Efficacy and Safety Study of Mavorixafor in Participants With Warts, Hypogammaglobulinemia, Infections, and Myelokathexis (WHIM) Syndrome

NCT ID: NCT03995108

Last Updated: 2025-07-29

Basic Information

• Recruitment Status: ACTIVE_NOT_RECRUITING
• Clinical Phase: PHASE3
• Total Enrollment: 31 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2019-10-24
• Study Completion Date: 2025-12-31

Brief 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

WHIM Syndrome

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: DOUBLE

Study Groups

Mavorixafor

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.

Group Type: EXPERIMENTAL

Mavorixafor

Intervention Type: DRUG

Mavorixafor provided as 100 mg capsules.

Placebo

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.

Group Type: PLACEBO_COMPARATOR

Mavorixafor

Intervention Type: DRUG

Mavorixafor provided as 100 mg capsules.

Placebo

Intervention Type: DRUG

Placebo matching to mavorixafor capsules

Interventions

Mavorixafor

Mavorixafor provided as 100 mg capsules.

Intervention Type: DRUG

Placebo

Placebo matching to mavorixafor capsules

Intervention Type: DRUG

Other Intervention Names

AMD11070; X4P-001

Eligibility Criteria

Inclusion Criteria

• Have signed the current approved informed consent form. Participants under 18 years of age (in the Netherlands and other applicable regions, participants under 16 years of age) will sign an approved informed assent form and must also have a signed parental/legal guardian consent.
• Have a genotype-confirmed mutation of chemokine (C-X-C motif) receptor 4 (CXCR4) consistent with WHIM phenotype.
• Agree to use a highly effective form of contraception.
• Be willing and able to comply with the protocol.
• Have confirmed ANC ≤400 cells/µL during screening, obtained while participant has no clinical evidence of infection.

• Completed the Randomized Period; or
• Granted Early Release from the Randomized Period.

Exclusion Criteria

• 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.

• Minimum Eligible Age: 12 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

X4 Pharmaceuticals

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Chief Medical Officer

Role: STUDY_DIRECTOR

X4 Pharmaceuticals

Locations

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)

Countries

United States; Australia; Austria; Denmark; France; Hungary; Israel; Italy; Netherlands; Russia; South Korea; Spain; Turkey (Türkiye)

References

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.

Reference Type: DERIVED

PMID: 38643510 (View on PubMed)

Other Identifiers

2019-001153-10

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

4WHIM

Identifier Type: OTHER

Identifier Source: secondary_id

X4P-001-103

Identifier Type: -

Identifier Source: org_study_id