Online survey study to explore patient and caregiver preferences for different spinal muscular atrophy treatments

Not Applicable

Completed

• Conditions: Musculoskeletal Diseases; Spinal muscular atrophy; SMA Type II and non-ambulatory Type III

• Registration Number: ISRCTN11372270
• Lead Sponsor: Roche (United States)

Brief Summary

2021 Results article in https://doi.org/10.1007/s40273-021-01118-2 (added 18/07/2022)

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2021-01-15
• Study Completion: 2021-04-30
• Last Update Posted: 5 August 2022

Recruitment & Eligibility

• Status: Completed
• Sex: All
• Target Recruitment: 70

Inclusion Criteria

1. A patient self-reported or caregiver proxy-reported diagnosis of SMA Type II: age of onset between 7 and 18 months OR SMA Type III: age of onset 18+ months and 17 years
2. Non-ambulatory, defined as unable to walk more than ten steps without a form of assistance
3. Adult patients aged 18+ OR caregivers of patients aged <18 years
4. Residents of the Netherlands, Finland, Ireland, Portugal or Belgium
5. Willing and able to give their informed consent

Exclusion Criteria

Does not meet inclusion criteria

Study & Design

• Study Type: Observational
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Relative importance of treatment characteristics measured using a DCE survey at a single time point

Secondary Outcome Measures

Name	Time	Method
There are no secondary outcome measures