Assessing health and economic outcomes in Friedreich Ataxia patients

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

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.

Why it matters

Who can join: the full rules

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 it's running

• 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

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• Nomlabofusp by injection for Friedreich's Ataxia: efficacy and safety

  This study tests whether subcutaneous nomlabofusp helps adults and children with Friedreich's Ataxia compared with placebo.

  Recruiting at 3 sites Phase 3 Larimar Therapeutics, Inc. · Tampa, Florida and 3 other locations · NCT07778836

• LX2006 Gene Therapy for Friedreich Ataxia Cardiomyopathy (FA-CM)

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  Recruiting Phase 2 Lexeo Therapeutics · Tampa, Florida · NCT07721025

• Nerve ultrasound in people with Friedreich ataxia

  This project uses routine peripheral nerve ultrasound to see if adults with genetically confirmed Friedreich ataxia have enlarged nerves.

  Recruiting Centre Hospitalier Universitaire de Nice · Nice, Alpes Maritimes · NCT07508631

• Omaveloxolone for swallowing problems in French people with Friedreich's ataxia

  This project will test whether omaveloxolone (SKYCLARYS) improves swallowing in French people aged 16 and older with genetically confirmed Friedreich's ataxia who have used the drug for at least six months.

  Recruiting Centre Hospitalier Universitaire de Nice · Nice, Alpes Maritimes · NCT07013292

• Testing DT-216P2 in patients with Friedreich's Ataxia

  This study is testing a new drug called DT-216P2 in adults with Friedreich's Ataxia to see how safe it is and how well it works in their bodies.

  Recruiting at 3 sites Phase 1/2 Design Therapeutics, Inc. · Sydney, New South Wales and 2 other locations · NCT06874010

• Clinical course of cardiomyopathy in people with Friedreich ataxia

  This study follows people with Friedreich ataxia who have cardiomyopathy to see how their heart disease changes over time and to identify who may later be eligible for a gene therapy study.

  Recruiting at 18 sites Lexeo Therapeutics · La Jolla, California and 18 other locations · NCT06865482

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.