chest pain
General disorders
MedDRA (17.1)
Systematic Assessment
EG0000 events0 affected61 at risk
EG0010 events0 affected59 at risk
EG0021 events1 affected61 at risk
EG003
Malaise
General disorders
MedDRA (17.1)
Systematic Assessment
EG0000 events0 affected61 at risk
EG0010 events0 affected59 at risk
EG0021 events1 affected61 at risk
EG003
Insomnia
Psychiatric disorders
MedDRA (17.1)
Systematic Assessment
EG0000 events0 affected61 at risk
EG0012 events2 affected59 at risk
EG0024 events4 affected61 at risk
EG003
Aspartate aminotransferase increased
Investigations
MedDRA (17.1)
Systematic Assessment
EG00048 events24 affected61 at risk
EG0010 events0 affected59 at risk
EG00263 events28 affected61 at risk
EG003
Alanine aminotransferase increased
Investigations
MedDRA (17.1)
Systematic Assessment
EG00044 events17 affected61 at risk
EG0011 events1 affected59 at risk
EG00253 events19 affected61 at risk
EG003
Blood alkaline phosphatase increased
Investigations
MedDRA (17.1)
Systematic Assessment
EG00026 events9 affected61 at risk
EG0010 events0 affected59 at risk
EG00230 events9 affected61 at risk
EG003
Blood lactate dehydrogenase increased
Investigations
MedDRA (17.1)
Systematic Assessment
EG00012 events7 affected61 at risk
EG0010 events0 affected59 at risk
EG00217 events7 affected61 at risk
EG003
White blood cell count decreased
Investigations
MedDRA (17.1)
Systematic Assessment
EG0000 events0 affected61 at risk
EG0011 events1 affected59 at risk
EG00211 events6 affected61 at risk
EG003
Weight increased
Investigations
MedDRA (17.1)
Systematic Assessment
EG0000 events0 affected61 at risk
EG0010 events0 affected59 at risk
EG0024 events3 affected61 at risk
EG003
Blood bilirubin increased
Investigations
MedDRA (17.1)
Systematic Assessment
EG0000 events0 affected61 at risk
EG0012 events1 affected59 at risk
EG0028 events4 affected61 at risk
EG003
Blood creatinine phosphokinase increased
Investigations
MedDRA (17.1)
Systematic Assessment
EG0000 events0 affected61 at risk
EG0010 events0 affected59 at risk
EG00216 events6 affected61 at risk
EG003
Bradycardia
Cardiac disorders
MedDRA (17.1)
Systematic Assessment
EG0000 events0 affected61 at risk
EG0010 events0 affected59 at risk
EG0020 events0 affected61 at risk
EG003
Anaemia
Blood and lymphatic system disorders
MedDRA (17.1)
Systematic Assessment
EG0003 events3 affected61 at risk
EG0012 events1 affected59 at risk
EG00212 events7 affected61 at risk
EG003
Neutropenia
Blood and lymphatic system disorders
MedDRA (17.1)
Systematic Assessment
EG0003 events3 affected61 at risk
EG0010 events0 affected59 at risk
EG00213 events4 affected61 at risk
EG003
Leukopenia
Blood and lymphatic system disorders
MedDRA (17.1)
Systematic Assessment
EG0002 events2 affected61 at risk
EG0010 events0 affected59 at risk
EG0027 events4 affected61 at risk
EG003
Thrombocytopenia
Blood and lymphatic system disorders
MedDRA (17.1)
Systematic Assessment
EG0002 events2 affected61 at risk
EG0010 events0 affected59 at risk
EG0022 events2 affected61 at risk
EG003
Cough
Respiratory, thoracic and mediastinal disorders
MedDRA (17.1)
Systematic Assessment
EG0000 events0 affected61 at risk
EG0013 events3 affected59 at risk
EG0026 events4 affected61 at risk
EG003
Dyspnoea
Respiratory, thoracic and mediastinal disorders
MedDRA (17.1)
Systematic Assessment
EG0000 events0 affected61 at risk
EG0010 events0 affected59 at risk
EG0024 events3 affected61 at risk
EG003
Oropharyngeal pain
Respiratory, thoracic and mediastinal disorders
MedDRA (17.1)
Systematic Assessment
EG0000 events0 affected61 at risk
EG0013 events2 affected59 at risk
EG0024 events4 affected61 at risk
EG003
Dysgeusia
Nervous system disorders
MedDRA (17.1)
Systematic Assessment
EG00024 events15 affected61 at risk
EG0011 events1 affected59 at risk
EG00229 events18 affected61 at risk
EG003
Headache
Nervous system disorders
MedDRA (17.1)
Systematic Assessment
EG00016 events11 affected61 at risk
EG00115 events11 affected59 at risk
EG00225 events15 affected61 at risk
EG003
Dizziness
Nervous system disorders
MedDRA (17.1)
Systematic Assessment
EG0007 events6 affected61 at risk
EG00112 events9 affected59 at risk
EG00214 events10 affected61 at risk
EG003
Paresthesia
Nervous system disorders
MedDRA (17.1)
Systematic Assessment
EG0000 events0 affected61 at risk
EG0011 events1 affected59 at risk
EG0027 events6 affected61 at risk
EG003
Memory impairment
Nervous system disorders
MedDRA (17.1)
Systematic Assessment
EG0000 events0 affected61 at risk
EG0011 events1 affected59 at risk
EG0022 events2 affected61 at risk
EG003
Neuropathy peripheral
Nervous system disorders
MedDRA (17.1)
Systematic Assessment
EG0000 events0 affected61 at risk
EG0010 events0 affected59 at risk
EG0025 events4 affected61 at risk
EG003
Periorbital oedema
Eye disorders
MedDRA (17.1)
Systematic Assessment
EG00013 events11 affected61 at risk
EG0011 events1 affected59 at risk
EG00223 events17 affected61 at risk
EG003
Eye oedema
Eye disorders
MedDRA (17.1)
Systematic Assessment
EG0006 events6 affected61 at risk
EG0012 events2 affected59 at risk
EG0026 events6 affected61 at risk
EG003
Eyelid oedema
Eye disorders
MedDRA (17.1)
Systematic Assessment
EG0000 events0 affected61 at risk
EG0010 events0 affected59 at risk
EG0025 events3 affected61 at risk
EG003
Vision blurred
Eye disorders
MedDRA (17.1)
Systematic Assessment
EG0000 events0 affected61 at risk
EG0010 events0 affected59 at risk
EG0025 events5 affected61 at risk
EG003
Tinnitus
Ear and labyrinth disorders
MedDRA (17.1)
Systematic Assessment
EG0001 events1 affected61 at risk
EG0010 events0 affected59 at risk
EG0022 events2 affected61 at risk
EG003
Vertigo
Ear and labyrinth disorders
MedDRA (17.1)
Systematic Assessment
EG0001 events1 affected61 at risk
EG0010 events0 affected59 at risk
EG0024 events4 affected61 at risk
EG003
Nausea
Gastrointestinal disorders
MedDRA (17.1)
Systematic Assessment
EG00040 events23 affected61 at risk
EG00127 events24 affected59 at risk
EG00266 events28 affected61 at risk
EG003
Diarrhea
Gastrointestinal disorders
MedDRA (17.1)
Systematic Assessment
EG00017 events13 affected61 at risk
EG00118 events15 affected59 at risk
EG00234 events19 affected61 at risk
EG003
Vomiting
Gastrointestinal disorders
MedDRA (17.1)
Systematic Assessment
EG00016 events12 affected61 at risk
EG0013 events3 affected59 at risk
EG00223 events16 affected61 at risk
EG003
Abdominal pain
Gastrointestinal disorders
MedDRA (17.1)
Systematic Assessment
EG00012 events10 affected61 at risk
EG0018 events6 affected59 at risk
EG00215 events13 affected61 at risk
EG003
Constipation
Gastrointestinal disorders
MedDRA (17.1)
Systematic Assessment
EG0008 events7 affected61 at risk
EG0013 events3 affected59 at risk
EG00213 events10 affected61 at risk
EG003
Dry mouth
Gastrointestinal disorders
MedDRA (17.1)
Systematic Assessment
EG0006 events6 affected61 at risk
EG0012 events2 affected59 at risk
EG00210 events8 affected61 at risk
EG003
Stomatitis
Gastrointestinal disorders
MedDRA (17.1)
Systematic Assessment
EG0004 events4 affected61 at risk
EG0012 events1 affected59 at risk
EG0025 events5 affected61 at risk
EG003
Hair color changes
Skin and subcutaneous tissue disorders
MedDRA (17.1)
Systematic Assessment
EG00046 events41 affected61 at risk
EG0012 events2 affected59 at risk
EG00253 events44 affected61 at risk
EG003
Pruritis
Skin and subcutaneous tissue disorders
MedDRA (17.1)
Systematic Assessment
EG00011 events10 affected61 at risk
EG0012 events2 affected59 at risk
EG00210 events10 affected61 at risk
EG003
Rash
Skin and subcutaneous tissue disorders
MedDRA (17.1)
Systematic Assessment
EG0009 events8 affected61 at risk
EG0014 events3 affected59 at risk
EG00228 events17 affected61 at risk
EG003
Rash maculo-papular
Skin and subcutaneous tissue disorders
MedDRA (17.1)
Systematic Assessment
EG0008 events6 affected61 at risk
EG0011 events1 affected59 at risk
EG00217 events10 affected61 at risk
EG003
Erythema
Skin and subcutaneous tissue disorders
MedDRA (17.1)
Systematic Assessment
EG0000 events0 affected61 at risk
EG0010 events0 affected59 at risk
EG0023 events2 affected61 at risk
EG003
Pruritis generalized
Skin and subcutaneous tissue disorders
MedDRA (17.1)
Systematic Assessment
EG0000 events0 affected61 at risk
EG0010 events0 affected59 at risk
EG0027 events6 affected61 at risk
EG003
Dry skin
Skin and subcutaneous tissue disorders
MedDRA (17.1)
Systematic Assessment
EG0000 events0 affected61 at risk
EG0012 events2 affected59 at risk
EG0026 events5 affected61 at risk
EG003
Alopecia
Skin and subcutaneous tissue disorders
MedDRA (17.1)
Systematic Assessment
EG0000 events0 affected61 at risk
EG0010 events0 affected59 at risk
EG0024 events4 affected61 at risk
EG003
Skin hypopigmentation
Skin and subcutaneous tissue disorders
MedDRA (17.1)
Systematic Assessment
EG0000 events0 affected61 at risk
EG0010 events0 affected59 at risk
EG0027 events5 affected61 at risk
EG003
Photosensitivity reaction
Skin and subcutaneous tissue disorders
MedDRA (17.1)
Systematic Assessment
EG0000 events0 affected61 at risk
EG0010 events0 affected59 at risk
EG0023 events3 affected61 at risk
EG003
Rash pruritic
Skin and subcutaneous tissue disorders
MedDRA (17.1)
Systematic Assessment
EG0000 events0 affected61 at risk
EG0011 events1 affected59 at risk
EG0021 events1 affected61 at risk
EG003
Dermatitis
Skin and subcutaneous tissue disorders
MedDRA (17.1)
Systematic Assessment
EG0000 events0 affected61 at risk
EG0010 events0 affected59 at risk
EG0020 events0 affected61 at risk
EG003
Arthralgia
Musculoskeletal and connective tissue disorders
MedDRA (17.1)
Systematic Assessment
EG00017 events14 affected61 at risk
EG00118 events15 affected59 at risk
EG00229 events19 affected61 at risk
EG003
Pain in extremity
Musculoskeletal and connective tissue disorders
MedDRA (17.1)
Systematic Assessment
EG0006 events4 affected61 at risk
EG0013 events3 affected59 at risk
EG00211 events9 affected61 at risk
EG003
Back pain
Musculoskeletal and connective tissue disorders
MedDRA (17.1)
Systematic Assessment
EG0000 events0 affected61 at risk
EG0010 events0 affected59 at risk
EG0027 events6 affected61 at risk
EG003
Decreased appetite
Metabolism and nutrition disorders
MedDRA (17.1)
Systematic Assessment
EG00013 events10 affected61 at risk
EG0016 events6 affected59 at risk
EG00215 events11 affected61 at risk
EG003
Hypercholesterolemia
Metabolism and nutrition disorders
MedDRA (17.1)
Systematic Assessment
EG0008 events5 affected61 at risk
EG0010 events0 affected59 at risk
EG00220 events7 affected61 at risk
EG003
Fluid retention
Metabolism and nutrition disorders
MedDRA (17.1)
Systematic Assessment
EG0000 events0 affected61 at risk
EG0010 events0 affected59 at risk
EG0026 events3 affected61 at risk
EG003
Hyperglycemia
Metabolism and nutrition disorders
MedDRA (17.1)
Systematic Assessment
EG0000 events0 affected61 at risk
EG0011 events1 affected59 at risk
EG0021 events1 affected61 at risk
EG003
Nasopharyngitis
Infections and infestations
MedDRA (17.1)
Systematic Assessment
EG0005 events4 affected61 at risk
EG0013 events3 affected59 at risk
EG0027 events5 affected61 at risk
EG003
Upper respiratory tract infection
Infections and infestations
MedDRA (17.1)
Systematic Assessment
EG0000 events0 affected61 at risk
EG0010 events0 affected59 at risk
EG00211 events8 affected61 at risk
EG003
Sinusitis
Infections and infestations
MedDRA (17.1)
Systematic Assessment
EG0000 events0 affected61 at risk
EG0010 events0 affected59 at risk
EG0029 events5 affected61 at risk
EG003
Cellulitis
Infections and infestations
MedDRA (17.1)
Systematic Assessment
EG0000 events0 affected61 at risk
EG0010 events0 affected59 at risk
EG0021 events1 affected61 at risk
EG003
Cystitis
Infections and infestations
MedDRA (17.1)
Systematic Assessment
EG0000 events0 affected61 at risk
EG0011 events1 affected59 at risk
EG0020 events0 affected61 at risk
EG003
Abdominal pain upper
Gastrointestinal disorders
MedDRA (17.1)
Systematic Assessment
EG0000 events0 affected61 at risk
EG0014 events4 affected59 at risk
EG0023 events3 affected61 at risk
EG003
Peripheral swelling
General disorders
MedDRA (17.1)
Systematic Assessment
EG0000 events0 affected61 at risk
EG0011 events1 affected59 at risk
EG0020 events0 affected61 at risk
EG003
Blood triglycerides increased
Investigations
MedDRA (17.1)
Systematic Assessment
EG0001 events1 affected61 at risk
EG0010 events0 affected59 at risk
EG0023 events1 affected61 at risk
EG003
Myalgia
Musculoskeletal and connective tissue disorders
MedDRA (17.1)
Systematic Assessment
EG0001 events1 affected61 at risk
EG0012 events2 affected59 at risk
EG0022 events2 affected61 at risk
EG003
Muscle spasms
Musculoskeletal and connective tissue disorders
MedDRA (17.1)
Systematic Assessment
EG0002 events2 affected61 at risk
EG0011 events1 affected59 at risk
EG0026 events4 affected61 at risk
EG003
neck pain
Musculoskeletal and connective tissue disorders
MedDRA (17.1)
Systematic Assessment
EG0001 events1 affected61 at risk
EG0011 events1 affected59 at risk
EG0021 events1 affected61 at risk
EG003
Hypoaesthesia
Nervous system disorders
MedDRA (17.1)
Systematic Assessment
EG0001 events1 affected61 at risk
EG0012 events2 affected59 at risk
EG0021 events1 affected61 at risk
EG003
Sciatica
Nervous system disorders
MedDRA (17.1)
Systematic Assessment
EG0000 events0 affected61 at risk
EG0011 events1 affected59 at risk
EG0020 events0 affected61 at risk
EG003
Influenza
Infections and infestations
MedDRA (17.1)
Systematic Assessment
EG0000 events0 affected61 at risk
EG0012 events2 affected59 at risk
EG0023 events2 affected61 at risk
EG003
Urinary tract infection
Infections and infestations
MedDRA (17.1)
Systematic Assessment
EG0000 events0 affected61 at risk
EG0015 events4 affected59 at risk
EG0022 events2 affected61 at risk
EG003
Herpes zoster
Infections and infestations
MedDRA (17.1)
Systematic Assessment
EG0000 events0 affected61 at risk
EG0010 events0 affected59 at risk
EG0020 events0 affected61 at risk
EG003
Lacrimation increased
Eye disorders
MedDRA (17.1)
Systematic Assessment
EG0003 events3 affected61 at risk
EG0010 events0 affected59 at risk
EG0024 events4 affected61 at risk
EG003
Hypophosphataemia
Metabolism and nutrition disorders
MedDRA (17.1)
Systematic Assessment
EG0003 events3 affected61 at risk
EG0011 events1 affected59 at risk
EG0026 events3 affected61 at risk
EG003
Hypertriglyceridaemia
Metabolism and nutrition disorders
MedDRA (17.1)
Systematic Assessment
EG0000 events0 affected61 at risk
EG0015 events4 affected59 at risk
EG0024 events2 affected61 at risk
EG003
Anxiety
Psychiatric disorders
MedDRA (17.1)
Systematic Assessment
EG0000 events0 affected61 at risk
EG0011 events1 affected59 at risk
EG0021 events1 affected61 at risk
EG003
Palpitations
Cardiac disorders
MedDRA (17.1)
Systematic Assessment
EG0002 events2 affected61 at risk
EG0010 events0 affected59 at risk
EG0025 events4 affected61 at risk
EG003
Tachycardia
Cardiac disorders
MedDRA (17.1)
Systematic Assessment
EG0000 events0 affected61 at risk
EG0010 events0 affected59 at risk
EG0023 events1 affected61 at risk
EG003
Haematuria
Renal and urinary disorders
MedDRA (17.1)
Systematic Assessment
EG0000 events0 affected61 at risk
EG0012 events2 affected59 at risk
EG0020 events0 affected61 at risk
EG003
Enrollment was stopped on 30 Sep 2016; no new participants received the study drug. After Part 1, those who wished to continue were un-blinded; those on placebo were discontinued.
Subject transitioned to another DS pexidartinib protocol
FG00015 subjects
FG0019 subjects
Surgical resection of tumor
FG0001 subjects
FG0011 subjects
Other
FG0000 subjects
FG0011 subjects
BG00057
BG00156
BG002113
>=65 years
BG0004
BG0013
BG0027
44.5
± 13.4
71
Male
BG00026
BG00123
BG00249
2
Asian
BG0001
BG0012
BG0023
Native Hawaiian or Other Pacific Islander
BG0002
BG0012
BG0024
Black or African American
BG0003
BG0011
BG0024
White
BG00052
BG00154
BG002106
More than one race
BG0001
BG0010
BG0021
Unknown or Not Reported
BG0000
BG0010
BG0020
5
Netherlands
Title
Measurements
BG0007
BG0014
BG00211
Hungary
Title
Measurements
BG0002
BG0011
BG0023
United States
Title
Measurements
BG00023
BG00122
BG00245
Denmark
Title
Measurements
BG0001
BG0012
BG0023
Poland
Title
Measurements
BG0002
BG0010
BG0022
Italy
Title
Measurements
BG0008
BG0019
BG00217
United Kingdom
Title
Measurements
BG0000
BG0011
BG0021
Australia
Title
Measurements
BG0005
BG0017
BG00212
France
Title
Measurements
BG0002
BG0015
BG0027
Germany
Title
Measurements
BG0004
BG0012
BG0026
Spain
Title
Measurements
BG0005
BG0013
BG0028
24.6
OG0010
Response (CR or PR)
Title
Measurements
OG00039.3
OG0010
Units
Counts
Participants
OG00061
OG00159
Title
Denominators
Categories
Baseline
ParticipantsOG00061
ParticipantsOG00158
Title
Measurements
OG00062.5± 3.2
OG00162.9± 2.9
Week 13
ParticipantsOG00052
ParticipantsOG00153
Title
Measurements
OG00013.0± 2.3
OG001
Week 25
ParticipantsOG00045
ParticipantsOG00143
Title
Measurements
OG00015.1± 2.1
OG001
Group IDs
Group Description
Statistical Method
Statistical Comment
P-Value
P-Value Comment
Parameter Type
Parameter Value
Dispersion Type
Dispersion Value
Confidence Interval Sides
Confidence Interval %
CI Lower Limit
CI Upper Limit
CI Lower Limit Comment
CI Upper Limit Comment
Estimate Comment
Tested Non-Inferiority
Non-Inferiority Type
Non-Inferiority Comment
Other Analysis Description
OG000
OG001
Treatment comparison between the pexidartinib and placebo groups at Week 25
Fisher's Exact Test
0.0043
Other
Treatment comparison analysis
Units
Counts
Participants
OG00061
OG00159
Title
Denominators
Categories
Complete Response (CR)
Title
Measurements
OG0004.9
OG0010
Partial Response (PR)
Title
Measurements
OG00050.8
OG0010
Response (CR or PR)
Title
Measurements
OG00055.7
OG0010
Group IDs
Group Description
Statistical Method
Statistical Comment
P-Value
P-Value Comment
Parameter Type
Parameter Value
Dispersion Type
Dispersion Value
Confidence Interval Sides
Confidence Interval %
CI Lower Limit
CI Upper Limit
CI Lower Limit Comment
CI Upper Limit Comment
Estimate Comment
Tested Non-Inferiority
Non-Inferiority Type
Non-Inferiority Comment
Other Analysis Description
OG000
OG001
Treatment comparison between the pexidartinib and placebo groups at Week 25
Fisher's Exact Test
<0.0001
Other
Treatment comparison analysis
Units
Counts
Participants
OG00061
OG00159
Title
Denominators
Categories
Week 9
ParticipantsOG00038
ParticipantsOG00141
Title
Measurements
OG0002.8± 1.0
OG001-0.4± 0.8
Week 17
ParticipantsOG00039
ParticipantsOG00140
Title
Measurements
OG0003.2± 1.1
OG001
Week 25
ParticipantsOG00038
ParticipantsOG00131
Title
Measurements
OG0004.1± 1.1
OG001
Group IDs
Group Description
Statistical Method
Statistical Comment
P-Value
P-Value Comment
Parameter Type
Parameter Value
Dispersion Type
Dispersion Value
Confidence Interval Sides
Confidence Interval %
CI Lower Limit
CI Upper Limit
CI Lower Limit Comment
CI Upper Limit Comment
Estimate Comment
Tested Non-Inferiority
Non-Inferiority Type
Non-Inferiority Comment
Other Analysis Description
OG000
OG001
Treatment comparison between pexidartinib and placebo groups at Week 25
Mixed effects model for repeated measure
0.0019
Other
Treatment comparison analysis
OG00061
OG00159
Title
Denominators
Categories
Baseline
ParticipantsOG00059
ParticipantsOG00158
Title
Measurements
OG0005.6± 0.2
OG0015.9± 0.3
Week 9
ParticipantsOG00030
ParticipantsOG00138
Title
Measurements
OG000-1.5± 0.3
OG001
Week 17
ParticipantsOG00037
ParticipantsOG00130
Title
Measurements
OG000-2.4± 0.3
OG001
Week 25
ParticipantsOG00033
ParticipantsOG00135
Title
Measurements
OG000-2.5± 0.3
OG001
Group IDs
Group Description
Statistical Method
Statistical Comment
P-Value
P-Value Comment
Parameter Type
Parameter Value
Dispersion Type
Dispersion Value
Confidence Interval Sides
Confidence Interval %
CI Lower Limit
CI Upper Limit
CI Lower Limit Comment
CI Upper Limit Comment
Estimate Comment
Tested Non-Inferiority
Non-Inferiority Type
Non-Inferiority Comment
Other Analysis Description
OG000
OG001
Treatment comparison between the pexidartinib and placebo groups at Week 25
Mixed effects model for repeated measure
<0.0001
Other
Treatment comparison analysis
OG00061
OG00159
Title
Denominators
Categories
Title
Measurements
OG00031.1
OG00115.3
All Pexidartinib Treated
All participants who received Pexidartinib in Part 1 and Part 2 as well as those who only received Pexidartinib in Part 2.
Units
Counts
Participants
OG00061
OG00130
OG00291
Title
Denominators
Categories
Number of responses
Title
Measurements
OG00023
OG00112
OG00235
Week 12 (Day 84); Without disease progression
Title
Measurements
OG00023
OG00112
OG00235
Week 12 (Day 84); With disease progression
Title
Measurements
OG0000
OG0010
OG0020
Week 24 (Day 168); Without disease progression
Title
Measurements
OG00023
OG00112
OG00235
Week 24 (Day 168); With disease progression
Title
Measurements
OG0000
OG0010
OG0020
Week 48 (Day 336); Without disease progression
Title
Measurements
OG00015
OG0019
OG00224
Week 48 (Day 336); With disease progression
Title
Measurements
OG0001
OG0010
OG0021
Week 72 (Day 504); Without disease progression
Title
Measurements
OG0009
OG0013
OG00212
Week 72 (Day 504); With disease progression
Title
Measurements
OG0001
OG0010
OG0021
Week 96 (Day 672); Without disease progression
Title
Measurements
OG0002
OG0011
OG0023
Week 96 (Day 672); With disease progression
Title
Measurements
OG0001
OG0010
OG0021
OG002
All Pexidartinib Treated
All participants who received pexidartinib in Part 1 and Part 2 as well as those who received pexidartinib in Part 2 only.
Units
Counts
Participants
OG00061
OG00130
OG00291
Title
Denominators
Categories
Number of responders
Title
Measurements
OG00034
OG00118
OG00252
Week 12 (Day 84); Without disease progression
Title
Measurements
OG00033
OG00118
OG00251
Week 12 (Day 84); With disease progression
Title
Measurements
OG0000
OG0010
OG0020
Week 24 (Day 168); Without disease progression
Title
Measurements
OG00032
OG00118
OG00250
Week 24 (Day 168); With disease progression
Title
Measurements
OG0000
OG0010
OG0020
Week 48 (Day 336); Without disease progression
Title
Measurements
OG00022
OG00113
OG00235
Week 48 (Day 336); With disease progression
Title
Measurements
OG0003
OG0011
OG0024
Week 72 (Day 504); Without disease progression
Title
Measurements
OG00013
OG0013
OG00216
Week 72 (Day 504); With disease progression
Title
Measurements
OG0003
OG0011
OG0024
Week 96 (Day 672); Without disease progression
Title
Measurements
OG0003
OG0011
OG0024
Week 96 (Day 672); With disease progression
Title
Measurements
OG0003
OG0011
OG0024
Week 120 (Day 840); Without disease progression
Title
Measurements
OG0001
OG0010
OG0021
Week 120 (Day 840); With disease progression
Title
Measurements
OG0003
OG0011
OG0024
Units
Counts
Participants
OG00037
OG00118
Title
Denominators
Categories
Title
Measurements
OG000NA(31.01 to NA)Median and upper limit 95% CI was not estimable due to insufficient number of events.
OG001NA(39.0 to NA)Median and upper limit 95% CI was not estimable due to insufficient number of events.
Units
Counts
Participants
OG00041
OG00121
Title
Denominators
Categories
Title
Measurements
OG00052.70(38.60 to NA)Upper limit 95% CI was not estimable due to insufficient number of events.
OG001NA(NA to NA)Median and 95% CIs was not estimable due to insufficient number of events.
Participants received treatment of Pexidartinib,1000 mg (5 capsules per day) for 2 weeks, then 800 mg (4 capsules per day) for 22 weeks and also received Pexidartinib in Part 2 at their prescribed dose.
Pexidartinib: Each capsule contains 200 mg of Pexidartinib for oral administration.
OG003
Placebo Part 1, Pexidartinib Part 2
Participants received treatment of matching placebo (5 capsules per day) for 2 weeks, then matching placebo (4 capsules per day) for 22 weeks in Part 1 and also received Pexidartinib in Part 2 at their prescribed dose.
Placebo: Placebo capsule matching Pexidartinib capsule for oral administration Pexidartinib: Each capsule contains 200 mg of Pexidartinib for oral administration.
OG004
All Pexidartinib Treated
All participants who received Pexidartinib in Part 1 and Part 2 as well as those who only received Pexidartinib in Part 2.
Units
Counts
Participants
OG00061
OG00159
OG00261
OG00330
OG00491
Title
Denominators
Categories
Any Hair color changes
Title
Measurements
OG00067.2
OG0013.4
OG00273.8
OG00383.3
OG00476.9
Grade ≥3 Hair color changes
Title
Measurements
OG0000
OG0010
OG0020
OG003
Any Pruritis
Title
Measurements
OG0009.8
OG0013.4
OG00216.4
OG003
Grade ≥3 Pruritis
Title
Measurements
OG0000
OG0010
OG0021.6
OG003
Any Rash maculopapular
Title
Measurements
OG0009.8
OG0011.7
OG00214.8
OG003
Grade ≥3 Rash maculopapular
Title
Measurements
OG0000
OG0010
OG0021.6
OG003
Any Pruritis generalized
Title
Measurements
OG0008.2
OG0010
OG0028.2
OG003
Grade ≥3 Pruritis generalized
Title
Measurements
OG0000
OG0010
OG0020
OG003
Any Erythema
Title
Measurements
OG0001.6
OG0010
OG0023.3
OG003
Grade ≥3 Erythema
Title
Measurements
OG0000
OG0010
OG0020
OG003
Any Dry skin
Title
Measurements
OG0003.3
OG0013.4
OG0026.6
OG003
Grade ≥3 Dry skin
Title
Measurements
OG0000
OG0010
OG0020
OG003
Any Photosensitivity reaction
Title
Measurements
OG0000
OG0010
OG0021.6
OG003
Grade ≥3 Photosensitivity reaction
Title
Measurements
OG0000
OG0010
OG0020
OG003
Any Nausea
Title
Measurements
OG00037.7
OG00140.7
OG00244.3
OG003
Grade ≥3 Nausea
Title
Measurements
OG0000
OG0010
OG0020
OG003
Any Diarrhea
Title
Measurements
OG00019.7
OG00125.4
OG00226.2
OG003
Grade ≥3 Diarrhea
Title
Measurements
OG0000
OG0010
OG0020
OG003
Any Vomiting
Title
Measurements
OG00019.7
OG0015.1
OG00223.0
OG003
Grade ≥3 Vomiting
Title
Measurements
OG0001.6
OG0010
OG0021.6
OG003
Any Abdominal Pain
Title
Measurements
OG00016.4
OG00110.2
OG00221.3
OG003
Grade ≥3 Abdominal Pain
Title
Measurements
OG0000
OG0010
OG0020
OG003
Any Dry mouth
Title
Measurements
OG0009.8
OG0013.4
OG00213.1
OG003
Grade ≥3 Dry mouth
Title
Measurements
OG0000
OG0010
OG0020
OG003
Any Constipation
Title
Measurements
OG00011.5
OG0015.1
OG00214.8
OG003
Grade ≥3 Constipation
Title
Measurements
OG0000
OG0010
OG0020
OG003
Any Stomatitis
Title
Measurements
OG0006.6
OG0011.7
OG0028.2
OG003
Grade ≥3 Stomatitis
Title
Measurements
OG0000
OG0010
OG0020
OG003
Any Fatigue
Title
Measurements
OG00054.1
OG00135.6
OG00255.7
OG003
Grade ≥3 Fatigue
Title
Measurements
OG0000
OG0010
OG0020
OG003
Any Edema peripheral
Title
Measurements
OG00013.1
OG0013.4
OG00216.4
OG003
Grade ≥3 Edema peripheral
Title
Measurements
OG0000
OG0010
OG0020
OG003
Any Face edema
Title
Measurements
OG00013.1
OG0011.7
OG00214.8
OG003
Grade ≥3 Face edema
Title
Measurements
OG0000
OG0010
OG0021.6
OG003
Any Asthenia
Title
Measurements
OG0009.8
OG0015.1
OG00211.5
OG003
Grade ≥3 Asthenia
Title
Measurements
OG0000
OG0010
OG0020
OG003
Any Pyrexia
Title
Measurements
OG0006.6
OG0011.7
OG0028.2
OG003
Grade ≥ Pyrexia
Title
Measurements
OG0000
OG0010
OG0020
OG003
Any AST increased
Title
Measurements
OG00039.3
OG0010
OG00244.3
OG003
Grade ≥3 AST increased
Title
Measurements
OG0009.8
OG0010
OG0029.8
OG003
Any ALT increased
Title
Measurements
OG00027.9
OG0011.7
OG00231.1
OG003
Grade ≥3 ALT increased
Title
Measurements
OG0009.8
OG0010
OG0029.8
OG003
Any ALP increased
Title
Measurements
OG00014.8
OG0010
OG00214.8
OG003
Grade ≥3 ALP increased
Title
Measurements
OG0006.6
OG0010
OG0026.6
OG003
Any LDH increased
Title
Measurements
OG00011.5
OG0010
OG00211.5
OG003
Grade ≥3 LDH increased
Title
Measurements
OG0001.6
OG0010
OG0021.6
OG003
Any Weight increased
Title
Measurements
OG0003.3
OG0010
OG0024.9
OG003
Grade ≥3 Weight increased
Title
Measurements
OG0000
OG0010
OG0020
OG003
Any Dysgeusia
Title
Measurements
OG00024.6
OG0011.7
OG00227.9
OG003
Grade ≥3 Dysgeusia
Title
Measurements
OG0000
OG0010
OG0020
OG003
Any Headache
Title
Measurements
OG00019.7
OG00118.6
OG00223.0
OG003
Grade ≥3 Headache
Title
Measurements
OG0000
OG0010
OG0021.6
OG003
Any Dizziness
Title
Measurements
OG0009.8
OG00115.3
OG00213.1
OG003
Grade ≥3 Dizziness
Title
Measurements
OG0001.6
OG0010
OG0021.6
OG003
Any Paresthesia
Title
Measurements
OG0001.6
OG0011.7
OG0028.2
OG003
Grade ≥3 Paresthesia
Title
Measurements
OG0000
OG0010
OG0020
OG003
Any Memory impairment
Title
Measurements
OG0000
OG0011.7
OG0021.6
OG003
Grade ≥3 Memory impairment
Title
Measurements
OG0000
OG0010
OG0020
OG003
Any Arthralgia
Title
Measurements
OG00023.0
OG00125.4
OG00227.9
OG003
Grade ≥3 Arthralgia
Title
Measurements
OG0003.3
OG0011.7
OG0023.3
OG003
Any Pain in extremity
Title
Measurements
OG0006.6
OG0016.8
OG0029.8
OG003
Grade ≥3 Pain in extremity
Title
Measurements
OG0000
OG0011.7
OG0020
OG003
Any Periorbital edema
Title
Measurements
OG00018.0
OG0011.7
OG00224.6
OG003
Grade ≥3 Periorbital edema
Title
Measurements
OG0001.6
OG0010
OG0021.6
OG003
Any Eyelid edema
Title
Measurements
OG0003.3
OG0010
OG0024.9
OG003
Grade ≥3 Eyelid edema
Title
Measurements
OG0000
OG0010
OG0020
OG003
Any Decreased appetite
Title
Measurements
OG00016.4
OG00110.2
OG00218.0
OG003
Grade ≥3 Decreased appetite
Title
Measurements
OG0000
OG0010
OG0020
OG003
Any Hypertension
Title
Measurements
OG00014.8
OG00110.2
OG00219.7
OG003
Grade ≥3 Hypertension
Title
Measurements
OG0004.9
OG0010
OG0024.9
OG003
Any Upper respiratory tract infection
Title
Measurements
OG0001.6
OG0010
OG00211.5
OG003
Grade ≥3 Upper respiratory tract infection
Title
Measurements
OG0000
OG0010
OG0020
OG003
Any Cough
Title
Measurements
OG0004.9
OG0015.1
OG0026.6
OG003
Grade ≥3 Cough
Title
Measurements
OG0000
OG0010
OG0020
OG003
Any Dyspnea
Title
Measurements
OG0001.6
OG0010
OG0024.9
OG003
Grade ≥3 Dyspnea
Title
Measurements
OG0000
OG0010
OG0020
OG003
Any Insomnia
Title
Measurements
OG0004.9
OG0013.4
OG0024.9
OG003
Grade ≥3 Insomnia
Title
Measurements
OG0000
OG0010
OG0020
OG003
Any Rash
Title
Measurements
OG00014.8
OG0015.1
OG00227.9
OG003
Grade ≥3 Rash
Title
Measurements
OG0001.6
OG0010
OG0021.6
OG003
OG002
All Pexidartinib Treated
All participants who received Pexidartinib in Part 1 and Part 2 as well as those who only received Pexidartinib in Part 2.
Units
Counts
Participants
OG00061
OG00130
OG00291
Title
Denominators
Categories
Complete Response (CR)
Title
Measurements
OG00024.6
OG00123.3
OG00224.2
Partial Response (PR)
Title
Measurements
OG00029.5
OG00130.0
OG00229.7
Response (CR or PR)
Title
Measurements
OG00054.1
OG00153.3
OG00253.8
OG002
All Pexidartinib Treated
All participants who received Pexidartinib in Part 1 and Part 2 as well as those who only received Pexidartinib in Part 2.
Units
Counts
Participants
OG00061
OG00130
OG00291
Title
Denominators
Categories
Baseline
ParticipantsOG00061
ParticipantsOG00130
ParticipantsOG00291
Title
Measurements
OG00062.5± 24.8
OG00166.5± 22.9
OG00263.8± 24.2
Week 25
ParticipantsOG00045
ParticipantsOG00124
ParticipantsOG00269
Title
Measurements
OG000
Week 49
ParticipantsOG00033
ParticipantsOG00122
ParticipantsOG00255
Title
Measurements
OG000
OG002
All Pexidartinib Treated
All participants who received Pexidartinib in Part 1 and Part 2 as well as those who only received Pexidartinib in Part 2.
Units
Counts
Participants
OG00061
OG00130
OG00291
Title
Denominators
Categories
Week 25
ParticipantsOG00038
ParticipantsOG00116
ParticipantsOG00254
Title
Measurements
OG0003.6± 4.9
OG0014.9± 6.3
OG0024.0± 5.4
Week 49
ParticipantsOG00025
ParticipantsOG00114
ParticipantsOG00239
Title
Measurements
OG000
OG002
All Pexidartinib Treated
All participants who received Pexidartinib in Part 1 and Part 2 as well as those who only received Pexidartinib in Part 2.
Units
Counts
Participants
OG00061
OG00130
OG00291
Title
Denominators
Categories
Baseline
ParticipantsOG00059
ParticipantsOG00126
ParticipantsOG00285
Title
Measurements
OG0005.6± 1.7
OG0015.7± 2.3
OG0025.6± 1.9
Week 25
ParticipantsOG00033
ParticipantsOG00118
ParticipantsOG00251
Title
Measurements
OG000
Week 49
ParticipantsOG00022
ParticipantsOG00110
ParticipantsOG00232
Title
Measurements
OG000
OG002
All Pexidartinib Treated
All participants who received Pexidartinib in Part 1 and Part 2 as well as those who only received Pexidartinib in Part 2.
Units
Counts
Participants
OG00061
OG00130
OG00291
Title
Denominators
Categories
Baseline
ParticipantsOG00059
ParticipantsOG00126
ParticipantsOG00285
Title
Measurements
OG0005.6± 1.6
OG0015.2± 2.5
OG0025.5± 1.9
Week 25
ParticipantsOG00033
ParticipantsOG00118
ParticipantsOG00251
Title
Measurements
OG000
Week 49
ParticipantsOG00022
ParticipantsOG00110
ParticipantsOG00232
Title
Measurements
OG000
Participants received treatment of matching placebo (5 capsules per day) for 2 weeks, then matching placebo (4 capsules per day) for 22 weeks in Part 1 and also received Pexidartinib in Part 2 at their prescribed dose.
Placebo: Placebo capsule matching Pexidartinib capsule for oral administration Pexidartinib: Each capsule contains 200 mg of Pexidartinib for oral administration.
OG002
All Pexidartinib Treated
All participants who received Pexidartinib in Part 1 and Part 2 as well as those who only received Pexidartinib in Part 2.