Safety and Efficacy of Oral Belumosudil in Black or African American, American Indian or Alaska Native, and Native Hawaiian or Other Pacific Islander Male and Female Participants Aged 12 Years and Above With Chronic Graft Versus Host Disease (cGVHD) After At Least 2 Prior Lines of Systemic Therapy

NCT05567406 | Withdrawn Phase 2 FDA Drug

• 3 other identifiers: SFY17661U1111-1277-6715KD025-218
• Study Type: interventional
• Target: N/A
• Locations: 1 country
• Sites: 3

Brief Summary

The purpose of this study is to measure safety and efficacy of oral belumosudil in Black or African American, American Indian or Alaska Native, and Native Hawaiian or Other Pacific Islander male and female participants with cGVHD who have previously been treated with at least 2 prior lines of systemic therapy aged 12 years and above. The duration of participants participation will be up to 4 weeks for screening, treatment until clinically significant progression of disease, and 4 weeks of safety follow-up, and then long-term follow-up every 12 weeks.1 Cycle = 28 days.

Conditions

Chronic Graft Versus Host Disease

Outcome Measures

Primary Outcomes (6)

• Number of participants with treatment emergent adverse events and serious adverse events

  Safety will be assessed by monitoring adverse events, physical Examinations, clinical laboratory evaluations, vital sign measurements, and ECG parameters.

  Up to approximately 48 months

• Number of participants with clinically significant laboratory abnormalities

  Up to approximately 12 months

• Change from baseline in systolic and diastolic blood pressure

  Baseline; up to approximately 12 months

• Change from baseline in heart rate

  Baseline; up to approximately 12 months

• Change from baseline in corrected QT interval using Fridericia's formula (QTc[F])

  Baseline; up to approximately 12 months

• Overall Response Rate (ORR)

  The ORR is defined as the proportion of participants meeting the overall response criteria assessment of Complete Response (CR) or Partial Response (PR) as defined by the 2014 NIH Consensus Development Project on Clinical Trials in cGVHD at any post-baseline response assessment.

  Up to approximately 12 months

Secondary Outcomes (15)

• Duration of Response (DOR)

  Up to approximately 12 months

• Change from baseline in the Lee Symptom Scale Score: Number of participants with a ≥ 7-point reduction

  Baseline; up to approximately 12 months

• Change from baseline in the Lee Symptom Scale Score: Number of participants with a ≥ 7-point reduction on 2 consecutive assessments

  Baseline; up to approximately 12 months

• Change from baseline in the Lee Symptom Scale Score: Duration of a ≥ 7 point reduction

  Baseline; up to approximately 12 months

• Response rate by organ system

  Up to approximately 12 months

Study Arms (1)

Belumosudil

EXPERIMENTAL

Participants will receive belumosudil orally, once daily (QD) or twice daily (BID) if they are taking strong CYP3A4 inducers or proton pump inhibitors.

Drug: Belumosudil

Interventions

Belumosudil DRUG

Pharmaceutical form: Tablet; Route of administration: Oral

Also known as: KD025, SAR445761, Rezurock

Belumosudil

Eligibility Criteria

• Age: 12 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Participants are included in the study if any of the following criteria apply:
• Participant is Black or African American, or American Indian or Alaska Native, or Native Hawaiian or Other Pacific Islander by self-identification.
• Previously received at least 2 and not more than 5 lines of systemic therapy for cGVHD.
• Receiving glucocorticoid therapy with a stable dose over the 2 weeks prior to screening.
• Have persistent cGVHD manifestations and systemic therapy is indicated.
• Karnofsky (if aged ≥ 16 years) / Lansky (if aged \< 16 years) Performance Score of ≥ 60.
• At least 12 years of age; weight ≥ 40 kilograms (kg).
• Alanine aminotransferase (ALT) and aspartate aminotransferase (AST) ≤ 3 x upper limit of normal (ULN).
• Total bilirubin ≤ 1.5 x ULN.
• Contraception (with double contraception methods) for male and female participants; not pregnant or breastfeeding for female participants
• Capable of giving signed informed consent.

You may not qualify if:

• Participants are excluded from the study if any of the following criteria apply:
• Participant has not been on a stable dose/regimen of systemic cGVHD treatment(s) for at least 2 weeks prior to screening. (Note: Concomitant corticosteroids, calcineurin inhibitors, sirolimus, MMF, methotrexate, rituximab, and ECP are acceptable. Systemic investigational GVHD treatments are not permitted).
• Histological relapse of the underlying cancer or post-transplant lymphoproliferative disease at the time of screening.
• Current treatment with ibrutinib or ruxolitinib. Prior treatment with ibrutinib or ruxolitinib is allowed with a washout of at least 28 days prior to enrollment.
• 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, poorly controlled psychiatric disease, or coronary artery disease).
• Corrected QT interval using Fridericia's formula (QTc\[F\]) \> 480 ms.
• Forced expiratory volume (in the first second; FEV1) ≤ 39% The above information is not intended to contain all considerations relevant to the potential participation in a clinical trial.

Sponsors & Collaborators

• Kadmon, a Sanofi Company: lead

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

Study Record Dates

• First Submitted: October 3, 2022
• First Posted: October 5, 2022
• Study Start: June 16, 2025
• Primary Completion (Estimated): August 31, 2026
• Study Completion (Estimated): August 31, 2026
• Last Updated: February 12, 2026

Record last verified: 2026-02

Data Sharing

• IPD Sharing: Will share

Locations

US (3)