Assessing health and economic outcomes in Friedreich Ataxia patients
Patient-reported, Health Economic and Psychosocial Outcomes in Friedreich Ataxia
This study is trying to understand how Friedreich Ataxia affects the lives and finances of patients by using a mobile app to gather information over six months.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 200 (estimated) |
| Ages | 12 Years and up |
| Sex | All |
| Sponsor | German Center for Neurodegenerative Diseases (DZNE) Academic / other |
| Locations | 6 sites (Innsbruck and 5 other locations) |
| Trial ID | NCT05943002 on ClinicalTrials.gov |
What this trial studies
The PROFA study is an international observational study aimed at understanding the psychosocial and economic impacts of Friedreich Ataxia (FA) on patients. It will recruit participants from six centers in Germany, Austria, and France, who will complete initial assessments through face-to-face interviews and ongoing evaluations via a mobile health app over six months. The study focuses on the feasibility of using the app for real-time data collection, healthcare costs, health-related quality of life, and the psychosocial effects of FA. By gathering comprehensive data, the study seeks to improve treatment and care for FA patients and their families.
Who should consider this trial
Good fit: Ideal candidates for this study are individuals aged 12 and older with a confirmed diagnosis of Friedreich Ataxia and a severity score of 30 or less on the Scale of the Assessment and Rating of Ataxia.
Not a fit: Patients with a severity score greater than 30 on the Scale of the Assessment and Rating of Ataxia or those unable to provide consent may not benefit from this study.
Why it matters
Potential benefit: If successful, this study could provide valuable insights that lead to improved treatment strategies and support for patients with Friedreich Ataxia.
How similar studies have performed: Previous studies on Friedreich Ataxia have been limited in scope, making this approach novel and potentially groundbreaking in understanding the condition's broader impacts.
Eligibility criteria
Show full inclusion / exclusion 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)
Where this trial is running
Innsbruck and 5 other locations
- Klinik für Neurologie, Medizinische Universität Innsbruck — Innsbruck, Austria (Recruiting)
- Paris Brain Institute — Paris, France (Recruiting)
- Department of Neurology, RWTH Aachen University — Aachen, Germany (Recruiting)
- German Center for Neuro-degenerative Diseases (DZNE) — Bonn, Germany (Recruiting)
- Friedrich-Baur-Institut an der Neurologischen Klinik und Poliklinik — Münich, Germany (Recruiting)
- Neurologische Klinik und Hertie-Institut für Klinische Hirnforschung, Universitätsklinik Tübingen — Tübingen, Germany (Not_yet_recruiting)
Study contacts
- Principal investigator: Bernhard Michalowsky, PD Dr. — German Center for Neurodegenerative Diseases (DZNE) Rostock/ Greifswald, Germany
- Study coordinator: Bernhard Michalowsky, PD Dr.
- Email: bernhard.michalowsky@dzne.de
- Phone: +49 3834 868530
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.