Patient-reported, Health Economic and Psychosocial Outcomes in Friedreich Ataxia
Trial Parameters
Brief Summary
The PROFA study is an international, multi-centric observational and validation study to assess the patient-reported, psychosocial and economic outcomes of patients with Friedreich Ataxia (FA). Eligible patients will be recruited from six study centers in Germany, Austria and France. Patients will complete a baseline assessment via face-to-face interviews at the study centers and multiple momentary follow-up assessments via a mobile-health app at home daily to monthly for six months. Study results will gain essential and in-depth insights into the daily life of patients with FA.
Eligibility Criteria
Inclusion Criteria: * FA confirmed by molecular genetic testing * Ataxia severity of ≤30 points according to the Scale of the Assessment and Rating of Ataxia (SARA) * Access to a smartphone or tablet and able to operate the device * Older than 12 years Exclusion Criteria: * Lack of ability to give consent * Ataxia severity \>30 according to the Scale of the Assessment and Rating of Ataxia (SARA)