A Clinical Study to Evaluate the Pharmacokinetics, Efficacy, and Safety of Belumosudil in Chinese Adolescents With cGVHD Who Have Had an Inadequate Response to Glucocorticoids or Other Systemic Therapies

NCT06616415 | Active Not Recruiting Phase 4

• 1 other identifier: ACT18369
• Study Type: interventional
• Target: 6
• Locations: 1 country
• Sites: 2

Brief Summary

This is a single group, Phase 4, single-arm post-marketing study for treatment. The purpose of this study is to verify the pharmacokinetics, efficacy, and safety of belumosudil mesylate tablets in Chinese adolescent participants (aged from 12 to less than 18) with cGVHD who have had an inadequate response to glucocorticoids or other systemic therapies. Participants will receive treatment with belumosudil tablets 200 mg once daily in 28-day cycles during the study.

Conditions

Chronic Graft Versus Host Disease

Outcome Measures

Primary Outcomes (1)

• Plasma concentrations of belumosudil at specified time points

  At Day 1 and Day 29(±3)

Secondary Outcomes (11)

• Overall response rate (ORR)

  Up to 18 month

• Duration of response (DoR)

  Up to 18 month

• System organ response rate

  Up to 18 month

• Number and proportion of participants with dose reduction in corticosteroid during the treatment period

  Up to 18 month

• Failure-free survival (FFS)

  Up to 18 month

Study Arms (1)

Belumosudil

EXPERIMENTAL

Participants will receive belumosudil 200 mg tablets orally QD in 28-day cycles until clinically significant progression of cGVHD

Drug: Belumosudil

Interventions

Belumosudil DRUG

Pharmaceutical form: Tablet Route of administration: Oral

Also known as: SAR445761/ KD025, Rezurock

Belumosudil

Eligibility Criteria

• Age: 12 Years - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Participant must be 12 to less than 18 years of age at the time of signing the informed consent.
• Participant has undergone allogeneic hematopoietic stem cell transplantation (allo-HSCT).
• Has active moderate to severe cGVHD.
• Has received at least one line of prior systemic therapy for cGVHD.
• Participant must receive a corticosteroid therapy for cGVHD with a stable dose for at least 2 weeks prior to the first dose of the IMP.
• Has a Lansky-Play performance score of ≥60.
• Participants should have an expected survival of longer than 6 months.
• Body weight of 30 kg and above.
• Contraceptive use by men and women 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.

You may not qualify if:

• Participants are excluded from the study if any of the following criteria apply:
• Recurrence of hematologic neoplasms (according to the corresponding criteria for recurrence of primary hematologic neoplasms) or post-transplant lymphoproliferative disease at screening.
• Received investigational systemic therapy for cGVHD within 28 days prior to enrollment, unless the prior treatment had been washed out for at least 28 days or 5 half-lives prior to enrollment, whichever is shorter.
• Absolute neutrophil count (ANC) \<1.0 × 10\^9/L.
• Platelet count \<50 × 10\^9/L.
• Alanine aminotransferase (ALT) \>3× the upper limit of normal (ULN), aspartate aminotransferase (AST) \>3 × ULN.
• Total bilirubin (TBIL) \>1.5 × ULN (\>3 ULN if Gilbert's syndrome).
• Estimated Glomerular Filtration Rate (eGFR) \<30 mL/min/1.73 m\^2 using the revised Bedside Schwartz formula . Revised Schwartz equation: CrCl (mL/min/1.73 m\^2) = 0.413 × (height \[in cm\])/Creatinine (in mg/dL) at screening visit.
• Participant 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.

Sponsors & Collaborators

• Sanofi: lead

Study Sites (2)

Investigational Site Number : 1560001

Beijing, 100045, China

Location

Investigational Site Number : 1560002

Shanghai, 200127, China

Location

MeSH Terms

Conditions

Bronchiolitis Obliterans Syndrome

Interventions

belumosudil; KD025

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

Study Officials

• Clinical Sciences & Operations

  Sanofi

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 24, 2024
• First Posted: September 27, 2024
• Study Start: December 4, 2024
• Primary Completion (Estimated): May 13, 2026
• Study Completion (Estimated): May 13, 2026
• Last Updated: June 22, 2025

Record last verified: 2025-06

Data Sharing

• IPD Sharing: Will share

Locations

CN (2)