Long-Term Follow-Up Study of Risdiplam in Participants With Spinal Muscular Atrophy (SMA) (WeSMA)
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|ClinicalTrials.gov Identifier: NCT05232929|
Recruitment Status : Recruiting
First Posted : February 10, 2022
Last Update Posted : September 13, 2023
|Condition or disease||Intervention/treatment||Phase|
|Spinal Muscular Atrophy||Drug: Risdiplam||Phase 4|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||500 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Long-Term Follow-Up Study of Patients With Spinal Muscular Atrophy Receiving Risdiplam Treatment|
|Actual Study Start Date :||June 14, 2022|
|Estimated Primary Completion Date :||June 1, 2029|
|Estimated Study Completion Date :||June 1, 2029|
Participants will receive risdiplam prescribed based on clinician judgment, as per the Evrysdi® USPI.
Participants will receive risdiplam orally.
Other Name: Evrysdi®
- Number of Participants With Adverse Events (AEs), Adverse Events of Special Interest (AESIs), and Serious Adverse Events (SAEs) [ Time Frame: Up to 5 years ]An adverse event (AE) is any untoward medical occurrence in a participant administered a pharmaceutical product and which does not necessarily have to have a causal relationship with the treatment. An adverse event can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding, for example), symptom, or disease temporally associated with the use of a pharmaceutical product, whether or not considered related to the pharmaceutical product. Pre-existing conditions which worsen during a study are also considered as adverse events.
- Percentage of Participants Considered Improved on the Clinical Global Impression of Change (CGI-C) Scale [ Time Frame: Up to 5 years ]The Clinical Global Impression of Change (CGI-C) is used to score a clinician's impression of a participant's change in global health. The CGI-C is a single item measure of change in global health, using seven response options: "very much improved", "much improved", "minimally improved", "no change", "minimally worse", "much worse", and "very much worse". Participants considered as "improved" include responses of "very much improved, "much improved" and "minimally improved".
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05232929
|Contact: Reference Study ID Number: ML43702 https://forpatients.roche.com/||888-662-6728 (U.S. Only)||email@example.com|
|Study Director:||Clinical Trials||Hoffmann-La Roche|