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

Genentech

Status and phase: Enrolling; 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 Insert (USPI) in adult and pediatric participants with spinal muscular atrophy (SMA). In this study, participants will be followed for up to 5 years 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.

Enrollment: 500 estimated patients

Sex: All

Volunteers: No Healthy Volunteers

Inclusion criteria

• Confirmed diagnosis of 5q-autosomal recessive 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])

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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500 participants in 1 patient group

Risdiplam

Experimental group

Description:

Participants will receive risdiplam prescribed based on clinician judgment, as per the Evrysdi® USPI.

Treatment:

Drug: Risdiplam