Mavorixafor for Neutropenia

Recruiting in Palo Alto (17 mi)

+50 other locations

Age: Any Age

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 3

Recruiting

Sponsor: X4 Pharmaceuticals

Must be taking: G-CSF

Must not be taking: Clozapine, Antiretrovirals, Antibiotics, others

Disqualifiers: Secondary neutropenia, Aplastic anemia, WHIM syndrome, others

Pivotal Trial (Near Approval)

Prior Safety Data

Trial Summary

What is the purpose of this trial?

The purpose of this study is to demonstrate the efficacy and evaluate the safety, and tolerability of mavorixafor in participants with congenital or acquired primary autoimmune and idiopathic chronic neutropenic disorders who are experiencing recurrent and/or serious infections as assessed by demonstrating its clinical benefit and increasing levels of circulating neutrophils.

Will I have to stop taking my current medications?

The trial requires that participants who are on G-CSF or other active background therapy must continue these medications at a stable dose and schedule throughout the study. If you are on other medications, the protocol does not specify whether you need to stop them.

What data supports the effectiveness of the drug Mavorixafor for neutropenia?

Mavorixafor has shown effectiveness in increasing white blood cell counts, including neutrophils, in patients with WHIM syndrome, a condition that involves low white blood cell counts. In a study, patients taking Mavorixafor experienced increased neutrophil levels and a reduction in infection rates, suggesting its potential benefit for conditions involving neutropenia (low levels of neutrophils). ^{1 2 3 4 5}

How is the drug Mavorixafor unique in treating neutropenia?

Mavorixafor is unique because it is an oral medication that selectively inhibits the CXCR4 receptor, which helps mobilize white blood cells from the bone marrow, potentially increasing neutrophil counts. This mechanism is different from other treatments like pegfilgrastim, which are typically injected and work by stimulating the production of white blood cells. ^{1 2 3 6 7}

Research Team

Chief Medical Officer

Principal Investigator

X4 Pharmaceuticals

Eligibility Criteria

This trial is for people with low white blood cell counts due to congenital or acquired neutropenic disorders, who have been on stable doses of standard treatments like G-CSF for at least a year. They must have had the disorder for over six months and show a specific low level of neutrophils without current infections.

Inclusion Criteria

Participants must maintain their G-CSF doses/regimens unchanged (unless for safety reasons) during the trial.

I have been on a stable dose of G-CSF or similar treatments for at least 4 weeks.

Key

Trial Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive mavorixafor or placebo orally once daily from Day 1 through Week 52

52 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

Interventions

• Mavorixafor (CXCR4 Inhibitor)

Trial Overview The study tests Mavorixafor's effectiveness in increasing white blood cells and reducing infection risks compared to a placebo. Participants will be randomly assigned to receive either Mavorixafor or an inactive substance while keeping their current treatment constant.

Participant Groups

Group I: Mavorixafor Experimental Treatment 1 Intervention

Participants will receive mavorixafor orally once daily starting from Day 1 through Week 52.

Group II: Placebo Placebo Group 1 Intervention

Participants will receive placebo to match mavorixafor orally once daily starting from Day 1 through Week 52.

Find a Clinic Near You

Research Locations Nearby Select from list below to view details:

Orso Health La Jolla, CA

Orso Health Torrance, CA

University of Michigan Ann Arbor, MI

Washington University Saint Louis, MO

Who Is Running the Clinical Trial?

X4 Pharmaceuticals

Lead Sponsor

Trials

11

Recruited

400+

References

1. United States pubmed.ncbi.nlm.nih.gov

Mavorixafor, an Orally Bioavailable CXCR4 Antagonist, Increases Immune Cell Infiltration and Inflammatory Status of Tumor Microenvironment in Patients with Melanoma. [2023]

2. United States pubmed.ncbi.nlm.nih.gov

Results of a phase 2 trial of an oral CXCR4 antagonist, mavorixafor, for treatment of WHIM syndrome. [2021]

3. United States pubmed.ncbi.nlm.nih.gov

A phase 1b trial of the CXCR4 inhibitor mavorixafor and nivolumab in advanced renal cell carcinoma patients with no prior response to nivolumab monotherapy. [2022]

4. England pubmed.ncbi.nlm.nih.gov

Severe neutropenia during cabazitaxel treatment is associated with survival benefit in men with metastatic castration-resistant prostate cancer (mCRPC): A post-hoc analysis of the TROPIC phase III trial. [2022]

5. United States pubmed.ncbi.nlm.nih.gov

Chemotherapy-Induced Neutropenia and Outcome in Patients With Metastatic Castration-Resistant Prostate Cancer Treated With First-Line Docetaxel. [2019]

6. China pubmed.ncbi.nlm.nih.gov

[Randomized controlled study of leucomax (recombinant human granulocyte-macrophage colony stimulating factor, rhGM-CSF) in the treatment of cancer chemotherapy-induced leucopenia]. [2006]

7. England pubmed.ncbi.nlm.nih.gov

Comparable efficacy and safety profiles of once-per-cycle pegfilgrastim and daily injection filgrastim in chemotherapy-induced neutropenia: a multicenter dose-finding study in women with breast cancer. [2022]