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

This study is currently recruiting participants.

Verified April 2010 by Genzyme

First Received on September 2, 2005. Last Updated on April 29, 2010 History of Changes

Sponsor:	Genzyme
Collaborator:	BioMarin/Genzyme LLC
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: laronidase	Phase IV

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:

Genetics Home Reference related topics: mucopolysaccharidosis type I Schindler disease

Drug Information available for: Laronidase

U.S. FDA Resources

Further study details as provided by Genzyme:

Primary Outcome Measures:

Urinary GAG (glycosamnioglycans) [ 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
Estimated Study Completion Date:	January 2012
Estimated Primary Completion Date:	January 2012 (Final data collection date for primary outcome measure)

Intervention Details:

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

Please refer to this study by its ClinicalTrials.gov identifier: NCT00144768

Contacts

Contact: Medical Information	800-745-4447	medinfo@genzyme.com
Contact: Medical Information	617-252-7832	medinfo@genzyme.com

Locations

United States, California
Children's Hospital Los Angeles	Completed
Los Angeles, California, United States, 90027
United States, Connecticut
Connecticut Children's Medical Center	Completed
Hartford, Connecticut, United States, 06106
United States, Minnesota
Regents of the University of Minnesota	Completed
Minneapolis, Minnesota, United States, 55455
United States, Wisconsin
Medical College of Wisconsin, Inc.	Recruiting
Milwaukee, Wisconsin, United States, 53226

Sponsors and Collaborators

Genzyme

BioMarin/Genzyme LLC

Investigators

Study Director:

Medical Monitor

Genzyme

More Information

No publications provided

Responsible Party:	Medical Monitor, Genzyme Corporation
ClinicalTrials.gov Identifier:	NCT00144768 History of Changes
Other Study ID Numbers:	ALID-020-03
Study First Received:	September 2, 2005
Last Updated:	April 29, 2010
Health Authority:	United States: Food and Drug Administration

Additional relevant MeSH terms:

Mucopolysaccharidosis I
Mucopolysaccharidoses
Carbohydrate Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn

Lysosomal Storage Diseases
Mucinoses
Connective Tissue Diseases
Metabolic Diseases

ClinicalTrials.gov processed this record on February 09, 2012