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
Large-scale testing (Phase 3)Looking for participantsNCT05371613What this trial is testingDetermine the Efficacy and Safety of Tividenofusp Alfa (DNL310) vs Idursulfase in Pediatric and Young Adult Participants With Neuronopathic (nMPS II) or Non-Neuronopathic Mucopolysaccharidosis Type II (nnMPS II)Who this might be right forMucopolysaccharidosis II Denali Therapeutics Inc. 63
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)Study completedNCT01645189What this trial is testingSafety and Efficacy of HunteraseWho this might be right forHunter Syndrome Green Cross Corporation 6
Large-scale testing (Phase 3)Study completedNCT03568175What this trial is testingJR-141 in Patients With Mucopolysaccharidosis IIWho this might be right forMucopolysaccharidosis II JCR Pharmaceuticals Co., Ltd. 28
Early research (Phase 1)Study completedNCT06475404What this trial is testingThe Tolerance, Safety, and Pharmacokinetics of GNR-055 in Healthy VolunteersWho this might be right forMucopolysaccharidosis Type IIMetabolic Diseases AO GENERIUM 18
Large-scale testing (Phase 3)Active Not RecruitingNCT06031259What this trial is testingExtension Study of Idursulfase-IT Along With Elaprase in Children and Adults With Hunter Syndrome and Cognitive ImpairmentWho this might be right forHunter Syndrome Takeda 6
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
Testing effectiveness (Phase 2)Study completedNCT01515956What this trial is testingStudy of BMN 110 in Pediatric Patients < 5 Years of Age With Mucopolysaccharidosis IVA (Morquio A Syndrome)Who this might be right forMucopolysaccharidosis IVAMorquio A SyndromeMPS IVA BioMarin Pharmaceutical 15
Large-scale testing (Phase 3)Enrolling By InvitationNCT05594992What this trial is testingAn Extension Study of JR-141 to Evaluate the Long-term Safety and Efficacy in MPS II (Hunter Syndrome) SubjectsWho this might be right forMucopolysaccharidosis II JCR Pharmaceuticals Co., Ltd. 80
Testing effectiveness (Phase 2)Active Not RecruitingNCT03708965What this trial is testingAn Extension Study of JR-141-BR21 in Patients With Mucopolysaccharidosis IIWho this might be right forMucopolysaccharidosis II JCR Pharmaceuticals Co., Ltd. 19
Testing effectiveness (Phase 2)Study completedNCT00884949What this trial is testingEvaluate the Safety, Tolerability and Efficacy of BMN 110 in Subjects With Mucopolysaccharidosis IVAWho this might be right forMPS IV A BioMarin Pharmaceutical 20
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
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 RecruitingNCT05665166What this trial is testingGene Therapy With Modified Autologous Hematopoietic Stem Cells for Patients With Mucopolysaccharidosis Type IIWho this might be right forMucopolysaccharidosis II University of Manchester 5
Not applicableLooking for participantsNCT05408715What this trial is testingA Natural History Study in Children With a Type II Collagen Disorder With Short StatureWho this might be right forSEDCHypochondrogenesisSemd, Strudwick Type+1 more Innoskel 60
Large-scale testing (Phase 3)Active Not RecruitingNCT07236606What this trial is testingRGX-121-3102 Gene Therapy in Participants With MPS II (Hunter Syndrome)Who this might be right forMPS IIHunter Syndrome (MPS II) REGENXBIO Inc. 2