Study of LX2006 Gene Therapy in Friedreich Ataxia Cardiomyopathy
Recruiting
Phase 2
Interventional Study
Friedreich Ataxia
Cardiomyopathy, Secondary
No Placebo Group
Every participant receives an active treatment — no one gets a placebo.
Prior Safety Data
This treatment has already been tested in at least one earlier human trial.
At a Glance
- Age
- 6 and older
- Sex
- Any
- Trial phase
- Phase 2
- Study type
- Interventional
- Purpose
- Treatment
- Participants needed
- 26 (estimated)
- Sponsor
- Lexeo Therapeutics · Industry
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About This Trial
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).
Trial Locations
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Eligibility Criteria
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 ventricula…
Contacts