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

NCT06056297 | Recruiting Phase 3 DMC FDA Drug

• 2 other identifiers: X4P-001-1102023-508482-32-00
• Study Type: interventional
• Target: 176
• Locations: 24 countries
• Sites: 112

Brief 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.

Conditions

Neutropenia

Keywords

Mavorixafor; Chronic neutropenia; Chronic idiopathic neutropenia; Severe Congenital Neutropenia (SCN); C-X-C chemokine receptor 4 (CXCR4); Congenital and acquired neutropenia; Autoimmune disease; Cohen syndrome; Barth syndrome; ELANE; G6PC3; GSD1b; Kostmann disease; Shwachman-Diamond syndrome; Autoimmune neutropenia (AIN)

Outcome Measures

Primary Outcomes (2)

• Co-primary Endpoint: Annualized Infection Rate Based on Infections Adjudicated by Blinded Infection Adjudication Committee (BIAC) During the Treatment Period

  Up to 52 Weeks

• Co-primary Endpoint: Number of Participants Meeting the Definition of a Positive Absolute Neutrophil Count (ANC) Response

  Positive ANC response: Increase of ANC \>500 cells/microliter (µL) from baseline.

  Up to 52 weeks

Secondary Outcomes (5)

• Infection Severity Based on Common Terminology Criteria for Adverse Events (CTCAE) Adjudicated by a BIAC During the Treatment Period

  Up to 52 Weeks

• Infection Duration Based on Duration of Infections Adjudicated by a BIAC During the Treatment Period in Those Participants who Developed Infections

  Up to 52 Weeks

• Antibiotic Use Due to Infection, Characterized by the Frequency of Antibiotic Use During the Treatment Period

  Up to 52 Weeks

• Oral Ulcers, as Assessed by Presence or Absence of Ulcers During the Treatment Period

  Up to 52 Weeks

• Change From Baseline in Patient Reported Outcomes Measurement Information System Short Form (PROMIS SF) Fatigue Questionnaire Total Score

  Baseline, Week 52

Study Arms (2)

Mavorixafor

EXPERIMENTAL

Participants will receive mavorixafor orally once daily starting from Day 1 through Week 52.

Drug: Mavorixafor

Placebo

PLACEBO COMPARATOR

Participants will receive placebo to match mavorixafor orally once daily starting from Day 1 through Week 52.

Drug: Placebo

Interventions

Placebo DRUG

Placebo will be administered per schedule specified in the arm description.

Placebo

Mavorixafor DRUG

Mavorixafor will be administered per schedule specified in the arm description.

Also known as: X4P-001

Mavorixafor

Eligibility Criteria

• Age: 12 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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:
• 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)
• Associated with extra-hematologic manifestations, for example, Barth syndrome, Cohen syndrome, glucose-6-phosphatase catalytic subunit 3 (G6PC3), Kostmann disease
• Associated with metabolic disorders, for example, glycogen storage disease 1b (GSD1b)
• Shwachman-Diamond syndrome
• Acquired Primary Neutropenia
• Chronic idiopathic neutropenia
• 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.

You may not qualify if:

• A diagnosis of secondary neutropenia including those due to:
• Hypersplenism
• Infection
• Malignancy
• Autoimmune disease, for example, systemic lupus erythematosus, rheumatoid arthritis, inflammatory bowel disease, graft-versus-host disease, thyroid disease
• Nutritional deficiency, for example, vitamin B12, folic acid, copper, caloric malnutrition
• Drug-induced cause, for example, chemotherapy, clozapine, antiretrovirals, antibiotics, monoclonal antibodies.
• A diagnosis of any of the following:
• Aplastic anemia
• Warts, hypogammaglobulinemia, infections, and myelokathexis (WHIM) syndrome
• Certain CNs, including but not limited to these classifications are excluded:
• Isolated with a cyclic presentation, for example, elastase, neutrophil expressed (ELANE)
• 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
• Associated with bone marrow failure, for example, Fanconi anemia, Diamond-Blackfan anemia
• 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.

Sponsors & Collaborators

• X4 Pharmaceuticals: lead

Study Sites (112)

Phoenix Children's Hospital

Phoenix, Arizona, 85016, United States

RECRUITING

UC Irvine

Irvine, California, 92697, United States

RECRUITING

Orso Health

La Jolla, California, 92037, United States

RECRUITING

University of California, Los Angeles-UCLA

Los Angeles, California, 90095, United States

RECRUITING

Orso Health

Torrance, California, 90505, United States

RECRUITING

University of Southern Florida

St. Petersburg, Florida, 33701, United States

RECRUITING

Children's Healthcare of Atlanta (CHOA)

Atlanta, Georgia, 30322, United States

RECRUITING

University of Iowa

Iowa City, Iowa, 52242, United States

RECRUITING

Boston Children's Cancer Center

Boston, Massachusetts, 02143, United States

RECRUITING

University of Michigan

Ann Arbor, Michigan, 48109, United States

RECRUITING

Washington University

St Louis, Missouri, 63130, United States

RECRUITING

Duke University Medical Center

Durham, North Carolina, 27710, United States

RECRUITING

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229, United States

RECRUITING

Cleveland Clinic

Cleveland, Ohio, 44195, United States

RECRUITING

The Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

RECRUITING

St. Jude Childrens; Research Hospital

Memphis, Tennessee, 38105, United States

RECRUITING

UT Southwestern Medical Center

Dallas, Texas, 75390, United States

RECRUITING

Cook Children's Medical Center

Fort Worth, Texas, 76104, United States

RECRUITING

Texas Children's Hospital/ Baylor College of Medicine

Houston, Texas, 77030, United States

RECRUITING

Sarah Cannon Research Institute

Woodland, Texas, 77380, United States

RECRUITING

University of Utah

Salt Lake City, Utah, 84112, United States

RECRUITING

Froedtert And Medical College Of Wisconsin

Milwaukee, Wisconsin, 53226, United States

RECRUITING

Hospital El Cruce

Buenos Aires, B1888AAE, Argentina

RECRUITING

Instituto de Investigaciones Clínicas Córdoba

Córdoba, X5000AAW, Argentina

RECRUITING

Santorio 9 de Julio

San Miguel de Tucumán, T4000IIH, Argentina

RECRUITING

Perth Children's Hospital

Nedlands, 6009, Australia

RECRUITING

Icon Cancer Centre Southport

Southport, 4215, Australia

RECRUITING

Ballarat Oncology And Haematology Services

Wendouree, 3355, Australia

RECRUITING

University of Alberta Hospital

Edmonton, AB T6G 2R3, Canada

RECRUITING

BC Children's Hospital

Vancouver, V5Z 4H4, Canada

RECRUITING

Clinica IMBANACO S.A.S

Cali, 76004, Colombia

RECRUITING

Clínica del Country

Chapinero, 0813, Colombia

RECRUITING

Hospital Pablo Tobón Uribe

Medellín, 500034, Colombia

RECRUITING

The University Hospital Brno

Brno, 62500, Czechia

RECRUITING

Motol University Hospital

Prague, 15006, Czechia

RECRUITING

Institute of Hematology and Pediatric Oncology - Lyon

Lyon, 69008, France

RECRUITING

Hôpital Saint Louis

Paris, 75010, France

RECRUITING

Hospital Armand Trousseau Ap-Hp

Paris, 75012, France

RECRUITING

Hôpital Necker - Enfants Malade

Paris, 75015, France

RECRUITING

Centre Hospitalier Universitaire de Rennes

Rennes, 75033, France

RECRUITING

Institut Universitaire de Cancérologie de Toulouse - Oncopôle

Toulouse, 31059, France

RECRUITING

LTD "Israeli-Georgian Medical Research Clinic Healthycore"

Tbilisi, 112, Georgia

RECRUITING

LEPL The First University Clinic of Tbilisi State Medical University

Tbilisi, 141, Georgia

RECRUITING

JSC K. Eristavi National Center of Experimental and Clinical Surgery

Tbilisi, 159, Georgia

RECRUITING

LTD Multiprofile Clinic Concilium Medulla

Tbilisi, 186, Georgia

RECRUITING

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

Dresden, 01307, Germany

RECRUITING

Universitaetsklinikum Carl Gustav Carus Tu Dresden

Dresden, 01307, Germany

RECRUITING

Hellenic Airforce 251 General Hospital

Athens, 10177, Greece

RECRUITING

University General Hospital of Heraklion

Heraklion, 71500, Greece

RECRUITING

The Olympion Private General Clinic

Pátrai, 26443, Greece

RECRUITING

AHEPA General Hospital of Thessaloniki

Thessaloniki, 54636, Greece

RECRUITING

CMC Deli Klinika

Budapest, 1012, Hungary

RECRUITING

Semmelweis University

Budapest, 1085, Hungary

RECRUITING

Teaching Hospital Markusovszky

Szombathely, 9700, Hungary

RECRUITING

Indira Gandhi Institute Of Medical Sciences

Patna, Bihar, 800014, India

WITHDRAWN

Nirmal Hospital Private Limited

Surat, Gujarat, 395002, India

WITHDRAWN

Pacific Medical College and Hospital

Udaipur, Rajasthan, 313001, India

WITHDRAWN

Yashoda Hospital

Hyderabad, Telangana, 500084, India

ACTIVE NOT RECRUITING

Ruth Rappaport Children's Hospital, Rambam Health Corporation

Haifa, 3109601, Israel

RECRUITING

Hadassah University Hospital-ein Kerem

Jerusalem, 9112001, Israel

RECRUITING

Schneider Children's Medical Center of Israel

Petah Tikva, 4920235, Israel

RECRUITING

The Chaim Sheba Medical Center

Ramat Gan, 5265601, Israel

RECRUITING

A. O. U. delle Marche

Torrette, Ancona, 60126, Italy

RECRUITING

ASST Spedali Civili di Brescia

Brescia, 25121, Italy

RECRUITING

IRCCS Instituto G. Gaslini

Genova, 16147, Italy

RECRUITING

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

Meldola, 47014, Italy

RECRUITING

IRCCS San Gerardo Dei Tintori

Monza, 20900, Italy

RECRUITING

Bambino Gesu Children's Hospital

Roma, PC 00165, Italy

RECRUITING

AOU Città della salute e della scienza di Torino

Torino, 10126, Italy

RECRUITING

Hospital Pakar Kanak-Kanak UKM

Kuala Lumpur, 56000, Malaysia

RECRUITING

Unidade Local de Saúde da Região de Aveiro

Aveiro, 3810-164, Portugal

RECRUITING

Unidade Local de Saude Coimbra - Paediatric Hospital

Coimbra, 3000-602, Portugal

RECRUITING

Unidade Local de Saude Coimbra - Quinta dos Vales

Coimbra, 3041-801, Portugal

RECRUITING

Unidade Local de Saude Santa Maria

Lisbon, 1649-028, Portugal

RECRUITING

Spitalul Clinic Colentina Site 1

Bucharest, 020125, Romania

RECRUITING

Spitalul Clinic Colentina Site 2

Bucharest, 020125, Romania

RECRUITING

Institutul Clinic Fundeni

Bucharest, 022328, Romania

RECRUITING

Institutul Clinic Fundeni

Bucharest, 22328, Romania

RECRUITING

Spitalul Clinic Județean de Urgență

Târgu Mureş, 540136, Romania

RECRUITING

University Clinical Centre of Serbia

Belgrade, 11000, Serbia

RECRUITING

ICO Badalona - Institut Català d'Oncologia

Barcelona, PC 08916, Spain

RECRUITING

Hospital Del Mar

Barcelona, PC08003, Spain

RECRUITING

Hospital San Pedro De Alcantara

Cáceres, 10003, Spain

RECRUITING

Hospital Universitario Reina Sofía

Córdoba, 14004, Spain

WITHDRAWN

Hospital Universitario de Gran Canaria Doctor Negrín

Las Palmas de Gran Canaria, 35010, Spain

RECRUITING

Hospital De La Princesa

Madrid, 28006, Spain

RECRUITING

Hospital Universitario Infanta Leonor

Madrid, 28031, Spain

WITHDRAWN

Hospital Universitario Fundacion Jimenez Diaz

Madrid, 28040, Spain

RECRUITING

Hospital Universitario Marqués de Valdecilla

Santander, 39008, Spain

RECRUITING

Hospital Universitario Virgen del Rocio

Seville, 41013, Spain

RECRUITING

Hospital Universitario y Politecnico de la Fe

Valencia, 46026, Spain

RECRUITING

Inselspital - University Hospital Bern Site 1

Bern, 3010, Switzerland

RECRUITING

Inselspital - University Hospital Bern Site 2

Bern, 3010, Switzerland

RECRUITING

Ramathibodi Hospital

Bangkok, 10400, Thailand

RECRUITING

Siriraj Hospital

Bangkok, 10700, Thailand

RECRUITING

Namik Kemal University (Tekirdağ Namık Kemal Üniversitesi)

Tekirdağ, Süleymanpaşa, 59030, Turkey (Türkiye)

RECRUITING

Dr. Abdurrahman Yurtaslan Ankara Oncology Training And Research Hospital

Ankara, 6000, Turkey (Türkiye)

RECRUITING

Ankara University School of Medicine

Ankara, 6620, Turkey (Türkiye)

RECRUITING

Uludag University Faculty Of Medicine

Nilufer, Turkey (Türkiye)

RECRUITING

Ondokuz Mayis University Medical Faculty Hospital

Samsun, 55200, Turkey (Türkiye)

RECRUITING

Lviv Regional Clinical Diagnostic Centre

Lviv, Oblast, 79010, Ukraine

RECRUITING

Medical Center Dobrobut-Clinic LLC

Kyiv, 03151, Ukraine

RECRUITING

Medical Center Ok Clinic of International Institute of Clinical Research LLC

Kyiv, 2091, Ukraine

RECRUITING

Llc Onto-Med

Kyiv, 3022, Ukraine

RECRUITING

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

Ternopil, 46000, Ukraine

RECRUITING

Medical Center The Clinic of Hospodarskyy LLC

Ternopil, 46002, Ukraine

RECRUITING

University Hospital of Wales

Cardiff, CF14 4XW, United Kingdom

RECRUITING

Cardiff And Vale University Health Board

London, CF14 4XW, United Kingdom

RECRUITING

Royal Free London Nhs Foundation Trust Royal Free Hospital

London, NW3 2QG, United Kingdom

RECRUITING

King's College Hospital

London, SE5 9RS, United Kingdom

RECRUITING

Imperial College Healthcare Nhs Trust - Hammersmith Hospital

London, W12 0HS, United Kingdom

RECRUITING

Great Ormond street Hospital for Children NHS Trust

London, WC1N 1DZ, United Kingdom

RECRUITING

MeSH Terms

Conditions

Neutropenia; Neutropenia, Severe Congenital, Autosomal Recessive 3; Autoimmune Diseases; Cohen syndrome; Barth Syndrome; Shwachman-Diamond Syndrome

Interventions

mavorixafor

Condition Hierarchy (Ancestors)

Agranulocytosis; Leukopenia; Cytopenia; Hematologic Diseases; Hemic and Lymphatic Diseases; Leukocyte Disorders; Immune System Diseases; Heart Defects, Congenital; Cardiovascular Abnormalities; Cardiovascular Diseases; Heart Diseases; Abnormalities, Multiple; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Lipid Metabolism, Inborn Errors; Metabolism, Inborn Errors; Lipid Metabolism Disorders; Metabolic Diseases; Nutritional and Metabolic Diseases; Exocrine Pancreatic Insufficiency; Pancreatic Diseases; Digestive System Diseases; Congenital Bone Marrow Failure Syndromes; Bone Marrow Failure Disorders; Bone Marrow Diseases; Lipomatosis

Study Officials

• Vice President Global Head of Clinical Development and Safety

  X4 Pharmaceuticals

  STUDY DIRECTOR

Central Study Contacts

Patient Affairs and Advocacy

CONTACT

857-529-5779; clinicaltrialinfo@x4pharma.com

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 20, 2023
• First Posted: September 28, 2023
• Study Start: June 6, 2024
• Primary Completion (Estimated): September 1, 2027
• Study Completion (Estimated): November 1, 2027
• Last Updated: April 13, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will not share

Locations

AR (3); IN (4); GB (6); GE (4); DE (2); GR (4); FR (6); TU (5); UA (6); IL (4); PT (4); TH (2); CA (2); AU (3); SE (1); ES (11); CZ (2); US (22); HU (3); CH (2); MY (1); RO (5); IT (7); CO (3)