5 completed, 1 no longer available, 1 recruiting, 1 terminated. 5 Phase 2, 2 Phase 1, 2 Phase 3.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 5 | Completed 4, Terminated 1 |
| Phase 1 | 2 | Completed 1, Recruiting 1 |
| Phase 3 | 2 | Completed 2 |
Follow Sebelipase alfa: new trials by email Literature Review of Sebelipase alfa trials Research Report on Sebelipase alfa
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 |
| 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 |
| Acid Lipase Replacement Investigating Safety and Efficacy (ARISE) in Participants With Lysosomal Acid Lipase Deficiency Sebelipase Alfa, Placebo - Lysosomal Acid Lipase Deficiency | Phase 3 | Completed | Alexion Pharmaceuticals, Inc. | 66 | 2013-01-22 | 2020-12-29 |
| 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 |
| An Expanded Access Protocol for Sebelipase Alfa for Patients With Lysosomal Acid Lipase Deficiency sebelipase alfa - Lysosomal Acid Lipase Deficiency | No longer available | Alexion Pharmaceuticals, Inc. | 2016-06-08 |
Wolman Disease (8) · Cholesterol Ester Storage Disease (7) · Lysosomal Storage Diseases (5) · Chanarin-Dorfman Syndrome (2) · Cirrhosis, Cryptogenic (2) · Liver Diseases, Alcoholic (2) · Niemann-Pick Diseases (2) · Non-alcoholic Fatty Liver Disease (2) · Gaucher Disease (1) · Genetic Diseases, Inborn (1) · Infant, Newborn, Diseases (1) · Lipid Metabolism Disorders (1) · Lipid Metabolism, Inborn Errors (1) · Lipidoses (1) · Metabolic Diseases (1)
Alexion Pharmaceuticals, Inc. (7) · University of California, San Francisco (1)
United States (7) · United Kingdom (6) · France (4) · Czechia (3) · Italy (3) · Australia (2) · Canada (2) · Croatia (2) · Germany (2) · Mexico (2) · Russia (2) · Spain (2) · Turkey (Türkiye) (2) · Argentina (1) · Belgium (1)
Florian Abel, MD (1) · Tippi MacKenzie, MD (1)
PMID 23348766, PMID 15324318, PMID 19948615, PMID 21637107, PMID 23510062, PMID 24993530, PMID 28374935, PMID 28657663, PMID 30337566, PMID 32102934, PMID 32657505, PMID 34774639, PMID 35442238, PMID 37955580, PMID 39891377, PMID 9918480