Phase 2 Study Comparing Axatilimab to Best Available Therapy in Children with Chronic Graft-Versus-Host Disease After Multiple Previous Treatments

What this Trial Is About

Researchers are studying pediatric participants aged 2 to under 18 years who have chronic graft-versus-host disease cGVHD that is moderate to severe and requires systemic immune suppression. The study compares the drug axatilimab to the best available therapy BAT after participants have already received at least two prior systemic treatments, including corticosteroids and ruxolitinib. This phase 2 trial aims to evaluate the response to these treatments in children and teens with this condition. Participants will be randomly assigned to receive either axatilimab at a protocol-defined dose or the best available therapy chosen by the investigator. The BAT options include specific drugs such as cyclosporine, tacrolimus, extracorporeal photopheresis, mycophenolate mofetil, mTOR inhibitors, rituximab, imatinib, methotrexate, or ibrutinib, excluding experimental agents. Both treatment groups will be monitored over time to assess responses and side effects. During the study, participants will be evaluated for objective response at 6 months as the primary outcome, with additional assessments up to 5 years including drug pharmacokinetics, overall response at 12 months, duration of response, organ-specific responses, and quality of life changes. Safety will be monitored by tracking treatment-emergent adverse events. The total study duration allows for long-term follow-up to better understand the effects and potential benefits of the treatments.

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)