NCT05445323

Brief Summary

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.

Trial Health

75
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
8

participants targeted

Target at below P25 for phase_1

Timeline
41mo left

Started Aug 2022

Longer than P75 for phase_1

Geographic Reach
1 country

3 active sites

Status
active not recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress53%
Aug 2022Sep 2029

First Submitted

Initial submission to the registry

June 23, 2022

Completed
13 days until next milestone

First Posted

Study publicly available on registry

July 6, 2022

Completed
2 months until next milestone

Study Start

First participant enrolled

August 24, 2022

Completed
7 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

September 1, 2029

Expected
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

September 1, 2029

Last Updated

December 23, 2025

Status Verified

December 1, 2025

Enrollment Period

7 years

First QC Date

June 23, 2022

Last Update Submit

December 19, 2025

Conditions

Friedreich AtaxiaCardiomyopathy, Secondary

Keywords

Friedreich's AtaxiaCardiomyopathyFAGene therapyFXN GeneFrataxin GeneLX2006

Outcome Measures

Primary Outcomes (1)

  • Treatment-emergent adverse events (TEAEs) and Treatment-emergent serious events (TESAEs)

    Change from baseline to end of year 5 post dose

Secondary Outcomes (5)

  • Change from baseline in LVMi

    Change from baseline to end of year 5 post dose

  • Change from baseline in LVEF

    Change from baseline to end of year 5 post dose

  • Change from baseline in cardiac fibrosis as measured by cardiac MRI

    Change from baseline to end of year 5 post dose

  • Change from baseline in measures of cardiopulmonary exercise tolerance

    Change from baseline to end of year 5 post dose

  • Presence and severity of cardiac arrythmias

    Change from baseline to end of year 5 post dose

Study Arms (1)

Cohort 1/ Cohort 2/ Cohort 3

EXPERIMENTAL
Genetic: Low dose LX2006Genetic: Mid Dose LX2006Genetic: High Dose LX2006

Interventions

Low dose LX2006GENETIC

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

Cohort 1/ Cohort 2/ Cohort 3
Mid Dose LX2006GENETIC

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

Cohort 1/ Cohort 2/ Cohort 3
High Dose LX2006GENETIC

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

Cohort 1/ Cohort 2/ Cohort 3

Eligibility Criteria

Age18 Years - 50 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64)

You may qualify if:

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

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (3)

Ataxia Center and HD Center of Excellence, University of California

Los Angeles, California, 90095, United States

Location

University of South Florida

Tampa, Florida, 33612, United States

Location

Mayo Clinic

Rochester, Minnesota, 55905, United States

Location

MeSH Terms

Conditions

Friedreich AtaxiaCardiomyopathies

Condition Hierarchy (Ancestors)

Spinocerebellar DegenerationsCerebellar DiseasesBrain DiseasesCentral Nervous System DiseasesNervous System DiseasesSpinal Cord DiseasesHeredodegenerative Disorders, Nervous SystemNeurodegenerative DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesMitochondrial DiseasesMetabolic DiseasesNutritional and Metabolic DiseasesHeart DiseasesCardiovascular Diseases

Study Officials

  • LEXEO Clinical Trials

    Lexeo Therapeutics

    STUDY DIRECTOR

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SEQUENTIAL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

June 23, 2022

First Posted

July 6, 2022

Study Start

August 24, 2022

Primary Completion (Estimated)

September 1, 2029

Study Completion (Estimated)

September 1, 2029

Last Updated

December 23, 2025

Record last verified: 2025-12

Data Sharing

IPD Sharing
Will not share

Locations

US(3)