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

This study has been completed.
Sponsor:
ClinicalTrials.gov Identifier:
NCT00146770
First Posted: September 7, 2005
Last Update Posted: April 3, 2015
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
Collaborator:
BioMarin/Genzyme LLC
Information provided by:
Sanofi
  Purpose
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.

Condition Intervention Phase
Mucopolysaccharidosis I Hurler's Syndrome Hurler-Scheie Syndrome Scheie Syndrome Biological: Aldurazyme Biological: placebo Phase 3

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Multicenter, Multinational, Open-Label Extension Study of the Safety and Efficacy of Aldurazyme® (Laronidase) in Patients With Mucopolysaccharidosis I

Resource links provided by NLM:


Further study details as provided by Sanofi:

Primary Outcome Measures:
  • 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.


Secondary Outcome Measures:
  • 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.


Other Outcome Measures:
  • Change From Baseline to Week 182 in Urinary GAG Level [ Time Frame: Baseline to Week 182 ]

    Urinary Glycosaminoglycan (GAG) Levels:

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



Enrollment: 45
Study Start Date: May 2001
Study Completion Date: March 2005
Primary Completion Date: March 2005 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Active Comparator: Placebo/Aldurazyme
Patients received placebo for 26 weeks in the Double-Blind Study then received 182 weeks of Aldurazyme (0.58 mg/kg every week) in this Extension Study; patients received a total of 182 weeks of Aldurazyme.
Biological: Aldurazyme
Placebo for 26 weeks then 0.58 mg/kg Aldurazyme every week for 182 weeks
Biological: placebo
Placebo for 26 weeks
Active Comparator: Aldurazyme/Aldurazyme
Patients received 26 weeks of Aldurazyme in the Double-Blind Study and then received 182 weeks of Aldurazyme in this Extension Study; patients received a total of 208 weeks of Aldurazyme.
Biological: Aldurazyme
0.58 mg/kg Aldurazyme every week for 208 weeks

  Eligibility

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Ages Eligible for Study:   Child, Adult, Senior
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • The patient or patient's legal guardian must provide written informed consent prior to any protocol-related procedures being performed.
  • The patient must have successfully completed Study ALID-003-99 (who received 21 of 26 consecutive weekly infusions).
  • The patient has not experienced any safety issues that would contraindicate participation in the Extension study.
  • A female patient of childbearing potential must have a negative pregnancy test at entry

Exclusion Criteria:

  • The patient is pregnant or lactating.
  • The patient has received an investigational drug within 30 days prior to the study enrollment.
  • The patient has a medical condition, serious intercurrent illness, or other extenuating circumstance that may significantly interfere with study compliance including all prescribed evaluations and follow-up activities
  Contacts and Locations
Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00146770


  Show 25 Study Locations
Sponsors and Collaborators
Genzyme, a Sanofi Company
BioMarin/Genzyme LLC
Investigators
Study Director: Medical Monitor Genzyme, a Sanofi Company
  More Information

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: Medical Monitor, Genzyme Corporation
ClinicalTrials.gov Identifier: NCT00146770     History of Changes
Other Study ID Numbers: ALID-006-01
First Submitted: September 2, 2005
First Posted: September 7, 2005
Results First Submitted: January 7, 2009
Results First Posted: June 16, 2009
Last Update Posted: April 3, 2015
Last Verified: March 2015

Additional relevant MeSH terms:
Mucopolysaccharidoses
Mucopolysaccharidosis I
Syndrome
Disease
Pathologic Processes
Carbohydrate Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Lysosomal Storage Diseases
Mucinoses
Connective Tissue Diseases
Metabolic Diseases


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