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

Study Overview
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Brief Summary
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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 Description
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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.

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

Conditions
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Hunter Syndrome

Intervention / Treatment
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* Biological: Idursulfase


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Patients must meet all of the following criteria to be considered eligible for enrollment: The patient is male and enrolled in the HOS (i.e., meets the entry criteria of a documented diagnosis of Hunter syndrome) The patient is ≥ 5 years-old The patient is on idursulfase treatment or scheduled to begin idursulfase treatment within 30 days of study enrollment The patient, patient's parent(s), or patient's legally authorized guardian must have voluntarily signed an Institutional Review Board (IRB)/Independent Ethics Committee (IEC)-approved informed consent form after all relevant aspects of the study have been explained and discussed with the patient, patient's parent(s), or patient's legally authorized guardian. Exclusion Criteria: Patients who meet any of the following criteria are not eligible for this study: The patient has received biologic/ERT products other than idursulfase, or other investigational product(s) for any reason within 30 days prior to study entry. The patient has a life expectancy of < 2 years The patient is unable to comply with the protocol, e.g., has a clinically relevant medical condition making implementation of the protocol difficult; has an uncooperative attitude; is unable to return for safety evaluations; or is otherwise unlikely to complete the study, as determined by the Investigator.

Ages Eligible for Study
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Minimum Age: 5 Years

Sexes Eligible for Study
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Male

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Elaprase<br>Idursulfase 0.5 mg/kg Weekly | Biological: Idursulfase<br>* 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.<br>* Other names: Elaprase;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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. | Baseline to 109 Weeks | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change From Baseline in uGAG Levels to 109 Weeks | Urine GAG | Baseline to 109 Weeks | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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MPS 2, MPSII, hunter's disease treatment, enzyme replacement therapy, hunter's disease, iduronate 2 sulfatase, mps diagnosis, mps symptoms, iduronate sulfatase, MPS II treatment, chronic ear infection, hunters syndrome, mps ii therapy, MPS2, hunter's syndrome, Hunter syndrome, elaprase, lysosomal storage disease, hunter syndrome therapy, mps society, hunter syndrome treatment, hunter's syndrome treatment, mucopolysaccharides, mps ii, idursulfase, hunters disease, enlarged adenoids, ert treatment, hunter disease, lysosomal storage disorder