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A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study of Mavorixafor in Patients With WHIM Syndrome With Open-Label Extension
Sponsor study identifier
Secondary identifiers
interventional
31
14 countries
23
Study Start
October 24, 2019
Actual (reported)Primary Completion
December 23, 2025
Actual (reported)Study Completion
December 23, 2025
Actual (reported)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.
Registry-reported information. Elapsed time and enrollment totals alone do not measure study progress or recruitment pace.
December 23, 2025
Actual (reported)Click on a node to explore related trials.
Some registry-reported names shown below are not yet linked in this graph.
Dates and qualifiers reported by the registry. Estimated dates do not confirm that a milestone has occurred.
June 19, 2019
Actual (reported)June 21, 2019
Actual (reported)October 24, 2019
Actual (reported)December 23, 2025
Actual (reported)December 23, 2025
Actual (reported)Randomized Placebo-Controlled Period: Time (in Hours) Above Threshold-Absolute Neutrophil Count (TAT-ANC in hours) of ≥ 500 Cells/Microliter (µL) over a 24-hour period
Time 0 (pre-dose, up to 15 minutes prior), 30, 60, and 90 min (each ± 5 min) and 2, 3, 4, 8, 12, 16, and 24 hours (each ± 15 min) post-dose at Baseline, Weeks 13, 26, 39, and 52
Open-Label Period: Percentage of Participants With Adverse Events (AEs)
From Day 1 (end of randomized period) up to end of study (30 days post-treatment in open-label period [Week 56 of open-label period])
Randomized Placebo-Controlled Period: Time (in Hours) Above Threshold-Absolute Lymphocyte Count (TAT-ALC) of ≥ 1000 Cells/µL over a 24-hour period
Time 0 (pre-dose, up to 15 minutes prior), 30, 60, and 90 minutes (each ± 5 minutes) and 2, 3, 4, 8, 12, 16, and 24 hours (each ± 15 minutes) post-dose at Baseline, Weeks 13, 26, 39, and 52
Randomized Placebo-Controlled Period: Composite Clinical Efficacy for Mavorixafor based on total infection score and total wart change score
Baseline up to Week 52
Randomized Placebo-Controlled Period: Change From Baseline in Total Warts Score at Week 52
Baseline, Week 52
Randomized Placebo-Controlled Period: Total Infection Score for Mavorixafor
Baseline up to Week 52
Randomized Placebo-Controlled Period: Time to Early Release
Baseline up to Week 52
Participants (adults and adolescents \[12 to 17 years of age weighing \>50 kilograms \[kg\]) will receive mavorixafor 400 milligrams (mg) once daily (QD) orally for 52 weeks in the Randomized Placebo-Controlled Period. Adolescents weighing ≤50 kg will receive mavorixafor 200 mg QD. Participants who complete the Randomized Placebo-Controlled Period or are granted Early Release due to recurrent or significant infections, as adjudicated by a blinded, independent adjudication committee (AC), will be offered the opportunity to enroll in the Open-Label Period and receive treatment with mavorixafor 400 mg once daily orally until commercial availability or study termination by the Sponsor.
Participants will receive placebo matching to mavorixafor QD orally for 52 weeks in the Randomized Placebo-Controlled Period. Participants who complete the Randomized Placebo-Controlled Period or are granted Early Release due to recurrent or significant infections, as adjudicated by a blinded, independent AC, will be offered the opportunity to enroll in the Open-Label Period and receive treatment with mavorixafor 400 mg once daily orally until commercial availability or study termination by the Sponsor.
Mavorixafor provided as 100 mg capsules.
Placebo matching to mavorixafor capsules
These entries preserve how the registry describes the intervention. Their treatment roles are shown separately from molecular identities.
Formulated product
Mavorixafor
The registry describes a specific formulation. Its exact chemical ingredients and approval status require separate evidence.
CAPSULE, HARD
View registry recordThe registry names these ingredients. The formulation’s exact chemical form has not been confirmed.
Mavorixafor
Named ingredient: mavorixafor
View registry recordUniversity of California San Diego Health/Rady Children's Hospital
San Diego, California, 92123, United States
California Dermatology Institute
Thousand Oaks, California, 91320, United States
University of Iowa
Iowa City, Iowa, 52242, United States
Johns Hopkins University Medical Center
Baltimore, Maryland, 21224, United States
University of Texas Southwestern Medical Center
Dallas, Texas, 75390-9020, United States
University of Washington Medical Center
Seattle, Washington, 98195, United States
Wesley Hospital
Badolato R, Alsina L, Azar A, Bertrand Y, Bolyard AA, Dale D, Deya-Martinez A, Dickerson KE, Ezra N, Hasle H, Kang HJ, Kiani-Alikhan S, Kuijpers TW, Kulagin A, Langguth D, Levin C, Neth O, Olbrich P, Peake J, Rodina Y, Rutten CE, Shcherbina A, Tarrant TK, Vossen MG, Wysocki CA, Belschner A, Bridger GJ, Chen K, Dubuc S, Hu Y, Jiang H, Li S, MacLeod R, Stewart M, Taveras AG, Yan T, Donadieu J. A phase 3 randomized trial of mavorixafor, a CXCR4 antagonist, for WHIM syndrome. Blood. 2024 Jul 4;144(1):35-45. doi: 10.1182/blood.2023022658.
Chief Medical Officer
X4 Pharmaceuticals
31 participants (actual enrollment reported by the registry).
First Submitted
June 19, 2019
First Posted
June 21, 2019
Study Start
October 24, 2019
Actual (reported)Registry-reported names are shown in full. Names without a link have not yet been matched to a Syfrah profile.
Contact the study team to confirm eligibility.
Auchenflower, Queensland, 4006, Australia
Children's Health Queensland Hospital
South Brisbane, Queensland, 4101, Australia
Medical University of Vienna - Medizinische Universität Wien
Vienna, 1090, Austria
Aarhus University Hospital
Aarhus, 8000, Denmark
CHU de Lyon, Institut d'Hematologie et d'Oncologie Pediatrique
Lyon, Rhne, 69008, France
CHU Paris Est, Hôpital d'Enfants Armand-Trousseau
Paris, 75571, France
Hopital Necker-Enfants Malades
Paris, 75743, France
University of Debrecen, Affiliated Department of Infectology
Debrecen, Hajdú-Bihar, H-4031, Hungary
HaEmek Medical Center
Afula, 1834111, Israel
Università degli Studi di Brescia, Scienze Cliniche e Sperimentali
Brescia, Piazza Del Mercato, 25123, Italy
Emma Children's Hospital Academic Medical Center (AMC)
Amsterdam, 1105 AZ, Netherlands
Dmitry Rogachev National Research Center of Pediatric Hematology, Oncology and Immunology
Moscow, 117997, Russia
Academician I.P. Pavlov First Saint Petersburg State Medical University
Saint Pertersburg, 197022, Russia
Seoul National University Hospital, Children's Hospital
Seoul, 03080, South Korea
Hospital Sant Joan de Deu Barcelona
Barcelona, Esplugues de Llobregat, 8950, Spain
Hospital Universitario Virgen del Rocío
Seville, Sevilla, 41013, Spain
Cukurova University Faculty of Medicine
Sarıçam, Adana, 1330, Turkey (Türkiye)
Primary Completion
December 23, 2025
Actual (reported)Study Completion
December 23, 2025
Actual (reported)Last Updated
July 6, 2026
Record last verified: 2026-07
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Source: ClinicalTrials.gov.
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