Experimental drug aims to boost immunity in rare WHIM syndrome
NCT ID NCT03995108
First seen Jul 06, 2026 · Last updated Jul 07, 2026 · Updated 1 time
Summary
This phase 3 trial tests whether mavorixafor can raise neutrophil levels in people with WHIM syndrome, a rare genetic condition that weakens the immune system. Participants receive either mavorixafor or a placebo to see if the drug helps maintain infection-fighting white blood cells above a safe threshold. The study also tracks safety and long-term effects in an open-label extension.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- mavorixafor
- What this could lead to
- If successful, mavorixafor could become the first approved treatment for WHIM syndrome, helping patients fight infections more effectively.
- What could go wrong
- This is a small trial (31 participants), and results may not apply to all patients. The drug may cause side effects or fail to show meaningful clinical benefit.
This is an AI summary of the original study and may miss details. Read our disclaimer.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Locations
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Aarhus University Hospital
Aarhus, 8000, Denmark
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Academician I.P. Pavlov First Saint Petersburg State Medical University
Saint Pertersburg, 197022, Russia
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CHU Paris Est, Hôpital d'Enfants Armand-Trousseau
Paris, 75571, France
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CHU de Lyon, Institut d'Hematologie et d'Oncologie Pediatrique
Lyon, Rhne, 69008, France
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California Dermatology Institute
Thousand Oaks, California, 91320, United States
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Children's Health Queensland Hospital
South Brisbane, Queensland, 4101, Australia
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Cukurova University Faculty of Medicine
Sarıçam, Adana, 1330, Turkey (Türkiye)
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Dmitry Rogachev National Research Center of Pediatric Hematology, Oncology and Immunology
Moscow, 117997, Russia
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Emma Children's Hospital Academic Medical Center (AMC)
Amsterdam, 1105 AZ, Netherlands
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HaEmek Medical Center
Afula, 1834111, Israel
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Hopital Necker-Enfants Malades
Paris, 75743, France
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Hospital Sant Joan de Deu Barcelona
Barcelona, Esplugues de Llobregat, 8950, Spain
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Hospital Universitario Virgen del Rocío
Seville, Sevilla, 41013, Spain
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Johns Hopkins University Medical Center
Baltimore, Maryland, 21224, United States
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Medical University of Vienna - Medizinische Universität Wien
Vienna, 1090, Austria
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Seoul National University Hospital, Children's Hospital
Seoul, 03080, South Korea
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University of California San Diego Health/Rady Children's Hospital
San Diego, California, 92123, United States
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University of Debrecen, Affiliated Department of Infectology
Debrecen, Hajdú-Bihar, H-4031, Hungary
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University of Iowa
Iowa City, Iowa, 52242, United States
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University of Texas Southwestern Medical Center
Dallas, Texas, 75390-9020, United States
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University of Washington Medical Center
Seattle, Washington, 98195, United States
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Università degli Studi di Brescia, Scienze Cliniche e Sperimentali
Brescia, Piazza Del Mercato, 25123, Italy
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Wesley Hospital
Auchenflower, Queensland, 4006, Australia
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