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 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
Testing effectiveness (Phase 2)Active Not RecruitingNCT03488394What this trial is testingGene Therapy With Modified Autologous Hematopoietic Stem Cells for the Treatment of Patients With Mucopolysaccharidosis Type I, Hurler VariantWho this might be right forMucopolysaccharidosis IH Orchard Therapeutics 8
Not applicableStudy completedNCT01870375What this trial is testingLongitudinal Studies of Brain Structure and Function in MPS DisordersWho this might be right forMucopolysaccharidosis Type IMucopolysaccharidosis Type IIMucopolysaccharidosis Type VI+2 more University of Minnesota 100
Early research (Phase 1)Ended earlyNCT00786968What this trial is testingExtension Study of Intrathecal Enzyme Replacement Therapy for MPS IWho this might be right forSpinal Cord CompressionMucopolysaccharidosis IHurler-Scheie Syndrome+2 more Patricia I. Dickson, M.D. 3
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
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
Early research (Phase 1)WithdrawnNCT05238324What this trial is testingSafety and Efficacy of HMI-203 in ERT-Treated Adults With MPS IIWho this might be right forMucopolysaccharidosis II Homology Medicines, Inc
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
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
Testing effectiveness (Phase 2)Study completedNCT03411161What this trial is testingS 81694 Plus Paclitaxel in Metastatic Breast CancerWho this might be right forMetastatic Breast CancerMetastatic Triple Negative Breast Cancer Institut de Recherches Internationales Servier 22
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 completedNCT02067650What this trial is testingUltrasound Findings of Finger, Wrist and Knee Joints in MucopolysaccharidosisWho this might be right forMPS IMPS IV Children's Hospital of Eastern Ontario 18
Testing effectiveness (Phase 2)Study completedNCT02437253What this trial is testingEffects of Adalimumab in Mucopolysaccharidosis Types I, II and VIWho this might be right forMucopolysaccharidosis Type IMucopolysaccharidosis Type IIMucopolysaccharidosis Type VI Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center 2
Testing effectiveness (Phase 2)WithdrawnNCT02715505What this trial is testingSafety and Exploratory Efficacy of HSC835 in Patients With Inherited Metabolic Disorders (IMD)Who this might be right forInherited Metabolic Disorders IMD Novartis Pharmaceuticals
Testing effectiveness (Phase 2)Study completedNCT01856218What this trial is testingAn Open-Label Phase 1/2 Study to Assess the Safety, Efficacy and Dose of Study Drug UX003 Recombinant Human Beta-glucuronidase (rhGUS) Enzyme Replacement Therapy in Patients With Mucopolysaccharidosis Type 7 (MPS 7)Who this might be right forMucopolysaccharidosis Type 7 Ultragenyx Pharmaceutical Inc 3
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
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
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
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