Actively Recruiting
A Phase 3, Randomized, Double-blind, Placebo-controlled Study of Mavorixafor in Participants With Congenital and Acquired Primary Autoimmune and Idiopathic Chronic Neutropenic Disorders Experiencing Recurrent or Serious Infections
Led by X4 Pharmaceuticals · Updated on 2026-06-05
176
Participants Needed
114
Research Sites
8 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are studying the effects of mavorixafor in people aged 12 and older who have congenital or acquired primary autoimmune and idiopathic chronic neutropenic disorders. These disorders cause low levels of neutrophils, a type of white blood cell, leading to recurrent or serious infections. The study aims to show if mavorixafor can increase neutrophil levels and reduce infections, while also evaluating the drug's safety and how well participants tolerate it. Participants will continue their current treatments, which may include granulocyte-colony stimulating factor (G-CSF), immunoglobulin replacement therapy, prophylactic antibiotics, or no active treatment. They will be randomly assigned to receive either mavorixafor or a placebo once daily by mouth for 52 weeks. The study is double-blind, meaning neither participants nor researchers will know which treatment is given until the study ends. Throughout the study, participants will be monitored for infections and neutrophil counts, with assessments including infection rates reviewed by a blinded committee. Other measures include infection severity, duration, antibiotic use, presence of oral ulcers, and fatigue levels from patient questionnaires. The research team will track safety and tolerability during the 52-week treatment period, with follow-up assessments to observe outcomes and any side effects.
CONDITIONS
Brief Title
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
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Diagnosis of congenital or acquired primary autoimmune or idiopathic chronic neutropenic disorder for at least 6 months not due to medications, infections, or malignancy
- Congenital neutropenia including isolated permanent forms, those with extra-hematologic manifestations, metabolic disorders, or Shwachman-Diamond syndrome
- Acquired primary neutropenia including chronic idiopathic or primary autoimmune neutropenia
- Absolute neutrophil count (ANC) below 1000 cells/µL during screening and confirmed low ANC at baseline without systemic infection
- History of at least 2 infections in the past 12 months requiring antibiotics or medical visits linked to neutropenia
- Stable use of granulocyte-colony stimulating factor (G-CSF) or other background therapy for at least 4 weeks prior to screening and willingness to maintain stable dosing during the study
You will not qualify if you...
- Diagnosis of secondary neutropenia caused by hypersplenism, infection, malignancy, autoimmune diseases, nutritional deficiency, or drug-induced causes
- Diagnosis of aplastic anemia, WHIM syndrome, or certain cyclic or immune dysregulation-associated congenital neutropenias
- Neutropenia linked to a Duffy-null phenotype unless accompanied by a pathogenic variant
- Medical or personal conditions that may affect safety or study completion
- Prior receipt of more than one dose of mavorixafor or other CXCR4 antagonists within 6 months
- Use of pegylated-G-CSF unless congenital neutropenia diagnosis is confirmed
- Use of other investigational drugs within 30 days or five half-lives before screening
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Up to 52 weeks
Participants receive either mavorixafor or placebo orally once daily while continuing their pre-study background therapy.
Regular visits throughout the 52 weeks as per study protocol
Trial Site Locations
Total: 114 locations
1
Phoenix Children's Hospital
Phoenix, Arizona, United States, 85016
Actively Recruiting
2
UC Irvine
Irvine, California, United States, 92697
Actively Recruiting
3
Orso Health
La Jolla, California, United States, 92037
Actively Recruiting
4
University of California, Los Angeles-UCLA
Los Angeles, California, United States, 90095
Actively Recruiting
5
Orso Health
Torrance, California, United States, 90505
Actively Recruiting
6
University of Southern Florida
St. Petersburg, Florida, United States, 33701
Actively Recruiting
7
Children's Healthcare of Atlanta (CHOA)
Atlanta, Georgia, United States, 30322
Actively Recruiting
8
University of Iowa
Iowa City, Iowa, United States, 52242
Actively Recruiting
9
Boston Children's Cancer Center
Boston, Massachusetts, United States, 02143
Actively Recruiting
10
University of Michigan
Ann Arbor, Michigan, United States, 48109
Actively Recruiting
11
Washington University
St Louis, Missouri, United States, 63130
Actively Recruiting
12
Duke University Medical Center
Durham, North Carolina, United States, 27710
Actively Recruiting
13
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States, 45229
Actively Recruiting
14
Cleveland Clinic
Cleveland, Ohio, United States, 44195
Actively Recruiting
15
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
Actively Recruiting
16
St. Jude Childrens; Research Hospital
Memphis, Tennessee, United States, 38105
Actively Recruiting
17
UT Southwestern Medical Center
Dallas, Texas, United States, 75390
Actively Recruiting
18
Cook Children's Medical Center
Fort Worth, Texas, United States, 76104
Actively Recruiting
19
Texas Children's Hospital/ Baylor College of Medicine
Houston, Texas, United States, 77030
Actively Recruiting
20
Sarah Cannon Research Institute
Woodland, Texas, United States, 77380
Actively Recruiting
21
University of Utah
Salt Lake City, Utah, United States, 84112
Actively Recruiting
22
Froedtert And Medical College Of Wisconsin
Milwaukee, Wisconsin, United States, 53226
Actively Recruiting
23
Hospital El Cruce
Buenos Aires, Argentina, B1888AAE
Actively Recruiting
24
Instituto de Investigaciones Clínicas Córdoba
Córdoba, Argentina, X5000AAW
Actively Recruiting
25
Santorio 9 de Julio
San Miguel de Tucumán, Argentina, T4000IIH
Actively Recruiting
26
Perth Children's Hospital
Nedlands, Australia, 6009
Actively Recruiting
27
Icon Cancer Centre Southport
Southport, Australia, 4215
Actively Recruiting
28
Ballarat Oncology And Haematology Services
Wendouree, Australia, 3355
Actively Recruiting
29
Universitair Ziekenhuis Leuven
Leuven, Belgium, 3000
Actively Recruiting
30
University of Alberta Hospital
Edmonton, Canada, AB T6G 2R3
Actively Recruiting
31
BC Children's Hospital
Vancouver, Canada, V5Z 4H4
Actively Recruiting
32
Fundacion Cardiovascular de Colombia-Instituto del Corazon Florida Blanca
Floridablanca, Santander Department, Colombia, 681003
Actively Recruiting
33
Clinica IMBANACO S.A.S
Cali, Colombia, 76004
Actively Recruiting
34
Clínica del Country
Chapinero, Colombia, 0813
Actively Recruiting
35
Hospital Pablo Tobón Uribe
Medellín, Colombia, 500034
Actively Recruiting
36
The University Hospital Brno
Brno, Czechia, 62500
Actively Recruiting
37
Motol University Hospital
Prague, Czechia, 15006
Actively Recruiting
38
Institute of Hematology and Pediatric Oncology - Lyon
Lyon, France, 69008
Actively Recruiting
39
Hôpital Saint Louis
Paris, France, 75010
Actively Recruiting
40
Hospital Armand Trousseau Ap-Hp
Paris, France, 75012
Actively Recruiting
41
Hôpital Necker - Enfants Malade
Paris, France, 75015
Actively Recruiting
42
Centre Hospitalier Universitaire de Rennes
Rennes, France, 75033
Actively Recruiting
43
Institut Universitaire de Cancérologie de Toulouse - Oncopôle
Toulouse, France, 31059
Actively Recruiting
44
LTD "Israeli-Georgian Medical Research Clinic Healthycore"
Tbilisi, Georgia, 112
Actively Recruiting
45
LEPL The First University Clinic of Tbilisi State Medical University
Tbilisi, Georgia, 141
Actively Recruiting
46
JSC K. Eristavi National Center of Experimental and Clinical Surgery
Tbilisi, Georgia, 159
Actively Recruiting
47
LTD Multiprofile Clinic Concilium Medulla
Tbilisi, Georgia, 186
Actively Recruiting
48
Klinik und Poliklinik fur Kinder- und Jugendmedizin, Universitatsklinikum Carl Gustav Carus an der TU
Dresden, Germany, 01307
Actively Recruiting
49
Universitaetsklinikum Carl Gustav Carus Tu Dresden
Dresden, Germany, 01307
Actively Recruiting
50
Hellenic Airforce 251 General Hospital
Athens, Greece, 10177
Actively Recruiting
51
University General Hospital of Heraklion
Heraklion, Greece, 71500
Actively Recruiting
52
The Olympion Private General Clinic
Pátrai, Greece, 26443
Actively Recruiting
53
AHEPA General Hospital of Thessaloniki
Thessaloniki, Greece, 54636
Actively Recruiting
54
CMC Deli Klinika
Budapest, Hungary, 1012
Actively Recruiting
55
Semmelweis University
Budapest, Hungary, 1085
Actively Recruiting
56
Teaching Hospital Markusovszky
Szombathely, Hungary, 9700
Actively Recruiting
57
Indira Gandhi Institute Of Medical Sciences
Patna, Bihar, India, 800014
Withdrawn
58
Nirmal Hospital Private Limited
Surat, Gujarat, India, 395002
Withdrawn
59
Pacific Medical College and Hospital
Udaipur, Rajasthan, India, 313001
Withdrawn
60
Yashoda Hospital
Hyderabad, Telangana, India, 500084
Active, Not Recruiting
61
Ruth Rappaport Children's Hospital, Rambam Health Corporation
Haifa, Israel, 3109601
Actively Recruiting
62
Hadassah University Hospital-ein Kerem
Jerusalem, Israel, 9112001
Actively Recruiting
63
Schneider Children's Medical Center of Israel
Petah Tikva, Israel, 4920235
Actively Recruiting
64
The Chaim Sheba Medical Center
Ramat Gan, Israel, 5265601
Actively Recruiting
65
A. O. U. delle Marche
Torrette, Ancona, Italy, 60126
Actively Recruiting
66
ASST Spedali Civili di Brescia
Brescia, Italy, 25121
Actively Recruiting
67
IRCCS Instituto G. Gaslini
Genova, Italy, 16147
Actively Recruiting
68
Istituto Scientifico Romagnolo Per Lo Studio E La Cura Dei Tumori (IRST) Dino Amadori
Meldola, Italy, 47014
Actively Recruiting
69
IRCCS San Gerardo Dei Tintori
Monza, Italy, 20900
Actively Recruiting
70
Bambino Gesu Children's Hospital
Roma, Italy, PC 00165
Actively Recruiting
71
AOU Città della salute e della scienza di Torino
Torino, Italy, 10126
Actively Recruiting
72
Hospital Pakar Kanak-Kanak UKM
Kuala Lumpur, Malaysia, 56000
Actively Recruiting
73
Unidade Local de Saúde da Região de Aveiro
Aveiro, Portugal, 3810-164
Actively Recruiting
74
Unidade Local de Saude Coimbra - Paediatric Hospital
Coimbra, Portugal, 3000-602
Actively Recruiting
75
Unidade Local de Saude Coimbra - Quinta dos Vales
Coimbra, Portugal, 3041-801
Actively Recruiting
76
Unidade Local de Saude Santa Maria
Lisbon, Portugal, 1649-028
Actively Recruiting
77
Spitalul Clinic Colentina Site 1
Bucharest, Romania, 020125
Actively Recruiting
78
Spitalul Clinic Colentina Site 2
Bucharest, Romania, 020125
Actively Recruiting
79
Institutul Clinic Fundeni
Bucharest, Romania, 022328
Actively Recruiting
80
Institutul Clinic Fundeni
Bucharest, Romania, 22328
Actively Recruiting
81
Spitalul Clinic Județean de Urgență
Târgu Mureş, Romania, 540136
Actively Recruiting
82
University Clinical Centre of Serbia
Belgrade, Serbia, 11000
Actively Recruiting
83
ICO Badalona - Institut Català d'Oncologia
Barcelona, Spain, PC 08916
Actively Recruiting
84
Hospital Del Mar
Barcelona, Spain, PC08003
Actively Recruiting
85
Hospital San Pedro De Alcantara
Cáceres, Spain, 10003
Actively Recruiting
86
Hospital Universitario Reina Sofía
Córdoba, Spain, 14004
Withdrawn
87
Hospital Universitario de Gran Canaria Doctor Negrín
Las Palmas de Gran Canaria, Spain, 35010
Actively Recruiting
88
Hospital De La Princesa
Madrid, Spain, 28006
Actively Recruiting
89
Hospital Universitario Infanta Leonor
Madrid, Spain, 28031
Withdrawn
90
Hospital Universitario Fundacion Jimenez Diaz
Madrid, Spain, 28040
Actively Recruiting
91
Hospital Universitario Marqués de Valdecilla
Santander, Spain, 39008
Actively Recruiting
92
Hospital Universitario Virgen del Rocio
Seville, Spain, 41013
Actively Recruiting
93
Hospital Universitario y Politecnico de la Fe
Valencia, Spain, 46026
Actively Recruiting
94
Inselspital - University Hospital Bern Site 1
Bern, Switzerland, 3010
Actively Recruiting
95
Inselspital - University Hospital Bern Site 2
Bern, Switzerland, 3010
Actively Recruiting
96
Ramathibodi Hospital
Bangkok, Thailand, 10400
Actively Recruiting
97
Siriraj Hospital
Bangkok, Thailand, 10700
Actively Recruiting
98
Namik Kemal University (Tekirdağ Namık Kemal Üniversitesi)
Tekirdağ, Süleymanpaşa, Turkey (Türkiye), 59030
Actively Recruiting
99
Dr. Abdurrahman Yurtaslan Ankara Oncology Training And Research Hospital
Ankara, Turkey (Türkiye), 6000
Actively Recruiting
100
Ankara University School of Medicine
Ankara, Turkey (Türkiye), 6620
Actively Recruiting
101
Uludag University Faculty Of Medicine
Nilufer, Turkey (Türkiye)
Actively Recruiting
102
Ondokuz Mayis University Medical Faculty Hospital
Samsun, Turkey (Türkiye), 55200
Actively Recruiting
103
Lviv Regional Clinical Diagnostic Centre
Lviv, Oblast, Ukraine, 79010
Actively Recruiting
104
Medical Center Dobrobut-Clinic LLC
Kyiv, Ukraine, 03151
Actively Recruiting
105
Medical Center Ok Clinic of International Institute of Clinical Research LLC
Kyiv, Ukraine, 2091
Actively Recruiting
106
Llc Onto-Med
Kyiv, Ukraine, 3022
Actively Recruiting
107
Communal non-commercial enterprise "Ternopil City Children's Communal Hospital"
Ternopil, Ukraine, 46000
Actively Recruiting
108
Medical Center The Clinic of Hospodarskyy LLC
Ternopil, Ukraine, 46002
Actively Recruiting
109
University Hospital of Wales
Cardiff, United Kingdom, CF14 4XW
Actively Recruiting
110
Cardiff And Vale University Health Board
London, United Kingdom, CF14 4XW
Actively Recruiting
111
Royal Free London Nhs Foundation Trust Royal Free Hospital
London, United Kingdom, NW3 2QG
Actively Recruiting
112
King's College Hospital
London, United Kingdom, SE5 9RS
Actively Recruiting
113
Imperial College Healthcare Nhs Trust - Hammersmith Hospital
London, United Kingdom, W12 0HS
Actively Recruiting
114
Great Ormond street Hospital for Children NHS Trust
London, United Kingdom, WC1N 1DZ
Actively Recruiting
Research Team
P
Patient Affairs and Advocacy
How is the study designed?
Study Type
INTERVENTIONAL
Masking
QUADRUPLE
Allocation
RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
2
Similar Trials
Frequently Asked Questions
Have more questions? Get in touch with our team for quick support
Not the Right Trial for You?
Explore thousands of other clinical trials that might be a better match.
Sign up to get personalized trial recommendations delivered to your inbox.
Already have an account? Log in here