Recruiting

schoolROCKBelumosudil for Children with Moderate-to-Severe Chronic Graft Versus Host Disease

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What is being tested

Belumosudil

Drug
Who is being recruted

Bronchiolitis Obliterans Syndrome+8

+ Organizing Pneumonia

+ Bronchial Diseases

From 1 to 18 Years
See all eligibility criteria
How is the trial designed

Treatment Study

Phase 2
Interventional
Study Start: December 2025
See protocol details

Summary

Principal SponsorSanofi
Study ContactTrial Transparency email recommended (Toll free for US & Canada)
Last updated: January 28, 2026
Sourced from a government-validated database.Claim as a partner

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.

Official TitleAn 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)
NCT07116031
Principal SponsorSanofi
Study ContactTrial Transparency email recommended (Toll free for US & Canada)
Last updated: January 28, 2026
Sourced from a government-validated database.Claim as a partner

Protocol

This section provides details of the study plan, including how the study is designed and what the study is measuring.
Design Details

37 patients to be enrolled

Total number of participants that the clinical trial aims to recruit.

Treatment Study

These studies test new ways to treat a disease, condition, or health issue. The goal is to see if a new drug, therapy, or approach works better or has fewer side effects than existing options.



Eligibility

Researchers look for people who fit a certain description, called eligibility criteria: person's general health condition or prior treatments.
Conditions
Criteria

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

Bronchiolitis Obliterans SyndromeOrganizing PneumoniaBronchial DiseasesBronchiolitisBronchiolitis ObliteransBronchitisGraft vs Host DiseaseImmune System DiseasesLung DiseasesLung Diseases, ObstructiveRespiratory Tract Diseases

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.
Treatment Groups
Study Objectives

One single intervention group is designated in this study

This study does not include a placebo group 

Treatment Groups

Group I

Experimental
Participant will take IMP with a meal approximately the same time each morning. IMP dose will be according to weight and will be increased to daily dose of twice a day (BID) in participants who concomitantly receive proton pump inhibitors (PPIs) or strong CYP3A4 inducers. No concomitant PPIs are allowed during Phase 1 up to and including Cycle 1 Day 15. From Day 16 onwards, PPIs will be permitted, resulting in an increased dose of Belumosudil to BID. No concomitant strong CYP3A4 inducers are allowed during Phase 1.

Study 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

Recruiting

Memorial Sloan Kettering Cancer Center- Site Number : 8400001

New York, United StatesOpen Memorial Sloan Kettering Cancer Center- Site Number : 8400001 in Google Maps
Recruiting

Texas Children's Hospital - Baylor - PIN- Site Number : 8400008

Houston, United States
Recruiting

Investigational Site Number : 0560003

Ghent, Belgium
Recruiting

Investigational Site Number : 0560001

Leuven, Belgium
Recruiting
20 Study Centers
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