Status | Study |
Recruiting |
Study Name: Novel Association of Cholesterol Ester Storage Disease Due to Lysosomal Acid Lipase Deficiency and Non-Alcoholic Fatty Liver Disease: A Prospective Clinical Study Condition: Non-alcoholic Fatty Liver Disease Cholesterol Ester Storage Disease Date: 2013-02-10 |
Active, not recruiting |
Study Name: A Multicenter Study of SBC-102 (Sebelipase Alfa) in Patients With Lysosomal Acid Lipase Deficiency/ ARISE (Acid Lipase Replacement Investigating Safety and Efficacy) Condition: Cholesterol Ester Storage Disease (CESD) Lysosomal Acid Lipase Date: 2012-12-17 Interventions: Drug: SBC-102 [sebelipase alfa] (1 mg/kg) |
Recruiting |
Study Name: Identification of Undiagnosed Lysosomal Acid Lipase Deficiency Condition: Lysosomal Acid Lipase Deficiency Cholesterol Ester Storage Disease Date: 2012-10-26 Interventions: Other: enzyme analysis |
Recruiting |
Study Name: Lysosomal Acid Lipase (LAL) Deficiency Registry Condition: Lysosomal Acid Lipase Deficiency Cholesterol Ester Storage Disease Date: 2012-06-29 |
Recruiting |
Study Name: Human Placental-Derived Stem Cell Transplantation Condition: Mucopolysaccharidosis I Mucopolysaccharidosis VI Adrenoleu Date: 2012-04-25 Interventions: Drug: Human Placental Derived Stem Cell Infusions of thawed HPDSC to be given following UCB infusion. |
Completed |
Study Name: An Observational Study of Patients With Lysosomal Acid Lipase Deficiency/Cholesteryl Ester Storage Disease Phenotype Condition: Cholesterol Ester Storage Disease(CESD) Lysosomal Acid Lipase D Date: 2012-02-01 |
Active, not recruiting |
Study Name: Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of SBC-102 (Sebelipase Alfa) in Adult Subjects With Lysosomal Acid Lipase Deficiency Condition: Cholesterol Ester Storage Disease(CESD) Lysosomal Acid Lipase D Date: 2011-11-26 Interventions: Drug: SBC-102 (sebelipase alfa) |
Terminated |
Study Name: Children With Lysosomal Acid Lipase Deficiency Who Previously Received Treatment With SBC-102 Condition: Lysosomal Acid Lipase Deficiency Wolman Disease Date: 2011-11-10 Interventions: Drug: SBC-102 SBC-102 is a recombinant human lysosomal acid lipase (rhLAL). The investigational medicina |
Active, not recruiting |
Study Name: SurVival of Lysosomal Acid Lipase Deficiency (LAL-D) Infants Treated With SebelipAse aLfa Condition: Lysosomal Acid Lipase Deficiency Wolman Disease Date: 2011-06-09 Interventions: Drug: Sebelipase alfa (SBC-102) Sebelipase alfa is a recombinant human lysosomal acid lipase (rhLAL). Th |
Completed |
Study Name: A Retrospective Natural History Study of Patients With Lysosomal Acid Lipase Deficiency/Wolman Phenotype Condition: Lysosomal Acid Lipase Deficiency Wolman Disease Date: 2011-04-13 |