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 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 This substudy was withdrawn upon implementation of protocol version 4.

Condition

  • Spinal Muscular Atrophy

Eligibility

Eligible Ages
All ages
Eligible Sex
All
Accepts Healthy Volunteers
No

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])

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

ArmDescriptionAssigned Intervention
Experimental
Risdiplam 		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.
  • Drug: Risdiplam
    Risdiplam taken by participants as prescribed by their physician.
    Other names:
    • Evrysdi®

More Details

Status
Active, not recruiting
Sponsor
Genentech, Inc.

Study Contact