Clinical Trial: Phase 3 Extension Study of the Safety and Efficacy of Aldurazyme® (Laronidase) in Mucopolysaccharidosis I (MPS I) Patients

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

Official Title: A Multicenter, Multinational, Open-Label Extension Study of the Safety and Efficacy of Aldurazyme® (Laronidase) in Patients With Mucopolysaccharidosis I

Brief Summary: This study is being conducted to collect additional long-term efficacy and safety data of Aldurazyme® (laronidase) patients with MPS I disease. Patients who were previously enrolled in the Phase 3 Double-Blind Study will be enrolled in this study.

Detailed Summary:
Sponsor: Genzyme, a Sanofi Company

Current Primary Outcome:

  • Change From Baseline to Week 182 in Percent Predicted Forced Vital Capacity (FVC) [ Time Frame: Baseline to Week 182 ]
    Percent Predicted Forced Vital Capacity: the maximal exhaled breath volume following a maximal inhaled breath. Overall change from Baseline to Week 182 in percent predicted FVC = (observed value)/(predicted value) * 100%). A higher value indicates a greater response.
  • Change From Baseline to Week 182 in Six Minute Walk Test (6MWT) [ Time Frame: Baseline to Week 182 ]
    Six Minute Walk Test: Distance walked (measured in Meters) in 6 minutes. A longer distance indicates a greater response.


Original Primary Outcome: To collect additional long-term efficacy and safety data of Aldurazyme® (laronidase) on patients previously enrolled in the phase 3 double-blind study.

Current Secondary Outcome:

  • Change From Baseline to Week 182 in Apnea/Hypopnea Index (AHI) [ Time Frame: Baseline to Week 182 ]
    Apnea/Hypopnea Index (AHI): Number of absent (apnea) and shallow (hypopnea) breaths per hour of sleep. A greater decrease in events indicates a greater response.
  • Change From Baseline to Week 182 in Liver Volume [ Time Frame: Baseline to Week 182 ]
    Liver Organ Volume: Volume of liver measured by Magnetic Resonance Imaging (MRI). Greater decrease in volume indicates a greater response.
  • Change From Baseline to Week 182 in Child Health Assessment Questionnaire/Health Assessment Questionnaire (CHAQ/HAQ) Disability Index Score [ Time Frame: Baseline to Week 182 ]
    CHAQ/HAQ = Patient questionnaire that measures the degree of disability on a scale of 0 (no disability) to 3 (maximal disability). A lower score indicates a greater response.
  • Change From Baseline to Week 182 in Active Joint Range of Motion (ROM) [ Time Frame: Baseline to Week182 ]
    Active Joint Range of Motion (ROM): Shoulder Flexion Ability to maximally raise one's arm overhead without assistance. Shoulder range of motion (mean of left and right arms) measured in degrees (0-180) by goniometry. Greater degree of flexion indicates greater response.


Original Secondary Outcome:

Information By: Sanofi

Dates:
Date Received: September 2, 2005
Date Started: May 2001
Date Completion:
Last Updated: March 17, 2015
Last Verified: March 2015