Study of LX2006 Gene Therapy in Friedreich Ataxia Cardiomyopathy

RecruitingPhase 2

A Phase 2, Multicenter, Open-label, Randomized, Controlled Study of LX2006 Gene Therapy in Participants With Friedreich Ataxia Cardiomyopathy (SUNRISE-FA 2)

Who can join

Ages 6 and older · All sexes

Full 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 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.

About the study

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).

What is being tested

LX2006 (genetic)
Usual Care (other)

Sponsor: Lexeo Therapeutics · Participants: 26 · Started: Jun 25, 2026

Contact the study team

Lexeo Clinical Trials · Phone: 212-547-9879

Official record on ClinicalTrials.gov — NCT07721025

Locations in the U.S.

Florida

University of South Florida, Tampa

Conditions

From ClinicalTrials.gov, data retrieved Sep 30, 2026. Each study sets its own eligibility; the study team decides who can join.