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).
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.
Study Type
OBSERVATIONAL
Enrollment
26
Patients received idursulfase as prescribed by their physician following locally approved prescribing information. Patients will not be provided idursulfase by Shire Human Genetic Therapies, Inc. or the HOS.
Children's Hospital & Research Center Oakland
Oakland, California, United States
Children's Hospitals and Clinics of Minnesota, Division of Genetics
Minneapolis, Minnesota, United States
Hospital de Clinicas de Porto Alegre, Servico de Genetica Medica
Porto Alegre, Rio Grande do Sul, Brazil
Birmingham Children's Hospital
Birmingham, United Kingdom
Infusion-Related Adverse Event (IRAE) Rates Between IgG Anti-idursulfase Antibody Positive (Ab+) and Anti-idursulfase IgG Antibody Negative (Ab-) Patients
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.
Time frame: Baseline to 109 Weeks
Change From Baseline in uGAG Levels to 109 Weeks
Urine GAG
Time frame: Baseline to 109 Weeks
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Great Ormond Street Hospital
London, United Kingdom
Central Manchester University Hospitals, St. Mary's Hospital
Manchester, United Kingdom