Not applicableStudy completedNCT03292887What this trial is testingHunter Outcome Survey (HOS)Who this might be right forHunter Syndrome Shire 1,443
Post-approval studies (Phase 4)Study completedNCT02455622What this trial is testingLong-term Evaluation on Height and Weight in Patients With MPS II Who Started Treatment at < 6 Years of AgeWho this might be right forHunter Syndrome Shire 21
Not applicableStudy completedNCT03529786What this trial is testingMucopolysaccharidosis Type II Natural HistoryWho this might be right forMucopolysaccharidosis II REGENXBIO Inc. 36
Large-scale testing (Phase 3)Active Not RecruitingNCT06031259What this trial is testingExtension Study of Idursulfase-IT Along With Elaprase in Children and Adults With Hunter Syndrome and Cognitive ImpairmentWho this might be right forHunter Syndrome Takeda 6
Not applicableStudy completedNCT03582449What this trial is testingIntensive Pharmacovigilance Program for Elaprase (SHP ELA-701)Who this might be right forHunter Syndrome Shire 5
Large-scale testing (Phase 3)Active Not RecruitingNCT04573023What this trial is testingA Phase III Study of JR-141 in Patients With Mucopolysaccharidosis II (STARLIGHT)Who this might be right forMucopolysaccharidosis II JCR Pharmaceuticals Co., Ltd. 86
Testing effectiveness (Phase 2)Active Not RecruitingNCT03153319What this trial is testingStudy to Evaluate the Safety and Efficacy of Adalimumab in MPS I, II, and VIWho this might be right forMucopolysaccharidosis IMucopolysaccharidosis IIMucopolysaccharidosis VI Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center 14
Testing effectiveness (Phase 2)Study completedNCT04571970What this trial is testingRGX-121 Gene Therapy in Children 5 Years of Age and Over With MPS II (Hunter Syndrome)Who this might be right forMucopolysaccharidosis Type II (MPS II) REGENXBIO Inc. 6
Not applicableStudy completedNCT05687474What this trial is testingBaby Detect : Genomic Newborn ScreeningWho this might be right forCongenital Adrenal HyperplasiaFamilial Hyperinsulinemic Hypoglycemia 1Phosphoglucomutase 1 Deficiency+121 more Centre Hospitalier Universitaire de Liege 6,824
Not applicableStudy completedNCT06022380What this trial is testingCore Outcome Set for Head, Neck and Respiratory Disease in Mucopolysaccharidosis IIWho this might be right forMucopolysaccharidosis Type II Manchester University NHS Foundation Trust 46
Not applicableStudy completedNCT01602601What this trial is testingTest the Possibility of Cross Reaction Induced by the Idursulfase Drug to GSK2788723Who this might be right forMucopolysaccharidosis II GlaxoSmithKline 10
Not applicableEnded earlyNCT04628871What this trial is testingLong Term Follow-up (LTFU) of Subjects Who Received SB-318, SB-913, or SB-FIXWho this might be right forHemophilia BMucopolysaccharidosis IMucopolysaccharidosis II Sangamo Therapeutics 13
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
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)Study completedNCT02412787What this trial is testingStudy of Long Term Safety and Clinical Outcomes of Idursulfase IT and Elaprase Treatment in Pediatric Participants Who Have Completed Study HGT-HIT-094Who this might be right forHunter Syndrome Shire 56
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
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