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A Study Investigating the Relationship Between the Development of Laronidase Antibody and Urinary GAG (Glycosaminoglycan) Levels in Aldurazyme® Treated Patients

This study has been completed.
Sponsor:
ClinicalTrials.gov Identifier:
NCT00144768
First Posted: September 5, 2005
Last Update Posted: May 5, 2014
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 (Responsible Party):
Sanofi ( Genzyme, a Sanofi Company )
  Purpose
The purpose of this study is to determine whether the development of antibodies to laronidase in patients with MPS I receiving Aldurazyme® impairs the clearance of GAG substrate.

Condition Intervention Phase
Mucopolysaccharidosis I Hurler's Syndrome Hurler-Scheie Syndrome Scheie's Syndrome Drug: laronidase Phase 4

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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).

Resource links provided by NLM:


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

Primary Outcome Measures:
  • Urinary GAG (glycosaminoglycans) [ Time Frame: Up to 4 years ]
  • Immunogenicity Testing [ Time Frame: Up to 4 years ]

Secondary Outcome Measures:
  • Safety [ Time Frame: Up to 4 years ]

Estimated Enrollment: 25
Study Start Date: July 2004
Study Completion Date: May 2007
Primary Completion Date: May 2007 (Final data collection date for primary outcome measure)
Intervention Details:
    Drug: laronidase
    dose of 0.58mg/kg body weight IV every week
    Other Name: Recombinant Human Alpha-L-Iduronidase, Aldurazyme®
  Eligibility

Information from the National Library of Medicine

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

Inclusion Criteria:

  • Have a documented 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 30 days prior to study enrollment
  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): NCT00144768


Locations
United States, California
Childrens Hospital Los Angeles
Los Angles, California, United States, 90027
United States, Connecticut
Connecticut Children's Medical Center
Hartford, Connecticut, United States, 06106
United States, Wisconsin
Children's Hospital of Wisconsin
Milwaukee, Wisconsin, United States, 53226
Sponsors and Collaborators
Genzyme, a Sanofi Company
BioMarin/Genzyme LLC
Investigators
Study Director: Medical Monitor Genzyme, a Sanofi Company
  More Information

Responsible Party: Genzyme, a Sanofi Company
ClinicalTrials.gov Identifier: NCT00144768     History of Changes
Other Study ID Numbers: ALID02003
First Submitted: September 2, 2005
First Posted: September 5, 2005
Last Update Posted: May 5, 2014
Last Verified: April 2013

Additional relevant MeSH terms:
Mucopolysaccharidosis I
Syndrome
Mucopolysaccharidoses
Disease
Pathologic Processes
Carbohydrate Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Lysosomal Storage Diseases
Mucinoses
Connective Tissue Diseases
Metabolic Diseases
Antibodies
Immunologic Factors
Physiological Effects of Drugs