Omaveloxolone for swallowing problems in French people with Friedreich's ataxia
Retrospective Bicentric Study in a Cohort of French Friedreich's Ataxia Patients on the 6-month Efficacy of Omaveloxolone Treatment on Dysphagia.
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.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 40 (estimated) |
| Ages | 16 Years and up |
| Sex | All |
| Sponsor | Centre Hospitalier Universitaire de Nice Academic / other |
| Locations | 1 site (Nice, Alpes Maritimes) |
| Trial ID | NCT07013292 on ClinicalTrials.gov |
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.
Who should consider this trial
Good fit: People aged 16 years or older with genetically confirmed Friedreich's ataxia in France who began omaveloxolone between February 2024 and May 2025 and completed baseline and six-month SSQ assessments.
Not a fit: Participants who stopped omaveloxolone before six months, who did not complete the required SSQ questionnaires, or whose dysphagia is primarily due to non-FA causes are unlikely to gain information or benefit from this project.
Why it matters
Potential benefit: If successful, it could show that omaveloxolone improves swallowing and reduces complications such as malnutrition, dehydration, and aspiration-related pneumonia in people with Friedreich's ataxia.
How similar studies have performed: Omaveloxolone has shown benefit on some neurological outcomes in prior Friedreich's ataxia trials, but targeted evidence specifically for improving dysphagia is limited, making this a relatively novel, focused assessment.
Eligibility criteria
Show full inclusion / exclusion criteria
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 this trial is running
Nice, Alpes Maritimes
- 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
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.