Not applicableStudy completedNCT01194141What this trial is testingExercise Training in Barth SyndromeWho this might be right forBarth Syndrome Washington University School of Medicine 4
Early research (Phase 1)Study completedNCT01380431What this trial is testingA Comparative Bioavailability Study of Lisinopril Tablets, 40 mg - Effect of Food StudyWho this might be right forTo Determine Bioequivalence Under Fed Conditions Par Pharmaceutical, Inc. 18
Not applicableStudy completedNCT00343577What this trial is testingAntiproteinuric Agents and Fabry DiseaseWho this might be right forFabry DiseaseProteinuria University of Alabama at Birmingham 12
Not applicableUnknownNCT01448577What this trial is testingStudy to Re-assess and Re-confirm Data Previously Recorded About the Incidence and Severity of Acute Abdominal "Pancreatitis" Episodes in Lipoprotein Lipase Deficient (LPLD) Subjects Previously Enrolled on AMT Clinical StudiesWho this might be right forLipoprotein Lipase Deficiency Amsterdam Molecular Therapeutics 22
Testing effectiveness (Phase 2)Study completedNCT01102686What this trial is testingPyrimethamine as a Treatment for Late-Onset GM2-gangliosidosis (Tay-Sachs and Sandhoff Disease)Who this might be right forGangliosidoses, GM2Sandhoff DiseaseTay-Sachs Disease The Hospital for Sick Children 20
Testing effectiveness (Phase 2)Ended earlyNCT04273269What this trial is testingA Safety and Efficacy Study of LYS-GM101 Gene Therapy in Patients With GM1 GangliosidosisWho this might be right forGM1 Gangliosidosis LYSOGENE 5
Not applicableStudy completedNCT00351156What this trial is testingStudy to Evaluate Blood Cell Lines From Patients With Gaucher DiseaseWho this might be right forGaucher Disease Amicus Therapeutics 50
Early research (Phase 1)Study completedNCT02422654What this trial is testingTaste Evaluation of Different Liquid Formulations With EliglustatWho this might be right forGaucher Disease Genzyme, a Sanofi Company 8
Not applicableLooking for participantsNCT04613089What this trial is testingNatural History and Longitudinal Clinical Assessments in NCL / Batten Disease, the International DEM-CHILD DatabaseWho this might be right forNeuronal Ceroid LipofuscinosisBatten DiseaseCLN1 Disease+12 more Universitätsklinikum Hamburg-Eppendorf 500
Testing effectiveness (Phase 2)Active Not RecruitingNCT05228145What this trial is testingGene Therapy Study for Children With CLN5 Batten DiseaseWho this might be right forNeuronal Ceroid Lipofuscinosis CLN5 Neurogene Inc. 6
Not applicableLooking for participantsNCT05473637What this trial is testingTaiwan Associated Genetic and Nongenetic Small Vessel DiseaseWho this might be right forCerebral Small Vessel DiseasesCadasilHTRA1-Related Autosomal Dominant Cerebral Angiopathy+5 more National Taiwan University Hospital 500
Large-scale testing (Phase 3)Study completedNCT05238519What this trial is testingImproved Diagnosis of Familial Hypercholesterolemia Across the Northland (ID-FH)Who this might be right forFamilial HypercholesterolemiaCholesterol, ElevatedGenetic Disease Essentia Health 257
Early research (Phase 1)WithdrawnNCT00679744What this trial is testingA Phase I Study of Pyrimethamine in Patients With GM2 GangliosidosisWho this might be right forG(M2) GangliosideTay-Sachs Disease GangliosideSandhoff Disease Ganglioside Exsar Corporation
Early research (Phase 1)Study completedNCT01554800What this trial is testingEffect of ACP-501 on Safety, Tolerability, Pharmacokinetics and Pharmacodynamics in Subjects With Coronary Artery DiseaseWho this might be right forCoronary Artery DiseaseAtherosclerosis MedImmune LLC 16
Large-scale testing (Phase 3)Study completedNCT04031742What this trial is testingEvaluate Safety and Efficacy of IBI306, a PCSK9 Monoclonal Antibody in Chinese Subjects With Homozygous Familial HypercholesterolemiaWho this might be right forHomozygous Familial Hypercholesterolemia Innovent Biologics (Suzhou) Co. Ltd. 18
Not applicableUnknownNCT05719337What this trial is testingMulti-Modality Echocardiographic Techniques in Pathological Left Ventricular Hypertrophy AdultsWho this might be right forLeft Ventricular Hypertrophy First Hospital of China Medical University 660
Large-scale testing (Phase 3)Study completedNCT05529992What this trial is testingVelaglucerase Alfa (VPRIV) in Chinese Children, Teenagers, and Adults With Type 1 Gaucher DiseaseWho this might be right forGaucher Disease Takeda 20
Not applicableWithdrawnNCT00018694What this trial is testingCholestanol in HumansWho this might be right forCerebrotendinous Xanthomatosis US Department of Veterans Affairs
Testing effectiveness (Phase 2)Study completedNCT02767531What this trial is testingOrlistat for the Treatment of Type I HyperlipoproteinemiaWho this might be right forHyperlipoproteinemia Type IHypertriglyceridemia University of Texas Southwestern Medical Center 2
Not applicableActive Not RecruitingNCT04101149What this trial is testingGenetic Causes of Familial HypercholesterolemiaWho this might be right forFamilial Hypercholesterolemia Region Örebro County 150