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Long-Term Follow-Up Study of Risdiplam in Participants With Spinal Muscular Atrophy (SMA) (WeSMA)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. Identifier: NCT05232929
Recruitment Status : Recruiting
First Posted : February 10, 2022
Last Update Posted : September 13, 2023
Information provided by (Responsible Party):
Genentech, Inc.

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. Package Insert (USPI) in adult and pediatric participants with spinal muscular atrophy (SMA). In this study, participants will be followed for up to 5 years 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.

Condition or disease Intervention/treatment Phase
Spinal Muscular Atrophy Drug: Risdiplam Phase 4

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 500 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Long-Term Follow-Up Study of Patients With Spinal Muscular Atrophy Receiving Risdiplam Treatment
Actual Study Start Date : June 14, 2022
Estimated Primary Completion Date : June 1, 2029
Estimated Study Completion Date : June 1, 2029

Arm Intervention/treatment
Experimental: Risdiplam
Participants will receive risdiplam prescribed based on clinician judgment, as per the Evrysdi® USPI.
Drug: Risdiplam
Participants will receive risdiplam orally.
Other Name: Evrysdi®

Primary Outcome Measures :
  1. Number of Participants With Adverse Events (AEs), Adverse Events of Special Interest (AESIs), and Serious Adverse Events (SAEs) [ Time Frame: Up to 5 years ]
    An adverse event (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 adverse event 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 adverse events.

Secondary Outcome Measures :
  1. Percentage of Participants Considered Improved on the Clinical Global Impression of Change (CGI-C) Scale [ Time Frame: Up to 5 years ]
    The Clinical Global Impression of Change (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".

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Confirmed diagnosis of 5q-autosomal recessive SMA
  • Prescribed or continued risdiplam based on clinical judgment of prescriber, as per the 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])

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT05232929

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Contact: Reference Study ID Number: ML43702 888-662-6728 (U.S. Only)

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Sponsors and Collaborators
Genentech, Inc.
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Study Director: Clinical Trials Hoffmann-La Roche
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Responsible Party: Genentech, Inc. Identifier: NCT05232929    
Other Study ID Numbers: ML43702
First Posted: February 10, 2022    Key Record Dates
Last Update Posted: September 13, 2023
Last Verified: September 2023
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
Plan Description: Qualified researchers may request access to individual patient level data through the clinical study data request platform ( Further details on Roche's criteria for eligible studies are available here ( For further details on Roche's Global Policy on the Sharing of Clinical Information and how to request access to related clinical study documents, see here (

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Product Manufactured in and Exported from the U.S.: No
Additional relevant MeSH terms:
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Muscular Atrophy
Muscular Atrophy, Spinal
Pathological Conditions, Anatomical
Neuromuscular Manifestations
Neurologic Manifestations
Nervous System Diseases
Spinal Cord Diseases
Central Nervous System Diseases
Motor Neuron Disease
Neurodegenerative Diseases
Neuromuscular Diseases
Neuromuscular Agents
Peripheral Nervous System Agents
Physiological Effects of Drugs