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Efficacy and Safety Study of Mavorixafor in Participants With Warts, Hypogammaglobulinemia, Infections, and Myelokathexis (WHIM) Syndrome
Sponsor: X4 Pharmaceuticals
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.
Official title: A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study of Mavorixafor in Patients With WHIM Syndrome With Open-Label Extension
Key Details
Gender
All
Age Range
12 Years - Any
Study Type
INTERVENTIONAL
Enrollment
31
Start Date
2019-10-24
Completion Date
2025-12
Last Updated
2025-07-29
Healthy Volunteers
No
Conditions
Interventions
Mavorixafor
Mavorixafor provided as 100 mg capsules.
Placebo
Placebo matching to mavorixafor capsules
Locations (23)
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)