Gene Therapy for Cardiomyopathy Associated With Friedreich's Ataxia

NCT05445323 · tracked via the Priya Life Science USA tracker

Sponsor

Lexeo Therapeutics

Phase

Phase 1/2

Started

2022-08-24

Last updated

2025-12-23

Condition(s) studied

Friedreich AtaxiaCardiomyopathy, Secondary

Investigational drug(s) / intervention(s)

Low dose LX2006Mid Dose LX2006High Dose LX2006

Low dose LX2006: Adeno-associated viral vector encoding the FXN gene (AAVrh.10hFXN)

Mid Dose LX2006: Adeno-associated viral vector encoding the FXN gene (AAVrh.10hFXN)

High Dose LX2006: Adeno-associated viral vector encoding the FXN gene (AAVrh.10hFXN)

Study summary

This is a Phase 1/2, open-label, dose-ascending, multicenter study of the safety and efficacy of LX2006 for participants who have Friedreich's Ataxia with evidence of cardiomyopathy. The study will evaluate up to three doses of single administration of LX2006 (AAVrh.10hFXN), an adeno-associated virus (AAV) gene therapy designed to intravenously deliver the human frataxin (hFXN) gene to cardiac cells over a 52-week period. Long-term safety and efficacy will be evaluated for an additional 4-years for a total of 5-years post LX2006 treatment.

Eligibility

Sex

ALL

Min age

18 Years

Max age

50 Years

Healthy volunteers

Inclusion Criteria: * Confirmed genetic diagnosis of FA, with onset being before 25 years of age * Protocol specified ranges for antibodies * Protocol specified measures of FA cardiomyopathy Exclusion Criteria: * Protocol specified ranges for left ventricular ejection fraction (LVEF) as measured by cardiac ECHO * Uncontrolled diabetes * Abnormal liver function * Active infection of any type, including hepatitis virus (A, B or C) or human immunodeficiency virus (HIV-1 and HIV-2) * Contraindication to cardiac MRI * Contraindications to cardiac biopsies * Participants who are receiving systemic corticosteroids or other immunosuppressive medications * History of significant coronary artery disease or any structural heart or vascular disease other than FA cardiomyopathy * Presence of clinically significant, hemodynamically unstable arrhythmias, requiring physician intervention * Presence of clinically significant abnormalities as determined by the investigator, other than ECG abnormalities related to FA * Uncontrolled psychiatric disease Other Inclusion/Exclusion criteria to be applied as per protocol.

Primary outcome measure(s)

Treatment-emergent adverse events (TEAEs) and Treatment-emergent serious events (TESAEs) — Change from baseline to end of year 5 post dose

Trial sites (3)

Facility	City	Region
Ataxia Center and HD Center of Excellence, University of California	Los Angeles	California
University of South Florida	Tampa	Florida
Mayo Clinic	Rochester	Minnesota

More Lexeo Therapeutics trials in the USA

Study of LX2006 Gene Therapy in Friedreich Ataxia Cardiomyopathy Recruiting

Gene Therapy for ACM Due to a PKP2 Pathogenic Variant Active, not recruiting

Long-term Follow-up Study of Gene Therapy for Arrhythmogenic Cardiomyopathy Due to a Plakophilin-2 Pathogenic Variant Enrolling by invitation

A Study to Assess Real-world Patient Characteristics and Clinical Course for Symptomatic Patients With PKP2-ACM Recruiting

Long-Term Follow-up of Gene Therapy for APOE4 Homozygote Alzheimer's Disease Active, not recruiting

Other trials for the same condition

A Study to Find Out How BIIB141 (Omaveloxolone) is Processed in the Body and to Learn More About Its Safety in Participants With Friedreich's Ataxia Aged 2 to 15 Years Old Active, not recruiting

Study of LX2006 Gene Therapy in Friedreich Ataxia Cardiomyopathy Recruiting

A Study to Assess the Safety and Efficacy of Vatiquinone in Participants With Friedreich Ataxia Active, not recruiting

A Study to Learn More About the Effects and Long-Term Safety of Omaveloxolone (BIIB141) in Children and Teens With Friedreich's Ataxia Recruiting

A Study to Learn More About the Long-Term Safety of BIIB141 (Omaveloxolone) in Participants With Friedreich's Ataxia Who Are Prescribed it by Their Own Doctors Recruiting

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT05445323 on ClinicalTrials.gov ↗ ← All trials in the USA