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

Primary Purpose

Spinal Muscular Atrophy

Status
Recruiting
Phase
Phase 4
Locations
International
Study Type
Interventional
Intervention
Risdiplam
Sponsored by
Genentech, Inc.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Spinal Muscular Atrophy

Eligibility Criteria

undefined - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

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])

Sites / Locations

  • Barrow Neurological InstituteRecruiting
  • Phoenix Children's HospitalRecruiting
  • University of Arkansas for Medical SciencesRecruiting
  • Children's Hospital Los AngelesRecruiting
  • Valley Children's HospitalRecruiting
  • University California - IrvineRecruiting
  • University of Colorado; Anschutz Medical Campus Department of NeurologyRecruiting
  • Advent Health OrlandoRecruiting
  • Rare Disease Research, LLCRecruiting
  • University of Kansas Medical CenterRecruiting
  • Norton Children's HospitalRecruiting
  • Helen DeVos Children's Hospital at Spectrum HealthRecruiting
  • Gillette Children's Specialty HealthcareRecruiting
  • Mayo ClinicRecruiting
  • Washington University; Wash Uni. Sch. Of Med; Barnes-Jewish HospitalRecruiting
  • NYU Hospital for Joint DiseasesRecruiting
  • Columbia University Med CenterRecruiting
  • Cincinnati Childrens HospitalRecruiting
  • Penn State Milton S. Hershey Medical CenterRecruiting
  • Childrens Hospital of PhiladelphiaRecruiting
  • Neurology & Neuromuscular Care CenterRecruiting
  • Central Texas Neurology ConsultantsRecruiting
  • University Of UtahRecruiting
  • University of Virginia Children?s Hospital; DevelopmentalRecruiting
  • MultiCare Health System Institute for Research and InnovationRecruiting
  • UBC (Remote Coordinating Center, no physical facility)Recruiting
  • Childrens Hospital of WisconsinRecruiting
  • Instituto de Rehabilitación del CaribeRecruiting

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Risdiplam

Arm Description

Participants will receive risdiplam prescribed based on clinician judgment, as per the Evrysdi® USPI.

Outcomes

Primary Outcome Measures

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.

Secondary Outcome Measures

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".

Full Information

First Posted
January 31, 2022
Last Updated
October 5, 2023
Sponsor
Genentech, Inc.
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1. Study Identification

Unique Protocol Identification Number
NCT05232929
Brief Title
Long-Term Follow-Up Study of Risdiplam in Participants With Spinal Muscular Atrophy (SMA)
Acronym
WeSMA
Official Title
Long-Term Follow-Up Study of Patients With Spinal Muscular Atrophy Receiving Risdiplam Treatment
Study Type
Interventional

2. Study Status

Record Verification Date
October 2023
Overall Recruitment Status
Recruiting
Study Start Date
June 14, 2022 (Actual)
Primary Completion Date
June 1, 2029 (Anticipated)
Study Completion Date
June 1, 2029 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Genentech, Inc.

4. Oversight

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

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Spinal Muscular Atrophy

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 4
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
500 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Risdiplam
Arm Type
Experimental
Arm Description
Participants will receive risdiplam prescribed based on clinician judgment, as per the Evrysdi® USPI.
Intervention Type
Drug
Intervention Name(s)
Risdiplam
Other Intervention Name(s)
Evrysdi®
Intervention Description
Participants will receive risdiplam orally.
Primary Outcome Measure Information:
Title
Number of Participants With Adverse Events (AEs), Adverse Events of Special Interest (AESIs), and Serious Adverse Events (SAEs)
Description
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.
Time Frame
Up to 5 years
Secondary Outcome Measure Information:
Title
Percentage of Participants Considered Improved on the Clinical Global Impression of Change (CGI-C) Scale
Description
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".
Time Frame
Up to 5 years

10. Eligibility

Sex
All
Accepts Healthy Volunteers
No
Eligibility Criteria
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])
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Reference Study ID Number: ML43702 https://forpatients.roche.com/
Phone
888-662-6728 (U.S. Only)
Email
global-roche-genentech-trials@gene.com
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Clinical Trials
Organizational Affiliation
Hoffmann-La Roche
Official's Role
Study Director
Facility Information:
Facility Name
Barrow Neurological Institute
City
Phoenix
State/Province
Arizona
ZIP/Postal Code
85013
Country
United States
Individual Site Status
Recruiting
Facility Name
Phoenix Children's Hospital
City
Phoenix
State/Province
Arizona
ZIP/Postal Code
85016
Country
United States
Individual Site Status
Recruiting
Facility Name
University of Arkansas for Medical Sciences
City
Little Rock
State/Province
Arkansas
ZIP/Postal Code
72103
Country
United States
Individual Site Status
Recruiting
Facility Name
Children's Hospital Los Angeles
City
Los Angeles
State/Province
California
ZIP/Postal Code
90010
Country
United States
Individual Site Status
Recruiting
Facility Name
Valley Children's Hospital
City
Madera
State/Province
California
ZIP/Postal Code
93636
Country
United States
Individual Site Status
Recruiting
Facility Name
University California - Irvine
City
Orange
State/Province
California
ZIP/Postal Code
92868
Country
United States
Individual Site Status
Recruiting
Facility Name
University of Colorado; Anschutz Medical Campus Department of Neurology
City
Aurora
State/Province
Colorado
ZIP/Postal Code
80045
Country
United States
Individual Site Status
Recruiting
Facility Name
Advent Health Orlando
City
Orlando
State/Province
Florida
ZIP/Postal Code
32804
Country
United States
Individual Site Status
Recruiting
Facility Name
Rare Disease Research, LLC
City
Atlanta
State/Province
Georgia
ZIP/Postal Code
30318
Country
United States
Individual Site Status
Recruiting
Facility Name
University of Kansas Medical Center
City
Kansas City
State/Province
Kansas
ZIP/Postal Code
66160
Country
United States
Individual Site Status
Recruiting
Facility Name
Norton Children's Hospital
City
Louisville
State/Province
Kentucky
ZIP/Postal Code
40202
Country
United States
Individual Site Status
Recruiting
Facility Name
Helen DeVos Children's Hospital at Spectrum Health
City
Grand Rapids
State/Province
Michigan
ZIP/Postal Code
49503
Country
United States
Individual Site Status
Recruiting
Facility Name
Gillette Children's Specialty Healthcare
City
Minnetonka
State/Province
Minnesota
ZIP/Postal Code
55343
Country
United States
Individual Site Status
Recruiting
Facility Name
Mayo Clinic
City
Rochester
State/Province
Minnesota
ZIP/Postal Code
55905
Country
United States
Individual Site Status
Recruiting
Facility Name
Washington University; Wash Uni. Sch. Of Med; Barnes-Jewish Hospital
City
Saint Louis
State/Province
Missouri
ZIP/Postal Code
63110
Country
United States
Individual Site Status
Recruiting
Facility Name
NYU Hospital for Joint Diseases
City
New York
State/Province
New York
ZIP/Postal Code
10003
Country
United States
Individual Site Status
Recruiting
Facility Name
Columbia University Med Center
City
New York
State/Province
New York
ZIP/Postal Code
10032
Country
United States
Individual Site Status
Recruiting
Facility Name
Cincinnati Childrens Hospital
City
Cincinnati
State/Province
Ohio
ZIP/Postal Code
45229
Country
United States
Individual Site Status
Recruiting
Facility Name
Penn State Milton S. Hershey Medical Center
City
Hershey
State/Province
Pennsylvania
ZIP/Postal Code
17033
Country
United States
Individual Site Status
Recruiting
Facility Name
Childrens Hospital of Philadelphia
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19104
Country
United States
Individual Site Status
Recruiting
Facility Name
Neurology & Neuromuscular Care Center
City
Denton
State/Province
Texas
ZIP/Postal Code
76208
Country
United States
Individual Site Status
Recruiting
Facility Name
Central Texas Neurology Consultants
City
Round Rock
State/Province
Texas
ZIP/Postal Code
78681
Country
United States
Individual Site Status
Recruiting
Facility Name
University Of Utah
City
Salt Lake City
State/Province
Utah
ZIP/Postal Code
84108
Country
United States
Individual Site Status
Recruiting
Facility Name
University of Virginia Children?s Hospital; Developmental
City
Charlottesville
State/Province
Virginia
ZIP/Postal Code
22903
Country
United States
Individual Site Status
Recruiting
Facility Name
MultiCare Health System Institute for Research and Innovation
City
Tacoma
State/Province
Washington
ZIP/Postal Code
98405
Country
United States
Individual Site Status
Recruiting
Facility Name
UBC (Remote Coordinating Center, no physical facility)
City
Morgantown
State/Province
West Virginia
ZIP/Postal Code
26508
Country
United States
Individual Site Status
Recruiting
Facility Name
Childrens Hospital of Wisconsin
City
Milwaukee
State/Province
Wisconsin
ZIP/Postal Code
53201
Country
United States
Individual Site Status
Recruiting
Facility Name
Instituto de Rehabilitación del Caribe
City
Santurce
ZIP/Postal Code
00912
Country
Puerto Rico
Individual Site Status
Recruiting

12. IPD Sharing Statement

Plan to Share IPD
Yes
IPD Sharing 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).

Learn more about this trial

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

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