Chronic Graft Versus Host Disease
Conditions
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.
Interventions
DRUGBelumosudil
Pharmaceutical form:Tablet-Route of administration:Oral
Sponsors
Sanofi
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
12 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* 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
Exclusion criteria
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 The above information is not intended to contain all considerations relevant to a participant's potential participation in a clinical trial.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 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
| Measure | Time frame | Description |
|---|---|---|
| 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. |
| 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 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 | — |
| Time taken for New Treatment (TTNT) or death | Baseline to end of study i.e. 12 months | — |
| Failure-free survival (FFS) | Baseline to end of study i.e. 12 months | — |
| Overall survival (OS) | Baseline to end of study i.e. 12 months | — |
| Change in Corticosteroid dose. | 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 |
| 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 | — |
Contacts
CONTACTTrial Transparency email recommended (Toll free for US & Canada)
Outcome results
None listed