Clinical trial • Phase III • Immunology|Haematology

MAVORIXAFOR for Chronic neutropenia|Congenital neutropenia|Autoimmune neutropenia|Idiopathic neutropenia

Phase III trial of MAVORIXAFOR for Chronic neutropenia|Congenital neutropenia|Autoimmune neutropenia|Idiopathic neutropenia.

Overview

Trial Therapeutic Area
Immunology|Haematology
Trial Disease
Chronic neutropenia|Congenital neutropenia|Autoimmune neutropenia|Idiopathic neutropenia
Trial Stage
Phase III
Drug Modality
Small molecule
Paediatric Trial
Yes

Key dates

Initial CTIS Submission Date
26-02-2024
First CTIS Authorization Date
14-06-2024

Trial design

Randomised, placebo (mavorixafor placebo capsules); no active comparator dose/schedule specified in the available documents-controlled Phase III trial in Portugal, Spain, France and others.

Randomised
Yes
Comparator
Placebo (Mavorixafor Placebo Capsules); no active comparator dose/schedule specified in the available documents
Target Sample Size
128
Trial Duration For Participant
365

Eligibility

Recruits 128 paediatric patients.

Pregnancy Exclusion
Participant is pregnant or breastfeeding.
Vulnerable Population
Includes participants aged 12 years and older (minors 12–17 years). Consent may be provided by the participant and/or parent and/or appropriate legally designated representative; assent is required for minors as described in the protocol (Appendix 1 Section 10.1.3). Age-specific ICF and assent documents are provided (adult ICF, parental ICF, assent forms for 12–15 and 16–17).

Inclusion criteria

  • {"criterion_text":"- Participants must be at least 12 years of age, at the time of signing the informed consent/assent, as per the local regulations and guidelines.\n- 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.\n- Bone marrow aspirate ± biopsy during the screening visit (or prior documentation of bone marrow aspirate ± biopsy within previous 9 months submitted for review and considered adequate for type of CN by central review hematopathologist) does not demonstrate evidence of hematologic malignancy or high risk for transformation by central review hematopathologist.\n- Body weight of ≥ 15 kg (inclusive).\n- Contraceptive use by men and women must be consistent with local regulations regarding the methods of contraception for those participating in clinical studies. Male Participants: •\tA male participant must agree to use highly effective contraception as detailed in Appendix 3 of this protocol during the treatment period and for at least 3 weeks after the last dose for participants early terminating the study or until EOS visit for participants completing the Week 52/Day 365 visit and refrain from donating sperm during this period. Female Participants: •\tA female participant is eligible to participate if she is not pregnant (see Appendix 3), not breastfeeding, and at least one of the following conditions applies: \tNot a woman of childbearing potential (WOCBP) as defined in Appendix 3. OR \tA WOCBP who agrees to follow the contraceptive guidance, as mentioned in Appendix 3 during the treatment period and for at least 3 weeks after the last dose of study drug for participants early terminating the study or until EOS visit for participants completing the Week 52/Day 365 visit.\n- Participant, parent, and/or appropriate legally designated representative is capable of giving signed ICF and/or assent as described in Appendix 1, Section 10.1.3, which includes compliance with the requirements and restrictions listed in the ICF and in this protocol.\n- 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. •\tCongenital Neutropenia, including but not limited to these classifications: a.\tIsolated with a permanent (non-cyclic) presentation, e.g., ELANE, CSF3R, CXCR2, WAS b.\tAssociated with extra-hematologic manifestations, e.g., Barth syndrome, Cohen syndrome, G6PC3, Kostmann disease c.\tAssociated with metabolic disorders, e.g., glycogen storage disease 1b (GSD1b) d.\tShwachman-Diamond syndrome •\tAcquired Primary Neutropenia a.\tChronic idiopathic neutropenia b.\tPrimary autoimmune neutropenia Other CN disorders that may be eligible for enrollment can be clarified and approved upon discussion with the study Medical Monitor\n- 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. Note: In the event of a systemic infection during the screening visit that may, in the opinion of the Investigator, have an effect on ANC, the baseline visit may be postponed or repeated, as deemed appropriate by the Investigator, to confirm trough ANC < 1000 cells/μL. Repeat measures should be justified with reason to believe the measure would change and should be limited to 3 times for any single type of event.\n- Prior history of recurrent and/or serious infections during the 12 months preceding the screening visit (i.e., suffering sequelae of CN), as defined by having at least 2 infections in the last 12 months that meet the following criteria: •\tInfection requiring the use of antibiotics (intravenous [IV]/oral); OR •\tInfection 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: •\tInfections considered by the Investigator to be likely related to the potential participant’s CN disorder. Note: Although oral ulcers (i.e., canker sores, aphthous ulcers) are sequelae of CN, they are not considered as de novo infections for the purpose of eligibility. If the finding of the oral ulcer(s) and/or oral mucositis are either thought to be exacerbated by or as a result of an infection, e.g., fungal etiology, then they can be considered an infection. Recurrent herpes simplex virus (HSV) or human papillomavirus (HPV) oral or genital lesions are NOT classified for the purpose of this study as infections. If such lesions have signs of secondary bacterial or fungal etiology, they can be considered infections. Gingivitis is NOT to be considered an infection. Periodontitis can be considered an infection.\n- 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 (Note: for participants receiving chronic G-CSF treatment, dosing modifications may be considered for safety reasons [e.g., if ANC > 10,000 cells/µL for ≥ 4 weeks; refer to Section 6.1.2])."}

Exclusion criteria

  • {"criterion_text":"- Participant is incapacitated and unable to comply with protocol-specific requirements\n- 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.\n- Participants who are awaiting HSCT due to somatic variants in genes associated with high risk for clonal proliferation.\n- An active malignancy or history (≤ 5 years prior to enrollment in the study) of solid or hematologic malignancy.\n- Exception: Adequately treated basal cell or squamous cell skin cancer, localized prostate cancer, carcinoma in situ of the cervix, , in situ ductal or lobular carcinoma of the breast, or stage 1, low-grade colon cancer after surgical treatment. Participants with a prior or concurrent solid tumor whose natural history or treatment does not have the potential to interfere with the safety or efficacy assessment of the investigational regimen, in the opinion of the Medical Monitor, may be eligible\n- Laboratory test results meeting ≥ 1 of the following criteria at the screening visit: •\tHemoglobin < 9.0 g/dL •\tPlatelets < 30,000/μL •\tEstimated glomerular filtration rate < 30 mL/min/1.73 m2, as estimated by the Chronic Kidney Disease Epidemiology Collaboration equation (age ≥ 18 years) or Schwartz equation (age 12-17 years). •\tSerum aspartate transaminase > 2.5 × upper limit of normal (ULN) •\tSerum alanine transaminase > 2.5 × ULN •\tTotal bilirubin > 1.5 × ULN (unless due to Gilbert’s syndrome, in which case total bilirubin ≥ 3.0 × ULN and direct bilirubin > 1.5 × ULN)\n- Prolonged corrected QT interval > 450 ms using Fridericia’s formula at the screening visit.\n- Participant is currently taking or has taken an investigational drug < 30 days prior to the screening visit, or 5 half-lives, whichever is longer.\n- Participant is pregnant or breastfeeding.\n- Unable and/or unwilling to swallow capsules.\n- Known systemic hypersensitivity to the mavorixafor drug substance, its inactive ingredients, or the placebo.\n- Grapefruit-containing products, which are variable inhibitors of CYP3A4, are prohibited from the day the first dose of study drug is given and during the study.\n- Diagnosed or suspected congenital long QT syndrome or any history of clinically significant (CS) ventricular arrhythmias (such as ventricular tachycardia, ventricular fibrillation, or torsades de pointes). Any history of arrhythmia will be discussed with the Medical Monitor before the participant’s entry into the study.\n- Receiving or requiring any medication/therapy that is prohibited (see Section 6.6.2.3).\n- Received more than 1 dose of mavorixafor in the past.\n- Received a CXCR4 antagonist (other than mavorixafor) in the past 6 months.\n- Participants taking pegylated-G-CSF unless they have a diagnosis of congenital neutropenia confirmed at the screening visit.\n- Unless a published drug metabolism exception is otherwise indicated by the Investigator following review, drugs which are (a) highly dependent on CYP2D6 for clearance are prohibited for a period starting 14 days or 5 half-lives, whichever is longer, prior to administration of study drug and during the study and (b) which are strong CYP3A4 inducers are prohibited for a period starting 7 days or 5 half-lives, whichever is longer, prior to the administration of study drug and during the study (see Appendix 5).\n- Systemic glucocorticoids (> 5 mg prednisone equivalent per day) are prohibited for a period starting 14 days or 5 half-lives, whichever is longer, prior to administration of study drug and during the study.\n- A diagnosis of secondary neutropenia including those due to: a.\tHypersplenism b.\tInfection c.\tMalignancy d.\tAutoimmune disease, e.g., systemic lupus erythematosus, rheumatoid arthritis, inflammatory bowel disease, graft-versus-host disease, thyroid disease e.\tNutritional deficiency, e.g., vitamin B12, folic acid, copper, caloric malnutrition f.\tDrug-induced cause, e.g., chemotherapy, clozapine, antiretrovirals, antibiotics, monoclonal antibodies.\n- Positive hepatitis C virus (HCV) antibodies with confirmation by HCV ribonucleic acid polymerase chain reaction reflex testing.\n- Positive hepatitis B surface antigen (HBsAg) or hepatitis B core antibody (HBcAb). If the participant tests HBsAg negative, HBcAb positive, and hepatitis B surface antibody positive upon reflex testing, the participant would be considered eligible.\n- A diagnosis of any of the following: •\tAplastic anemia •\tWHIM syndrome •\tCertain CNs, including but not limited to these classifications, are excluded: a.\tIsolated with a cyclic presentation, e.g., ELANE b.\tAssociated with immune dysregulation, e.g., CVID, ALPS, familial hemophagocytic lymphohistiocytosis, Chédiak-Higashi syndrome, GATA2 deficiency syndrome c.\tAssociated with bone marrow failure, e.g., Fanconi anemia, Diamond-Blackfan anemia •\tNeutropenia 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.\n- A history of HIV and an acquired immunodeficiency syndrome-defining condition other than CD4+ count < 200 cells/µL. Participants with HIV may be enrolled if viral load as determined by routinely used tests has been undetectable for at least 6 months prior to the screening visit; if the participant is taking effective antiretroviral therapy (ART), regimen must have been stable for > 4 weeks prior to the screening visit.\n- Known active COVID 19 infection or a positive test within the local accepted clinical and governmental guidelines for a communicable window. Note: Participants with prior COVID 19 exposure are permitted to enroll if they have a negative test and conform with local guidelines.\n- Major surgery (defined as any invasive procedure that involves penetrating or exposing a body cavity) ≤ 6 weeks before the baseline visit requiring general anesthesia or which, in the opinion of the Investigator, may compromise the safety of the participant."}

Endpoints

Primary endpoints

  • {"endpoint_text":"- Annualized infection rate based on infections adjudicated by a BIAC during the 52-week treatment period","definition_or_measurement_approach":"Annualized infection rate measured based on infections adjudicated by a BIAC during the 52-week treatment period."}
  • {"endpoint_text":"- 2a: Proportion of ANC responders. An ANC responder is a participant meeting the definition of a positive ANC response for at least 3 out of 6 visits [Weeks 4, 8, 13, 26, 39, and 52] during the 52-week treatment period, where a positive ANC response is defined as: (please see next \"ID 3\")","definition_or_measurement_approach":"Proportion of participants meeting positive ANC response at ≥3 of the 6 scheduled visits (Weeks 4, 8, 13, 26, 39, 52) during the 52-week treatment period."}
  • {"endpoint_text":"- 2b: (continued from ID 2) •\tANC ≥ 1500 cells/µL, with the exception of participants with baseline ANC < 500 cells/µL •\t≥ 2-fold increase in ANC from baseline, for participants with baseline ANC < 500 cells/µL","definition_or_measurement_approach":"A positive ANC response is defined as ANC ≥1500 cells/µL (except for participants with baseline ANC <500 cells/µL, where a ≥2-fold increase from baseline is the criterion). ANC measurements use trough mean ANC (mean of multiple ANC measurements over 6 hours where applicable)."}

Secondary endpoints

  • {"endpoint_text":"- Infection severity based on CTCAE grading, adjudicated by a BIAC during the 52-week treatment period","definition_or_measurement_approach":"Severity graded per CTCAE and adjudicated by a BIAC during the 52-week treatment period."}
  • {"endpoint_text":"- Infection duration based on duration of infections adjudicated by a BIAC during the 52-week treatment period","definition_or_measurement_approach":"Duration measured as duration of infections adjudicated by a BIAC during the 52-week treatment period."}
  • {"endpoint_text":"- Antibiotic use due to infection, characterized by the frequency of antibiotic use during the 52-week treatment period","definition_or_measurement_approach":"Characterized by frequency of antibiotic use for infections during the 52-week treatment period."}
  • {"endpoint_text":"- Oral ulcers, as assessed by presence or absence of ulcers, during the 52-week treatment period","definition_or_measurement_approach":"Presence/absence assessment of oral ulcers during the 52-week treatment period."}
  • {"endpoint_text":"- Change from baseline to Week 52/Day 365 in PROMIS SF Fatigue Questionnaire total score","definition_or_measurement_approach":"Change from baseline to Week 52/Day 365 in total score of the PROMIS SF Fatigue questionnaire (PRO)."}

Recruitment

Digital Remote Recruitment
True, recruitment includes digital/remote methods (LeapCure digital recruitment materials, online pre-screener questionnaires, telephone pre-screening / Pre-ICF telephone data and digital advertisement materials as listed in recruitment documents).
Planned Sample Size
128
Recruitment Window Months
21
Consent Approach
Informed consent obtained from the participant and/or parent and/or appropriate legally designated representative as applicable; assent required for minors per protocol (age-specific forms: assent 12–15, 16–17); multiple language ICF/assent documents provided (e.g., English, Portuguese, Spanish, French, German, Italian, Hungarian, Romanian, Czech, Greek, Polish, Dutch as reflected in published ICF and patient-facing documents).

Methods

  • LeapCure: recruitment support and advertisement including digital recruitment materials and pre-screener questionnaires (documents: 'LeapCure Digital Recruitment Materials', 'LeapCure Pre-screener Questionnaire', 'LeapCure Patient Journey')
  • Scout Clinical: patient travel concierge and reimbursement support (contact: rianna.jackson@scoutclinical.com)
  • Third-party site- and country-specific recruitment arrangements (K1 documents) and pre-study handouts for potential participants (documents associated with multiple Member States)

Geography

Total Number Of Sites
49
Total Number Of Participants
86

Portugal

Earliest CTIS Part Ii Submission Date
02-08-2024
Latest Decision Or Authorization Date
16-02-2026
Processing Time Days
593
Number Of Sites
4
Number Of Participants
8

Sites

Site Name
Unidade Local De Saude De Santa Maria E.P.E.
Department Name
Inmunoallergology
Principal Investigator Name
Susana Lopes da Silva
Principal Investigator Email
susanasilva@medicina.ulisboa.pt
Contact Person Name
Susana Lopes da Silva
Site Name
Unidade Local De Saude Da Regiao De Aveiro E.P.E.
Department Name
Hematology
Principal Investigator Name
Dino Luiz
Principal Investigator Email
dino.luis.71474@chbv.min-saude.pt
Contact Person Name
Dino Luiz
Site Name
Unidade Local De Saude De Coimbra E.P.E.
Department Name
Hematology
Principal Investigator Name
Joana Azevedo
Principal Investigator Email
joana.azevedo@ulscoimbra.min-saude.pt
Contact Person Name
Joana Azevedo
Site Name
Unidade Local de Saúde de Coimbra, EPE - Hospital Geral
Department Name
Hematology
Principal Investigator Name
Joana Azevedo
Principal Investigator Email
joana.azevedo@ulscoimbra.min-saude.pt
Contact Person Name
Joana Azevedo

Spain

Earliest CTIS Part Ii Submission Date
24-05-2024
Latest Decision Or Authorization Date
16-02-2026
Processing Time Days
633
Number Of Sites
12
Number Of Participants
8

Sites

Site Name
University Hospital Marqués de Valdecilla
Department Name
Hematology
Principal Investigator Name
Miguel Ángel Cortés Vázquez
Principal Investigator Email
miguelangel.cortes@scsalud.es
Contact Person Name
Miguel Ángel Cortés Vázquez
Contact Person Email
miguelangel.cortes@scsalud.es
Site Name
El Hospital Universitario De Gran Canaria Dr. Negrin
Department Name
Hematology
Principal Investigator Name
Juan Francisco López
Principal Investigator Email
juanfra5226@gmail.com
Contact Person Name
Juan Francisco López
Contact Person Email
juanfra5226@gmail.com
Site Name
University Hospital Son Espases
Department Name
Haematology
Principal Investigator Name
Laura Lo Riso
Principal Investigator Email
laura.loriso@ssib.es
Contact Person Name
Laura Lo Riso
Contact Person Email
laura.loriso@ssib.es
Site Name
Hospital Universitario De La Princesa
Department Name
Hematology
Principal Investigator Name
Adrián Alegre Amor
Principal Investigator Email
adrian.alegre@salud.madrid.org
Contact Person Name
Adrián Alegre Amor
Contact Person Email
adrian.alegre@salud.madrid.org
Site Name
Hospital San Pedro de Alcantara
Department Name
Hematology
Principal Investigator Name
Baldomero Javier Moriano Palacios
Principal Investigator Email
javier.moriano@salud-juntaex.es
Contact Person Name
Baldomero Javier Moriano Palacios
Site Name
University Hospital Virgen Del Rocio S.L.
Department Name
Pediatric Immunodeficiencies
Principal Investigator Name
Olaf Neth
Principal Investigator Email
olafw.nethsspa@juntadeandalucia.es
Contact Person Name
Olaf Neth
Site Name
Institut Catala D'oncologia
Department Name
Haematology
Principal Investigator Name
Inés Hernandez Rodriguez
Principal Investigator Email
ihernandez@iconcologia.net
Contact Person Name
Inés Hernandez Rodriguez
Contact Person Email
ihernandez@iconcologia.net
Site Name
Hospital Universitario Y Politecnico La Fe
Department Name
Aparicio
Principal Investigator Name
Bienvenida Argilés
Principal Investigator Email
argiles_bie@gva.es
Contact Person Name
Bienvenida Argilés
Contact Person Email
argiles_bie@gva.es
Site Name
Hospital Universitario Infanta Leonor
Department Name
Mori
Principal Investigator Name
Karen del Carmen Marín
Principal Investigator Email
karen.marin@salud.madrid.org
Contact Person Name
Karen del Carmen Marín
Contact Person Email
karen.marin@salud.madrid.org
Site Name
Hospital Del Mar
Department Name
Haematology
Principal Investigator Name
Sara Garcia Avila
Principal Investigator Email
sgarciaavila@psmar.cat
Contact Person Name
Sara Garcia Avila
Contact Person Email
sgarciaavila@psmar.cat
Site Name
Hospital General Universitario Reina Sofia
Department Name
Immunology
Principal Investigator Name
Aurora Jurado Roger
Principal Investigator Email
Aurora.jurado.sspa@juntadeandalucia.es
Contact Person Name
Aurora Jurado Roger
Site Name
Hospital Universitario Fundacion Jimenez Diaz
Department Name
Calvo
Principal Investigator Name
Nuria Revilla
Principal Investigator Email
nuria.revilla@quironsalud.es
Contact Person Name
Nuria Revilla
Contact Person Email
nuria.revilla@quironsalud.es

France

Earliest CTIS Part Ii Submission Date
29-08-2024
Latest Decision Or Authorization Date
17-02-2026
Processing Time Days
537
Number Of Sites
6
Number Of Participants
20

Sites

Site Name
Hospices Civils De Lyon
Department Name
IHOPe
Principal Investigator Name
Nathalie Garnier
Principal Investigator Email
nathalie.garnier@ihope.fr
Contact Person Name
Nathalie Garnier
Contact Person Email
nathalie.garnier@ihope.fr
Site Name
Assistance Publique Hopitaux De Paris
Department Name
Hematology
Principal Investigator Name
Flore Sicre De Fontbrune
Principal Investigator Email
flore.sicre-de-fontbrune@aphp.fr
Contact Person Name
Flore Sicre De Fontbrune
Site Name
Trousseau Hospital
Department Name
Hemato Oncology
Principal Investigator Name
Jean Donadieu
Principal Investigator Email
jean.donadieu@aphp.fr
Contact Person Name
Jean Donadieu
Contact Person Email
jean.donadieu@aphp.fr
Site Name
Centre Hospitalier Universitaire De Rennes
Department Name
Hematology
Principal Investigator Name
Aline Moignet Autrel
Principal Investigator Email
aline.moignet@chu-rennes.fr
Contact Person Name
Aline Moignet Autrel
Contact Person Email
aline.moignet@chu-rennes.fr
Site Name
Hopital Necker Enfants Malades
Department Name
Hematology
Principal Investigator Name
Felipe Suarez
Principal Investigator Email
felipe.suarez@aphp.fr
Contact Person Name
Felipe Suarez
Contact Person Email
felipe.suarez@aphp.fr
Site Name
Centre Hospitalier Universitaire De Toulouse
Department Name
Hématologue
Principal Investigator Name
Jérémie Dion
Principal Investigator Email
drci.toulouse@chu-toulouse.fr
Contact Person Name
Jérémie Dion
Contact Person Email
drci.toulouse@chu-toulouse.fr

Hungary

Earliest CTIS Part Ii Submission Date
24-05-2024
Latest Decision Or Authorization Date
24-02-2026
Processing Time Days
641
Number Of Sites
3
Number Of Participants
4

Sites

Site Name
Complex Medical Center Déli Klinika
Department Name
Hematology
Principal Investigator Name
Gergely Kriván
Principal Investigator Email
info@deliklinika.hu
Contact Person Name
Gergely Kriván
Contact Person Email
info@deliklinika.hu
Site Name
Vas Varmegyei Markusovszky Egyetemi Oktatokorhaz
Department Name
Hematology Department
Principal Investigator Name
Márk Plander
Principal Investigator Email
plander.mark@markusovszky.hu
Contact Person Name
Márk Plander
Contact Person Email
plander.mark@markusovszky.hu
Site Name
Semmelweis University
Department Name
Hematology
Principal Investigator Name
Zsolt György Nagy
Principal Investigator Email
nagy.zsolt@med.semmelweis-univ.hu
Contact Person Name
Zsolt György Nagy

Belgium

Earliest CTIS Part Ii Submission Date
13-08-2025
Latest Decision Or Authorization Date
16-02-2026
Processing Time Days
187
Number Of Sites
1
Number Of Participants
5

Sites

Site Name
UZ Leuven
Department Name
Pediatric Immunodeficiencies
Principal Investigator Name
Isabelle Meyts
Principal Investigator Email
Isabelle.Meyts@uzleuven.be
Contact Person Name
Isabelle Meyts
Contact Person Email
Isabelle.Meyts@uzleuven.be

Germany

Earliest CTIS Part Ii Submission Date
24-05-2024
Latest Decision Or Authorization Date
17-02-2026
Processing Time Days
634
Number Of Sites
2
Number Of Participants
4

Sites

Site Name
Universitaetsklinikum Carl Gustav Carus Dresden an der Technischen Universitaet Dresden AöR
Department Name
Medizinische Klinik und Poliklinik 1
Principal Investigator Name
Katja Sockel
Principal Investigator Email
katja.sockel@uniklinikum-dresden.de
Contact Person Name
Katja Sockel
Site Name
Universitaetsklinikum Carl Gustav Carus Dresden an der Technischen Universitaet Dresden AöR
Department Name
Department of Pediatrics, Dresden University
Principal Investigator Name
Catharina Schuetz
Principal Investigator Email
catharina.schuetz@uniklinikum-dresden.de
Contact Person Name
Catharina Schuetz

Romania

Earliest CTIS Part Ii Submission Date
20-05-2024
Latest Decision Or Authorization Date
24-02-2026
Processing Time Days
645
Number Of Sites
5
Number Of Participants
4

Sites

Site Name
Spitalul Clinic Judetean De Urgenta Targu Mures
Department Name
Internal Medicine 1 - Hematology
Principal Investigator Name
Ioan Macarie
Principal Investigator Email
theodora.benedek@umfst.ro
Contact Person Name
Ioan Macarie
Contact Person Email
theodora.benedek@umfst.ro
Site Name
Institutul Clinic Fundeni
Department Name
Hematology 3
Principal Investigator Name
Ana-Maria Moldovianu
Principal Investigator Email
secretariat@icfundeni.ro
Contact Person Name
Ana-Maria Moldovianu
Contact Person Email
secretariat@icfundeni.ro
Site Name
Spitalul Clinic Colentina Bucuresti
Department Name
Hematology
Principal Investigator Name
Georgeta Daniela Georgescu
Principal Investigator Email
secretariat@spitalulcolentina.ro
Contact Person Name
Georgeta Daniela Georgescu
Site Name
Spitalul Clinic Colentina Bucuresti
Department Name
Hematology
Principal Investigator Name
Viola-Maria Popov
Principal Investigator Email
secretariat@spitalulcolentina.ro
Contact Person Name
Viola-Maria Popov
Site Name
Institutul Clinic Fundeni
Department Name
Pediatric Clinic
Principal Investigator Name
Anca Colita
Principal Investigator Email
secretariat@icfundeni.ro
Contact Person Name
Anca Colita
Contact Person Email
secretariat@icfundeni.ro

Ireland

Earliest CTIS Part Ii Submission Date
01-09-2025
Latest Decision Or Authorization Date
16-02-2026
Processing Time Days
168
Number Of Sites
1
Number Of Participants
5

Sites

Site Name
Children's Health Ireland
Department Name
Haematology
Principal Investigator Name
Melanie Cotter
Principal Investigator Email
melanie.cotter1@childrenshealthireland.ie
Contact Person Name
Melanie Cotter

Italy

Earliest CTIS Part Ii Submission Date
21-08-2024
Latest Decision Or Authorization Date
25-02-2026
Processing Time Days
553
Number Of Sites
7
Number Of Participants
14

Sites

Site Name
IRCCS Istituto Giannina Gaslini
Department Name
Haematology Department
Principal Investigator Name
Francesca Fioredda
Principal Investigator Email
francescafioredda@gaslini.org
Contact Person Name
Francesca Fioredda
Contact Person Email
francescafioredda@gaslini.org
Site Name
Istituto Romagnolo Per Lo Studio Dei Tumori Dino Amadori IRST S.r.l.
Department Name
Medical Oncology
Principal Investigator Name
Alessandro Lucchesi
Principal Investigator Email
alessandro.lucchesi@irst.emr.it
Contact Person Name
Alessandro Lucchesi
Site Name
Azienda Socio Sanitaria Territoriale Degli Spedali Civili Di Brescia
Department Name
Pediatric Clinic
Principal Investigator Name
Raffaele Badolato
Principal Investigator Email
raffaele.badolato@unibs.it
Contact Person Name
Raffaele Badolato
Contact Person Email
raffaele.badolato@unibs.it
Site Name
Azienda Ospedaliero Universitaria Ospedali Riuniti Umberto I G M Lancisi G Salesi
Department Name
Hematology Department
Principal Investigator Name
Antonella Poloni
Principal Investigator Email
a.poloni@staff.univpm.it
Contact Person Name
Antonella Poloni
Contact Person Email
a.poloni@staff.univpm.it
Site Name
Fondazione IRCCS San Gerardo Dei Tintori
Department Name
Haematology Department
Principal Investigator Name
Filippo Achille Brioschi
Contact Person Name
Filippo Achille Brioschi
Site Name
Azienda Ospedaliera Universitaria Citta Della Salute E Della Scienza Di Torino
Department Name
Hematology
Principal Investigator Name
Chiara Frairia
Principal Investigator Email
cfrairia@cittadellasalute.to.it
Contact Person Name
Chiara Frairia
Site Name
Ospedale Pediatrico Bambino Gesu
Department Name
Paediatric Onco-Haematology Department
Principal Investigator Name
Franco Locatelli
Principal Investigator Email
franco.locatelli@opbg.net
Contact Person Name
Franco Locatelli
Contact Person Email
franco.locatelli@opbg.net

Poland

Earliest CTIS Part Ii Submission Date
11-08-2025
Latest Decision Or Authorization Date
24-03-2026
Processing Time Days
225
Number Of Sites
1
Number Of Participants
4

Sites

Site Name
Samodzielny Publiczny Zaklad Opieki Zdrowotnej Centralny Szpital Kliniczny Uniwersytetu Medycznego W Lodzi
Department Name
Klinika Pediatrii, Onkologii i Hematologii
Principal Investigator Name
Wojciech Młynarski
Principal Investigator Email
poczta@csk.umed.pl
Contact Person Name
Wojciech Młynarski
Contact Person Email
poczta@csk.umed.pl

Greece

Earliest CTIS Part Ii Submission Date
18-09-2024
Latest Decision Or Authorization Date
13-05-2026
Processing Time Days
602
Number Of Sites
4
Number Of Participants
8

Sites

Site Name
University General Hospital Of Heraklion
Department Name
Haematology
Principal Investigator Name
Helen Papadaki
Principal Investigator Email
researchprot@pagni.gr
Contact Person Name
Helen Papadaki
Contact Person Email
researchprot@pagni.gr
Site Name
251 Air Force General Hospital
Department Name
Haematology department
Principal Investigator Name
Dimitrios Christoulas
Principal Investigator Email
pr.hafgs@haf.gr
Contact Person Name
Dimitrios Christoulas
Contact Person Email
pr.hafgs@haf.gr
Site Name
University General Hospital Of Thessaloniki Ahepa
Department Name
First Propedeutic Department of Internal Medicine
Principal Investigator Name
Georgia Kaiafa
Principal Investigator Email
ahepahos@n3.syzefxis.gov.gr
Contact Person Name
Georgia Kaiafa
Contact Person Email
ahepahos@n3.syzefxis.gov.gr
Site Name
Olympion Therapeftirio General Clinic Of Patras S.A.
Department Name
Hematology
Principal Investigator Name
Anargyros Symeonidis
Principal Investigator Email
info@olympion-sa.gr
Contact Person Name
Anargyros Symeonidis
Contact Person Email
info@olympion-sa.gr

Czechia

Earliest CTIS Part Ii Submission Date
21-05-2024
Latest Decision Or Authorization Date
09-04-2026
Processing Time Days
688
Number Of Sites
3
Number Of Participants
2

Sites

Site Name
Fakultni Nemocnice Brno
Department Name
Oddělení dětské hematologie a biochemie
Principal Investigator Name
Pavel Mazánek
Principal Investigator Email
mazanek.pavel@fnbrno.cz
Contact Person Name
Pavel Mazánek
Contact Person Email
mazanek.pavel@fnbrno.cz
Site Name
Fakultni Nemocnice V Motole
Department Name
Klinika dětské hematologie a onkologie
Principal Investigator Name
Martina Suková
Principal Investigator Email
studie@fnmotol.cz
Contact Person Name
Martina Suková
Contact Person Email
studie@fnmotol.cz
Site Name
Ustav Hematologie A krevni Transfuze
Department Name
Ambulance
Principal Investigator Name
Marie Lauermannová
Principal Investigator Email
Marie.Lauermannova@uhkt.cz
Contact Person Name
Marie Lauermannová
Contact Person Email
Marie.Lauermannova@uhkt.cz

Sponsor

Primary sponsor

Full Name
X4 Pharmaceuticals Inc.
Organisation Type
Pharmaceutical company
Country Of Registered Address
United States

Contract research organisations

Name
Novotech Clinical Research (Cyprus) Limited
Responsibilities
Project management / local CRO duties (codes include 1,12,2,5 as listed in sponsorDuties)
Name
Almac Clinical Services Limited
Responsibilities
IMP labeling and distribution
Name
Clario
Responsibilities
eCOA and adjudication committee support
Name
Primevigilance USA Inc.
Responsibilities
Pharmacovigilance
Name
Marken LLP
Responsibilities
Home healthcare services/logistics

Third parties

  • {"country":"United States","full_name":"Scout Clinical","duties_or_roles":"Patient travel concierge and reimbursement","organisation_type":"Hospital/Clinic/Other health care facility"}
  • {"country":"Argentina","full_name":"Resolution Latin America","duties_or_roles":"sponsorDuties codes: 1,12,2","organisation_type":"Industry"}
  • {"country":"United Kingdom","full_name":"Acm Global Central Laboratory Limited","duties_or_roles":"EU Central Laboratory","organisation_type":"Laboratory/Research/Testing facility"}
  • {"country":"Sweden","full_name":"TATAA Biocenter AB","duties_or_roles":"sponsorDuties code: 4","organisation_type":"Industry"}
  • {"country":"Cyprus","full_name":"Novotech Clinical Research (Cyprus) Limited","duties_or_roles":"sponsorDuties codes: 1,12,2,5","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Clario","duties_or_roles":"Electronic Clinical Outcome Assessment (eCOA); Adjudication Committee","organisation_type":"Health care"}
  • {"country":"United Kingdom","full_name":"The Doctors Laboratory Limited","duties_or_roles":"sponsorDuties code: 4","organisation_type":"Laboratory/Research/Testing facility"}
  • {"country":"United States","full_name":"Merative US LP","duties_or_roles":"sponsorDuties code: 7","organisation_type":"Health care"}
  • {"country":"United States","full_name":"Almac Clinical Technologies LLC","duties_or_roles":"sponsorDuties code: 3","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Leapcure Inc.","duties_or_roles":"Recruitment support and advertisement","organisation_type":"Industry"}
  • {"country":"Portugal","full_name":"Evidenze Portugal Unipessoal Lda.","duties_or_roles":"sponsorDuties codes: 1,2","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Immunologix","duties_or_roles":"sponsorDuties code: 4","organisation_type":"Health care"}
  • {"country":"United Kingdom (Northern Ireland)","full_name":"Almac Clinical Services Limited","duties_or_roles":"Investigational Medicinal Product labeling and distribution","organisation_type":"Pharmaceutical company"}
  • {"country":"Finland","full_name":"Blueprint Genetics Oy","duties_or_roles":"External/ Specialized laboratory NGS - Next Generation Sequencing","organisation_type":"Hospital/Clinic/Other health care facility"}
  • {"country":"United States","full_name":"Primevigilance USA Inc.","duties_or_roles":"Pharmacovigilance","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Marken LLP","duties_or_roles":"Home Healthcare Services","organisation_type":"Pharmaceutical company"}
  • {"country":"Greece","full_name":"Argyri Tsakanika","duties_or_roles":"sponsorDuties codes: 1,12","organisation_type":"SME"}
  • {"country":"United States","full_name":"Mayo Collaborative Services LLC","duties_or_roles":"sponsorDuties code: 4","organisation_type":"Laboratory/Research/Testing facility"}
  • {"country":"Italy","full_name":"Evidenze Health S.r.l.","duties_or_roles":"sponsorDuties codes: 1,2","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Charles River Laboratories Inc.","duties_or_roles":"External/ Specialized laboratory PK analysis","organisation_type":"Pharmaceutical company"}
  • {"country":"Australia","full_name":"Nexomics","duties_or_roles":"Bone marrow analysis","organisation_type":"Industry"}

Investigational products

Investigational Product Name
Mavorixafor
Active Substance
MAVORIXAFOR
Modality
Small molecule
Routes Of Administration
ORAL
Route
ORAL
Authorisation Status
Authorised product record present in CTIS product dictionary (prodAuthStatus: 1)
Maximum Dose
400 mg daily (maxDailyDoseAmount: 400)
Investigational Product Name
Mavorixafor Placebo Capsules
Modality
Other
Authorisation Status
Not applicable / placebo

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