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Gene Therapy for Cardiomyopathy Associated With Friedreich's Ataxia

NCT05445323 · Status: ACTIVE_NOT_RECRUITING · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 8

Last updated 2025-12-23

No results posted yet for this 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.

Conditions

  • Friedreich Ataxia
  • Cardiomyopathy, Secondary

Interventions

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)

Sponsors & Collaborators

  • Lexeo Therapeutics

    lead INDUSTRY

Principal Investigators

  • LEXEO Clinical Trials · Lexeo Therapeutics

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SEQUENTIAL

Eligibility

Min Age
18 Years
Max Age
50 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2022-08-24
Primary Completion
2029-09-30
Completion
2029-09-30
FDA Drug
Yes

Countries

  • United States

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05445323 on ClinicalTrials.gov