Study of LX2006 Gene Therapy in Friedreich Ataxia Cardiomyopathy

NCT07721025 | Recruiting Phase 2 DMC FDA Drug

• 3 other identifiers: LX2006-032025-524970-40-002025
• Study Type: interventional
• Target: 26
• Locations: 1 country
• Sites: 1

Brief Summary

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).

Conditions

Friedreich Ataxia; Cardiomyopathy, Secondary

Keywords

Friedreich Ataxia Cardiomyopathy; FA-CM; Friedreich Ataxia; Friedreich's Ataxia; FA; FRDA; Cardiomyopathy; Genetic Cardiomyopathy; Mitochondrial Cardiomyopathy; Cardiac Disease; Left Ventricular Hypertrophy; Hypertrophic Cardiomyopathy (Non-Obstructive); HCM; Gene Therapy; FXN Gene; FXN; Frataxin Gene; LX2006; SUNRISE-FA 2; SUNRISE-FA; CLARITY

Outcome Measures

Primary Outcomes (9)

• Cohort 1: Percent change from baseline in left ventricular mass index by cardiac MRI

  At Week 26

• Cohort 2: Incidence and severity of treatment-emergent adverse events

  Through Month 60

• Cohort 2: Change in clinical routine safety laboratory tests as assessed by standardized clinical laboratory reference ranges

  Through Month 60

• Cohort 2: Change in clinical vital signs (body temperature [°C])

  Through Month 60

• Cohort 2: Change in clinical vital signs (systolic and diastolic blood pressure [mmHg])

  Through Month 60

• Cohort 2: Change in clinical vital signs (heart rate [beats per minute])

  Through Month 60

• Cohort 2: Change in clinical vital signs (respiratory rate [breaths per minute])

  Through Month 60

• Cohort 2: Change in clinical vital signs (oxygen saturation [%])

  Through Month 60

• Cohort 2: Change in clinical 12-lead ECG findings (ECG machine automatically calculates the heart rate [beats per minute] and measures PR, QT, QT interval corrected using Fridericia's method intervals, and QRS complex [milliseconds])

  Through Month 60

Secondary Outcomes (15)

• Cohorts 1 and 2: Change from baseline in high-sensitivity troponin I

  At Week 26

• Cohort 1: Change from baseline in maximal wall thickness by cardiac MRI

  At Week 26

• Cohort 1: Number of the following cardiovascular events as collected from the participant by the study doctor during scheduled study visits

  At Month 60

• Cohort 1: Change from baseline in modified Friedreich's Ataxia Rating Scale

  At Week 26, at Week 52, and through Month 60

• Cohort 1: Change from baseline in Kansas City Cardiomyopathy Questionnaire

  At Week 26, at Week 52, and through Month 60

Study Arms (2)

LX2006

EXPERIMENTAL

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.

Genetic: LX2006

Usual Care

OTHER

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).

Other: Usual Care

Interventions

LX2006 GENETIC

Adeno-associated viral vector encoding the FXN gene (AAVrh.10hFXN)

LX2006

Usual Care OTHER

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).

Usual Care

Eligibility Criteria

• Age: 6 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Lexeo Therapeutics: lead

Study Sites (1)

University of South Florida

Tampa, Florida, 33612, United States

RECRUITING

MeSH Terms

Conditions

Friedreich Ataxia; Cardiomyopathies; Heart Diseases; Hypertrophy, Left Ventricular; Cardiomyopathy, Hypertrophic

Condition Hierarchy (Ancestors)

Spinocerebellar Degenerations; Cerebellar Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Spinal Cord Diseases; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Mitochondrial Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases; Cardiovascular Diseases; Cardiomegaly; Hypertrophy; Pathological Conditions, Anatomical; Pathological Conditions, Signs and Symptoms; Aortic Stenosis, Subvalvular; Aortic Valve Stenosis; Aortic Valve Disease; Heart Valve Diseases

Study Officials

• Lexeo Clinical Trials

  Lexeo Therapeutics, Inc.

  STUDY DIRECTOR

Central Study Contacts

Lexeo Clinical Trials

CONTACT

212-547-9879; clinicaltrials@lexeotx.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: SINGLE
• Who Masked: OUTCOMES ASSESSOR
• Masking Details: Clinical outcomes of interest will be read with blinded assessors using standardized procedures. All post-baseline centrally read imaging and cardiac biomarker high-sensitivity troponin I will be blinded to the investigator, sponsor, and the reader (and the participant).
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 24, 2026
• First Posted: July 22, 2026
• Study Start: June 25, 2026
• Primary Completion (Estimated): June 1, 2032
• Study Completion (Estimated): June 1, 2032
• Last Updated: September 2, 2026

Record last verified: 2026-08

Locations

US (1)