An open-label, Phase 1/2, multicenter study of belumosudil in children aged 1 to <18 years requiring systemic treatment for active moderate-to-severe chronic graft versus host disease (cGVHD).

Condition studied

Chronic graft versus host disease.

Investigational medicinal product(s)

Eligibility

Primary endpoint

Phase 1: AUC, Phase 2: Proportion of participants who achieve an overall response (partial response [PR] or complete response [CR]) by Week 25 or Cycle 7 Day 1 whichever is first

Endpoint detail

Phase 1: Number of participants with treatment- emergent adverse events [TEAEs], serious TEAEs, and adverse events of special interest (AESIs), Phase 1: Cmax, Phase 1: AUC0-6h, Phase 1: ORR, Phase 1: DOR, Phase 1: response by organ, Phase 1: failure-free survival (FFS), Phase 1: overall survival (OS), Phase 1: time to response (TTR), Phase 2: Number of participants with treatment- emergent adverse events [TEAEs], serious TEAEs, and adverse events of special interest (AESIs), Phase 2: Ctrough of belumosudil, Phase 2: DOR, Phase 2: response by organ, Phase 2: FFS, Phase 2: OS, Phase 2: time to response (TTR), Phase 1: time to next treatment (TTNT), Phase 2: time to next treatment (TTNT)