Study of LX2006 Gene Therapy in Friedreich Ataxia Cardiomyopathy (NCT07721025) | Clinical Trial Compass
RecruitingPhase 2
Study of LX2006 Gene Therapy in Friedreich Ataxia Cardiomyopathy
United States26 participantsStarted 2026-06-25
Plain-language summary
The purpose of Study LX2006-03, a multicenter, Phase 2, open-label, randomized, controlled study, is to evaluate the efficacy and safety of LX2006 gene therapy in participants with Friedreich ataxia (FA) cardiomyopathy (CM).
Who can participate
Age range
6 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:
* Male or female, age at least 6 years at the time of signing the informed consent (and assent, if applicable).
* Diagnosis of FA, based on clinical phenotype and genotype (GAA expansion on the frataxin gene)
* Onset of FA on or before 25 years of age
* Confirmed left ventricular hypertrophy and abnormal left ventricular mass index
* Left ventricular ejection fraction at least 30%
* Anti-AAVrh.10 total antibody titer less than the protocol-specified maximum level
Exclusion Criteria:
* Presence of other forms of cardiomyopathy that contribute to heart failure
* Current use of inotrope infusion or presence of a ventricular assist device
* Contraindication to cardiac MRI
* Prior organ transplant
* Previous gene transfer or cell therapy
* Poorly controlled diabetes (hemoglobin A1c ≥8%)
* Active hematologic or solid organ cancer
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
Percent change from baseline in left ventricular mass index by cardiac MRI