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NCT Number: NCT07721025

Study of LX2006 Gene Therapy in Friedreich Ataxia Cardiomyopathy

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).

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Key information

Conditions

Friedreich Ataxia Aortic Stenosis, Subvalvular Aortic Valve Disease Aortic Valve Stenosis Brain Diseases Cardiomegaly Cardiomyopathies Cardiomyopathy, Hypertrophic Cardiomyopathy, Secondary Cardiovascular Diseases Central Nervous System Diseases Cerebellar Diseases Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Heart Diseases Heart Valve Diseases Heredodegenerative Disorders, Nervous System Hypertrophy Hypertrophy, Left Ventricular Metabolic Diseases Mitochondrial Diseases Nervous System Diseases Neurodegenerative Diseases Nutritional and Metabolic Diseases Pathological Conditions, Anatomical Pathological Conditions, Signs and Symptoms Spinal Cord Diseases Spinocerebellar Degenerations

Age range

6 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

University of South Florida

Tampa, Florida, 33612, United States

Location status: Recruiting

Location contact

Lucretia Campbell

CONTACT

[email protected]

813-974-5633

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

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).

Primary outcomes

  1. Percent change from baseline in left ventricular mass index by cardiac MRI

    Time frame: At Week 26

Secondary outcomes

  1. Change from baseline in high-sensitivity troponin I

    Time frame: At Week 26 and through Month 60

  2. Change from baseline in left ventricular wall thickness and additional imaging outcomes

    Time frame: At Week 26 and through Month 60

  3. Change from baseline in modified Friedreich's Ataxia Rating Scale

    Time frame: At Week 26 and through Month 60

    A neurological assessment tool for Friedreich ataxia that is measured on a scale of 0 to 93 with high scores indicating greater physical impairment

  4. Change from baseline in Kansas City Cardiomyopathy Questionnaire

    Time frame: Through Month 60

    A cardiac assessment tool scored from 0 to 100, with higher scores indicating better health status and lower scores reflecting more severe heart failure symptoms and functional limitations

  5. Healthcare resource use

    Time frame: Through Month 60

    Time from baseline to first occurrence of any of the following as captured via electronic data capture system:

    • Death due to any cause, and
    • Any of the following due to cardiovascular events:
    • Death
    • Hospitalization for heart failure
    • Non-fatal stroke
    • Non-fatal heart attack
    • Non-fatal life-threatening rapid or irregular heartbeat
    • Heart transplant
    • Insertion of a left ventricular assist device (pump) in the heart

  6. Cardiovascular events

    Time frame: Through Month 60

    Number of the following cardiovascular events as collected from the patient by the study doctor during scheduled study visits:

    • Hospitalization for heart failure
    • Non-fatal stroke
    • Non-fatal heart attack
    • Non-fatal life-threatening rapid or irregular heartbeat
    • Heart transplant
    • Insertion of a left ventricular assist device (pump) in the heart

  7. Patient Global Impressions scales

    Time frame: Through Month 60

  8. Incidence and severity of treatment-emergent adverse events

    Time frame: Through Month 60

  9. Change in clinical routine safety laboratory tests as assessed by standardized clinical laboratory reference ranges

    Time frame: Through Month 60

  10. Change in clinical vital signs (body temperature [°C])

    Time frame: Through Month 60

  11. Change in clinical vital signs (systolic and diastolic blood pressure [mmHg])

    Time frame: Through Month 60

  12. Change in clinical vital signs (heart rate [beats per minute])

    Time frame: Through Month 60

  13. Change in clinical vital signs (respiratory rate [breaths per minute])

    Time frame: Through Month 60

  14. Change in clinical vital signs (oxygen saturation [%])

    Time frame: Through Month 60

  15. Change in clinical 12-lead ECG findings

    Time frame: Through Month 60

    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]

Study contacts

Contact information is provided by the study sponsor or research team.

Lexeo Clinical Trials

CONTACT

[email protected]

212-547-9879

Sponsors and collaborators

Lead sponsor

Lexeo Therapeutics

Industry

Registry information

Official study title

A Phase 2, Multicenter, Open-label, Randomized, Controlled Study of LX2006 Gene Therapy in Participants With Friedreich Ataxia Cardiomyopathy (SUNRISE-FA 2)

Acronym: SUNRISE-FA 2

Important dates

Study start
2026
Primary completion
2032
Study completion
2032
First posted
Jul 22, 2026
Registry last updated
Jul 22, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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