Testing effectiveness (Phase 2)Study completedNCT03370653What this trial is testingA Study in MPS VI to Assess Safety and Efficacy of OdiparcilWho this might be right forMucopolysaccharidosis VI Inventiva Pharma 20
Not applicableEnrolling By InvitationNCT03655223What this trial is testingEarly Check: Expanded Screening in NewbornsWho this might be right forSpinal Muscular AtrophyFragile X SyndromeFragile X - Premutation+182 more RTI International 30,000
Large-scale testing (Phase 3)Ended earlyNCT00654433What this trial is testingALD-101 Adjuvant Therapy of Unrelated Umbilical Cord Blood Transfusion (UCBT) in Patients With Inherited Metabolic DiseasesWho this might be right forInherited Metabolic DiseasesLysosomal Storage DisordersPeroxisomal Storage Diseases+2 more Aldagen 40
Testing effectiveness (Phase 2)Study completedNCT00176917What this trial is testingStem Cell Transplantation for HurlerWho this might be right forMucopolysaccharidosis IMucopolysaccharidosis VIMannosidosis+1 more Masonic Cancer Center, University of Minnesota 41
Not applicableStudy completedNCT01707433What this trial is testingDiagnosis of Mucopolysaccharidosis Disorders in Patients Presenting With Bilateral Hip DiseaseWho this might be right forMucopolysaccharidosis IV AMucopolysaccharidosis VI Children's Hospitals and Clinics of Minnesota 22
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
Testing effectiveness (Phase 2)Ended earlyNCT00668564What this trial is testingHematopoietic Stem Cell Transplantation (HCT) for Inborn Errors of MetabolismWho this might be right forHurler's SyndromeMaroteaux-Lamy SyndromeSly Syndrome+8 more Masonic Cancer Center, University of Minnesota 18
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
Post-approval studies (Phase 4)Study completedNCT00299000What this trial is testingTwo Dose Level Study of Naglazyme(TM) (Galsulfase) in Infants With MPS VIWho this might be right forMucopolysaccharidosis VIMaroteaux-Lamy Syndrome BioMarin Pharmaceutical 4
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)Study completedNCT01586455What this trial is testingHuman Placental-Derived Stem Cell TransplantationWho this might be right forMucopolysaccharidosis IMucopolysaccharidosis VIAdrenoleukodystrophy+13 more New York Medical College 43
Large-scale testing (Phase 3)Study completedNCT00067470What this trial is testingStudy of Recombinant Human N-acetylgalactosamine 4-sulfatase (rhASB) in Patients With MPS VIWho this might be right forMucopolysaccharidosis VI BioMarin Pharmaceutical 39
Large-scale testing (Phase 3)Study completedNCT00104234What this trial is testingStudy of rhASB in Patients With Mucopolysaccharidosis VIWho this might be right forMucopolysaccharidosis VI BioMarin Pharmaceutical 39
Testing effectiveness (Phase 2)Study completedNCT00048711What this trial is testingOpen-Label Study of Efficacy and Safety of Recombinant Human N-acetylgalactosamine 4-sulfatase in Patients With MPS VIWho this might be right forMucopolysaccharidosis VI BioMarin Pharmaceutical
Not applicableStudy completedNCT01586871What this trial is testingCarotid Structure and Function in MPS Syndromes: A Multicenter Study of the Lysosomal Disease NetworkWho this might be right forMucopolysaccharidoses University of Minnesota 1,147
Not applicableEnded earlyNCT02156674What this trial is testingNaglazyme After Allo Transplant for Maroteaux-Lamy SyndromeWho this might be right forMaroteaux-Lamy Syndrome Masonic Cancer Center, University of Minnesota 1
Early research (Phase 1)Study completedNCT00048620What this trial is testingStudy of Recombinant Human N-Acetylgalactosamine 4-Sulfatase in Patients With MPS VIWho this might be right forMucopolysaccharidosis VI BioMarin Pharmaceutical
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 applicableUnknownNCT00005900What this trial is testingStudy of Pulmonary Complications in Pediatric Patients With Storage Disorders Undergoing Allogeneic Hematopoietic Stem Cell TransplantationWho this might be right forI Cell DiseaseFucosidosisGloboid Cell Leukodystrophy+9 more Fairview University Medical Center 10
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