Aldurazyme^® Clinical Trials summary

The efficiency of the uptake mechanism and the efficacy of the enzyme replacement in MPS I in vivo has been established in a series of preclinical studies using recombinant human alpha-L-iduronidase. The results of these studies form the basis for clinical studies, which use a dose that has been established from canine MPS I studies.

Two clinical trials evaluated the clinical pharmacology, efficacy, and safety of weekly intravenous administration of Aldurazyme^® (laronidase). These trials include a Phase I/II open-label study ^[1] with an extension study, and a Phase III ^[2] randomized, double-blind, placebo-controlled study, with an open-label extension study.

Please see the full prescribing information in Dutch or in French (PDF) for Aldurazyme^®.

References

1. Kakkis E, Muenzer J, Tiller GE, Waber L, Belmont J, Passage M, et al. Enzyme replacement therapy in mucopolysaccharidosis I. N Engl J Med 2001; 344(3):182-8.

2. Wraith JE, Clarke LA, Beck M et al. Enzyme replacement therapy for mucopolysaccharidosis I: A randomized, double-blinded, placebo-controlled, multinational study of recombinant human α-L-Iduronidase (Laronidase) J. Pediatr 2004; 144:581-8