ClinicalTrials.gov record NCT07013292

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

Record copied from ClinicalTrials.gov: Oct 10, 2026AI summaries generated: Sep 7, 2026

Retrospective Bicentric Study in a Cohort of French Friedreich's Ataxia Patients on the 6-month Efficacy of Omaveloxolone Treatment on Dysphagia.

StatusRecruitingThe study is currently enrolling participants. Only the study team can say whether a particular person can join.
Study typeObservationalA study in which researchers do not assign participants to a treatment or other intervention. They observe or measure what happens, which may include study visits, tests, questionnaires or a review of medical records.
Age16 years old and older
SexAll
LocationFrance
AI summaryWhat this study is about, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

This retrospective cohort study looks at people with genetically confirmed Friedreich Ataxia who received omaveloxolone under early access and completed six months of treatment. Swallowing difficulty (dysphagia) is measured by the Sydney Swallow Questionnaire (SSQ) at treatment start and after six months. The study also compares SSQ results with ataxia severity (SARA), ambulatory status, and GAA repeat size.

What it is studying. The study examines changes in dysphagia severity (measured by the Sydney Swallow Questionnaire) from treatment start to six months in people with Friedreich Ataxia who received omaveloxolone during early access.

What the researchers hope to learn. Measure change in dysphagia after six months of omaveloxolone treatment; compare swallowing scores (SSQ) with ataxia severity (SARA); assess correlations with ambulatory status and GAA expansion size.

What this study is about

The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.

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.

Interventions being studied

Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.

Drug
  • SKYCLARYS (omaveloxolone)

Eligibility

The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.

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.

Study description and what participation involves

The registry record has no detailed description beyond the summary above.

AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

This is a retrospective review of existing clinical data: researchers will use SSQ scores taken at treatment start (baseline) and after six months of omaveloxolone and other information already in medical records. The study also uses existing SARA (ataxia) scores and GAA expansion size from patient files when available. No new testing procedures, samples, or drug administration are described in the registry record for this study.

How the study is structured. A retrospective observational cohort review: researchers use medical records and patient-completed SSQs from the start of omaveloxolone treatment and again after six months. All participants already received the drug as part of early access; the study does not assign treatments or randomize participants. Analyses compare SSQ scores over time and against other clinical measures (SARA score, ambulatory status, GAA repeat size).

Questions you could ask

These questions are suggestions for a conversation with the study team or your own clinician, not advice.

  • This is an observational study, so the study itself gives no treatment. What would taking part involve for me, and would it change my usual care?
  • The listed sites are in France. Is any of them reachable for me, and does the study help with travel?
  • Which of the eligibility criteria would need to be checked with tests before I could join?

More on Friedreich ataxia

Clinical trials26 trials listed for Friedreich ataxiaEvery trial the registry lists for Friedreich ataxia, with recruitment status, phase and sites, updated from ClinicalTrials.gov.See all trials →Research updatesWhat changed in Friedreich ataxia researchLatest monthly update: September 2026. Includes: Heart change stages. New research summarized each month in plain language, with links to every source paper.Read the latest update →

Important dates

Study start
Jun 1, 2025
Primary completion
Jun 30, 2026 (estimated)
Study completion
Jun 30, 2026 (estimated)
Target enrollment
40 participants (estimated)
Last updated on registry
Apr 29, 2026
Data verified
Oct 10, 2026

Sponsor and research team

The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.

Lead sponsor
Centre Hospitalier Universitaire de Nice

Central study contact

If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.

Participating sites (1)

Terms used on this page · explanations by MedicalUpdates.ai

life expectancy
Some studies require that, in the investigator's judgement, a participant is likely to live long enough to complete the study period. It is a study requirement, not a prediction about any individual.
cohort
A group of people who share a characteristic, such as a diagnosis, and are studied together. 'Prospective' means they are followed forward from enrolment; 'retrospective' means existing records are reviewed.
baseline
Measurements taken at the start of the study, before any study treatment, so that later results can be compared with them.
Inclusion Criteria
The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
Exclusion Criteria
The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.

The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.

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