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NCT07124078PHASE2Recruiting

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)

Incyte Corporation

Start Date

5/20/2026

Completion Date

7/31/2029

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.

Eligibility Criteria

Age Range: 2 years to 17 years

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.

Interventions

DRUG

INCA034176

DRUG

Best available Treatment (BAT)

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Conditions

Chronic Graft-versus-host-disease

Locations

City of Hope Medical Center

Duarte, California 91010

United States

Children'S Hospital Los Angeles Specialt

Los Angeles, California 90027

United States

Lucile Packard Child Hospital End

Palo Alto, California 94304

United States

Childrens National Medical Center

Washington D.C., District of Columbia 20010

United States

Adventhealth Pediatric Cellular Therapy and Blood and Marrow Transplant

Orlando, Florida 32804

United States

Childrens Healthcare of Atlanta

Atlanta, Georgia 30329

United States

Memorial Sloan Kettering Cancer Center

New York, New York 10065

United States

Levine Cancer Institute

Charlotte, North Carolina 28203

United States

Duke Cancer Center

Durham, North Carolina 27710

United States

Childrens Hospital of Pittsburgh

Pittsburgh, Pennsylvania 15224

United States

Fred Hutchinson Cancer Research Center

Seattle, Washington 98109

United States

Hôpital Universitaire Des Enfants Reine Fabiola

Brussels,

Belgium

Universite Catholique de Louvain (Ucl) - Cliniques Universitaires Saint-Luc

Woluwe-Saint-Lambert, 01200

Belgium

Charite Campus Virchow

Berlin, 13353

Germany

Klinikum Der Johann Wolfgang Goethe-Universitaet

Frankfurt am Main, 60590

Germany

Universitaetsklinikum Freiburg Zentrum Fuer Kinderheilkunde- Und Jugendmedizin Klinik Iv: Paediatris

Freiburg im Breisgau, 79106

Germany

Universitaetsklinikum Hamburg-Eppendorf

Hamburg, 20246

Germany

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

Hanover, 30625

Germany

University Hospital Regensburg

Regensburg, 93053

Germany

Irccs - Aou Di Bologna - Sant'Orsola Malpighi

Bologna, 40138

Italy

Irccs Istituto G. Gaslini, Universita Di Genova

Genova, 16147

Italy

Fondazione Irccs San Gerardo Dei Tintori

Monza, 20900

Italy

Azienda Ospedaliera Universitaria Di Padova

Padua, 35128

Italy

Fondazione Irccs Policlinico San Matteo Di Pavia Oncoematologia Pediatrica, Universita Di Pavia

Pavia, 27100

Italy

Irccs Ospedale Pediatrico Bambino Gesu

Rome, 00165

Italy

Hospital Universitario Vall D'Hebron

Barcelona, 08035

Spain

Hospital Sant Joan de Deu Barcelona - Children'S Hospital

Esplugues de Llobregat, 08950

Spain

Hospital Infantil Unversitario Nino Jesus

Madrid, 28009

Spain

Hospital Universitario La Paz

Madrid, 28046

Spain

Hospital Regional Universitario de Malaga- Hospital Materno Infantil

Málaga, 29011

Spain

Hospital Universitario Virgen de La Arrixaca

Murcia, 30120

Spain

Universitat de Valencia - Hospital Universitari I Politecnic La Fe de Valencia (Hospital La Fe Bulev

Valencia, 46026

Spain

Birmingham Children'S Hospital - Birmingham Women'S and Children'S Nhs Foundation Trust

Birmingham, B4 6NH

United Kingdom

Addenbrooke'S Hospital - Cambridge University Hospitals Nhs Foundation Trust

Cambridge, CB2 0QQ

United Kingdom

The Royal Hospital For Children - Glasgow Health Board

Glasgow, G51 4TF

United Kingdom

Leeds Childrens Hospital

Leeds, LS1 3EX

United Kingdom

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

London, W2 1NY

United Kingdom

Great Ormond Street Hospital Nhs Trust

London, WC1N 3JH

United Kingdom

Royal Manchester Children'S Hospital - Manchester University Nhs Foundation Trust

Manchester, M139WL

United Kingdom

Royal Victoria Infirmary - the Newcastle Upon Tyne Hospitals Nhs Foundation Trust

Newcastle, NE1 4LP

United Kingdom

Sheffield Children'S Hospital - Sheffield Children'S Nhs Foundation Trust

Sheffield, S10 2TH

United Kingdom