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

Information source: Sanofi
ClinicalTrials.gov processed this data on August 20, 2015
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: Completed

Sponsored by: Genzyme, a Sanofi Company

Official(s) and/or principal investigator(s):
Medical Monitor, Study Director, Affiliation: Genzyme, a Sanofi Company


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.

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 (glycosaminoglycans)

Immunogenicity Testing

Secondary outcome: Safety


Minimum age: N/A. Maximum age: N/A. Gender(s): Both.


Inclusion Criteria:

- Have a documented diagnosis of MPS I confirmed by measurable signs and symptoms and

deficient ╬▒-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


- 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

Childrens Hospital Los Angeles, Los Angles, California 90027, United States

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

Children's Hospital of Wisconsin, Milwaukee, Wisconsin 53226, United States

Additional Information

Starting date: July 2004
Last updated: May 2, 2014

Page last updated: August 20, 2015

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