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

NCT00144768 | Completed Phase 4

• 1 other identifier: ALID02003
• Study Type: interventional
• Target: 25
• Locations: 1 country
• Sites: 3

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.

Conditions

Mucopolysaccharidosis I; Hurler's Syndrome; Hurler-Scheie Syndrome; Scheie's Syndrome

Outcome Measures

Primary Outcomes (2)

• Urinary GAG (glycosaminoglycans)

  Up to 4 years

• Immunogenicity Testing

  Up to 4 years

Secondary Outcomes (1)

• Safety

  Up to 4 years

Interventions

laronidase DRUG

dose of 0.58mg/kg body weight IV every week

Also known as: Recombinant Human Alpha-L-Iduronidase, Aldurazyme®

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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

Sponsors & Collaborators

• Genzyme, a Sanofi Company: lead
• BioMarin/Genzyme LLC: collaborator

Study Sites (3)

Childrens Hospital Los Angeles

Los Angles, California, 90027, United States

Location

Connecticut Children's Medical Center

Hartford, Connecticut, 06106, United States

Location

Children's Hospital of Wisconsin

Milwaukee, Wisconsin, 53226, United States

Location

MeSH Terms

Conditions

Mucopolysaccharidosis I

Interventions

Iduronidase

Condition Hierarchy (Ancestors)

Mucopolysaccharidoses; Carbohydrate Metabolism, Inborn Errors; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Lysosomal Storage Diseases; Mucinoses; Connective Tissue Diseases; Skin and Connective Tissue Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases

Intervention Hierarchy (Ancestors)

Glycoside Hydrolases; Hydrolases; Enzymes; Enzymes and Coenzymes

Study Officials

• Medical Monitor

  Genzyme, a Sanofi Company

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 2, 2005
• First Posted: September 5, 2005
• Study Start: July 1, 2004
• Primary Completion: May 1, 2007
• Study Completion: May 1, 2007
• Last Updated: May 5, 2014

Record last verified: 2013-04

Locations

US (3)