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

NCT07116031 | Recruiting Phase 2 FDA Drug

• 3 other identifiers: DFI17893U1111-1281-01032024-511508-18
• Study Type: interventional
• Target: 37
• Locations: 12 countries
• Sites: 29

Brief Summary

This is an open-label, single group, Phase 1/2, 1-arm study for treatment of children aged 1 to \<18 years with active moderate-to-severe cGVHD that is refractory to or recurred after at least 2 prior lines of systemic therapy for cGVHD. The purpose of Phase 1 is to determine the PK profiles and to establish the Recommended Pediatric Equivalent Dose (RPED) of belumosudil in participants aged 1 to \<12 years with active moderate to severe cGVHD. Upon completion and evaluation of Phase 1, Phase 2 will commence with the purpose of determining safety and efficacy (ORR by 24 weeks) of belumosudil in participants aged 1 to \<18 years. Study details include: The end of study is defined as 3 years after the last participant is recruited or all participants have discontinued treatment, or have died, whichever comes first. Minimum of 6 participants ages 1 to 6 years will be enrolled for each phase of study Individual participant duration on study will consist of: Up to 4 weeks for screening. Treatment until clinically significant progression of cGVHD, relapse/recurrence of the underlying disease, start of a new systemic treatment for cGVHD, experience of an unacceptable adverse event, request from participant or Investigator, or until the end of the study is reached, whichever comes first. 4 weeks of post treatment safety follow-up. Long-term follow-up until death or end of study, whichever occurs first.

Conditions

Chronic Graft Versus Host Disease

Outcome Measures

Primary Outcomes (2)

• Phase 1: AUC

  PK parameter (AUC at steady state)

  Cycle 1 Day 15 after the last participant dosed in the phase 1 part.

• 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 first

  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 first, as defined by the National Institute of Health (NIH) Consensus response criteria

  Up to 3 years after the last participant enrolled

Secondary Outcomes (16)

• Phase 1: Number of participants with treatment-emergent adverse events [TEAEs], serious TEAEs, and adverse events of special interest (AESIs)

  Up to 3 years after the last participant enrolled

• Phase 1: Cmax

  Cycle 1 Day 15 after the last participant dosed in the phase 1 part

• Phase 1: AUC0-6h

  Cycle 1 Day 15 after the last participant dosed in the phase 1 part

• Phase 1: ORR

  Up to 3 years after the last participant enrolled

• Phase 1: DOR

  Up to 3 years after the last participant enrolled

Study Arms (1)

belumosudil

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.

Drug: Belumosudil

Interventions

Belumosudil DRUG

Pharmaceutical form:Oral suspension -Route of administration:Oral or nasogastric tube

Also known as: SAR445761, REZUROCK

belumosudil

Eligibility Criteria

• Age: 1 Year - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify 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.
• 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

You may not qualify if:

• 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)

Sponsors & Collaborators

• Sanofi: lead
• Meiji Seika Pharma Co., Ltd.: collaborator

Study Sites (29)

Children's Hospital Los Angeles- Site Number : 8400009

Los Angeles, California, 90027, United States

RECRUITING

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

Washington D.C., District of Columbia, 20010, United States

RECRUITING

Memorial Sloan Kettering Cancer Center - New York - York Avenue- Site Number : 8400001

New York, New York, 10065, United States

RECRUITING

Texas Children's Hospital- Site Number : 8400008

Houston, Texas, 77030, United States

RECRUITING

Investigational Site Number : 0560003

Ghent, 9000, Belgium

RECRUITING

Investigational Site Number : 0560001

Leuven, 3000, Belgium

RECRUITING

Investigational Site Number : 1240002

Toronto, Ontario, M5G 1X8, Canada

RECRUITING

Investigational Site Number : 1560003

Beijing, 100045, China

RECRUITING

Investigational Site Number : 1560001

Shanghai, 200040, China

RECRUITING

Investigational Site Number : 1560004

Shenzhen, 518026, China

RECRUITING

Investigational Site Number : 1560002

Suzhou, 215025, China

RECRUITING

Investigational Site Number : 2500002

Marseille, 13885, France

RECRUITING

Investigational Site Number : 2500001

Paris, 75019, France

RECRUITING

Investigational Site Number : 2760001

Berlin, 13353, Germany

RECRUITING

Investigational Site Number : 3760005

Haifa, 3109601, Israel

RECRUITING

Investigational Site Number : 3760002

Jerusalem, 9112001, Israel

RECRUITING

Investigational Site Number : 3760004

Petah Tikva, 4920235, Israel

RECRUITING

Investigational Site Number : 3760003

Ramat Gan, 5262100, Israel

RECRUITING

Investigational Site Number : 3760001

Tel Aviv, 6423906, Israel

RECRUITING

Investigational Site Number : 3800002

Milan, Milano, 20122, Italy

RECRUITING

Investigational Site Number : 3800001

Rome, Roma, 00165, Italy

RECRUITING

Investigational Site Number : 5280001

Utrecht, 3584 CS, Netherlands

RECRUITING

Investigational Site Number : 7240003

Esplugues de Llobregat, Barcelona [Barcelona], 08950, Spain

RECRUITING

Investigational Site Number : 7240001

Barcelona, 08035, Spain

RECRUITING

Investigational Site Number : 7240002

Madrid, 28009, Spain

RECRUITING

Investigational Site Number : 7920003

Ankara, 06800, Turkey (Türkiye)

RECRUITING

Investigational Site Number : 7920001

Izmir, 35100, Turkey (Türkiye)

RECRUITING

Investigational Site Number : 8260002

Newcastle upon Tyne, England, NE2 4HH, United Kingdom

RECRUITING

Investigational Site Number : 8260001

London, London, City of, WC1N 3JH, United Kingdom

RECRUITING

Related Links

• DFI17893 Plain Language Results Summary

MeSH Terms

Conditions

Bronchiolitis Obliterans Syndrome

Interventions

belumosudil

Condition Hierarchy (Ancestors)

Organizing Pneumonia; Bronchiolitis Obliterans; Bronchiolitis; Bronchitis; Bronchial Diseases; Respiratory Tract Diseases; Lung Diseases, Obstructive; Lung Diseases; Graft vs Host Disease; Immune System Diseases

Central Study Contacts

Trial Transparency email recommended (Toll free for US & Canada)

CONTACT

800-633-1610; contact-us@sanofi.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 25, 2025
• First Posted: August 11, 2025
• Study Start: December 2, 2025
• Primary Completion (Estimated): February 28, 2031
• Study Completion (Estimated): February 28, 2031
• Last Updated: April 30, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will share

Locations

US (4); TU (2); CA (1); FR (2); IL (5); BE (2); CN (4); IT (2); NL (1); ES (3); DE (1); GB (2)