Long-term Follow-up Study of Risdiplam in Participants With Spinal Muscular Atrophy (SMA) (WeSMA)

Genentech

Status and phase: Active, not recruiting; Phase 4

Conditions: Spinal Muscular Atrophy

Treatments: Drug: Risdiplam

Study type: Interventional

Funder types: Industry

Identifiers

NCT05232929

ML43702

Details and patient eligibility

About

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 Information (USPI) in adult and pediatric participants with SMA. In this study, participants will be followed for approximately 24 months from enrollment 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.

An optional sub-study was planned to assess the feasibility, acceptability, and adherence of remote assessment of motor and bulbar functions in participants with SMA using wearable and smartphone-based biosensors. This substudy was withdrawn upon implementation of protocol version 4.

Enrollment: 404 patients

Sex: All

Volunteers: No Healthy Volunteers

Inclusion criteria

• Provide informed consent or assent when appropriate, as determined by the participant's age and individual site and local standards
• Prescribed or continued risdiplam based on clinical judgment of prescriber, as per the risdiplam (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])

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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404 participants in 1 patient group

Risdiplam

Experimental group

Description:

Participants who are taking risdiplam prescribed based on clinician judgment, as per the Evrysdi® USPI are enrolled in this study. A Remote Virtual Site option is available in the study allowing enrollment of participants with SMA who are prescribed risdiplam treatment even if their physician is not a participating investigator. A central investigator will be responsible for data collection and data entry for participants who do not have a participating investigator.

Treatment:

Drug: Risdiplam