Contact SCGE




Gene Therapy Trial Report

Summary

Study of LX2006 Gene Therapy in Friedreich Ataxia Cardiomyopathy


NCTID NCT07721025 (View at clinicaltrials.gov)
Description
Development Status 🔄 Active
Indication 🔄 Friedreich Ataxia with Secondary Cardiomyopathy
Disease Ontology Term 🔄 DOID:0111218
Drug Product Name 🔄 LX2006
Drug Product Description 🔄 AAVrh.10-CAG-hFXN
Sponsor Lexeo Therapeutics
Funder Type Industry
Recruitment Status
Enrollment Count 26 (ESTIMATED)
Results Posted Not Available

Therapy Information


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 🔄 Adeno-associated virus
Viral Vector Subtype 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 🔄 Pivotal dose: 1.2E12 vg/kg

Study Record Dates


Current Phase Phase2
Submit Date 2026-06-24
Completion Date 2032-06
Last Update 2026-07-22

Participation Criteria


Eligible Age >=6 Years
Standard Ages Child, Adult, Older adult
Sexes Eligible for Study ALL
Eligibility Criteria
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.
View Inclusion and Exclusion Criteria at ClinicalTrials.gov

Locations


No.of Trial Sites 1
Locations United States

Regulatory Information


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

Resources/Links