← Back to searchRecruitingRecruiting
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)
NCT07124078 · Incyte Corporation
In plain English
Click the button to translate this study into plain language — what it is, who qualifies, and what participation looks like.
Official title
A Phase 2, Randomized, Open-Label Study of 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)
About this study
This study will be conducted to compare Axatilimab Versus Best Available Therapy in Pediatric Participants With Chronic Graft Versus Host Disease After at Least 2 Prior Lines of Systemic Therapy.
Eligibility criteria
Inclusion Criteria:
* Aged ≥ 2 to \< 18 years at the time of signing the informed consent.
* 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:
* Receipt of more than 1 prior allo-HCT. Prior autologous HCT is allowed.
* 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.
Other protocol-defined Inclusion/Exclusion Criteria may apply.
Study design
Enrollment target: 60 participants
Allocation: randomized
Masking: none
Age groups: child
Timeline
Starts: 2026-05-31
Estimated completion: 2029-07-31
Last updated: 2026-04-14
Interventions
Drug: INCA034176Drug: Best available Treatment (BAT)
Primary outcomes
- • Objective Response (OR) at 6 months (6 months)
Sponsor
Incyte Corporation · industry
Contacts & investigators
ContactIncyte Corporation Call Center (US) · contact · medinfo@incyte.com · 1.855.463.3463
ContactIncyte Corporation Call Center (ex-US) · contact · eumedinfo@incyte.com · +800 00027423
InvestigatorIncyte Medical Monitor · study_director, Incyte Corporation
All locations (42)
City of Hope Medical CenterRecruiting
Duarte, California, United States
Lucile Packard Child Hospital EndNot Yet Recruiting
Palo Alto, California, United States
Childrens National Medical CenterNot Yet Recruiting
Washington D.C., District of Columbia, United States
Adventhealth Pediatric Cellular Therapy and Blood and Marrow TransplantNot Yet Recruiting
Orlando, Florida, United States
Childrens Healthcare of AtlantaNot Yet Recruiting
Atlanta, Georgia, United States
Congenital Heart Center At Mott Children'S HospitalWithdrawn
Ann Arbor, Michigan, United States
Memorial Sloan Kettering Cancer CenterRecruiting
New York, New York, United States
Levine Cancer InstituteNot Yet Recruiting
Charlotte, North Carolina, United States
Childrens Hospital of PittsburghNot Yet Recruiting
Pittsburgh, Pennsylvania, United States
Fred Hutchinson Cancer Research CenterNot Yet Recruiting
Seattle, Washington, United States
Hôpital Universitaire Des Enfants Reine FabiolaNot Yet Recruiting
Brussels, Belgium
Universite Catholique de Louvain (Ucl) - Cliniques Universitaires Saint-LucNot Yet Recruiting
Woluwe-Saint-Lambert, Belgium
Charite Campus VirchowNot Yet Recruiting
Berlin, Germany
Klinikum Der Johann Wolfgang Goethe-UniversitaetRecruiting
Frankfurt am Main, Germany
Universitaetsklinikum Freiburg Zentrum Fuer Kinderheilkunde- Und Jugendmedizin Klinik Iv: PaediatrisNot Yet Recruiting
Freiburg im Breisgau, Germany
Universitaetsklinikum Hamburg-EppendorfNot Yet Recruiting
Hamburg, Germany
Medizinische Hochschule Hannover (Mhh) - Zentrum Fuer Kinderheilkunde Und Jugendmedizin - Klinik FueNot Yet Recruiting
Hanover, Germany
Universitaeres Krebszentrum Leipzig (Uccl)-Universitatsklinikum Leipzig AorWithdrawn
Leipzig, Germany
University Hospital RegensburgNot Yet Recruiting
Regensburg, Germany
Universitaetsklinikum TuebingenWithdrawn
Tübingen, Germany
Irccs - Aou Di Bologna - Sant'Orsola MalpighiNot Yet Recruiting
Bologna, Italy
Irccs Istituto G. Gaslini, Universita Di GenovaNot Yet Recruiting
Genova, Italy
Fondazione Irccs San Gerardo Dei TintoriNot Yet Recruiting
Monza, Italy
Azienda Ospedaliera Universitaria Di PadovaNot Yet Recruiting
Padua, Italy
Fondazione Irccs Policlinico San Matteo Di Pavia Oncoematologia Pediatrica, Universita Di PaviaNot Yet Recruiting
Pavia, Italy
Irccs Ospedale Pediatrico Bambino GesuRecruiting
Rome, Italy
Hospital Universitario Vall D'HebronRecruiting
Barcelona, Spain
Hospital Universitario La PazRecruiting
Madrid, Spain
Hospital Regional Universitario de Malaga- Hospital Materno InfantilRecruiting
Málaga, Spain
Hospital Universitario Virgen de La ArrixacaRecruiting
Murcia, Spain
Universitat de Valencia - Hospital Universitari I Politecnic La Fe de Valencia (Hospital La Fe BulevRecruiting
Valencia, Spain
Birmingham Children'S Hospital - Birmingham Women'S and Children'S Nhs Foundation TrustNot Yet Recruiting
Birmingham, United Kingdom
Addenbrooke'S Hospital - Cambridge University Hospitals Nhs Foundation TrustRecruiting
Cambridge, United Kingdom
The Royal Hospital For Children - Glasgow Health BoardRecruiting
Glasgow, United Kingdom
Leeds Childrens HospitalRecruiting
Leeds, United Kingdom
University College London Hospitals - University College London Hospitals Nhs Foundation TrustWithdrawn
London, United Kingdom
Royal Marsden Hospital (Sutton) - Royal Marsden Nhs Foundation TrustWithdrawn
London, United Kingdom
St Mary'S Hospital, Paddington - Imperial College Healthcare Nhs TrustRecruiting
London, United Kingdom
Great Ormond Street Hospital Nhs Foundation TrustRecruiting
London, United Kingdom
Royal Manchester Children'S Hospital - Manchester University Nhs Foundation TrustRecruiting
Manchester, United Kingdom
Royal Victoria Infirmary - the Newcastle Upon Tyne Hospitals Nhs Foundation TrustNot Yet Recruiting
Newcastle, United Kingdom
Sheffield Children'S Hospital - Sheffield Children'S Nhs Foundation TrustRecruiting
Sheffield, United Kingdom