A study to observe if Risdiplam treatment is safe and effective in Spinal Muscular Atrophy Patients in India.
- Conditions
- Health Condition 1: G129- Spinal muscular atrophy, unspecified
- Registration Number
- CTRI/2021/10/037251
- Lead Sponsor
- Roche Products India Pvt Ltd
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Open to Recruitment
- Sex
- Not specified
- Target Recruitment
- 0
1. Have signed the informed consent/assent (as applicable). In the case of minors, a parent or authorized adult will be required to sign the Informed Consent Form on behalf of the patient. The informed consent/assent will be obtained in accordance with local requirements
2. Patients aged =2 months at enrollment (for preterm infants this is the corrected age)
3. Confirmed diagnosis of 5q autosomal recessive SMA including genetic confirmation of homozygous deletion or compound heterozygosity that is predictive of loss of function of the survival motor neuron 1 (SMN1) gene and/or SMN2 gene
4. Clinical history, signs or symptoms attributable to SMA in patients of age 2 months and older
5. Patients who have been prescribed and received at least one dose of risdiplam for SMA.
Not provided
Study & Design
- Study Type
- PMS
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method The primary objective of the study is to assess the safety and effectiveness of risdiplam for the treatment of SMA in patients of age 2 months and older in India.Timepoint: Three years
- Secondary Outcome Measures
Name Time Method To describe the profile of SMA patients (2 months of age and older) in India based on demographics and familial, clinical, and genetic characteristics.Timepoint: Three years