Not applicableLooking for participantsNCT03564561What this trial is testingNatural History of Pompe DiseaseWho this might be right forGlycogen Storage Disease Type II, Adult Assistance Publique - Hôpitaux de Paris 20
Not applicableUnknownNCT02000310What this trial is testingMolecular and Cellular Mechanisms of Lysosomal Storage DiseasesWho this might be right forLysosomal Storage Disorders O & O Alpan LLC 80
Not applicableEnded earlyNCT04008849What this trial is testingA Follow-up Study in Patients With Inherited Metabolic Disorders (IMD) Who Underwent Hematopoietic Stem Cell Transplantation (HSCT) With MGTA-456Who this might be right forInherited Metabolic Disorders (IMD) Magenta Therapeutics, Inc. 3
Post-approval studies (Phase 4)Study completedNCT00144768What this trial is testingA Study Investigating the Relationship Between the Development of Laronidase Antibody and Urinary GAG (Glycosaminoglycan) Levels in Aldurazyme® Treated PatientsWho this might be right forMucopolysaccharidosis IHurler's SyndromeHurler-Scheie Syndrome+1 more Genzyme, a Sanofi Company 25
Not applicableActive Not RecruitingNCT04848779What this trial is testingA Prospective Study to Observe & Describe Clinical Outcomes of Alglucosidase Alfa Treatment in Patients ≤6 Months of Age With Infantile-onset Pompe Disease (IOPD)Who this might be right forGlycogen Storage Disease Type II Sanofi 16
Testing effectiveness (Phase 2)WithdrawnNCT02715505What this trial is testingSafety and Exploratory Efficacy of HSC835 in Patients With Inherited Metabolic Disorders (IMD)Who this might be right forInherited Metabolic Disorders IMD Novartis Pharmaceuticals
Large-scale testing (Phase 3)Study completedNCT01275066What this trial is testingStudy to Evaluate the Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)Who this might be right forMPS IV A BioMarin Pharmaceutical 177
Not applicableUnknownNCT04476550What this trial is testingClinical Specimen Collection From Pompe Disease PatientsWho this might be right forPompe Disease Serhat Gumrukcu, MD PhD 12
Not applicableLooking for participantsNCT06396546What this trial is testing'Glycogen Storage Diseases (GSDs) in Indian Children- Establishing an Indian GSD (I-GSD) Registry'Who this might be right forGlycogen Storage Diseases Institute of Liver and Biliary Sciences, India 250
Not applicableLooking for participantsNCT06539169What this trial is testingFLOWER: Following Longitudinal Outcomes With Epidemiology for Rare DiseasesWho this might be right forAlpha-ThalassemiaBeta-ThalassemiaAmyloidosis+14 more xCures 1,000
Not applicableStudy completedNCT04081272What this trial is testingEffect of G6PD Deficiency on Red Blood Cell StorageWho this might be right forG6PD Deficiency Columbia University 40
Not applicableLooking for participantsNCT06967831What this trial is testingDrug Repurposing for Mitochondrial Disorders Using iPSCs Derived Neural CellsWho this might be right forLeigh Syndrome (Maternally Inherited, MILS)Leigh Syndrome (AR, AD, XR) Charite University, Berlin, Germany 80
Early research (Phase 1)Looking for participantsNCT06567769What this trial is testingPhase 1 Study of GC1130A in Patients With Sanfilippo Syndrome Type A (MPS IIIA)Who this might be right forSanfilippo Syndrome Type A GC Biopharma Corp 9
Testing effectiveness (Phase 2)Study completedNCT03370653What this trial is testingA Study in MPS VI to Assess Safety and Efficacy of OdiparcilWho this might be right forMucopolysaccharidosis VI Inventiva Pharma 20
Not applicableActive Not RecruitingNCT05634512What this trial is testingEvaluation of Intravenous Laronidase Pharmacokinetics Before and After Hematopoietic Cell Transplantation in Patients With Mucopolysaccharidosis Type IH.Who this might be right forHematopoietic Cell TransplantationMucopolysaccharidosis Type I Masonic Cancer Center, University of Minnesota 13
Not applicableLooking for participantsNCT07239856What this trial is testingGoat vs. Cow Milk Digestive ToleranceWho this might be right forMilk IntoleranceMilk HypersensitivityCow Milk Protein Sensitivity Institut National de Recherche pour l'Agriculture, l'Alimentation et l'Environnement 20
Large-scale testing (Phase 3)Study completedNCT01145339What this trial is testingA Non-inferiority, Multicenter and Randomized, Single-Dose Study About a Treatment to Hypolactasia (LAILAI)Who this might be right forHypolactasia Eurofarma Laboratorios S.A. 140
Not applicableNot Yet RecruitingNCT06931379What this trial is testingScreening of Lactose Intolerance Among IBS Patients Diarrhea DominantWho this might be right forLactose Intolerance Assiut University 70
Post-approval studies (Phase 4)Study completedNCT02878876What this trial is testingIncidence of Lactose Intolerance Among Self-reported Lactose Intolerant PeopleWho this might be right forLactose Intolerance a2 Milk Company Ltd. 600
Not applicableLooking for participantsNCT05031507What this trial is testingStudy of Skeletal DisordersWho this might be right forSkeletal Disorders Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) 100