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Clear Cell Renal Cell CarcinomaPhase 1/2NCT02923531

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

A Phase 1/2 study testing X4P-001 and Nivolumab in people with Clear Cell Renal Cell Carcinoma, run by X4 Pharmaceuticals at 4 sites in United States.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record
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Trial summary

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. Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Clear Cell Renal Cell Carcinoma
What is being tested?
X4P-001 and Nivolumab
Could I receive a placebo?
No, everyone receives an active treatment
How many people
9 participants
Where
4 sitesUnited States
Study dates
7 Dec 2016to 8 Aug 2018
Phase
Phase 1/2
Are there results?
Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Histologically confirmed diagnosis of Renal Cell Carcinoma with a documented clear cell component (ccRCC).
  • Currently receiving nivolumab and considered by Investigator to have the potential to derive clinical benefit from continuing treatment with nivolumab.
  • Based on Response Evaluation Criteria in Solid Tumors (RECIST) Version 1.1 criteria on current nivolumab treatment (prior to initiation of this study), has a best response of confirmed stable disease (SD) or confirmed progressive disease (PD). Confirmed SD or confirmed PD refers to a response that is confirmed by a second scan which is at least 4 weeks apart from the previous scan.
  • At least one extra-renal measurable target lesion meeting the criteria of RECIST Version 1.1.
  • Agree to use contraception from screening, through the study, and for at least 5 months after the last dose of nivolumab as follows: for women of childbearing potential agree to use highly-effective contraceptive methods; for males, agree to use a condom with sexual partner.

Who cannot join

  • Pregnant or nursing.
  • Life expectancy of less than 3 months.
  • Performance status greater than or equal to (≥) 2 (Eastern Cooperative Oncology Group [ECOG] criteria).
  • New York Heart Association (NYHA) Class III or IV, uncontrolled hypertension, or clinically significant arrhythmia.
  • Previously received X4P-001.
  • Has a second malignancy. Except: malignancies that were treated curatively and have not recurred within 2 years prior to study treatment; completely resected basal cell and squamous cell skin cancers; any malignancy considered to be indolent and that has never required therapy; and completely resected carcinoma in situ of any type.
Show all 12Show fewer
  • Has active central nervous system (CNS) metastases (including evidence of cerebral edema by Magnetic Resonance Imaging [MRI], or progression from prior imaging study, or any requirement for steroids, or clinical symptoms of/from CNS metastases) within 28 days prior to study treatment. Subjects with known CNS metastases must have a baseline MRI scan within 28 days of study treatment.
  • Ongoing clinical adverse events National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) Grade greater than (>) 2 resulting from prior cancer therapies.
  • Known history of Human Immunodeficiency Virus (HIV) or Acquired Immunodeficiency Syndrome (AIDS); or positive test for hepatitis C virus (HCV), or hepatitis B surface antigen (HBsAg).
  • History of clinically significant or uncontrolled cardiac, hepatic, or pulmonary disease.
  • 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 (NCI CTCAE Grade 3 or 4), chronic liver disease (meeting criteria for Child-Pugh Class B or C), or organ transplantation.
  • Inadequate hematologic, hepatic, or renal function.

Trial locations and contacts

4 sites in 1 country.

United States4 sites

  • Study site

    Boston, Massachusetts

  • Study site

    Chapel Hill, North Carolina

  • Study site

    Hackensack, New Jersey

  • Study site

    Washington D.C., District of Columbia

Other trials in United States

About this study

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

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.

Being tested
X4P-001, Nivolumab
Medical subject headings
Carcinoma, Renal Cell

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • X4P-001Drug

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

  • NivolumabDrug

    Also called Opdivo

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

Study groups in NCT02923531
Study groupRoleWhat they receive
X4P-001 Plus NivolumabExperimentalParticipants 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.
Receives: Drug: X4P-001; Drug: Nivolumab

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. 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.

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

Secondary outcomes (10)

  1. 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.

    Measured: 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

  2. 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.

    Measured: 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

  3. Minimum Plasma Concentration (Cmin) of X4P-001

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

    Measured: 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

  4. Time to Reach Cmax (Tmax) of X4P-001

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

    Measured: 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

  5. 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.

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

  6. 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.

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

  7. 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.

    Measured: From administration of first dose of study medication (Day 1) until first appearance of CR or PR (up to 15 months)

  8. 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.

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

  9. 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.

    Measured: From administration of first dose of study medication (Day 1) until disease progression or death from any cause (up to 15 months)

  10. 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.

    Measured: From administration of first dose of study medication (Day 1) until disease progression (up to 15 months)

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted4 Oct 2016 (estimated)
  2. Study start7 Dec 2016
  3. Primary completion8 Aug 2018
  4. Study completion8 Aug 2018

Why it stopped: Due to low enrollment, the study was terminated early.

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 1/2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
9 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT02923531
Sponsor's protocol ID
X4P-001-RCCB
Status last verified
Nov 2022
First submitted
30 Sep 2016
Last updated
29 Dec 2022
FDA-regulated drug
Yes
FDA-regulated device
No

Publications

  • Choueiri TK, Atkins MB, Rose TL, Alter RS, Ju Y, Niland K, Wang Y, Arbeit R, Parasuraman S, Gan L, McDermott DF. A phase 1b trial of the CXCR4 inhibitor mavorixafor and nivolumab in advanced renal cell carcinoma patients with no prior response to nivolumab monotherapy. Invest New Drugs. 2021 Aug;39(4):1019-1027. doi: 10.1007/s10637-020-01058-2. Epub 2021 Jan 28.

    PubMed 33507454

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing X4P-001 and Nivolumab in people with Clear Cell Renal Cell Carcinoma, run by X4 Pharmaceuticals at 4 sites in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 5 inclusion and 12 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: stopped early, as of the sponsor's last update on 29 Dec 2022. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 4 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this combined phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 29 Dec 2022). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Number of Participants With Treatment-Emergent Adverse Events (TEAEs) (time frame: From administration of first dose of study medication (Day 1) up to 30 days after last dose (up to 16 months)). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT02923531, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 29 Dec 2022.

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