Health Condition 1: D811- Severe combined immunodeficiency [SCID] with low T- and B-cell numbers
Conditions
Interventions
Intervention1: REZUROCK /belumosudil
: 200 mg tablet taken orally once daily with food for 24 weeks
Control Intervention1: NA: NA
Sponsors
Sanofi Healthcare India Pvt Limited (SHIPL)
Eligibility
Inclusion criteria
Inclusion criteria: I 01 Male or female patients at least 12 years of age inclusive, at the time of signing the informed consent Type of participant and disease characteristics I 02 Participants who had an allogeneic hematopoietic cell transplantation (HCT) Sex, contraceptive/barrier method and pregnancy testing requirements/breastfeeding I 03. Male or Female -Female: A female participant is eligible to participate if she is not pregnant, not breastfeeding, and at least 1 of the following conditions applies- --Not a woman of childbearing potential (WOCBP). OR --WOCBP who agrees to follow the contraceptive guidance which is highly effective (as described in Section XX) during the study intervention period and for at least 90 days after the last administration of study intervention - Male participants are eligible to participate if they agree during the study intervention period and for at least 90 days after the last administration of study intervention: --Either to be abstinent from heterosexual intercourse as their preferred and usual lifestyle (abstinent on a long term and persistent basis) and agree to remain abstinent; OR must agree to use contraception/barrier (a male condom and an additional highly effective contraceptive method (as described in Section XX) when having sexual intercourse with a WOCBP who is not currently pregnant and capable of giving signed informed consent (including compliance with the requirements and restrictions listed in the informed consent form (ICF) and in the protocol) Informed Consent I 04. Capable of giving signed informed consent as described in Appendix 1 of the protocol which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol. Other inclusion criteria I 05. Previously received at least 2 prior lines of systemic therapy for cGVHD; I 06. Received glucocorticoid therapy with a stable dose over the 2 weeks prior to screening. Note: Concomitant corticosteroids, calcineurin inhibitors, sirolimus, MMF, methotrexate, rituximab, and extracorporeal photopheresis (ECP) are acceptable. Systemic investigational GVHD treatments are not permitted; I 07. Had persistent cGVHD manifestations and systemic therapy was indicated; I 08. Karnofsky (if aged more than equal to 16 years)/Lansky (if aged more than equal to 16 years)/Lansky (if aged less than 16 years) Performance Score of more than equal to 60
Exclusion criteria
Exclusion criteria: Participants are excluded from the study if any of the following criteria apply- Diagnostic assessments E 01. Absolute neutrophil count less than equal to 1.5 X 109/L; E 02. Platelet count less than equal to 50 X 109/L; E 03. Alanine aminotransferase (ALT) and aspartate aminotransferase (AST) more than equal to 3 X upper limit of normal (ULN) (more than 5 ULN if due to cGVHD).; E 04. Total bilirubin more than equal to 1.5 X ULN; E 05. Glomerular filtration rate (GFR) less than equal to 30 mL/min/1.73m3 E 06. Positive hepatitis B surface antigen, or hepatitis C antibody or HIV antibody at screening E 07. FEV1 less than equal to 39percent or has lung score of 3 Other exclusion criteria E 08. Diagnosed with another malignancy (other than malignancy for which transplant was performed) within 3 years of enrolment E 09. Current enrolment or past participation within the last 30 days before signing of informed consent in any other clinical study involving an investigational study treatment or any other type of medical research E 10. Participation in a prior belumosudil clinical trial or any prior use of belumosudil E 11. Has a history of or ongoing drug or alcohol abuse E 12. Sensitivity to any of the study interventions, or components thereof, or drug or other allergy that, in the opinion of the Investigator, contraindicates participation in the study E 13. Individuals accommodated in an institution because of regulatory or legal order; prisoners or participants who are legally institutionalized E 14. 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 E 15. Participants are employees of the clinical study site or other individuals directly involved in the conduct of the study, or immediate family members of such individuals E 16. Any specific situation during study implementation/course that may raise ethical concerns.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| To evaluate the safety of belumosudil in Indian patients, 12 years and older, with chronic graft versus-host disease (cGVHD) after failure of at least two prior lines of systemic therapy.Timepoint: 24 weeks | — |
Secondary
| Measure | Time frame |
|---|---|
| To describe the efficacy of belumosudil in Indian patients, 12 years and older, with chronic graft versus-host disease (cGVHD) after failure of at least two prior lines of systemic therapy.Timepoint: 12 months | — |
Countries
India
Contacts
Public ContactMr Swapnil Sonje
Sanofi Healthcare India Pvt Limited (SHIPL)
Outcome results
None listed