Mucopolysaccharidoses: all 144 studies
Page 2 of 2. Open to participants first (11), then studies with results posted, then the rest; newest registration first within each.
- Safety and Dose Ranging Study of Insulin Receptor MAb-IDUA Fusion Protein in Patients With MPS ICompletedNCT02371226Phase 1
- Biomarker for Hurler Disease (BioHurler)WithdrawnNCT02298712
- A Multicenter, Multinational, Observational Morquio A Registry Study (MARS)CompletedNCT02294877
- Natural History Study to Characterise the Course of Disease Progression in Participants With Mucopolysaccharidosis Type IIIBTerminatedNCT02293408
- A Retrospective Chart Review of Deceased Patients With Mucopolysaccharidosis Type IIIBCompletedNCT02293382
- Safety and Dose Ranging Study of Insulin Receptor MoAb-IDS Fusion Protein in Patients With Hunter SyndromeCompletedNCT02262338Phase 1
- Psychological Concomitants of Morquio A Syndrome - Longitudinal Effects of Enzyme Replacement Therapy (The MAPLE Study)CompletedNCT02208661
- Dynamic Gait Analysis in Children With Mucopolysaccharidosis Type IVaWithdrawnNCT02153255
- Mucopolysaccharidosis (MPS) I, II, and VI Screening in a High-Risk Population With Previous Surgical Repair or Presence of Inguinal and/or Umbilical Hernia in Combination With Pediatric ENT Surgery (The HATT Project)TerminatedNCT02095015
- Ultrasound Findings of Finger, Wrist and Knee Joints in MucopolysaccharidosisCompletedNCT02067650
- Long-term Follow-up of Sanfilippo Type A Patients Treated by Intracerebral SAF-301 Gene TherapyCompletedNCT02053064Phase 1/2
- The Long-term Safety Study of Idursulfase-beta in Hunter Syndrome(Mucopolysaccharidosis II) PatientsStatus unknownNCT02044692
- Natural History Studies of Mucopolysaccharidosis IIICompletedNCT02037880
- BMN 110 Phase 3B in Australian PatientsCompletedNCT01966029Phase 3
- Lysosomal Storage Disease: Health, Development, and Functional Outcome Surveillance in Preschool ChildrenCompletedNCT01938014
- Longitudinal Studies of Brain Structure and Function in MPS DisordersCompletedNCT01870375
- BMN 110 US Expanded Access ProgramApproved for marketingNCT01858103
- Observational Study to Evaluate Neurodevelopmental Status in Pediatric Patients With Hunter Syndrome (MPS II)CompletedNCT01822184
- Psychological Concomitants of Morquio Syndrome (The MAP Study)CompletedNCT01752296
- Discovering New Biomarkers For Monitoring Disease Progression in Patients With Mucopolysaccharidosis IVATerminatedNCT01733615
- Diagnosis of Mucopolysaccharidosis Disorders in Patients Presenting With Bilateral Hip DiseaseCompletedNCT01707433
- Non-invasive Assessment of Intraocular Pressure in MPS by Use of the Ocular Response Analyzer.Status unknownNCT01695161
- Study to Detect Unrecognized Mucopolysaccharidosis in Children Visiting Rheumatology, Hand or Skeletal Dysplasia ClinicsTerminatedNCT01675674
- A Study to Test the Possibility of Cross Reaction Induced by the Idursulfase Drug to GSK2788723CompletedNCT01602601
- Human Placental-Derived Stem Cell TransplantationCompletedNCT01586455Phase 1
- Laronidase (Aldurazyme TM) Enzyme Replacement Therapy With Hematopoietic Stem Cell Transplant for Hurler SyndromeTerminatedNCT01572636
- Longitudinal Study of Bone Disease in Children with Mucopolysaccharidoses (MPS) I, II, and VICompletedNCT01521429
- Natural History Study of Patients With Mucopolysaccharidosis Type IIIB (MPS IIIB, Sanfilippo Syndrome Type B)CompletedNCT01509768
- Intracerebral Gene Therapy for Sanfilippo Type A SyndromeCompletedNCT01474343Phase 1/2
- Biomarkers for Hunter SyndromeTerminatedNCT01330277
- Hematopoietic Stem Cell Transplantation for MucopolysaccharidosisStatus unknownNCT01238328Phase 2/3
- A Study of Intrathecal Enzyme Therapy for Cognitive Decline in MPS ICompletedNCT00852358Not applicable
- A Clinical Assessment Study of Subjects With Mucopolysaccharidosis IVA (Morquio Syndrome)TerminatedNCT00787995
- Extension Study of Intrathecal Enzyme Replacement Therapy for MPS ITerminatedNCT00786968Phase 1
- Long Term Follow Up Of Patients Who Have Received Gene Therapy Or Gene Marked ProductsCompletedNCT00695279
- ALD-101 Adjuvant Therapy of Unrelated Umbilical Cord Blood Transfusion (UCBT) in Patients With Inherited Metabolic DiseasesTerminatedNCT00654433Phase 3
- Intrathecal Enzyme Replacement for Hurler SyndromeCompletedNCT00638547Phase 1
- Intrathecal Enzyme Replacement Therapy for Spinal Cord Compression in Mucopolysaccharidosis (MPS) ITerminatedNCT00215527Phase 1
- Mucopolysaccharidosis (MPS) VI Clinical Surveillance Program (CSP)CompletedNCT00214773
- A Study Investigating the Relationship Between the Development of Laronidase Antibody and Urinary GAG (Glycosaminoglycan) Levels in Aldurazyme® Treated PatientsCompletedNCT00144768Phase 4
- Open-Label Study of Efficacy and Safety of Recombinant Human N-acetylgalactosamine 4-sulfatase in Patients With MPS VICompletedNCT00048711Phase 2
- Study of Recombinant Human N-Acetylgalactosamine 4-Sulfatase in Patients With MPS VICompletedNCT00048620Phase 1
- Study of Pulmonary Complications in Pediatric Patients With Storage Disorders Undergoing Allogeneic Hematopoietic Stem Cell TransplantationStatus unknownNCT00005900
- Phase I/II Study of Retroviral-Mediated Transfer of Iduronate-2-Sulfatase Gene Into Lymphocytes of Patients With Mucopolysaccharidosis II (Mild Hunter Syndrome)CompletedNCT00004454Phase 1/2