A Study Investigating the Relationship Between the Development of Laronidase Antibody and Urinary GAG (Glycosaminoglycan) Levels in Aldurazyme� Treated Patients
Information source: Genzyme
Information obtained from ClinicalTrials.gov on December 08, 2011
Link to the current ClinicalTrials.gov record.
Condition(s) targeted: Mucopolysaccharidosis I; Hurler's Syndrome; Hurler-Scheie Syndrome; Scheie's Syndrome
Intervention: laronidase (Drug)
Phase: Phase 4
Status: Recruiting
Sponsored by: Genzyme Official(s) and/or principal investigator(s):
Medical Monitor, Study Director, Affiliation: Genzyme
Overall contact:
Medical Information, Phone: 800-745-4447, Email: medinfo@genzyme.com
Summary
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.
Clinical Details
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).
Study design: Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Primary outcome: Urinary GAG (glycosamnioglycans)Immunogenicity Testing
Secondary outcome: Safety
Eligibility
Minimum age: N/A.
Maximum age: N/A.
Gender(s): Both.
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
Locations and Contacts
Medical Information, Phone: 800-745-4447, Email: medinfo@genzyme.com
Children's Hospital Los Angeles, Los Angeles, California 90027, United States; Completed
Connecticut Children's Medical Center, Hartford, Connecticut 06106, United States; Completed
Regents of the University of Minnesota, Minneapolis, Minnesota 55455, United States; Completed
Medical College of Wisconsin, Inc., Milwaukee, Wisconsin 53226, United States; Recruiting
Additional Information
Starting date: July 2004
Last updated: April 29, 2010
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