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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00882921
Recruitment Status : Completed
First Posted : April 17, 2009
Results First Posted : July 30, 2014
Last Update Posted : January 9, 2019
Information provided by (Responsible Party):

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).

Condition or disease Intervention/treatment
Hunter Syndrome Biological: Idursulfase

Detailed Description:
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.

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Study Type : Observational
Actual Enrollment : 26 participants
Observational Model: Cohort
Time Perspective: Prospective
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 Idursulfase Enzyme Replacement Therapy
Actual Study Start Date : October 14, 2008
Actual Primary Completion Date : February 8, 2013
Actual Study Completion Date : February 8, 2013

Group/Cohort Intervention/treatment
Idursulfase 0.5 mg/kg Weekly
Biological: Idursulfase
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.
Other Name: Elaprase

Primary Outcome Measures :
  1. 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.

Secondary Outcome Measures :
  1. Change From Baseline in uGAG Levels to 109 Weeks [ Time Frame: Baseline to 109 Weeks ]
    Urine GAG

Biospecimen Retention:   Samples Without DNA
Blood and urine

Information from the National Library of Medicine

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Ages Eligible for Study:   5 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Patients with Hunter syndrome

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT00882921

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United States, California
Children's Hospital & Research Center Oakland
Oakland, California, United States, 94609
United States, Minnesota
Children's Hospitals and Clinics of Minnesota, Division of Genetics
Minneapolis, Minnesota, United States, 55404
Hospital de Clinicas de Porto Alegre, Servico de Genetica Medica
Porto Alegre, RS, Brazil, 90035-903
United Kingdom
Birmingham Children's Hospital
Birmingham, United Kingdom, B46NH
Great Ormond Street Hospital
London, United Kingdom, WC1N 3JH
Central Manchester University Hospitals, St. Mary's Hospital
Manchester, United Kingdom, M139WL
Sponsors and Collaborators
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Study Director: Study Director Shire

Publications of Results:
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Responsible Party: Shire Identifier: NCT00882921     History of Changes
Other Study ID Numbers: HGT-ELA-042
First Posted: April 17, 2009    Key Record Dates
Results First Posted: July 30, 2014
Last Update Posted: January 9, 2019
Last Verified: December 2018

Keywords provided by Shire:
hunter's disease treatment
hunter's disease
iduronate 2 sulfatase
MPS II treatment
hunters syndrome
mps ii therapy
hunter's syndrome
Hunter syndrome
hunter syndrome therapy
hunter syndrome treatment
hunter's syndrome treatment
mps ii
hunters disease
hunter disease
enzyme replacement therapy
mps diagnosis
mps symptoms
iduronate sulfatase
chronic ear infection
lysosomal storage disease
mps society
enlarged adenoids
ert treatment
lysosomal storage disorder

Additional relevant MeSH terms:
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Mucopolysaccharidosis II
Pathologic Processes
Mental Retardation, X-Linked
Intellectual Disability
Neurobehavioral Manifestations
Neurologic Manifestations
Nervous System Diseases
Genetic Diseases, X-Linked
Genetic Diseases, Inborn
Heredodegenerative Disorders, Nervous System
Carbohydrate Metabolism, Inborn Errors
Metabolism, Inborn Errors
Lysosomal Storage Diseases
Connective Tissue Diseases
Metabolic Diseases
Immunologic Factors
Physiological Effects of Drugs