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

Status: Recruiting
Location: See all (97) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 3
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
Participation Requirements
Sex: All
Minimum Age: 12
Healthy Volunteers: f
View:

• 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 and Sponsor.

• 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.

Locations
United States
Arizona
Phoenix Children's Hospital
RECRUITING
Phoenix
California
UC Irvine
RECRUITING
Irvine
Orso Health
RECRUITING
La Jolla
Orso Health
RECRUITING
Torrance
Florida
University of Southern Florida
RECRUITING
St. Petersburg
Iowa
University of Iowa
RECRUITING
Iowa City
Michigan
University of Michigan
RECRUITING
Ann Arbor
Missouri
Washington University
RECRUITING
St Louis
North Carolina
Duke University Medical Center
RECRUITING
Durham
Ohio
Cincinnati Children's Hospital Medical Center
RECRUITING
Cincinnati
Pennsylvania
The Children's Hospital of Philadelphia
RECRUITING
Philadelphia
Texas
UT Southwestern Medical Center
RECRUITING
Dallas
Cook Children's Medical Center
RECRUITING
Fort Worth
Utah
University of Utah
RECRUITING
Salt Lake City
Other Locations
Argentina
Hospital El Cruce
RECRUITING
Buenos Aires
Instituto de Investigaciones Clínicas Córdoba
RECRUITING
Córdoba
Santorio 9 de Julio
RECRUITING
San Miguel De Tucumán
Australia
Perth Children's Hospital
RECRUITING
Nedlands
Icon Cancer Centre Southport
RECRUITING
Southport
Ballarat Oncology And Haematology Services
RECRUITING
Wendouree
Canada
University of Alberta Hospital
RECRUITING
Edmonton
Colombia
Clinica IMBANACO S.A.S
RECRUITING
Santiago De Cali
France
Institute of Hematology and Pediatric Oncology - Lyon
RECRUITING
Lyon
Hôpital Necker - Enfants Malade
RECRUITING
Paris
Hôpital Saint Louis
RECRUITING
Paris
Hospital Armand Trousseau Ap-Hp
RECRUITING
Paris
Centre Hospitalier Universitaire de Rennes
RECRUITING
Rennes
Georgia
JSC K. Eristavi National Center of Experimental and Clinical Surgery
RECRUITING
Tbilisi
LEPL The First University Clinic of Tbilisi State Medical University
RECRUITING
Tbilisi
LTD Israeli-Georgian Medical Research Clinic Healthycore
RECRUITING
Tbilisi
LTD Multiprofile Clinic Concilium Medulla
RECRUITING
Tbilisi
Germany
Klinik und Poliklinik fur Kinder- und Jugendmedizin, Universitatsklinikum Carl Gustav Carus an der TU
RECRUITING
Dresden
Universitaetsklinikum Carl Gustav Carus Tu Dresden
RECRUITING
Dresden
Greece
Hellenic Airforce 251 General Hospital
RECRUITING
Athens
University General Hospital of Heraklion
RECRUITING
Heraklion
The Olympion Private General Clinic
RECRUITING
Pátrai
AHEPA General Hospital of Thessaloniki
RECRUITING
Thessaloniki
Hungary
CMC Deli Klinika
RECRUITING
Budapest
Semmelweis University
RECRUITING
Budapest
Teaching Hospital Markusovszky
RECRUITING
Szombathely
India
Yashoda Hospital
RECRUITING
Hyderabad
Indira Gandhi Institute Of Medical Sciences
RECRUITING
Patna
Nirmal Hospital Private Limited
RECRUITING
Surat
Pacific Medical College and Hospital
RECRUITING
Udaipur
Israel
Ruth Rappaport Children's Hospital, Rambam Health Corporation
RECRUITING
Haifa
Hadassah University Hospital-ein Kerem
RECRUITING
Jerusalem
Schneider Children's Medical Center of Israel
RECRUITING
Petah Tikva
The Chaim Sheba Medical Center
RECRUITING
Ramat Gan
Italy
ASST Spedali Civili di Brescia
RECRUITING
Brescia
IRCCS Instituto G. Gaslini
RECRUITING
Genova
Istituto Scientifico Romagnolo Per Lo Studio E La Cura Dei Tumori (IRST) Dino Amadori
RECRUITING
Meldola
IRCCS San Gerardo Dei Tintori
RECRUITING
Monza
Bambino Gesu Children's Hospital
RECRUITING
Roma
A. O. U. delle Marche
RECRUITING
Torrette
Malaysia
Hospital Pakar Kanak-Kanak UKM
RECRUITING
Kuala Lumpur
Portugal
Unidade Local de Saúde da Região de Aveiro
RECRUITING
Aveiro
Unidade Local de Saude Coimbra - Paediatric Hospital
RECRUITING
Coimbra
Unidade Local de Saude Coimbra - Quinta dos Vales
RECRUITING
Coimbra
Unidade Local de Saude Santa Maria
RECRUITING
Lisbon
Romania
Institutul Clinic Fundeni
RECRUITING
Bucharest
Institutul Clinic Fundeni
RECRUITING
Bucharest
Spitalul Clinic Colentina Site 1
RECRUITING
Bucharest
Spitalul Clinic Colentina Site 2
RECRUITING
Bucharest
Spitalul Clinic Județean de Urgență
RECRUITING
Târgu Mureş
Serbia
University Clinical Centre of Serbia
RECRUITING
Belgrade
Spain
Hospital Del Mar
RECRUITING
Barcelona
ICO Badalona - Institut Català d'Oncologia
RECRUITING
Barcelona
Hospital San Pedro De Alcantara
RECRUITING
Cáceres
Hospital Universitario Reina Sofía
RECRUITING
Córdoba
Hospital Universitario de Gran Canaria Doctor Negrín
RECRUITING
Las Palmas De Gran Canaria
Hospital De La Princesa
RECRUITING
Madrid
Hospital Universitario Fundacion Jimenez Diaz
RECRUITING
Madrid
Hospital Universitario Infanta Leonor
RECRUITING
Madrid
Hospital Universitario Marqués de Valdecilla
RECRUITING
Santander
Hospital Universitario Virgen del Rocio
RECRUITING
Seville
Hospital Universitario y Politecnico de la Fe
RECRUITING
Valencia
Switzerland
Inselspital - University Hospital Bern Site 1
RECRUITING
Bern
Inselspital - University Hospital Bern Site 2
RECRUITING
Bern
Thailand
Ramathibodi Hospital
RECRUITING
Bangkok
Siriraj Hospital
RECRUITING
Bangkok
Turkey
Ankara University School of Medicine
RECRUITING
Ankara
Dr. Abdurrahman Yurtaslan Ankara Oncology Training And Research Hospital
RECRUITING
Ankara
Uludag University Faculty Of Medicine
RECRUITING
Nilufer
Ondokuz Mayis University Medical Faculty Hospital
RECRUITING
Samsun
Namik Kemal University (Tekirdağ Namık Kemal Üniversitesi)
RECRUITING
Tekirdağ
Ukraine
Llc Onto-Med
RECRUITING
Kyiv
Medical Center Dobrobut-Clinic LLC
RECRUITING
Kyiv
Medical Center Ok Clinic of International Institute of Clinical Research LLC
RECRUITING
Kyiv
Lviv Regional Clinical Diagnostic Centre
RECRUITING
Lviv
Communal non-commercial enterprise Ternopil City Children's Communal Hospital
RECRUITING
Ternopil
Medical Center The Clinic of Hospodarskyy LLC
RECRUITING
Ternopil
United Kingdom
University Hospital of Wales
RECRUITING
Cardiff
Cardiff And Vale University Health Board
RECRUITING
London
Great Ormond street Hospital for Children NHS Trust
RECRUITING
London
Imperial College Healthcare Nhs Trust - Hammersmith Hospital
RECRUITING
London
King's College Hospital
RECRUITING
London
Royal Free London Nhs Foundation Trust Royal Free Hospital
RECRUITING
London
Contact Information
Primary
Patient Affairs and Advocacy
clinicaltrialinfo@x4pharma.com
857-529-5779
Time Frame
Start Date: 2024-06-06
Estimated Completion Date: 2026-08
Participants
Target number of participants: 150
Treatments
Experimental: Mavorixafor
Participants will receive mavorixafor orally once daily starting from Day 1 through Week 52.
Placebo_comparator: Placebo
Participants will receive placebo to match mavorixafor orally once daily starting from Day 1 through Week 52.
Sponsors
Leads: X4 Pharmaceuticals

This content was sourced from clinicaltrials.gov