The study enrolled 90 patients at eight primary sites in the US and Europe. Participants received either Myozyme or a placebo every other week for 18 months. The average age of study participants was 44 years. The primary efficacy endpoints of the study sought to determine the effect of Myozyme on functional endurance as measured by the six-minute walk test and to determine the effect of Myozyme on pulmonary function as measured by percent predicted forced vital capacity.
The results showed that, at 18 months, patients treated with Myozyme increased their distance walked in six minutes by an average of approximately 30 meters as compared with the placebo group (P=0.0283; Wilcoxon test). The placebo group did not show any improvement from baseline. The average baseline distance walked in six minutes in both groups was approximately 325 meters.
Richard Moscicki, senior vice president and chief medical officer for Genzyme, said: “The preservation of pulmonary function is extremely important because respiratory failure is the major cause of mortality in this progressive and life-threatening neuromuscular disease. This trial has broader implications, in that it is one of the first large randomized trials to show benefit in a degenerative neuromuscular disease. On behalf of Genzyme, I want to thank the patients and physicians who participated in this study, whose commitment to ensuring its successful completion was a service to the entire Pompe community.”