Efficacy of Omaveloxolone Treatment for Dysphagia in French Patients With Friedreich's Ataxia

Recruiting now

Conditions studied: Friedreich Ataxia

In brief

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.

Key facts

Study ID

NCT07013292

Run by

Centre Hospitalier Universitaire de Nice

People needed

40

Starts

2025-06-01

Expected to finish

2026-06-30

Last updated by the study team

2026-04-29

Who can join

Age: 16 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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.

You may not qualify if…

• 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 is running

• CHU NICE — Nice, Alpes Maritimes, France (enrolling)

Full record on ClinicalTrials.gov

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