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Efficacy and Safety Study of Mavorixafor in Participants With Warts, Hypogammaglobulinemia, Infections, and Myelokathexis (WHIM) Syndrome
NCT03995108 · View on ClinicalTrials.gov ↗
Study Summary
This study has a double-blind, Randomized Placebo-Controlled Period and an Open-Label Period. The primary objective of the Randomized Placebo-Controlled Period is to demonstrate the efficacy of mavorixafor in participants with WHIM syndrome as assessed by increasing levels of circulating neutrophils compared with placebo, and relative to a clinically meaningful threshold. The primary objective of the Open-Label Period is to evaluate the safety and tolerability of mavorixafor in participants with WHIM syndrome. Participants are allowed to continue treatment in the Open-Label Period, if regionally applicable, until mavorixafor becomes commercially available, or until the study is terminated by the Sponsor.
Conditions Studied
Interventions
- DRUG Placebo
- DRUG Mavorixafor
Study Locations (20)
Other
- Medical University of Vienna - Medizinische Universität Wien — Vienna
- Aarhus University Hospital — Aarhus
- CHU Paris Est, Hôpital d'Enfants Armand-Trousseau — Paris
- Hopital Necker-Enfants Malades — Paris
- HaEmek Medical Center — Afula
- Emma Children's Hospital Academic Medical Center (AMC) — Amsterdam
- Dmitry Rogachev National Research Center of Pediatric Hematology, Oncology and Immunology — Moscow
- Academician I.P. Pavlov First Saint Petersburg State Medical University — Saint Pertersburg
- Seoul National University Hospital, Children's Hospital — Seoul
California
- University of California San Diego Health/Rady Children's Hospital — San Diego
- California Dermatology Institute — Thousand Oaks
Queensland
- Wesley Hospital — Auchenflower
- Children's Health Queensland Hospital — South Brisbane
Iowa
- University of Iowa — Iowa City
Maryland
- Johns Hopkins University Medical Center — Baltimore
Texas
- University of Texas Southwestern Medical Center — Dallas
Washington
- University of Washington Medical Center — Seattle
Rhne
- CHU de Lyon, Institut d'Hematologie et d'Oncologie Pediatrique — Lyon
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 31 participants |
| Start Date | 2019-10-24 |
| Est. Completion | 2025-12 |
| Phase | Phase 3 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT03995108
The ClinicalTrials.gov registry entry for NCT03995108 describes a study currently listed as active not recruiting. It is categorized as Phase 3, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 31 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is X4 Pharmaceuticals, which has 5 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.
The record links to 1 condition, with WHIM Syndrome appearing as the primary indexed condition, and to 2 interventions — of which Placebo is the first listed. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.
Geographic footprint matters for practical reasons: NCT03995108 reports 20 study locations spanning 10 distinct geographic areas — top geographies include Other, California, Queensland. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.
Frequently Asked Questions
What is clinical trial NCT03995108 about?
NCT03995108 is a clinical study titled "Efficacy and Safety Study of Mavorixafor in Participants With Warts, Hypogammaglobulinemia, Infections, and Myelokathexis (WHIM) Syndrome". This study has a double-blind, Randomized Placebo-Controlled Period and an Open-Label Period. The primary objective of the Randomized Placebo-Controlled Period is to demonstrate the efficacy of mavorixafor in participants with WHIM syndrome as assessed by increasing levels of circulating neutrophils...
What is the current status of trial NCT03995108?
This trial is currently active not recruiting. It is a Phase 3 study. The enrollment target is 31 participants. The study started on 2019-10-24. Estimated completion is 2025-12.
What conditions does trial NCT03995108 study?
This clinical trial studies the following conditions: WHIM Syndrome. These conditions were identified from the trial registry and reflect the primary focus areas of the research.
What interventions are being tested in trial NCT03995108?
The interventions under investigation include: Placebo (DRUG), Mavorixafor (DRUG). Each intervention is being evaluated for safety and efficacy as part of this clinical study.
Who is sponsoring clinical trial NCT03995108?
This trial is sponsored by X4 Pharmaceuticals, which has 5 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.
Where is trial NCT03995108 being conducted?
This trial has 20 study locations across California, Iowa, Maryland, Texas, Washington. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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