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TrialConditionsStatusPhaseUpdatedMore infoNCT ID
A Phase 1 AAV Gene Therapy Trial Evaluating Safety and Preliminary Efficacy of RP-A701 in Subjects With BAG3 Dilated CardiomyopathyCardiomegaly, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dilatation, Pathologic, Pathological Conditions, Anatomical, Pathological Conditions, Signs and Symptoms, cardiomyopathy, cardiovascular disorder, dilated cardiomyopathy, heart disorder, heart failure, hereditary disease, laminopathyRecruitingPhase 12026-10-01NCT07137338
A Study Observing Disease Progression in a Patient With Hutchinson-Gilford Progeria Syndrome (HGPS)Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Nutritional and Metabolic Diseases, hereditary disease, inborn errors of metabolism, laminopathy, metabolic disease, progeroid syndromeNot yet recruitingNot stated2026-09-24NCT07839351
Observatoire Des Patients Atteints de Laminopathies et Emerinopathies (Observatory for PAtients With Laminopathies and Emerinopathies)Autosomal Emery-Dreifuss Muscular Dystrophy, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Emery-Dreifuss Muscular Dystrophy 1, Emery-Dreifuss muscular dystrophy, Muscular Disorders, Atrophic, X-linked Emery-Dreifuss muscular dystrophy, X-linked disease, hereditary disease, laminopathy, muscle tissue disorder, muscular dystrophy, musculoskeletal system disorder, nervous system disorder, neuromuscular diseaseRecruitingNot stated2026-09-21NCT03058185
Observational Study of Natural History of BAG3 Gene Mutation-Associated Dilated Cardiomyopathy in Chinese AdultsCardiomegaly, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, cardiomyopathy, cardiovascular disorder, dilated cardiomyopathy, heart disorder, hereditary disease, laminopathyRecruitingNot stated2026-09-16NCT07646600
Efficacy and Safety of Mibavademab in Adult and Pediatric Patients With Generalized LipodystrophyCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Nutritional and Metabolic Diseases, Skin Diseases, Metabolic, Skin and Connective Tissue Diseases, congenital generalized lipodystrophy, familial partial lipodystrophy, hereditary disease, inborn errors of metabolism, laminopathy, lipodystrophy, metabolic disease, skin disorderRecruitingPhase 32026-09-11NCT07220785
Creating Stem Cells From a Progeria Patient's Blood to Test Candidate Treatments in the LabCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Nutritional and Metabolic Diseases, hereditary disease, inborn errors of metabolism, laminopathy, metabolic disease, progeroid syndromeNot yet recruitingNot stated2026-09-10NCT07803432
RHYthm and Myocardial Function Relationship Evaluation in Heart DiseasesArrhythmias, Cardiac, Cardiomegaly, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Pathologic Processes, Pathological Conditions, Signs and Symptoms, atrial fibrillation, cardiomyopathy, cardiovascular disorder, dilated cardiomyopathy, heart disorder, heart failure, hereditary disease, laminopathyRecruitingNot stated2026-09-10NCT07491237
A Study of AZD4063 in PLN R14del Dilated CardiomyopathyCardiomegaly, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, cardiomyopathy, cardiovascular disorder, dilated cardiomyopathy, heart disorder, hereditary disease, laminopathyRecruitingPhase 12026-09-04NCT07241104
Sexual Quality of Life in Female Patients With Lipodystrophy Syndromes or Turner's Syndrome: a Case-control StudyCardiovascular Abnormalities, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Male Urogenital Diseases, Nutritional and Metabolic Diseases, Sex Chromosome Disorders, Skin Diseases, Metabolic, Skin and Connective Tissue Diseases, Turner syndrome, Urogenital Abnormalities, Urogenital Diseases, cardiovascular disorder, chromosomal disorder, congenital heart disease, disorder of sexual differentiation, endocrine system disorder, familial partial lipodystrophy, gonadal disorder, gonadal dysgenesis, heart disorder, hereditary disease, laminopathy, lipodystrophy, metabolic disease, sex chromosome disorder of sex development, skin disorderNot yet recruitingNot stated2026-09-02NCT07799012
Natural History Study in Pediatric Patients With MYBPC3 Mutation-associated CardiomyopathyCardiomegaly, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, aortic valve disorder, aortic valve stenosis, cardiomyopathy, cardiovascular disorder, dilated cardiomyopathy, heart disorder, heart valve disorder, hereditary disease, hypertrophic cardiomyopathy, laminopathy, restrictive cardiomyopathy, subvalvular aortic stenosisActive, not recruitingNot stated2026-09-02NCT05112237
An AAV Gene Therapy Trial of AFTX-201 in Adults With BAG3-Associated Dilated Cardiomyopathy (DCM)Cardiomegaly, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, cardiomyopathy, cardiovascular disorder, dilated cardiomyopathy, heart disorder, hereditary disease, laminopathyRecruitingPhase 1 / Phase 22026-09-01NCT07426419
Study of the Progression of Chronic Cardiovascular ConditionsCardiomegaly, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Ventricular Outflow Obstruction, aortic valve disorder, aortic valve stenosis, cardiomyopathy, cardiovascular disorder, dilated cardiomyopathy, familial dilated cardiomyopathy, heart disorder, heart failure, heart valve disorder, hereditary disease, hypertensive disorder, hypertrophic cardiomyopathy, laminopathy, subvalvular aortic stenosis, vascular disorderRecruitingNot stated2026-08-12NCT07420907
Performance of the Cardiac Microcurrent (C-MIC) System With a Less Invasively Placed Left Ventricular LeadCardiomegaly, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, cardiomyopathy, cardiovascular disorder, dilated cardiomyopathy, heart disorder, heart failure, hereditary disease, idiopathic cardiomyopathy, laminopathyRecruitingNot applicable2026-08-11NCT06920030
Identification of Women With Severe Insulin Resistant Syndromes of Genetic Origin Among Patients With "Classic" Polycystic Ovary Syndrome (PCOS)Adnexal Diseases, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Cysts, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Genital Diseases, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Nutritional and Metabolic Diseases, Skin Diseases, Metabolic, Skin and Connective Tissue Diseases, Urogenital Diseases, endocrine system disorder, familial partial lipodystrophy, female reproductive system disorder, gonadal disorder, hereditary disease, laminopathy, lipodystrophy, metabolic disease, neoplasm, ovarian cyst, ovarian disorder, polycystic ovary syndrome, skin disorderRecruitingNot applicable2026-08-10NCT07412028
A Study With NVC-001 in Patients With LMNA-related Dilated Cardiomyopathy (SUNBEAM-LMNA)Cardiomegaly, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, cardiomyopathy, cardiovascular disorder, dilated cardiomyopathy, heart disorder, hereditary disease, laminopathyRecruitingPhase 1 / Phase 22026-08-07NCT07606274
MyocarditIRM-Génétique - Genetic Sub-study of the French Cohort of Acute Myocarditis Cases Diagnosed by MRICardiomegaly, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, acute myocarditis, cardiomyopathy, cardiovascular disorder, dilated cardiomyopathy, heart disorder, hereditary disease, laminopathyActive, not recruitingNot stated2026-07-21NCT07717073
Open-label Extension Study to Evaluate Metreleptin in Patients With Partial LipodystrophyCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Nutritional and Metabolic Diseases, Skin Diseases, Metabolic, Skin and Connective Tissue Diseases, familial partial lipodystrophy, hereditary disease, laminopathy, lipodystrophy, metabolic disease, skin disorderRecruitingPhase 32026-07-16NCT06679270
Safety and Efficacy of FAP iCDC in End-stage Dilated CardiomyopathyCardiomegaly, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, cardiomyopathy, cardiovascular disorder, dilated cardiomyopathy, heart disorder, heart failure, hereditary disease, laminopathyRecruitingPhase 1 / Phase 22026-07-14NCT06902896
the Clinical Outcome of Empagliflozin in Pediatric Heart Failure PatientsCardiomegaly, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, cardiomyopathy, cardiovascular disorder, dilated cardiomyopathy, heart disorder, heart failure, hereditary disease, laminopathyRecruitingPhase 22026-07-09NCT07692256
Safety and Efficacy of Second Infusion of FAP iCDC in End-stage Dilated CardiomyopathyCardiomegaly, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, cardiomyopathy, cardiovascular disorder, dilated cardiomyopathy, heart disorder, heart failure, hereditary disease, laminopathyRecruitingPhase 12026-07-02NCT07516288
A Phase 1 Open-Label Study of the Safety of Intravenous Allogeneic Neonatal Mesenchymal Cells (nMSCs) in Young Adult (1A) and Pediatric (1B) Patients With Dilated Cardiomyopathy (DCM)Cardiomegaly, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, cardiomyopathy, cardiovascular disorder, dilated cardiomyopathy, heart disorder, hereditary disease, laminopathyRecruitingPhase 12026-06-26NCT06464588
Pediatric Cardiomyopathy Mutation AnalysisCardiomegaly, Cardiovascular Abnormalities, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Hypertrophy, Pathological Conditions, Anatomical, Pathological Conditions, Signs and Symptoms, aortic valve disorder, aortic valve stenosis, arrhythmogenic right ventricular cardiomyopathy, cardiomyopathy, cardiovascular disorder, congenital heart disease, dilated cardiomyopathy, heart disorder, heart failure, heart valve disorder, hereditary disease, hypertrophic cardiomyopathy, laminopathy, left ventricular noncompaction, restrictive cardiomyopathy, subvalvular aortic stenosisRecruitingNot stated2026-06-22NCT02432092
A Study About the Natural History in Adults With BAG3 Dilated Cardiomyopathy (a Type of Heart Disease) (BAG3 DCM)Cardiomegaly, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, cardiomyopathy, cardiovascular disorder, dilated cardiomyopathy, heart disorder, hereditary disease, laminopathyActive, not recruitingNot stated2026-06-12NCT05981092
AI-Enabled Direct-from-ECG Ejection Fraction (EF) Severity Assessment Using COR ECG Wearable MonitorArrhythmias, Cardiac, Atrial Remodeling, Cardiomegaly, Cardiotoxicity, Chemically-Induced Disorders, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Consciousness Disorders, Drug-Related Side Effects and Adverse Reactions, Infarction, Necrosis, Neurobehavioral Manifestations, Neurologic Manifestations, Pathologic Processes, Pathological Conditions, Anatomical, Pathological Conditions, Signs and Symptoms, Signs and Symptoms, Syncope, Unconsciousness, Ventricular Dysfunction, Ventricular Dysfunction, Left, cardiomyopathy, cardiovascular disorder, dilated cardiomyopathy, heart disorder, heart failure, heart valve disorder, hereditary disease, injury, ischemic disease, laminopathy, myocardial infarction, myocardial ischemia, nervous system disorder, radiation injury, vascular disorderRecruitingNot stated2026-06-01NCT06699056
Mainstreaming Genetic Testing for Non-Ischemic Cardiomyopathy in Western CanadaCardiomegaly, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, cardiomyopathy, cardiovascular disorder, dilated cardiomyopathy, heart disorder, hereditary disease, laminopathyEnrolling by invitationNot applicable2026-05-08NCT07345338