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 Criteria:
Exclusion Criteria:
Other inclusion/exclusion criteria to be applied as per protocol.
clinicaltrials@lexeotx.com212-547-9879
Clinical outcomes of interest will be read with blinded assessors using standardized procedures. All post-baseline centrally read imaging and cardiac biomarkers will be blinded to the investigator, sponsor, and the reader (and the participant).
Cohort 1: Participants ≥16 years of age with FA-CM Cohort 2: Participants ≥6 to \<16 years of age with FA-CM. As Cohort 2 will not be randomized, all participants will receive LX2006 upon enrollment. Participants in Cohort 2 will be enrolled after safety is assessed in a group of participants in Cohort 1.
Cohort 1: Participants ≥16 years of age with FA-CM Participants will receive usual care for 26 weeks before receiving treatment with LX2006 (single crossover).
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