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

• Sponsor: Genzyme, a Sanofi Company (Industry)
• Overall Status: Completed
• CT.gov ID: NCT00144768
• Collaborator: BioMarin/Genzyme LLC (Industry)
• Enrollment: 25
• Locations: 3
• Duration (Months): 34
• Patients Per Site: 8.3
• Patients Per Site Per Month: 0.2

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
Mucopolysaccharidosis I; Hurler's Syndrome; Hurler-Scheie Syndrome; Scheie's Syndrome	Drug: laronidase	Phase 4

Study Design

• Study Type: Interventional
• Anticipated Enrollment: 25 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• Masking: None (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).
• Study Start Date: Jul 1, 2004
• Actual Primary Completion Date: May 1, 2007
• Actual Study Completion Date: May 1, 2007

Outcome Measures

Primary Outcome Measures

1. Urinary GAG (glycosaminoglycans) [Up to 4 years]

2. Immunogenicity Testing [Up to 4 years]

Secondary Outcome Measures

1. Safety [Up to 4 years]

Eligibility Criteria

Criteria

• Ages Eligible for Study: N/A and Older
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

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

Locations

	Site	City	State	Country	Postal Code
1	Childrens Hospital Los Angeles	Los Angles	California	United States	90027
2	Connecticut Children's Medical Center	Hartford	Connecticut	United States	06106
3	Children's Hospital of Wisconsin	Milwaukee	Wisconsin	United States	53226

Sponsors and Collaborators

• Genzyme, a Sanofi Company
• BioMarin/Genzyme LLC

Investigators

• Study Director: Medical Monitor, Genzyme, a Sanofi Company

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• Responsible Party: Genzyme, a Sanofi Company
• ClinicalTrials.gov Identifier: NCT00144768
• Other Study ID Numbers: ALID02003
• First Posted: Sep 5, 2005
• Last Update Posted: May 5, 2014
• Last Verified: Apr 1, 2013

• Additional relevant MeSH terms: Mucopolysaccharidoses; Mucopolysaccharidosis I; Syndrome

Study Results

No Results Posted as of May 5, 2014