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The purpose of the study is to test different doses of X4P-001 given alone and in combination with axitinib in participants diagnosed with advanced renal cell carcinoma. The goals of the study are to determine the safety and tolerability of X4P-001, as well as the potential effect it may have on the body and the cancer tumor.
X4P-001 is an orally bioavailable C-X-C chemokine receptor type 4 (CXCR4) antagonist that has demonstrated activity in various tumor models. CXCR4 is the receptor for C-X-C chemokine ligand type 12 (CXCL12). CXCL12 has potent chemotactic activity for lymphocytes and myeloid-derived suppressor cells (MDSCs), and is important in homing of hematopoietic stem cells to the bone marrow. CXCR4 is also expressed and active on multiple types of human cancers, including clear cell Renal Cell Carcinoma (ccRCC), ovarian cancer, and melanoma, and increased expression of CXCR4 on tumor cells has been associated with significantly decreased overall participant survival.
Multiple observations implicate the CXCL12/CXCR4 axis in contributing to the lack (or loss) of tumor responsiveness to angiogenesis inhibitors (also referred to as "angiogenic escape"). In animal cancer models, interference with CXCR4 function has been demonstrated to disrupt the tumor microenvironment and unmask the tumor to immune attack by multiple mechanisms, including:
The hypothesis and evidence of published data support is that effective CXCR4 antagonism by X4P-001 would be of potential benefit in participants with advanced ccRCC and other cancers by multiple mechanisms:
This initial clinical trial in participants with advanced ccRCC will evaluate X4P-001 both as a single agent (monotherapy) and also in combination with axitinib, a small molecule tyrosine kinase inhibitor (TKI) approved for second-line treatment of participants with ccRCC. This combination has the potential to further improve outcomes by reducing the angiogenic escape that typically occurs with TKI therapy.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Dose Escalation (Part A): X4P-001 200 mg BID with Axitinib | Experimental | Participants will receive X4P-001 200 milligrams (mg) orally twice daily (BID) with axitinib at 5 mg orally BID. |
|
| Dose Escalation (Part A): X4P-001 400 mg QD with Axitinib | Experimental | Participants will receive X4P-001 400 mg orally once daily (QD) with axitinib at 5 mg orally BID. |
|
| Dose Escalation (Part A): X4P-001 600 mg QD with Axitinib | Experimental | Participants will receive X4P-001 600 mg orally QD with axitinib at 5 mg orally BID. |
|
| Dose Expansion (Part B): X4P-001 400 mg QD With Axitinib | Experimental | Participants received X4P-001 400 mg orally QD with axitinib at 5 mg orally BID. |
|
| Dose Escalation and Expansion (Part C): X4P-001 600 mg QD Monotherapy | Experimental | Participants will receive X4P-001 600 mg orally QD. |
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| X4P-001 | Drug | Continuous, oral dosing |
|
| Measure | Description | Time Frame |
|---|---|---|
| Parts A, B, and C: Number of Participants With Treatment-emergent Adverse Events (TEAEs) | An AE was as any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug related. An SAE was an AE that met at least 1 of the following criteria: resulted in death, was life-threatening, required inpatient hospitalization or prolongation of existing hospitalization for the AE, persistent or significant disability/incapacity or substantial disruption of the ability to conduct normal life functions, congenital anomaly/birth defect (in the child of a participant who was exposed to the study drug), important medical event or reaction. Adverse events with onset after administration of the first dose of study drug up to 10 days after last dosing date were considered TEAEs. A summary of all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the 'Reported Adverse Events' Section. | From first dose of study drug up to 10 days after last dosing (maximum exposure: 52.1 months) |
| Measure | Description | Time Frame |
|---|---|---|
| Parts A and B: Objective Response Rate (ORR), as Assessed Using Response Evaluation Criteria in Solid Tumors (RECIST) Version 1.1 | The ORR was defined as percentage of participants with best overall response of complete response (CR) or partial response (PR). CR: Disappearance of all target and non-target lesions. Any pathological lymph nodes (whether target or non-target) with a reduction in short axis to <10 millimeters (mm). PR: At least a 30% decrease in the sum of diameters of target lesions, taking as reference the baseline sum diameters. |
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Inclusion Criteria:
Exclusion Criteria:
Has life expectancy of less than 3 months.
Has performance status Grade >2 (Eastern Cooperative Oncology Group [ECOG] criteria).
Has New York Heart Association (NYHA) Class III or IV heart failure or uncontrolled hypertension (systolic blood pressure [SBP] ≥160 millimeters of mercury [mm Hg]; diastolic blood pressure [DBP] ≥100 mm Hg).
Has previously received X4P-001.
Parts A and B only: Has received a prior course of axitinib.
Parts A and B only: Has received mechanistic target of rapamycin (mTOR) inhibitor(s) as their only prior treatment for ccRCC.
Has a prior history or current evidence of intracranial (CNS) metastatic RCC, except for
≤3 lesions treated by CyberKnife or excisional surgery, clinically stable for at least 4 weeks, and without evidence of recurrence on MRI imaging at screening.
Has ongoing acute clinical adverse events National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) Grade >1 resulting from prior cancer therapies (except alopecia, tyrosine kinase inhibitor [TKI]-related hand-foot syndrome, or thyroid dysfunction).
Has had within the past 6 months the occurrence or persistence of one or more of the following medical conditions that could not be controlled with usual medical care (for example, required emergency care or hospitalization): hypertension, angina, congestive heart failure, diabetes, seizure disorder.
Has had within the past 6 months the occurrence of one or more of the following events: myocardial infarction, cerebrovascular accident, deep vein thrombosis, pulmonary embolism, hemorrhage (CTC Grade 3 or 4), chronic liver disease (meeting criteria for Child-Pugh Class B or C), a second active malignancy (excluding basal cell carcinoma and cervical carcinoma in situ), organ transplantation.
Has had within the 4 weeks prior to initiation of study drug, or is expected to have during the study period, surgery requiring general anesthesia.
Has, at screening, serologic laboratory tests meeting one or more of the following criteria:
An indeterminate or positive test for antibody to human immunodeficiency virus (HIV)-1 or -2.
An indeterminate or positive test for antibody to hepatitis C virus (HCV), unless documented to have no detectable viral load on two independent samples.
A positive test for hepatitis B surface antigen (HBsAg).
Has, at screening, safety laboratory tests meeting one or more of the following criteria:
Hemoglobin <8.0 grams (g)/deciliter (dL)
Absolute neutrophil count (ANC) <1,500/microliter (μL)
Platelets <75,000/μL
Creatinine >2.0x upper limit of normal (ULN)
Serum aspartate transaminase (AST) >2.5x ULN
Serum alanine transaminase (ALT) >2.5x ULN
Total bilirubin >1.5x ULN (unless due to Gilbert's Syndrome)
International normalized ratio (INR) >1.5x ULN
Has received other anti-cancer therapy within the following specified intervals prior to Day 1:
TKI within 2 weeks.
Radiation therapy within 2 weeks.
Bevacizumab within 4 weeks.
Other chemotherapy (for example, mitomycin-C, nitrosourea) or immunotherapy (for example, antibody, cytokine) within 4 weeks
For investigational anti-cancer therapies, the interval will be determined in consultation with the Medical Monitor.
Has, within 2 weeks prior to Day 1, received a medication prohibited based on cytochrome P3A4 (CYP3A4) interaction
Has, within 2 weeks prior to Day 1, received systemic corticosteroids exceeding prednisone 10 mg per day or equivalent; for other immunosuppressive agents, the exclusionary dose and duration will be determined in consultation with the Medical Monitor.
Is, within 2 weeks prior to Day 1, nursing.
Has, at the planned initiation of study drug, an uncontrolled infection.
Has any other medical or personal condition that, in the opinion of the Investigator, may potentially compromise the safety or compliance of the participant, or may preclude the participant's successful completion of the clinical trial.
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Scottsdale | Arizona | 85259 | United States | |||
This study comprised 3 parts: Part A (Phase 1: Dose escalation cohorts), Part B (Phase 2: Dose expansion cohort), and Part C (Dose escalation and expansion cohort). As planned, the efficacy and safety data collected per dose level not per study part.
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| ID | Title | Description |
|---|---|---|
| FG000 | Dose Escalation (Part A): X4P-001 200 mg BID With Axitinib | Participants received X4P-001 200 milligrams (mg) orally twice daily (BID) with axitinib at 5 mg orally BID. |
| FG001 | Dose Escalation (Part A): X4P-001 400 mg QD With Axitinib |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Dose Escalation (Part A) |
|
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Jun 8, 2018 | Jul 23, 2024 |
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|
| axitinib | Drug | Continuous, oral dosing |
|
|
| From administration of first dose of study medication until disease progression, study completion or early termination (up to 80 weeks) |
| Parts A and B: Time to Objective Response, as Assessed Using RECIST v1.1 | Time to objective response was defined as time from first administration of combination regimen to first CR or PR whichever came first. CR: Disappearance of all target and non-target lesions. Any pathological lymph nodes (whether target or non-target) with a reduction in short axis to <10 mm. PR: At least a 30% decrease in the sum of diameters of target lesions, taking as reference the baseline sum diameters. | From administration of first dose of study medication until first appearance of CR or PR (up to 80 weeks) |
| Parts A and B: Duration of Objective Response (DOR), as Assessed Using RECIST v1.1 | The DOR was defined as the time from first CR or PR whichever came first until the time of disease progression or death by any cause. CR: Disappearance of all target and non-target lesions. Any pathological lymph nodes (whether target or non-target) with a reduction in short axis to <10 mm. PR: At least a 30% decrease in the sum of diameters of target lesions, taking as reference the baseline sum diameters. Disease progression: At least a 20% increase in the sum of diameters of target lesions, taking as reference the smallest sum on study. In addition to the relative increase of 20%, the sum also demonstrated an absolute increase of at least 5 mm. Unequivocal progression of existing non-target lesions. The appearance of 1 or more new lesions was also considered progression. | Time from first CR or PR until the time of disease progression or death due to any cause (up to 80 weeks) |
| Parts A and B: Disease Control Rate (DCR), as Assessed Using RECIST v1.1 | The DCR was defined as the percentage of participants with best overall response of CR, PR or stable disease (SD). CR: Disappearance of all target and non-target lesions. Any pathological lymph nodes (whether target or non-target) with a reduction in short axis to <10 mm. PR: At least a 30% decrease in the sum of diameters of target lesions, taking as reference the baseline sum diameters. SD: Neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for progressive disease (PD), taking as reference the smallest sum diameters while on study. PD: At least a 20% increase in the sum of diameters of target lesions, taking as reference the smallest sum on study. In addition to the relative increase of 20%, the sum also demonstrated an absolute increase of at least 5 mm. Unequivocal progression of existing non-target lesions. The appearance of 1 or more new lesions was also considered progression. | From administration of first dose of study medication until disease progression, study completion or early termination (up to 80 weeks) |
| Parts A and B: Time to Progression (TTP), as Assessed Using RECIST v1.1 | The TTP was defined as the time from first administration of combination regimen until PD. PD: At least a 20% increase in the sum of diameters of target lesions, taking as reference the smallest sum on study In addition to the relative increase of 20%, the sum also demonstrated an absolute increase of at least 5 mm. Unequivocal progression of existing non-target lesions or the appearance of 1 or more new lesions was also considered progression. | From administration of first dose of study medication until disease progression (up to 80 weeks) |
| Parts A and B: Progression Free Survival (PFS), as Assessed Using RECIST v1.1 | The PFS was defined as the time from first administration of combination regimen until disease progression or death from any cause. Disease progression: At least a 20% increase in the sum of diameters of target lesions, taking as reference the smallest sum on study. In addition to the relative increase of 20%, the sum also demonstrated an absolute increase of at least 5 mm. An unequivocal progression of existing non-target lesions or the appearance of 1 or more new lesions was also considered progression. | From administration of first dose of study medication until disease progression or death from any cause (up to 80 weeks) |
| Parts A and B: Maximum Plasma Concentration (Cmax) of X4P-001 | Day 1 and Day 15 of Cycle 1 |
| Area Under the Curve From Time 0 to the Last Quantifiable Timepoint (AUC0-tlast) | Day 1 and Day 15 of Cycle 1 |
| Washington D.C. |
| District of Columbia |
| 20057 |
| United States |
| Jacksonville | Florida | 32224 | United States |
| Chicago | Illinois | 60637 | United States |
| Indianapolis | Indiana | 46237 | United States |
| Iowa City | Iowa | 52242 | United States |
| New Orleans | Louisiana | 70121 | United States |
| Boston | Massachusetts | 02215 | United States |
| Detroit | Michigan | 48201 | United States |
| Saint Paul | Minnesota | 55101 | United States |
| St Louis | Missouri | 63110 | United States |
| Hackensack | New Jersey | 07601 | United States |
| New York | New York | 10029 | United States |
| The Bronx | New York | 10461 | United States |
| Toledo | Ohio | 43623 | United States |
| Philadelphia | Pennsylvania | 19104 | United States |
| Greenville | South Carolina | 29605 | United States |
| Houston | Texas | 77030 | United States |
| Gyeyang-gu | Seoul | 06351 | South Korea |
| Seongdu | Seoul | 03722 | South Korea |
Participants received X4P-001 400 mg orally once daily (QD) with axitinib at 5 mg orally BID. |
| FG002 | Dose Escalation (Part A): X4P-001 600 mg QD With Axitinib | Participants received X4P-001 600 mg orally QD with axitinib at 5 mg orally BID. |
| FG003 | Dose Expansion (Part B): X4P-001 400 mg QD With Axitinib | Participants received X4P-001 400 mg orally QD with axitinib at 5 mg orally BID. |
| FG004 | Dose Escalation and Expansion (Part C): X4P-001 600 mg QD Monotherapy | Participants received X4P-001 600 mg orally QD. |
| Received at Least 1 Dose of Study Drug |
|
| COMPLETED |
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| NOT COMPLETED |
|
|
| Dose Expansion (Part B) |
|
|
| Dose Escalation and Expansion (Part C) |
|
|
Safety population included all participants who received at least 1 dose of study drug. As planned, Baseline Characteristics data collected per dose level not per study part.
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| ID | Title | Description |
|---|---|---|
| BG000 | X4P-001 200 mg BID With Axitinib | Participants received X4P-001 200 mg orally BID with axitinib at 5 mg orally BID in Part A. |
| BG001 | X4P-001 400 mg QD With Axitinib | Participants received X4P-001 400 mg orally QD with axitinib at 5 mg orally BID in either Part A or Part B. |
| BG002 | X4P-001 600 mg QD With Axitinib | Participants received X4P-001 600 mg orally QD with axitinib at 5 mg orally BID in Part A. |
| BG003 | X4P-001 600 mg QD Monotherapy | Participants received X4P-001 600 mg orally QD in Part C. |
| BG004 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Customized | Count of Participants | Participants |
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| Sex: Female, Male | Count of Participants | Participants |
| ||||||||||||||||
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||
| Race (NIH/OMB) | Count of Participants | Participants |
|
| 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 | Parts A, B, and C: Number of Participants With Treatment-emergent Adverse Events (TEAEs) | An AE was as any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug related. An SAE was an AE that met at least 1 of the following criteria: resulted in death, was life-threatening, required inpatient hospitalization or prolongation of existing hospitalization for the AE, persistent or significant disability/incapacity or substantial disruption of the ability to conduct normal life functions, congenital anomaly/birth defect (in the child of a participant who was exposed to the study drug), important medical event or reaction. Adverse events with onset after administration of the first dose of study drug up to 10 days after last dosing date were considered TEAEs. A summary of all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the 'Reported Adverse Events' Section. | Safety population included all participants who received at least 1 dose of study drug. | Posted | Count of Participants | Participants | From first dose of study drug up to 10 days after last dosing (maximum exposure: 52.1 months) |
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| Secondary | Parts A and B: Objective Response Rate (ORR), as Assessed Using Response Evaluation Criteria in Solid Tumors (RECIST) Version 1.1 | The ORR was defined as percentage of participants with best overall response of complete response (CR) or partial response (PR). CR: Disappearance of all target and non-target lesions. Any pathological lymph nodes (whether target or non-target) with a reduction in short axis to <10 millimeters (mm). PR: At least a 30% decrease in the sum of diameters of target lesions, taking as reference the baseline sum diameters. | Clinically evaluable population included all participants who received at least 1 dose of study medication, who had at least 1 valid post-baseline response (CR/PR/stable disease [SD]/progressive disease [PD]) and had no major protocol deviations which impacted the efficacy assessment. Due to dose-limiting toxicities (DLTs) experienced by participants, the sample size was too small for data to be collected for this Outcome Measure for the "X4P-001 600 mg QD With Axitinib" arm. | Posted | Number | 95% Confidence Interval | percentage of participants | From administration of first dose of study medication until disease progression, study completion or early termination (up to 80 weeks) |
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| Secondary | Parts A and B: Time to Objective Response, as Assessed Using RECIST v1.1 | Time to objective response was defined as time from first administration of combination regimen to first CR or PR whichever came first. CR: Disappearance of all target and non-target lesions. Any pathological lymph nodes (whether target or non-target) with a reduction in short axis to <10 mm. PR: At least a 30% decrease in the sum of diameters of target lesions, taking as reference the baseline sum diameters. | Clinically evaluable population included all participants who received at least 1 dose of study medication, who had at least 1 valid post-baseline response (CR/PR/SD/PD) and had no major protocol deviations which impacted the efficacy assessment. Due to DLTs experienced by participants, the sample size was too small for data to be collected for this Outcome Measure for the "X4P-001 600 mg QD With Axitinib" arm. | Posted | Median | 95% Confidence Interval | months | From administration of first dose of study medication until first appearance of CR or PR (up to 80 weeks) |
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| Secondary | Parts A and B: Duration of Objective Response (DOR), as Assessed Using RECIST v1.1 | The DOR was defined as the time from first CR or PR whichever came first until the time of disease progression or death by any cause. CR: Disappearance of all target and non-target lesions. Any pathological lymph nodes (whether target or non-target) with a reduction in short axis to <10 mm. PR: At least a 30% decrease in the sum of diameters of target lesions, taking as reference the baseline sum diameters. Disease progression: At least a 20% increase in the sum of diameters of target lesions, taking as reference the smallest sum on study. In addition to the relative increase of 20%, the sum also demonstrated an absolute increase of at least 5 mm. Unequivocal progression of existing non-target lesions. The appearance of 1 or more new lesions was also considered progression. | Clinically evaluable population included all participants who received at least 1 dose of study medication, who had at least 1 valid post-baseline response (CR/PR/SD/PD) and had no major protocol deviations which impacted efficacy assessment. 'Overall number of participants analyzed' = participants evaluable for this outcome measure. Due to DLTs experienced by participants, the sample size was too small for data to be collected for this Outcome Measure for "X4P-001 600 mg QD With Axitinib" arm. | Posted | Median | 95% Confidence Interval | months | Time from first CR or PR until the time of disease progression or death due to any cause (up to 80 weeks) |
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| Secondary | Parts A and B: Disease Control Rate (DCR), as Assessed Using RECIST v1.1 | The DCR was defined as the percentage of participants with best overall response of CR, PR or stable disease (SD). CR: Disappearance of all target and non-target lesions. Any pathological lymph nodes (whether target or non-target) with a reduction in short axis to <10 mm. PR: At least a 30% decrease in the sum of diameters of target lesions, taking as reference the baseline sum diameters. SD: Neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for progressive disease (PD), taking as reference the smallest sum diameters while on study. PD: At least a 20% increase in the sum of diameters of target lesions, taking as reference the smallest sum on study. In addition to the relative increase of 20%, the sum also demonstrated an absolute increase of at least 5 mm. Unequivocal progression of existing non-target lesions. The appearance of 1 or more new lesions was also considered progression. | Clinically evaluable population included all participants who received at least 1 dose of study medication, who had at least 1 valid post-baseline response (CR/PR/SD/PD) and had no major protocol deviations which impacted the efficacy assessment. Due to DLTs experienced by participants, the sample size was too small for data to be collected for this Outcome Measure for the "X4P-001 600 mg QD With Axitinib" arm. | Posted | Number | 95% Confidence Interval | percentage of participants | From administration of first dose of study medication until disease progression, study completion or early termination (up to 80 weeks) |
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| Secondary | Parts A and B: Time to Progression (TTP), as Assessed Using RECIST v1.1 | The TTP was defined as the time from first administration of combination regimen until PD. PD: At least a 20% increase in the sum of diameters of target lesions, taking as reference the smallest sum on study In addition to the relative increase of 20%, the sum also demonstrated an absolute increase of at least 5 mm. Unequivocal progression of existing non-target lesions or the appearance of 1 or more new lesions was also considered progression. | Clinically evaluable population included all participants who received at least 1 dose of study medication, who had at least 1 valid post-baseline response (CR/PR/SD/PD) and had no major protocol deviations which impacted the efficacy assessment. Due to DLTs experienced by participants, the sample size was too small for data to be collected for this Outcome Measure for the "X4P-001 600 mg QD With Axitinib" arm. | Posted | Median | 95% Confidence Interval | months | From administration of first dose of study medication until disease progression (up to 80 weeks) |
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| Secondary | Parts A and B: Progression Free Survival (PFS), as Assessed Using RECIST v1.1 | The PFS was defined as the time from first administration of combination regimen until disease progression or death from any cause. Disease progression: At least a 20% increase in the sum of diameters of target lesions, taking as reference the smallest sum on study. In addition to the relative increase of 20%, the sum also demonstrated an absolute increase of at least 5 mm. An unequivocal progression of existing non-target lesions or the appearance of 1 or more new lesions was also considered progression. | Clinically evaluable population included all participants who received at least 1 dose of study medication, who had at least 1 valid post-baseline response (CR/PR/SD/PD) and had no major protocol deviations which impacted the efficacy assessment. Due to DLTs experienced by participants, the sample size was too small for data to be collected for this Outcome Measure for the "X4P-001 600 mg QD With Axitinib" arm. | Posted | Median | 95% Confidence Interval | months | From administration of first dose of study medication until disease progression or death from any cause (up to 80 weeks) |
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| Secondary | Parts A and B: Maximum Plasma Concentration (Cmax) of X4P-001 | The pharmacokinetic (PK) population included all participants who had at least one evaluable concentration. Here 'Overall number of participants analyzed' = participants evaluable for this outcome measure. 'Number analyzed' = participants evaluable at specified timepoint. | Posted | Geometric Mean | Geometric Coefficient of Variation | nanograms (ng)/milliliter (mL) | Day 1 and Day 15 of Cycle 1 |
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| Secondary | Area Under the Curve From Time 0 to the Last Quantifiable Timepoint (AUC0-tlast) | The PK population included all participants who had at least one evaluable concentration. Here 'Overall number of participants analyzed' = participants evaluable for this outcome measure. 'Number analyzed' = participants evaluable at specified timepoint. | Posted | Geometric Mean | Geometric Coefficient of Variation | hours*ng/mL | Day 1 and Day 15 of Cycle 1 |
|
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From first dose of study drug up to 10 days after last dosing (maximum exposure: 52.1 months)
Safety population included all participants who received at least 1 dose of study drug. As planned, safety data collected per dose level not per study part.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | X4P-001 200 mg BID With Axitinib | Participants received X4P-001 200 mg orally BID with axitinib at 5 mg orally BID in Part A. | 1 | 3 | 1 | 3 | 3 | 3 |
| EG001 | X4P-001 400 mg QD With Axitinib | Participants received X4P-001 400 mg orally QD with axitinib at 5 mg orally BID in either Part A or Part B. | 2 | 62 | 26 | 62 | 62 | 62 |
| EG002 | X4P-001 600 mg QD With Axitinib | Participants received X4P-001 600 mg orally QD with axitinib at 5 mg orally BID in Part A. | 1 | 6 | 1 | 6 | 6 | 6 |
| EG003 | X4P-001 600 mg QD Monotherapy | Participants received X4P-001 600 mg orally QD in Part C. | 3 | 3 | 2 | 3 | 3 | 3 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Diarrhea | Gastrointestinal disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Abdominal distension | Gastrointestinal disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Ascites | Gastrointestinal disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Pleural effusion | Respiratory, thoracic and mediastinal disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Acquired tracheooesophageal fistula | Respiratory, thoracic and mediastinal disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Asthma | Respiratory, thoracic and mediastinal disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Pneumothorax | Respiratory, thoracic and mediastinal disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Acute kidney injury | Renal and urinary disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Chronic kidney disease | Renal and urinary disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Ureterolithiasis | Renal and urinary disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Urinary retention | Renal and urinary disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Pain | General disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Fatigue | General disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA 19.0 | Systematic Assessment |
| |
| Pyelonephritis acute | Infections and infestations | MedDRA 19.0 | Systematic Assessment |
| |
| Sepsis | Infections and infestations | MedDRA 19.0 | Systematic Assessment |
| |
| Femur fracture | Injury, poisoning and procedural complications | MedDRA 19.0 | Systematic Assessment |
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| Pelvic fracture | Injury, poisoning and procedural complications | MedDRA 19.0 | Systematic Assessment |
| |
| Spinal fracture | Injury, poisoning and procedural complications | MedDRA 19.0 | Systematic Assessment |
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| Hyperkalaemia | Metabolism and nutrition disorders | MedDRA 19.0 | Systematic Assessment |
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| Hypercalcaemia | Metabolism and nutrition disorders | MedDRA 19.0 | Systematic Assessment |
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| Blood creatinine increased | Investigations | MedDRA 19.0 | Systematic Assessment |
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| Hepatic enzyme increased | Investigations | MedDRA 19.0 | Systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA 19.0 | Systematic Assessment |
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| Back pain | Musculoskeletal and connective tissue disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Spinal cord compression | Nervous system disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Hypertension | Vascular disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Ventricular tachycardia | Cardiac disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Cholestatic liver injury | Hepatobiliary disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Metastases to central nervous system | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 19.0 | Systematic Assessment |
| |
| Device failure | Product Issues | MedDRA 19.0 | Systematic Assessment |
| |
| Hyperglycemia | Metabolism and nutrition disorders | MedDRA 19.0 | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Diarrhoea | Gastrointestinal disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Dyspepsia | Gastrointestinal disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Stomatitis | Gastrointestinal disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Dry mouth | Gastrointestinal disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Gastrooesophageal reflux disease | Gastrointestinal disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Abdominal pain upper | Gastrointestinal disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Dysphagia | Gastrointestinal disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Abdominal distension | Gastrointestinal disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Abdominal discomfort | Gastrointestinal disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Aphthous ulcer | Gastrointestinal disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Flatulence | Gastrointestinal disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Glossodynia | Gastrointestinal disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Oral pain | Gastrointestinal disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Abdominal pain lower | Gastrointestinal disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Anal fistula | Gastrointestinal disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Gastritis | Gastrointestinal disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Gingival bleeding | Gastrointestinal disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Glossitis | Gastrointestinal disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Haematemesis | Gastrointestinal disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Haematochezia | Gastrointestinal disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Hypoaesthesia oral | Gastrointestinal disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Lip pain | Gastrointestinal disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Oesophagitis | Gastrointestinal disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Oral dysaesthesia | Gastrointestinal disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Oral papule | Gastrointestinal disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Proctalgia | Gastrointestinal disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Toothache | Gastrointestinal disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Umbilical hernia | Gastrointestinal disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Weight decreased | Investigations | MedDRA 19.0 | Systematic Assessment |
| |
| Blood creatinine increased | Investigations | MedDRA 19.0 | Systematic Assessment |
| |
| Alanine aminotransferase increased | Investigations | MedDRA 19.0 | Systematic Assessment |
| |
| Aspartate aminotransferase increased | Investigations | MedDRA 19.0 | Systematic Assessment |
| |
| Blood alkaline phosphatase increased | Investigations | MedDRA 19.0 | Systematic Assessment |
| |
| Lipase increased | Investigations | MedDRA 19.0 | Systematic Assessment |
| |
| White blood cell count increased | Investigations | MedDRA 19.0 | Systematic Assessment |
| |
| Blood thyroid stimulating hormone increased | Investigations | MedDRA 19.0 | Systematic Assessment |
| |
| Lymphocyte count decreased | Investigations | MedDRA 19.0 | Systematic Assessment |
| |
| Weight increased | Investigations | MedDRA 19.0 | Systematic Assessment |
| |
| Amylase increased | Investigations | MedDRA 19.0 | Systematic Assessment |
| |
| Blood creatine phosphokinase increased | Investigations | MedDRA 19.0 | Systematic Assessment |
| |
| Blood fibrinogen increased | Investigations | MedDRA 19.0 | Systematic Assessment |
| |
| Blood uric acid increased | Investigations | MedDRA 19.0 | Systematic Assessment |
| |
| Blood bilirubin increased | Investigations | MedDRA 19.0 | Systematic Assessment |
| |
| Blood cholesterol increased | Investigations | MedDRA 19.0 | Systematic Assessment |
| |
| Electrocardiogram QT prolonged | Investigations | MedDRA 19.0 | Systematic Assessment |
| |
| Neutrophil count increased | Investigations | MedDRA 19.0 | Systematic Assessment |
| |
| Hepatic enzyme increased | Investigations | MedDRA 19.0 | Systematic Assessment |
| |
| Tri-iodothyronine decreased | Investigations | MedDRA 19.0 | Systematic Assessment |
| |
| Thyroid function test abnormal | Investigations | MedDRA 19.0 | Systematic Assessment |
| |
| Activated partial thromboplastin time prolonged | Investigations | MedDRA 19.0 | Systematic Assessment |
| |
| Blood albumin increased | Investigations | MedDRA 19.0 | Systematic Assessment |
| |
| Blood bicarbonate decreased | Investigations | MedDRA 19.0 | Systematic Assessment |
| |
| Blood potassium decreased | Investigations | MedDRA 19.0 | Systematic Assessment |
| |
| Blood urea increased | Investigations | MedDRA 19.0 | Systematic Assessment |
| |
| Creatinine renal clearance decreased | Investigations | MedDRA 19.0 | Systematic Assessment |
| |
| Electrocardiogram abnormal | Investigations | MedDRA 19.0 | Systematic Assessment |
| |
| Haemoglobin increased | Investigations | MedDRA 19.0 | Systematic Assessment |
| |
| Lymphocyte count increased | Investigations | MedDRA 19.0 | Systematic Assessment |
| |
| Neutrophil count decreased | Investigations | MedDRA 19.0 | Systematic Assessment |
| |
| Platelet count decreased | Investigations | MedDRA 19.0 | Systematic Assessment |
| |
| Troponin increased | Investigations | MedDRA 19.0 | Systematic Assessment |
| |
| Vitamin d decreased | Investigations | MedDRA 19.0 | Systematic Assessment |
| |
| Fatigue | General disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Chills | General disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Non-cardiac chest pain | General disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Influenza like illness | General disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Asthenia | General disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Oedema peripheral | General disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Mucosal inflammation | General disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Chest pain | General disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Gait disturbance | General disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Pain | General disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Chest discomfort | General disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Facial pain | General disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Decreased activity | General disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Feeling abnormal | General disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Malaise | General disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Peripheral swelling | General disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Suprapubic pain | General disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Temperature intolerance | General disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Decreased appetite | Metabolism and nutrition disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Hyperkalaemia | Metabolism and nutrition disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Dehydration | Metabolism and nutrition disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Abnormal loss of weight | Metabolism and nutrition disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Hyponatraemia | Metabolism and nutrition disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Hyperuricaemia | Metabolism and nutrition disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Hyperglycaemia | Metabolism and nutrition disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Hypomagnesaemia | Metabolism and nutrition disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Hypoalbuminaemia | Metabolism and nutrition disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Hypercalcaemia | Metabolism and nutrition disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Hypophosphataemia | Metabolism and nutrition disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Hypoglycaemia | Metabolism and nutrition disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Acidosis | Metabolism and nutrition disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Gout | Metabolism and nutrition disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Hypertriglyceridaemia | Metabolism and nutrition disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Hypocalcaemia | Metabolism and nutrition disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Hypokalaemia | Metabolism and nutrition disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Dysphonia | Respiratory, thoracic and mediastinal disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Epistaxis | Respiratory, thoracic and mediastinal disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Nasal congestion | Respiratory, thoracic and mediastinal disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Nasal dryness | Respiratory, thoracic and mediastinal disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Productive cough | Respiratory, thoracic and mediastinal disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Haemoptysis | Respiratory, thoracic and mediastinal disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Pleural effusion | Respiratory, thoracic and mediastinal disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Sinus pain | Respiratory, thoracic and mediastinal disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Aspiration | Respiratory, thoracic and mediastinal disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Dyspnoea exertional | Respiratory, thoracic and mediastinal disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Paranasal sinus discomfort | Respiratory, thoracic and mediastinal disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Pulmonary embolism | Respiratory, thoracic and mediastinal disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Rhinalgia | Respiratory, thoracic and mediastinal disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Rhinitis allergic | Respiratory, thoracic and mediastinal disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Rhinorrhoea | Respiratory, thoracic and mediastinal disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Sinus congestion | Respiratory, thoracic and mediastinal disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Throat irritation | Respiratory, thoracic and mediastinal disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Upper-airway cough syndrome | Respiratory, thoracic and mediastinal disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Wheezing | Respiratory, thoracic and mediastinal disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Myalgia | Musculoskeletal and connective tissue disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Muscular weakness | Musculoskeletal and connective tissue disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Musculoskeletal chest pain | Musculoskeletal and connective tissue disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Flank pain | Musculoskeletal and connective tissue disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Neck pain | Musculoskeletal and connective tissue disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Bone pain | Musculoskeletal and connective tissue disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Arthritis | Musculoskeletal and connective tissue disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Joint swelling | Musculoskeletal and connective tissue disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Muscle spasms | Musculoskeletal and connective tissue disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Musculoskeletal pain | Musculoskeletal and connective tissue disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Pain in jaw | Musculoskeletal and connective tissue disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Bursitis | Musculoskeletal and connective tissue disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Joint stiffness | Musculoskeletal and connective tissue disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Muscle twitching | Musculoskeletal and connective tissue disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Dizziness | Nervous system disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Dysgeusia | Nervous system disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Memory impairment | Nervous system disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Hypoaesthesia | Nervous system disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Paraesthesia | Nervous system disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Cognitive disorder | Nervous system disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Presyncope | Nervous system disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Sciatica | Nervous system disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Balance disorder | Nervous system disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Cerebrospinal fluid leakage | Nervous system disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Hyperaesthesia | Nervous system disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Lethargy | Nervous system disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Muscle spasticity | Nervous system disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Neuropathy peripheral | Nervous system disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Peripheral sensory neuropathy | Nervous system disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Somnolence | Nervous system disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Spinal cord compression | Nervous system disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Taste disorder | Nervous system disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Hypertension | Vascular disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Hot flush | Vascular disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Hypotension | Vascular disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Deep vein thrombosis | Vascular disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Palmar-plantar erythrodysaesthesia syndrome | Skin and subcutaneous tissue disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Rash | Skin and subcutaneous tissue disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Dry skin | Skin and subcutaneous tissue disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Urticaria | Skin and subcutaneous tissue disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Rash pruritic | Skin and subcutaneous tissue disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Pruritus | Skin and subcutaneous tissue disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Skin lesion | Skin and subcutaneous tissue disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Hyperkeratosis | Skin and subcutaneous tissue disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Rash maculo-papular | Skin and subcutaneous tissue disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Blister | Skin and subcutaneous tissue disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Alopecia | Skin and subcutaneous tissue disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Dermatitis acneiform | Skin and subcutaneous tissue disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Dermatitis contact | Skin and subcutaneous tissue disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Erythema | Skin and subcutaneous tissue disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Hair colour changes | Skin and subcutaneous tissue disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Hyperhidrosis | Skin and subcutaneous tissue disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Petechiae | Skin and subcutaneous tissue disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Photosensitivity reaction | Skin and subcutaneous tissue disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Rash follicular | Skin and subcutaneous tissue disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Skin exfoliation | Skin and subcutaneous tissue disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Dry eye | Eye disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Vision blurred | Eye disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Eye pain | Eye disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Cataract | Eye disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Eye pruritus | Eye disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Lacrimation increased | Eye disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Age-related macular degeneration | Eye disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Eye irritation | Eye disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Macular oedema | Eye disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Ocular hyperaemia | Eye disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Retinal haemorrhage | Eye disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Retinal pigmentation | Eye disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Retinal vein occlusion | Eye disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Visual impairment 0 | Eye disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA 19.0 | Systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA 19.0 | Systematic Assessment |
| |
| Sinusitis | Infections and infestations | MedDRA 19.0 | Systematic Assessment |
| |
| Herpes zoster | Infections and infestations | MedDRA 19.0 | Systematic Assessment |
| |
| Influenza | Infections and infestations | MedDRA 19.0 | Systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA 19.0 | Systematic Assessment |
| |
| Skin infection | Infections and infestations | MedDRA 19.0 | Systematic Assessment |
| |
| Candida infection | Infections and infestations | MedDRA 19.0 | Systematic Assessment |
| |
| Cellulitis | Infections and infestations | MedDRA 19.0 | Systematic Assessment |
| |
| Conjunctivitis | Infections and infestations | MedDRA 19.0 | Systematic Assessment |
| |
| Dermatophytosis of nail | Infections and infestations | MedDRA 19.0 | Systematic Assessment |
| |
| Furuncle | Infections and infestations | MedDRA 19.0 | Systematic Assessment |
| |
| Laryngitis | Infections and infestations | MedDRA 19.0 | Systematic Assessment |
| |
| Localised infection | Infections and infestations | MedDRA 19.0 | Systematic Assessment |
| |
| Otitis media | Infections and infestations | MedDRA 19.0 | Systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA 19.0 | Systematic Assessment |
| |
| Pyuria | Infections and infestations | MedDRA 19.0 | Systematic Assessment |
| |
| Staphylococcal infection | Infections and infestations | MedDRA 19.0 | Systematic Assessment |
| |
| Proteinuria | Renal and urinary disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Acute kidney injury | Renal and urinary disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Haematuria | Renal and urinary disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Azotaemia | Renal and urinary disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Pollakiuria | Renal and urinary disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Urinary tract pain | Renal and urinary disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Nephrolithiasis | Renal and urinary disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Chronic kidney disease | Renal and urinary disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Micturition urgency | Renal and urinary disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Stress urinary incontinence | Renal and urinary disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Urge incontinence | Renal and urinary disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Urinary incontinence | Renal and urinary disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Hypothyroidism | Endocrine disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Hyperthyroidism | Endocrine disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Anaemia | Blood and lymphatic system disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Lymph node pain | Blood and lymphatic system disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Lymphadenopathy | Blood and lymphatic system disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Lymphopenia | Blood and lymphatic system disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Polycythaemia | Blood and lymphatic system disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Thrombocytopenia | Blood and lymphatic system disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Insomnia | Psychiatric disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Anxiety | Psychiatric disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Depression | Psychiatric disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Confusional state | Psychiatric disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Hallucination | Psychiatric disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Stress | Psychiatric disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Fall | Injury, poisoning and procedural complications | MedDRA 19.0 | Systematic Assessment |
| |
| Contusion | Injury, poisoning and procedural complications | MedDRA 19.0 | Systematic Assessment |
| |
| Animal scratch | Injury, poisoning and procedural complications | MedDRA 19.0 | Systematic Assessment |
| |
| Ankle fracture | Injury, poisoning and procedural complications | MedDRA 19.0 | Systematic Assessment |
| |
| Corneal abrasion | Injury, poisoning and procedural complications | MedDRA 19.0 | Systematic Assessment |
| |
| Peroneal nerve injury | Injury, poisoning and procedural complications | MedDRA 19.0 | Systematic Assessment |
| |
| Procedural pain | Injury, poisoning and procedural complications | MedDRA 19.0 | Systematic Assessment |
| |
| Scapula fracture | Injury, poisoning and procedural complications | MedDRA 19.0 | Systematic Assessment |
| |
| Skin abrasion | Injury, poisoning and procedural complications | MedDRA 19.0 | Systematic Assessment |
| |
| Skin laceration | Injury, poisoning and procedural complications | MedDRA 19.0 | Systematic Assessment |
| |
| Sunburn | Injury, poisoning and procedural complications | MedDRA 19.0 | Systematic Assessment |
| |
| Traumatic haematoma | Injury, poisoning and procedural complications | MedDRA 19.0 | Systematic Assessment |
| |
| Pelvic pain | Reproductive system and breast disorders | MedDRA 19.0 | Systematic Assessment | This is a sex-specific AE. Only female participants were at risk. |
|
| Benign prostatic hyperplasia | Reproductive system and breast disorders | MedDRA 19.0 | Systematic Assessment | This is a sex-specific AE. Only male participants were at risk. |
|
| Gynaecomastia | Reproductive system and breast disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Scrotal oedema | Reproductive system and breast disorders | MedDRA 19.0 | Systematic Assessment | This is a sex-specific AE. Only male participants were at risk. |
|
| Vulvovaginal pruritus | Reproductive system and breast disorders | MedDRA 19.0 | Systematic Assessment | This is a sex-specific AE. Only female participants were at risk. |
|
| Cardiac disorder | Cardiac disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Sinus bradycardia | Cardiac disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Sinus tachycardia | Cardiac disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Ear pain | Ear and labyrinth disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Hypoacusis | Ear and labyrinth disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Tinnitus | Ear and labyrinth disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Hepatobiliary disorders | Hepatobiliary disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Biliary colic | Hepatobiliary disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Hepatic vascular thrombosis | Hepatobiliary disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Hypersensitivity | Immune system disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Tooth extraction | Surgical and medical procedures | MedDRA 19.0 | Systematic Assessment |
| |
| Electrocardiogram Q wave abnormal | Investigations | MedDRA 19.0 | Systematic Assessment |
| |
| Pharyngitis | Infections and infestations | MedDRA 19.0 | Systematic Assessment |
| |
| Diabetes Mellitus | Metabolism and nutrition disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Localised Oedema | General disorders | MedDRA 19.0 | Systematic Assessment |
|
Not provided
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Chief Medical Officer | X4 Pharmaceuticals | (857) 529-8300 | patientinfo@x4pharma.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Jun 21, 2022 | Jul 23, 2024 | SAP_001.pdf |
| ID | Term |
|---|---|
| D002292 | Carcinoma, Renal Cell |
| ID | Term |
|---|---|
| D000230 | Adenocarcinoma |
| D002277 | Carcinoma |
| D009375 | Neoplasms, Glandular and Epithelial |
| D009370 | Neoplasms by Histologic Type |
| D009369 | Neoplasms |
| D007680 | Kidney Neoplasms |
| D014571 | Urologic Neoplasms |
| D014565 | Urogenital Neoplasms |
| D009371 | Neoplasms by Site |
| D052776 | Female Urogenital Diseases |
| D005261 | Female Urogenital Diseases and Pregnancy Complications |
| D000091642 | Urogenital Diseases |
| D007674 | Kidney Diseases |
| D014570 | Urologic Diseases |
| D052801 | Male Urogenital Diseases |
Not provided
Not provided
| ID | Term |
|---|---|
| C494414 | mavorixafor |
| D000077784 | Axitinib |
| ID | Term |
|---|---|
| D001549 | Benzamides |
| D000577 | Amides |
| D009930 | Organic Chemicals |
| D001565 | Benzoates |
| D000146 | Acids, Carbocyclic |
| D002264 | Carboxylic Acids |
| D001555 | Benzene Derivatives |
| D006841 | Hydrocarbons, Aromatic |
| D006844 | Hydrocarbons, Cyclic |
| D006838 | Hydrocarbons |
| D007191 | Indazoles |
| D011720 | Pyrazoles |
| D001393 | Azoles |
| D006573 | Heterocyclic Compounds, 1-Ring |
| D006571 | Heterocyclic Compounds |
| D006574 | Heterocyclic Compounds, 2-Ring |
| D000072471 | Heterocyclic Compounds, Fused-Ring |
Not provided
Not provided
| Clinical Deterioration |
|
| Disease Progression |
|
| Withdrawal by Subject |
|
| DLT |
|
| ≥65 Years |
|
| Male |
|
| Not Hispanic or Latino |
|
| Unknown or Not Reported |
|
| Asian |
|
| Native Hawaiian or Other Pacific Islander |
|
| Black or African American |
|
| White |
|
| More than one race |
|
| Unknown or Not Reported |
|
|
|
| Units | Counts |
|---|---|
| Participants |
|
|
| OG001 | X4P-001 400 mg QD With Axitinib | Participants received X4P-001 400 mg orally QD with axitinib at 5 mg orally BID in either Part A or Part B. |
|
|
| OG001 | X4P-001 400 mg QD With Axitinib | Participants received X4P-001 400 mg orally QD with axitinib at 5 mg orally BID in either Part A or Part B. |
|
|
| Units | Counts |
|---|---|
| Participants |
|
|
|
|
|
|
|
|