Gene Therapy LX2006 Tested in Friedreich Ataxia Heart Disease
Tests LX2006 gene therapy against usual care in people with Friedreich ataxia heart disease.
Plain-language title generated from the registry record
Registry title: Study of LX2006 Gene Therapy in Friedreich Ataxia Cardiomyopathy
Key facts
- Where
- Tampa, United StatesSee the site
- Phase
- Phase 2
Where in United States
- University of South FloridaTampa, United StatesTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- Lexeo Therapeutics
- Trial code
- NCT07721025EU 2025-524970-40-00
- Record updated
- 2 September 2026
- Original description
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).Read full original descriptionShow less
A Phase 2, Multicenter, Open-label, Randomized, Controlled Study of LX2006 Gene Therapy in Participants With Friedreich Ataxia Cardiomyopathy (SUNRISE-FA 2)
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).
Inclusion: 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: Prior organ transplant; Previous gene transfer or cell therapy; Presence of other forms of cardiomyopathy that contribute to heart failure; Contraindication to cardiac MRI; Active hematologic or solid organ cancer; Other inclusion/exclusion criteria to be applied as per protocol; Poorly controlled diabetes (hemoglobin A1c ≥8%); Current use of inotrope infusion or presence of a ventricular assist device.