Study of LX2006 Gene Therapy in Friedreich Ataxia Cardiomyopathy (SUNRISE-FA 2)

Lexeo Therapeutics

Status and phase: Enrolling; Phase 2

Conditions: Cardiomyopathy, Secondary; Friedreich Ataxia

Treatments: Genetic: LX2006; Other: Usual Care

Study type: Interventional

Funder types: Industry

Identifiers

NCT07721025

2025-524970-40-00 (EU Trial (CTIS) Number)

LX2006-03

Details and patient eligibility

About

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).

Enrollment: 26 estimated patients

Sex: All

Ages: 6+ years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Crossover Assignment

Masking: Single Blind

---

26 participants in 2 patient groups

LX2006

Experimental group

Description:

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.

Treatment:

Genetic: LX2006

Usual Care

Other group

Description:

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).

Treatment:

Other: Usual Care