Long-term Follow-up Study of Risdiplam in Participants With Spinal Muscular Atrophy (SMA)
Purpose
A multi-center, longitudinal, prospective, non-comparative study to investigate the long-term safety and effectiveness of risdiplam, prescribed based on clinician judgment as per the Evrysdi® U.S. Package Insert (USPI) in adult and pediatric participants with SMA. In this study, participants will be followed for the duration of the study or until withdrawal of consent, loss to follow-up, or death. Participants who discontinue risdiplam may still remain in the study, if they agree to continue participating in the follow-up assessments.
Condition
- Spinal Muscular Atrophy
Eligibility
- Eligible Ages
- All ages
- Eligible Genders
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- Clinical diagnosis of SMA - Prescribed or continued risdiplam based on clinical judgment of prescriber, as per the Evrysdi® USPI, after U.S. FDA approval (07 August 2020)
Exclusion Criteria
- Hypersensitivity to risdiplam - Participated in a registrational trial for risdiplam (i.e., Firefish [NCT02913482], Sunfish [NCT02908685], Jewelfish [NCT03032172], and Rainbowfish [NCT03779334])
Study Design
- Phase
- Phase 4
- Study Type
- Interventional
- Allocation
- N/A
- Intervention Model
- Single Group Assignment
- Primary Purpose
- Treatment
- Masking
- None (Open Label)
Arm Groups
| Arm | Description | Assigned Intervention |
|---|---|---|
|
Experimental Risdiplam |
Participants will receive risdiplam prescribed based on clinician judgment, as per the Evrysdi® USPI. |
|
Recruiting Locations
Medstar Georgetown University Hospital
Washington, District of Columbia 20007
Washington, District of Columbia 20007
More Details
- Status
- Recruiting
- Sponsor
- Genentech, Inc.
Study Contact
Reference Study ID Number: ML43702 https://forpatients.roche.com/888-662-6728 (U.S. Only)
global-roche-genentech-trials@gene.com