Not applicableEnrolling By InvitationNCT06103487What this trial is testingLong Term Follow-Up for RGX-111Who this might be right forMucopolysaccharidosis I REGENXBIO Inc. 7
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 applicableStudy completedNCT01521429What this trial is testingLongitudinal Study of Bone Disease in Children with Mucopolysaccharidoses (MPS) I, II, and VIWho this might be right forMucopolysaccharidoses Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center 55
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
Testing effectiveness (Phase 2)Study completedNCT00146757What this trial is testingA Study Evaluating the Safety and Pharmacokinetics of Aldurazyme® (Laronidase) in MPS I Patients Less Than 5 Years OldWho this might be right forMucopolysaccharidosis IHurler SyndromeHurler-Scheie Syndrome+1 more Genzyme, a Sanofi Company 20
Post-approval studies (Phase 4)Study completedNCT05134571What this trial is testingChina Post-marketing Surveillance (PMS) Study of Aldurazyme®Who this might be right forMucopolysaccharidosis I Genzyme, a Sanofi Company 12
Large-scale testing (Phase 3)Ended earlyNCT00748969What this trial is testingClinical Trial of Growth Hormone in MPS I, II, and VIWho this might be right forMucopolysaccharidosis IMucopolysaccharidosis IIMucopolysaccharidosis VI Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center 2
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
Not applicableLooking for participantsNCT00144794What this trial is testingMucopolysaccharidosis I (MPS I) RegistryWho this might be right forMucopolysaccharidosis I (MPS I) Genzyme, a Sanofi Company 1,500
Not applicableStudy completedNCT00695279What this trial is testingLong Term Follow Up Of Patients Who Have Received Gene Therapy Or Gene Marked ProductsWho this might be right forSevere Combined ImmunodeficiencyMalignancy, HematologicNeuroblastoma+2 more St. Jude Children's Research Hospital 41
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
Large-scale testing (Phase 3)Looking for participantsNCT02716246What this trial is testingPhase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSHWho this might be right forMPS IIIASanfilippo SyndromeSanfilippo A+1 more Ultragenyx Pharmaceutical Inc 36
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 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
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 applicableStudy completedNCT03161171What this trial is testingParental Coping With Challenging Behavior in Mucopolysaccharidosis Type I-IIIWho this might be right forMucopolysaccharidosis Type IMucopolysaccharidosis Type IIMucopolysaccharidosis Type III+2 more University Hospital Heidelberg 34
Testing effectiveness (Phase 2)Study completedNCT02324049What this trial is testingSafety, Pharmacokinetics, and Pharmacodynamics/Efficacy of SBC-103 in Mucopolysaccharidosis III, Type B (MPS IIIB)Who this might be right forMucopolysaccharidosis IIIB Alexion Pharmaceuticals, Inc. 11
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
Testing effectiveness (Phase 2)Ended earlyNCT02702115What this trial is testingAscending Dose Study of Genome Editing by the Zinc Finger Nuclease (ZFN) Therapeutic SB-318 in Subjects With MPS IWho this might be right forMPS I Sangamo Therapeutics 3
Not applicableEnded earlyNCT01675674What this trial is testingStudy to Detect Unrecognized Mucopolysaccharidosis in Children Visiting Rheumatology, Hand or Skeletal Dysplasia ClinicsWho this might be right forMucopolysaccharidosesMucopolysaccharidosis IMucopolysaccharidosis II+2 more National MPS Society 3,000