Not applicableStudy completedNCT04163848What this trial is testingCARbon Impact of aNesthesic GasWho this might be right forGas; InhalationAnesthesia Ciusss de L'Est de l'Île de Montréal 58
Not applicableStudy completedNCT02090179What this trial is testingEvaluation of Blood Brain Barrier Integrity and Structural Abnormalities in MPS IIIB Patients Using Multimodal Magnetic Resonance ImagingWho this might be right forMPS IIIB (Sanfilippo B Syndrome) Alexion Pharmaceuticals, Inc. 5
Testing effectiveness (Phase 2)Active Not RecruitingNCT03488394What this trial is testingGene Therapy With Modified Autologous Hematopoietic Stem Cells for the Treatment of Patients With Mucopolysaccharidosis Type I, Hurler VariantWho this might be right forMucopolysaccharidosis IH Orchard Therapeutics 8
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 applicableUnknownNCT01695161What this trial is testingNon-invasive Assessment of Intraocular Pressure in MPS by Use of the Ocular Response Analyzer.Who this might be right forMucopolysaccharidosisFabry Disease Johannes Gutenberg University Mainz 75
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
Post-approval studies (Phase 4)Study completedNCT05019560What this trial is testingAwareness in Old Aged Patients During Laryngoscopy and Intubation Using Isolated Forearm TechniqueWho this might be right forAwareness, AnesthesiaRecall PhenomenonInhalation; Gas Ain Shams University 50
Not applicableStudy completedNCT02746341What this trial is testingNatural History Study of Patients With MPS IIIAWho this might be right forMucopolysaccharidosis IIIA LYSOGENE 23
Not applicableLooking for participantsNCT06551961What this trial is testingEffect of Chronic Feeding of Inulin And Methylcellulose on Colonic FermentationWho this might be right forIrritable Bowel Syndrome University of Nottingham 35
Early research (Phase 1)Study completedNCT02371226What this trial is testingSafety and Dose Ranging Study of Insulin Receptor MAb-IDUA Fusion Protein in Patients With MPS IWho this might be right forMucopolysaccharidosis I ArmaGen, Inc 3
Not applicableUnknownNCT06002373What this trial is testingAssessment of Artificial Intelligence for Treatment Decision Recommendation of Adult Skeletal Class III PatientsWho this might be right forSkeletal DysplasiaReverse Overjet Cairo University 150
Not applicableLooking for participantsNCT05619900What this trial is testingRegistry of Patients Diagnosed With Lysosomal Storage DiseasesWho this might be right forMucopolysaccharidosis IMucopolysaccharidosis IIMucopolysaccharidosis IV A+5 more University of California, San Francisco 250
Not applicableStudy completedNCT01873911What this trial is testingNeurobehavioral Phenotypes in MPS IIIWho this might be right forSanfilippo Syndrome Type ASanfilippo Syndrome Type BHurler Syndrome University of Minnesota 30
Post-approval studies (Phase 4)Ended earlyNCT00418821What this trial is testingThe Effect of Aldurazyme® (Laronidase) Treatment on Lactation in Female Patients With Mucopolysaccharidosis I (MPS I) and Their Breastfed InfantsWho this might be right forMucopolysaccharidosis IHurler's SyndromeHurler-Scheie Syndrome+1 more Genzyme, a Sanofi Company 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
Not applicableEnded earlyNCT01733615What this trial is testingDiscovering New Biomarkers For Monitoring Disease Progression in Patients With Mucopolysaccharidosis IVAWho this might be right forMucopolysaccharidosis Type IVA Emory University 3
Testing effectiveness (Phase 2)Looking for participantsNCT01962415What this trial is testingReduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCTWho this might be right forPrimary Immunodeficiency (PID)Congenital Bone Marrow Failure SyndromesInherited Metabolic Disorders (IMD)+4 more Paul Szabolcs 100
Post-approval studies (Phase 4)Enrolling By InvitationNCT05492799What this trial is testingSafety, Tolerability and Efficacy of ICV AX 250 Treatment in MPS IIIB -OLEWho this might be right forMPS III B Allievex Corporation 15
Not applicableStudy completedNCT02208661What this trial is testingPsychological Concomitants of Morquio A Syndrome - Longitudinal Effects of Enzyme Replacement Therapy (The MAPLE Study)Who this might be right forMorquio A SyndromeMucopolysaccharidosis IV A Nadia Ali, PhD 12
Early research (Phase 1)Active Not RecruitingNCT06567769What 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