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

March 28, 2024 updated by: Genentech, Inc.

Long-Term Follow-Up Study of Patients With Spinal Muscular Atrophy Receiving Risdiplam Treatment

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.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Estimated)

500

Phase

  • Phase 4

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • Puerto Rico
      • Santurce, Puerto Rico, 00912
        • Recruiting
        • Instituto de Rehabilitación del Caribe
  • United States
    • Arizona
      • Phoenix, Arizona, United States, 85016
        • Recruiting
        • Phoenix Children's Hospital
      • Phoenix, Arizona, United States, 85013
        • Recruiting
        • Barrow Neurological Institute
    • Arkansas
      • Little Rock, Arkansas, United States, 72103
        • Recruiting
        • University of Arkansas for Medical Sciences
    • California
      • Loma Linda, California, United States, 92354
        • Recruiting
        • Loma Linda University Health Care; Clinical Trial Center
      • Los Angeles, California, United States, 90010
        • Recruiting
        • Children's Hospital Los Angeles
      • Madera, California, United States, 93636
        • Recruiting
        • Valley Children's Hospital
      • Orange, California, United States, 92868
        • Recruiting
        • University California - Irvine
      • Sacramento, California, United States, 95817
        • Recruiting
        • University of California Davis Medical Center
    • Colorado
      • Aurora, Colorado, United States, 80045
        • Recruiting
        • University of Colorado; Anschutz Medical Campus Department of Neurology
    • Florida
      • Orlando, Florida, United States, 32804
        • Recruiting
        • Advent Health Orlando
      • Orlando, Florida, United States, 32827
        • Recruiting
        • Nemour's Children's Hospital, Florida
    • Georgia
      • Atlanta, Georgia, United States, 30318
        • Recruiting
        • Rare Disease Research, LLC
    • Kansas
      • Kansas City, Kansas, United States, 66160
        • Recruiting
        • University of Kansas Medical Center
    • Kentucky
      • Lexington, Kentucky, United States, 40536
        • Recruiting
        • University of Kentucky Medical Center
      • Louisville, Kentucky, United States, 40202
        • Recruiting
        • Norton Children's Hospital
    • Massachusetts
      • Boston, Massachusetts, United States, 02114
        • Recruiting
        • Massachusetts General Hospital; Neurological Clinical Research Institute (NCRI)
    • Michigan
      • Ann Arbor, Michigan, United States, 48109
        • Recruiting
        • University of Michigan Pediatric Rehabilitation Center
      • Grand Rapids, Michigan, United States, 49503
        • Recruiting
        • Helen DeVos Children's Hospital at Spectrum Health
    • Minnesota
      • Minnetonka, Minnesota, United States, 55343
        • Recruiting
        • Gillette Children's Specialty Healthcare
      • Rochester, Minnesota, United States, 55905
        • Recruiting
        • Mayo Clinic
    • Missouri
      • Saint Louis, Missouri, United States, 63110
        • Recruiting
        • Washington University; Wash Uni. Sch. Of Med; Barnes-Jewish Hospital
    • New York
      • New York, New York, United States, 10003
        • Recruiting
        • NYU Hospital for Joint Diseases
      • New York, New York, United States, 10032
        • Recruiting
        • Columbia University Med Center
    • Ohio
      • Cincinnati, Ohio, United States, 45229
        • Recruiting
        • Cincinnati Childrens Hospital
    • Pennsylvania
      • Hershey, Pennsylvania, United States, 17033
        • Recruiting
        • Penn State Milton S. Hershey Medical Center
      • Philadelphia, Pennsylvania, United States, 19104
        • Recruiting
        • Childrens Hospital of Philadelphia
    • Texas
      • Austin, Texas, United States, 78723
        • Recruiting
        • University of Texas at Austin Health sciences, Dell Medical School
      • Denton, Texas, United States, 76208
        • Recruiting
        • Neurology & Neuromuscular Care Center
      • Round Rock, Texas, United States, 78681
        • Recruiting
        • Central Texas Neurology Consultants
      • San Antonio, Texas, United States, 78229
        • Recruiting
        • Methodist Children's Hospital of South Texas
    • Utah
      • Salt Lake City, Utah, United States, 84108
        • Recruiting
        • University of Utah
    • Virginia
      • Charlottesville, Virginia, United States, 22903
        • Recruiting
        • University of Virginia Children?s Hospital; Developmental
    • Washington
      • Tacoma, Washington, United States, 98405
        • Recruiting
        • MultiCare Health System Institute for Research and Innovation
    • West Virginia
      • Morgantown, West Virginia, United States, 26508
        • Recruiting
        • UBC (Remote Coordinating Center, no physical facility)
    • Wisconsin
      • Milwaukee, Wisconsin, United States, 53201
        • Recruiting
        • Childrens Hospital of Wisconsin

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

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])

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / 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 Names:
  • Evrysdi®

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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.
Up to 5 years

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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".
Up to 5 years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Genentech, Inc.

Investigators

  • Study Director: Clinical Trials, Hoffmann-La Roche

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

June 14, 2022

Primary Completion (Estimated)

June 1, 2029

Study Completion (Estimated)

June 1, 2029

Study Registration Dates

First Submitted

January 31, 2022

First Submitted That Met QC Criteria

January 31, 2022

First Posted (Actual)

February 10, 2022

Study Record Updates

Last Update Posted (Actual)

April 1, 2024

Last Update Submitted That Met QC Criteria

March 28, 2024

Last Verified

March 1, 2024

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • ML43702

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

YES

IPD Plan Description

Qualified researchers may request access to individual patient level data through the clinical study data request platform (www.vivli.org). Further details on Roche's criteria for eligible studies are available here (https://vivli.org/ourmember/roche/). 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 (https://www.roche.com/research_and_development/who_we_are_how_we_work/clinical_trials/our_commitment_to_data_sharing.htm).

Drug and device information, study documents

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

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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