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

This study has been completed.
Sponsor:
Collaborator:
Genzyme-Biomarin LLC
Information provided by:
Genzyme, a Sanofi Company
ClinicalTrials.gov Identifier:
NCT00912925
First received: June 2, 2009
Last updated: August 21, 2009
Last verified: May 2009
  Purpose

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


Condition Intervention Phase
Mucopolysaccharidosis I
Hurlers Syndrome
Hurler Scheie Syndrome
Scheie
Biological: rhIDU (recombinant human-Alpha-L-Iduronidase)
Biological: Placebo
Phase 3

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Randomized, Double-Blind, Placebo-Controlled, Multicenter, Multinational, Clinical Study of Recombinant Human Alpha L-Iduronidase In Patients With Mucopolysaccharidosis I

Resource links provided by NLM:


Further study details as provided by Genzyme, a Sanofi Company:

Primary Outcome Measures:
  • Overall Change From Baseline to Week 26 in Percent Predicted Forced Vital Capacity (FVC) [ Time Frame: Baseline to Week 26 ] [ Designated as safety issue: No ]
  • Overall Change From Baseline to Week 26 in Six Minute Walk Test (6MWT) [ Time Frame: Baseline to Week 26 ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Overall Change From Baseline to Week 26 in Apnea/Hypopnea Index (AHI) [ Time Frame: Baseline to Week 26 ] [ Designated as safety issue: No ]
  • Overall Percent Change From Baseline to Week 26 in Liver Volume [ Time Frame: Baseline to Week 26 ] [ Designated as safety issue: No ]
  • 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 ] [ Designated as safety issue: No ]
  • Overall Change From Baseline to Week 26 in Active Joint Range of Motion (ROM) [ Time Frame: Baseline to Week 26 ] [ Designated as safety issue: No ]
  • Overall Percent Change From Baseline to Week 26 in Urinary Glycosaminoglycan (GAG) Levels [ Time Frame: Baseline to Week 26 ] [ Designated as safety issue: No ]

Enrollment: 45
Study Start Date: December 2000
Study Completion Date: September 2001
Primary Completion Date: September 2001 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Placebo Comparator: Placebo
Patients in the placebo-control group were administered a solution of 100 millimolar (mM) sodium phosphate, 150 mM sodium chloride, and 0.001% polysorbate-80, adjusted to a pH of 5.8 administered intravenously over approximately 4 hours once weekly for 26 weeks.
Biological: Placebo
Patients in the Placebo-control group were administered a solution of 100mM sodium phosphate , 150mM sodium chloride, and 0.001% polysorbate-80, adjusted to a pH of 5.8 administered intravenously over a time period of approximately 4 hours once weekly for 26 weeks.
Active Comparator: Aldurazyme treatment
Patients in the active treatment group received Aldurazyme intravenously at a dose of 100 Units/kg (approximately 0.58 mg/kg = labeled dose) administered intravenously over approximately 4 hours once weekly for 26 weeks.
Biological: rhIDU (recombinant human-Alpha-L-Iduronidase)
Patients in the active treatment group received Aldurazyme intravenously at a dose of 100 units/kg (approximately 0.58mg/kg) administered intravenously over approximately 4 hours once weekly for 26 weeks.

  Eligibility

Ages Eligible for Study:   5 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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.
  Contacts and Locations
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, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00912925

Locations
United States, New York
New York, New York, United States
United States, North Carolina
Chapel Hill, North Carolina, United States
Canada, British Columbia
Vancouver, British Columbia, Canada
Germany
Mainz, Germany
Sponsors and Collaborators
Genzyme, a Sanofi Company
Genzyme-Biomarin LLC
Investigators
Study Director: Medical Monitor Genzyme, a Sanofi Company
  More Information

No publications provided

Responsible Party: Medical Monitor, Genzyme Corporation
ClinicalTrials.gov Identifier: NCT00912925     History of Changes
Other Study ID Numbers: ALID-003-99
Study First Received: June 2, 2009
Results First Received: July 2, 2009
Last Updated: August 21, 2009
Health Authority: United States: Food and Drug Administration
Canada: Health Canada
European Union: European Medicines Agency
Germany: Federal Institute for Drugs and Medical Devices

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

ClinicalTrials.gov processed this record on September 16, 2014