150 Participants Needed

Mavorixafor for Neutropenia

Recruiting at 82 trial locations
PA
Overseen ByPatient Affairs and Advocacy
Pivotal Trial (Near Approval)This treatment is in the last trial phase before FDA approval
Prior Safety DataThis treatment has passed at least one previous human trial

Trial Summary

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).12345

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.12367

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.

Research Team

CM

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.
I have been diagnosed with a chronic neutropenic disorder not caused by medications, infections, or cancer for over 6 months.
See 1 more

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
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
2Treatment groups
Experimental Treatment
Placebo Group
Group I: MavorixaforExperimental Treatment1 Intervention
Participants will receive mavorixafor orally once daily starting from Day 1 through Week 52.
Group II: PlaceboPlacebo Group1 Intervention
Participants will receive placebo to match mavorixafor orally once daily starting from Day 1 through Week 52.

Find a Clinic Near You

Who Is Running the Clinical Trial?

X4 Pharmaceuticals

Lead Sponsor

Trials
11
Recruited
400+

Findings from Research

Mavorixafor, an oral CXCR4 inhibitor, significantly increased immune cell infiltration and activation in melanoma tumors, enhancing key immune signatures after 3 weeks of treatment.
The combination of mavorixafor with pembrolizumab further boosted immune responses, while maintaining an acceptable safety profile with mostly mild adverse events, suggesting its potential as a treatment for patients not responding to existing therapies.
Mavorixafor, an Orally Bioavailable CXCR4 Antagonist, Increases Immune Cell Infiltration and Inflammatory Status of Tumor Microenvironment in Patients with Melanoma.Andtbacka, RHI., Wang, Y., Pierce, RH., et al.[2023]
Mavorixafor, an oral CXCR4 antagonist, was well tolerated in a phase 2 study involving 8 adult patients with WHIM syndrome, showing no serious treatment-related adverse events.
The treatment led to significant increases in white blood cell counts and a reduction in yearly infection rates from 4.63 to 2.27 events, along with a 75% decrease in cutaneous warts, indicating its potential efficacy in managing WHIM syndrome.
Results of a phase 2 trial of an oral CXCR4 antagonist, mavorixafor, for treatment of WHIM syndrome.Dale, DC., Firkin, F., Bolyard, AA., et al.[2021]
In a study involving 9 patients with metastatic clear cell renal cell carcinoma (ccRCC) who were unresponsive to nivolumab, the combination of mavorixafor and nivolumab showed potential antitumor activity, with one patient achieving a partial response and others experiencing stable disease for a median duration of 6.7 months.
Mavorixafor, an allosteric CXCR4 inhibitor, appears to enhance immune responses by increasing the infiltration of activated cytotoxic T cells and correlating with elevated levels of CXCL9, suggesting its mechanism may improve treatment outcomes when combined with PD-1 blockade.
A phase 1b trial of the CXCR4 inhibitor mavorixafor and nivolumab in advanced renal cell carcinoma patients with no prior response to nivolumab monotherapy.Choueiri, TK., Atkins, MB., Rose, TL., et al.[2022]

References

Mavorixafor, an Orally Bioavailable CXCR4 Antagonist, Increases Immune Cell Infiltration and Inflammatory Status of Tumor Microenvironment in Patients with Melanoma. [2023]
Results of a phase 2 trial of an oral CXCR4 antagonist, mavorixafor, for treatment of WHIM syndrome. [2021]
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]
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]
Chemotherapy-Induced Neutropenia and Outcome in Patients With Metastatic Castration-Resistant Prostate Cancer Treated With First-Line Docetaxel. [2019]
[Randomized controlled study of leucomax (recombinant human granulocyte-macrophage colony stimulating factor, rhGM-CSF) in the treatment of cancer chemotherapy-induced leucopenia]. [2006]
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]
Unbiased ResultsWe believe in providing patients with all the options.
Your Data Stays Your DataWe only share your information with the clinical trials you're trying to access.
Verified Trials OnlyAll of our trials are run by licensed doctors, researchers, and healthcare companies.
Back to top
Terms of Service·Privacy Policy·Cookies·Security