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

Phase 4

Completed

• Conditions: Mucopolysaccharidosis I; Hurler's Syndrome; Hurler-Scheie Syndrome; Scheie's Syndrome

• Registration Number: NCT00144768
• Lead Sponsor: Genzyme, a Sanofi Company

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.

Detailed Description

Not available

Basic Information
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Recruitment & Eligibility
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Study & Design
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Trial Locations

Study Timeline

• Study Start: 2004-07
• Study First Submitted: 2005-09-02
• Study First Submitted QC: 2005-09-02
• Study First Posted: 2005-09-05
• Primary Completion: 2007-05
• Study Completion: 2007-05
• Status Verified: 2013-04
• Last Update Submitted: 2014-05-02
• Last Update Posted: 2014-05-05

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 25

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.

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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

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Primary Outcome Measures

Name	Time	Method
Immunogenicity Testing	Up to 4 years
Urinary GAG (glycosaminoglycans)	Up to 4 years

Secondary Outcome Measures

Name	Time	Method
Safety	Up to 4 years

Trial Locations

Locations (3)

Connecticut Children's Medical Center; 🇺🇸 Hartford, Connecticut, United States

Children's Hospital of Wisconsin; 🇺🇸 Milwaukee, Wisconsin, United States

Childrens Hospital Los Angeles; 🇺🇸 Los Angles, California, United States