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

Mucopolysaccharidosis I Clinical Trial

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).

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00144768
• Other study ID #: ALID02003
• Status: Completed
• Phase: Phase 4
• First received: September 2, 2005
• Last updated: May 2, 2014
• Start date: July 2004
• Est. completion date: May 2007

Study information

• Verified date: April 2013
• Source: Sanofi
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Food and Drug Administration
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 25
• Est. completion date: May 2007
• Est. primary completion date: May 2007
• Accepts healthy volunteers: No
• Gender: Both
• Age group: N/A and older

Eligibility

Inclusion Criteria:

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

Study Design

Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Hurler's Syndrome
• Hurler-Scheie Syndrome
• Mucopolysaccharidoses
• Mucopolysaccharidosis I
• Scheie's Syndrome
• Syndrome

Intervention

Drug:
• laronidase
dose of 0.58mg/kg body weight IV every week

Locations

Country	Name	City	State
United States	Connecticut Children's Medical Center	Hartford	Connecticut
United States	Childrens Hospital Los Angeles	Los Angles	California
United States	Children's Hospital of Wisconsin	Milwaukee	Wisconsin

Sponsors (2)

Lead Sponsor	Collaborator
Genzyme, a Sanofi Company	BioMarin/Genzyme LLC

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Urinary GAG (glycosaminoglycans)		Up to 4 years	No
Primary	Immunogenicity Testing		Up to 4 years	No
Secondary	Safety		Up to 4 years	Yes