A Study to Investigate Safety of Belumosudil in Participants Aged 12 Years and Above, With Chronic Graft-versus-host Disease (cGVHD)
- Conditions
- Chronic Graft Versus Host Disease
- Interventions
- Registration Number
- NCT07135973
- Lead Sponsor
- Sanofi
- Brief Summary
This is an interventional phase IV clinical study which is single-arm study for assessing the safety of belumosudil in Indian patients who are12 years and older.
Study details include:
* The study duration will be up to 12 months per participant.
* The treatment duration will be up to 24 weeks.
* The number of visits will be 7.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- NOT_YET_RECRUITING
- Sex
- All
- Target Recruitment
- 26
Male or female patients at least 12 years of age inclusive, at the time of signing the informed consent Participants who had an allogeneic hematopoietic cell transplantation (HCT) Previously received at least 2 prior lines of systemic therapy for cGVHD Received glucocorticoid therapy with a stable dose over the 2 weeks prior to screening Had persistent cGVHD manifestations and systemic therapy was indicated Karnofsky (if aged ≥ 16 years)/Lansky (if aged <16 years) Performance Score of ≥ 60
Capable of giving signed informed consent as described in the protocol
Participants are excluded from the study if any of the following criteria apply:
Absolute neutrophil count ≤1.5 X 109/L Platelet count ≤ 50 X 109/L Alanine aminotransferase (ALT) and aspartate aminotransferase (AST) ≥ 3 X upper limit of normal (ULN) (>5 × ULN if due to cGVHD) Total bilirubin ≥ 1.5 X ULN Glomerular filtration rate (GFR) ≤ 30 mL/min/1.73m3 Positive hepatitis B surface antigen, or hepatitis C antibody or HIV antibody at screening FEV1 ≤39% or has lung score of 3
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description belumosudil Belumosudil 200 mg taken orally once daily with food for 24 weeks
- Primary Outcome Measures
Name Time Method Drug related Grade ≥ 3 treatment emergent adverse events (TEAE) From baseline to 24 weeks Drug related Grade ≥ 3 treatment emergent adverse events (TEAE) during the treatment period (24 weeks)
- Secondary Outcome Measures
Name Time Method Best overall Response (BoR) Baseline to end of study i.e. 12 months Best overall Response (BoR), defined as the proportion of patients who achieved complete response (CR) or partial response (PR) according to the NIH Consensus Criteria.
Time to Response (TTR) Baseline to end of study i.e. 12 months Response by organ system (including GSR) Baseline to end of study i.e. 12 months Change in CNI dose Baseline to end of study i.e. 12 months Symptomatic improvement from baseline during treatment based on modified Lee Symptom Scale Baseline to end of study i.e. 12 months defined as a ≥7-point reduction from baseline in total symptom score on a scale of 0 to 100 which measures the symptoms of chronic GVHD, with higher scores indicating worse symptoms
Duration of Response (DOR) Baseline to end of study i.e. 12 months Duration of Response (DOR) calculated from time of initial PR or CR until documented progression, death, or new systemic therapy.
Time taken for New Treatment (TTNT) or death Baseline to end of study i.e. 12 months Change in Corticosteroid dose. Baseline to end of study i.e. 12 months Failure-free survival (FFS) Baseline to end of study i.e. 12 months Pulmonary Function Test (PFT) Baseline to end of study i.e. 12 months Total lung capacity, Residual volume, Forced expiratory volume, Tidal volume
Number of participants with treatment-emergent adverse events [TEAEs], serious TEAEs, and adverse events of special interest (AESIs) Baseline to end of study i.e. 12 months Overall survival (OS) Baseline to end of study i.e. 12 months