NCT05943002Recruiting
Patient-reported, Health Economic and Psychosocial Outcomes in Friedreich Ataxia
Source: ClinicalTrials.gov · Last updated 1 Apr 2026
Brief summary (registry, in English)
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.
- Status (registry)
- Recruiting
- Study type
- Observational
- Enrollment
- 200 (estimated)
- First submitted
- 16 Mar 2023
- Start date
- Jun 2023
- Primary completion date
- Jun 2028
- Lead sponsor
- German Center for Neurodegenerative Diseases (DZNE)
- Diseases
- Friedreich ataxia
- NCT number
- NCT05943002
What you commit to
- Study type: Observational
- Planned participants: 200
- From Jun 2023 to the primary completion date, Jun 2028
- 6 sites in 3 countries
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Other
- Lack of ability to give consent
- Ataxia severity \>30 according to the Scale of the Assessment and Rating of Ataxia (SARA)
Who the study is looking for
- 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
Age in the registry: 12 years to - · Sex: All · Does not accept healthy volunteers
Sites (6)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| Klinik für Neurologie, Medizinische Universität Innsbruck Coordinator of this site? Confirm its status | Innsbruck | Austria | Recruiting |
| Department of Neurology, RWTH Aachen University Coordinator of this site? Confirm its status | Aachen | Germany | Recruiting |
| German Center for Neuro-degenerative Diseases (DZNE) Coordinator of this site? Confirm its status | Bonn | Germany | Recruiting |
| Friedrich-Baur-Institut an der Neurologischen Klinik und Poliklinik Coordinator of this site? Confirm its status | Münich | Germany | Recruiting |
| Neurologische Klinik und Hertie-Institut für Klinische Hirnforschung, Universitätsklinik Tübingen Coordinator of this site? Confirm its status | Tübingen | Germany | Not yet recruiting |
| Paris Brain Institute Coordinator of this site? Confirm its status | Paris | France | Recruiting |
This page is information, not medical advice. Discuss any trial with your doctor.