Efficacy and Safety Study of Mavorixafor in Participants With Warts, Hypogammaglobulinemia, Infections, and Myelokathexis (WHIM) Syndrome
Completed · Phase 3 · Has a placebo group
Conditions studied: WHIM Syndrome
In brief
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.
Key facts
- Study ID
- NCT03995108
- Run by
- X4 Pharmaceuticals
- People needed
- 31
- Starts
- 2019-10-24
- Expected to finish
- 2025-12-23
- Last updated by the study team
- 2026-07-06
Who can join
Age: 12 and older. Sex: any. Healthy volunteers: not accepted.
You may not qualify if…
- Has known systemic hypersensitivity to the mavorixafor drug substance, its inactive ingredients, or the placebo.
- Is pregnant or breastfeeding.
- Has any medical or personal condition, which in the opinion of the Investigator may potentially compromise the safety or compliance of the participant or may preclude the participant's successful completion of the clinical study.
Where it is running
- University of California San Diego Health/Rady Children's Hospital — San Diego, California, United States
- California Dermatology Institute — Thousand Oaks, California, United States
- University of Iowa — Iowa City, Iowa, United States
- Johns Hopkins University Medical Center — Baltimore, Maryland, United States
- University of Texas Southwestern Medical Center — Dallas, Texas, United States
- University of Washington Medical Center — Seattle, Washington, United States
- Wesley Hospital — Auchenflower, Queensland, Australia
- Children's Health Queensland Hospital — South Brisbane, Queensland, Australia
- Medical University of Vienna - Medizinische Universität Wien — Vienna, Austria
- Aarhus University Hospital — Aarhus, Denmark
- CHU de Lyon, Institut d'Hematologie et d'Oncologie Pediatrique — Lyon, Rhne, France
- CHU Paris Est, Hôpital d'Enfants Armand-Trousseau — Paris, France
- Hopital Necker-Enfants Malades — Paris, France
- University of Debrecen, Affiliated Department of Infectology — Debrecen, Hajdú-Bihar, Hungary
- HaEmek Medical Center — Afula, Israel
- Università degli Studi di Brescia, Scienze Cliniche e Sperimentali — Brescia, Piazza Del Mercato, Italy
- Emma Children's Hospital Academic Medical Center (AMC) — Amsterdam, Netherlands
- Dmitry Rogachev National Research Center of Pediatric Hematology, Oncology and Immunology — Moscow, Russia
- Academician I.P. Pavlov First Saint Petersburg State Medical University — Saint Pertersburg, Russia
- Seoul National University Hospital, Children's Hospital — Seoul, South Korea
- Hospital Sant Joan de Deu Barcelona — Barcelona, Esplugues de Llobregat, Spain
- Hospital Universitario Virgen del Rocío — Seville, Sevilla, Spain
- Cukurova University Faculty of Medicine — Sarıçam, Adana, Turkey (Türkiye)
Full record on ClinicalTrials.gov
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