Not applicableWithdrawnNCT02298673What this trial is testingBiomarker for Mucolipidosis Disorder Type I, II, III, IV (BioML)Who this might be right forSkeletal AbnormalitiesPsychomotor Retardation CENTOGENE GmbH Rostock
Not applicableStudy completedNCT00015782What this trial is testingThe Natural History and Pathogenesis of Mucolipidosis Type IVWho this might be right forMucolipidosis Type IV National Institute of Neurological Disorders and Stroke (NINDS) 30
Not applicableStudy completedNCT01416467What this trial is testingCharacterization of the Patient Population With GalactosialidosisWho this might be right forGalactosialidosis St. Jude Children's Research Hospital 3
Testing effectiveness (Phase 2)Study completedNCT00383448What this trial is testingHSCT for High Risk Inherited Inborn ErrorsWho this might be right forAdrenoleukodystrophyMetachromatic LeukodystrophyGloboid Cell Leukodystrophy+6 more Masonic Cancer Center, University of Minnesota 38
Not applicableLooking for participantsNCT06316752What this trial is testingThe Natural History of Sialidosis Type IWho this might be right forObservational Study National Taiwan University Hospital 30
Not applicableLooking for participantsNCT00029965What this trial is testingNatural History of Glycosphingolipid Storage Disorders and Glycoprotein DisordersWho this might be right forNeurological RegressionMyoclonusCherry Red Spot+1 more National Human Genome Research Institute (NHGRI) 200
Testing effectiveness (Phase 2)Study completedNCT00176917What this trial is testingStem Cell Transplantation for HurlerWho this might be right forMucopolysaccharidosis IMucopolysaccharidosis VIMannosidosis+1 more Masonic Cancer Center, University of Minnesota 41
Not applicableUnknownNCT00005900What this trial is testingStudy of Pulmonary Complications in Pediatric Patients With Storage Disorders Undergoing Allogeneic Hematopoietic Stem Cell TransplantationWho this might be right forI Cell DiseaseFucosidosisGloboid Cell Leukodystrophy+9 more Fairview University Medical Center 10
Not applicableEnded earlyNCT01067742What this trial is testingThe Natural History of Mucolipidosis Type IVWho this might be right forMucolipidosis Type IV Baylor Research Institute 7
Not applicableUnknownNCT04624789What this trial is testingRegistry GangliosidosesWho this might be right forGangliosidosesGM1 GangliosidosisMorquio B Disease+6 more SphinCS Lyso Gemeinnutzige UG (Haftungsbeschrankt) 40
Early research (Phase 1)Enrolling By InvitationNCT07398872What this trial is testingSafety and Efficacy of AAV9. hMCOLN1co For Patients With Mucolipidosis Type IVWho this might be right forMucolipidosis Type IV The Children's Hospital of Zhejiang University School of Medicine 1
Large-scale testing (Phase 3)Ended earlyNCT00654433What this trial is testingALD-101 Adjuvant Therapy of Unrelated Umbilical Cord Blood Transfusion (UCBT) in Patients With Inherited Metabolic DiseasesWho this might be right forInherited Metabolic DiseasesLysosomal Storage DisordersPeroxisomal Storage Diseases+2 more Aldagen 40
Not applicableStudy completedNCT01891422What this trial is testingLongitudinal Studies of the GlycoproteinosesWho this might be right forAspartylglucosaminuriaFucosidosisGalactosialidosis+6 more Greenwood Genetic Center 100
Not applicableLooking for participantsNCT05782387What this trial is testingMucolipidosis Type IV Natural History StudyWho this might be right forMucolipidosis Type IV Massachusetts General Hospital 50
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 applicableStudy completedNCT01626092What this trial is testingReduced-Intensity Hematopoietic Stem Cell Transplant for High Risk Lysosomal and Peroxisomal DisordersWho this might be right forLysosomal Storage DiseasePeroxisomal Disorder Masonic Cancer Center, University of Minnesota 3
Not applicableLooking for participantsNCT01793168What this trial is testingRare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at SanfordWho this might be right forRare DisordersUndiagnosed DisordersDisorders of Unknown Prevalence+340 more Sanford Health 20,000