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:
Collaborator:
BioMarin/Genzyme LLC
Information provided by (Responsible Party):
Sanofi ( Genzyme, a Sanofi Company )
ClinicalTrials.gov Identifier:
NCT00144768
First received: September 2, 2005
Last updated: May 2, 2014
Last verified: April 2013
  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: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: 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:

Primary Outcome Measures:
  • Urinary GAG (glycosaminoglycans) [ Time Frame: Up to 4 years ] [ Designated as safety issue: No ]
  • 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
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

Genders Eligible for Study:   Both
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
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: 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

No publications provided

Responsible Party: Sanofi ( Genzyme, a Sanofi Company )
ClinicalTrials.gov Identifier: NCT00144768     History of Changes
Other Study ID Numbers: ALID02003
Study First Received: September 2, 2005
Last Updated: May 2, 2014
Health Authority: United States: Food and Drug Administration

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

ClinicalTrials.gov processed this record on October 23, 2014