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CXCR4 Antagonist

X4P-001 for Williams-Beuren Syndrome

Phase 2
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
Be older than 18 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up time 0 (-15 minutes [min] pre-dose), 30, 60, and 90 min (each ± 5 min) and 2, 3, 4, 8, 12, 16, and 24 hours (each ±15 min) at weeks 5, 13, and 21
Awards & highlights

Study Summary

This trial is testing a new drug for people with WHIM syndrome, a rare genetic disorder. The goal is to find a safe dose and see how well the drug works. People in the trial can keep taking the drug after the trial if it's helping them.

Eligible Conditions
  • Williams-Beuren Syndrome

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~time 0 (-15 minutes [min] pre-dose), 30, 60, and 90 min (each ± 5 min) and 2, 3, 4, 8, 12, 16, and 24 hours (each ±15 min) at weeks 5, 13, and 21
This trial's timeline: 3 weeks for screening, Varies for treatment, and time 0 (-15 minutes [min] pre-dose), 30, 60, and 90 min (each ± 5 min) and 2, 3, 4, 8, 12, 16, and 24 hours (each ±15 min) at weeks 5, 13, and 21 for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Mean AUCANC and/or AUCALC

Trial Design

1Treatment groups
Experimental Treatment
Group I: X4P-001Experimental Treatment1 Intervention
Initial Treatment Phase: Participants will initiate treatment with mavorixafor at 50 milligrams (mg) once daily (QD) orally or a higher dose, with potential escalation based on area under the curve for absolute neutrophil count and absolute leukocyte count (AUCANC/ALC) values to a maximum total daily dose of 400 mg. Participants are expected to receive treatment for 24 weeks in the initial Treatment Period or until development of a treatment-limiting toxicity (TLT). Extension Phase: All participants will receive mavorixafor; the dose will not exceed 400 mg. In the Extension Phase, treatment may continue until mavorixafor becomes available via an alternative mechanism (for example, drug is commercially available, an expanded access program, etc.) or until the study is terminated 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
354 Total Patients Enrolled

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
Recent research and studies
BloodJournal
Results of a Phase 2 Trial of an Oral CXCR4 Antagonist, Mavorixafor, for Treatment of WHIM Syndrome
Dale, David C., Frank Firkin, Audrey Anna Bolyard, Merideth Kelley, Vahagn Makaryan, Kenneth J. Gorelick, Tarek Ebrahim, et al.. 2020. “Results of a Phase 2 Trial of an Oral CXCR4 Antagonist, Mavorixafor, for Treatment of WHIM Syndrome”. Blood. American Society of Hematology. doi:10.1182/blood.2020007197.
clinicaltrials.govStudy
A Dose Determination and Safety Study of X4P-001 (Mavorixafor) in Participants With Warts, Hypogammaglobulinemia, Infections, and Myelokathexis (WHIM) Syndrome
2016. "A Dose Determination and Safety Study of X4P-001 (Mavorixafor) in Participants With Warts, Hypogammaglobulinemia, Infections, and Myelokathexis (WHIM) Syndrome". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT03005327.
~1 spots leftby Apr 2025
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