A Study of Belumosudil in Children With Chronic Graft Versus Host Disease (schoolROCK) (NCT07116031) | Clinical Trial Compass
RecruitingPhase 2
A Study of Belumosudil in Children With Chronic Graft Versus Host Disease (schoolROCK)
United States, Belgium, Canada37 participantsStarted 2025-12-02
Plain-language summary
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.
Who can participate
Age range
1 Year – 18 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion Criteria:
* 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 years) or Karnofsky (if aged ≥16 years) 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
* The participant or their legally authorized representative (LAR) must be capable of giving signed informed consent
* Life expectancy of \>6 months
* Participants can take the IMP orally or via a nasogastric tube
Exclusion Criteria:
* Progressive underlying disease or post-transplant lymphoproliferative disease within 4 weeks prior to the first dose of the IM…
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Phase 1: AUC
Timeframe: Cycle 1 Day 15 after the last participant dosed in the phase 1 part.
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
Timeframe: Last participant completing 24 weeks (Week 25 visit or Cycle 7 Day 1 visit, whichever comes first) in the study
Trial details
NCT IDNCT07116031
SponsorSanofi
Sponsor typeINDUSTRY
Study typeINTERVENTIONAL
Primary completion2028-09-25
Contact for this trial
Trial Transparency email recommended (Toll free for US & Canada)