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

Launched by GENENTECH, INC. · Jan 31, 2022

Keywords

ClinConnect Summary

This clinical trial is studying the long-term safety and effectiveness of a medication called risdiplam for people with spinal muscular atrophy (SMA). SMA is a genetic condition that affects muscle strength and movement. Participants in this study, which is open to both adults and children, will be followed for up to 5 years to see how well the medication works over time. To be eligible, individuals must have a confirmed diagnosis of a specific type of SMA and must have been prescribed risdiplam based on their doctor’s recommendation after it was approved by the FDA in August 2020.

During the study, participants will regularly check in with researchers to assess their health and how they are responding to the treatment. Even if someone stops taking risdiplam, they can still participate in the study as long as they agree to continue with the follow-up assessments. This trial is currently recruiting participants, and it aims to gather valuable information about the medication to help improve care for those living with SMA.

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• • 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 Criteria:
• • 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