A study of belumosudil in children with chronic graft versus host disease (schoolROCK).

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Sanofi-Aventis Recherche & Developpement

Participant type

Pediatric, Patients

Age range

0-17 years, 18-64 years

Gender

Male and Female

Therapeutic area

Diseases [C] - Immune System Diseases [C20]

Trial duration

5 Jan 2026 → ongoing

Decision date (initial)

2025-10-01

Transition trial

No

Low-intervention

No

Rare-disease indication

No

Vulnerable population

No

External identifiers

EU CT number

2024-511508-18-00

WHO UTN

U1111-1281-0103

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Pharmacokinetic, Dose response, Safety, Efficacy

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)

Secondary objectives 17

1. Phase 1: To evaluate safety
2. Phase 1: To evaluate the overall response rate (ORR) by 24 weeks
3. Phase 1: To evaluate the duration of response (DOR)
4. Phase 1: To evaluate the response by organ
5. Phase 1: To evaluate the failure-free survival (FFS)
6. Phase 1: To evaluate the overall survival (OS)
7. Phase 1: To evaluate time to response (TTR)
8. Phase 1: To evaluate PK
9. Phase 2: To evaluate safety
10. Phase 2: To evaluate PK
11. Phase 2: To evaluate the DOR
12. Phase 2: To evaluate the response by organ
13. Phase 2: To evaluate the FFS
14. Phase 2: To evaluate the OS
15. Phase 2: To evaluate time to response (TTR)
16. Phase 1: To evaluate the time to next treatment (TTNT) for cGVHD
17. Phase 2: To evaluate the time to next treatment (TTNT) for cGVHD

Conditions and MedDRA coding

Chronic graft versus host disease.

Regulatory references

EMA paediatric investigation plan (PIP)

EMEA-003425-PIP01-23

Plan to share IPD

Yes

IPD plan description

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

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 12

1. 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.
2. Life expectancy of >6 months
3. Participants can take the IMP orally or via a nasogastric tube
4. Participant has undergone an allogeneic HCT
5. Has active moderate to severe cGVHD, defined using the NIH Consensus diagnosis and staging criteria for which systemic therapy is required
6. cGVHD is refractory to or has recurred after at least 2 prior lines of systemic treatment
7. Has received at least two lines of prior systemic therapy for cGVHD, but no more than 5 lines.
8. 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
9. Has a Lansky-Play (if aged <16 years) or Karnofsky (if aged ≥16 years) performance scale of ≥60
10. Body weight of 8 kg and above
11. 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
12. The participant or their legally authorized representative (LAR) must be capable of giving signed informed consent

Exclusion criteria 23

1. Progressive underlying disease or post-transplant lymphoproliferative disease within 4 weeks prior to the first dose of the IMP.
2. 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
3. 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.
4. 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.
5. 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
6. 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)
7. Alanine aminotransferase (ALT) and/or aspartate aminotransferase (AST) >3× upper limit of normal (ULN) (> 5x ULN if abnormalities are due to cGVHD)
8. Total bilirubin >1.5 × ULN (>3 x ULN if Gilbert’s syndrome or if abnormalities are due to cGVHD)
9. Glomerular filtration rate (GFR) <30 mL/min/1.73 m2 using the revised Bedside Schwartz calculator
10. Participants with an active viral disease including hepatitis B virus (HBV) and hepatitis C virus (HCV)
11. Active uncontrolled Cytomegalovirus (CMV) or Epstein-Barr virus (EBV) infection
12. Diagnosed with another malignancy (other than malignancy for which transplant was performed) within 3 years prior to the first dose of the IMP
13. Known history of human immunodeficiency virus (HIV)
14. 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
15. 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)
16. Has a forced expiratory volume (in the first second; FEV1) ≤39% or has lung score of 3
17. Female participants who are pregnant or breastfeeding
18. The use of herbal and recreational drugs within 7 days before the start of study intervention
19. Participant has had previous exposure to belumosudil
20. 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
21. 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.
22. 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)
23. 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.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 2

1. Phase 1: AUC
2. Phase 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

Secondary endpoints 18

1. Phase 1: Number of participants with treatment- emergent adverse events [TEAEs], serious TEAEs, and adverse events of special interest (AESIs)
2. Phase 1: Cmax
3. Phase 1: AUC0-6h
4. Phase 1: ORR
5. Phase 1: DOR
6. Phase 1: response by organ
7. Phase 1: failure-free survival (FFS)
8. Phase 1: overall survival (OS)
9. Phase 1: time to response (TTR)
10. Phase 2: Number of participants with treatment- emergent adverse events [TEAEs], serious TEAEs, and adverse events of special interest (AESIs)
11. Phase 2: Ctrough of belumosudil
12. Phase 2: DOR
13. Phase 2: response by organ
14. Phase 2: FFS
15. Phase 2: OS
16. Phase 2: time to response (TTR)
17. Phase 1: time to next treatment (TTNT)
18. Phase 2: time to next treatment (TTNT)

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 2

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Sanofi-Aventis Recherche & Developpement

Sponsor organisation

Sanofi-Aventis Recherche & Developpement

Address

13 Quai Jules Guesde

City

Vitry Sur Seine

Postcode

94400

Country

France

Scientific contact point

Organisation

Sanofi-Aventis Recherche & Developpement

Contact name

Clinical Sciences and Operations

Public contact point

Organisation

Sanofi-Aventis Recherche & Developpement

Contact name

Clinical Sciences and Operations

Third parties 10

Locations

6 EU/EEA countries · 13 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 102 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

8 submissions — initial application plus substantial / non-substantial modifications