Status:
COMPLETED
A Study Investigating the Relationship Between the Development of Laronidase Antibody and Urinary GAG (Glycosaminoglycan) Levels in Aldurazyme® Treated Patients
Lead Sponsor:
Genzyme, a Sanofi Company
Collaborating Sponsors:
BioMarin/Genzyme LLC
Conditions:
Mucopolysaccharidosis I
Hurler's Syndrome
Eligibility:
All Genders
Phase:
PHASE4
Brief Summary
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.
Eligibility Criteria
Inclusion
- 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
- 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
Key Trial Info
Start Date :
July 1 2004
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
May 1 2007
Estimated Enrollment :
25 Patients enrolled
Trial Details
Trial ID
NCT00144768
Start Date
July 1 2004
End Date
May 1 2007
Last Update
May 5 2014
Active Locations (3)
Enter a location and click search to find clinical trials sorted by distance.
1
Childrens Hospital Los Angeles
Los Angles, California, United States, 90027
2
Connecticut Children's Medical Center
Hartford, Connecticut, United States, 06106
3
Children's Hospital of Wisconsin
Milwaukee, Wisconsin, United States, 53226