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

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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)

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

Trial contacts and locations

0

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Central trial contact

Reference Study ID Number: ML43702 https://forpatients.roche.com/

Data sourced from clinicaltrials.gov

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