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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 confirmed ANC ≤400 cells/µL during screening, obtained while participant has no clinical evidence of infection.
Have a genotype-confirmed mutation of chemokine (C-X-C motif) receptor 4 (CXCR4) consistent with WHIM phenotype.
Timeline
Screening 3 weeks
Treatment Varies
Follow Up baseline up to week 52
Awards & highlights
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.
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.
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 belowSelect...
My white blood cell count is very low without any signs of infection.
Select...
My condition is confirmed to have a CXCR4 mutation linked to WHIM syndrome.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ baseline up to week 52
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~baseline up to week 52
Treatment Details
Study Objectives
Study objectives can provide a clearer picture of what you can expect from a treatment.Secondary study objectives
Randomized Placebo-Controlled Period: Composite Clinical Efficacy (Total Infection Score and Total Wart Change Score) for Participants With Non-Ig Use
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
331 Total Patients Enrolled
Chief Medical OfficerStudy DirectorX4 Pharmaceuticals
124 Previous Clinical Trials
21,800 Total Patients Enrolled
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- I have signed the consent form, or if under 18 (or 16 in certain regions), I and my guardian have both signed.My white blood cell count is very low without any signs of infection.You have finished the randomized period of the study or have been released early from it.You agree to use a very effective method of birth control during the study.My condition is confirmed to have a CXCR4 mutation linked to WHIM syndrome.You agree to follow the study guidelines.
Research Study Groups:
This trial has the following groups:- Group 1: Placebo
- Group 2: Mavorixafor
Awards:
This trial has 1 awards, including:- Pivotal Trial - The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
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