2 completed. 2 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 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 |
|---|---|---|---|---|---|---|
| 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 |
| 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 |
Mucopolysaccharidoses (2) · Mucopolysaccharidosis VII (2) · Adrenoleukodystrophy (1) · Aspartylglucosaminuria (1) · Fucosidosis (1) · Leukodystrophy, Globoid Cell (1) · Leukodystrophy, Metachromatic (1) · Lysosomal Storage Diseases (1) · Metabolic Diseases (1) · Mucopolysaccharidosis I (1) · Mucopolysaccharidosis II (1) · Mucopolysaccharidosis VI (1) · Peroxisomal Disorders (1) · Rare Diseases (1) · Sphingolipidoses (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) · Adrenal Gland Diseases (1) · Adrenal Insufficiency (1) · Brain Diseases (1) · Brain Diseases, Metabolic (1) · Brain Diseases, Metabolic, Inborn (1) · Central Nervous System Diseases (1) · Demyelinating Diseases (1)
Alemtuzumab (1) · Busulfan (1) · Cyclophosphamide (1) · Cyclosporine (1) · Mycophenolic Acid (1) · Stem Cell Transplantation (1) · vestronidase alfa (1)
Masonic Cancer Center, University of Minnesota (1) · Ultragenyx Pharmaceutical Inc (1)
United States (2) · Portugal (1) · Spain (1)
Paul Orchard, MD (1)
PMID 30467742, PMID 35331634, PMID 37415957
2015 (1)