1 completed, 1 recruiting. 1 Phase 3.
| Phase | Trials | Status |
|---|---|---|
| Phase 3 | 1 | Completed 1 |
Follow MPS 7: new trials by email Literature Review of MPS 7 trials Research Report on MPS 7
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 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 |
Mucopolysaccharidosis VII (2) · Lysosomal Storage Diseases (1) · Metabolic Diseases (1) · Mucopolysaccharidoses (1) · Rare Diseases (1)
Carbohydrate Metabolism, Inborn Errors (2) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (2) · Connective Tissue Diseases (2) · Genetic Diseases, Inborn (2) · Metabolism, Inborn Errors (2) · Mucinoses (2) · Nutritional and Metabolic Diseases (2) · Skin and Connective Tissue Diseases (2) · Disease Attributes (1) · Lysosomal Storage Diseases (1) · Metabolic Diseases (1) · Mucopolysaccharidoses (1) · Pathologic Processes (1) · Pathological Conditions, Signs and Symptoms (1)
Ultragenyx Pharmaceutical Inc (2)
United States (2) · Argentina (1) · Brazil (1) · France (1) · Germany (1) · Netherlands (1) · Portugal (1) · Spain (1)
Chester Whitley, MD (1) · Mislen Bauer, MD (1) · Paul Harmatz, MD (1) · Raymond Wang, MD (1)
PMID 29478819, PMID 30467742, PMID 32063397, PMID 33874971, PMID 37415957