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)
This is an open-label, single group, Phase 1/2, 1-arm study for treatment of children aged 1 to \<18 years with active moderate-to-severe cGVHD that is refractory to or recurred after at least 2 prior lines of systemic therapy for cGVHD. The purpose of Phase 1 is to determine the PK profiles and to establish the Recommended Pediatric Equivalent Dose (RPED) of belumosudil in participants aged 1 to \<12 years with active moderate to severe cGVHD. Upon completion and evaluation of Phase 1, Phase 2 will commence with the purpose of determining safety and efficacy (ORR by 24 weeks) of belumosudil in participants aged 1 to \<18 years. Study details include: The end of study is defined as 3 years after the last participant is recruited or all participants have discontinued treatment, or have died, whichever comes first. Minimum of 6 participants ages 1 to 6 years will be enrolled for each phase of study Individual participant duration on study will consist of: Up to 4 weeks for screening. Treatment until clinically significant progression of cGVHD, relapse/recurrence of the underlying disease, start of a new systemic treatment for cGVHD, experience of an unacceptable adverse event, request from participant or Investigator, or until the end of the study is reached, whichever comes first. 4 weeks of post treatment safety follow-up. Long-term follow-up until death or end of study, whichever occurs first.
• Participant must be 1 to \<18 years of age, at the time the consent/assent is signed. For Phase 1: participant must be 1 to \<12 years of age, at the time the consent/assent is signed. For Phase 2: participant must be 1 to \<18 years of age, at the time the consent/assent is signed.
• Participant has undergone an allogeneic HCT
• Has active moderate to severe cGVHD, defined using the NIH Consensus diagnosis and staging criteria for which systemic therapy is required
• cGVHD is refractory to or has recurred after at least 2 prior lines of systemic treatment
• Has received at least two lines of prior systemic therapy for cGVHD, but no more than 5 lines.
• If participant receives corticosteroid therapy for cGVHD, the dose must be stable for at least 2 weeks prior to the first dose of the IMP
• Has a Lansky-Play (if aged ≤16) or Karnofsky (if aged \>16) performance scale of ≥60
• Body weight of 8 kg and above
• Contraceptive use by sexually active male and female should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies
• Life expectancy of \>6 months
• Participants can take the IMP orally or via a nasogastric tube