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A study of belumosudil in children with chronic graft versus host disease (schoolROCK).
EU CTIS ID: 2024-511508-18-00
What this study is testing
Phase 1: To establish the Recommended Pediatric Equivalent Dose (RPED) of belumosudil Phase 2: To evaluate overall response rate (ORR) by 24 weeks (Week 25 visit or Cycle 7 Day 1 whichever is first)
- Phase I and Phase II (Integrated)- Other
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- 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.
- 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
You likely can't join if
- Progressive underlying disease or post-transplant lymphoproliferative disease within 4 weeks prior to the first dose of the IMP.
- 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
- Diagnosed with another malignancy (other than malignancy for which transplant was performed) within 3 years prior to the first dose of the IMP
- 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
See the full eligibility criteria
Who can join
- 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.
- 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
- 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
What rules you out
- Progressive underlying disease or post-transplant lymphoproliferative disease within 4 weeks prior to the first dose of the IMP.
- 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
- Diagnosed with another malignancy (other than malignancy for which transplant was performed) within 3 years prior to the first dose of the IMP
- 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
- 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
- The use of herbal and recreational drugs within 7 days before the start of study intervention
- Participant has had previous exposure to belumosudil
- 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
- 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
- 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 who meet any of the following criteria regarding systemic GVHD treatments: - 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 who meet any of the following criteria regarding systemic GVHD treatments:- 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. Note: Corticosteroids and calcineurin inhibitors may continue throughout the study.
- 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
The study team makes the final eligibility decision.
Where it's taking place
- Canada
- Japan
- Turkey
- United Kingdom
- China
- United States
- Israel
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18-64 years, 0-17 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Canada; Japan; Turkey; United Kingdom; China; United States and 1 more location(s). Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.