2 completed. 2 Phase 3.
| Phase | Trials | Status |
|---|---|---|
| Phase 3 | 2 | Completed 2 |
Follow MPS VII: new trials by email Literature Review of MPS VII trials Research Report on MPS VII
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 |
|---|---|---|---|---|---|---|
| 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 |
| 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 |
Lysosomal Storage Diseases (2) · Metabolic Diseases (2) · Mucopolysaccharidoses (2) · Mucopolysaccharidosis VII (2) · Rare Diseases (2)
Carbohydrate Metabolism, Inborn Errors (2) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (2) · Connective Tissue Diseases (2) · Disease Attributes (2) · Genetic Diseases, Inborn (2) · Metabolism, Inborn Errors (2) · Mucinoses (2) · Nutritional and Metabolic Diseases (2) · Pathologic Processes (2) · Pathological Conditions, Signs and Symptoms (2) · Skin and Connective Tissue Diseases (2)
Ultragenyx Pharmaceutical Inc (2)
United States (2) · Brazil (1) · Mexico (1) · Portugal (1)
Chester Whitley, MD (1) · Mislen Bauer, MD (1) · Paul Harmatz, MD (1) · Raymond Wang, MD (1)
PMID 32063397, PMID 33874971, PMID 37415957, PMID 29478819, PMID 30467742
2015 (1)