Neoplasms

All Conditions

Urinary Tract, Sexual Organs, and Pregnancy Conditions

Rare Diseases

Carcinoma

Carcinoma, Renal Cell

Neoplasms, Glandular and Epithelial

Neoplasms by Histologic Type

Adenocarcinoma

Kidney Neoplasms

Urologic Neoplasms

Urogenital Neoplasms

Urogenital Diseases

Female Urogenital Diseases

Pregnancy Complications

Female Urogenital Diseases and Pregnancy Complications

Kidney Diseases

Urologic Diseases

Male Urogenital Diseases

Renal Cell Carcinoma

Clear Cell Renal Cell Carcinoma

Antineoplastic Agents

All Drugs and Chemicals

Nivolumab

Antineoplastic Agents, Immunological

Immune Checkpoint Inhibitors

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.

X4P-001 will be administered as per the dose and schedule specified in the arm.

X4P-001

Nivolumab will be administered as per the dose and schedule specified in the arm.

Chief Medical Officer

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.

Addition of X4P-001 to Nivolumab Treatment in Participants With Renal Cell Carcinoma

A Phase 1B/2A Trial Adding X4P-001 in Patients Receiving Nivolumab for Treatment of Advanced Clear Cell Renal Cell Carcinoma

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.

Samples were to be analyzed for X4P-001 concentration using reversed-phase high performance liquid chromatography (RP-HPLC) with tandem mass spectrometry (MS) detection.

Samples were to be analyzed for X4P-001 concentration using RP-HPLC with tandem MS detection.

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.

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 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.

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.

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.

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.

X4 Pharmaceuticals

Safety population included all participants who received at least 1 dose of study medication.

Participants received X4P-001 400 mg (as 4 capsules of 100 mg each) orally once daily in combination with nivolumab 240 mg IV infusion (over 60 minutes) every 2 weeks. Study treatment was administered in 28-day cycles and continued until treatment-limiting toxicity or disease progression.

X4P-001 Plus Nivolumab

Participants received X4P-001 400 mg (as 4 capsules of 100 mg each) orally once daily in combination with nivolumab 240 mg IV infusion (over 60 minutes) every 2 weeks. Study medication was administered in 28-day cycles and continued until treatment-limiting toxicity or disease progression.

Age, Continuous

Sex: Female, Male

Ethnicity (NIH/OMB)

Race (NIH/OMB)

Due to low enrollment, the study was terminated early. Data for some pre-registered endpoints were therefore not collected or analysed and could not be reported.

Any TEAEs

Serious TEAEs

X4P-001-Related TEAEs

Nivolumab-Related TEAEs

Number of Participants With Treatment-Emergent Adverse Events (TEAEs)

The study was terminated and the pharmacokinetic (PK) samples collected could not be analyzed due to insufficient data available to assess the outcome measure as per the prespecified method of analysis.

Maximum Observed Plasma Concentration (Cmax) of X4P-001

The study was terminated and the PK samples collected could not be analyzed due to insufficient data available to assess the outcome measure as per the prespecified method of analysis.

Area Under the Plasma Concentration Versus Time Curve (AUC) of X4P-001

Minimum Plasma Concentration (Cmin) of X4P-001

The study was terminated and the PK samples collected could not be analyzed due to insufficient data available to as per the prespecified method of analysis.

Time to Reach Cmax (Tmax) of X4P-001

Intent-to-treat (ITT) population included all participants who received at least 1 dose of study medication.

Objective Response Rate (ORR): Percentage of Participants With Objective Response, Evaluated Using Response Evaluation Criteria in Solid Tumors (RECIST) Version 1.1

ITT population included all participants who received at least 1 dose of study medication.

Duration of Objective Response (DOR), Evaluated Using RECIST Version 1.1

Time to Objective Response, Evaluated Using RECIST Version 1.1

Disease Control Rate: Percentage of Participants With CR or PR or Stable Disease (SD), Evaluated Using RECIST Version 1.1

Progression Free Survival (PFS), Evaluated Using RECIST Version 1.1

Time to Progression (TTP), Evaluated Using RECIST Version 1.1

Participants received 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 was administered in 28-day cycles and continued until treatment-limiting toxicity or disease progression.

Spots Global Cancer Trial Database for Addition of X4P-001 to Nivolumab Treatment in Participants With Renal Cell Carcinoma

Trial Identification

Conditions

Interventions

Study Description

Keywords

Eligibility

Locations

Contact Details

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