Study of LX2006 Gene Therapy in Friedreich Ataxia Cardiomyopathy

NCT07721025 · Status: RECRUITING · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 26

Last updated 2026-07-22

No results posted yet for this study

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

Interventions

GENETIC

LX2006

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

OTHER

Usual Care

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).

Sponsors & Collaborators

  • Lexeo Therapeutics

    lead INDUSTRY

Principal Investigators

  • Lexeo Clinical Trials · Lexeo Therapeutics, Inc.

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
SINGLE
Model
CROSSOVER

Eligibility

Min Age
6 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2026-06-25
Primary Completion
2032-06-30
Completion
2032-06-30
FDA Drug
Yes

Countries

  • United States

Study Locations

More Related Trials

Entities

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 NCT07721025 on ClinicalTrials.gov