A Study of Belumosudil in Children With Chronic Graft Versus Host Disease (schoolROCK)
ConditionChronic Graft Versus Host Disease
Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age1-18
SponsorSanofi
About this trial
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.
30 days of post treatment safety follow-up. Long-term follow-up until death or end of study, whichever occurs first.
Eligibility criteria
Qualifiers
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
Disqualifiers
Progressive underlying disease or post-transplant lymphoproliferative disease within 4 weeks prior to the first dose of the IMP.
Diagnosed with another malignancy (other than malignancy for which transplant was performed) within 3 years prior to the first dose of the IMP
History or other evidence of severe illness or any other conditions that would make the participant, in the opinion of the Investigator, unsuitable for the study (such as malabsorption syndromes, active, uncontrolled infections, or poorly controlled psychiatric disease)
Has a forced expiratory volume (in the first second; FEV1) ≤39% or has lung score of 3
Trial design
Treatments tested in this trial
- Belumosudil
- Belumosudil
Treatment groups
37 Participants
are divided into 1 treatment groupSponsors and collaborators
Sanofi
Lead sponsor
Meiji Seika Pharma Co., Ltd.
Collaborator
Source ClinicalTrials.gov