EFC17757 GvHD_ROCKnrol-1
Research type
Research Study
Full title
A randomized, double-blind, multicenter, Phase 3 study to evaluate efficacy and safety of belumosudil in combination with corticosteroids versus placebo in combination with corticosteroids in participants at least 12 years of age with newly diagnosed chronic graft versus host disease (cGVHD)
IRAS ID
1008347
Contact name
Patrick Maury
Contact email
Sponsor organisation
Sanofi-Aventis Recherche & Développement
Eudract number
2023-505394-32
Research summary
This study will consider how effective and safe belumosudil (study drug) with prednisone or prednisolone (a steroid), is compared to a placebo (a similar-looking tablet having no medicine) with prednisone or prednisolone to treat patients at least 12 years old newly diagnosed with chronic graft versus host disease (cGVHD).
Belumosudil is a study drug developed by Sanofi (sponsor of the study) as a possible treatment in patients with newly diagnosed cGVHD. The study drug weakens immune reactions caused by donor cells (bone marrow cells from a healthy person, injected into a sick person) which may play a role in the progression of cGVHD. This stops the immune system from causing inflammation and scarring in normal tissues.
This study is a randomized, double-blind (ie, neither the people taking part nor the study doctors know who is given the study medicine or the placebo), parallel, 2-arm study where each person participates for up to 3 years from the date of last participant in.
Treatment is randomly chosen for each participant ie, selected by chance using a computer program. Participants take prednisone with 200-mg tablets of belumosudil or placebo once daily by mouth until treatment failure or the end of study. The starting dose of prednisone is 1 mg/kg/day and may be gradually reduced if response to treatment is good.
The benefits of belumosudil in cGVHD are being tested. Belumosudil has been approved for cGVHD in several countries for use in adult and pediatric patients 12 years and older with at least 2 prior therapies. Belumosudil was well tolerated by participants in numerous clinical studies. Participants may be at risk for infections and disorders due to belumosudil’s effect on the immune system. Participants may experience temporary spikes in liver enzymes which are usually resolved after proper management.REC name
South Central - Berkshire Research Ethics Committee
REC reference
23/SC/0290
Date of REC Opinion
25 Oct 2023
REC opinion
Further Information Favourable Opinion