Clinical Trial: An Observational Study Evaluating Anti-Idursulfase Serum Antibody Response in Hunter Syndrome Patients

Study Status: Completed
Recruit Status: Completed
Study Type: Observational

Official Title: A Multi-Center Observational Study Evaluating Anti-Idursulfase Serum Antibody Response in Hunter Syndrome Patients Enrolled in the Hunter Outcome Survey (HOS) Receiving Id

Brief Summary: The objective of this study is to evaluate the effect of anti-idursulfase antibodies on idursulfase safety (measured by infusion related adverse events) between patients who develop anti-idursulfase antibodies and patients who do not after long-term idursulfase enzyme replacement therapy (ERT).

Detailed Summary: This study is being conducted to satisfy post-marketing commitments to monitor anti-idursulfase antibody development in Hunter syndrome patients after long-term idursulfase enzyme replacement therapy. The study will be conducted as a sub-study within the Hunter Outcome Survey (HOS). Hunter syndrome patients in the HOS who have previously received idursulfase as well as treatment-naive patients who will begin idursulfase treatment within 30 days of study enrollment will be included.
Sponsor: Shire

Current Primary Outcome: Infusion-Related Adverse Event (IRAE) Rates Between IgG Anti-idursulfase Antibody Positive (Ab+) and Anti-idursulfase IgG Antibody Negative (Ab-) Patients [ Time Frame: Baseline to 109 Weeks ]

The primary analysis of how presence of antibodies affected IRAE rates was performed based on a negative binomial regression model. This was done to account for potentially differential follow-up time between antibody groups.


Original Primary Outcome: To evaluate the relative risk of experiencing an infusion-related adverse event given anti-idursulfase antibody positive status relative to anti-idursulfase antibody negative status. [ Time Frame: 2 years ]

Current Secondary Outcome: Change From Baseline in uGAG Levels to 109 Weeks [ Time Frame: Baseline to 109 Weeks ]

Urine GAG


Original Secondary Outcome: To measure the mean (and percent) difference in urinary GAG level between the groups of IgG anti-idursulfase antibody positive and anti-idursulfase IgG antibody negative patients. [ Time Frame: 2 years ]

Information By: Shire

Dates:
Date Received: April 16, 2009
Date Started: October 2008
Date Completion:
Last Updated: August 4, 2014
Last Verified: July 2014