Patient-reported, Health Economic and Psychosocial Outcomes in Friedreich Ataxia

Launched by GERMAN CENTER FOR NEURODEGENERATIVE DISEASES (DZNE) · Jul 5, 2023

Keywords

ClinConnect Summary

The PROFA study is a research project looking into the experiences of people living with Friedreich Ataxia (FA), a rare genetic condition that affects movement and coordination. The goal of the study is to understand how FA impacts daily life, including mental health and economic factors. To participate, you must be diagnosed with FA through genetic testing, have a certain level of movement difficulty, and be over the age of 12. You will need access to a smartphone or tablet to take part in the study.

If you join the study, you will first meet with researchers for an interview at one of the study centers in Germany, Austria, or France. After that, you’ll use a mobile app to share your experiences daily to monthly for six months. This research is important because it will provide valuable insights into the challenges faced by individuals with FA, helping to improve support and resources for those affected by the condition. The study is currently recruiting participants, and everyone is welcome to apply.

Gender

ALL

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)