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Belumosudil treatment for children with chronic graft-versus-host disease (schoolROCK)

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)

Phase 2 Interventional Sanofi · NCT07116031

This trial will test whether the oral drug belumosudil helps children ages 1 to under 18 with moderate-to-severe chronic graft-versus-host disease that has not improved after at least two prior systemic treatments.

Quick facts

PhasePhase 2
Study typeInterventional
Enrollment37 (estimated)
Ages1 Year to 18 Years
SexAll
SponsorSanofi Industry-sponsored
Drugs / interventionsrituximab, ibrutinib, ruxolitinib, imatinib, methotrexate
Locations29 sites (Los Angeles, California and 28 other locations)
Trial IDNCT07116031 on ClinicalTrials.gov

What this trial studies

This is an open-label, single-group Phase 1/2 study enrolling children who developed moderate-to-severe cGVHD after allogeneic transplant and who have failed at least two prior systemic therapies. Phase 1 (ages 1 to <12) determines pharmacokinetics and establishes a recommended pediatric equivalent dose, then Phase 2 (ages 1 to <18) measures safety and overall response rate at 24 weeks. At least six participants aged 1 to 6 years will be enrolled in each phase and participants undergo screening before starting oral belumosudil. The study will follow participants until treatment discontinuation and the study end is defined as three years after the last participant is recruited or all have discontinued or died.

Who should consider this trial

Good fit: Children aged 1 to under 18 who had an allogeneic hematopoietic cell transplant and have active moderate-to-severe cGVHD that is refractory to or recurred after at least two prior systemic treatments, meet weight and performance requirements, and can tolerate study procedures are ideal candidates.

Not a fit: Children with mild cGVHD, those who have received more than five prior systemic therapies, weigh under 8 kg, or have very low performance status would not be eligible and are unlikely to benefit from this protocol.

Why it matters

Potential benefit: If successful, belumosudil could provide an effective oral treatment option for children with refractory moderate-to-severe cGVHD who have limited alternatives.

How similar studies have performed: Belumosudil has demonstrated efficacy in adult cGVHD studies and is approved for that population, but pediatric data are limited and this study aims to expand pediatric dosing and safety information.

Eligibility criteria

Show full inclusion / exclusion criteria 
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) 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

Exclusion Criteria:

* 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
* Current treatment with systemic agents for cGVHD (apart from corticosteroids and calcineurin inhibitors), such as ibrutinib, ruxolitinib, sirolimus, mycophenolate (MMF), methotrexate, rituximab, imatinib, extracorporeal photopheresis (ECP) and any investigational cGVHD treatment. Prior treatment with these agents and/or therapy is allowed with a washout of at least 28 days or 5 half-lives, whichever is shorter, prior to the first dose of the IMP
* 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 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)
* 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

The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.

Where this trial is running

Los Angeles, California and 28 other locations

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions Chronic Graft Versus Host Disease
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.