Global study on the natural history of Friedreich Ataxia

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 UNIFAI study is a global, multicenter, prospective, longitudinal observational study aimed at understanding the natural progression of Friedreich Ataxia, a rare genetic disorder. Participants with genetically confirmed Friedreich Ataxia will be assessed annually for up to 25 years, with data collected from medical records, clinical evaluations, and patient-reported outcomes. This study seeks to harmonize existing natural history studies to enhance the understanding of clinical measures that can quantitatively assess the disease.

Why it matters

Who can join: the full rules

Inclusion Criteria:

1. Both males and females of any age
2. Individuals with Friedreich ataxia (FA): Participants that meet genetically confirmed diagnosis of Friedreich ataxia
3. Written informed consent provided

   1. Informed consent must be obtained for all participants
   2. For underage participants, they and the parent/ legally authorized representative have to sign the informed consent form, child assent (if applicable)
   3. Persons who are not legally competent require the informed consent of their legally authorized representative

   Exclusion Criteria:
4. Unable or unwilling to provide informed consent
5. Acute or ongoing medical or other conditions that would interfere with the conduct and assessments of the study
6. For any reason in the opinion of the investigator, participant would be unlikely or unable to comply with study protocol requirements.

Where it's running

• UCLA Ataxia Center
  Los Angeles, California, United States Recruiting
• University of Colorado
  Denver, Colorado, United States Recruiting
• University of Florida - Neurology
  Gainesville, Florida, United States Recruiting
• USF Ataxia Research Center
  Tampa, Florida, United States Recruiting
• Emory University Hospital - Neurology
  Atlanta, Georgia, United States Recruiting
• University of Iowa, Stead Family Children's Hospital
  Iowa City, Iowa, United States Recruiting

Study contacts

• Principal investigator: David Lynch, MD — Children's Hospital of Philadelphia
• Study coordinator: Mackenzi Coker
• Email: fagcc-clinops@curefa.org
• Phone: +14848796160

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)

  A Phase 2 study tests LX2006 gene therapy in people with Friedreich ataxia who also have cardiomyopathy to see if it helps heart function and safety.

  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.