Clinical Study of Aldurazyme in Patients With Mucopolysaccharidosis (MPS) I

A Randomized, Double-Blind, Placebo-Controlled, Multicenter, Multinational, Clinical Study of Recombinant Human Alpha L-Iduronidase In Patients With Mucopolysaccharidosis I

This study is being conducted to demonstrate the safety and clinical efficacy of Aldurazyme treatment in MPS I patients

Inclusion Criteria:

• The patient had a documented diagnosis of MPS I confirmed by measurable clinical signs and symptoms of MPS I and a fibroblast or leukocyte alpha-L-iduronidase enzyme activity level of less than 10% of the lower limit of the normal range of the measuring laboratory.
• Female patients of childbearing potential had a negative pregnancy test (urine-beta-human chorionic gonadotropin (hCG)) at baseline (all female patients of childbearing potential and sexually mature male patients were advised to use a medically accepted method of contraception throughout the study).
• The patient was capable of standing independently for 6 minutes and walking a minimum of 5 meters within 6 minutes.
• The patient was capable of performing a reproducible FVC maneuver.
• The patient had a baseline FVC value that was less than or equal to 80% of the patient's predicted normal FVC value based on polgar predicted values for standing height for children 5 through 7 years of age and the Hankinson predicted values for ages 8 and above.

Exclusion Criteria:

• The patient had undergone a tracheostomy.
• The patient had previously undergone a bone marrow transplantation.
• The patient was pregnant or lactating.
• The patient has received an investigational drug within 30 days prior to study enrollment.
• The patient had a medical condition, serious intercurrent illness, or other extenuating circumstance that could have significantly interfered with study compliance including all prescribing evaluations and follow-up activities.
• The patient had a known hypersensitivity to rhIDU or to components of the active or placebo test solutions.