Extrait

The overall objective is to evaluate the safety, efficacy, and pharmacokinetics (PK) of Myozyme treatment in patients with late-onset Pompe disease as compared to placebo. The primary objectives of the study are: 1) to evaluate the safety profile of Myozyme; 2) to determine the effect of Myozyme treatment on functional endurance as measured by the Six Minute Walk Test (6MWT) 3) to determine the effect of Myozyme treatment on respiratory muscle weakness as measured by Forced Vital Capacity (FVC) in the upright position ; and 4) to determine in a subset of patients the PK profile of Myozyme in patients with late-onset Pompe disease. The study wil be considered to have met its primary efficacy objective if a statistically significant treatment effect of Myozyme over placebo is demonstrated in the 6 MWT distance walked.

Critère d'inclusion

• Glycogen Storage Disease type II (Pompe´s disease)

Liens

• ictrp
• euctr