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TrialConditionsStatusPhaseUpdatedMore infoNCT ID
Study of LX2006 Gene Therapy in Friedreich Ataxia CardiomyopathyCardiomegaly, Cardiomyopathies, Secondary, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Friedreich ataxia, Hypertrophy, Hypertrophy, Left Ventricular, Nutritional and Metabolic Diseases, Pathological Conditions, Anatomical, Pathological Conditions, Signs and Symptoms, Spinocerebellar Degenerations, aortic valve disorder, aortic valve stenosis, brain disorder, cardiomyopathy, cardiovascular disorder, central nervous system disorder, cerebellar disorder, heart disorder, heart valve disorder, hereditary disease, hypertrophic cardiomyopathy, inborn mitochondrial metabolism disorder, inherited neurodegenerative disorder, metabolic disease, nervous system disorder, neurodegenerative disease, spinal cord disorder, subvalvular aortic stenosisRecruitingPhase 22026-09-02NCT07721025
Clinical Course Of Disease In Participants With FA-CMCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Friedreich ataxia, Nutritional and Metabolic Diseases, Spinocerebellar Degenerations, brain disorder, cardiomyopathy, cardiovascular disorder, central nervous system disorder, cerebellar disorder, heart disorder, hereditary disease, inborn mitochondrial metabolism disorder, inherited neurodegenerative disorder, metabolic disease, nervous system disorder, neurodegenerative disease, spinal cord disorderRecruitingNot stated2026-09-02NCT06865482
Gene Therapy for Cardiomyopathy Associated With Friedreich's AtaxiaCardiomyopathies, Secondary, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Friedreich ataxia, Nutritional and Metabolic Diseases, Spinocerebellar Degenerations, brain disorder, cardiomyopathy, cardiovascular disorder, central nervous system disorder, cerebellar disorder, heart disorder, hereditary disease, inborn mitochondrial metabolism disorder, inherited neurodegenerative disorder, metabolic disease, nervous system disorder, neurodegenerative disease, spinal cord disorderActive, not recruitingPhase 1 / Phase 22025-12-23NCT05445323
Long-term Follow-up Study of Gene Therapy for Arrhythmogenic Cardiomyopathy Due to a Plakophilin-2 Pathogenic VariantCardiovascular Abnormalities, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Death, Death, Sudden, Death, Sudden, Cardiac, Pathologic Processes, Pathological Conditions, Signs and Symptoms, arrhythmogenic right ventricular cardiomyopathy, cardiac arrest, cardiomyopathy, cardiovascular disorder, congenital heart disease, heart disorderEnrolling by invitationNot stated2025-11-12NCT07050160
Gene Therapy for ACM Due to a PKP2 Pathogenic VariantCardiovascular Abnormalities, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Death, Death, Sudden, Death, Sudden, Cardiac, Pathologic Processes, Pathological Conditions, Signs and Symptoms, arrhythmogenic right ventricular cardiomyopathy, cardiac arrest, cardiomyopathy, cardiovascular disorder, congenital heart disease, heart disorderActive, not recruitingPhase 1 / Phase 22025-11-12NCT06109181
Long-Term Follow-up of Gene Therapy for APOE4 Homozygote Alzheimer's DiseaseAlzheimer disease, brain disorder, central nervous system disorder, cognitive disorder, dementia, nervous system disorder, neurodegenerative disease, psychiatric disorder, tauopathyActive, not recruitingPhase 12025-07-01NCT05400330
A Study to Assess Real-world Patient Characteristics and Clinical Course for Symptomatic Patients With PKP2-ACMCardiovascular Abnormalities, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Death, Death, Sudden, Death, Sudden, Cardiac, Pathologic Processes, Pathological Conditions, Signs and Symptoms, arrhythmogenic right ventricular cardiomyopathy, cardiac arrest, cardiomyopathy, cardiovascular disorder, congenital heart disease, heart disorderRecruitingNot stated2025-05-16NCT06976606