A Study of Mavorixafor in Participants With Congenital and Acquired Primary Autoimmune and Idiopathic Chronic Neutropenic Disorders Who Are Experiencing Recurrent and/or Serious Infections

Last updated: April 14, 2025
Sponsor: X4 Pharmaceuticals
Overall Status: Active - Recruiting

Phase

3

Condition

Neutropenia

Treatment

Placebo

Mavorixafor

Clinical Study ID

NCT06056297
X4P-001-110
2023-508482-32-00
  • Ages > 12
  • All Genders

Study Summary

The purpose of this study is to demonstrate the efficacy and evaluate the safety and tolerability of mavorixafor in participants with congenital or acquired primary autoimmune and idiopathic chronic neutropenic disorders who are experiencing recurrent and/or serious infections as assessed by demonstrating its clinical benefit and increasing levels of circulating neutrophils.

Eligibility Criteria

Inclusion

Key Inclusion Criteria:

  • Diagnosis of congenital or acquired primary autoimmune and idiopathic chronicneutropenic disorder ≥6 months prior to the screening visit that is not attributableto 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), Kostmanndisease

  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 thatmay be eligible for enrollment can be clarified and approved upon discussionwith study Medical Monitor and Sponsor.

  • Have an ANC <1000 cells/µL during screening (single ANC value from hematology) andconfirmed trough mean ANC (mean value of multiple ANC measurements over 6 hours) atbaseline visit, with no clinical evidence of systemic infection.

  • Prior history of recurrent and/or serious infections during the 12 months precedingthe screening visit (that is, suffering sequelae of chronic neutropenia), as definedby having at least 2 infections in the last 12 months that meet the followingcriteria:

  • Infection requiring the use of antibiotics (intravenous [IV]/oral); OR

  • Infection requiring a visit to healthcare facility (including but not limited toemergency room visit, urgent care facility, primary care physician's office, orin-patient hospitalization);

AND for all potential participants:

  • Infections considered by the Investigator to be likely related to the potentialparticipant's CN disorder.

  • Participants who are on G-CSF or other active background therapy must have beenreceiving these therapies during the previous 12 months while continuing to sufferfrom infections, be on a stable dose and dosing schedule for ≥4 weeks prior toscreening 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 therapydoses/regimens stable (other than for safety reasons) for the duration of the study.

Exclusion

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, rheumatoidarthritis, inflammatory bowel disease, graft-versus-host disease, thyroiddisease

  5. Nutritional deficiency, for example, vitamin B12, folic acid, copper, caloricmalnutrition

  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:

  4. Isolated with a cyclic presentation, for example, elastase, neutrophilexpressed (ELANE)

  5. Associated with immune dysregulation, for example, common variableimmunodeficiency (CVID), autoimmune lymphoproliferative syndrome (ALPS),familial hemophagocytic lymphohistiocytosis, Chédiak-Higashi syndrome,GATA-binding protein 2 (GATA2) deficiency syndrome

  6. Associated with bone marrow failure, for example, Fanconi anemia,Diamond-Blackfan anemia

  7. Neutropenia associated with a Duffy-null phenotype (formerly known as benignethnic neutropenia). However, a participant with an autosomal dominantpathogenic variant in a gene associated with CN on a Duffy-null background maybe eligible for inclusion

  • A medical or personal condition that may potentially compromise the safety of theparticipant, may preclude the participant's successful completion of the clinicalstudy, or could, in the opinion of the Investigator or the Sponsor, interfere withthe 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) inthe past 6 months.

  • Participants taking pegylated-G-CSF unless they have a diagnosis of congenitalneutropenia confirmed at screening.

  • Participant is currently taking or has taken other investigational drug <30 daysprior to the screening visit or 5 half-lives, whichever is longer.

Note: Other protocol-defined inclusion and exclusion criteria may apply.

Study Design

Total Participants: 150
Treatment Group(s): 2
Primary Treatment: Placebo
Phase: 3
Study Start date:
June 06, 2024
Estimated Completion Date:
August 31, 2026

Study Description

All participants will continue their pre-study background therapy, defined as the participant's current treatment regimen. Options include, but are not limited to, granulocyte-colony stimulating factor (G-CSF), immunoglobulin replacement therapy, prophylactic antibiotics, or "watchful waiting".

Connect with a study center

  • Instituto de Investigaciones Clínicas Córdoba

    Córdoba, X5000AAW
    Argentina

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  • Santorio 9 de Julio

    San Miguel de Tucumán, T4000IIH
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  • Icon Cancer Centre Southport

    Southport, 4215
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  • University of Alberta Hospital

    Edmonton, AB T6G 2R3
    Canada

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  • The University Hospital Brno

    Brno, 62500
    Czechia

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  • Motol University Hospital

    Prague, 15006
    Czechia

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  • Institute of Hematology and Pediatric Oncology - Lyon

    Lyon, 69008
    France

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  • Hospital Armand Trousseau Ap-Hp

    Paris, 75012
    France

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  • Hospital Saint Louis

    Paris, 75010
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  • Hôpital Necker - Enfants Malade

    Paris, 75015
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  • Hôpital Saint Louis

    Paris, 75010
    France

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  • Hôspital Necker - Enfants Malade

    Paris, 75015
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  • Centre Hospitalier Universitaire de Rennes

    Rennes, 75033
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  • JSC K. Eristavi National Center of Experimental and Clinical Surgery

    Tbilisi, 159
    Georgia

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  • LEPL The First University Clinic of Tbilisi State Medical University

    Tbilisi, 141
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  • LTD "Israeli-Georgian Medical Research Clinic Healthycore"

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  • LTD Multiprofile Clinic Concilium Medulla

    Tbilisi, 186
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  • Klinik und Poliklinik fur Kinder- und Jugendmedizin, Universitatsklinikum Carl Gustav Carus an der TU

    Dresden, 01307
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  • Universitaetsklinikum Carl Gustav Carus Tu Dresden

    Dresden, 01307
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  • Hellenic Airforce 251 General Hospital

    Athens, 10177
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  • University General Hospital of Heraklion

    Iraklio, 71500
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  • AHEPA General Hospital of Thessaloniki

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  • CMC Deli Klinika

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  • Semmelweis University

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  • Teaching Hospital Markusovszky

    Szombathely, 9700
    Hungary

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  • Indira Gandhi Institute Of Medical Sciences

    Patna, Bihar 800014
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    Hyderabad, Telangana 500084
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  • Ruth Rappaport Children's Hospital, Rambam Health Corporation

    Haifa, 3109601
    Israel

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  • A. O. U. delle Marche

    Torrette, Ancona 60126
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  • Ospedale Riuniti Torrette di Ancona ( Riuniti Torrette Hospital in Ancona)

    Torrette, Ancona 60126
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  • ASST Spedali Civili di Brescia

    Brescia, 25121
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  • IRCCS Instituto G. Gaslini

    Genova, 16147
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  • Istituto Scientifico Romagnolo Per Lo Studio E La Cura Dei Tumori (IRST) Dino Amadori

    Meldola, 47014
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  • IRCCS San Gerardo Dei Tintori

    Monza, 20900
    Italy

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  • Unidade Local de Saúde da Região de Aveiro

    Aveiro, 3810-164
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  • Unidade Local de Saude Coimbra - Paediatric Hospital

    Coimbra, 3000-602
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  • Unidade Local de Saude Coimbra - Quinta dos Vales

    Coimbra, 3041-801
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  • Institutul Clinic Fundeni

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  • Spitalul Clinic Colentina Site 1

    Bucharest, 020125
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  • Spitalul Clinic Colentina Site 2

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  • Spitalul Clinic Județean de Urgență

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  • University Clinical Centre of Serbia

    Belgrade, 11000
    Serbia

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  • Hospital Del Mar

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    Spain

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  • ICO Badalona - Institut Català d'Oncologia

    Barcelona, PC 08916
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  • Hospital San Pedro De Alcantara

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  • Hospital Universitario Reina Sofía

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  • Hospital De La Princesa

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    Spain

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  • Hospital Universitario Fundacion Jimenez Diaz

    Madrid, 28040
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  • Hospital Universitario Infanta Leonor

    Madrid, 28031
    Spain

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  • Hospital Universitario Son Espases

    Mallorca, PC07120
    Spain

    Site Not Available

  • Hospital Universitario Marqués de Valdecilla

    Santander, 39008
    Spain

    Active - Recruiting

  • Hospital Universitario Virgen del Rocio

    Sevilla, 41013
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  • Inselspital - University Hospital Bern Site 1

    Bern, 3010
    Switzerland

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  • Inselspital - University Hospital Bern Site 2

    Bern, 3010
    Switzerland

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  • Ramathibodi Hospital

    Bangkok, 10400
    Thailand

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  • Siriraj Hospital

    Bangkok, 10700
    Thailand

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  • Ankara University School of Medicine

    Ankara, 6620
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  • Dr. Abdurrahman Yurtaslan Ankara Oncology Training And Research Hospital

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  • Uludag University Faculty Of Medicine

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  • Ondokuz Mayis University Medical Faculty Hospital

    Samsun, 55200
    Turkey

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  • Lviv Regional Clinical Diagnostic Centre

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  • Llc Onto-Med

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  • Medical Center Dobrobut-Clinic LLC

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

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  • Medical Center The Clinic of Hospodarskyy LLC

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  • Cardiff And Vale University Health Board

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  • Imperial College Healthcare Nhs Trust - Hammersmith Hospital

    London, W12 0HS
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  • King's College Hospital

    London, SE5 9RS
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  • Phoenix Children's Hospital

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  • Orso Health

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  • Orso Health

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  • Duke University Medical Center

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  • Cincinnati Children's Hospital Medical Center

    Cincinnati, Ohio 45229
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