3 completed, 2 recruiting, 1 available. 2 Phase 3, 1 Phase 1, 1 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 3 | 2 | Completed 2 |
| Phase 1 | 1 | Recruiting 1 |
| Phase 2 | 1 | Completed 1 |
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 |
|---|---|---|---|---|---|---|
| PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders) Aldurazyme (laronidase) - MPS I, MPS II | Phase 1 | Recruiting | University of California, San Francisco | 10 | 2021-07-01 | 2026-03-17 |
| 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 |
| Expanded Access to Mepsevii Mepsevii - MPS VII, Mucopolysaccharidosis VII | Available | Ultragenyx Pharmaceutical Inc | 2026-07-30 |
Mucopolysaccharidosis VII (6) · Lysosomal Storage Diseases (3) · Metabolic Diseases (3) · Mucopolysaccharidoses (3) · Rare Diseases (3) · Gaucher Disease (1) · Mucopolysaccharidosis IV (1) · Mucopolysaccharidosis VI (1) · Sudden Infant Death (1) · Wolman Disease (1)
Carbohydrate Metabolism, Inborn Errors (6) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (6) · Connective Tissue Diseases (6) · Genetic Diseases, Inborn (6) · Metabolism, Inborn Errors (6) · Mucinoses (6) · Nutritional and Metabolic Diseases (6) · Skin and Connective Tissue Diseases (6) · Pathologic Processes (4) · Pathological Conditions, Signs and Symptoms (4) · Disease Attributes (3) · Lysosomal Storage Diseases (3) · Metabolic Diseases (3) · Mucopolysaccharidoses (3) · Brain Diseases (1)
vestronidase alfa (5) · GAA protein, human (1) · GALNS protein, human (1) · Iduronidase (1) · Sebelipase alfa (1) · galsulfase (1) · idursulfase (1)
Ultragenyx Pharmaceutical Inc (5) · University of California, San Francisco (1)
United States (5) · Portugal (3) · Brazil (2) · Spain (2) · Argentina (1) · France (1) · Germany (1) · Mexico (1) · Netherlands (1)
Chester Whitley, MD (1) · Mislen Bauer, MD (1) · Paul Harmatz, MD (1) · Raymond Wang, MD (1) · Tippi MacKenzie, MD (1)
PMID 37415957, PMID 30467742, PMID 32063397, PMID 33874971, PMID 15324318, PMID 19948615, PMID 21637107, PMID 23510062, PMID 28374935, PMID 28657663, PMID 29478819, PMID 30337566, PMID 32102934, PMID 35331634, PMID 37955580, PMID 39891377, PMID 9918480
2015 (2)