NCT05445323

Gene Therapy for Cardiomyopathy Associated With Friedreich's Ataxia

A Phase 1/2 Study of the Safety and Efficacy of LX2006 Gene Therapy in Participants With Cardiomyopathy Associated With Friedreich's Ataxia

• Status: Active Not Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 8 (actual)
• Sponsor: Lexeo Therapeutics · Industry
• Sex: All
• Age: 18 Years – 50 Years
• Healthy volunteers: Not accepted

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.

Detailed description

Friedreich's ataxia (FA) is a rare, autosomal recessive disease caused by a mutation in the autosomal frataxin (FXN) gene. Progressive cardiomyopathy with cardiac hypertrophy and fibrosis is observed in most individuals with FA. The disease is more severe in those with earlier onset. Presently, there is no therapy that alters the progression of cardiomyopathy in FA, which is responsible for 59% of FA-related deaths. The primary objective of this dose escalation study is to assess the safety and tolerability of three ascending doses of LX2006 in patients with FA-associated cardiomyopathy. LX2006 is designed to restore hFXN levels in order to improve mitochondrial function. Assessments of cardiac function, biomarkers and other preliminary efficacy endpoints are also included in this study.

Conditions

• Friedreich Ataxia
• Cardiomyopathy, Secondary

Interventions

Type	Name	Description
GENETIC	Low dose LX2006	Adeno-associated viral vector encoding the FXN gene (AAVrh.10hFXN)
GENETIC	Mid Dose LX2006	Adeno-associated viral vector encoding the FXN gene (AAVrh.10hFXN)
GENETIC	High Dose LX2006	Adeno-associated viral vector encoding the FXN gene (AAVrh.10hFXN)

Timeline

• Start date: 2022-08-24
• Primary completion: 2029-09-01
• Completion: 2029-09-01
• First posted: 2022-07-06
• Last updated: 2025-12-23

Locations

3 sites across 1 country: United States

Regulatory

• FDA-regulated drug study