The main criteria for participation
The UNIFAI study is recruiting 3,000 participants of all ages with Friedreich’s ataxia in several countries around the world, including France, including those included in the patient registry of the European Consortium for Translational Studies of Friedreich’s Ataxia (EFACTS), as well as healthy volunteers.
The objective
This is an observational study that assesses the spontaneous progression (natural history) of the disease over time, measured through neurological examinations, timed walking tests, upper limb function measurements, and patient-reported quality of life assessments. Some research centers conduct additional assessments related to speech, vision, fatigue, balance, and cognition. These data are supplemented with information on the patient’s history and management, including any treatments.
Through this study, the researchers aim to determine patterns, trends, and potential variations in natural history across patient subgroups, as well as the effectiveness of therapies or interventions. They also aim to identify biomarkers and new endpoints for future clinical trials.
This type of study is essential for understanding the mechanisms of the disease, but also for being able to subsequently evaluate the effects of a drug candidate: it will be considered effective if it modifies the natural history.
In practice
Each patient is monitored for 25 years with one assessment per year.
France has two research centers at the Brain Institute in Paris and at the Hautepierre Hospital in Strasbourg.
Where are we?
Recruitment has not yet started in France.
Contacts :
Elodie Petit (Paris Brain Institute – ICM)
elodie.petit@icm-institute.org
Tel. : 01 57 27 45 31
Mathieu Anheim (Hautepierre Hospital, Neurology Department, Strasbourg)
mathieu.anheim@chru-strasbourg.fr
Tel. : 03 88 12 87 11