Omaveloxolone for swallowing problems in French people with Friedreich's 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

This is an observational, single-center project at CHU Nice that follows people with genetically confirmed Friedreich's ataxia treated with omaveloxolone via France's early-access program. Eligible participants started omaveloxolone between February 2024 and May 2025 and must have completed the Sydney Swallow Questionnaire (SSQ) at baseline and after six months of treatment. The main approach is to compare patient-reported swallowing scores before and after at least six months on the drug to identify changes in dysphagia. There is no randomized control arm, so findings will reflect real-world outcomes in treated patients followed at this center.

Why it matters

Who can join: the full rules

Inclusion Criteria:

* Aged ≥ 16 Years
* Confirmed diagnosis of Friedreich's ataxia, genetically verified.
* Omaveloxolone therapy between February 2024 and May 2025, having receive treatment for at least 6 months.

Exclusion Criteria:

* Participants who interrupted treatment permanently before 6 months.
* Participants who did not complete the SSQ (Sydney swallow Questionnaire) at baseline and after 6 months of treatment.

Where it's running

• Chu Nice
  Nice, Alpes Maritimes, France Recruiting

Study contacts

• Study coordinator: Andra EZARU
• Email: ezaru.a@chu-nice.fr
• Phone: 04 92 03 96 55

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

• 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

• Health effects of omaveloxolone taken during pregnancy or breastfeeding in people with Friedreich's Ataxia

  See if taking BIIB141 (omaveloxolone) during pregnancy or while breastfeeding affects the health of people with Friedreich's Ataxia and their babies.

  Recruiting Biogen · Morrisville, North Carolina · NCT06628687

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.