drugset / Trial / NCT00146757
A Study Evaluating the Safety and Pharmacokinetics of Aldurazyme® (Laronidase) in MPS I Patients Less Than 5 Years Old
Non-randomizedSingle-groupOpen-labelTreatment
Summary
The main objectives of this study are to evaluate the safety and pharmacokinetics (PK) of enzyme replacement therapy with recombinant human alpha-L-iduronidase \[Aldurazyme® (laronidase)\] in mucopolysaccharidosis I (MPS I) patients less than 5 years old. Efficacy measurements will also be evaluated in this study.
Timeline
- Start
- 2002-10
- Primary completion
- 2005-05
- Completion
- 2005-05
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | laronidase | Protein / enzyme biologic | 100 iu/kg | — |
| Subject | laronidase | Protein / enzyme biologic | 200 iu/kg | — |