Phase
Condition
Neutropenia
Treatment
Placebo
Mavorixafor
Clinical Study ID
Ages > 12 All Genders
Study Summary
Eligibility Criteria
Inclusion
Key Inclusion Criteria:
Diagnosis of congenital or acquired primary autoimmune and idiopathic chronicneutropenic disorder ≥6 months prior to the screening visit that is not attributableto 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), Kostmanndisease
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 thatmay be eligible for enrollment can be clarified and approved upon discussionwith study Medical Monitor and Sponsor.
Have an ANC <1000 cells/µL during screening (single ANC value from hematology) andconfirmed trough mean ANC (mean value of multiple ANC measurements over 6 hours) atbaseline visit, with no clinical evidence of systemic infection.
Prior history of recurrent and/or serious infections during the 12 months precedingthe screening visit (that is, suffering sequelae of chronic neutropenia), as definedby having at least 2 infections in the last 12 months that meet the followingcriteria:
Infection requiring the use of antibiotics (intravenous [IV]/oral); OR
Infection requiring a visit to healthcare facility (including but not limited toemergency room visit, urgent care facility, primary care physician's office, orin-patient hospitalization);
AND for all potential participants:
Infections considered by the Investigator to be likely related to the potentialparticipant's CN disorder.
Participants who are on G-CSF or other active background therapy must have beenreceiving these therapies during the previous 12 months while continuing to sufferfrom infections, be on a stable dose and dosing schedule for ≥4 weeks prior toscreening 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 therapydoses/regimens stable (other than for safety reasons) for the duration of the study.
Exclusion
Key Exclusion Criteria:
- A diagnosis of secondary neutropenia including those due to:
Hypersplenism
Infection
Malignancy
Autoimmune disease, for example, systemic lupus erythematosus, rheumatoidarthritis, inflammatory bowel disease, graft-versus-host disease, thyroiddisease
Nutritional deficiency, for example, vitamin B12, folic acid, copper, caloricmalnutrition
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, neutrophilexpressed (ELANE)
Associated with immune dysregulation, for example, common variableimmunodeficiency (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
Neutropenia associated with a Duffy-null phenotype (formerly known as benignethnic neutropenia). However, a participant with an autosomal dominantpathogenic variant in a gene associated with CN on a Duffy-null background maybe eligible for inclusion
A medical or personal condition that may potentially compromise the safety of theparticipant, may preclude the participant's successful completion of the clinicalstudy, or could, in the opinion of the Investigator or the Sponsor, interfere withthe 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) inthe past 6 months.
Participants taking pegylated-G-CSF unless they have a diagnosis of congenitalneutropenia confirmed at screening.
Participant is currently taking or has taken other investigational drug <30 daysprior to the screening visit or 5 half-lives, whichever is longer.
Note: Other protocol-defined inclusion and exclusion criteria may apply.
Study Design
Study Description
Connect with a study center
Instituto de Investigaciones Clínicas Córdoba
Córdoba, X5000AAW
ArgentinaActive - Recruiting
Santorio 9 de Julio
San Miguel de Tucumán, T4000IIH
ArgentinaActive - Recruiting
Icon Cancer Centre Southport
Southport, 4215
AustraliaActive - Recruiting
University of Alberta Hospital
Edmonton, AB T6G 2R3
CanadaActive - Recruiting
The University Hospital Brno
Brno, 62500
CzechiaActive - Recruiting
Motol University Hospital
Prague, 15006
CzechiaActive - Recruiting
Institute of Hematology and Pediatric Oncology - Lyon
Lyon, 69008
FranceActive - Recruiting
Hospital Armand Trousseau Ap-Hp
Paris, 75012
FranceActive - Recruiting
Hospital Saint Louis
Paris, 75010
FranceActive - Recruiting
Hôpital Necker - Enfants Malade
Paris, 75015
FranceActive - Recruiting
Hôpital Saint Louis
Paris, 75010
FranceActive - Recruiting
Hôspital Necker - Enfants Malade
Paris, 75015
FranceActive - Recruiting
Centre Hospitalier Universitaire de Rennes
Rennes, 75033
FranceActive - Recruiting
JSC K. Eristavi National Center of Experimental and Clinical Surgery
Tbilisi, 159
GeorgiaActive - Recruiting
LEPL The First University Clinic of Tbilisi State Medical University
Tbilisi, 141
GeorgiaActive - Recruiting
LTD "Israeli-Georgian Medical Research Clinic Healthycore"
Tbilisi, 112
GeorgiaActive - Recruiting
LTD Multiprofile Clinic Concilium Medulla
Tbilisi, 186
GeorgiaActive - Recruiting
Klinik und Poliklinik fur Kinder- und Jugendmedizin, Universitatsklinikum Carl Gustav Carus an der TU
Dresden, 01307
GermanyActive - Recruiting
Universitaetsklinikum Carl Gustav Carus Tu Dresden
Dresden, 01307
GermanyActive - Recruiting
Hellenic Airforce 251 General Hospital
Athens, 10177
GreeceActive - Recruiting
University General Hospital of Heraklion
Iraklio, 71500
GreeceActive - Recruiting
The Olympion Private General Clinic
Patra, 26443
GreeceActive - Recruiting
AHEPA General Hospital of Thessaloniki
Thessaloniki, 54636
GreeceActive - Recruiting
CMC Deli Klinika
Budapest, 1012
HungaryActive - Recruiting
Semmelweis University
Budapest, 1085
HungaryActive - Recruiting
Teaching Hospital Markusovszky
Szombathely, 9700
HungaryActive - Recruiting
Indira Gandhi Institute Of Medical Sciences
Patna, Bihar 800014
IndiaActive - Recruiting
Nirmal Hospital Private Limited
Surat, Gujarat 395002
IndiaActive - Recruiting
Pacific Medical College and Hospital
Udaipur, Rajasthan 313001
IndiaActive - Recruiting
Yashoda Hospital
Hyderabad, Telangana 500084
IndiaActive - Recruiting
Ruth Rappaport Children's Hospital, Rambam Health Corporation
Haifa, 3109601
IsraelActive - Recruiting
A. O. U. delle Marche
Torrette, Ancona 60126
ItalyActive - Recruiting
Ospedale Riuniti Torrette di Ancona ( Riuniti Torrette Hospital in Ancona)
Torrette, Ancona 60126
ItalyActive - Recruiting
ASST Spedali Civili di Brescia
Brescia, 25121
ItalyActive - Recruiting
IRCCS Instituto G. Gaslini
Genova, 16147
ItalyActive - Recruiting
Istituto Scientifico Romagnolo Per Lo Studio E La Cura Dei Tumori (IRST) Dino Amadori
Meldola, 47014
ItalyActive - Recruiting
IRCCS San Gerardo Dei Tintori
Monza, 20900
ItalyActive - Recruiting
Unidade Local de Saúde da Região de Aveiro
Aveiro, 3810-164
PortugalActive - Recruiting
Unidade Local de Saude Coimbra - Paediatric Hospital
Coimbra, 3000-602
PortugalActive - Recruiting
Unidade Local de Saude Coimbra - Quinta dos Vales
Coimbra, 3041-801
PortugalActive - Recruiting
Institutul Clinic Fundeni
Bucharest, 022328
RomaniaActive - Recruiting
Spitalul Clinic Colentina Site 1
Bucharest, 020125
RomaniaActive - Recruiting
Spitalul Clinic Colentina Site 2
Bucharest, 020125
RomaniaActive - Recruiting
Spitalul Clinic Județean de Urgență
Târgu-Mureş, 540136
RomaniaActive - Recruiting
University Clinical Centre of Serbia
Belgrade, 11000
SerbiaActive - Recruiting
Hospital Del Mar
Barcelona, PC08003
SpainActive - Recruiting
ICO Badalona - Institut Català d'Oncologia
Barcelona, PC 08916
SpainActive - Recruiting
Hospital San Pedro De Alcantara
Cáceres, 10003
SpainActive - Recruiting
Hospital Universitario Reina Sofía
Córdoba, 14004
SpainActive - Recruiting
Hospital De La Princesa
Madrid, 28006
SpainActive - Recruiting
Hospital Universitario Fundacion Jimenez Diaz
Madrid, 28040
SpainActive - Recruiting
Hospital Universitario Infanta Leonor
Madrid, 28031
SpainActive - Recruiting
Hospital Universitario Son Espases
Mallorca, PC07120
SpainSite Not Available
Hospital Universitario Marqués de Valdecilla
Santander, 39008
SpainActive - Recruiting
Hospital Universitario Virgen del Rocio
Sevilla, 41013
SpainActive - Recruiting
Inselspital - University Hospital Bern Site 1
Bern, 3010
SwitzerlandActive - Recruiting
Inselspital - University Hospital Bern Site 2
Bern, 3010
SwitzerlandActive - Recruiting
Ramathibodi Hospital
Bangkok, 10400
ThailandActive - Recruiting
Siriraj Hospital
Bangkok, 10700
ThailandActive - Recruiting
Ankara University School of Medicine
Ankara, 6620
TurkeyActive - Recruiting
Dr. Abdurrahman Yurtaslan Ankara Oncology Training And Research Hospital
Ankara, 6000
TurkeyActive - Recruiting
Uludag University Faculty Of Medicine
Nilufer,
TurkeyActive - Recruiting
Ondokuz Mayis University Medical Faculty Hospital
Samsun, 55200
TurkeyActive - Recruiting
Lviv Regional Clinical Diagnostic Centre
Lviv, Oblast 79010
UkraineActive - Recruiting
Llc Onto-Med
Kyiv, 3022
UkraineActive - Recruiting
Medical Center Dobrobut-Clinic LLC
Kyiv, 03151
UkraineActive - Recruiting
Medical Center Ok Clinic of International Institute of Clinical Research LLC
Kyiv, 2091
UkraineActive - Recruiting
Communal non-commercial enterprise "Ternopil City Children's Communal Hospital"
Ternopil, 46000
UkraineActive - Recruiting
Medical Center The Clinic of Hospodarskyy LLC
Ternopil, 46002
UkraineActive - Recruiting
Cardiff And Vale University Health Board
London, CF14 4XW
United KingdomActive - Recruiting
Great Ormond street Hospital for Children NHS Trust
London, WC1N 1DZ
United KingdomActive - Recruiting
Imperial College Healthcare Nhs Trust - Hammersmith Hospital
London, W12 0HS
United KingdomActive - Recruiting
King's College Hospital
London, SE5 9RS
United KingdomActive - Recruiting
Royal Free London Nhs Foundation Trust Royal Free Hospital
London, NW3 2QG
United KingdomActive - Recruiting
Phoenix Children's Hospital
Phoenix, Arizona 85016
United StatesActive - Recruiting
Orso Health
La Jolla, California 92037
United StatesActive - Recruiting
Orso Health
Torrance, California 90505
United StatesActive - Recruiting
University of Michigan
Ann Arbor, Michigan 48109
United StatesActive - Recruiting
Washington University
Saint Louis, Missouri 63130
United StatesActive - Recruiting
Duke University Medical Center
Durham, North Carolina 27710
United StatesActive - Recruiting
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio 45229
United StatesActive - Recruiting
UT Southwestern Medical Center
Dallas, Texas 75390
United StatesActive - Recruiting
Cook Children's Medical Center
Fort Worth, Texas 76104
United StatesActive - Recruiting
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