Testing effectiveness (Phase 2)WithdrawnNCT05665036What this trial is testingSafety and Efficacy of Encapsulated Allogeneic MPS-1 TherapyWho this might be right forMPS IMPS1-SMPS1-HS Sigilon Therapeutics, Inc.
Large-scale testing (Phase 3)Study completedNCT02432144What this trial is testingUX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Therapy in Subjects With Mucopolysaccharidosis Type 7, Sly Syndrome (MPS 7)Who this might be right forSly SyndromeMPS VIIMucopolysaccharidosis+1 more Ultragenyx Pharmaceutical Inc 12
Large-scale testing (Phase 3)Enrolling By InvitationNCT05594992What this trial is testingAn Extension Study of JR-141 to Evaluate the Long-term Safety and Efficacy in MPS II (Hunter Syndrome) SubjectsWho this might be right forMucopolysaccharidosis II JCR Pharmaceuticals Co., Ltd. 80
Not applicableStudy completedNCT01938014What this trial is testingLysosomal Storage Disease: Health, Development, and Functional Outcome Surveillance in Preschool ChildrenWho this might be right forMucopolysaccharidosis Type I (MPS I)Mucopolysaccharidosis Type II (MPS II)Mucopolysaccharidosis Type III (MPS III)+2 more University of Chicago 19
Large-scale testing (Phase 3)Study completedNCT00146770What this trial is testingPhase 3 Extension Study of the Safety and Efficacy of Aldurazyme® (Laronidase) in Mucopolysaccharidosis I (MPS I) PatientsWho this might be right forMucopolysaccharidosis IHurler's SyndromeHurler-Scheie Syndrome+1 more Genzyme, a Sanofi Company 45
Testing effectiveness (Phase 2)Study completedNCT02418455What this trial is testingStudy of UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Treatment in Mucopolysaccharidosis Type 7, Sly Syndrome (MPS 7) Patients Less Than 5 Years of AgeWho this might be right forSly SyndromeMPS VIIMucopolysaccharidosis+1 more Ultragenyx Pharmaceutical Inc 8
Large-scale testing (Phase 3)UnknownNCT03612869What this trial is testingStudy of AAVrh10-h.SGSH Gene Therapy in Patients With Mucopolysaccharidosis Type IIIA (MPS IIIA)Who this might be right forMucopolysaccharidosis Type IIIA LYSOGENE 20
Not applicableUnknownNCT04637646What this trial is testingEvaluation of Cardiac Affections in Patients With Mucopolysaccharidosis (MPS) in Assuit University Children Hospital (AUCH)Who this might be right forMucopolysaccharidoses Assiut University 25
Not applicableUnknownNCT03227042What this trial is testingA Prospective Natural History Study of Mucopolysaccharidosis Type IIIB (MPS IIIB)Who this might be right forMucopolysaccharidosis Type IIIB Allievex Corporation 44
Not applicableStudy completedNCT00144794What this trial is testingMucopolysaccharidosis I (MPS I) RegistryWho this might be right forMucopolysaccharidosis I (MPS I) Genzyme, a Sanofi Company 1,406
Early research (Phase 1)Active Not RecruitingNCT05422482What this trial is testingEvaluate the Safety, Tolerability, PK and PD of Intracerebroventricular GC1123 in Patients with MPS ⅡWho this might be right forMucopolysaccharidosis IIHunter Syndrome GC Biopharma Corp 12
Large-scale testing (Phase 3)Active Not RecruitingNCT07236606What this trial is testingRGX-121-3102 Gene Therapy in Participants With MPS II (Hunter Syndrome)Who this might be right forMPS IIHunter Syndrome (MPS II) REGENXBIO Inc. 2
Testing effectiveness (Phase 2)Study completedNCT00884949What this trial is testingEvaluate the Safety, Tolerability and Efficacy of BMN 110 in Subjects With Mucopolysaccharidosis IVAWho this might be right forMPS IV A BioMarin Pharmaceutical 20
Testing effectiveness (Phase 2)Ended earlyNCT02618512What this trial is testingA Open Label Study in Previously Studied, SBC-103 Treatment Naïve MPS IIIB Subjects to Investigate the Safety, Pharmacokinetics, and Pharmacodynamics/Efficacy of SBC-103 Administered IntravenouslyWho this might be right forMucopolysaccharidosis III, Type B (MPS IIIB)Sanfilippo B Alexion Pharmaceuticals, Inc. 3
Not applicableLooking for participantsNCT03604835What this trial is testingMucopolysaccharidosis VII Disease Monitoring ProgramWho this might be right forMucopolysaccharidosis VIIMPS VIIMPS 7+1 more Ultragenyx Pharmaceutical Inc 50
Large-scale testing (Phase 3)Study completedNCT02055118What this trial is testingStudy of Intrathecal Idursulfase-IT Administered in Conjunction With Elaprase® in Pediatric Patients With Hunter Syndrome and Early Cognitive ImpairmentWho this might be right forHunter Syndrome Shire 58
Not applicableLooking for participantsNCT06036693What this trial is testingMPS (RaDiCo Cohort) (RaDiCo-MPS)Who this might be right forMucopolysaccharidosis IMucopolysaccharidosis IIMucopolysaccharidosis III+5 more Institut National de la Santé Et de la Recherche Médicale, France 1,000
Not applicableWithdrawnNCT02298712What this trial is testingBiomarker for Hurler Disease (BioHurler)Who this might be right forMucopolysaccharidosis Type IGargoylismMetabolism, Inborn Errors CENTOGENE GmbH Rostock
Early research (Phase 1)Study completedNCT01173016What this trial is testingAdministration of IV Laronidase Post Bone Marrow Transplant in HurlerWho this might be right forHurler Syndrome Masonic Cancer Center, University of Minnesota 11
Testing effectiveness (Phase 2)Study completedNCT02060526What this trial is testingRandomized, Controlled, Open-label, Multicenter, Safety and Efficacy Study of rhHNS Administration Via an IDDD in Pediatric Patients With Early Stage MPS IIIA DiseaseWho this might be right forSanfilippo Syndrome Shire 21