Studies daily life and feelings of people with Friedreich ataxia
Follows people with Friedreich ataxia through daily life using questionnaires and a phone app.
Plain-language title generated from the registry record
Registry title: Patient-reported, Health Economic and Psychosocial Outcomes in Friedreich Ataxia
Key facts
- Where
- Paris, FranceSee the site
Where in France
- Paris Brain InstituteParis, FranceTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- German Center for Neurodegenerative Diseases (DZNE)
- Trial code
- NCT05943002
- Record updated
- 1 April 2026
- Original description
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.Read full original descriptionShow less
Patient-reported, Health Economic and Psychosocial Outcomes in Friedreich Ataxia
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.
Inclusion: 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: Lack of ability to give consent; Ataxia severity >30 according to the Scale of the Assessment and Rating of Ataxia (SARA).