ALID02003: A Study Investigating the Relationship Between the Development of Laronidase Antibody and Urinary GAG Levels in Aldurazyme® Treated Patients

This study is currently recruiting patients.

Sponsored By:	Genzyme BioMarin/Genzyme LCC
Information Provided By:	Genzyme
ClinicalTrials.gov Identifier:	NCT00144768

Purpose

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.

Condition	Intervention	Phase
Mucopolysaccharidosis I Hurler's Syndrome Hurler-Scheie Syndrome Scheie's Syndrome	Drug: Recombinant Human Alpha-L-Iduronidase, Aldurazyme® (laronidase)	Phase IV

Study Type: Interventional

Study Design: Treatment, Open Label, Uncontrolled, Single Group Assignment, Safety/Efficacy Study

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).

Further Study Details:

Primary Outcomes: To evaluate clinical significance of anti-laronidase IgG antibody formation to Aldurazyme® (laronidase) therapy.

Study start: July 2004; Expected completion: July 2011

Last follow-up: December 2010; Data entry closure: March 2011

Eligibility

Genders Eligible for Study: Both

Criteria

Inclusion Criteria:

Have a document 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

Location Information

Please refer to this study by ClinicalTrials.gov identifier NCT00144768
Medical Information, 800-745-4447, medinfo@genzyme.com
Medical Information, 617-252-7832, medinfo@genzyme.com

California
Children's Hospital Los Angeles, Los Angeles, California, 90027, United States; Recruiting

Connecticut
Connecticut Children's Medical Center, Hartford, Connecticut, 06106, United States; Recruiting

Minnesota
Regents of the University of Minnesota, Minneapolis, Minnesota, 55455, United States; Recruiting

Wisconsin
Medical College of Wisconsin, Inc., Milwaukee, Wisconsin, 53226, United States; Recruiting

More Information

US FDA Approved Full Prescribing Information for Aldurazyme®

Study ID Numbers: ALID02003
ClinicalTrials.gov Identifier: NCT00144768
Health Authority: United States: Food and Drug Administration