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

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ClinicalTrials.gov Identifier: NCT00144781
Recruitment Status : Completed
First Posted : September 5, 2005
Results First Posted : June 16, 2009
Last Update Posted : April 3, 2015
Sponsor:
Collaborator:
BioMarin/Genzyme LLC
Information provided by:
Sanofi

September 2, 2005
September 5, 2005
March 27, 2009
June 16, 2009
April 3, 2015
December 2004
January 2006   (Final data collection date for primary outcome measure)
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.
To evaluate differences in the pharmacodynamic response and safety of Aldurazyme® (laronidase) does regimens
Complete list of historical versions of study NCT00144781 on ClinicalTrials.gov Archive Site
  • 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.
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A Dose-optimization Study of Aldurazyme® (Laronidase) in Patients With Mucopolysaccharidosis I (MPS I) Disease
A Multicenter, Multinational, Randomized, Dose-Optimization Study of the Safety and Pharmacodynamic Response of Aldurazyme® (Laronidase) in Patients With Mucopolysaccharidosis I
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).
Not Provided
Interventional
Phase 4
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
  • Mucopolysaccharidosis I
  • Hurler's Syndrome
  • Hurler-Scheie Syndrome
  • Scheie Syndrome
  • Biological: Aldurazyme (Recombinant Human Alpha-L-Iduronidase)
    0.58 mg/kg every week
  • Biological: Aldurazyme (Recombinant Human Alpha-L-Iduronidase)
    1.2 mg/kg every week
  • Biological: Aldurazyme (Recombinant Human Alpha-L-Iduronidase)
    1.2 mg/kg every other week
  • Biological: Aldurazyme (Recombinant Human Alpha-L-Iduronidase)
    1.8 mg/kg every other week
  • Active Comparator: 1
    0.58 mg Aldurazyme/kg of body weight (100 U/kg) administered every week (labeled dose). Final Visit is Week 27 for patients randomized to every week regimen.
    Intervention: Biological: Aldurazyme (Recombinant Human Alpha-L-Iduronidase)
  • Active Comparator: 2
    1.2 mg Aldurazyme/kg of body weight (200 U/kg) administered every week. Final Visit is Week 27 for patients randomized to every week regimen.
    Intervention: Biological: Aldurazyme (Recombinant Human Alpha-L-Iduronidase)
  • Active Comparator: 3
    1.2 mg Aldurazyme/kg of body weight (200 U/kg) administered every 2 weeks. Final Visit is Week 26 for patients randomized to every 2 week regimen.
    Intervention: Biological: Aldurazyme (Recombinant Human Alpha-L-Iduronidase)
  • Active Comparator: 4
    1.8 mg Aldurazyme/kg of body weight (300 U/kg) administered every 2 weeks. Final Visit is Week 26 for patients randomized to every 2 week regimen.
    Intervention: Biological: Aldurazyme (Recombinant Human Alpha-L-Iduronidase)
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*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Completed
34
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January 2006
January 2006   (Final data collection date for primary outcome measure)

Inclusion Criteria:

  • Have a documented diagnosis of MPS I, confirmed by measurable clinical signs and symptoms of MPS I, and a documented fibroblast or leukocyte α-L-iduronidase enzyme activity level of less than 10% of the lower limit of the normal range of the measuring laboratory.
  • Weigh at least 12.5 kg.
  • Have serum creatinine and blood urea nitrogen (BUN) values within age appropriate normal ranges.
  • Provide signed, written informed consent prior to any protocol-related procedures being performed. Consent of a legally authorized guardian(s) is (are) required for patients under 18 years. If the patient is under 18 years old and can understand the consent, written informed consent will be required from both the patient and the authorized guardian(s).

Exclusion Criteria:

  • Have previously received Aldurazyme® (laronidase).
  • Have a suspected hypersensitivity to Aldurazyme® (laronidase) or known hypersensitivity to components of the infusion solution.
  • Have previously undergone hematopoietic stem cell transplantation (HSCT; i.e., from bone marrow [BMT], peripheral blood, or umbilical cord blood) or other major organ transplantation.
  • Have a medical condition, serious inter-current illness, or other extenuating circumstance that may interfere with study compliance including all prescribed evaluations and follow-up activities, except the 6MWT. (Note: All patients may not be capable of performing the 6MWT due to age and/or maturity level. Exemption from performing the 6MWT must be obtained in writing by the investigator from the sponsor's medical monitor prior to enrollment).
  • Have an acute illness that requires surgical intervention, and/or anticipates surgery during study participation, and/or has had surgery within 30 days prior to study enrollment.
  • Have received an investigational drug within 30 days prior to study enrollment.
  • Is pregnant or lactating. Female patients of childbearing potential must have a negative pregnancy test [urine β-human chronic gonadotropin (hCG)] at entry (prior to the first infusion). Note: All female patients of childbearing potential and sexually mature males must be advised to use a medically accepted method of contraception throughout the study.
Sexes Eligible for Study: All
Child, Adult, Older Adult
No
Contact information is only displayed when the study is recruiting subjects
Brazil,   Canada
 
 
NCT00144781
ALID-017-03
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Medical Monitor, Genzyme Corporation
Genzyme, a Sanofi Company
BioMarin/Genzyme LLC
Study Director: Medical Information Genzyme, a Sanofi Company
Sanofi
March 2015

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP