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

A Multicenter, Open-label, Single-arm, Phase 4 Clinical Study to Evaluate the Pharmacokinetics, Efficacy, and Safety of Belumosudil Mesylate Tablets in Chinese Adolescents (Aged From 12 to Less Than 18 Years) With Chronic Graft-versus-host Disease (cGVHD) Who Have Had an Inadequate Response to Glucocorticoids or Other Systemic Therapies

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.

Study Overview

• Status: Completed
• Conditions: Chronic Graft Versus Host Disease
• Intervention / Treatment: Drug: Belumosudil

• Study Type: Interventional
• Enrollment (Actual): 6
• Phase: Phase 4

Contacts and Locations

Study Locations

• China
  • Beijing, China, 100045
    • Investigational Site Number : 1560001
  • Shanghai, China, 200127
    • Investigational Site Number : 1560002

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• 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.

Exclusion Criteria:

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Belumosudil; Participants will receive belumosudil 200 mg tablets orally QD in 28-day cycles until clinically significant progression of cGVHD	Drug: Belumosudil; Pharmaceutical form: Tablet Route of administration: Oral; Other Names: Rezurock; SAR445761/ KD025

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Plasma concentrations of belumosudil at specified time points	At Day 1 and Day 29(±3)

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Overall response rate (ORR)	Overall response rate (ORR, including complete response [CR] and partial response [PR]), which will be assessed by the investigator according to the NIH Consensus Criteria (2014) at any time before the start of new systemic treatment for cGVHD.	Up to 18 month
Duration of response (DoR)	Duration of response (DoR): time from the date of the first response to the date of cGVHD progression as defined by 2014 NIH consensus response criteria, start of new systemic treatment for cGVHD, or death, whichever occurs first. DOR is determined only for participants who achieved overall response (PR or CR) as per 2014 NIH consensus response criteria.	Up to 18 month
System organ response rate	System organ response rate: proportion of participants who achieve an overall response (CR or PR) for each of the nine individual organs (Skin, Eyes, Mouth, Esophagus, Upper GI, Lower GI, Liver, Lungs, and Joints and fascia) as per 2014 NIH consensus response criteria at any time before the start of new systemic treatment for cGVHD	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)	Failure-free survival (FFS): time from the date of the first administration of IMP to 1) the date of initiation of new systemic treatment for cGVHD, or 2) date of death, or 3) date of recurrent underlying disease, whichever occurs first	Up to 18 month
Overall survival (OS)	Overall survival (OS): time from the date of the first administration of IMP to the date of death due to any cause	Up to 18 month
Change in cGVHD severity based on physician-reported overall cGVHD activity		Up to 18 month
Change in symptom activity assessed based on patient-reported cGVHD activity		Up to 18 month
Number and proportion of participants with calcineurin inhibitor (CNI) dose reduction or discontinuation during the treatment period		Up to 18 month
Number of participants with treatment-emergent adverse events (TEAEs), serious TEAEs, and adverse events of special interest (AESIs)		Up to 18 month
Modified Lee cGVHD Symptom Scale change	The scale contains 30 items grouped in 7 subscales (skin, eye, mouth, lung, nutrition, energy, and psychological). Patients report how "bothered" they feel about each symptom using a five-point Likert scale from "not at all" (0) to "extremely" (4). A higher score indicating worse symptoms, reporting score will be normalizing to a 0 to 100 scale.	Up to 18 month

Collaborators and Investigators

• Sponsor: Sanofi
• Investigators: Study Director: Clinical Sciences & Operations, Sanofi

Publications and helpful links

Helpful Links

• ACT18369 Plain Language Results Summary

Study record dates

Study Major Dates

• Study Start (Actual): December 4, 2024
• Primary Completion (Actual): May 12, 2026
• Study Completion (Actual): May 12, 2026

Study Registration Dates

• First Submitted: September 24, 2024
• First Submitted That Met QC Criteria: September 24, 2024
• First Posted (Actual): September 27, 2024

Study Record Updates

• Last Update Posted (Actual): May 27, 2026
• Last Update Submitted That Met QC Criteria: May 21, 2026
• Last Verified: May 1, 2026

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Organizing Pneumonia; Immune System Diseases; Respiratory Tract Diseases; Lung Diseases; Bronchial Diseases; Lung Diseases, Obstructive; Bronchiolitis Obliterans; Bronchiolitis; Bronchitis; Graft vs Host Disease; Bronchiolitis Obliterans Syndrome; Molecular Mechanisms of Pharmacological Action; Enzyme Inhibitors; Protein Kinase Inhibitors; belumosudil; KD025
• Other Study ID Numbers: ACT18369

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (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

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No