Clear Cell Renal Cell CarcinomaPhase 1/2NCT02667886
Trial of X4P-001 in Participants With Advanced Renal Cell Carcinoma
A Phase 1/2 study testing X4P-001 and axitinib in people with Clear Cell Renal Cell Carcinoma, run by X4 Pharmaceuticals at 20 sites in 2 countries.
Trial summary
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… 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 axitinib
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 74 participants
- Where
- 20 sitesin 2 countries
- Study dates
- 27 Apr 2016to 14 Apr 2022
- 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
- Have a histologically confirmed diagnosis of predominant ccRCC.
- Have received at least one prior course of treatment for ccRCC. Part C only: Prior treatment must include at least 1 course of vascular endothelial growth factor (VEGF)-directed therapy.
- Have on computed tomography (CT) imaging done within 28 days of Day 1 findings consistent with advanced ccRCC, including at least one extra-renal measurable target lesion meeting the criteria of Response Evaluation Criteria in Solid Tumors (RECIST) Version 1.1.
- For women of childbearing potential and men, agree to use effective contraceptive methods from screening, through the study, and for at least 4 weeks after the last dose of study drug.
- For women of childbearing potential, have a negative pregnancy test (serum or urine) on Day 1 prior to initiating study treatment.
- Be willing and able to comply with the protocol
Who cannot join
- 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.
Show all 36Show fewer
- 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.
Trial locations and contacts
20 sites in 2 countries.
20 sites in 2 countries
United States18 sites
Study site
Boston, Massachusetts, 02215
Study site
Chicago, Illinois, 60637
Study site
Detroit, Michigan, 48201
Study site
Greenville, South Carolina, 29605
Study site
Hackensack, New Jersey, 07601
Study site
Houston, Texas, 77030
Study site
Indianapolis, Indiana, 46237
Study site
Iowa City, Iowa, 52242
Study site
Jacksonville, Florida, 32224
Study site
New Orleans, Louisiana, 70121
Study site
New York, New York, 10029
Study site
Philadelphia, Pennsylvania, 19104
Study site
Saint Paul, Minnesota, 55101
Study site
Scottsdale, Arizona, 85259
Study site
St Louis, Missouri, 63110
Study site
The Bronx, New York, 10461
Study site
Toledo, Ohio, 43623
Study site
Washington D.C., District of Columbia, 20057
South Korea2 sites
Study site
Gyeyang-gu, Seoul, 06351
Study site
Seongdu, Seoul, 03722
About this study
Official title: A Phase 1/2 Trial of X4P-001 as Single Agent and in Combination With Axitinib in Patients With Advanced Renal Cell Carcinoma
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.
Read the detailed description
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:
- Eliminating tumor re-vascularization
- Decreasing the infiltration of MDSCs
- Increasing the ratio of cluster of differentiation 8+ (CD8+) T cells to T regulatory (Treg) cells
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:
- Decreased recruitment of MDSCs, resulting in increased anti-tumor immune attack
- Sustained decrease in neoangiogenesis and tumor vascular supply
- Interference with the autocrine effect of increased expression by ccRCC of both CXCR4 and CXCL12, its only ligand, thereby, potentially reducing cancer cell metastasis
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.
- Conditions
- Clear Cell Renal Cell Carcinoma
- Being tested
- X4P-001, axitinib
- 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
Also called Mavorixafor, AMD11070
Continuous, oral dosing
axitinibDrug
Also called Inlyta
Continuous, oral dosing
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: Drug: X4P-001; Drug: axitinib |
| 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. Receives: Drug: X4P-001; Drug: axitinib |
| 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. Receives: Drug: X4P-001; Drug: axitinib |
| 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. Receives: Drug: X4P-001; Drug: axitinib |
| Dose Escalation and Expansion (Part C): X4P-001 600 mg QD Monotherapy | Experimental | Participants will receive X4P-001 600 mg orally QD. Receives: Drug: X4P-001 |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: From first dose of study drug up to 10 days after last dosing (maximum exposure: 52.1 months)
Secondary outcomes (8)
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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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
Measured: Day 1 and Day 15 of Cycle 1
Area Under the Curve From Time 0 to the Last Quantifiable Timepoint (AUC0-tlast)
Measured: Day 1 and Day 15 of Cycle 1
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Jan 2016 (estimated)
- Study start27 Apr 2016
- Primary completion14 Apr 2022
- Study completion14 Apr 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Clear Cell Renal Cell CarcinomaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
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
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 74 participants (actual)
Sponsor and investigators
- Lead sponsor
- X4 Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02667886
- Sponsor's protocol ID
- X4P-001-RCCA
- Status last verified
- Oct 2024
- First submitted
- 20 Jan 2016
- Last updated
- 3 Oct 2024
- Results posted
- Yes, on ClinicalTrials.gov
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 3 Oct 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1/2 Trial of X4P-001 as Single Agent and in Combination With Axitinib in Patients With Advanced Renal Cell Carcinoma
- ClinicalTrials.gov ID
- NCT02667886
- Acronym / sponsor ID
- X4P-001-RCCA
- Conditions
- Clear Cell Renal Cell Carcinoma
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 74 participants (actual)
- Lead sponsor
- X4 PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 27 Apr 2016 (actual)
- Primary completion
- 14 Apr 2022 (actual)
- Study completion
- 14 Apr 2022 (actual)
- First posted
- 29 Jan 2016 (estimated)
- Results
- Posted 14 Aug 2024
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
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:
- Eliminating tumor re-vascularization
- Decreasing the infiltration of MDSCs
- Increasing the ratio of cluster of differentiation 8+ (CD8+) T cells to T regulatory (Treg) cells
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:
- Decreased recruitment of MDSCs, resulting in increased anti-tumor immune attack
- Sustained decrease in neoangiogenesis and tumor vascular supply
- Interference with the autocrine effect of increased expression by ccRCC of both CXCR4 and CXCL12, its only ligand, thereby, potentially reducing cancer cell metastasis
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 5
| Arm | Type | Receives |
|---|---|---|
| Dose Escalation (Part A): X4P-001 200 mg BID with Axitinib | Experimental | Drug: X4P-001, Drug: axitinibParticipants 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 | Drug: X4P-001, Drug: axitinibParticipants 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 | Drug: X4P-001, Drug: axitinibParticipants 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 | Drug: X4P-001, Drug: axitinibParticipants 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 | Drug: X4P-001Participants will receive X4P-001 600 mg orally QD. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- Have a histologically confirmed diagnosis of predominant ccRCC.
- Have received at least one prior course of treatment for ccRCC. Part C only: Prior treatment must include at least 1 course of vascular endothelial growth factor (VEGF)-directed therapy.
- Have on computed tomography (CT) imaging done within 28 days of Day 1 findings consistent with advanced ccRCC, including at least one extra-renal measurable target lesion meeting the criteria of Response Evaluation Criteria in Solid Tumors (RECIST) Version 1.1.
- For women of childbearing potential and men, agree to use effective contraceptive methods from screening, through the study, and for at least 4 weeks after the last dose of study drug.
- For women of childbearing potential, have a negative pregnancy test (serum or urine) on Day 1 prior to initiating study treatment.
Key exclusion criteria (36 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| X4P-001Also: Mavorixafor, AMD11070Continuous, oral dosing | Drug | Dose Escalation (Part A): X4P-001 200 mg BID with Axitinib, Dose Escalation (Part A): X4P-001 400 mg QD with Axitinib, Dose Escalation (Part A): X4P-001 600 mg QD with Axitinib, Dose Escalation and Expansion (Part C): X4P-001 600 mg QD Monotherapy, Dose Expansion (Part B): X4P-001 400 mg QD With Axitinib |
| axitinibAlso: InlytaContinuous, oral dosing | Drug | Dose Escalation (Part A): X4P-001 200 mg BID with Axitinib, Dose Escalation (Part A): X4P-001 400 mg QD with Axitinib, Dose Escalation (Part A): X4P-001 600 mg QD with Axitinib, Dose Expansion (Part B): X4P-001 400 mg QD With Axitinib |
Primary outcome
Parts A, B, and C: Number of Participants With Treatment-emergent Adverse Events (TEAEs)
Time frame: From first dose of study drug up to 10 days after last dosing (maximum exposure: 52.1 months)
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.
8 secondary outcomes
Parts A and B: Objective Response Rate (ORR), as Assessed Using Response Evaluation Criteria in Solid Tumors (RECIST) Version 1.1
Time frame: 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 frame: 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
Time frame: 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
Time frame: 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
Time frame: 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
Time frame: 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
Time frame: Day 1 and Day 15 of Cycle 1
Area Under the Curve From Time 0 to the Last Quantifiable Timepoint (AUC0-tlast)
Time frame: Day 1 and Day 15 of Cycle 1
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Oct 2024.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
20 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing X4P-001 and axitinib in people with Clear Cell Renal Cell Carcinoma, run by X4 Pharmaceuticals at 20 sites in 2 countries.
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 6 inclusion and 36 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: finished, as of the sponsor's last update on 3 Oct 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 20 sites in United States, South Korea. 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 14 Aug 2024). 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?
Parts A, B, and C: Number of Participants With Treatment-emergent Adverse Events (TEAEs) (time frame: From first dose of study drug up to 10 days after last dosing (maximum exposure: 52.1 months)). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 3 Oct 2024.