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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) | Clinical Trials | Clareo Health

RECRUITINGPHASE2

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)

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)

NCT07124078•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.

Eligibility

Age

2 - 17 years

Sex

ALL

Healthy Volunteers

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.

Locations (38)

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

Key Dates

Start Date

March 31, 2026

Primary Completion Date

January 31, 2029

Completion Date

July 31, 2029

Last Updated

March 20, 2026

Enrollment

ESTIMATED participants

Conditions

Chronic Graft-versus-host-disease

Interventions

INCA034176

DRUG

Best available Treatment (BAT)

DRUG

Contacts

Incyte Corporation Call Center (US)

CONTACT

1.855.463.3463 medinfo@incyte.com

Incyte Corporation Call Center (ex-US)

CONTACT

+800 00027423 eumedinfo@incyte.com

Efficacy and Safety of KD025 in Subjects With cGVHD After At Least 2 Prior Lines of Systemic Therapy

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COMPLETEDPHASE1

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Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: March 20, 2026Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

View ClinicalTrials.gov Terms and Conditions

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)

Inclusion Criteria

Exclusion Criteria

Efficacy and Safety of KD025 in Subjects With cGVHD After At Least 2 Prior Lines of Systemic Therapy

Study of Ruxolitinib for Acute and Chronic Graft Versus Host Disease

Ibrutinib Plus Rituximab for cGVHD Following Allo-SCT