Clinical Trials

Friedreich's ataxia

Displaying 4 studies

  • Gene Therapy for Cardiomyopathy Associated With Friedreich's Ataxia Rochester, MN

    The primary purpose of this dose-ranging study is to assess the safety and tolerability of 3 ascending doses of LX2006 gene therapy for the purpose of selecting the appropriate dose for further clinical development. In addition, assessments of biomarkers and preliminary efficacy are included in this study.

    The primary purpose of the LTFU is to assess the long-term safety and tolerability of LX2006 up to 5 years post-treatment. Additionally, efficacy assessments will be evaluated quarterly during Year 2 and annually up to 5 years post-treatment.

  • (+) Epicatechin to Treat Friedreich's Ataxia Rochester, MN

    The purpose of this study is to test the safety and effectiveness of synthetically produced (+) Epicatechin for treating patients who have Friedreich's Ataxia, a neurological disorder.

  • Individualized Medicine Biobank for Mitochondrial Diseases Rochester, MN

    The Mitochondrial Disease Biobank is a place to store blood and tissue samples from people with symptoms of mitochondrial disease. Health information about each donor will be attached to the samples.

  • A Study on Mayo Clinic Patients with Friedreich Ataxia Rochester, MN

    The purpose of this study is to create a clinical registry of patients with FRDA seen at Mayo Clinic.

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