RecruitingPhase 3NCT06056297

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

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

Sponsor

X4 Pharmaceuticals

Enrollment

176 participants

Start Date

Jun 6, 2024

Study Type

INTERVENTIONAL

Conditions

Neutropenia

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

Min Age: 12 Years

Plain Language Summary

Simplified for easier understanding

This study is testing whether a drug called mavorixafor can reduce serious and recurrent infections in people who have chronic neutropenia — a condition where the body doesn't produce enough white blood cells (neutrophils) to fight off infections. **You may be eligible if...** - You have a confirmed diagnosis of congenital or acquired primary chronic neutropenia that has been present for at least 6 months - Your neutropenia is not caused by medications, recent infections, or cancer - You have experienced recurrent or serious infections as a result - Specific types include: Severe Congenital Neutropenia, Autoimmune Neutropenia, Cyclic Neutropenia, or Idiopathic Neutropenia **You may NOT be eligible if...** - Your low white blood cell count is caused by a medication, active infection, or malignancy - You have conditions that exclude you from the study per protocol criteria - You are pregnant or breastfeeding Talk to your doctor to see if this trial is right for you.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Interventions

DRUGMavorixafor

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

DRUGPlacebo

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

Locations(112)

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

Boston Children's Cancer Center

Boston, Massachusetts, 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

St. Jude Childrens; Research Hospital

Memphis, Tennessee, 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

AOU Città della salute e della scienza di Torino

Torino, 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

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NCT06056297

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