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
NCT ID: NCT06056297
Last Updated: 2025-11-14
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
PHASE3
176 participants
INTERVENTIONAL
2024-06-06
2027-11-30
Brief Summary
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Detailed Description
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Conditions
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Keywords
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
QUADRUPLE
Study Groups
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Mavorixafor
Participants will receive mavorixafor orally once daily starting from Day 1 through Week 52.
Mavorixafor
Mavorixafor will be administered per schedule specified in the arm description.
Placebo
Participants will receive placebo to match mavorixafor orally once daily starting from Day 1 through Week 52.
Placebo
Placebo will be administered per schedule specified in the arm description.
Interventions
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Mavorixafor
Mavorixafor will be administered per schedule specified in the arm description.
Placebo
Placebo will be administered per schedule specified in the arm description.
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* 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.
Exclusion Criteria
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.
12 Years
ALL
No
Sponsors
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X4 Pharmaceuticals
INDUSTRY
Responsible Party
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Principal Investigators
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Vice President Global Head of Clinical Development and Safety
Role: STUDY_DIRECTOR
X4 Pharmaceuticals
Locations
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Phoenix Children's Hospital
Phoenix, Arizona, United States
UC Irvine
Irvine, California, United States
Orso Health
La Jolla, California, United States
University of California, Los Angeles-UCLA
Los Angeles, California, United States
Orso Health
Torrance, California, United States
University of Southern Florida
St. Petersburg, Florida, United States
University of Iowa
Iowa City, Iowa, United States
University of Michigan
Ann Arbor, Michigan, United States
Washington University
St Louis, Missouri, United States
Duke University Medical Center
Durham, North Carolina, United States
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
UT Southwestern Medical Center
Dallas, Texas, United States
Cook Children's Medical Center
Fort Worth, Texas, United States
University of Utah
Salt Lake City, Utah, United States
Froedtert And Medical College Of Wisconsin
Milwaukee, Wisconsin, United States
Hospital El Cruce
Buenos Aires, , Argentina
Instituto de Investigaciones Clínicas Córdoba
Córdoba, , Argentina
Santorio 9 de Julio
San Miguel de Tucumán, , Argentina
Perth Children's Hospital
Nedlands, , Australia
Icon Cancer Centre Southport
Southport, , Australia
Ballarat Oncology And Haematology Services
Wendouree, , Australia
University of Alberta Hospital
Edmonton, , Canada
Clinica IMBANACO S.A.S
Cali, , Colombia
The University Hospital Brno
Brno, , Czechia
Motol University Hospital
Prague, , Czechia
Institute of Hematology and Pediatric Oncology - Lyon
Lyon, , France
Hôpital Saint Louis
Paris, , France
Hospital Armand Trousseau Ap-Hp
Paris, , France
Hôpital Necker - Enfants Malade
Paris, , France
Centre Hospitalier Universitaire de Rennes
Rennes, , France
LTD "Israeli-Georgian Medical Research Clinic Healthycore"
Tbilisi, , Georgia
LEPL The First University Clinic of Tbilisi State Medical University
Tbilisi, , Georgia
JSC K. Eristavi National Center of Experimental and Clinical Surgery
Tbilisi, , Georgia
LTD Multiprofile Clinic Concilium Medulla
Tbilisi, , Georgia
Klinik und Poliklinik fur Kinder- und Jugendmedizin, Universitatsklinikum Carl Gustav Carus an der TU
Dresden, , Germany
Universitaetsklinikum Carl Gustav Carus Tu Dresden
Dresden, , Germany
Hellenic Airforce 251 General Hospital
Athens, , Greece
University General Hospital of Heraklion
Heraklion, , Greece
The Olympion Private General Clinic
Pátrai, , Greece
AHEPA General Hospital of Thessaloniki
Thessaloniki, , Greece
CMC Deli Klinika
Budapest, , Hungary
Semmelweis University
Budapest, , Hungary
Teaching Hospital Markusovszky
Szombathely, , Hungary
Indira Gandhi Institute Of Medical Sciences
Patna, Bihar, India
Nirmal Hospital Private Limited
Surat, Gujarat, India
Pacific Medical College and Hospital
Udaipur, Rajasthan, India
Yashoda Hospital
Hyderabad, Telangana, India
Ruth Rappaport Children's Hospital, Rambam Health Corporation
Haifa, , Israel
Hadassah University Hospital-ein Kerem
Jerusalem, , Israel
Schneider Children's Medical Center of Israel
Petah Tikva, , Israel
The Chaim Sheba Medical Center
Ramat Gan, , Israel
A. O. U. delle Marche
Torrette, Ancona, Italy
ASST Spedali Civili di Brescia
Brescia, , Italy
IRCCS Instituto G. Gaslini
Genova, , Italy
Istituto Scientifico Romagnolo Per Lo Studio E La Cura Dei Tumori (IRST) Dino Amadori
Meldola, , Italy
IRCCS San Gerardo Dei Tintori
Monza, , Italy
Bambino Gesu Children's Hospital
Roma, , Italy
Hospital Pakar Kanak-Kanak UKM
Kuala Lumpur, , Malaysia
Unidade Local de Saúde da Região de Aveiro
Aveiro, , Portugal
Unidade Local de Saude Coimbra - Paediatric Hospital
Coimbra, , Portugal
Unidade Local de Saude Coimbra - Quinta dos Vales
Coimbra, , Portugal
Unidade Local de Saude Santa Maria
Lisbon, , Portugal
Spitalul Clinic Colentina Site 1
Bucharest, , Romania
Spitalul Clinic Colentina Site 2
Bucharest, , Romania
Institutul Clinic Fundeni
Bucharest, , Romania
Institutul Clinic Fundeni
Bucharest, , Romania
Spitalul Clinic Județean de Urgență
Târgu Mureş, , Romania
University Clinical Centre of Serbia
Belgrade, , Serbia
ICO Badalona - Institut Català d'Oncologia
Barcelona, , Spain
Hospital Del Mar
Barcelona, , Spain
Hospital San Pedro De Alcantara
Cáceres, , Spain
Hospital Universitario Reina Sofía
Córdoba, , Spain
Hospital Universitario de Gran Canaria Doctor Negrín
Las Palmas de Gran Canaria, , Spain
Hospital De La Princesa
Madrid, , Spain
Hospital Universitario Infanta Leonor
Madrid, , Spain
Hospital Universitario Fundacion Jimenez Diaz
Madrid, , Spain
Hospital Universitario Marqués de Valdecilla
Santander, , Spain
Hospital Universitario Virgen del Rocio
Seville, , Spain
Hospital Universitario y Politecnico de la Fe
Valencia, , Spain
Inselspital - University Hospital Bern Site 1
Bern, , Switzerland
Inselspital - University Hospital Bern Site 2
Bern, , Switzerland
Ramathibodi Hospital
Bangkok, , Thailand
Siriraj Hospital
Bangkok, , Thailand
Namik Kemal University (Tekirdağ Namık Kemal Üniversitesi)
Tekirdağ, Süleymanpaşa, Turkey (Türkiye)
Dr. Abdurrahman Yurtaslan Ankara Oncology Training And Research Hospital
Ankara, , Turkey (Türkiye)
Ankara University School of Medicine
Ankara, , Turkey (Türkiye)
Uludag University Faculty Of Medicine
Nilufer, , Turkey (Türkiye)
Ondokuz Mayis University Medical Faculty Hospital
Samsun, , Turkey (Türkiye)
Lviv Regional Clinical Diagnostic Centre
Lviv, Oblast, Ukraine
Medical Center Dobrobut-Clinic LLC
Kyiv, , Ukraine
Medical Center Ok Clinic of International Institute of Clinical Research LLC
Kyiv, , Ukraine
Llc Onto-Med
Kyiv, , Ukraine
Communal non-commercial enterprise "Ternopil City Children's Communal Hospital"
Ternopil, , Ukraine
Medical Center The Clinic of Hospodarskyy LLC
Ternopil, , Ukraine
University Hospital of Wales
Cardiff, , United Kingdom
Cardiff And Vale University Health Board
London, , United Kingdom
Royal Free London Nhs Foundation Trust Royal Free Hospital
London, , United Kingdom
King's College Hospital
London, , United Kingdom
Imperial College Healthcare Nhs Trust - Hammersmith Hospital
London, , United Kingdom
Great Ormond street Hospital for Children NHS Trust
London, , United Kingdom
Countries
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Central Contacts
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Other Identifiers
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2023-508482-32-00
Identifier Type: CTIS
Identifier Source: secondary_id
X4P-001-110
Identifier Type: -
Identifier Source: org_study_id