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Belumosudil for Children with Chronic Graft Versus Host Disease

This study, called schoolROCK, is testing a drug called belumosudil in children aged 1 to under 18 years old who have chronic graft versus host disease (cGVHD). This is a condition where donated cells attack the patient's body after a transplant. Participants must have moderate-to-severe cGVHD that hasn't responded to at least two other treatments. The study aims to find the right dose of belumosudil for younger children (Phase 1) and then to see how safe and effective it is in a wider age range (Phase 2). Success will be measured by how many participants show an overall improvement in their cGVHD by 24 weeks.

Study design
This is an open-label, single-group study with one treatment arm, meaning all participants receive belumosudil. It is a Phase 1/2 study and plans to enroll 37 participants.
What's involved
Not specified in the trial record.
Compensation
Not stated in the trial record.
Follow-up
The primary endpoint for response is measured at 24 weeks (Week 25 visit or Cycle 7 Day 1 visit, whichever comes first).

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.

NCT07116031

A Study of Belumosudil in Children With Chronic Graft Versus Host Disease (schoolROCK)

Recruiting
PHASE2Ages 1–18InterventionalTreatment
Sanofi
~37 participants
Updated 2026-08-24 on ClinicalTrials.gov
What's tested:Belumosudil

At a glance

Recruiting sites
33 of 33 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Phase 1: AUC
Measured over Cycle 1 Day 15 after the last participant dosed in the phase 1 part.
+1 more outcome measured

Conditions

Chronic Graft Versus Host Disease

Explore related trials

Chronic Graft Versus Host Disease

NCT07116031

Where you'd take part

This study runs at 33 sites. They're the same protocol — you choose where, and that choice sets who your contact draft is addressed to.

  • Children's Hospital Los Angeles- Site Number : 8400009

    Los Angeles, Californiano site contact published

    Recruiting

  • Children's National Medical Center - Washington- Site Number : 8400005

    Washington D.C., District of Columbiano site contact published

    Recruiting

  • Fred Hutchinson Cancer Research Center- Site Number : 8400002

    Seattle, Washingtonno site contact published

    Recruiting

  • Investigational Site Number : 0560001

    Leuven, Belgiumno site contact published

    Recruiting

  • Investigational Site Number : 0560003

    Ghent, Belgiumno site contact published

    Recruiting

  • Investigational Site Number : 1240001

    Vancouver, British Columbia, Canadano site contact published

    Recruiting

  • Investigational Site Number : 1240002

    Toronto, Ontario, Canadano site contact published

    Recruiting

  • Investigational Site Number : 1560001

    Shanghai, Chinano site contact published

    Recruiting

Sites open and close at different times, so the status above is per site — it can differ from the study's overall status.

Who to contact

Trial Transparency email recommended (Toll free for US & Canada)
[email protected]
800-633-1610 · ext. option 6
Email the study team

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Eligibility criteria

Inclusion

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

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
Female participants who are pregnant or breastfeeding
Participants who meet any of the following criteria regarding systemic GVHD treatments:
Participants who newly initiated any systemic GVHD treatment within 14 days prior to the first dose of belumosudil.
Participants receiving systemic GVHD treatments ibrutinib, ruxolitinib, mycophenolate (MMF), methotrexate, rituximab, axatilimab, or imatinib who are unable to meet the following requirements:
No dose increases from 14 days prior to belumosudil initiation and continuing for the first 14 days of belumosudil treatment (dose reductions and discontinuations are permitted during this period)
Ability to discontinue these therapies within 14 days after initiating belumosudil (allowing for a maximum overlap period of up to 14 days with belumosudil treatment)
Participants receiving other systemic GVHD treatments (apart from corticosteroids and calcineurin inhibitors) including investigational treatments who have not completed a washout period of at least 28 days or 5 half-lives (whichever is shorter) prior to the first dose of belumosudil. No washout period is required for extracorporeal photopheresis (ECP) or sirolimus therapy, but these must be discontinued before study treatment initiation.
The use of herbal and recreational drugs within 7 days before the start of study intervention
Participant has had previous exposure to belumosudil
Administration of live or live-attenuated vaccines is prohibited within 28 days or 5 elimination half-lives of the respective vaccine, whichever is longer, prior to IMP administration and until study intervention discontinuation
Treatment with any non-GVHD investigational agent, or any investigational device or procedure, within 28 days (or 5 half-lives, whichever is longer) of enrollment, prior to the first dose of the IMP
For Phase 1 only: Administration with strong CYP3A4 inducers is not allowed within 14 days or 5 half-lives (whichever is longer) of the first dose of IMP until the study intervention discontinuation.
For Phase 1 only: PPIs are not allowed within 1 day or 5 half-lives (whichever is longer) of the first dose of IMP and Day 15 of Cycle 1. They can be restarted on Cycle 1 Day 16.
Absolute neutrophil count \<1.0 × 109/L. The use of granulocyte-colony stimulating factor (G-CSF) is not allowed within 7 days prior to the ANC test to reach this level during screening
Platelet count \<25 × 109/L. Platelet transfusions are not allowed within 72 hours before hematology screening test. Participants with platelet transfusion refractoriness will be excluded. (Participants who have suboptimal responses to at least 2 transfusions will be considered as platelet transfusion refractory)
Alanine aminotransferase (ALT) and/or aspartate aminotransferase (AST) \>3× upper limit of normal (ULN) (\> 5x ULN if abnormalities are due to cGVHD)
Total bilirubin \>1.5 × ULN (\>3 x ULN if Gilbert's syndrome or if abnormalities are due to cGVHD)
Glomerular filtration rate (GFR) \<30 mL/min/1.73 m2 using the revised Bedside Schwartz calculator
Participants with an active viral disease including hepatitis B virus (HBV) and hepatitis C virus (HCV)
Active uncontrolled Cytomegalovirus (CMV) or Epstein-Barr virus (EBV) infection
Known history of human immunodeficiency virus (HIV)
Not suitable for participation, whatever the reason, as judged by the Investigator, including medical or clinical conditions, or participants potentially at risk of noncompliance to study procedures

What this trial measures

  • Phase 1: AUCCycle 1 Day 15 after the last participant dosed in the phase 1 part.

    PK parameter (AUC at steady state)

  • 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 firstLast participant completing 24 weeks (Week 25 visit or Cycle 7 Day 1 visit, whichever comes first) in the study

    Proportion of participants who achieve an overall response (partial response \[PR\] or complete response \[CR\]) with up to 24 weeks of therapy (i.e. by the Week 25 or Cycle 7 Day 1 visit whichever is first), as defined by the National Institute of Health (NIH) Consensus response criteria