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Patient Registry of the European Friedreich's Ataxia Consortium for Translational Studies (EFACTS)

Launched by EUROPEAN FRIEDREICH'S ATAXIA CONSORTIUM FOR TRANSLATIONAL STUDIES · Feb 20, 2014

Trial Information

Current as of July 21, 2025

Unknown status

Keywords

Friedreich's Ataxia Efacts Frda

ClinConnect Summary

The EFACTS trial is a study focused on understanding Friedreich's Ataxia (FRDA), a rare genetic condition that affects coordination and movement. This research is being conducted across multiple centers in Europe and aims to gather important information about how FRDA progresses over time. By observing both patients with FRDA and a control group without the condition, the study hopes to improve clinical assessments, develop reliable ways to measure changes in patients’ health, and help with future clinical trials.

To participate in this study, individuals must have a confirmed genetic diagnosis of FRDA. There’s also a need for control participants who have been genetically tested and do not have FRDA. Participants can expect to contribute to important research that could lead to better treatments and care for people with FRDA. The study is currently recruiting individuals of all ages and genders, making it an inclusive opportunity for those affected by this condition.

Gender

ALL

Eligibility criteria

  • Inclusion Criteria:
  • Genetic diagnosis of FRDA
  • For control research participants: genetically confirmed absence of FRDA

About European Friedreich's Ataxia Consortium For Translational Studies

The European Friedreich's Ataxia Consortium for Translational Studies (EFACTS) is a collaborative initiative dedicated to advancing research and treatment options for Friedreich's Ataxia, a rare genetic disorder. Comprising leading academic institutions, healthcare professionals, and patient advocacy groups across Europe, EFACTS aims to facilitate innovative clinical trials and foster the development of new therapeutic strategies. By leveraging a multidisciplinary approach and promoting data sharing, the consortium seeks to enhance understanding of the disease mechanisms, improve patient outcomes, and accelerate the translation of scientific discoveries into clinical practice.

Locations

Paris, , France

Bruxelles, , Belgium

Milan, , Italy

Innsbruck, , Austria

Strasbourg, , France

Aachen, , Germany

Bonn, , Germany

Marburg, , Germany

Munich, , Germany

Tübingen, , Germany

Madrid, , Spain

London, , United Kingdom

Bruxelles, , Belgium

Praha, , Czechia

Paris, , France

Strasbourg, , France

Munich, , Germany

Athens, , Greece

Dublin, , Ireland

Roma, , Italy

Barcelona, , Spain

London, , United Kingdom

Patients applied

0 patients applied

Trial Officials

Jörg B. Schulz, MD

Principal Investigator

University Hospital Aachen, Dept. of Neurology

Paola Giunti, MD

Principal Investigator

University College of London, Institute of Neurology

Caterina Mariotti, MD

Principal Investigator

Fondazione IRCCS Istituto Neurologico Carlo Besta, Unit of Genetics of Neurodegenerative and Metabolic Diseases

Francisco J. Rodriguez de Rivera Garrido, MD

Principal Investigator

La Paz University Hospital, Universidad Autónoma de Madrid, Dept. of Neurology,

Alexandra Durr, MD

Principal Investigator

Hôpital Pitié Salpêtrière, ICM

Thomas Klopstock, MD

Principal Investigator

University of Munich, Dept. of Neurology with Friedrich-Baur-Institute

Sylvia Boesch, MD

Principal Investigator

Medical University Innsbruck, Department of Neurology

Gilles Naeije, MD

Principal Investigator

Université Libre de Bruxelles, Service de Neurologie

Ludger Schöls, MD

Principal Investigator

University of Tübingen, Dept. of Neurodegenerative Diseases, Hertie-Institute for Clinical Brain Research

Thomas Klockgether, MD

Principal Investigator

University Hospital Bonn, Dept. of Neurology

Georgios Koutsis, MD

Principal Investigator

National and Kapodistrian University of Athens, Neurogenetics Unit

Sinead Murphy, MD

Principal Investigator

Tallaght University Hospital, Department of Neurology

Richard Walsh, MD

Principal Investigator

Tallaght University Hospital, Department of Neurology

Enrico Bertini, MD

Principal Investigator

Bambino Gesù Children's Hospital, Department of Neurosciences

Francesc Palau Martinez, MD

Principal Investigator

Hospital Sant Joan de Déu, Servicio de Neurología

Martin Vyhnálek, MD

Principal Investigator

Motol University Hospital, Centre for Hereditary Ataxias

Mathieu Anheim, MD

Principal Investigator

Hôpital de Hautepierre, Service de Neurologie

Bart van de Warrenburg, MD

Principal Investigator

STICHTING RADBOUD UNIVERSITAIR MEDISCH CENTRUM

Kathrin Reetz, MD

Principal Investigator

University Hospital Aachen, Dept. of Neurology

Timeline

First submit

Trial launched

Trial updated

Estimated completion

Not reported

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