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

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

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

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

Detailed Summary:
Sponsor: Genzyme, a Sanofi Company

Current Primary Outcome:

  • Overall Change From Baseline to Week 26 in Percent Predicted Forced Vital Capacity (FVC) [ Time Frame: Baseline to Week 26 ]
    Percent Predicted Forced Vital Capacity (FVC): the maximal exhaled breathe volume following a maximal inhaled breath. Overall Change from Baseline to Week 26 in percent predicted FVC = (observed value)/(predicted value) * 100%). A higher value indicates a greater response.
  • Overall Change From Baseline to Week 26 in Six Minute Walk Test (6MWT) [ Time Frame: Baseline to Week 26 ]
    Six Minute Walk Test (6MWT): Distance walked (measured in meters) in 6 minutes. A longer distance indicates a greater response.


Original Primary Outcome:

  • Forced Vital Capacity (FVC) [ Time Frame: 26 weeks ]
  • Six Minute Walk Test (6MWT) [ Time Frame: 26 weeks ]


Current Secondary Outcome:

  • Overall Change From Baseline to Week 26 in Apnea/Hypopnea Index (AHI) [ Time Frame: Baseline to Week 26 ]
    Apnea/Hypopnea Index (AHI): Number of absent (apnea) and shallow (hypopnea) breaths per hour of sleep. Overall change from Baseline to Week 26 in AHI. A greater decrease in events indicates a greater response.
  • Overall Percent Change From Baseline to Week 26 in Liver Volume [ Time Frame: Baseline to Week 26 ]
    Liver Organ Volume: Volume of liver measured by Magnetic Resonance Imaging (MRI). Greater decrease in volume indicates a greater response.
  • Overall Change From Baseline to Week 26 in Child Health Assessment Questionnaire/Health Assessment Questionnaire (CHAQ/HAQ) Disability Index Score [ Time Frame: Baseline to week 26 ]
    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.
  • Overall Change From Baseline to Week 26 in Active Joint Range of Motion (ROM) [ Time Frame: Baseline to Week 26 ]
    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.
  • Overall Percent Change From Baseline to Week 26 in Urinary Glycosaminoglycan (GAG) Levels [ Time Frame: Baseline to Week 26 ]
    Urinary Glycosaminoglycan (GAG) Levels: Concentration of GAG relative to creatinine in urine. A greater decrease in GAG level indicates a greater response.


Original Secondary Outcome:

  • Apnea/Hypopnea Index (AHI) [ Time Frame: 26 weeks ]
  • Liver Organ Volume (hepatomegaly) [ Time Frame: 26 weeks ]
  • CHAQ/HAQ Disability Index Score [ Time Frame: 26 weeks ]
  • Shoulder Flexion [ Time Frame: 26 weeks ]
  • uGAG [ Time Frame: 26 weeks ]


Information By: Sanofi

Dates:
Date Received: June 2, 2009
Date Started: December 2000
Date Completion:
Last Updated: March 19, 2015
Last Verified: March 2015