schoolROCKBelumosudil for Children with Moderate-to-Severe Chronic Graft Versus Host Disease
Belumosudil
Bronchiolitis Obliterans Syndrome+8
+ Organizing Pneumonia
+ Bronchial Diseases
Treatment Study
Summary
Study start date: December 2, 2025
Actual date on which the first participant was enrolled.This clinical trial is designed to help children aged 1 to under 18 years who have a condition called chronic graft versus host disease (cGVHD). This disease can occur after a bone marrow transplant when the donated cells attack the recipient's body. The study focuses on children with a moderate to severe form of this disease that hasn't responded to at least two different treatments. The goal is to find out if a drug called belumosudil can be an effective treatment for these children, particularly focusing on its safety and how well it works, as well as determining the appropriate dose for younger children under 12 years old. Participants in this study will receive belumosudil and will be monitored closely. Treatment will continue until the disease progresses significantly, another treatment is needed, or if any serious side effects occur. Initially, the study will focus on determining how the drug is processed in the body and establishing a suitable dose for younger children. Later, it will evaluate the overall safety and effectiveness of the treatment in the broader age group up to 18 years. The study involves a screening period of up to 4 weeks, treatment until specific criteria are met, and a follow-up period of 4 weeks after treatment. Long-term follow-up will continue until the participant's death or the study ends, which is planned for 3 years after the last participant joins.
Protocol
This section provides details of the study plan, including how the study is designed and what the study is measuring.37 patients to be enrolled
Total number of participants that the clinical trial aims to recruit.Treatment Study
Eligibility
Researchers look for people who fit a certain description, called eligibility criteria: person's general health condition or prior treatments.Any sex
Biological sex of participants that are eligible to enroll.From 1 to 18 Years
Range of ages for which participants are eligible to join.Healthy volunteers not allowed
If individuals who are healthy and do not have the condition being studied can participate.Conditions
Pathology
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
Study Plan
Find out more about all the medication administered in this study, their detailed description and what they involve.One single intervention group is designated in this study
This study does not include a placebo group
Treatment Groups
Group I
ExperimentalStudy Objectives
Primary Objectives
Secondary Objectives
Study Centers
These are the hospitals, clinics, or research facilities where the trial is being conducted. You can find the location closest to you and its status.This study has 20 locations
Memorial Sloan Kettering Cancer Center- Site Number : 8400001
New York, United StatesOpen Memorial Sloan Kettering Cancer Center- Site Number : 8400001 in Google MapsTexas Children's Hospital - Baylor - PIN- Site Number : 8400008
Houston, United StatesInvestigational Site Number : 0560003
Ghent, BelgiumInvestigational Site Number : 0560001
Leuven, Belgium