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Experimental drug aims to boost immunity in rare WHIM syndrome

NCT ID NCT03995108

Disease control Sponsor: X4 Pharmaceuticals Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed This study
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

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.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

31 people

The number who actually took part.

Started

Oct 2019

Finished

Dec 2025

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

12 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria for the Randomized Placebo-Controlled Period : * 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. Inclusion Criteria for the Open-Label Period: * 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.

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Conditions

The condition(s) this trial relates to.

WHIM syndrome WHIM syndrome 1

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Whim syndrome

Contacts and locations

Locations

  • Aarhus University Hospital

    Aarhus, 8000, Denmark

  • Academician I.P. Pavlov First Saint Petersburg State Medical University

    Saint Pertersburg, 197022, Russia

  • CHU Paris Est, Hôpital d'Enfants Armand-Trousseau

    Paris, 75571, France

  • CHU de Lyon, Institut d'Hematologie et d'Oncologie Pediatrique

    Lyon, Rhne, 69008, France

  • California Dermatology Institute

    Thousand Oaks, California, 91320, United States

  • Children's Health Queensland Hospital

    South Brisbane, Queensland, 4101, Australia

  • Cukurova University Faculty of Medicine

    Sarıçam, Adana, 1330, Turkey (Türkiye)

  • Dmitry Rogachev National Research Center of Pediatric Hematology, Oncology and Immunology

    Moscow, 117997, Russia

  • Emma Children's Hospital Academic Medical Center (AMC)

    Amsterdam, 1105 AZ, Netherlands

  • HaEmek Medical Center

    Afula, 1834111, Israel

  • Hopital Necker-Enfants Malades

    Paris, 75743, France

  • Hospital Sant Joan de Deu Barcelona

    Barcelona, Esplugues de Llobregat, 8950, Spain

  • Hospital Universitario Virgen del Rocío

    Seville, Sevilla, 41013, Spain

  • Johns Hopkins University Medical Center

    Baltimore, Maryland, 21224, United States

  • Medical University of Vienna - Medizinische Universität Wien

    Vienna, 1090, Austria

  • Seoul National University Hospital, Children's Hospital

    Seoul, 03080, South Korea

  • University of California San Diego Health/Rady Children's Hospital

    San Diego, California, 92123, United States

  • University of Debrecen, Affiliated Department of Infectology

    Debrecen, Hajdú-Bihar, H-4031, Hungary

  • University of Iowa

    Iowa City, Iowa, 52242, United States

  • University of Texas Southwestern Medical Center

    Dallas, Texas, 75390-9020, United States

  • University of Washington Medical Center

    Seattle, Washington, 98195, United States

  • Università degli Studi di Brescia, Scienze Cliniche e Sperimentali

    Brescia, Piazza Del Mercato, 25123, Italy

  • Wesley Hospital

    Auchenflower, Queensland, 4006, Australia

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