Gene Therapy for Cardiomyopathy in Friedreich's Ataxia

Recruiting in Palo Alto (17 mi)

+3 other locations

Age: 18 - 65

Sex: Any

Travel: May be covered

Time Reimbursement: Varies

Trial Phase: Phase 1 & 2

Waitlist Available

Sponsor: Lexeo Therapeutics

No Placebo Group

Trial Summary

What is the purpose of this trial?This is a Phase 1/2, open-label, dose-ascending, multicenter study of the safety and efficacy of LX2006 for participants who have Friedreich's Ataxia with evidence of cardiomyopathy. The study will evaluate up to three doses of single administration of LX2006 (AAVrh.10hFXN), an adeno-associated virus (AAV) gene therapy designed to intravenously deliver the human frataxin (hFXN) gene to cardiac cells over a 52-week period. Long-term safety and efficacy will be evaluated for an additional 4-years for a total of 5-years post LX2006 treatment.

Eligibility Criteria

This trial is for individuals with Friedreich's Ataxia diagnosed before age 25 who have heart issues related to the condition. They must meet specific criteria for cardiomyopathy and antibody levels. Excluded are those with significant coronary disease, uncontrolled diabetes or psychiatric conditions, abnormal liver function, certain infections like hepatitis or HIV, unstable heart rhythms needing intervention, or on immunosuppressive drugs.

Inclusion Criteria

I was diagnosed with Fanconi anemia before I turned 25.

Exclusion Criteria

My diabetes is not under control.

I have heart disease not related to FA cardiomyopathy.

My heart's pumping ability is within the required range.

I cannot have heart tissue samples taken due to health risks.

I do not have any active infections, including hepatitis or HIV.

I am currently taking steroids or other medications that weaken my immune system.

My liver isn't working properly.

I have serious heart rhythm problems needing doctor's care.

Participant Groups

The study tests LX2006 gene therapy at different doses in people with Friedreich's Ataxia-related cardiomyopathy. It involves a single administration of an AAV vector carrying the human frataxin gene to cardiac cells and monitors safety and effectiveness over one year, with a follow-up period extending to five years.

1Treatment groups

Experimental Treatment

Group I: Cohort 1/ Cohort 2/ Cohort 3Experimental Treatment3 Interventions