Gene Therapy for Cardiomyopathy Associated With Friedreich's Ataxia (NCT05445323) | Clinical Trial Compass
Active — Not RecruitingPhase 1/2
Gene Therapy for Cardiomyopathy Associated With Friedreich's Ataxia
United States8 participantsStarted 2022-08-24
Plain-language summary
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.
Who can participate
Age range
18 Years – 50 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion Criteria:
* Confirmed genetic diagnosis of FA, with onset being before 25 years of age
* Protocol specified ranges for antibodies
* Protocol specified measures of FA cardiomyopathy
Exclusion Criteria:
* Protocol specified ranges for left ventricular ejection fraction (LVEF) as measured by cardiac ECHO
* Uncontrolled diabetes
* Abnormal liver function
* Active infection of any type, including hepatitis virus (A, B or C) or human immunodeficiency virus (HIV-1 and HIV-2)
* Contraindication to cardiac MRI
* Contraindications to cardiac biopsies
* Participants who are receiving systemic corticosteroids or other immunosuppressive medications
* History of significant coronary artery disease or any structural heart or vascular disease other than FA cardiomyopathy
* Presence of clinically significant, hemodynamically unstable arrhythmias, requiring physician intervention
* Presence of clinically significant abnormalities as determined by the investigator, other than ECG abnormalities related to FA
* Uncontrolled psychiatric disease
Other Inclusion/Exclusion criteria to be applied as per protocol.
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Treatment-emergent adverse events (TEAEs) and Treatment-emergent serious events (TESAEs)
Timeframe: Change from baseline to end of year 5 post dose