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)

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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)

Status
Active
Cancer Type
Unknown Primary
Trial Phase
Phase II
Eligibility
2 - 17 Years, Male and Female
Study Type
Treatment
NCT ID
NCT07124078
Protocol IDs
INCA034176-256 (primary)
NCI-2025-07537
Study Sponsor
Incyte Corporation

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

  1. Aged 2 to < 18 years at the time of randomization.
  2. Active, moderate to severe cGVHD, requiring systemic immune suppression.
  3. Participants with refractory or recurrent cGVHD who have received at least 2 lines of systemic therapy, including corticosteroids and ruxolitinib.
  4. 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.
  5. 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, ibrutinib, or pentostatin.
  6. 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.

Treatment Sites in Georgia

Winship Cancer Institute of Emory University


1365 Clifton Road NE
Building C
Atlanta, GA 30322
winshipcancer.emory.edu

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