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
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
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
Urinary GAG (glycosaminoglycans)
Secondary outcome: Safety
Minimum age: N/A.
Maximum age: N/A.
- 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.
- 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
Starting date: July 2004
Last updated: May 2, 2014