Study of LX2006 Gene Therapy in Friedreich Ataxia Cardiomyopathy

Part of paid clinical trials in Tampa, Florida.

Sponsor
Lexeo Therapeutics
Study ID
NCT07721025
Phase
PHASE2
Status
Recruiting
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Conditions

  • Cardiomyopathy, Secondary
  • Friedreich Ataxia

Eligibility Criteria

Sex
ALL
Age
6 Years - N/A
Healthy Volunteers
Not accepted

Interventions

  • LX2006 — GENETIC
    Adeno-associated viral vector encoding the FXN gene (AAVrh.10hFXN)
  • 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).

Study Details

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).

Key Dates

First listed
Jul 22, 2026
Start date
Jun 25, 2026
Status verified
Jul 2026
Primary completion
Jun 30, 2032
Completion
Jun 30, 2032

Study Design

Enrollment
26 participants (estimated)
Allocation
RANDOMIZED
Intervention model
CROSSOVER
Primary purpose
TREATMENT

Arms

  • Experimental: LX2006
    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.
  • 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).

Primary Outcome Measure

Percent change from baseline in left ventricular mass index by cardiac MRI [ Time Frame: At Week 26 ]

Central Contacts

Locations (1)

FacilityCityStateZIPSite coordinators
University of South FloridaTampaFlorida33612
Lucretia Campbell
[email protected]
813-974-5633

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By research site
University of South Florida· Tampa, FL

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