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

Study Identifier:
ML43702
ClinicalTrials.gov Identifier:
NCT05232929
EudraCT Identifier:
N/A
EU CT Number:
N/A
Sponsor:
Genentech, Inc.
Active, not recruiting

Study Details

Medical Condition
  • All Spinal Muscular Atrophies
Study Drug
  • Drug: Risdiplam
Date
Mar 2022 - Dec 2026
Phase 1
Phase 2
Phase 3
Phase 4
Patient Requirements
Sex: Female & Male
Age: N/A
Requirements Information
Inclusion and Exclusion Criteria
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 \[NCT03779334\])

Protocol Summary

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 This substudy was withdrawn upon implementation of protocol version 4.

Trial Locations

Location
Status
Location
Barrow Neurological Institute
Phoenix, Arizona, United States, 85013
Status
N/A
Location
Phoenix Children's Hospital
Phoenix, Arizona, United States, 85016
Status
N/A
Location
University of Arkansas for Medical Sciences
Little Rock, Arkansas, United States, 72103
Status
N/A
Location
Loma Linda University Health Care
Loma Linda, California, United States, 92354
Status
N/A
Location
Children's Hospital Los Angeles
Los Angeles, California, United States, 90010
Status
N/A
Location
Valley Children's Hospital
Madera, California, United States, 93636
Status
N/A
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