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)

Recruiting now · Phase 2

Conditions studied: Chronic Graft-versus-host-disease

In brief

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.

Key facts

Study ID

NCT07124078

Run by

Incyte Corporation

People needed

60

Starts

2026-05-20

Expected to finish

2029-07-31

Last updated by the study team

2026-06-02

Who can join

Age: 2 and older, up to 17. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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.

You may not qualify if…

• 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.

Where it is running

• Sheffield Children'S Hospital - Sheffield Children'S Nhs Foundation Trust — Sheffield, United Kingdom (enrolling)
• Fondazione Irccs Policlinico San Matteo Di Pavia Oncoematologia Pediatrica, Universita Di Pavia — Pavia, Italy (enrolling)
• Irccs Ospedale Pediatrico Bambino Gesu — Rome, Italy (enrolling)
• Hospital Universitario Vall D'Hebron — Barcelona, Spain (enrolling)
• Hospital Universitario La Paz — Madrid, Spain (enrolling)
• Hospital Regional Universitario de Malaga- Hospital Materno Infantil — Málaga, Spain (enrolling)
• Hospital Universitario Virgen de La Arrixaca — Murcia, Spain (enrolling)
• Universitat de Valencia - Hospital Universitari I Politecnic La Fe de Valencia (Hospital La Fe Bulev — Valencia, Spain (enrolling)
• Birmingham Children'S Hospital - Birmingham Women'S and Children'S Nhs Foundation Trust — Birmingham, United Kingdom (enrolling)
• Addenbrooke'S Hospital - Cambridge University Hospitals Nhs Foundation Trust — Cambridge, United Kingdom (enrolling)
• The Royal Hospital For Children - Glasgow Health Board — Glasgow, United Kingdom (enrolling)
• Leeds Childrens Hospital — Leeds, United Kingdom (enrolling)
• St Mary'S Hospital, Paddington - Imperial College Healthcare Nhs Trust — London, United Kingdom (enrolling)
• Great Ormond Street Hospital Nhs Trust — London, United Kingdom (enrolling)
• Royal Manchester Children'S Hospital - Manchester University Nhs Foundation Trust — Manchester, United Kingdom (enrolling)
• Royal Victoria Infirmary - the Newcastle Upon Tyne Hospitals Nhs Foundation Trust — Newcastle, United Kingdom (enrolling)
• City of Hope Medical Center — Duarte, California, United States (enrolling)
• Childrens National Medical Center — Washington D.C., District of Columbia, United States (enrolling)
• Memorial Sloan Kettering Cancer Center — New York, New York, United States (enrolling)
• Levine Cancer Institute — Charlotte, North Carolina, United States (enrolling)
• Hôpital Universitaire Des Enfants Reine Fabiola — Brussels, Belgium (enrolling)
• Universite Catholique de Louvain (Ucl) - Cliniques Universitaires Saint-Luc — Woluwe-Saint-Lambert, Belgium (enrolling)
• Klinikum Der Johann Wolfgang Goethe-Universitaet — Frankfurt am Main, Germany (enrolling)
• Irccs Istituto G. Gaslini, Universita Di Genova — Genova, Italy (enrolling)
• Azienda Ospedaliera Universitaria Di Padova — Padua, Italy (enrolling)

Full record on ClinicalTrials.gov

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