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NCT03995108 · ClinicalTrials.gov registry record · Phase 3
Efficacy and Safety Study of Mavorixafor in Participants With Warts, Hypogammaglobulinemia, Infections, and Myelokathexis (WHIM) Syndrome
A Phase 3 study of WHIM Syndrome, sponsored by X4 Pharmaceuticals.
- Active
- Registry status
- Phase 3
- Development phase
- 31
- Enrollment target
- 20
- Study locations
NCT03995108: Active Phase 3 study of WHIM Syndrome, sponsored by X4 Pharmaceuticals.
NCT03995108 is a Phase 3 study of WHIM Syndrome that is active but no longer recruiting, run by X4 Pharmaceuticals. The registered enrollment target is 31 participants, below the 772-participant average among 15,282 other Phase 3 trials with a reported enrollment target (96% lower). The trial reports 20 study locations across 10 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT03995108, a Phase 3 study of WHIM Syndrome, is active but no longer recruiting, sponsored by X4 Pharmaceuticals.
- ACTIVE NOT RECRUITING
- Registry status
- Phase 3
- Development phase
- 31 participants
- Enrollment target
- 20
- Study locations
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 |
What the registry record for NCT03995108 still lists
NCT03995108 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 31 participants, a relatively small participant target, below the 772-participant average among 15,282 other Phase 3 trials with a reported enrollment target (96% lower).
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.
NCT03995108 names 20 study sites across 10 states, led by Other, California, Queensland.
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.
What interventions are being tested in trial NCT03995108?
The interventions under investigation include: Placebo (DRUG), Mavorixafor (DRUG).
Who is sponsoring clinical trial NCT03995108?
This trial is sponsored by X4 Pharmaceuticals, which has 11 total clinical trials registered on ClinicalTrials.gov.
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.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT03995108's enrollment target sits among peer trials
31 1914th of 2000 higher than 84 of 2,000 other Phase 3 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 3 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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