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Clinical Trials in the USA / NCT06056297
Recruiting Phase 3

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 · tracked via the Priya Life Science USA tracker
Sponsor
X4 Pharmaceuticals
Phase
Phase 3
Started
2024-06-06
Last updated
2026-08-06

Condition(s) studied

Neutropenia

Investigational drug(s) / intervention(s)

MavorixaforPlacebo

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

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

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

Sex
ALL
Min age
12 Years
Max age
Healthy volunteers
No
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.

Primary outcome measure(s)

Trial sites (114)

FacilityCityRegionStatus
Phoenix Children's Hospital Phoenix Arizona Recruiting
UC Irvine Irvine California Recruiting
Orso Health La Jolla California Recruiting
University of California, Los Angeles-UCLA Los Angeles California Recruiting
Orso Health Torrance California Withdrawn
University of Southern Florida St. Petersburg Florida Recruiting
Children's Healthcare of Atlanta (CHOA) Atlanta Georgia Recruiting
University of Iowa Iowa City Iowa Recruiting
Boston Children's Cancer Center Boston Massachusetts Recruiting
University of Michigan Ann Arbor Michigan Recruiting
Washington University St Louis Missouri Recruiting
Duke University Medical Center Durham North Carolina Recruiting
Cincinnati Children's Hospital Medical Center Cincinnati Ohio Recruiting
Cleveland Clinic Cleveland Ohio Recruiting
The Children's Hospital of Philadelphia Philadelphia Pennsylvania Recruiting
St. Jude Childrens; Research Hospital Memphis Tennessee Recruiting
UT Southwestern Medical Center Dallas Texas Recruiting
Cook Children's Medical Center Fort Worth Texas Recruiting
Texas Children's Hospital/ Baylor College of Medicine Houston Texas Recruiting
Sarah Cannon Research Institute Woodland Texas Recruiting
University of Utah Salt Lake City Utah Recruiting
Froedtert And Medical College Of Wisconsin Milwaukee Wisconsin Recruiting
Hospital El Cruce Buenos Aires Argentina Recruiting
Instituto de Investigaciones Clínicas Córdoba Córdoba Argentina Recruiting
Santorio 9 de Julio San Miguel de Tucumán Argentina Recruiting
Perth Children's Hospital Nedlands Australia Recruiting
Icon Cancer Centre Southport Southport Australia Recruiting
Ballarat Oncology And Haematology Services Wendouree Australia Recruiting
Universitair Ziekenhuis Leuven Leuven Belgium Recruiting
University of Alberta Hospital Edmonton Canada Recruiting
BC Children's Hospital Vancouver Canada Recruiting
Fundacion Cardiovascular de Colombia-Instituto del Corazon Florida Blanca Floridablanca Santander Department Recruiting
Clinica IMBANACO S.A.S Cali Colombia Recruiting
Clínica del Country Chapinero Colombia Recruiting
Hospital Pablo Tobón Uribe Medellín Colombia Recruiting
The University Hospital Brno Brno Czechia Recruiting
Motol University Hospital Prague Czechia Recruiting
Institute of Hematology and Pediatric Oncology - Lyon Lyon France Recruiting
Hôpital Saint Louis Paris France Recruiting
Hospital Armand Trousseau Ap-Hp Paris France Recruiting

+ 74 more sites — see the full list on the official registry below.

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT06056297 on ClinicalTrials.gov ↗ ← All trials in the USA