A Phase 2 Study of Axatilimab versus BAT in Pediatric Participants with cGVHD

• Research type

  Research Study

• Full 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)

• IRAS ID

  1012207

• Contact name

  Carol Penning

• Contact email

  cpenning@incyte.com

• Sponsor organisation

  Incyte Corporation

• Research summary

  Chronic graft-versus-host disease (cGVHD) happens when healthy transplanted (graft) “donor” stem cells see the recipient's (host) cells as foreign and start to destroy them. The usual treatment for moderate to severe cGVHD involves corticosteroids, but these don’t always work well, and the disease can return.

  This study is being done to compare the effects of a drug called axatilimab with one of the “Best Available Therapies” (BAT) options, for children and teens aged 2 to under 18 years, with moderate or severe cGVHD who have already been treated with corticosteroids and another drug called ruxolitinib.

  In this study, both the participants and their doctors will know which treatment they are getting.

  The study has three parts:
  1. Screening Period: This lasts 28 days and involves initial tests and assessments.

  2. Treatment Period: Participants will receive either axatilimab or a BAT treatment in 4-week cycles. There will be weekly visits for the first month, then visits every two weeks for the next five months. After that, visits will be every 2 or 4 weeks for those on axatilimab, or every 4 weeks for those on other treatments. Treatment continues until the participant stops the study treatment, with a final visit after the last dose.

  3. Follow-Up Period: About 30 days after the last dose, there will be a follow-up visit. Additional visits will occur over the next two years to check on cGVHD symptoms. Study staff will also contact participants every three months for up to five years (phone, email, or visits).

  The study will last up to five years for each participant and will include around 60 paediatric patients. Participants will undergo various tests and exams throughout the study as outlined in the participant information sheet.

• REC name

  London - Chelsea Research Ethics Committee

• REC reference

  25/LO/0539

• Date of REC Opinion

  12 Sep 2025

• REC opinion

  Further Information Favourable Opinion