Status | Study |
Active, not recruiting |
Study Name: A Long-Term Open-Label Treatment and Extension Study of UX003 rhGUS Enzyme Replacement Therapy in Subjects With MPS 7 Condition: Sly Syndrome MPS VII Date: 2015-04-22 Interventions: Drug: UX003 UX003 is a sterile liquid buffered saline formulation of rhGUS that contains enzyme at a con |
Active, not recruiting |
Study Name: An Open-Label Study of UX003-rhGUS Enzyme Replacement Treatment in MPS 7 Patients Less Than 5 Years of Age Condition: Sly Syndrome MPS VII Date: 2015-04-12 Interventions: Drug: UX003 Other Names: recombinant human beta-glucoronidase |
Recruiting |
Study Name: Biomarker for Sly Disease Condition: Mucopolysaccharidosis VII Sly Disease Date: 2014-10-23 |
Completed |
Study Name: A Phase 3 Study of UX003 rhGUS Enzyme Replacement Therapy in Patients With MPS 7 Condition: MPS 7 Sly Syndrome Date: 2014-08-22 Interventions: Drug: UX003 UX003 is a sterile |
Recruiting |
Study Name: MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis Condition: Mucopolysaccharidosis Disorders Hurler Syndrome Hunter Syn Date: 2014-06-20 Interventions: Biological: Stem Cell Transplantation |
No longer available |
Study Name: An Open-Label Treatment Protocol With UX003 rhGUS Enzyme Replacement Therapy for an Advanced Stage MPS 7 Patient Condition: Mucopolysaccharidosis Type 7 Date: 2014-03-24 Interventions: Drug: UX003 Open Label Other Names: |
Completed |
Study Name: An 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 MPS 7 Condition: Mucopolysaccharidosis Type 7 Date: 2013-05-08 Interventions: Drug: UX003 |
Recruiting |
Study Name: Longitudinal Studies of Brain Structure and Function in MPS Disorders Condition: Mucopolysaccharidosis Type I Mucopolysaccharidosis Type II Date: 2011-08-02 |
Active, not recruiting |
Study Name: Allogeneic Bone Marrow Transplant for Inherited Metabolic Disorders Condition: Mucopolysaccharidosis Hurler Syndrome Hunter Syndrome Date: 2010-01-05 Interventions: Drug: Campath-1H Administered |
Terminated |
Study Name: Hematopoietic Stem Cell Transplantation (HCT) for Inborn Errors of Metabolism Condition: Hurler's Syndrome Maroteaux-Lamy Syndrome Date: 2008-04-25 Interventions: Procedure: Stem Cell Transplantation |