A Phase 1/2 interventional study of X4P-001 and Nivolumab in Clear Cell Renal Cell Carcinoma, sponsored by X4 Pharmaceuticals. Terminated at 4 sites in United States. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2022-12-29.
Sponsored by X4 Pharmaceuticals · Phase 1/2, Interventional, and Treatment
The purpose of this study is to determine if the combination of X4P-001 plus nivolumab is safe and tolerable. Secondly, the study will investigate if adding X4P-001 to nivolumab treatment has an effect on the body and the cancer tumor, in participants receiving nivolumab but not exhibiting a radiological response.
6,741 studies on the registry are indexed under Carcinoma; 1,161 are open to participants now.
This study's enrollment of 9 is below the median of 45 across 5,170 interventional studies indexed under Carcinoma.
Browse Carcinoma studies →X4 Pharmaceuticals is the lead sponsor of 12 studies on the registry; 1 is open to participants now.
Of its 10 completed or terminated interventional studies of FDA-regulated products, 3 (30%) have results posted.
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Exclusion Criteria:
Participants will receive X4P-001 400 milligrams (mg) (as 4 capsules of 100 mg each) orally once daily in combination with nivolumab 240 mg intravenous (IV) infusion (over 60 minutes) every 2 weeks. Study medication will be administered in 28-day cycles and will continue until treatment-limiting toxicity or disease progression.
Drug: X4P-001 · Drug: Nivolumab
X4P-001 will be administered as per the dose and schedule specified in the arm.
Nivolumab will be administered as per the dose and schedule specified in the arm.
Also known as: Opdivo
Number of Participants With Treatment-Emergent Adverse Events (TEAEs)
An adverse event (AE) was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. Drug-related TEAEs: TEAEs with possible, probable, definite, or missing relationship to study medication (X4P-001 or Nivolumab). Serious AEs: death, a life-threatening AE, inpatient hospitalization or prolongation of existing hospitalization, persistent or significant disability or incapacity, a congenital anomaly or birth defect, or an important medical event that jeopardized participant and required medical intervention to prevent 1 of the outcomes listed in this definition. TEAEs were defined as events occurring on or after the first dose of study drug through 30 days after the last dose. Any TEAEs included both serious and non-serious TEAEs. A summary of other non-serious AEs and all serious AEs, regardless of causality, is located in Reported AE section.
Time frame: From administration of first dose of study medication (Day 1) up to 30 days after last dose (up to 16 months)
Maximum Observed Plasma Concentration (Cmax) of X4P-001
Samples were to be analyzed for X4P-001 concentration using reversed-phase high performance liquid chromatography (RP-HPLC) with tandem mass spectrometry (MS) detection.
Time frame: Predose (-30 minutes) on Day 1 of Cycle 1; predose (-10 minutes) on Days 8, 15, and 22 of Cycle 1, and Day 1 of Cycle 3; postdose at 60 and 90 minutes, and 2, 3 4, and 8 hours on Day 22 of Cycle 1
Area Under the Plasma Concentration Versus Time Curve (AUC) of X4P-001
Samples were to be analyzed for X4P-001 concentration using RP-HPLC with tandem MS detection.
Time frame: Predose (-30 minutes) on Day 1 of Cycle 1; predose (-10 minutes) on Days 8, 15, and 22 of Cycle 1, and Day 1 of Cycle 3; postdose at 60 and 90 minutes, and 2, 3 4, and 8 hours on Day 22 of Cycle 1
Minimum Plasma Concentration (Cmin) of X4P-001
Samples were to be analyzed for X4P-001 concentration using RP-HPLC with tandem MS detection.
Time frame: Predose (-30 minutes) on Day 1 of Cycle 1; predose (-10 minutes) on Days 8, 15, and 22 of Cycle 1, and Day 1 of Cycle 3; postdose at 60 and 90 minutes, and 2, 3 4, and 8 hours on Day 22 of Cycle 1
Time to Reach Cmax (Tmax) of X4P-001
Samples were to be analyzed for X4P-001 concentration using RP-HPLC with tandem MS detection.
Time frame: Predose (-30 minutes) on Day 1 of Cycle 1; predose (-10 minutes) on Days 8, 15, and 22 of Cycle 1, and Day 1 of Cycle 3; postdose at 60 and 90 minutes, and 2, 3 4, and 8 hours on Day 22 of Cycle 1
Objective Response Rate (ORR): Percentage of Participants With Objective Response, Evaluated Using Response Evaluation Criteria in Solid Tumors (RECIST) Version 1.1
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 less than (\<) 10 millimeters (mm). PR: At least a 30 percent (%) decrease in the sum of diameters of target lesions, taking as reference the baseline sum diameters.
Time frame: From administration of first dose of study medication (Day 1) until disease progression, study completion or early termination (up to 15 months)
Duration of Objective Response (DOR), Evaluated Using RECIST Version 1.1
DOR was defined as the time from first CR or PR whichever comes first until the time of disease progression by RECIST v1.1 or death due to 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 frame: Time from first CR or PR until the time of disease progression or death due to any cause (up to 15 months)
Time to Objective Response, Evaluated Using RECIST Version 1.1
Time to objective response was defined as time from first administration of combination regimen to first CR or PR whichever comes 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.
Time frame: From administration of first dose of study medication (Day 1) until first appearance of CR or PR (up to 15 months)
Disease Control Rate: Percentage of Participants With CR or PR or Stable Disease (SD), Evaluated Using RECIST Version 1.1
Disease control rate was defined as percentage of participants with best overall response of CR or PR or 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.
Time frame: From administration of first dose of study medication (Day 1) until disease progression, study completion or early termination (up to 15 months)
Progression Free Survival (PFS), Evaluated Using RECIST Version 1.1
PFS was defined as the time from first administration of study medication until objective tumor progression or death from any cause. Tumor 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.
Time frame: From administration of first dose of study medication (Day 1) until disease progression or death from any cause (up to 15 months)
Time to Progression (TTP), Evaluated Using RECIST Version 1.1
TTP was defined as the time from first administration of study medication until objective tumor progression. Tumor 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 or the appearance of 1 or more new lesions was also considered progression.
Time frame: From administration of first dose of study medication (Day 1) until disease progression (up to 15 months)
| Milestone | X4P-001 Plus Nivolumab |
|---|---|
| Started | 9 |
| Received at least 1 dose of study medication | 9 |
| Completed | 0 |
| Not completed | 9 |
| Withdrew: Adverse event | 4 |
| Withdrew: Clinical deterioration | 1 |
| Withdrew: Disease progression | 3 |
| Withdrew: Study termination | 1 |
An adverse event (AE) was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. Drug-related TEAEs: TEAEs with possible, probable, definite, or missing relationship to study medication (X4P-001 or Nivolumab). Serious AEs: death, a life-threatening AE, inpatient hospitalization or prolongation of existing hospitalization, persistent or significant disability or incapacity, a congenital anomaly or birth defect, or an important medical event that jeopardized participant and required medical intervention to prevent 1 of the outcomes listed in this definition. TEAEs were defined as events occurring on or after the first dose of study drug through 30 days after the last dose. Any TEAEs included both serious and non-serious TEAEs. A summary of other non-serious AEs and all serious AEs, regardless of causality, is located in Reported AE section.
| Participants | X4P-001 Plus Nivolumab |
|---|---|
| Any TEAEs | 9 |
| Serious TEAEs | 2 |
| X4P-001-Related TEAEs | 9 |
| Nivolumab-Related TEAEs | 9 |
Samples were to be analyzed for X4P-001 concentration using reversed-phase high performance liquid chromatography (RP-HPLC) with tandem mass spectrometry (MS) detection.
No measurements were reported for this outcome.
Samples were to be analyzed for X4P-001 concentration using RP-HPLC with tandem MS detection.
No measurements were reported for this outcome.
Samples were to be analyzed for X4P-001 concentration using RP-HPLC with tandem MS detection.
No measurements were reported for this outcome.
Samples were to be analyzed for X4P-001 concentration using RP-HPLC with tandem MS detection.
No measurements were reported for this outcome.
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 less than (\<) 10 millimeters (mm). PR: At least a 30 percent (%) decrease in the sum of diameters of target lesions, taking as reference the baseline sum diameters.
| percentage of participants | X4P-001 Plus Nivolumab |
|---|---|
| Objective Response Rate (ORR): Percentage of Participants With Objective Response, Evaluated Using Response Evaluation Criteria in Solid Tumors (RECIST) Version 1.1 | 11.1 (0.3 to 48.2) |
DOR was defined as the time from first CR or PR whichever comes first until the time of disease progression by RECIST v1.1 or death due to 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.
| months | X4P-001 Plus Nivolumab |
|---|---|
| Duration of Objective Response (DOR), Evaluated Using RECIST Version 1.1 | NA (NA to NA) |
Time to objective response was defined as time from first administration of combination regimen to first CR or PR whichever comes 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.
| months | X4P-001 Plus Nivolumab |
|---|---|
| Time to Objective Response, Evaluated Using RECIST Version 1.1 | NA (NA to NA) |
Disease control rate was defined as percentage of participants with best overall response of CR or PR or 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.
| percentage of participants | X4P-001 Plus Nivolumab |
|---|---|
| Disease Control Rate: Percentage of Participants With CR or PR or Stable Disease (SD), Evaluated Using RECIST Version 1.1 | 88.9 (51.8 to 99.7) |
PFS was defined as the time from first administration of study medication until objective tumor progression or death from any cause. Tumor 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.
| months | X4P-001 Plus Nivolumab |
|---|---|
| Progression Free Survival (PFS), Evaluated Using RECIST Version 1.1 | NA (NA to NA) |
TTP was defined as the time from first administration of study medication until objective tumor progression. Tumor 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 or the appearance of 1 or more new lesions was also considered progression.
| months | X4P-001 Plus Nivolumab |
|---|---|
| Time to Progression (TTP), Evaluated Using RECIST Version 1.1 | NA (NA to NA) |
Collected over From administration of first dose of study medication (Day 1) up to 30 days after last dose (up to 16 months). Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| X4P-001 Plus Nivolumab | 0/9 (0%) | 2/9 (22.2%) | 9/9 (100%) |
| Event | X4P-001 Plus Nivolumab |
|---|---|
| Alanine aminotransferase increasedInvestigations | 1/9 |
| Aspartate aminotransferase increasedInvestigations | 1/9 |
| Mucosal inflammationGeneral disorders | 1/9 |
| Autoimmune hepatitisHepatobiliary disorders | 1/9 |
| Rash maculo-papularSkin and subcutaneous tissue disorders | 1/9 |
| EmbolismVascular disorders | 1/9 |
| Event | X4P-001 Plus Nivolumab |
|---|---|
| DiarrhoeaGastrointestinal disorders | 6/9 |
| Nasal congestionRespiratory, thoracic and mediastinal disorders | 5/9 |
| Dry eyeEye disorders | 4/9 |
| CoughRespiratory, thoracic and mediastinal disorders | 4/9 |
| FatigueGeneral disorders | 4/9 |
| HeadacheNervous system disorders | 4/9 |
| NauseaGastrointestinal disorders | 3/9 |
| Weight decreasedInvestigations | 3/9 |
| PruritusSkin and subcutaneous tissue disorders | 3/9 |
| ArthralgiaMusculoskeletal and connective tissue disorders | 3/9 |
Safety population included all participants who received at least 1 dose of study medication.
| Age, Continuous(years) | X4P-001 Plus Nivolumab |
|---|---|
| Mean | 62.7 ± 8.85 |
| Sex: Female, Male(Participants) | X4P-001 Plus Nivolumab |
|---|---|
| Female | 1 |
| Male | 8 |
| Ethnicity (NIH/OMB)(Participants) | X4P-001 Plus Nivolumab |
|---|---|
| Hispanic or Latino | 0 |
| Not Hispanic or Latino | 8 |
| Unknown or Not Reported | 1 |
| Race (NIH/OMB)(Participants) | X4P-001 Plus Nivolumab |
|---|---|
| American Indian or Alaska Native | 0 |
| Asian | 0 |
| Native Hawaiian or Other Pacific Islander | 0 |
| Black or African American | 0 |
| White | 9 |
| More than one race | 0 |
| Unknown or Not Reported | 0 |
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