A Study to Evaluate Axatilimab Versus Best Available Therapy in Pediatric Participants With Chronic Graft-Versus-Host Disease After at Least 2 Prior Lines of Systemic Therapy (AGAVE-256)
NCT ID: NCT07124078
Last Updated: 2025-12-31
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
PHASE2
60 participants
INTERVENTIONAL
2026-01-31
2029-07-31
Brief Summary
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Detailed Description
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Conditions
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Keywords
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
NONE
Study Groups
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Axatilimab
Axatilimab at the protocol-defined dose.
INCA034176
Axatilimab at the protocol-defined dose.
Best available Treatment (BAT)
Best Available Therapy (BAT) will be selected by the Investigator for each participant. BAT may not include experimental agents (i.e. those not approved for the treatment of any indication) as well as a limited number of other selected drugs in accordance with the protocol-defined requirements.
Best available Treatment (BAT)
Best Available Therapy (BAT) will be selected by the Investigator for each participant. BAT may not include experimental agents (i.e. those not approved for the treatment of any indication) as well as a limited number of other selected drugs in accordance with the protocol-defined requirements.
Interventions
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INCA034176
Axatilimab at the protocol-defined dose.
Best available Treatment (BAT)
Best Available Therapy (BAT) will be selected by the Investigator for each participant. BAT may not include experimental agents (i.e. those not approved for the treatment of any indication) as well as a limited number of other selected drugs in accordance with the protocol-defined requirements.
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* Active, moderate to severe cGVHD, requiring systemic immune suppression.
* Participants with refractory or recurrent cGVHD who have received at least 2 lines of systemic therapy, including corticosteroids and ruxolitinib.
* Concomitant use of systemic corticosteroids is allowed. Participants on systemic corticosteroids must be on a stable dose of corticosteroids for at least 2 weeks prior to C1D1. Topical and inhaled corticosteroid agents are allowed.
* Participants must accept to be treated with one of the following BAT options on C1D1: CNI (cyclosporine or tacrolimus), ECP, MMF, an mTOR inhibitor (everolimus or sirolimus), rituximab, imatinib, methotrexate, or ibrutinib.
* History of allo-HCT from any donor HLA type (related or unrelated donor with any degree of HLA matching) using any graft source (bone marrow, peripheral blood stem cells, or cord blood). Recipients of myeloablative, nonmyeloablative, or reduced-intensity conditioning are eligible.
Exclusion Criteria
* Evidence of relapse of hematologic disease or treatment for relapse after the allo-SCT was performed, including DLI for the treatment of molecular relapse. Note: Participants who have received a scheduled DLI as part of their transplant procedure and not for management of malignancy relapse are eligible.
* Systemic treatment with CNIs or mTOR inhibitors started within 2 weeks prior to C1D1.
* Severe renal impairment, that is, GFR \< 30 mL/min/1.73 m2 as estimated using modified Schwartz formula, or end-stage renal disease on dialysis.
* Impaired liver function, defined as total bilirubin \> 1.5 × ULN and/or ALT and AST \> 3 × ULN in participants with no evidence of liver cGVHD.
* History of acute or chronic pancreatitis.
* Active, symptomatic myositis.
* Female adolescent participants who are pregnant or breastfeeding.
2 Years
17 Years
ALL
No
Sponsors
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Incyte Corporation
INDUSTRY
Responsible Party
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Principal Investigators
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Incyte Medical Monitor
Role: STUDY_DIRECTOR
Incyte Corporation
Locations
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City of Hope Medical Center
Duarte, California, United States
Stanford University School of Medicine
Stanford, California, United States
Childrens National Medical Center
Washington D.C., District of Columbia, United States
Adventhealth Pediatric Cellular Therapy and Blood and Marrow Transplant
Orlando, Florida, United States
Emory Childrens Center
Atlanta, Georgia, United States
Memorial Sloan Kettering
New York, New York, United States
Levine Cancer Institute
Charlotte, North Carolina, United States
Childrens Hospital of Pittsburgh
Pittsburgh, Pennsylvania, United States
Fred Hutchinson Cancer Research Center
Seattle, Washington, United States
Hôpital Universitaire Des Enfants Reine Fabiola
Brussels, , Belgium
Universite Catholique de Louvain (Ucl) - Cliniques Universitaires Saint-Luc
Woluwe-Saint-Lambert, , Belgium
Charite Campus Virchow
Berlin, , Germany
Klinikum Der Johann Wolfgang Goethe-Universitaet
Frankfurt am Main, , Germany
Universitaetsklinikum Freiburg Zentrum Fuer Kinderheilkunde Und Jugendmedizin Klinik Iv
Freiburg im Breisgau, , Germany
Universitaetsklinikum Hamburg-Eppendorf
Hamburg, , Germany
Medizinische Hochschule Hannover (Mhh) - Zentrum Fuer Kinderheilkunde Und Jugendmedizin - Klinik Fue
Hanover, , Germany
Universitaeres Krebszentrum Leipzig (Uccl)-Universitatsklinikum Leipzig Aor
Leipzig, , Germany
University Hospital Regensburg
Regensburg, , Germany
Irccs - Aou Di Bologna - Sant'Orsola Malpighi
Bologna, , Italy
Irccs Istituto G. Gaslini, Universita Di Genova
Genova, , Italy
Fondazione Irccs San Gerardo Dei Tintori
Monza, , Italy
Azienda Ospedaliera Universitaria Di Padova
Padua, , Italy
Fondazione Irccs Policlinico San Matteo Di Pavia Oncoematologia Pediatrica, Universita Di Pavia
Pavia, , Italy
Irccs Ospedale Pediatrico Bambino Gesu
Rome, , Italy
Hospital Universitario Vall D'Hebron
Barcelona, , Spain
Hospital Universitario La Paz
Madrid, , Spain
Hospital Regional Universitario de Malaga- Hospital Materno Infantil
Málaga, , Spain
Hospital Universitario Virgen de La Arrixaca
Murcia, , Spain
Universitat de Valencia - Hospital Universitari I Politecnic La Fe de Valencia
Valencia, , Spain
Birmingham Children'S Hospital - Birmingham Women'S and Children'S Nhs Foundation Trust
Birmingham, , United Kingdom
Addenbrooke'S Hospital - Cambridge University Hospitals Nhs Foundation Trust
Cambridge, , United Kingdom
The Royal Hospital For Children - Glasgow Health Board
Glasgow, , United Kingdom
Leeds Childrens Hospital
Leeds, , United Kingdom
University College London Hospitals - University College London Hospitals Nhs Foundation Trust
London, , United Kingdom
St Mary'S Hospital, Paddington - Imperial College Healthcare Nhs Trust
London, , United Kingdom
Great Ormond Street Hospital For Children Nhs Foundation Trust
London, , United Kingdom
Royal Manchester Children'S Hospital - Manchester University Nhs Foundation Trust
Manchester, , United Kingdom
Royal Victoria Infirmary - the Newcastle Upon Tyne Hospitals Nhs Foundation Trust
Newcastle, , United Kingdom
Sheffield Children'S Hospital - Sheffield Children'S Nhs Foundation Trust
Sheffield, , United Kingdom
Countries
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Central Contacts
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Related Links
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A Study to Evaluate Axatilimab Versus Best Available Therapy in Pediatric Participants With Chronic Graft-Versus-Host Disease After at Least 2 Prior Lines of Systemic Therapy (AGAVE-256)
Other Identifiers
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INCA034176-256
Identifier Type: -
Identifier Source: org_study_id