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RECRUITING

NCT07124078

PHASE2

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)

Sponsor: Incyte Corporation

View on ClinicalTrials.gov

Summary

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.

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)

Key Details

Gender

All

Age Range

2 Years - 17 Years

Study Type

INTERVENTIONAL

Enrollment

Start Date

2026-05-31

Completion Date

2029-07-31

Last Updated

2026-04-09

Healthy Volunteers

Conditions

Chronic Graft-versus-host-disease

Interventions

DRUG

INCA034176

Axatilimab at the protocol-defined dose.

DRUG

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.

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.

Locations (37)

City of Hope Medical Center

Duarte, California, United States

Lucile Packard Child Hospital End

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

Childrens Healthcare of Atlanta

Atlanta, Georgia, United States

Memorial Sloan Kettering Cancer Center

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: Paediatris

Freiburg im Breisgau, Germany

Universitaetsklinikum Hamburg-Eppendorf

Hamburg, Germany

Medizinische Hochschule Hannover (Mhh) - Zentrum Fuer Kinderheilkunde Und Jugendmedizin - Klinik Fue

Hanover, 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 (Hospital La Fe Bulev

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

St Mary'S Hospital, Paddington - Imperial College Healthcare Nhs Trust

London, United Kingdom

Great Ormond Street Hospital 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