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

Spinal Muscular Atrophy Clinical Trial

— WeSMA  

Official title:

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05232929
• Other study ID #: ML43702
• Status: Recruiting
• Phase: Phase 4
• Start date: June 14, 2022
• Est. completion date: June 1, 2029

Study information

• Verified date: May 2024
• Source: Genentech, Inc.
• Contact: Reference Study ID Number: ML43702 https://forpatients.roche.com
• Phone: 888-662-6728 (U.S. Only)
• Email: global-roche-genentech-trials[@]gene.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 500
• Est. completion date: June 1, 2029
• Est. primary completion date: June 1, 2029
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A and older

Eligibility

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])

Related Conditions & MeSH terms

• Atrophy
• Muscular Atrophy
• Muscular Atrophy, Spinal
• Spinal Muscular Atrophy

Intervention

Drug:
• Risdiplam
Participants will receive risdiplam orally.

Locations

Country	Name	City	State
Puerto Rico	Instituto de Rehabilitación del Caribe	Santurce
United States	University of Michigan Pediatric Rehabilitation Center	Ann Arbor	Michigan
United States	Rare Disease Research, LLC	Atlanta	Georgia
United States	University of Colorado; Anschutz Medical Campus Department of Neurology	Aurora	Colorado
United States	University of Texas at Austin Health sciences, Dell Medical School	Austin	Texas
United States	Massachusetts General Hospital; Neurological Clinical Research Institute (NCRI)	Boston	Massachusetts
United States	University of Virginia Children?s Hospital; Developmental	Charlottesville	Virginia
United States	Cincinnati Childrens Hospital	Cincinnati	Ohio
United States	Neurology & Neuromuscular Care Center	Denton	Texas
United States	Helen DeVos Children's Hospital at Spectrum Health	Grand Rapids	Michigan
United States	Penn State Milton S. Hershey Medical Center	Hershey	Pennsylvania
United States	University of Kansas Medical Center	Kansas City	Kansas
United States	University of Kentucky Medical Center	Lexington	Kentucky
United States	University of Arkansas for Medical Sciences	Little Rock	Arkansas
United States	Loma Linda University Health Care; Clinical Trial Center	Loma Linda	California
United States	Children's Hospital Los Angeles	Los Angeles	California
United States	Norton Children's Hospital	Louisville	Kentucky
United States	Valley Children's Hospital	Madera	California
United States	Childrens Hospital of Wisconsin	Milwaukee	Wisconsin
United States	Gillette Children's Specialty Healthcare	Minnetonka	Minnesota
United States	UBC (Remote Coordinating Center, no physical facility)	Morgantown	West Virginia
United States	Columbia University Med Center	New York	New York
United States	NYU Hospital for Joint Diseases	New York	New York
United States	University California - Irvine	Orange	California
United States	Advent Health Orlando	Orlando	Florida
United States	Nemour's Children's Hospital, Florida	Orlando	Florida
United States	Childrens Hospital of Philadelphia	Philadelphia	Pennsylvania
United States	Barrow Neurological Institute	Phoenix	Arizona
United States	Phoenix Children's Hospital	Phoenix	Arizona
United States	Mayo Clinic	Rochester	Minnesota
United States	Central Texas Neurology Consultants	Round Rock	Texas
United States	University of California Davis Medical Center	Sacramento	California
United States	Washington University; Wash Uni. Sch. Of Med; Barnes-Jewish Hospital	Saint Louis	Missouri
United States	University Of Utah	Salt Lake City	Utah
United States	Methodist Children's Hospital of South Texas	San Antonio	Texas
United States	MultiCare Health System Institute for Research and Innovation	Tacoma	Washington
United States	Children's National Hospital	Washington	District of Columbia
United States	Medstar Georgetown University Hospital	Washington	District of Columbia

Sponsors (1)

Lead Sponsor	Collaborator
Genentech, Inc.

Countries where clinical trial is conducted

United States,  Puerto Rico, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Number of Participants With Adverse Events (AEs), Adverse Events of Special Interest (AESIs), and Serious Adverse Events (SAEs)	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	Percentage of Participants Considered Improved on the Clinical Global Impression of Change (CGI-C) Scale	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