Recruiting
Phase 2

LX2006

Sponsor:

Lexeo Therapeutics

Code:

NCT07721025

Conditions

Friedreich Ataxia

Cardiomyopathy, Secondary

Eligibility Criteria

Sex: All

Age: 6+

Healthy Volunteers: Not accepted

Interventions

LX2006

Usual Care

Study Details

Brief 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

Study ID

NCT07721025

Start date

Jun 25, 2026

Status verified date

Aug, 2026

Completion date

Jun, 2032

Anticipated

Primary completion date

Jun, 2032

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 6+

Healthy Volunteers: Not accepted

Inclusion Criteria:

  • Male or female, age at least 6 years at the time of signing the informed consent (and assent, if applicable).
  • Diagnosis of FA, based on clinical phenotype and genotype (GAA expansion on the frataxin gene)
  • Onset of FA on or before 25 years of age
  • Confirmed left ventricular hypertrophy and abnormal left ventricular mass index
  • Left ventricular ejection fraction at least 30%
  • Anti-AAVrh.10 total antibody titer less than the protocol-specified maximum level

Exclusion Criteria:

  • Presence of other forms of cardiomyopathy that contribute to heart failure
  • Current use of inotrope infusion or presence of a ventricular assist device
  • Contraindication to cardiac MRI
  • Prior organ transplant
  • Previous gene transfer or cell therapy
  • Poorly controlled diabetes (hemoglobin A1c ≥8%)
  • Active hematologic or solid organ cancer

Other inclusion/exclusion criteria to be applied as per protocol.

Study Design

Enrollment

26 participants

Anticipated

Allocation

Randomized

Intervention Model

Crossover

Primary purpose

Treatment

Interventions and Outcome Measures

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).

Interventions

LX2006

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

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

  • Cohort 1: Percent change from baseline in left ventricular mass index by cardiac MRI [ Time Frame: At Week 26 ]
  • Cohort 2: Incidence and severity of treatment-emergent adverse events [ Time Frame: Through Month 60 ]
  • Cohort 2: Change in clinical routine safety laboratory tests as assessed by standardized clinical laboratory reference ranges [ Time Frame: Through Month 60 ]
  • Cohort 2: Change in clinical vital signs (body temperature [°C]) [ Time Frame: Through Month 60 ]
  • Cohort 2: Change in clinical vital signs (systolic and diastolic blood pressure [mmHg]) [ Time Frame: Through Month 60 ]
  • Cohort 2: Change in clinical vital signs (heart rate [beats per minute]) [ Time Frame: Through Month 60 ]
  • Cohort 2: Change in clinical vital signs (respiratory rate [breaths per minute]) [ Time Frame: Through Month 60 ]
  • Cohort 2: Change in clinical vital signs (oxygen saturation [%]) [ Time Frame: Through Month 60 ]
  • Cohort 2: Change in clinical 12-lead ECG findings (ECG machine automatically calculates the heart rate [beats per minute] and measures PR, QT, QT interval corrected using Fridericia's method intervals, and QRS complex [milliseconds]) [ Time Frame: Through Month 60 ]

Central Contacts and Locations

Central contacts

Lexeo Clinical Trials

212-547-9879clinicaltrials@lexeotx.com

Locations

University of South Florida

Recruiting

Tampa, Florida, United States, 33612

Contacts

Lucretia Campbell

813-974-5633lcampbel@usf.edu

More Information

Sponsor

Lexeo Therapeutics

Last update posted

Sep 2, 2026

Last verified

Aug, 2026

Keywords

  • Friedreich Ataxia Cardiomyopathy
  • FA-CM
  • Friedreich Ataxia
  • Friedreich's Ataxia
  • FA
  • FRDA
  • Cardiomyopathy
  • Genetic Cardiomyopathy
  • Mitochondrial Cardiomyopathy
  • Cardiac Disease
  • Left Ventricular Hypertrophy
  • Hypertrophic Cardiomyopathy (Non-Obstructive)
  • HCM
  • Gene Therapy
  • FXN Gene
  • FXN
  • Frataxin Gene
  • LX2006
  • SUNRISE-FA 2
  • SUNRISE-FA
  • CLARITY

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-10. This information was provided to ClinicalTrials.gov by Lexeo Therapeutics on 2026-09-02.