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Small Molecule

Mavorixafor for WHIM Syndrome

Phase 3
Waitlist Available
Research Sponsored by X4 Pharmaceuticals
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Have signed the current approved informed consent form. Participants under 18 years of age (in the Netherlands and other applicable regions, participants under 16 years of age) will sign an approved informed assent form and must also have a signed parental/legal guardian consent.
Have confirmed ANC ≤400 cells/µL during screening, obtained while participant has no clinical evidence of infection.
Timeline
Screening 3 weeks
Treatment Varies
Follow Up year 1 of open-label period
Awards & highlights

Study Summary

This trial has two parts: a double-blind controlled period where half the participants receive a placebo and half receive the drug being tested, and an open-label period where all participants receive the drug. The primary objective of the first part is to see if the drug being tested is effective at increasing levels of circulating neutrophils compared to placebo, and the second part is to see if the drug is safe and well-tolerated.

Who is the study for?
This trial is for individuals with WHIM syndrome, a rare genetic condition. Participants must have a confirmed CXCR4 mutation and low neutrophil counts without infection. They should agree to use effective contraception and comply with the study protocol. It's open to adults and some minors who provide consent.Check my eligibility
What is being tested?
The trial is testing Mavorixafor's ability to increase neutrophils in WHIM patients compared to a placebo. Initially, it's double-blind—neither participants nor researchers know who gets what—and then all get Mavorixafor in an Open-Label Period until it’s commercially available or the study ends.See study design
What are the potential side effects?
While specific side effects are not listed here, typically such trials monitor for any adverse reactions ranging from mild symptoms like headaches or nausea to more serious issues affecting organ function or causing allergic reactions.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I have signed the consent form, or if under 18 (or 16 in certain regions), I and my guardian have both signed.
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My white blood cell count is very low without any signs of infection.
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My condition is confirmed to have a CXCR4 mutation linked to WHIM syndrome.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~year 1 of open-label period
This trial's timeline: 3 weeks for screening, Varies for treatment, and year 1 of open-label period for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Open-Label Period: Percentage of Participants With Adverse Events (AEs)
Randomized Placebo-Controlled Period: Time (in Hours) Above Threshold-Absolute Neutrophil Count (TAT-ANC in hours) of ≥ 500 Cells/Microliter (µL) over a 24-hour period
Secondary outcome measures
Absolute and Fold Change From Baseline in Absolute T, B and Natural Killer Lymphocyte at Week 52
Leukocytes
Open-Label Period: Change From Baseline in Cutaneous Warts at Week 52, Based on Central Review of CGI-C
+49 more

Trial Design

2Treatment groups
Experimental Treatment
Placebo Group
Group I: MavorixaforExperimental Treatment1 Intervention
Participants (adults and adolescents [12 to 17 years of age weighing >50 kilograms [kg]) will receive mavorixafor 400 milligrams (mg) once daily (QD) orally for 52 weeks in the Randomized Placebo-Controlled Period. Adolescents weighing ≤50 kg will receive mavorixafor 200 mg QD. Participants who complete the Randomized Placebo-Controlled Period or are granted Early Release due to recurrent or significant infections, as adjudicated by a blinded, independent adjudication committee (AC), will be offered the opportunity to enroll in the Open-Label Period and receive treatment with mavorixafor 400 mg once daily orally until commercial availability or study termination by the Sponsor.
Group II: PlaceboPlacebo Group2 Interventions
Participants will receive placebo matching to mavorixafor QD orally for 52 weeks in the Randomized Placebo-Controlled Period. Participants who complete the Randomized Placebo-Controlled Period or are granted Early Release due to recurrent or significant infections, as adjudicated by a blinded, independent AC, will be offered the opportunity to enroll in the Open-Label Period and receive treatment with mavorixafor 400 mg once daily orally until commercial availability or study termination by the Sponsor.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
AMD-070
Not yet FDA approved

Find a Location

Who is running the clinical trial?

X4 PharmaceuticalsLead Sponsor
9 Previous Clinical Trials
338 Total Patients Enrolled
Chief Medical OfficerStudy DirectorX4 Pharmaceuticals
122 Previous Clinical Trials
21,493 Total Patients Enrolled

Media Library

Mavorixafor (Small Molecule) Clinical Trial Eligibility Overview. Trial Name: NCT03995108 — Phase 3
Williams-Beuren Syndrome Research Study Groups: Placebo, Mavorixafor
Williams-Beuren Syndrome Clinical Trial 2023: Mavorixafor Highlights & Side Effects. Trial Name: NCT03995108 — Phase 3
Mavorixafor (Small Molecule) 2023 Treatment Timeline for Medical Study. Trial Name: NCT03995108 — Phase 3

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
~6 spots leftby Mar 2025