Trial of X4P-001 in Participants With Advanced Renal Cell Carcinoma

NCT02667886 | Completed Phase 1 Results FDA Drug

• 1 other identifier: X4P-001-RCCA
• Study Type: interventional
• Target: 74
• Locations: 2 countries
• Sites: 20

Brief 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 cancer tumor.

Conditions

Clear Cell Renal Cell Carcinoma

Outcome Measures

Primary Outcomes (1)

• 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.

  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

  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

  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 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

  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

  From administration of first dose of study medication until disease progression (up to 80 weeks)

Study Arms (5)

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.

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.

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.

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.

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.

Drug: X4P-001

Interventions

X4P-001 DRUG

Continuous, oral dosing

Also known as: Mavorixafor, AMD11070

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

axitinib DRUG

Continuous, oral dosing

Also known as: Inlyta

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

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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.
• 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.

Sponsors & Collaborators

• X4 Pharmaceuticals: lead

Study Sites (20)

Unknown Facility

Scottsdale, Arizona, 85259, United States

Location

Unknown Facility

Washington D.C., District of Columbia, 20057, United States

Location

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Jacksonville, Florida, 32224, United States

Location

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Chicago, Illinois, 60637, United States

Location

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Indianapolis, Indiana, 46237, United States

Location

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Iowa City, Iowa, 52242, United States

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New Orleans, Louisiana, 70121, United States

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Boston, Massachusetts, 02215, United States

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Detroit, Michigan, 48201, United States

Location

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Saint Paul, Minnesota, 55101, United States

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St Louis, Missouri, 63110, United States

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Hackensack, New Jersey, 07601, United States

Location

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New York, New York, 10029, United States

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The Bronx, New York, 10461, United States

Location

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Toledo, Ohio, 43623, United States

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Philadelphia, Pennsylvania, 19104, United States

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Greenville, South Carolina, 29605, United States

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Houston, Texas, 77030, United States

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Gyeyang-gu, Seoul, 06351, South Korea

Location

Unknown Facility

Seongdu, Seoul, 03722, South Korea

Location

MeSH Terms

Conditions

Carcinoma, Renal Cell

Interventions

mavorixafor; Axitinib

Condition Hierarchy (Ancestors)

Adenocarcinoma; Carcinoma; Neoplasms, Glandular and Epithelial; Neoplasms by Histologic Type; Neoplasms; Kidney Neoplasms; Urologic Neoplasms; Urogenital Neoplasms; Neoplasms by Site; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Kidney Diseases; Urologic Diseases; Male Urogenital Diseases

Intervention Hierarchy (Ancestors)

Benzamides; Amides; Organic Chemicals; Benzoates; Acids, Carbocyclic; Carboxylic Acids; Benzene Derivatives; Hydrocarbons, Aromatic; Hydrocarbons, Cyclic; Hydrocarbons; Indazoles; Pyrazoles; Azoles; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Heterocyclic Compounds, 2-Ring; Heterocyclic Compounds, Fused-Ring

Results Point of Contact

• Title: Chief Medical Officer
• Organization: X4 Pharmaceuticals

patientinfo@x4pharma.com

(857) 529-8300

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: GT60
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 20, 2016
• First Posted: January 29, 2016
• Study Start: April 27, 2016
• Primary Completion: April 14, 2022
• Study Completion: April 14, 2022
• Last Updated: October 3, 2024
• Results First Posted: August 14, 2024

Record last verified: 2024-10

Locations

US (18); KR (2)