2 completed. 2 Phase 1, 1 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 1 | 2 | Completed 2 |
| Phase 2 | 1 | Completed 1 |
Follow Cholesterol Ester Storage Disease: new trials by email Literature Review of Cholesterol Ester Storage Disease trials Research Report on Cholesterol Ester Storage Disease
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 |
|---|---|---|---|---|---|---|
| Human Placental-Derived Stem Cell Transplantation Human Placental Derived Stem Cell - Mucopolysaccharidosis I, Mucopolysaccharidosis VI | Phase 1 | Completed | New York Medical College | 43 | 2013-04-01 | 2022-10-25 |
| Safety, Tolerability and Pharmacokinetics of SBC-102 (Sebelipase Alfa) in Adult Participants With Lysosomal Acid Lipase Deficiency Sebelipase alfa 0.35 mg/kg, Sebelipase alfa 1 mg/kg, Sebelipase alfa 3 mg/kg - Cholesterol Ester Storage Disease(CESD), Lysosomal Acid Lipase Deficiency | Phase 1 / Phase 2 | Completed | Alexion Pharmaceuticals, Inc. | 9 | 2011-04-25 | 2018-12-11 |
Wolman Disease (2) · Adrenoleukodystrophy (1) · Anemia, Aplastic (1) · Anemia, Diamond-Blackfan (1) · Fucosidosis (1) · Gaucher Disease (1) · Leukemia, Myeloid, Acute (1) · Leukodystrophy, Globoid Cell (1) · Leukodystrophy, Metachromatic (1) · Lysosomal Storage Diseases (1) · Metabolism, Inborn Errors (1) · Mucopolysaccharidosis I (1) · Mucopolysaccharidosis VI (1) · Myelodysplastic Syndromes (1)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (2) · Genetic Diseases, Inborn (2) · Infant, Newborn, Diseases (2) · Lipid Metabolism Disorders (2) · Lipid Metabolism, Inborn Errors (2) · Lipidoses (2) · Metabolic Diseases (2) · Nutritional and Metabolic Diseases (2) · Adrenal Gland Diseases (1) · Adrenal Insufficiency (1) · Anemia (1) · Anemia, Hypoplastic, Congenital (1) · Bone Marrow Diseases (1) · Bone Marrow Failure Disorders (1) · Brain Diseases (1)
Sebelipase alfa (1)
Alexion Pharmaceuticals, Inc. (1) · New York Medical College (1)
United States (2) · Czechia (1) · France (1) · United Kingdom (1)