Not applicableEnrolling By InvitationNCT06103487What this trial is testingLong Term Follow-Up for RGX-111Who this might be right forMucopolysaccharidosis I REGENXBIO Inc. 21
Not applicableLooking for participantsNCT07361536What this trial is testingCardiac Structure and Function in MPSWho this might be right forMPS IMPS IV AMPS - Mucopolysaccharidosis+1 more Children's Hospital of Orange County 240
Large-scale testing (Phase 3)Study completedNCT00912925What this trial is testingClinical Study of Aldurazyme in Patients With Mucopolysaccharidosis (MPS) IWho this might be right forMucopolysaccharidosis IHurlers SyndromeHurler-Scheie Syndrome Genzyme, a Sanofi Company 45
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 applicableEnded earlyNCT04628871What 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
Early research (Phase 1)Ended earlyNCT00215527What this trial is testingIntrathecal Enzyme Replacement Therapy for Spinal Cord Compression in Mucopolysaccharidosis (MPS) IWho this might be right forMucopolysaccharidosis ILysosomal Storage DiseasesSpinal Cord Compression Patricia I. Dickson, M.D. 4
Not applicableStudy completedNCT05073783What this trial is testingAssess the Safety of Myozyme® and of Aldurazyme® in Male and Female Participants of Any Age Group With Pompe Disease or With Mucopolysaccharidosis Type I (MPS I) in a Home-care SettingWho this might be right forPompe DiseaseMucopolysaccharidosis Type I (MPS I) Sanofi 57
Not applicableStudy completedNCT00144794What this trial is testingMucopolysaccharidosis I (MPS I) RegistryWho this might be right forMucopolysaccharidosis I (MPS I) Genzyme, a Sanofi Company 1,406
Testing effectiveness (Phase 2)Study completedNCT04453085What this trial is testingAn Extension Study of JR-171-101 Study in Patients With Mucopolysaccharidosis Type I (MPS I)Who this might be right forMucopolysaccharidosis I JCR Pharmaceuticals Co., Ltd. 14
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