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

Launched by CENTRE HOSPITALIER UNIVERSITAIRE DE NICE · Jun 5, 2025

Keywords

ClinConnect Summary

This clinical trial is studying the effectiveness of a medication called Omaveloxolone in treating dysphagia, which is difficulty swallowing, in patients with Friedreich's ataxia (FA). FA is a rare genetic condition that affects movement and coordination, and it can lead to serious problems like malnutrition and social isolation due to swallowing difficulties. The trial will look at how well Omaveloxolone works over six months for patients aged 16 and older who have received early access to this treatment in France.

To participate in the trial, candidates must be at least 16 years old and have a confirmed diagnosis of Friedreich's ataxia. Participants will take Omaveloxolone for at least six months and complete a simple questionnaire about their swallowing abilities at the beginning and end of the trial. This study aims to provide valuable insights into how this medication can help improve swallowing difficulties in FA patients, an area that has not been extensively researched before.

Gender

ALL

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.