Clinical Trial: Immune Tolerance Study With Aldurazyme® (Laronidase)

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

Official Title: A Trial of Antigen-specific Immune Tolerance Induction in Mucopolysaccharidosis I (MPS I) Patients Initiating Enzyme Replacement Therapy With Aldurazyme® (Laronidase)

Brief Summary: The purpose of this study is to see if treatment with an antigen-specific immunosuppressive can decrease or stop an antibody response to laronidase (Aldurazyme®) during enzyme replacement therapy with laronidase in severe Mucopolysaccharidosis I (MPS I) participants.

Detailed Summary:
Sponsor: Genzyme, a Sanofi Company

Current Primary Outcome: Number of Participants Who Achieved Immune Tolerance Induction [ Time Frame: 24 weeks after start of full-dose laronidase therapy ]

Immune tolerance induction success was defined as development of an anti-laronidase immunoglobulin G (IgG) antibody titer less than or equal to (<=) 1:3200 after 24 weeks of receiving full-dose (0.58 mg/kg) laronidase therapy.


Original Primary Outcome: Antibody titer to laronidase less than or equal to 1:3200 [ Time Frame: 24 weeks ]

Current Secondary Outcome: Percent Reduction of Urinary Glycosaminoglycan (uGAG) Level From Baseline to the End of Treatment/Early Withdrawal [ Time Frame: Baseline, end of treatment/early withdrawal (up to 24 weeks after start of full-dose laronidase therapy) ]

Urinary Glycosaminoglycan (uGAG) Levels: concentration of glycosaminoglycan (GAG) relative to creatinine in urine. A greater decrease in uGAG level indicates a greater response.


Original Secondary Outcome: Mean uGAG reduction [ Time Frame: 39 weeks ]

Information By: Sanofi

Dates:
Date Received: August 13, 2008
Date Started: September 2008
Date Completion:
Last Updated: June 2, 2014
Last Verified: June 2014