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Chronic Graft Versus Host DiseasePhase 2schoolROCKNCT07116031

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

A Phase 2 study testing Belumosudil in people with Chronic Graft Versus Host Disease, run by Sanofi at 35 sites in 13 countries.

Recruiting

Looking for participants now. Contact the study team or a site below to ask about taking part.

Sites and contacts
Official record

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Trial 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. Read more about this study

Who is it for?
Ages 1 year to 18 years
Conditions the study is for
Chronic Graft Versus Host Disease
What is being tested?
Belumosudil
Type of treatment
Pill or oral medicine
Could I receive a placebo?
No, everyone receives an active treatment
How many people
37 participants
Where
35 sitesin 13 countries
Study dates
2 Dec 2025to 28 Feb 2031
Phase
Phase 2

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 1 year to 18 years
  • All sexes
  • No healthy volunteers
  • Child, Adult

Who may be able to 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.
  • 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
Show all 12Show fewer
  • Has a Lansky-Play (if aged <16 years) or Karnofsky (if aged ≥16 years) 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
  • 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

Who cannot join

  • 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
  • Participants who meet any of the following criteria regarding systemic GVHD treatments:
Show all 28Show fewer
  • Participants who newly initiated any systemic GVHD treatment within 14 days prior to the first dose of belumosudil.
  • 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 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.
  • 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 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
  • 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.

Trial locations and contacts

35 sites in 13 countries. Contact the study team or a site near you to ask about taking part.

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

800-633-1610 ext. option 6

contact-us@sanofi.com

35 sites in 13 countries

United States7 sites, 7 recruiting

  • Children's Healthcare of Atlanta - Egleston Hospital- Site Number : 8400003

    Atlanta, Georgia, 30322

    Recruiting

  • Dana-Farber Cancer Institute- Site Number : 8400007

    Boston, Massachusetts, 02215

    Recruiting

  • Texas Children's Hospital- Site Number : 8400008

    Houston, Texas, 77030

    Recruiting

  • Children's Hospital Los Angeles- Site Number : 8400009

    Los Angeles, California, 90027

    Recruiting

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

    New York, New York, 10065

    Recruiting

  • Fred Hutchinson Cancer Research Center- Site Number : 8400002

    Seattle, Washington, 98109

    Recruiting

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

    Washington D.C., District of Columbia, 20010

    Recruiting

Other trials in United States

Israel5 sites, 5 recruiting

  • Investigational Site Number : 3760005

    Haifa, 3109601

    Recruiting

  • Investigational Site Number : 3760002

    Jerusalem, 9112001

    Recruiting

  • Investigational Site Number : 3760004

    Petah Tikva, 4920235

    Recruiting

  • Investigational Site Number : 3760003

    Ramat Gan, 5262100

    Recruiting

  • Investigational Site Number : 3760001

    Tel Aviv, 6423906

    Recruiting

Other trials in Israel

China4 sites, 4 recruiting

  • Investigational Site Number : 1560003

    Beijing, 100045

    Recruiting

  • Investigational Site Number : 1560001

    Shanghai, 200040

    Recruiting

  • Investigational Site Number : 1560004

    Shenzhen, 518026

    Recruiting

  • Investigational Site Number : 1560002

    Suzhou, 215025

    Recruiting

Other trials in China

Italy3 sites, 3 recruiting

  • Investigational Site Number : 3800002

    Monza, 20900

    Recruiting

  • Investigational Site Number : 3800001

    Rome, Roma, 00165

    Recruiting

  • Investigational Site Number : 3800003

    Turin, Torino, 10126

    Recruiting

Other trials in Italy

Spain3 sites, 3 recruiting

  • Investigational Site Number : 7240001

    Barcelona, Catalunya [Cataluña], 08035

    Recruiting

  • Investigational Site Number : 7240003

    Esplugues de Llobregat, Barcelona [Barcelona], 08950

    Recruiting

  • Investigational Site Number : 7240002

    Madrid, 28009

    Recruiting

Other trials in Spain

Belgium2 sites, 2 recruiting

  • Investigational Site Number : 0560003

    Ghent, 9000

    Recruiting

  • Investigational Site Number : 0560001

    Leuven, 3000

    Recruiting

Other trials in Belgium

Canada2 sites, 2 recruiting

  • Investigational Site Number : 1240002

    Toronto

    Recruiting

  • Investigational Site Number : 1240001

    Vancouver, British Columbia, V6H 3N1

    Recruiting

Other trials in Canada

France2 sites, 2 recruiting

  • Investigational Site Number : 2500002

    Marseille, 13885

    Recruiting

  • Investigational Site Number : 2500001

    Paris, 75019

    Recruiting

Other trials in France

Turkey (Türkiye)2 sites, 2 recruiting

  • Investigational Site Number : 7920003

    Ankara, 06800

    Recruiting

  • Investigational Site Number : 7920001

    Izmir, 35100

    Recruiting

Other trials in Turkey (Türkiye)

United Kingdom2 sites, 2 recruiting

  • Investigational Site Number : 8260001

    London, London, City of, WC1N 3JH

    Recruiting

  • Investigational Site Number : 8260002

    Newcastle upon Tyne, England, NE2 4HH

    Recruiting

Other trials in United Kingdom

Germany1 site, 1 recruiting

  • Investigational Site Number : 2760001

    Berlin, 13353

    Recruiting

Other trials in Germany

Japan1 site, 1 recruiting

  • Investigational Site Number : 3920002

    Tokyo, 157-8535

    Recruiting

Other trials in Japan

Netherlands1 site, 1 recruiting

  • Investigational Site Number : 5280001

    Utrecht, 3584 CS

    Recruiting

Other trials in Netherlands

About this study

Official title: 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)

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.

Read more

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.

30 days of post treatment safety follow-up. Long-term follow-up until death or end of study, whichever occurs first.

Being tested
Belumosudil
Medical subject headings
Bronchiolitis Obliterans Syndrome

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • BelumosudilDrug

    Also called SAR445761, REZUROCK

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

  • BelumosudilDrug

    Also called SAR445761, REZUROCK

    Pharmaceutical form:Tablet formulation-Route of administration:Oral

Study groups in NCT07116031
Study groupRoleWhat they receive
belumosudilExperimentalParticipant 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.
Receives: Drug: Belumosudil

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Phase 1: AUC

    PK parameter (AUC at steady state)

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

  2. 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]) with up to 24 weeks of therapy (i.e. by the Week 25 or Cycle 7 Day 1 visit whichever is first), as defined by the National Institute of Health (NIH) Consensus response criteria

    Measured: Last participant completing 24 weeks (Week 25 visit or Cycle 7 Day 1 visit, whichever comes first) in the study

Secondary outcomes (18)

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

    Safety

    Measured: Up to 3 years after the last participant enrolled

  2. Phase 1: Cmax

    Steady-state belumosudil PK parameters

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

  3. Phase 1: AUC0-6h

    Steady-state belumosudil PK parameters

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

  4. Phase 1: ORR

    Proportion of participants who achieve an overall response (complete response [CR] or partial response [PR]) by 24 weeks, as defined by the 2014 National Institute of Health (NIH) Consensus response criteria

    Measured: last participant completing 24 weeks (Week 25 visit or Cycle 7 Day 1 visit, whichever comes first) in the study.

  5. Phase 1: DOR

    Time from the date of first response to the date of progression of cGVHD, initiation of a new systemic treatment for cGVHD, or death, whichever comes first. DOR is determined only for participants who achieved overall response (PR or CR) as per 2014 NIH Consensus response criteria

    Measured: Up to 3 years after the last participant enrolled

  6. Phase 1: response by organ

    As defined by the 2014 NIH Consensus response criteria

    Measured: Up to 3 years after the last participant enrolled

  7. Phase 1: failure-free survival (FFS)

    Time from the date of the first investigational medicinal product (IMP) administration to the date of initiation of a new systemic treatment for cGVHD, relapse or recurrence of the underlying disease, or death, whichever occurs first

    Measured: Up to 3 years after the last participant enrolled

  8. Phase 1: overall survival (OS)

    Time from the date of first IMP administration to the date of death due to any cause

    Measured: Up to 3 years after the last participant enrolled

  9. Phase 1: time to response (TTR)

    Time from the date of the first IMP administration to the first documented response (either CR or PR) according to the 2014 NIH Consensus Criteria for cGVHD

    Measured: Up to 3 years after the last participant enrolled

  10. Phase 2: Number of participants with treatment-emergent adverse events [TEAEs], serious TEAEs, and adverse events of special interest (AESIs)

    Safety

    Measured: Up to 3 years after the last participant enrolled

  11. Phase 2: Ctrough of belumosudil

    Measured: Cycle 2 Day 1 and Cycle 4 Day 1 after the last participant enrolled

  12. Phase 2: DOR

    Time from first response to the date of progression of cGVHD, initiation of a new systemic treatment for cGVHD, or death, whichever comes first. DOR is determined only for participants who achieved overall response (PR or CR) as per NIH Consensus response criteria

    Measured: Up to 3 years after the last participant enrolled

  13. Phase 2: response by organ

    response by organ as defined by the 2014 NIH Consensus response criteria

    Measured: Up to 3 years after the last participant enrolled

  14. Phase 2: FFS

    Time from the date of the first IMP administration to the date of initiation of a new systemic treatment for cGVHD, relapse or recurrence of the underlying disease, or death, whichever occurs first

    Measured: Up to 3 years after the last participant enrolled

  15. Phase 2: OS

    Time from the date of first IMP administration to the date of death due to any cause

    Measured: Up to 3 years after the last participant enrolled

  16. Phase 2: time to response (TTR)

    Time from the date of the first IMP administration to the first documented response (either CR or PR) according to the 2014 NIH Consensus Criteria for cGVHD

    Measured: Up to 3 years after the last participant enrolled

  17. Phase 1: time to next treatment (TTNT)

    Time from the date of the first IMP administration to initiating the next line of systemic therapy, or death whichever is first

    Measured: Up to 3 years after the last participant enrolled

  18. Phase 2: time to next treatment (TTNT)

    Time from the date of the first IMP administration to initiating the next line of systemic therapy, or death whichever is first

    Measured: Up to 3 years after the last participant enrolled

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted11 Aug 2025
  2. Study start2 Dec 2025
  3. Primary completion25 Sep 2028 (estimated)
  4. Study completion28 Feb 2031 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
37 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT07116031
Sponsor's protocol ID
DFI17893
Other IDs
U1111-1281-0103, 2024-511508-18
Acronym
schoolROCK
Status last verified
Sep 2026
First submitted
25 Jul 2025
Last updated
22 Sep 2026
Results posted
No
Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
Yes: Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org

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