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Gene Therapy for Cardiomyopathy Associated With Friedreich's Ataxia

About this research study:

Lexeo Therapeutics is sponsoring a clinical trial to learn about the safety and tolerability of 2 different doses of an investigational gene therapy in participants with cardiomyopathy associated with Friedreich’s Ataxia. This investigational gene therapy is specifically designed to deliver a normal copy of the human FXN (Frataxin) gene to the body which may lead to improvement in the cardiomyopathy associated with Friedreich’s Ataxia. All eligible participants will receive one of the two doses of gene therapy; there will no placebo in this study.

This study is being conducted at multiple different sites and will last approximately 5 years in total. The screening period will take up to 3 months, and then there will be 16 office visits during the first year following the investigational therapy administration. After that, there will be a long-term follow up period consisting of an additional 10 office visits over the next 4 years.

  • Immediately after the investigational gene therapy is given, participants will need to stay in the hospital for at least two nights
  • Participants will also need to reside close to the study site for up to 4 weeks after the investigational gene therapy is given
The investigational gene therapy and all study-related assessments will be provided at no cost. You may be reimbursed for study-related expenses, such as parking, meals, and other reasonable accommodations. Travel and accommodations will be arranged for participants.

To learn more about gene therapy please visit:

  1. FARA’s Roadmap to Gene Therapy Readiness
  2. Materials from the Cornell/Lexeo community webinar:
    1. Slides
    2. Unanswered audience questions
    3. FAQs
You may be eligible if you:

  • Are male or female 18 - 40 years of age
  • Have a confirmed diagnosis of Friedreich’s Ataxia, with onset of disease before 25 years of age
  • Have evidence of cardiomyopathy
  • Are willing and able to undergo cardiac biopsies and MRIs
There are additional eligibility criteria that will be assessed and discussed with you by the study doctor at the time of screening.

Additional information can be found on:

How to participate:

This study is currently recruiting at University of California Los Angeles and University of South Florida. Other sites, including University of Iowa, and Mayo Clinic, will start recruiting at a later date.

If you are interested in participation and would like to learn more about the study, please contact:

UCLA - Aaron Fisher 310-206-8153
USF - Shaila Ghanekar 813-974-5909

Anyone considering participating in a clinical trial should discuss the matter with his or her physician. FARA does not endorse or recommend any particular studies.

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