6 completed, 2 terminated. 8 Phase 2, 2 Phase 3, 1 Phase 1.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 8 | Completed 6, Terminated 2 |
| 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 |
| Clinical Study In Infants With Rapidly Progressive Lysosomal Acid Lipase Deficiency Sebelipase Alfa - Lysosomal Acid Lipase Deficiency | Phase 2 | Terminated | Alexion Pharmaceuticals, Inc. | 10 | 2014-06-06 | 2019-11-18 |
| 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 |
| Hematopoietic Stem Cell Transplantation (HCT) for Inborn Errors of Metabolism Stem Cell Transplantation, Cyclophosphamide, Campath-1H - Hurler's Syndrome, Maroteaux-Lamy Syndrome | Phase 2 | Terminated | Masonic Cancer Center, University of Minnesota | 18 | 2008-03-01 | 2017-12-28 |
| 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 (8) · Leukodystrophy, Globoid Cell (3) · Leukodystrophy, Metachromatic (3) · Lysosomal Storage Diseases (3) · Metabolism, Inborn Errors (3) · Niemann-Pick Diseases (3) · Adrenoleukodystrophy (2) · Chanarin-Dorfman Syndrome (2) · Cirrhosis, Cryptogenic (2) · Fucosidosis (2) · Gangliosidosis, GM1 (2) · Liver Diseases, Alcoholic (2) · Non-alcoholic Fatty Liver Disease (2) · Sandhoff Disease (2)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (8) · Nutritional and Metabolic Diseases (8) · Genetic Diseases, Inborn (7) · Infant, Newborn, Diseases (7) · Lipid Metabolism Disorders (7) · Lipid Metabolism, Inborn Errors (7) · Lipidoses (7) · Metabolic Diseases (7) · Brain Diseases (5) · Brain Diseases, Metabolic (5) · Brain Diseases, Metabolic, Inborn (5) · Central Nervous System Diseases (5) · Lysosomal Storage Diseases (5) · Lysosomal Storage Diseases, Nervous System (5) · Metabolism, Inborn Errors (5)
Sebelipase alfa (5) · Alemtuzumab (2) · Bone Marrow Transplantation (2) · Busulfan (2) · Cyclophosphamide (2) · Stem Cell Transplantation (2) · Antilymphocyte Serum (1) · Clofarabine (1) · Hematopoietic Stem Cell Transplantation (1) · Hydroxyurea (1) · Melphalan (1) · Whole-Body Irradiation (1)
Alexion Pharmaceuticals, Inc. (5) · Masonic Cancer Center, University of Minnesota (3)
United States (8) · United Kingdom (5) · France (3) · Canada (2) · Czechia (2) · Italy (2) · Australia (1) · Belgium (1) · Brazil (1) · Croatia (1) · Denmark (1) · Egypt (1) · Finland (1) · Germany (1) · Ireland (1)
Paul Orchard, MD (3)