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

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 This substudy was withdrawn upon implementation of protocol version 4.

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This study is active but is not currently recruiting participants.

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Key information

Conditions

Spinal Muscular Atrophy Central Nervous System Diseases Motor Neuron Disease Muscular Atrophy, Spinal Nervous System Diseases Neurodegenerative Diseases Neuromuscular Diseases Spinal Cord Diseases

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 4

Primary location

Instituto de Rehabilitación del Caribe, Santurce, Puerto Rico

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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])

Treatment and study plan

Risdiplam

Drug

Risdiplam taken by participants as prescribed by their physician.

Other names: Evrysdi®

Primary outcomes

Number of Participants With Adverse Events (AEs), Adverse Events of Special Interest (AESIs), and Serious Adverse Events (SAEs)

Time frame: From enrollment up to the duration of the study or until withdrawal of consent, loss to follow-up, or death (up to approximately 24 months)

An AE is any untoward medical occurrence in a participant administered a pharmaceutical product and which does not necessarily have to have a causal relationship with the treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding, for example), symptom, or disease temporally associated with the use of a pharmaceutical product, whether or not considered related to the pharmaceutical product. Pre-existing conditions which worsen during a study are also considered as AE.

Secondary outcomes

Percentage of Participants Considered Improved on the Clinical Global Impression of Change (CGI-C) Scale

Time frame: From enrollment up to the duration of the study or until withdrawal of consent, loss to follow-up, or death (up to approximately 24 months)

The CGI-C is used to score a clinician's impression of a participant's change in global health. The CGI-C is a single item measure of change in global health, using seven response options: "very much improved", "much improved", "minimally improved", "no change", "minimally worse", "much worse", and "very much worse". Participants considered as "improved" include responses of "very much improved, "much improved" and "minimally improved".

Sponsors and collaborators

Lead sponsor

Genentech, Inc.

Industry

Registry information

Official study title

Long-term Follow-up Study of Patients With Spinal Muscular Atrophy Receiving Risdiplam Treatment

Acronym: WeSMA

Important dates

Study start: 2022
Primary completion: 2026
Study completion: 2026
First posted: Feb 10, 2022
Registry last updated: Jul 16, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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