Clinical Trial: A Dose-optimization Study of Aldurazyme® (Laronidase) in Patients With Mucopolysaccharidosis I (MPS I) Disease

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

Official Title: A Multicenter, Multinational, Randomized, Dose-Optimization Study of the Safety and Pharmacodynamic Response of Aldurazyme® (Laronidase) in Patients With Mucopolysaccharidosis I

Brief Summary: The main purpose of this study is to evaluate differences in the pharmacodynamic response of 4 Aldurazyme® (laronidase) dose regimens in patients with Mucopolysaccharidosis I (MPS I).

Detailed Summary:
Sponsor: Genzyme, a Sanofi Company

Current Primary Outcome: Percent Change From Baseline to Week 26 in Urinary Glycosaminoglycan (GAG) Level [ Time Frame: Baseline to 26 Weeks ]

Urinary GAG Level - Concentration of GAG relative to creatinine in urine. A greater decrease in GAG level indicates a greater response.


Original Primary Outcome: To evaluate differences in the pharmacodynamic response and safety of Aldurazyme® (laronidase) does regimens

Current Secondary Outcome:

  • Percent Change From Baseline to Week 26 in Liver Organ Volume [ Time Frame: Baseline to 26 Weeks ]
    A greater decrease in liver volume indicates a greater response.
  • Change From Baseline to Week 26 in Six Minute Walk Test (6MWT) [ Time Frame: Baseline to 26 Weeks ]
    Six Minute Walk Test: Distance walked (measured in Meters) in 6 minutes. A longer distance indicates a greater response.


Original Secondary Outcome:

Information By: Sanofi

Dates:
Date Received: September 2, 2005
Date Started: December 2004
Date Completion:
Last Updated: March 17, 2015
Last Verified: March 2015