Gene Therapy for Cardiomyopathy Associated With Friedreich's Ataxia

NCTID	NCT05445323 (View at clinicaltrials.gov)
Description	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. (Show More)
Development Status	Active
Indication	Friedreich Ataxia, Cardiomyopathy, Secondary
Disease Ontology Term	DOID:0111218
Drug Product Name	LX2006
Drug Product Description 🔄	AAVrh.10-CAG-hFXN
Sponsor	Lexeo Therapeutics
Funder Type	Industry
Recruitment Status	Active not recruiting
Enrollment Count	8 (ACTUAL)
Results Posted	Not Available

Target Gene/Variant	FXN
Therapeutic Modality	Gene transfer
Therapy Route	in vivo
Mechanism of Action	Functional gene replacement
Route of Administration	Intravenous
Drug Product Type	Viral vector
Gene Delivery System Type	Viral transduction
Vector Type	AAVrh10
Dose 1 🔄	Dose escalation study: 1.8E11 vg/kg (n=6), 5.6E11 vg/kg (n=7),1.2E12 vg/kg (n=4)
Dose 2 🔄	Planned pivotal dose: 1.2E12 vg/kg

Eligible Age	18 Years - 50 Years
Standard Ages	Adult
Sexes Eligible for Study	ALL
Eligibility Criteria	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. View Inclusion and Exclusion Criteria at ClinicalTrials.gov

No.of Trial Sites	3
Locations	United States

Has US IND	True
FDA Designations	Breakthrough Therapy, Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation, Regenerative Medicine Advanced Therapy (RMAT)
Recent Updates	Finalized pivotal trial protocol and statistical analysis plan in June 2026, 1.2E12 will be pivotal dose, topline data readout expected 2H2027