Gene Therapy for Cardiomyopathy in Friedreich's Ataxia
What You Need to Know Before You Apply
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.
Do I need to stop my current medications for the trial?
The trial excludes participants who are taking systemic corticosteroids or other immunosuppressive medications, so you may need to stop these if you are currently taking them. The protocol does not specify about other medications.
Is the gene therapy treatment LX2006 generally safe in humans?
How does the treatment LX2006 differ from other treatments for Friedreich's Ataxia cardiomyopathy?
LX2006 is a gene therapy that uses an adeno-associated virus (AAV) to deliver a normal copy of the frataxin gene directly to heart cells, aiming to correct the underlying genetic cause of cardiomyopathy in Friedreich's Ataxia. This approach is unique because it targets the root cause of the disease at the genetic level, unlike other treatments that may only address symptoms.678910
What data supports the effectiveness of the treatment LX2006 for cardiomyopathy in Friedreich's Ataxia?
Research on similar gene therapies using adeno-associated viruses (AAV) for Duchenne muscular dystrophy (DMD) shows promise in improving heart function and reducing cardiac issues. These studies suggest that AAV-based gene therapy can be effective in treating heart-related conditions, which may indicate potential benefits for cardiomyopathy in Friedreich's Ataxia.1231112
Who Is on the Research Team?
LEXEO Clinical Trials
Principal Investigator
Lexeo Therapeutics
Are You a Good Fit for This Trial?
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
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive a single administration of LX2006 gene therapy, with dose escalation across cohorts
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- LX2006
Find a Clinic Near You
Who Is Running the Clinical Trial?
Lexeo Therapeutics
Lead Sponsor