Study of LX2006 Gene Therapy in Friedreich Ataxia Cardiomyopathy
Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age6+
SponsorLexeo Therapeutics
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).
Eligibility criteria
Qualifiers
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
Disqualifiers
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
Trial design
Treatments tested in this trial
- LX2006
- Usual Care
Treatment groups
26 Participants
are divided into 2 treatment groupsSponsors and collaborators
Source ClinicalTrials.gov