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 applicableLooking for participantsNCT05537753What this trial is testingEncore PFO Closure Device - The PerFOrm TrialWho this might be right forPatent Foramen OvaleCryptogenic Stroke Encore Medical Inc. 500
Not applicableUnknownNCT03315637What this trial is testingFetal Endoscopic Surgery for Spina BifidaWho this might be right forNeural Tube DefectsSpina Bifida, OpenMyelomeningocele+5 more Hospital Universitari Vall d'Hebron Research Institute 50
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 applicableLooking for participantsNCT07099651What this trial is testingAutosomal Dominant Spinocerebellar Ataxias and Social CognitionWho this might be right forAutosomal Dominant Spinocerebellar Ataxia (SCA1, 2,3,6,7,27B) University Hospital, Angers 160
Not applicableStudy completedNCT03358823What this trial is testingStudy on the Brain Network of Angelman SyndromeWho this might be right forAngelman Syndrome Fudan University 150
Not applicableUnknownNCT03901261What this trial is testingCohort Study of Adult Patients With Down Syndrome at Risk of Developing Alzheimer's Disease (TriAL21)Who this might be right forDown SyndromeAlzheimer Disease Institut Jerome Lejeune 200
Not applicableStudy completedNCT01983670What this trial is testingThe Investigation of the Pre-movement Facilitation of Agonist-antagonist Muscles and the Effect of the Feedforward Rehabilitation in Individuals With HypermetriaWho this might be right forSpinocerebellar Atrophy (SCA) Chang Gung University 40
Early research (Phase 1)Study completedNCT00101270What this trial is testingOxaliplatin and Irinotecan in Treating Young Patients With Refractory Solid Tumors or LymphomasWho this might be right forChildhood Burkitt LymphomaChildhood Central Nervous System Germ Cell TumorChildhood Diffuse Large Cell Lymphoma+26 more National Cancer Institute (NCI) 24
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
Testing effectiveness (Phase 2)Looking for participantsNCT04753970What this trial is testingRetina is a Marker for Cerebrovascular HeathWho this might be right forCerebral Small Vessel DiseasesCerebral Autosomal Dominant Arteriopathy With Subcortical Infarcts and LeukoencephalopathyCerebral Microbleeding+1 more Mayo Clinic 100
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
Large-scale testing (Phase 3)Study completedNCT00206323What this trial is testingA Randomized, Placebo-controlled, Tourette Syndrome Study.Who this might be right forTourette Syndrome Baylor College of Medicine 29
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 applicableUnknownNCT05790473What this trial is testingArtificial Intelligence Algorithm for the Screening of Abnormal Fetal Brain Findings at First Trimester Ultrasound ScanWho this might be right forFetal AnomalyBrain Malformation Fondazione Policlinico Universitario Agostino Gemelli IRCCS 10,000