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)

Description
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)

Objective
Primary
Demonstrate the superiority of belumosudil in combination with prednisone vs placebo in combination with prednisone in Event-Free Survival (EFS)

Secondary
-Demonstrate the superiority of belumosudil in combination with prednisone vs placebo in combination with prednisone in durable overall response rate (ORR), modified Lee Symptom Scale (mLSS) improvement, and in the rate of corticosteroid withdrawal
-Demonstrate the superiority of belumosudil in combination with prednisone vs placebo in combination with prednisone in durable overall response rate (ORR)
-Demonstrate the superiority of belumosudil in combination with prednisone vs placebo in combination with prednisone in the rate of corticosteroid withdrawal

Treatment This is a parallel, Phase 3, two-arm study for the treatment of newly diagnosed moderate or severe chronic GVHD.

The study duration for a participant includes up to 4 weeks for screening; a treatment period until clinically meaningful cGVHD progression (defined as progression requiring addition of new systemic treatment for cGVHD), relapse/recurrence of the underlying disease, participant starts new systemic treatment for cGVHD or experiences an unacceptable toxicity, at the request of the participants or the investigators, or until the end of study is reached, whichever comes first; at least 30 days follow-up of adverse events (AEs) after the last dose until resolution or stabilization, if applicable; and long-term follow-up until death or study close-out, whichever comes first.

Key Eligibility Inclusion Criteria:

Patients must be at least 12 years of age inclusive, at the time of signing the informed consent

Participants who have undergone allogenic HCT with newly diagnosed moderate to severe cGVHD according to NIH consensus diagnosis and staging criteria (2014)

Participants who require systemic treatment with corticosteroids for cGVHD

Participants who have not received any prior systemic treatment for cGVHD (including ECP)

If participants are receiving other immunosuppressive agents for the prophylaxis or treatment of acute GVHD, the dose should be under the threshold pre-defined in protocol

Body weight ≥ 40kg

Contraceptive use by men and women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies.

Participants or their legally authorized representative must be capable of giving signed informed consent

Applicable Disease Sites
Leukemia

Participating Institutions
UW Health 1 S. Park Medical Center; UW Health University Hospital