Not applicableStudy completedNCT02851862What this trial is testingA Natural History of Late Onset Tay-Sachs DiseaseWho this might be right forGM2 Gangliosidosis Massachusetts General Hospital 10
Testing effectiveness (Phase 2)Study completedNCT00283959What this trial is testingA 12-Week Safety and Pharmacodynamic Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry DiseaseWho this might be right forFabry Disease Amicus Therapeutics 4
Testing effectiveness (Phase 2)Study completedNCT03580122What this trial is testingThe Effect of Arginine on Classic GalactosemiaWho this might be right forClassic Galactosemia Academisch Ziekenhuis Maastricht 4
Not applicableStudy completedNCT00005479What this trial is testingDiabetes, Lipoproteins and Accelerated Vascular DiseaseWho this might be right forCardiovascular DiseasesHeart DiseasesCoronary Disease+4 more Columbia University 164
Not applicableUnknownNCT04388969What this trial is testingWorld Data on Ambroxol for Patients With GD and GBA Related PDWho this might be right forGaucher DiseaseParkinson DiseaseGBA Gene Mutation Shaare Zedek Medical Center 300
Not applicableUnknownNCT03806894What this trial is testingClopidogrel Resistance in Stroke Patients From Different EthnicitiesWho this might be right forClopidogrel, Poor Metabolism ofIschemic Stroke Ziv Hospital 300
Not applicableLooking for participantsNCT06094205What this trial is testingFeasibility of the BrainGate2 Neural Interface System in Persons With Tetraplegia (BG-Speech-02)Who this might be right forAnarthriaDysarthriaTetraplegia+5 more Leigh R. Hochberg, MD, PhD. 2
Not applicableStudy completedNCT04353466What this trial is testingAssessing the Impact of Elelyso on Bone Involvement Currently Treated With Other ERTsWho this might be right forGaucher Disease, Type 1 Shaare Zedek Medical Center 30
Not applicableWithdrawnNCT02385162What this trial is testingBiomarker for Glycogen Storage Diseases (BioGlycogen)Who this might be right forFructose Metabolism, Inborn ErrorsGlycogen Storage DiseaseGlycogen Storage Disease Type I+7 more CENTOGENE GmbH Rostock
Not applicableEnded earlyNCT02360046What this trial is testingThe Influence of Different Hydrocortisone Replacement Doses on the Partitioning and Flexibility of Ectopic Lipids in Patients With Corticotropic HypopituitarismWho this might be right forHypopituitarismHydrocortisoneLipids+2 more Insel Gruppe AG, University Hospital Bern 30
Testing effectiveness (Phase 2)Study completedNCT00925054What this trial is testingDose-Finding Study to Evaluate the Safety, Efficacy, & Tolerability of Multiple Doses of rAvPAL-PEG in Subjects With PKUWho this might be right forPhenylketonuria BioMarin Pharmaceutical 40
Large-scale testing (Phase 3)Study completedNCT02319005What this trial is testingENDEAVOUR: Phase 3 Multicenter Study of Revusiran (ALN-TTRSC) in Patients With Transthyretin (TTR) Mediated Familial Amyloidotic Cardiomyopathy (FAC)Who this might be right forTransthyretin (TTR) Mediated Familial Amyloidotic Cardiomyopathy (FAC)Amyloidosis, HereditaryAmyloid Neuropathies, Familial+3 more Alnylam Pharmaceuticals 206
Not applicableStudy completedNCT00005151What this trial is testingPawtucket Heart Health ProgramWho this might be right forCardiovascular DiseasesHeart DiseasesHypercholesterolemia+5 more National Heart, Lung, and Blood Institute (NHLBI)
Not applicableStudy completedNCT01196156What this trial is testingFollow-up to Adult Height of a Cohort of Subjects Born Small for the Gestational Age and Treated With Growth HormoneWho this might be right forInfant, Small for Gestational Age Merck KGaA, Darmstadt, Germany 443
Not applicableUnknownNCT02795910What this trial is testingIntegrated Primary Care for Diabetes and Cardiovascular DiseaseWho this might be right forDiabetes MellitusHypertensionHeart Failure+2 more University of Sao Paulo General Hospital 40,577
Testing effectiveness (Phase 2)Study completedNCT02771366What this trial is testingThe Efficient Brain StudyWho this might be right forMetabolism - Cerebral Energy University of Florida 30
Testing effectiveness (Phase 2)Ended earlyNCT01195753What this trial is testingHuman Heterologous Liver Cells for Infusion in Children With Urea Cycle DisordersWho this might be right forUrea Cycle Disorders Cytonet GmbH & Co. KG 10
Not applicableUnknownNCT06367127What this trial is testingUtility of the Clamping Bean Test (CBT) for Covert Hepatic Encephalopathy ScreeningWho this might be right forHepatic EncephalopathyCovert Hepatic Encephalopathy Shanghai Changzheng Hospital 200
Large-scale testing (Phase 3)Study completedNCT00697073What this trial is testingStudy to Assess the Safety and Tolerability of Idebenone in the Treatment of Friedreich's Ataxia PatientsWho this might be right forFriedreich's Ataxia Santhera Pharmaceuticals 68
Not applicableUnknownNCT02868905What this trial is testingIdentification of Epigenetic Markers Common to Obesity and Alzheimer's Disease in WomenWho this might be right forObesityAlzheimer's Disease Central Hospital, Nancy, France 160