4 completed, 1 active not recruiting, 1 available, 1 recruiting. 4 Phase 2, 2 Phase 3.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 4 | Completed 2, Active not recruiting 1, Terminated 1 |
| Phase 3 | 2 | Completed 2 |
Follow Sly Syndrome: new trials by email Literature Review of Sly Syndrome trials Research Report on Sly Syndrome
Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.
| Trial | Phase | Status | Sponsor | Enrollment | Start | Updated |
|---|---|---|---|---|---|---|
| Mucopolysaccharidosis VII Disease Monitoring Program No Intervention - Mucopolysaccharidosis VII, MPS VII | Recruiting | Ultragenyx Pharmaceutical Inc | 50 | 2018-01-29 | 2026-08-27 | |
| A Study of UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Therapy in Subjects With Mucopolysaccharidosis Type 7, Sly Syndrome (MPS 7) UX003 - Sly Syndrome, MPS VII | Phase 3 | Completed | Ultragenyx Pharmaceutical Inc | 12 | 2015-11-10 | 2020-07-30 |
| Study of UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Treatment in Mucopolysaccharidosis Type 7, Sly Syndrome (MPS 7) Patients Less Than 5 Years of Age UX003 - Sly Syndrome, MPS VII | Phase 2 | Completed | Ultragenyx Pharmaceutical Inc | 8 | 2015-07-21 | 2019-10-30 |
| A Phase 3 Study of UX003 Recombinant Human Betaglucuronidase (rhGUS) Enzyme Replacement Therapy in Patients With Mucopolysaccharidosis Type 7 (MPS 7) UX003, Placebo - MPS 7, Sly Syndrome | Phase 3 | Completed | Ultragenyx Pharmaceutical Inc | 12 | 2014-12-01 | 2020-07-30 |
| MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis Stem Cell Transplantation, IMD Preparative Regimen, Osteopetrosis Only Preparative Regimen - Mucopolysaccharidosis Disorders, Hurler Syndrome | Phase 2 | Active not recruiting | Masonic Cancer Center, University of Minnesota | 149 | 2014-07-10 | 2026-11-03 |
| Allogeneic Bone Marrow Transplant for Inherited Metabolic Disorders Campath-1H, Cyclophosphamide, Busulfan - Mucopolysaccharidosis, Hurler Syndrome | Phase 2 | Completed | Masonic Cancer Center, University of Minnesota | 46 | 2009-12-01 | 2018-02-05 |
| Hematopoietic Stem Cell Transplantation (HCT) for Inborn Errors of Metabolism Stem Cell Transplantation, Cyclophosphamide, Campath-1H - Hurler's Syndrome, Maroteaux-Lamy Syndrome | Phase 2 | Terminated | Masonic Cancer Center, University of Minnesota | 18 | 2008-03-01 | 2017-12-28 |
| Expanded Access to Mepsevii Mepsevii - MPS VII, Mucopolysaccharidosis VII | Available | Ultragenyx Pharmaceutical Inc | 2026-07-30 |
Mucopolysaccharidosis VII (8) · Mucopolysaccharidoses (4) · Aspartylglucosaminuria (3) · Fucosidosis (3) · Leukodystrophy, Globoid Cell (3) · Leukodystrophy, Metachromatic (3) · Lysosomal Storage Diseases (3) · Metabolic Diseases (3) · Mucopolysaccharidosis I (3) · Mucopolysaccharidosis VI (3) · Peroxisomal Disorders (3) · Rare Diseases (3) · Sphingolipidoses (3) · alpha-Mannosidosis (3) · Mucopolysaccharidosis II (2)
Carbohydrate Metabolism, Inborn Errors (8) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (8) · Connective Tissue Diseases (8) · Genetic Diseases, Inborn (8) · Mucinoses (8) · Nutritional and Metabolic Diseases (8) · Skin and Connective Tissue Diseases (8) · Metabolism, Inborn Errors (7) · Lysosomal Storage Diseases (5) · Metabolic Diseases (5) · Mucopolysaccharidoses (4) · Brain Diseases (3) · Brain Diseases, Metabolic (3) · Brain Diseases, Metabolic, Inborn (3) · Central Nervous System Diseases (3)
vestronidase alfa (4) · Stem Cell Transplantation (3) · Alemtuzumab (2) · Busulfan (2) · Cyclophosphamide (2) · Bone Marrow Transplantation (1) · Cyclosporine (1) · Mycophenolic Acid (1) · Regimen B (1)
Ultragenyx Pharmaceutical Inc (5) · Masonic Cancer Center, University of Minnesota (3)
United States (7) · Portugal (3) · Brazil (2) · Spain (2) · Argentina (1) · France (1) · Germany (1) · Mexico (1) · Netherlands (1)
Paul Orchard, MD (2) · Chester Whitley, MD (1) · Mislen Bauer, MD (1) · Paul Harmatz, MD (1) · Paul Orchard, M.D. (1) · Raymond Wang, MD (1)
PMID 37415957, PMID 30467742, PMID 32063397, PMID 33874971, PMID 21586746, PMID 29478819, PMID 35331634
2015 (2)