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RECRUITING

NCT06056297

PHASE3

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

Sponsor: X4 Pharmaceuticals

View on ClinicalTrials.gov

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.

Official title: A Phase 3, Randomized, Double-blind, Placebo-controlled, Multicenter Study of Mavorixafor in Participants With Congenital and Acquired Primary Autoimmune and Idiopathic Chronic Neutropenic Disorders Who Are Experiencing Recurrent and/or Serious Infections

Key Details

Gender

All

Age Range

12 Years - Any

Study Type

INTERVENTIONAL

Enrollment

176

Start Date

2024-06-06

Completion Date

2027-11

Last Updated

2026-03-02

Healthy Volunteers

Conditions

Neutropenia

Interventions

DRUG

Mavorixafor

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

DRUG

Placebo

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

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.

Locations (109)

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

Children's Healthcare of Atlanta (CHOA)

Atlanta, Georgia, 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

Cleveland Clinic

Cleveland, 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

Texas Children's Hospital/ Baylor College of Medicine

Houston, Texas, United States

Sarah Cannon Research Institute

Woodland, 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

BC Children's Hospital

Vancouver, Canada

Clinica IMBANACO S.A.S

Cali, Colombia

Clínica del Country

Chapinero, Colombia

Hospital Pablo Tobón Uribe

Medellín, 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

Institut Universitaire de Cancérologie de Toulouse - Oncopôle

Toulouse, 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

Clinical Research Directory

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

Summary

Key Details

Conditions

Interventions

Eligibility Criteria

Locations (109)