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A Study Investigating the Relationship Between the Development of Laronidase Antibody and Urinary GAG Levels in Aldurazyme® Treated Patients
This study is currently recruiting participants.
Verified by Genzyme, July 2008
Sponsors and Collaborators: Genzyme
BioMarin/Genzyme LLC
Information provided by: Genzyme
ClinicalTrials.gov Identifier: NCT00144768
  Purpose

The purpose of this study is to determine whether the development of antibodies to Aldurazyme® (laronidase) in patients with MPS I receiving Aldurazyme® (laronidase) impairs the clearance of GAG substrate.


Condition Intervention Phase
Mucopolysaccharidosis I
Hurler's Syndrome
Hurler-Scheie Syndrome
Scheie's Syndrome
Drug: Aldurazyme® (laronidase)
Phase IV

Genetics Home Reference related topics: mucopolysaccharidosis type I
Drug Information available for: Immunoglobulins Globulin, Immune Laronidase
U.S. FDA Resources
Study Type: Interventional
Study Design: Treatment, Open Label, Single Group Assignment, Safety/Efficacy Study
Official Title: A Multicenter, Multinational, Open-Label Study of Anti-Laronidase Antibody Formation and Urinary GAG Levels in Patients With Mucopolysaccharidosis I (MPS I) Being Treated With Aldurazyme® (Laronidase).

Further study details as provided by Genzyme:

Primary Outcome Measures:
  • Urinary GAG and Immunogenicity Testing [ Time Frame: Up to 4 years ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Safety [ Time Frame: Up to 4 years ] [ Designated as safety issue: Yes ]

Estimated Enrollment: 25
Study Start Date: July 2004
Estimated Study Completion Date: January 2012
Intervention Details:
    Drug: Aldurazyme® (laronidase)
    dose of 0.58mg/kg body weight IV every week
  Eligibility

Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Have a document diagnosis of MPS I confirmed by measurable signs and symptoms and deficient α-L-iduronidase activity (<10% of the lower limit of normal).
  • For a patients receiving Aldurazyme therapy prior to study entry: Have available as baseline data the results of urinary GAG levels and IgG antibody titers collected prior to the patient's first Aldurazyme infusion.
  • 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.

Exclusion Criteria:

  • Have previously received Aldurazyme without the collection of baseline samples as specified.
  • Have a suspected hypersensitivity to Aldurazyme or a know hypersensitivity to components of infusion solution.
  • Have received a Hematopoietic Stem Cell Transplantation, injection fibroblast therapy, or major organ transplant.
  • Are receiving chronic immunosuppressant therapy.
  • Have a medical condition, serious intercurrent illness, or other extenuating circumstances that may interfere with study compliance including all prescribed evaluations and follow-up activities.
  • Are pregnant or lactating
  • Have received investigational drug within 30days prior to study enrollment
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00144768

Contacts
Contact: Medical Information 800-745-4447 medinfo@genzyme.com
Contact: Medical Information 617-252-7832 medinfo@genzyme.com

Locations
United States, California
Children's Hospital Los Angeles Active, not recruiting
Los Angeles, California, United States, 90027
United States, Connecticut
Connecticut Children's Medical Center Active, not recruiting
Hartford, Connecticut, United States, 06106
United States, Minnesota
Regents of the University of Minnesota Active, not recruiting
Minneapolis, Minnesota, United States, 55455
United States, Wisconsin
Medical College of Wisconsin, Inc. Recruiting
Milwaukee, Wisconsin, United States, 53226
Sponsors and Collaborators
Genzyme
BioMarin/Genzyme LLC
  More Information

US FDA Approved Full Prescribing Information for Aldurazyme®  This link exits the ClinicalTrials.gov site

Responsible Party: Genzyme ( Medical Monitor )
Study ID Numbers: ALID-020-03
Study First Received: September 2, 2005
Last Updated: July 29, 2008
ClinicalTrials.gov Identifier: NCT00144768  
Health Authority: United States: Food and Drug Administration

Study placed in the following topic categories:
Metabolic Diseases
Lysosomal Storage Diseases
Mucopolysaccharidosis I
Alpha-L-iduronidase deficiency
Metabolism, Inborn Errors
Mucopolysaccharidoses
Antibodies
Genetic Diseases, Inborn
Connective Tissue Diseases
Mucopolysaccharidosis
Metabolic disorder
Hurler syndrome
Immunoglobulins

Additional relevant MeSH terms:
Pathologic Processes
Disease
Syndrome
Mucinoses
Carbohydrate Metabolism, Inborn Errors

ClinicalTrials.gov processed this record on January 16, 2009