Study of LX2006 Gene Therapy in Friedreich Ataxia Cardiomyopathy
NCT07721025 · Status: RECRUITING · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 26
Last updated 2026-07-22
Summary
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).
Conditions
- Friedreich Ataxia
- Cardiomyopathy, Secondary
Interventions
- GENETIC
-
LX2006
Adeno-associated viral vector encoding the FXN gene (AAVrh.10hFXN)
- OTHER
-
Usual Care
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).
Sponsors & Collaborators
-
Lexeo Therapeutics
lead INDUSTRY
Principal Investigators
-
Lexeo Clinical Trials · Lexeo Therapeutics, Inc.
Study Design
- Allocation
- RANDOMIZED
- Purpose
- TREATMENT
- Masking
- SINGLE
- Model
- CROSSOVER
Eligibility
- Min Age
- 6 Years
- Sex
- ALL
- Healthy Volunteers
- No
Timeline & Regulatory
- Start
- 2026-06-25
- Primary Completion
- 2032-06-30
- Completion
- 2032-06-30
- FDA Drug
- Yes
Countries
- United States
Study Locations
More Related Trials
-
Characterization of the Interruptions of the GAA Expansion and Study of Their Influence on the Severity of Friedreich's Ataxia
NCT04346238 ·Status: COMPLETED
-
A Gene Therapy Study in Patients With Gaucher Disease Type 1
NCT05324943 ·Status: COMPLETED ·Phase: PHASE1
-
Long-Term Follow-up of Subjects Who Were Treated With ST-920
NCT05039866 ·Status: ENROLLING_BY_INVITATION
-
A Study to Characterize the Cardiac Phenotype of Individuals With Friedreich's Ataxia (CARFA Study)
NCT02840669 ·Status: COMPLETED ·Phase: NA
-
A Study to Investigate the Effects and Safety of SPG601 for the Treatment of Fragile X Syndrome in Male Participants
NCT07439510 ·Status: NOT_YET_RECRUITING ·Phase: PHASE2/PHASE3
-
Evaluation of the Long-term Safety, Pharmacodynamics, and Exploratory Efficacy of GZ/SAR402671 in Treatment-Naïve Adult Male Patients With Fabry Disease
NCT02489344 ·Status: COMPLETED ·Phase: PHASE2
-
Open Label, Study Of Efficacy and Safety Of AVR-RD-01 for Treatment-Naive Subjects With Classic Fabry Disease
NCT03454893 ·Status: TERMINATED ·Phase: PHASE1/PHASE2
-
FRIEDREICH ATAXIA- STEROIDOGENESIS
NCT07123142 ·Status: COMPLETED
-
A Fabry Disease Gene Therapy Study
NCT04040049 ·Status: TERMINATED ·Phase: PHASE1/PHASE2
-
GM1 and GM2 Gangliosidosis PROspective Neurological Disease TrajectOry Study (PRONTO)
NCT05109793 ·Status: COMPLETED
-
Friedreich Ataxia Nerve Ultrasund
NCT07508631 ·Status: RECRUITING
-
Evaluate the Safety and Preliminary Efficacy of EXG110 in Subjects With Fabry Disease
NCT06539624 ·Status: RECRUITING ·Phase: NA
-
Long-Term Follow-up Study of Subjects With Fabry Disease Who Received Lentiviral Gene Therapy in Study AVRO-RD-01-201
NCT04999059 ·Status: TERMINATED
-
Gene Therapy for Gaucher's and Fabry Disease Using Viruses and Blood-Forming Cells
NCT00001234 ·Status: COMPLETED ·Phase: PHASE1
-
Clinical Study of AAV1-gamma-sarcoglycan Gene Therapy for Limb Girdle Muscular Dystrophy Type 2C
NCT01344798 ·Status: COMPLETED ·Phase: PHASE1
-
Natural History, Genetic Bases and Phenotype-genotype Correlations in Autosomal Dominant Spinocerebellar Degenerations
NCT00136630 ·Status: COMPLETED
-
Implementation and Evaluation of a Rare Disease Algorithm to Identify Persons at Risk of Gaucher Disease Using Data From Electronic Health Records (EHRs) in the United States (Project Searchlight)
NCT05908656 ·Status: COMPLETED ·Phase: NA
-
Long Read Analysis in Spinal Muscular Atrophy - LOREASI
NCT07332702 ·Status: RECRUITING ·Phase: NA
-
Monitoring of Early Disease Progression in Hereditary Transthyretin Amyloidosis
NCT03431896 ·Status: COMPLETED
-
Safety and Efficacy Study of Fx-1006A in Patients With Familial Amyloidosis
NCT00409175 ·Status: COMPLETED ·Phase: PHASE2/PHASE3
-
Sun May Arise on SMA : Newborn Screening of Spinal Muscular Atrophy in Belgium
NCT03554343 ·Status: COMPLETED
-
Evaluation of the Safety and Efficacy of Late-onset Pompe Disease Gene Therapy Drug
NCT06391736 ·Status: RECRUITING ·Phase: PHASE1/PHASE2
-
Fabry and Cardiomyopathy (FaCard)
NCT01429597 ·Status: WITHDRAWN
-
Analysis of Oculo-motor Deficiencies Associated With FMR1 Gene Expression (Genetic Abnormality Predisposing to a Neurodegenerative Disease)
NCT00763191 ·Status: TERMINATED ·Phase: NA
-
Epidemiological Study of Fabry Disease in Taiwan Young Stroke Patients
NCT03596398 ·Status: UNKNOWN