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TrialConditionsStatusPhaseUpdatedMore infoNCT ID
A Gaucher Disease Gene Therapy Trial With FLT201Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Gaucher Disease, Type 1, Gaucher disease, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Lysosomal Storage Diseases, Nervous System, Nutritional and Metabolic Diseases, brain disorder, central nervous system disorder, hereditary disease, inborn errors of metabolism, lysosomal lipid storage disorder, lysosomal storage disease, metabolic disease, nervous system disorder, sphingolipidosisRecruitingPhase 32026-09-03NCT07223944
RCT of Implantable Defibrillators in Patients With Non Ischemic Cardiomyopathy, Scar and Severe Systolic Heart Failurecardiovascular disorder, heart disorder, heart failureRecruitingNot applicable2026-06-16NCT05568069
Study of VGA039 in Healthy Volunteers and Patients With Von Willebrand Disease (VIVID)Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Hemic and Lymphatic Diseases, blood coagulation disease, blood platelet disease, cardiovascular disorder, coagulation protein disease, hematologic disorder, hemorrhagic disease, hereditary disease, inherited blood coagulation disorder, vascular disorder, vascular hemostatic disease, von Willebrand disease (hereditary or acquired)RecruitingPhase 1 / Phase 22025-08-26NCT05776069