Not applicableWithdrawnNCT01284257What this trial is testingObservational Registry Study of Renal Transplant PatientsWho this might be right forKidney Transplant Novartis Pharmaceuticals
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 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
Testing effectiveness (Phase 2)UnknownNCT03632213What this trial is testingEvaluation of Losartan on Cardiovascular Disease in Patients With Mucopolysaccharidoses IV A and VIWho this might be right forMucopolysaccharidosis IV AMucopolysaccharidosis VIMucopolysaccharidoses+6 more Hospital de Clinicas de Porto Alegre 10
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
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 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
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 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
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
Not applicableLooking for participantsNCT03333200What this trial is testingLongitudinal Study of Neurodegenerative DisordersWho this might be right forMLDKrabbe DiseaseALD+24 more University of Pittsburgh 1,500
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
Large-scale testing (Phase 3)Study completedNCT01415427What this trial is testingLong-Term Efficacy and Safety Extension Study of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)Who this might be right forMucopolysaccharidosis IV AMorquio A SyndromeMPS IVA BioMarin Pharmaceutical 173
Large-scale testing (Phase 3)Study completedNCT01966029What this trial is testingBMN 110 Phase 3B in Australian PatientsWho this might be right forMucopolysaccharidosis IVA (Morquio A Syndrome) BioMarin Pharmaceutical 13
Not applicableStudy completedNCT01961518What this trial is testingScreening an Orthopedic Population for Mildly-affected Individuals With Morquio Syndrome A and Maroteaux-Lamy SyndromeWho this might be right forMorquio Syndrome AMaroteaux Lamy SyndromeMPS IVA+1 more Greenwood Genetic Center 17
Not applicableStudy completedNCT01870375What this trial is testingLongitudinal Studies of Brain Structure and Function in MPS DisordersWho this might be right forMucopolysaccharidosis Type IMucopolysaccharidosis Type IIMucopolysaccharidosis Type VI+2 more University of Minnesota 100
Not applicableLooking for participantsNCT05284006What this trial is testingNon-invasive Functional Assessment and Pathogenesis of Morquio AWho this might be right forMucopolysaccharidosis IV Type AMorquio A SyndromeMPS IVA Nemours Children's Clinic 60
Not applicableEnded earlyNCT00787995What this trial is testingA Clinical Assessment Study of Subjects With Mucopolysaccharidosis IVA (Morquio Syndrome)Who this might be right forMPS IV AMucopolysaccharidosis IVAMorquio A Syndrome BioMarin Pharmaceutical 353
Not applicableStudy completedNCT01920828What this trial is testingGait Analysis in MPS IVAWho this might be right forMPS IVAMorquio Syndrome Oregon Health and Science University 10
Testing effectiveness (Phase 2)Study completedNCT01515956What this trial is testingStudy of BMN 110 in Pediatric Patients < 5 Years of Age With Mucopolysaccharidosis IVA (Morquio A Syndrome)Who this might be right forMucopolysaccharidosis IVAMorquio A SyndromeMPS IVA BioMarin Pharmaceutical 15