Not applicableAvailableNCT05795361What this trial is testingPost-trial Access Program of Idursulfase-IT Along With Elaprase in Children With Hunter SyndromeWho this might be right forHunter Syndrome Takeda
Not applicableNot Yet RecruitingNCT07666269What this trial is testingMorphology in Oral Rare Syndromes & Artificial Intelligence for Clinical DiagnosisWho this might be right forOsteogenesis ImperfectaRare Bone DisordersHypophosphatemia+6 more University Hospital, Bordeaux 240
Not applicableStudy completedNCT06064656What this trial is testingA Non-Interventional Study of Clinical Characteristics and Mortality of US Patients With Fibrodysplasia Ossificans Progressiva (FOP)Who this might be right forFibrodysplasia Ossificans Progressiva (FOP) Regeneron Pharmaceuticals 131
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
Large-scale testing (Phase 3)Enrolling By InvitationNCT06075537What this trial is testingAn Extension Study of the Long-Term Safety, Tolerability, and Efficacy of Tividenofusp Alfa (DNL310) in Participants With Mucopolysaccharidosis Type II (MPS II) From Study DNLI-E-0002 or Study DNLI-E-0007Who this might be right forMucopolysaccharidosis II Denali Therapeutics Inc. 99
Large-scale testing (Phase 3)Active Not RecruitingNCT05208281What this trial is testingA Multi-cohort Study of Safety, Efficacy, PK and PD of GNR-055 in Patients With Mucopolysaccharidosis Type IIWho this might be right forMucopolysaccharidosis Type IIMetabolic Diseases AO GENERIUM 32
Testing effectiveness (Phase 2)Ended earlyNCT01609062What this trial is testingSafety and Exercise Study of Two Doses of BMN 110 for Morquio A SyndromeWho this might be right forMucopolysaccharidosis IVAMorquio A SyndromeMPS IVA BioMarin Pharmaceutical 25
Not applicableStudy completedNCT02936713What this trial is testingEffect of Controlled Diet Combined With a Fermented Milk Product on Gas-related SymptomsWho this might be right forFunctional Gastrointestinal Disorders Danone Global Research & Innovation Center 126
Testing effectiveness (Phase 2)Ended earlyNCT03315182What this trial is testingGene Transfer Clinical Trial for Mucopolysaccharidosis (MPS) IIIBWho this might be right forMucopolysaccharidosis Type 3 B Abeona Therapeutics, Inc 11
Not applicableAvailableNCT03775174What this trial is testingExpanded Access to MepseviiWho this might be right forMPS VIIMucopolysaccharidosis VIISly Syndrome Ultragenyx Pharmaceutical Inc
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
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
Not applicableStudy completedNCT03292887What this trial is testingHunter Outcome Survey (HOS)Who this might be right forHunter Syndrome Shire 1,443
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
Testing effectiveness (Phase 2)Study completedNCT01043640What this trial is testingAllogeneic Bone Marrow Transplant for Inherited Metabolic DisordersWho this might be right forMucopolysaccharidosisHurler SyndromeHunter Syndrome+10 more Masonic Cancer Center, University of Minnesota 46
Testing effectiveness (Phase 2)Study completedNCT01155778What this trial is testingSafety, Tolerability, Ascending Dose and Dose Frequency Study of rhHNS Via an IDDD in MPS IIIA PatientsWho this might be right forMucopolysaccharidosis (MPS) Shire 12
Not applicableStudy completedNCT03150069What this trial is testingPregnancy With Morquio Syndrome - What Are Patients' Perspectives and Has ERT Changed Them?Who this might be right forMorquio DiseaseMPS - Mucopolysaccharidosis Emory University 18
Not applicableNot Yet RecruitingNCT07712003What this trial is testingNatural History to Assess Disease in Patients With MPS IIICWho this might be right forSanfilippo Syndrome Type CMucopolysaccharidosis (MPS) IIIC Phoenix Nest 35
Not applicableUnknownNCT06177847What this trial is testingEfficacy of BIS Monitoring in Deep Hypnotic StateWho this might be right forHypnotic; Withdrawal State With DeliriumNeurological DisorderPostoperative Delirium+4 more Universitas Indonesia 50
Large-scale testing (Phase 3)Study completedNCT01645189What this trial is testingSafety and Efficacy of HunteraseWho this might be right forHunter Syndrome Green Cross Corporation 6