Trial Outcomes & Findings for Addition of X4P-001 to Nivolumab Treatment in Participants With Renal Cell Carcinoma (NCT NCT02923531)

Last Updated: 2022-12-29

Results Overview

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.

Recruitment status

TERMINATED

Study phase

PHASE1/PHASE2

Target enrollment

9 participants

Primary outcome timeframe

From administration of first dose of study medication (Day 1) up to 30 days after last dose (up to 16 months)

Results posted on

2022-12-29

Participant Flow

Participant milestones

Participant milestones
Measure	X4P-001 Plus Nivolumab 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.
Overall Study STARTED	9
Overall Study Received at Least 1 Dose of Study Medication	9
Overall Study COMPLETED	0
Overall Study NOT COMPLETED	9

Reasons for withdrawal

Reasons for withdrawal
Measure	X4P-001 Plus Nivolumab 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.
Overall Study Adverse Event	4
Overall Study Clinical Deterioration	1
Overall Study Disease Progression	3
Overall Study Study Termination	1

Baseline Characteristics

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

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	X4P-001 Plus Nivolumab n=9 Participants 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	62.7 years STANDARD_DEVIATION 8.85 • n=5 Participants
Sex: Female, Male Female	1 Participants n=5 Participants
Sex: Female, Male Male	8 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	8 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	1 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants
Race (NIH/OMB) White	9 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants

PRIMARY outcome

Timeframe: From administration of first dose of study medication (Day 1) up to 30 days after last dose (up to 16 months)

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

Outcome measures

Outcome measures
Measure	X4P-001 Plus Nivolumab n=9 Participants 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.
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) Any TEAEs	9 Participants
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) Serious TEAEs	2 Participants
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) X4P-001-Related TEAEs	9 Participants
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) Nivolumab-Related TEAEs	9 Participants

SECONDARY outcome

Timeframe: 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

Population: 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.

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

Outcome measures

Outcome data not reported

SECONDARY outcome

Population: 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.

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

Outcome measures

Outcome data not reported

SECONDARY outcome

Population: 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.

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

Outcome measures

Outcome data not reported

SECONDARY outcome

Population: 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.

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

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: From administration of first dose of study medication (Day 1) until disease progression, study completion or early termination (up to 15 months)

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

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.

Outcome measures

Outcome measures
Measure	X4P-001 Plus Nivolumab n=9 Participants 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.
Objective Response Rate (ORR): Percentage of Participants With Objective Response, Evaluated Using Response Evaluation Criteria in Solid Tumors (RECIST) Version 1.1	11.1 percentage of participants Interval 0.3 to 48.2

SECONDARY outcome

Timeframe: Time from first CR or PR until the time of disease progression or death due to any cause (up to 15 months)