drugset / Trial / NCT00410566
Safety Study of rhASM Enzyme Replacement Therapy in Adults With Acid Sphingomyelinase Deficiency (Niemann-Pick Disease)
Non-randomizedParallel-groupOpen-labelTreatment
Summary
The purpose of this study is to determine the safe range of single doses of rhASM administered to adults with ASM deficiency.
Timeline
- Start
- 2006-12
- Primary completion
- 2009-03
- Completion
- 2009-04
Outcome
Outcome not reported
Stopped: “Terminated by sponsor - Single dose safety objective achieved.”
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | Olipudase alfa | Protein / enzyme biologic | 0.03 mg/kg | Intravenous |
| Subject | Olipudase alfa | Protein / enzyme biologic | 0.1 mg/kg | Intravenous |
| Subject | Olipudase alfa | Protein / enzyme biologic | 0.3 mg/kg | Intravenous |
| Subject | Olipudase alfa | Protein / enzyme biologic | 0.6 mg/kg | Intravenous |
| Subject | Olipudase alfa | Protein / enzyme biologic | 1 mg/kg | Intravenous |