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
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 participantsNCT00144794What this trial is testingMucopolysaccharidosis I (MPS I) RegistryWho this might be right forMucopolysaccharidosis I (MPS I) Genzyme, a Sanofi Company 1,500
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
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 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
Testing effectiveness (Phase 2)Active Not RecruitingNCT02171104What this trial is testingMT2013-31: Allo HCT for Metabolic Disorders and Severe OsteopetrosisWho this might be right forMucopolysaccharidosis DisordersHurler SyndromeHunter Syndrome+26 more Masonic Cancer Center, University of Minnesota 149
Early research (Phase 1)Looking for participantsNCT04532047What this trial is testingPEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders)Who this might be right forMPS IMPS IIMPS IVA+6 more University of California, San Francisco 10
Not applicableActive Not RecruitingNCT04628871What this trial is testingLong Term Follow-up (LTFU) of Subjects Who Received SB-318, SB-913, or SB-FIXWho this might be right forHemophilia BMucopolysaccharidosis IMucopolysaccharidosis II Sangamo Therapeutics 13
Testing effectiveness (Phase 2)Study completedNCT00730314What this trial is testingUnrelated Hematopoietic Stem Cell Transplantation(HSCT) for Genetic Diseases of Blood CellsWho this might be right forSickle Cell DiseaseThalassemiaAnemia+9 more Children's Hospital Los Angeles 25
Not applicableUnknownNCT04958070What this trial is testingThe Intensively Follow-up Examinations for Asymptomatic MPS I Infants in TaiwanWho this might be right forMPS I Mackay Memorial Hospital 20
Testing effectiveness (Phase 2)Ended earlyNCT01372228What this trial is testingPhase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic DisordersWho this might be right forHurler Syndrome (MPS I)Hurler-Scheie SyndromeHunter Syndrome (MPS II)+9 more Talaris Therapeutics Inc. 3
Early research (Phase 1)Study completedNCT00692926What this trial is testingUnrelated Umbilical Cord Blood Transplantation Augmented With ALDHbr Umbilical Cord Blood CellsWho this might be right forMDSAnemia, AplasticInborn Errors of Metabolism+2 more Joanne Kurtzberg, MD 37
Not applicableStudy completedNCT03576729What this trial is testingMRS to Determine Neuroinflammation and Oxidative Stress in MPS IWho this might be right forMucopolysaccharidosis Type I University of Minnesota 30