Status:
ACTIVE_NOT_RECRUITING
Long-term Follow-up Study of Risdiplam in Participants With Spinal Muscular Atrophy (SMA)
Lead Sponsor:
Genentech, Inc.
Conditions:
Spinal Muscular Atrophy
Eligibility:
All Genders
Phase:
PHASE4
Brief 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. Pac...
Eligibility Criteria
Inclusion
- Clinical diagnosis of SMA
- Prescribed or continued risdiplam based on clinical judgment of prescriber, as per the Evrysdi® USPI, after U.S. FDA approval (07 August 2020)
- Sub study:
- Participants in the main study (ML43702) are eligible to be included in the sub study only if all of the following criteria apply:
- Age ≥ 10 years at the time of signing Informed Consent Form
- Willingness and ability to use smartphone technology
- Fluency in English (written and spoken as per the judgment of the investigator)
- Willingness and ability to complete all aspects of the sub study, including respiration and swallowing measurements using respiratory inductance plethysmography (RIP) belts and surface electromyography (sEMGs)
- Hammersmith Functional Motor Scale-Expanded (HFMSE) \> 10
- Functional Oral Intake Scale (FOIS) \>1
- Willingness to be video recorded during in-clinic SMA-DAT tasks and ADAM sensor assessments and training
- Availability of a caregiver who is willing to participate throughout this sub study
Exclusion
- Hypersensitivity to risdiplam
- Participated in a registrational trial for risdiplam (i.e., Firefish \[NCT02913482\], Sunfish \[NCT02908685\], Jewelfish \[NCT03032172\], and Rainbowfish \[NCT03779334\])
- Sub study:
- Potential participants will be excluded from the sub study if they meet any of the following criteria:
- Current respiratory infection that, in the opinion of the investigator, would interfere with the conduct of the sub study
- History or known presence of any significant psychiatric disorder such as schizophrenia, bipolar disorder, or substance use disorders
- Current active clinically significant anxiety or depressive disorder, as judged by the investigator, that is likely to impede a participant's ability to participate in the sub study
- Wearing a pacemaker (due to incompatibility with the ADAM sensor)
- Inability to tolerate the performance of sub study procedures
Key Trial Info
Start Date :
March 29 2022
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
December 31 2026
Estimated Enrollment :
402 Patients enrolled
Trial Details
Trial ID
NCT05232929
Start Date
March 29 2022
End Date
December 31 2026
Last Update
November 28 2025
Active Locations (39)
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1
Barrow Neurological Institute
Phoenix, Arizona, United States, 85013
2
Phoenix Children's Hospital
Phoenix, Arizona, United States, 85016
3
University of Arkansas for Medical Sciences
Little Rock, Arkansas, United States, 72103
4
Loma Linda University Health Care
Loma Linda, California, United States, 92354