NCT07013292 Efficacy of Omaveloxolone Treatment for Dysphagia in French Patients With Friedreich's Ataxia
| NCT ID | NCT07013292 |
| Status | Recruiting |
| Phase | — |
| Sponsor | Centre Hospitalier Universitaire de Nice |
| Condition | Friedreich Ataxia |
| Study Type | OBSERVATIONAL |
| Enrollment | 40 participants |
| Start Date | 2025-06-01 |
| Primary Completion | 2026-06-30 |
Eligibility & Interventions
Eligibility Fast-Check
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What to Expect as a Participant
This is an observational study. You will not receive an experimental treatment; researchers will collect data based on your existing condition or standard treatment.
This trial targets 40 participants in total. It began in 2025-06-01 with a primary completion date of 2026-06-30.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief Summary
Friedreich's ataxia (FA) is a rare, inherited neurodegenerative disease that typically begins in children and young people. It primarily affects the spinal cord, peripheral nerves and cerebellum of the brain. Clinical manifestations include progressive gait and limb ataxia, auditory and optic neuropathy, cardiomyopathy, scoliosis, dysarthria, and dysphagia. In advanced stages, individuals may become wheelchair-dependent, leading to a severe loss of autonomy and reduced life expectancy. To date, there are no effective treatments known to reverse or halt disease progression. Heart disease remains the leading cause of death in individuals with FA. In January 2024, Omaveloxolone was approved for early access in France to treat FA in patients aged 16 years and older. Dysphagia is a central manifestation in FA, and may lead to severe complications such as malnutrition, dehydration, and aspiration-related pneumonia, as well as reduced self-esteem and social isolation. Despite its clinical relevance, dysphagia remains underexplored in clinical trials, including in major Omaveloxolone studies where no specific tool for measuring dysphagia has been incorporated. This study aimed to comprehensively evaluate the effect of Omaveloxolone on dysphagia after six months of treatment, in a cohort of French patients with Friedreich's ataxia who benefited from early access to treatment between February 2024 and May 2025. The severity of dysphagia will be assessed using the Sydney Swallow Questionnaire (SSQ), completed by patients at baseline and after six months of Omaveloxolone treatment.
Eligibility 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.
Contact & Investigator
Frequently Asked Questions
Who can join the NCT07013292 clinical trial?
This trial is open to participants of all sexes, aged 16 Years or older, studying Friedreich Ataxia. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
Is NCT07013292 currently recruiting?
Yes, NCT07013292 is actively recruiting participants. Contact the research team at ezaru.a@chu-nice.fr for enrollment information.
Where is the NCT07013292 trial being conducted?
This trial is being conducted at Nice, France.
Who is sponsoring the NCT07013292 clinical trial?
NCT07013292 is sponsored by Centre Hospitalier Universitaire de Nice. The trial plans to enroll 40 participants.