Clinical trial • Phase II • Immunology

AXATILIMAB for Chronic graft-versus-host disease

Phase II trial of AXATILIMAB for Chronic graft-versus-host disease. Randomised, open-label. 50 participants.

Overview

Trial Therapeutic Area
Immunology
Trial Disease
Chronic graft-versus-host disease
Trial Stage
Phase II
Drug Modality
Monoclonal antibody|Small molecule
Paediatric Trial
Yes

Key dates

Initial CTIS Submission Date
17-05-2024
First CTIS Authorization Date
09-09-2024

Trial design

Randomised, open-label Phase II trial in Spain, Germany, Belgium and others.

Randomised
Yes
Open Label
Yes
Target Sample Size
50

Eligibility

Recruits 50 paediatric patients.

Pregnancy Exclusion
Pregnant or breastfeeding
Vulnerable Population
Minors aged 12 to <18 years are eligible (minimum age 12); note participants aged 12 to <18 will not be enrolled in Germany, Italy, and the UK. Parental/guardian informed consent and age-appropriate assent/consent documents are provided (Parental ICF, Assent, Consent 12-17 and related ICF documents available in submission).

Inclusion criteria

  • {"criterion_text":"- 1.\tAt least 12 years of age at the time of informed consent. Note: Participants aged 12 to ˂ 18 years will not be enrolled in Germany, Italy, and the UK.\n- New-onset moderate or severe cGVHD, as defined by the 2014 NIH Consensus Criteria\n- History of 1 allo-SCT\n- Willingness to avoid pregnancy or fathering children"}

Exclusion criteria

  • {"criterion_text":"- Participants who received more than 1 prior allo-SCT. Prior autologous HCT is allowed.\n- 2.\tHas overlap cGVHD, defined as the presence of features or characteristics of aGVHD with simultaneous diagnostic and/or distinctive features of cGVHD.\n- Prior systemic treatment for cGVHD, including systemic corticosteroids and extracorporeal photopheresis.\n- Received systemic corticosteroids within 2 weeks prior to C1D1, regardless of indication, except for physiological replacement doses of corticosteroids (ie, < 10 mg/day prednisone equivalent) for adrenal insufficiency.\n- Initiated systemic treatment with CNIs or mTOR inhibitors within 2 weeks prior to C1D1.\n- Prior treatment with a JAK inhibitor within 8 weeks before randomization\n- Pregnant or breastfeeding\n- Known allergies, hypersensitivity, or intolerance to any of the study medications, excipients, or similar compounds."}

Endpoints

Primary endpoints

  • {"endpoint_text":"- Overall response (OR) at 6 months, defined for each treatment group as CR or PR at 6 months in the absence of new systemic therapy for cGVHD. Response assessment will be based on the 2014 NIH Criteria","definition_or_measurement_approach":"Response assessment will be based on the 2014 NIH Criteria; OR at 6 months defined as CR or PR at 6 months in the absence of new systemic therapy for cGVHD."}

Secondary endpoints

  • {"endpoint_text":"- Safety and tolerability will be assessed by evaluating the frequency and severity of AEs (including SAEs) and changes in clinical and laboratory assessments.","definition_or_measurement_approach":"Assessment by frequency and severity of adverse events (including SAEs) and changes in clinical and laboratory assessments."}
  • {"endpoint_text":"- DOR (in responders only)","definition_or_measurement_approach":""}
  • {"endpoint_text":"- Proportion of participants with ≥ 7-point improvement in mLSS total score.","definition_or_measurement_approach":"Proportion of participants achieving at least a 7-point improvement in modified Lee Symptom Scale (mLSS) total score."}
  • {"endpoint_text":"- Best overall response in the first 6 months and on study, based on the 2014 NIH Consensus Criteria","definition_or_measurement_approach":"Assessment of best overall response using 2014 NIH Consensus Criteria within first 6 months and throughout study."}
  • {"endpoint_text":"- OR at 12 months, defined as CR or PR at 12 months","definition_or_measurement_approach":"Overall response at 12 months defined as CR or PR at 12 months."}
  • {"endpoint_text":"- PK parameters for axatilimab and ruxolitinib","definition_or_measurement_approach":"Pharmacokinetic parameter assessment for axatilimab and ruxolitinib (PK sampling and analysis as per protocol)."}
  • {"endpoint_text":"- \tEFS, defined as the time from the date of randomization to the date of addition or initiation of new systemic therapy for cGVHD, death due to any cause, or Day 1 in the case of treatment failure (ie, non-CR or non-PR by Month 6), whichever occurs first.","definition_or_measurement_approach":"Event-free survival (EFS) measured as time from randomization to addition/initiation of new systemic therapy for cGVHD, death from any cause, or Day 1 for treatment failure (non-CR/non-PR by Month 6)."}

Recruitment

Digital Remote Recruitment
Yes
Planned Sample Size
70
Recruitment Window Months
64
Consent Approach
Adults provide informed consent using the Main ICF. For minors (age 12-17) parental/guardian consent plus assent is used (Consent 12-17, Parental ICF, Assent documents present). ICFs and assent materials are provided in multiple languages (English, French, Dutch, Spanish, German, Italian as per submitted patient-facing documents).

Methods

  • Email communications via Scout (Scout Email Communication documents listed in submission)
  • Reloadable ScoutPass mailer (Reloadable ScoutPass Mailer/Brochure documents)
  • Printed brochures and mailers in multiple languages (Scout Brochure, Reloadable ScoutPass Brochure files in ENG/FR/NL/DE/IT/ES variants)
  • General Practitioner letters to support recruitment (General Practitioner Letter documents)

Geography

Total Number Of Sites
34
Total Number Of Participants
70

Spain

Earliest CTIS Part Ii Submission Date
25-06-2024
Latest Decision Or Authorization Date
28-04-2025
Processing Time Days
307
Number Of Sites
13
Number Of Participants
26

Sites

Site Name
University Clinical Hospital Virgen De La Arrixaca
Department Name
Hematology
Principal Investigator Name
Andres Sanchez
Principal Investigator Email
asanchez2005@gmail.com
Contact Person Name
Andres Sanchez
Contact Person Email
asanchez2005@gmail.com
Site Name
University Hospital Virgen Del Rocio S.L.
Department Name
Hematology
Principal Investigator Name
Jose Antonio Perez
Principal Investigator Email
josea.perez.simon.sspa@juntadeandalucia.es
Contact Person Name
Jose Antonio Perez
Site Name
Hospital Universitario 12 De Octubre
Department Name
Hematology
Principal Investigator Name
Maria Calbacho
Principal Investigator Email
mcalbachorobles@gmail.com
Contact Person Name
Maria Calbacho
Contact Person Email
mcalbachorobles@gmail.com
Site Name
Hospital Universitario Marques De Valdecilla
Department Name
Hematology
Principal Investigator Name
Arancha Bermudez
Principal Investigator Email
maranzazu.bermudez@scsalud.es
Contact Person Name
Arancha Bermudez
Contact Person Email
maranzazu.bermudez@scsalud.es
Site Name
Hospital Clinico Universitario De Valencia
Department Name
Hematology
Principal Investigator Name
Jose Luis Piñana
Principal Investigator Email
jlpinana@gmail.com
Contact Person Name
Jose Luis Piñana
Contact Person Email
jlpinana@gmail.com
Site Name
Hospital Clinic De Barcelona
Department Name
Hematology
Principal Investigator Name
Carmen Martinez
Principal Investigator Email
cmarti@clinic.cat
Contact Person Name
Carmen Martinez
Contact Person Email
cmarti@clinic.cat
Site Name
Hospital Universitario Virgen De Las Nieves
Department Name
Hematology
Principal Investigator Name
Manuel Jurado
Principal Investigator Email
manuel.jurado.sspa@juntadeandalucia.es
Contact Person Name
Manuel Jurado
Site Name
Hospital Universitario Regional De Malaga
Department Name
Hematology
Principal Investigator Name
Maria Jesus Pascual
Principal Investigator Email
mjcascon@gmail.com
Contact Person Name
Maria Jesus Pascual
Contact Person Email
mjcascon@gmail.com
Site Name
Institut Catala D'oncologia
Department Name
Hematology
Principal Investigator Name
Alberto Mussetti
Principal Investigator Email
amussetti@iconcologia.net
Contact Person Name
Alberto Mussetti
Contact Person Email
amussetti@iconcologia.net
Site Name
Hospital General Universitario Gregorio Maranon
Department Name
Hematology
Principal Investigator Name
Mi Kwon
Principal Investigator Email
mi.kwon@salud.madrid.org
Contact Person Name
Mi Kwon
Contact Person Email
mi.kwon@salud.madrid.org
Site Name
Hospital Universitario Y Politecnico La Fe
Department Name
Hematology
Principal Investigator Name
Jaime Sanz
Principal Investigator Email
jaime.sanz@uv.es
Contact Person Name
Jaime Sanz
Contact Person Email
jaime.sanz@uv.es
Site Name
Hospital Universitario Ramon Y Cajal
Department Name
Hematology
Principal Investigator Name
Anabelle Chinea
Principal Investigator Email
anabellechip@hotmail.com
Contact Person Name
Anabelle Chinea
Contact Person Email
anabellechip@hotmail.com
Site Name
Hospital Universitario De Salamanca
Department Name
Hematology
Principal Investigator Name
Lucia Lopez
Principal Investigator Email
lucialopezcorral@usal.es
Contact Person Name
Lucia Lopez
Contact Person Email
lucialopezcorral@usal.es

Germany

Earliest CTIS Part Ii Submission Date
23-08-2024
Latest Decision Or Authorization Date
23-04-2025
Processing Time Days
243
Number Of Sites
6
Number Of Participants
14

Sites

Site Name
Universitaetsklinikum Regensburg AöR
Department Name
Internal Medicine III, Hematology and Oncology
Principal Investigator Name
Daniel Wolff
Principal Investigator Email
daniel.wolff@klinik.uni-regensburg.de
Contact Person Name
Daniel Wolff
Site Name
Medical Center - University Of Freiburg
Department Name
Internal Medicine I, Hematology, Oncology and Stem Cell Transplantation
Principal Investigator Name
Robert Zeiser
Principal Investigator Email
robert.zeiser@uniklinik-freiburg.de
Contact Person Name
Robert Zeiser
Site Name
Goethe University Frankfurt
Department Name
Hematology/Oncology
Principal Investigator Name
Gesine Bug
Principal Investigator Email
studienservice@med.uni-frankfurt.de
Contact Person Name
Gesine Bug
Site Name
Universitaetsklinikum Mannheim GmbH
Department Name
Hematology and Oncology
Principal Investigator Name
Sebastian Kreil
Principal Investigator Email
sebastian.kreil@medma.uni-heidelberg.de
Contact Person Name
Sebastian Kreil
Site Name
Institut fuer Klinische Transfusionsmedizin Jena gGmbH
Department Name
Hematology and Internal Oncology
Principal Investigator Name
Inken Hilgendorf
Principal Investigator Email
inken.hilgendorf@med.uni-jena.de
Contact Person Name
Inken Hilgendorf
Site Name
University Medical Center Hamburg-Eppendorf
Department Name
Stem Cell Transplantation, Center of Oncology
Principal Investigator Name
Silke Heidenreich
Principal Investigator Email
s.heidenreich@uke.de
Contact Person Name
Silke Heidenreich
Contact Person Email
s.heidenreich@uke.de

Belgium

Earliest CTIS Part Ii Submission Date
09-08-2024
Latest Decision Or Authorization Date
22-04-2025
Processing Time Days
256
Number Of Sites
7
Number Of Participants
14

Sites

Site Name
Centre hospitalier universitaire de Liege
Department Name
Hematology
Principal Investigator Name
Frédéric Baron
Principal Investigator Email
f.baron@uliege.be
Contact Person Name
Frédéric Baron
Contact Person Email
f.baron@uliege.be
Site Name
UZ Leuven
Department Name
Hematology
Principal Investigator Name
Hélène Schoemans
Principal Investigator Email
helene.schoemans@uzleuven.be
Contact Person Name
Hélène Schoemans
Contact Person Email
helene.schoemans@uzleuven.be
Site Name
Jessa Ziekenhuis
Department Name
Hematology
Principal Investigator Name
Koen Theunissen
Principal Investigator Email
koen.theunissen@jessazh.be
Contact Person Name
Koen Theunissen
Contact Person Email
koen.theunissen@jessazh.be
Site Name
Antwerp University Hospital
Department Name
Haematology, Heamostatis and Stem Cell Therapy
Principal Investigator Name
Anke Verlinden
Principal Investigator Email
anke.verlinden@uza.be
Contact Person Name
Anke Verlinden
Contact Person Email
anke.verlinden@uza.be
Site Name
Algemeen Ziekenhuis Delta
Department Name
Hematology
Principal Investigator Name
Dries Deeren
Principal Investigator Email
dries.deeren@azdelta.be
Contact Person Name
Dries Deeren
Contact Person Email
dries.deeren@azdelta.be
Site Name
Az St-Jan Brugge-Oostende A.V.
Department Name
Hematology
Principal Investigator Name
Alexander Schauwvlieghe
Principal Investigator Email
alexander.schauwvlieghe@azsintjan.be
Contact Person Name
Alexander Schauwvlieghe
Site Name
UZ Leuven (Paediatric Haematology-Oncology and Stem Cell Transplantation)
Department Name
Paediatric Haematology-Oncology and Stem Cell Transplantation
Principal Investigator Name
Heidi Segers
Principal Investigator Email
heidi.segers@uzleuven.be
Contact Person Name
Heidi Segers
Contact Person Email
heidi.segers@uzleuven.be

Italy

Earliest CTIS Part Ii Submission Date
05-08-2024
Latest Decision Or Authorization Date
23-01-2026
Processing Time Days
536
Number Of Sites
8
Number Of Participants
16

Sites

Site Name
Azienda Ospedaliera Universitaria Citta Della Salute E Della Scienza Di Torino
Department Name
Departmental of Hematology, Internal Medicine
Principal Investigator Name
Luisa Giaccone
Principal Investigator Email
luisa.giaccone@unito.it
Contact Person Name
Luisa Giaccone
Contact Person Email
luisa.giaccone@unito.it
Site Name
Azienda Socio Sanitaria Territoriale Papa Giovanni XXIII
Department Name
Departmental of Oncology and Hematology
Principal Investigator Name
Alessandra Algarotti
Principal Investigator Email
aalgarotti@asst-pg23.it
Contact Person Name
Alessandra Algarotti
Contact Person Email
aalgarotti@asst-pg23.it
Site Name
Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico
Department Name
Medical Area Department - S.C. HEMATOLOGY
Principal Investigator Name
Giorgia Saporiti
Principal Investigator Email
giorgia.saporiti@policlinico.mi.it
Contact Person Name
Giorgia Saporiti
Site Name
Humanitas Mirasole S.p.A.
Department Name
Departmental of Oncology and Hematology
Principal Investigator Name
Stefania Bramanti
Principal Investigator Email
stefania.bramanti@humanitas.it
Contact Person Name
Stefania Bramanti
Contact Person Email
stefania.bramanti@humanitas.it
Site Name
Azienda Ospedaliero-Universitaria Di Bologna IRCCS Istituto Di Ricerca E Di Cura A Carattere Scientifico
Department Name
Departmental of Hematology / Advanced Cellular Therapies
Principal Investigator Name
Francesca Bonifazi
Principal Investigator Email
francesca.bonifazi@unibo.it
Contact Person Name
Francesca Bonifazi
Contact Person Email
francesca.bonifazi@unibo.it
Site Name
Azienda Ospedaliera Universitaria Citta Della Salute E Della Scienza Di Torino (Piazza Polonia)
Department Name
Dep of Pathology and Child Care and Pediatric Oncohematology-Regina Margherita Children's Hospital
Principal Investigator Name
Franca Fagioli
Principal Investigator Email
franca.fagioli@unito.it
Contact Person Name
Franca Fagioli
Contact Person Email
franca.fagioli@unito.it
Site Name
Ospedale Pediatrico Bambino Gesu
Department Name
Departmental of Oncohaematology and Cell Therapy Studies
Principal Investigator Name
Franco Locatelli
Principal Investigator Email
franco.locatelli@opbg.net
Contact Person Name
Franco Locatelli
Contact Person Email
franco.locatelli@opbg.net
Site Name
Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Department Name
Departmental of Hematology
Principal Investigator Name
Simona Sica
Principal Investigator Email
simona.sica@unicatt.it
Contact Person Name
Simona Sica
Contact Person Email
simona.sica@unicatt.it

Sponsor

Primary sponsor

Full Name
Incyte Corp.
Organisation Type
Pharmaceutical company
Country Of Registered Address
United States

Contract research organisations

Name
Syneos Health Inc.
Responsibilities
SDTM conversion
Name
Iqvia Biotech LLC
Responsibilities
DMC management
Name
Suvoda LLC

Third parties

  • {"country":"United States","full_name":"Q Squared Solutions Holdings LLC","duties_or_roles":"Central lab","organisation_type":"Laboratory/Research/Testing facility"}
  • {"country":"United States","full_name":"Syneos Health Inc.","duties_or_roles":"SDTM conversion","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Iqvia Biotech LLC","duties_or_roles":"DMC management","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Suvoda LLC","duties_or_roles":"","organisation_type":"Non-Pharmaceutical company"}
  • {"country":"Belgium","full_name":"CellCarta Biosciences","duties_or_roles":"Central lab for WB PBMC","organisation_type":"Pharmaceutical company"}

Investigational products

Investigational Product Name
Axatilimab (INCA034176)
Active Substance
AXATILIMAB
Modality
Monoclonal antibody
Routes Of Administration
INTRAVENOUS (IV) OR SUBCUTANEOUS (SC)
Route
INTRAVENOUS (IV) OR SUBCUTANEOUS (SC)
Authorisation Status
1
Maximum Dose
0.3 mg/kg
Investigational Product Name
Jakavi 5 mg tablets
Active Substance
RUXOLITINIB
Modality
Small molecule
Routes Of Administration
ORAL
Route
ORAL
Authorisation Status
2
Maximum Dose
20 mg
Investigational Product Name
PREDNISONE
Active Substance
PREDNISONE
Modality
Small molecule
Routes Of Administration
ORAL
Route
ORAL
Authorisation Status
2
Maximum Dose
100 mg
Investigational Product Name
METHYLPREDNISOLONE
Active Substance
METHYLPREDNISOLONE
Modality
Small molecule
Routes Of Administration
ORAL AND IV
Route
ORAL AND IV
Authorisation Status
2
Maximum Dose
100 mg
Combination Treatment
Yes

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