SMA Type II and non-ambulatory Type III Musculoskeletal Diseases Spinal muscular atrophy
Conditions
Interventions
Approximately 70 survey participants will be recruited through participant associations within the five target countries. Potential participants will be contacted by e-mail with information about the
the second part of the survey will be a DCE consisting of a series of choices between hypothetical treatments varying in terms of treatment attributes, including treatment benefits, side effects, mode
Sponsors
Roche (United States)
Eligibility
Sex/Gender
All
Inclusion criteria
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
Exclusion criteria: Does not meet inclusion criteria
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Relative importance of treatment characteristics measured using a DCE survey at a single time point | — |
Secondary
| Measure | Time frame |
|---|---|
| There are no secondary outcome measures | — |
Countries
Belgium, Finland, Ireland, Netherlands, Portugal, United States of America
Contacts
Public ContactClinical Trials
Outcome results
None listed