A Phase 3, Open-Label Study of Axatilimab Versus Best Available Therapy in cGVHD

• Research type

  Research Study

• Full title

  A Phase 3, Randomized, Open-Label Study of Axatilimab Versus Best Available Therapy in Participants with Chronic Graft-Versus-Host Disease After at Least 2 Prior Lines of Systemic Therapy

• IRAS ID

  1011188

• Contact name

  Carol Penning

• Contact email

  cpenning@incyte.com

• Sponsor organisation

  Incyte Corporation

• Research summary

  Chronic graft-versus-host disease (cGVHD) is a complication that can occur after an allogeneic (donor) stem cell transplant. The usual treatment for moderate to severe cGVHD involves corticosteroids, but these don’t work well enough and the disease can come back.
  This study is looking at a new drug called axatilimab and comparing it to “Best Available Therapies” (BAT) for individuals with moderate or severe cGVHD who have already been treated with corticosteroids and another drug called ruxolitinib. In this study, both the participants and the doctors will know which treatment is being given.

  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 another 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 he 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 tears (phone, email, or visits).

  The study will last up to five years for each participant and will include around 300 people. Participants will undergo various tests and exams throughout the study as outlined in the participant information sheet.

• REC name

  South Central - Hampshire B Research Ethics Committee

• REC reference

  25/SC/0024

• Date of REC Opinion

  21 Feb 2025

• REC opinion

  Further Information Favourable Opinion