NeutropeniaPhase 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 study testing Mavorixafor in people with Neutropenia, run by X4 Pharmaceuticals at 114 sites in 25 countries.
Trial 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… Read more about this study
- Who is it for?
- Ages 12 years and older
- Conditions the study is for
- Neutropenia
- What is being tested?
- Mavorixafor
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 176 participants
- Where
- 114 sitesin 25 countries
- Study dates
- 6 Jun 2024to Nov 2027
- Phase
- Phase 3
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 12 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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
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- 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.
- 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.
Who cannot join
- A diagnosis of secondary neutropenia including those due to:
- Hypersplenism
- Infection
- Malignancy
- Autoimmune disease, for example, systemic lupus erythematosus, rheumatoid arthritis, inflammatory bowel disease, graft-versus-host disease, thyroid disease
- Nutritional deficiency, for example, vitamin B12, folic acid, copper, caloric malnutrition
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- 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, neutrophil expressed (ELANE)
- 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
- Associated with bone marrow failure, for example, Fanconi anemia, Diamond-Blackfan anemia
- 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.
Trial locations and contacts
114 sites in 25 countries. Contact the study team or a site near you to ask about taking part.
114 sites in 25 countries
United States22 sites, 21 recruiting
- Recruiting
University of Michigan
Ann Arbor, Michigan, 48109
- Recruiting
Children's Healthcare of Atlanta (CHOA)
Atlanta, Georgia, 30322
- Recruiting
Boston Children's Cancer Center
Boston, Massachusetts, 02143
- Recruiting
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
- Recruiting
Cleveland Clinic
Cleveland, Ohio, 44195
- Recruiting
UT Southwestern Medical Center
Dallas, Texas, 75390
- Recruiting
Duke University Medical Center
Durham, North Carolina, 27710
- Recruiting
Cook Children's Medical Center
Fort Worth, Texas, 76104
- Recruiting
Texas Children's Hospital/ Baylor College of Medicine
Houston, Texas, 77030
- Recruiting
University of Iowa
Iowa City, Iowa, 52242
- Recruiting
UC Irvine
Irvine, California, 92697
- Recruiting
Orso Health
La Jolla, California, 92037
- Recruiting
University of California, Los Angeles-UCLA
Los Angeles, California, 90095
- Recruiting
St. Jude Childrens; Research Hospital
Memphis, Tennessee, 38105
- Recruiting
Froedtert And Medical College Of Wisconsin
Milwaukee, Wisconsin, 53226
- Recruiting
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
- Recruiting
Phoenix Children's Hospital
Phoenix, Arizona, 85016
- Recruiting
University of Utah
Salt Lake City, Utah, 84112
- Recruiting
Washington University
St Louis, Missouri, 63130
- Recruiting
University of Southern Florida
St. Petersburg, Florida, 33701
- Withdrawn
Orso Health
Torrance, California, 90505
- Recruiting
Sarah Cannon Research Institute
Woodland, Texas, 77380
Spain11 sites, 9 recruiting
- Recruiting
ICO Badalona - Institut Català d'Oncologia
Barcelona, PC 08916
- Recruiting
Hospital Del Mar
Barcelona, PC08003
- Recruiting
Hospital San Pedro De Alcantara
Cáceres, 10003
- Withdrawn
Hospital Universitario Reina Sofía
Córdoba, 14004
- Recruiting
Hospital Universitario de Gran Canaria Doctor Negrín
Las Palmas de Gran Canaria, 35010
- Recruiting
Hospital De La Princesa
Madrid, 28006
- Withdrawn
Hospital Universitario Infanta Leonor
Madrid, 28031
- Recruiting
Hospital Universitario Fundacion Jimenez Diaz
Madrid, 28040
- Recruiting
Hospital Universitario Marqués de Valdecilla
Santander, 39008
- Recruiting
Hospital Universitario Virgen del Rocio
Seville, 41013
- Recruiting
Hospital Universitario y Politecnico de la Fe
Valencia, 46026
Italy7 sites, 7 recruiting
- Recruiting
ASST Spedali Civili di Brescia
Brescia, 25121
- Recruiting
IRCCS Instituto G. Gaslini
Genova, 16147
- Recruiting
Istituto Scientifico Romagnolo Per Lo Studio E La Cura Dei Tumori (IRST) Dino Amadori
Meldola, 47014
- Recruiting
IRCCS San Gerardo Dei Tintori
Monza, 20900
- Recruiting
Bambino Gesu Children's Hospital
Roma, PC 00165
- Recruiting
AOU Città della salute e della scienza di Torino
Torino, 10126
- Recruiting
A. O. U. delle Marche
Torrette, Ancona, 60126
France6 sites, 6 recruiting
- Recruiting
Institute of Hematology and Pediatric Oncology - Lyon
Lyon, 69008
- Recruiting
Hôpital Saint Louis
Paris, 75010
- Recruiting
Hospital Armand Trousseau Ap-Hp
Paris, 75012
- Recruiting
Hôpital Necker - Enfants Malade
Paris, 75015
- Recruiting
Centre Hospitalier Universitaire de Rennes
Rennes, 75033
- Recruiting
Institut Universitaire de Cancérologie de Toulouse - Oncopôle
Toulouse, 31059
Ukraine6 sites, 6 recruiting
- Recruiting
Medical Center Dobrobut-Clinic LLC
Kyiv, 03151
- Recruiting
Medical Center Ok Clinic of International Institute of Clinical Research LLC
Kyiv, 2091
- Recruiting
Llc Onto-Med
Kyiv, 3022
- Recruiting
Lviv Regional Clinical Diagnostic Centre
Lviv, Oblast, 79010
- Recruiting
Communal non-commercial enterprise "Ternopil City Children's Communal Hospital"
Ternopil, 46000
- Recruiting
Medical Center The Clinic of Hospodarskyy LLC
Ternopil, 46002
United Kingdom6 sites, 6 recruiting
- Recruiting
University Hospital of Wales
Cardiff, CF14 4XW
- Recruiting
Cardiff And Vale University Health Board
London, CF14 4XW
- Recruiting
Royal Free London Nhs Foundation Trust Royal Free Hospital
London, NW3 2QG
- Recruiting
King's College Hospital
London, SE5 9RS
- Recruiting
Imperial College Healthcare Nhs Trust - Hammersmith Hospital
London, W12 0HS
- Recruiting
Great Ormond street Hospital for Children NHS Trust
London, WC1N 1DZ
Romania5 sites, 5 recruiting
- Recruiting
Spitalul Clinic Colentina Site 1
Bucharest, 020125
- Recruiting
Spitalul Clinic Colentina Site 2
Bucharest, 020125
- Recruiting
Institutul Clinic Fundeni
Bucharest, 022328
- Recruiting
Institutul Clinic Fundeni
Bucharest, 22328
- Recruiting
Spitalul Clinic Județean de Urgență
Târgu Mureş, 540136
Turkey (Türkiye)5 sites, 5 recruiting
- Recruiting
Dr. Abdurrahman Yurtaslan Ankara Oncology Training And Research Hospital
Ankara, 6000
- Recruiting
Ankara University School of Medicine
Ankara, 6620
- Recruiting
Uludag University Faculty Of Medicine
Nilufer
- Recruiting
Ondokuz Mayis University Medical Faculty Hospital
Samsun, 55200
- Recruiting
Namik Kemal University (Tekirdağ Namık Kemal Üniversitesi)
Tekirdağ, Süleymanpaşa, 59030
Colombia4 sites, 4 recruiting
- Recruiting
Clinica IMBANACO S.A.S
Cali, 76004
- Recruiting
Clínica del Country
Chapinero, 0813
- Recruiting
Fundacion Cardiovascular de Colombia-Instituto del Corazon Florida Blanca
Floridablanca, Santander Department, 681003
- Recruiting
Hospital Pablo Tobón Uribe
Medellín, 500034
Georgia4 sites, 4 recruiting
- Recruiting
LTD "Israeli-Georgian Medical Research Clinic Healthycore"
Tbilisi, 112
- Recruiting
LEPL The First University Clinic of Tbilisi State Medical University
Tbilisi, 141
- Recruiting
JSC K. Eristavi National Center of Experimental and Clinical Surgery
Tbilisi, 159
- Recruiting
LTD Multiprofile Clinic Concilium Medulla
Tbilisi, 186
Greece4 sites, 4 recruiting
- Recruiting
Hellenic Airforce 251 General Hospital
Athens, 10177
- Recruiting
University General Hospital of Heraklion
Heraklion, 71500
- Recruiting
The Olympion Private General Clinic
Pátrai, 26443
- Recruiting
AHEPA General Hospital of Thessaloniki
Thessaloniki, 54636
India4 sites
- Terminated
Yashoda Hospital
Hyderabad, Telangana, 500084
- Withdrawn
Indira Gandhi Institute Of Medical Sciences
Patna, Bihar, 800014
- Withdrawn
Nirmal Hospital Private Limited
Surat, Gujarat, 395002
- Withdrawn
Pacific Medical College and Hospital
Udaipur, Rajasthan, 313001
Israel4 sites, 4 recruiting
- Recruiting
Ruth Rappaport Children's Hospital, Rambam Health Corporation
Haifa, 3109601
- Recruiting
Hadassah University Hospital-ein Kerem
Jerusalem, 9112001
- Recruiting
Schneider Children's Medical Center of Israel
Petah Tikva, 4920235
- Recruiting
The Chaim Sheba Medical Center
Ramat Gan, 5265601
Portugal4 sites, 4 recruiting
- Recruiting
Unidade Local de Saúde da Região de Aveiro
Aveiro, 3810-164
- Recruiting
Unidade Local de Saude Coimbra - Paediatric Hospital
Coimbra, 3000-602
- Recruiting
Unidade Local de Saude Coimbra - Quinta dos Vales
Coimbra, 3041-801
- Recruiting
Unidade Local de Saude Santa Maria
Lisbon, 1649-028
Argentina3 sites, 3 recruiting
- Recruiting
Hospital El Cruce
Buenos Aires, B1888AAE
- Recruiting
Instituto de Investigaciones Clínicas Córdoba
Córdoba, X5000AAW
- Recruiting
Santorio 9 de Julio
San Miguel de Tucumán, T4000IIH
Australia3 sites, 3 recruiting
- Recruiting
Perth Children's Hospital
Nedlands, 6009
- Recruiting
Icon Cancer Centre Southport
Southport, 4215
- Recruiting
Ballarat Oncology And Haematology Services
Wendouree, 3355
Hungary3 sites, 3 recruiting
- Recruiting
CMC Deli Klinika
Budapest, 1012
- Recruiting
Semmelweis University
Budapest, 1085
- Recruiting
Teaching Hospital Markusovszky
Szombathely, 9700
Canada2 sites, 2 recruiting
- Recruiting
University of Alberta Hospital
Edmonton, AB T6G 2R3
- Recruiting
BC Children's Hospital
Vancouver, V5Z 4H4
Czechia2 sites, 2 recruiting
- Recruiting
The University Hospital Brno
Brno, 62500
- Recruiting
Motol University Hospital
Prague, 15006
Germany2 sites, 2 recruiting
- Recruiting
Klinik und Poliklinik fur Kinder- und Jugendmedizin, Universitatsklinikum Carl Gustav Carus an der TU
Dresden, 01307
- Recruiting
Universitaetsklinikum Carl Gustav Carus Tu Dresden
Dresden, 01307
Switzerland2 sites, 2 recruiting
- Recruiting
Inselspital - University Hospital Bern Site 1
Bern, 3010
- Recruiting
Inselspital - University Hospital Bern Site 2
Bern, 3010
Thailand2 sites, 2 recruiting
- Recruiting
Ramathibodi Hospital
Bangkok, 10400
- Recruiting
Siriraj Hospital
Bangkok, 10700
Belgium1 site, 1 recruiting
- Recruiting
Universitair Ziekenhuis Leuven
Leuven, 3000
Malaysia1 site, 1 recruiting
- Recruiting
Hospital Pakar Kanak-Kanak UKM
Kuala Lumpur, 56000
Serbia1 site, 1 recruiting
- Recruiting
University Clinical Centre of Serbia
Belgrade, 11000
About this study
Official title: 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
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.
Read the detailed description
All participants will continue their pre-study background therapy, defined as the participant's current treatment regimen. Options include, but are not limited to, granulocyte-colony stimulating factor (G-CSF), immunoglobulin replacement therapy, prophylactic antibiotics, or "watchful waiting".
- Conditions
- Neutropenia
- Being tested
- Mavorixafor, Placebo
- Keywords
- Mavorixafor, Chronic neutropenia, Chronic idiopathic neutropenia, Severe Congenital Neutropenia (SCN), C-X-C chemokine receptor 4 (CXCR4), Congenital and acquired neutropenia, Autoimmune disease, Cohen syndrome, Barth syndrome, ELANE, G6PC3, GSD1b, Kostmann disease, Shwachman-Diamond syndrome, Autoimmune neutropenia (AIN)
- Medical subject headings
- Neutropenia, Neutropenia, Severe Congenital, Autosomal Recessive 3, Autoimmune Diseases, Cohen syndrome, Barth Syndrome, Shwachman-Diamond Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MavorixaforDrug
Also called X4P-001
Mavorixafor will be administered per schedule specified in the arm description.
PlaceboDrug
Placebo will be administered per schedule specified in the arm description.
| Study group | Role | What they receive |
|---|---|---|
| Mavorixafor | Experimental | Participants will receive mavorixafor orally once daily starting from Day 1 through Week 52. Receives: Drug: Mavorixafor |
| Placebo | Placebo comparator | Participants will receive placebo to match mavorixafor orally once daily starting from Day 1 through Week 52. Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Co-primary Endpoint: Annualized Infection Rate Based on Infections Adjudicated by Blinded Infection Adjudication Committee (BIAC) During the Treatment Period
Measured: Up to 52 Weeks
Co-primary Endpoint: Number of Participants Meeting the Definition of a Positive Absolute Neutrophil Count (ANC) Response
Positive ANC response: Increase of ANC >500 cells/microliter (µL) from baseline.
Measured: Up to 52 weeks
Secondary outcomes (5)
Infection Severity Based on Common Terminology Criteria for Adverse Events (CTCAE) Adjudicated by a BIAC During the Treatment Period
Measured: Up to 52 Weeks
Infection Duration Based on Duration of Infections Adjudicated by a BIAC During the Treatment Period in Those Participants who Developed Infections
Measured: Up to 52 Weeks
Antibiotic Use Due to Infection, Characterized by the Frequency of Antibiotic Use During the Treatment Period
Measured: Up to 52 Weeks
Oral Ulcers, as Assessed by Presence or Absence of Ulcers During the Treatment Period
Measured: Up to 52 Weeks
Change From Baseline in Patient Reported Outcomes Measurement Information System Short Form (PROMIS SF) Fatigue Questionnaire Total Score
Measured: Baseline, Week 52
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Sep 2023
- Study start6 Jun 2024
- Primary completionSep 2027 (estimated)
- Study completionNov 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for NeutropeniaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 176 participants (estimated)
Sponsor and investigators
- Lead sponsor
- X4 Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Vice President Global Head of Clinical Development and Safety, Study director, X4 Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06056297
- Sponsor's protocol ID
- X4P-001-110
- Other IDs
- 2023-508482-32-00, 2023
- Status last verified
- Aug 2026
- First submitted
- 20 Sep 2023
- Last updated
- 6 Aug 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No