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New drug aims to boost immune cells in rare blood disorder

NCT ID NCT06056297

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 study
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
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 Jun 24, 2026 · Last updated Aug 07, 2026 · Updated 3 times

Summary

This Phase 3 study tests whether the drug mavorixafor can reduce serious infections and increase neutrophil levels in people with chronic neutropenia—a condition where the body doesn't make enough infection-fighting white blood cells. About 176 participants will receive either mavorixafor or a placebo. The goal is to see if the drug safely lowers infection rates and improves blood cell counts.

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 a new treatment option to reduce serious infections and improve quality of life for people with chronic neutropenia.
What could go wrong
This is a Phase 3 trial, but results are not guaranteed. The drug may not work for all types of neutropenia, and side effects are possible. Success depends on showing clear benefit over placebo.
Why investors are watching

X4 Pharmaceuticals is running a phase 3 trial of its drug mavorixafor in 176 people with chronic neutropenic disorders, conditions where the body has too few infection-fighting white blood cells. For a micro-cap company, this late-stage readout is a central test of whether the drug works and can reach the market, so the outcome will heavily shape the company's future.

If it works: If the trial shows mavorixafor raises neutrophil levels and reduces infections, X4 could gain a path to regulatory approval and its first commercial product. That result would validate the drug's value for a patient group with few treatment options.

If it fails: Phase 3 trials often fail, and a negative or delayed result could leave X4 without a clear path forward, hurting its ability to fund further development. The company's small size means it has limited room to absorb a setback.

AI-written from the trial record. Speculative, and not investment advice.

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

About 176 people

The number the study aims to enrol. It can still change while the study runs.

Started

Jun 2024

Expected to finish

Nov 2027

An estimate. End dates often move.

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.

Key Inclusion Criteria: * Diagnosis of congenital or acquired primary autoimmune and idiopathic chronic neutropenic disorder ≥6 months prior to the screening visit that is not attributable to medications, active or recent infections or malignancy. * Congenital Neutropenia, including but not limited to these classifications: 1. Isolated with a permanent (non-cyclic) presentation, for example, elastase, neutrophil expressed (ELANE), colony stimulating factor 3 receptor (CSF3R), C-X-C chemokine receptor 2 (CXCR2), Wiskott-Aldrich syndrome (WAS) 2. Associated with extra-hematologic manifestations, for example, Barth syndrome, Cohen syndrome, glucose-6-phosphatase catalytic subunit 3 (G6PC3), Kostmann disease 3. Associated with metabolic disorders, for example, glycogen storage disease 1b (GSD1b) 4. Shwachman-Diamond syndrome * Acquired Primary Neutropenia 1. Chronic idiopathic neutropenia 2. Primary autoimmune neutropenia. Other chronic neutropenia (CN) disorders that may be eligible for enrollment can be clarified and approved upon discussion with study Medical Monitor. * Have an ANC \<1000 cells/µL during screening (single ANC value from hematology) and confirmed trough mean ANC (mean value of multiple ANC measurements over 6 hours) at baseline visit, with no clinical evidence of systemic infection. * Prior history of recurrent and/or serious infections during the 12 months preceding the screening visit (that is, suffering sequelae of chronic neutropenia), as defined by having at least 2 infections in the last 12 months that meet the following criteria: * Infection requiring the use of antibiotics (intravenous \[IV\]/oral); OR * Infection requiring a visit to healthcare facility (including but not limited to emergency room visit, urgent care facility, primary care physician's office, or in-patient hospitalization); AND for all potential participants: * Infections considered by the Investigator to be likely related to the potential participant's CN disorder. * Participants who are on G-CSF or other active background therapy must have been receiving these therapies during the previous 12 months while continuing to suffer from infections, be on a stable dose and dosing schedule for ≥4 weeks prior to screening visit and remain on this dose and dosing schedule throughout the study (unless ANC \>10,000 cells/µL for ≥4 weeks). * Participants must be willing to keep their G-CSF or other background therapy doses/regimens stable (other than for safety reasons) for the duration of the study. Key Exclusion Criteria: * A diagnosis of secondary neutropenia including those due to: 1. Hypersplenism 2. Infection 3. Malignancy 4. Autoimmune disease, for example, systemic lupus erythematosus, rheumatoid arthritis, inflammatory bowel disease, graft-versus-host disease, thyroid disease 5. Nutritional deficiency, for example, vitamin B12, folic acid, copper, caloric malnutrition 6. Drug-induced cause, for example, chemotherapy, clozapine, antiretrovirals, antibiotics, monoclonal antibodies. * A diagnosis of any of the following: 1. Aplastic anemia 2. Warts, hypogammaglobulinemia, infections, and myelokathexis (WHIM) syndrome 3. Certain CNs, including but not limited to these classifications are excluded: 1. Isolated with a cyclic presentation, for example, elastase, neutrophil expressed (ELANE) 2. Associated with immune dysregulation, for example, common variable immunodeficiency (CVID), autoimmune lymphoproliferative syndrome (ALPS), familial hemophagocytic lymphohistiocytosis, Chédiak-Higashi syndrome, GATA-binding protein 2 (GATA2) deficiency syndrome 3. Associated with bone marrow failure, for example, Fanconi anemia, Diamond-Blackfan anemia 4. Neutropenia associated with a Duffy-null phenotype (formerly known as benign ethnic neutropenia). However, a participant with an autosomal dominant pathogenic variant in a gene associated with CN on a Duffy-null background may be eligible for inclusion * A medical or personal condition that may potentially compromise the safety of the participant, may preclude the participant's successful completion of the clinical study, or could, in the opinion of the Investigator or the Medical Monitor, interfere with the objectives of the study. * Received more than 1 dose of mavorixafor in the past. * Received C-X-C chemokine receptor 4 (CXCR4) antagonist (other than mavorixafor) in the past 6 months. * Participants taking pegylated-G-CSF unless they have a diagnosis of congenital neutropenia confirmed at screening. * Participant is currently taking or has taken other investigational drug \<30 days prior to the screening visit or 5 half-lives, whichever is longer. Note: Other protocol-defined inclusion and exclusion criteria may apply.

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Conditions

The condition(s) this trial relates to.

autoimmune disease Barth syndrome Cohen syndrome constitutional neutropenia Kostmann syndrome neutropenia nonimmune chronic idiopathic neutropenia of adults Shwachman-Diamond Syndrome

As listed by the trial registrant

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

Neutropenia

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The study's own enquiry address

    This study publishes an address for enquiries. See it below .

  2. The places running it

    107 sites in 24 countries. The list below names each one and where it is.

  3. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  4. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Study contacts

  • Contact

    Email: •••••@•••••

Locations

  • A. O. U. delle Marche

    RECRUITING

    Torrette, Ancona, 60126, Italy

  • AHEPA General Hospital of Thessaloniki

    RECRUITING

    Thessaloniki, 54636, Greece

  • AOU Città della salute e della scienza di Torino

    RECRUITING

    Torino, 10126, Italy

  • ASST Spedali Civili di Brescia

    RECRUITING

    Brescia, 25121, Italy

  • Ankara University School of Medicine

    RECRUITING

    Ankara, 6620, Turkey (Türkiye)

  • BC Children's Hospital

    RECRUITING

    Vancouver, V5Z 4H4, Canada

  • Ballarat Oncology And Haematology Services

    RECRUITING

    Wendouree, 3355, Australia

  • Bambino Gesu Children's Hospital

    RECRUITING

    Roma, PC 00165, Italy

  • Boston Children's Cancer Center

    RECRUITING

    Boston, Massachusetts, 02143, United States

  • CMC Deli Klinika

    RECRUITING

    Budapest, 1012, Hungary

  • Cardiff And Vale University Health Board

    RECRUITING

    London, CF14 4XW, United Kingdom

  • Centre Hospitalier Universitaire de Rennes

    RECRUITING

    Rennes, 75033, France

  • Children's Healthcare of Atlanta (CHOA)

    RECRUITING

    Atlanta, Georgia, 30322, United States

  • Cincinnati Children's Hospital Medical Center

    RECRUITING

    Cincinnati, Ohio, 45229, United States

  • Cleveland Clinic

    RECRUITING

    Cleveland, Ohio, 44195, United States

  • Clinica IMBANACO S.A.S

    RECRUITING

    Cali, 76004, Colombia

  • Clínica del Country

    RECRUITING

    Chapinero, 0813, Colombia

  • Communal non-commercial enterprise "Ternopil City Children's Communal Hospital"

    RECRUITING

    Ternopil, 46000, Ukraine

  • Cook Children's Medical Center

    RECRUITING

    Fort Worth, Texas, 76104, United States

  • Dr. Abdurrahman Yurtaslan Ankara Oncology Training And Research Hospital

    RECRUITING

    Ankara, 6000, Turkey (Türkiye)

  • Duke University Medical Center

    RECRUITING

    Durham, North Carolina, 27710, United States

  • Froedtert And Medical College Of Wisconsin

    RECRUITING

    Milwaukee, Wisconsin, 53226, United States

  • Fundacion Cardiovascular de Colombia-Instituto del Corazon Florida Blanca

    RECRUITING

    Floridablanca, Santander Department, 681003, Colombia

  • Great Ormond street Hospital for Children NHS Trust

    RECRUITING

    London, WC1N 1DZ, United Kingdom

  • Hadassah University Hospital-ein Kerem

    RECRUITING

    Jerusalem, 9112001, Israel

  • Hellenic Airforce 251 General Hospital

    RECRUITING

    Athens, 10177, Greece

  • Hospital Armand Trousseau Ap-Hp

    RECRUITING

    Paris, 75012, France

  • Hospital De La Princesa

    RECRUITING

    Madrid, 28006, Spain

  • Hospital Del Mar

    RECRUITING

    Barcelona, PC08003, Spain

  • Hospital El Cruce

    RECRUITING

    Buenos Aires, B1888AAE, Argentina

  • Hospital Pablo Tobón Uribe

    RECRUITING

    Medellín, 500034, Colombia

  • Hospital Pakar Kanak-Kanak UKM

    RECRUITING

    Kuala Lumpur, 56000, Malaysia

  • Hospital San Pedro De Alcantara

    RECRUITING

    Cáceres, 10003, Spain

  • Hospital Universitario Fundacion Jimenez Diaz

    RECRUITING

    Madrid, 28040, Spain

  • Hospital Universitario Infanta Leonor

    WITHDRAWN

    Madrid, 28031, Spain

  • Hospital Universitario Marqués de Valdecilla

    RECRUITING

    Santander, 39008, Spain

  • Hospital Universitario Reina Sofía

    WITHDRAWN

    Córdoba, 14004, Spain

  • Hospital Universitario Virgen del Rocio

    RECRUITING

    Seville, 41013, Spain

  • Hospital Universitario de Gran Canaria Doctor Negrín

    RECRUITING

    Las Palmas de Gran Canaria, 35010, Spain

  • Hospital Universitario y Politecnico de la Fe

    RECRUITING

    Valencia, 46026, Spain

  • Hôpital Necker - Enfants Malade

    RECRUITING

    Paris, 75015, France

  • Hôpital Saint Louis

    RECRUITING

    Paris, 75010, France

  • ICO Badalona - Institut Català d'Oncologia

    RECRUITING

    Barcelona, PC 08916, Spain

  • IRCCS Instituto G. Gaslini

    RECRUITING

    Genova, 16147, Italy

  • IRCCS San Gerardo Dei Tintori

    RECRUITING

    Monza, 20900, Italy

  • Icon Cancer Centre Southport

    RECRUITING

    Southport, 4215, Australia

  • Imperial College Healthcare Nhs Trust - Hammersmith Hospital

    RECRUITING

    London, W12 0HS, United Kingdom

  • Indira Gandhi Institute Of Medical Sciences

    WITHDRAWN

    Patna, Bihar, 800014, India

  • Inselspital - University Hospital Bern Site 1

    RECRUITING

    Bern, 3010, Switzerland

  • Inselspital - University Hospital Bern Site 2

    RECRUITING

    Bern, 3010, Switzerland

  • Institut Universitaire de Cancérologie de Toulouse - Oncopôle

    RECRUITING

    Toulouse, 31059, France

  • Institute of Hematology and Pediatric Oncology - Lyon

    RECRUITING

    Lyon, 69008, France

  • Instituto de Investigaciones Clínicas Córdoba

    RECRUITING

    Córdoba, X5000AAW, Argentina

  • Institutul Clinic Fundeni

    RECRUITING

    Bucharest, 022328, Romania

  • Institutul Clinic Fundeni

    RECRUITING

    Bucharest, 22328, Romania

  • Istituto Scientifico Romagnolo Per Lo Studio E La Cura Dei Tumori (IRST) Dino Amadori

    RECRUITING

    Meldola, 47014, Italy

  • JSC K. Eristavi National Center of Experimental and Clinical Surgery

    RECRUITING

    Tbilisi, 159, Georgia

  • King's College Hospital

    RECRUITING

    London, SE5 9RS, United Kingdom

  • Klinik und Poliklinik fur Kinder- und Jugendmedizin, Universitatsklinikum Carl Gustav Carus an der TU

    RECRUITING

    Dresden, 01307, Germany

  • LEPL The First University Clinic of Tbilisi State Medical University

    RECRUITING

    Tbilisi, 141, Georgia

  • LTD "Israeli-Georgian Medical Research Clinic Healthycore"

    RECRUITING

    Tbilisi, 112, Georgia

  • LTD Multiprofile Clinic Concilium Medulla

    RECRUITING

    Tbilisi, 186, Georgia

  • Llc Onto-Med

    RECRUITING

    Kyiv, 3022, Ukraine

  • Lviv Regional Clinical Diagnostic Centre

    RECRUITING

    Lviv, Oblast, 79010, Ukraine

  • Medical Center Dobrobut-Clinic LLC

    RECRUITING

    Kyiv, 03151, Ukraine

  • Medical Center Ok Clinic of International Institute of Clinical Research LLC

    RECRUITING

    Kyiv, 2091, Ukraine

  • Medical Center The Clinic of Hospodarskyy LLC

    RECRUITING

    Ternopil, 46002, Ukraine

  • Motol University Hospital

    RECRUITING

    Prague, 15006, Czechia

  • Namik Kemal University (Tekirdağ Namık Kemal Üniversitesi)

    RECRUITING

    Tekirdağ, Süleymanpaşa, 59030, Turkey (Türkiye)

  • Nirmal Hospital Private Limited

    WITHDRAWN

    Surat, Gujarat, 395002, India

  • Ondokuz Mayis University Medical Faculty Hospital

    RECRUITING

    Samsun, 55200, Turkey (Türkiye)

  • Orso Health

    RECRUITING

    La Jolla, California, 92037, United States

  • Orso Health

    WITHDRAWN

    Torrance, California, 90505, United States

  • Pacific Medical College and Hospital

    WITHDRAWN

    Udaipur, Rajasthan, 313001, India

  • Perth Children's Hospital

    RECRUITING

    Nedlands, 6009, Australia

  • Phoenix Children's Hospital

    RECRUITING

    Phoenix, Arizona, 85016, United States

  • Ramathibodi Hospital

    RECRUITING

    Bangkok, 10400, Thailand

  • Royal Free London Nhs Foundation Trust Royal Free Hospital

    RECRUITING

    London, NW3 2QG, United Kingdom

  • Ruth Rappaport Children's Hospital, Rambam Health Corporation

    RECRUITING

    Haifa, 3109601, Israel

  • Santorio 9 de Julio

    RECRUITING

    San Miguel de Tucumán, T4000IIH, Argentina

  • Sarah Cannon Research Institute

    RECRUITING

    Woodland, Texas, 77380, United States

  • Schneider Children's Medical Center of Israel

    RECRUITING

    Petah Tikva, 4920235, Israel

  • Semmelweis University

    RECRUITING

    Budapest, 1085, Hungary

  • Siriraj Hospital

    RECRUITING

    Bangkok, 10700, Thailand

  • Spitalul Clinic Colentina Site 1

    RECRUITING

    Bucharest, 020125, Romania

  • Spitalul Clinic Colentina Site 2

    RECRUITING

    Bucharest, 020125, Romania

  • Spitalul Clinic Județean de Urgență

    RECRUITING

    Târgu Mureş, 540136, Romania

  • St. Jude Childrens; Research Hospital

    RECRUITING

    Memphis, Tennessee, 38105, United States

  • Teaching Hospital Markusovszky

    RECRUITING

    Szombathely, 9700, Hungary

  • Texas Children's Hospital/ Baylor College of Medicine

    RECRUITING

    Houston, Texas, 77030, United States

  • The Chaim Sheba Medical Center

    RECRUITING

    Ramat Gan, 5265601, Israel

  • The Children's Hospital of Philadelphia

    RECRUITING

    Philadelphia, Pennsylvania, 19104, United States

  • The Olympion Private General Clinic

    RECRUITING

    Pátrai, 26443, Greece

  • The University Hospital Brno

    RECRUITING

    Brno, 62500, Czechia

  • UC Irvine

    RECRUITING

    Irvine, California, 92697, United States

  • UT Southwestern Medical Center

    RECRUITING

    Dallas, Texas, 75390, United States

  • Uludag University Faculty Of Medicine

    RECRUITING

    Nilufer, Turkey (Türkiye)

  • Unidade Local de Saude Coimbra - Paediatric Hospital

    RECRUITING

    Coimbra, 3000-602, Portugal

  • Unidade Local de Saude Coimbra - Quinta dos Vales

    RECRUITING

    Coimbra, 3041-801, Portugal

  • Unidade Local de Saude Santa Maria

    RECRUITING

    Lisbon, 1649-028, Portugal

  • Unidade Local de Saúde da Região de Aveiro

    RECRUITING

    Aveiro, 3810-164, Portugal

  • Universitaetsklinikum Carl Gustav Carus Tu Dresden

    RECRUITING

    Dresden, 01307, Germany

  • Universitair Ziekenhuis Leuven

    RECRUITING

    Leuven, 3000, Belgium

  • University Clinical Centre of Serbia

    RECRUITING

    Belgrade, 11000, Serbia

  • University General Hospital of Heraklion

    RECRUITING

    Heraklion, 71500, Greece

  • University Hospital of Wales

    RECRUITING

    Cardiff, CF14 4XW, United Kingdom

  • University of Alberta Hospital

    RECRUITING

    Edmonton, AB T6G 2R3, Canada

  • University of California, Los Angeles-UCLA

    RECRUITING

    Los Angeles, California, 90095, United States

  • University of Iowa

    RECRUITING

    Iowa City, Iowa, 52242, United States

  • University of Michigan

    RECRUITING

    Ann Arbor, Michigan, 48109, United States

  • University of Southern Florida

    RECRUITING

    St. Petersburg, Florida, 33701, United States

  • University of Utah

    RECRUITING

    Salt Lake City, Utah, 84112, United States

  • Washington University

    RECRUITING

    St Louis, Missouri, 63130, United States

  • Yashoda Hospital

    TERMINATED

    Hyderabad, Telangana, 500084, India

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