Not applicableEnrolling By InvitationNCT06103487What this trial is testingLong Term Follow-Up for RGX-111Who this might be right forMucopolysaccharidosis I REGENXBIO Inc. 7
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
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 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)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
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
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
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
Early research (Phase 1)Study completedNCT00638547What this trial is testingIntrathecal Enzyme Replacement for Hurler SyndromeWho this might be right forHurler Syndrome Masonic Cancer Center, University of Minnesota 26
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
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
Early research (Phase 1)Study completedNCT01917708What this trial is testingBone Marrow Transplant With Abatacept for Non-Malignant DiseasesWho this might be right forHurler SyndromeFanconi AnemiaGlanzmann Thrombasthenia+12 more Emory University 10
Not applicableNo Longer AvailableNCT03639844What this trial is testingBPX-501 T Cells Infused Post Stem Cell Transplant in Pediatrics With Non-Malignant Disorders Ineligible for BPU004 StudyWho this might be right forHurler SyndromeInherited Metabolic DisorderLysosomal Storage Disorder+2 more Bellicum Pharmaceuticals
Not applicableEnded earlyNCT04008849What this trial is testingA Follow-up Study in Patients With Inherited Metabolic Disorders (IMD) Who Underwent Hematopoietic Stem Cell Transplantation (HSCT) With MGTA-456Who this might be right forInherited Metabolic Disorders (IMD) Magenta Therapeutics, Inc. 3
Not applicableWithdrawnNCT00286689What this trial is testingEffects of Growth Hormone in Chronically Ill ChildrenWho this might be right forHurler SyndromeCerebral PalsyJuvenile Rheumatoid Arthritis+2 more University of Texas Southwestern Medical Center
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
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 applicableWithdrawnNCT02298712What this trial is testingBiomarker for Hurler Disease (BioHurler)Who this might be right forMucopolysaccharidosis Type IGargoylismMetabolism, Inborn Errors CENTOGENE GmbH Rostock
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
Not applicableLooking for participantsNCT04528355What this trial is testingData Collection Study of Patients With Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT With RICWho this might be right forPrimary Immunodeficiency (PID)Congenital Bone Marrow Failure SyndromesInherited Metabolic Disorders (IMD)+2 more Paul Szabolcs 50