6 completed. 6 Phase 2, 2 Phase 3, 1 Phase 1.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 6 | Completed 6 |
| Phase 3 | 2 | Completed 2 |
| Phase 1 | 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 |
|---|---|---|---|---|---|---|
| Safety and Efficacy Study of Sebelipase Alfa in Participants With Lysosomal Acid Lipase Deficiency Sebelipase Alfa - Lysosomal Acid Lipase Deficiency | Phase 2 | Completed | Alexion Pharmaceuticals, Inc. | 31 | 2014-06-24 | 2019-12-04 |
| Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of SBC-102 (Sebelipase Alfa) in Adult Subjects With Lysosomal Acid Lipase Deficiency sebelipase alfa - Cholesterol Ester Storage Disease (CESD), Lysosomal Acid Lipase Deficiency | Phase 2 | Completed | Alexion Pharmaceuticals, Inc. | 8 | 2011-12-12 | 2018-07-20 |
| Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of Sebelipase Alfa in Children With Growth Failure Due to Lysosomal Acid Lipase Deficiency Sebelipase alfa (SBC-102) - Lysosomal Acid Lipase Deficiency, Wolman Disease | Phase 2 / Phase 3 | Completed | Alexion Pharmaceuticals, Inc. | 9 | 2011-05-04 | 2019-01-30 |
| 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 |
| HSCT for High Risk Inherited Inborn Errors Clofarabine, Total body Irradiation, Melphalan - Adrenoleukodystrophy, Metachromatic Leukodystrophy | Phase 2 | Completed | Masonic Cancer Center, University of Minnesota | 38 | 2006-09-01 | 2019-07-11 |
| Stem Cell Transplant for Inborn Errors of Metabolism Stem Cell Transplant, Busulfan, Cyclophosphamide, Antithymocyte Globulin - Adrenoleukodystrophy, Metachromatic Leukodystrophy | Phase 2 / Phase 3 | Completed | Masonic Cancer Center, University of Minnesota | 135 | 1995-01-01 | 2017-12-28 |
Wolman Disease (6) · Lysosomal Storage Diseases (3) · Adrenoleukodystrophy (2) · Gangliosidosis, GM1 (2) · Leukodystrophy, Globoid Cell (2) · Leukodystrophy, Metachromatic (2) · Metabolism, Inborn Errors (2) · Niemann-Pick Diseases (2) · Sandhoff Disease (2) · Tay-Sachs Disease (2) · Chanarin-Dorfman Syndrome (1) · Cirrhosis, Cryptogenic (1) · Fucosidosis (1) · Gaucher Disease (1)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (6) · Nutritional and Metabolic Diseases (6) · Genetic Diseases, Inborn (5) · Infant, Newborn, Diseases (5) · Lipid Metabolism Disorders (5) · Lipid Metabolism, Inborn Errors (5) · Lipidoses (5) · Metabolic Diseases (5) · Metabolism, Inborn Errors (4) · Brain Diseases (3) · Brain Diseases, Metabolic (3) · Brain Diseases, Metabolic, Inborn (3) · Central Nervous System Diseases (3) · Lysosomal Storage Diseases (3) · Lysosomal Storage Diseases, Nervous System (3)
Sebelipase alfa (4) · Alemtuzumab (1) · Antilymphocyte Serum (1) · Bone Marrow Transplantation (1) · Busulfan (1) · Clofarabine (1) · Cyclophosphamide (1) · Hematopoietic Stem Cell Transplantation (1) · Hydroxyurea (1) · Melphalan (1) · Stem Cell Transplantation (1) · Whole-Body Irradiation (1)
Alexion Pharmaceuticals, Inc. (4) · Masonic Cancer Center, University of Minnesota (2)
United States (6) · United Kingdom (4) · France (3) · Canada (2) · Czechia (2) · Australia (1) · Belgium (1) · Brazil (1) · Croatia (1) · Denmark (1) · Egypt (1) · Germany (1) · Ireland (1) · Italy (1) · Mexico (1)
Paul Orchard, MD (2)