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Active, Not Recruiting

NCT Number: NCT05445323

Gene Therapy for Cardiomyopathy Associated With Friedreich's Ataxia

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.

Active, Not Recruiting

This study is active but is not currently recruiting participants.

Key information

Conditions

Friedreich Ataxia Brain Diseases Cardiomyopathies Cardiomyopathy, Secondary Cardiovascular Diseases Central Nervous System Diseases Cerebellar Diseases Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Heart Diseases Heredodegenerative Disorders, Nervous System Metabolic Diseases Mitochondrial Diseases Nervous System Diseases Neurodegenerative Diseases Nutritional and Metabolic Diseases Spinal Cord Diseases Spinocerebellar Degenerations

Age range

18 year–50 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1 / Phase 2

Primary location

Ataxia Center and HD Center of Excellence, University of California, Los Angeles, California, United States

Loading trial locations.

About this study

Friedreich's ataxia (FA) is a rare, autosomal recessive disease caused by a mutation in the autosomal frataxin (FXN) gene. Progressive cardiomyopathy with cardiac hypertrophy and fibrosis is observed in most individuals with FA. The disease is more severe in those with earlier onset. Presently, there is no therapy that alters the progression of cardiomyopathy in FA, which is responsible for 59% of FA-related deaths.

The primary objective of this dose escalation study is to assess the safety and tolerability of three ascending doses of LX2006 in patients with FA-associated cardiomyopathy. LX2006 is designed to restore hFXN levels in order to improve mitochondrial function. Assessments of cardiac function, biomarkers and other preliminary efficacy endpoints are also included in this study.

Who can participate

Healthy volunteers accepted: No

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

Inclusion criteria

  • Confirmed genetic diagnosis of FA, with onset being before 25 years of age
  • Protocol specified ranges for antibodies
  • Protocol specified measures of FA cardiomyopathy

Exclusion criteria

  • Protocol specified ranges for left ventricular ejection fraction (LVEF) as measured by cardiac ECHO
  • Uncontrolled diabetes
  • Abnormal liver function
  • Active infection of any type, including hepatitis virus (A, B or C) or human immunodeficiency virus (HIV-1 and HIV-2)
  • Contraindication to cardiac MRI
  • Contraindications to cardiac biopsies
  • Participants who are receiving systemic corticosteroids or other immunosuppressive medications
  • History of significant coronary artery disease or any structural heart or vascular disease other than FA cardiomyopathy
  • Presence of clinically significant, hemodynamically unstable arrhythmias, requiring physician intervention
  • Presence of clinically significant abnormalities as determined by the investigator, other than ECG abnormalities related to FA
  • Uncontrolled psychiatric disease

Other Inclusion/Exclusion criteria to be applied as per protocol.

Treatment and study plan

Low dose LX2006

Genetic

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

Mid Dose LX2006

Genetic

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

High Dose LX2006

Genetic

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

Primary outcomes

  1. Treatment-emergent adverse events (TEAEs) and Treatment-emergent serious events (TESAEs)

    Time frame: Change from baseline to end of year 5 post dose

Secondary outcomes

  1. Change from baseline in LVMi

    Time frame: Change from baseline to end of year 5 post dose

  2. Change from baseline in LVEF

    Time frame: Change from baseline to end of year 5 post dose

  3. Change from baseline in cardiac fibrosis as measured by cardiac MRI

    Time frame: Change from baseline to end of year 5 post dose

  4. Change from baseline in measures of cardiopulmonary exercise tolerance

    Time frame: Change from baseline to end of year 5 post dose

  5. Presence and severity of cardiac arrythmias

    Time frame: Change from baseline to end of year 5 post dose

Sponsors and collaborators

Lead sponsor

Lexeo Therapeutics

Industry

Registry information

Official study title

A Phase 1/2 Study of the Safety and Efficacy of LX2006 Gene Therapy in Participants With Cardiomyopathy Associated With Friedreich's Ataxia

Important dates

Study start
2022
Primary completion
2029
Study completion
2029
First posted
Jul 6, 2022
Registry last updated
Dec 23, 2025

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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