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

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Shire
ClinicalTrials.gov Identifier:
NCT00882921
First received: April 16, 2009
Last updated: August 4, 2014
Last verified: July 2014
  Purpose

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 Intervention
Hunter Syndrome
Biological: Idursulfase

Study Type: Observational
Study Design: 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

Resource links provided by NLM:


Further study details as provided by Shire:

Primary Outcome Measures:
  • 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 ] [ Designated as safety issue: Yes ]
    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:
  • Change From Baseline in uGAG Levels to 109 Weeks [ Time Frame: Baseline to 109 Weeks ] [ Designated as safety issue: No ]
    Urine GAG


Biospecimen Retention:   Samples Without DNA

Blood and urine


Enrollment: 26
Study Start Date: October 2008
Study Completion Date: February 2013
Primary Completion Date: February 2013 (Final data collection date for primary outcome measure)
Groups/Cohorts Assigned Interventions
Elaprase
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

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.

  Eligibility

Ages Eligible for Study:   5 Years and older
Genders Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population

Patients with Hunter syndrome

Criteria

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.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00882921

Locations
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
Brazil
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
Shire
Investigators
Principal Investigator: Paul R Harmatz, MD Children's Hospital & Research Center Oakland
Principal Investigator: James E Wraith, MD Central Manchester University Hospitals, St. Mary's Hospital
Principal Investigator: Suresh Vijay, MD Birmingham Children's Hospital
Principal Investigator: Roberto Giugliani, MD, PhD Hospital de Clinicas de Porto Alegre
Principal Investigator: Nancy J Mendelsohn, MD Children's Hospitals and Clinics of Minnesota
Principal Investigator: Ashok Vellodi, MD Great Ormond Street Hospital
Study Director: Arian Pano, MD, MPH Shire Human Genetic Therapies, Inc.
  More Information

No publications provided

Responsible Party: Shire
ClinicalTrials.gov Identifier: NCT00882921     History of Changes
Other Study ID Numbers: HGT-ELA-042
Study First Received: April 16, 2009
Results First Received: June 23, 2014
Last Updated: August 4, 2014
Health Authority: United States: Food and Drug Administration

Keywords provided by Shire:
MPS2
MPS 2
mps 2
Hunter syndrome
hunters syndrome
hunter's syndrome
hunter disease
hunters disease
hunter's disease
MPS II
MPSII
mps ii
mucopolysaccharides
lysosomal storage disease
lysosomal storage disorder
chronic ear infection
enlarged adenoids
mps symptoms
mps diagnosis
mps ii therapy
MPS II therapy
MPS II treatment
ert treatment
elaprase
idursulfase
iduronate sulfatase
iduronate 2 sulfatase
enzyme replacement therapy
hunter syndrome treatment
hunter's syndrome treatment

Additional relevant MeSH terms:
Mucopolysaccharidosis II
Syndrome
Mental Retardation, X-Linked
Intellectual Disability
Neurobehavioral Manifestations
Neurologic Manifestations
Nervous System Diseases
Genetic Diseases, X-Linked
Genetic Diseases, Inborn
Heredodegenerative Disorders, Nervous System
Mucopolysaccharidoses
Carbohydrate Metabolism, Inborn Errors
Metabolism, Inborn Errors
Lysosomal Storage Diseases
Mucinoses
Connective Tissue Diseases
Metabolic Diseases
Disease
Pathologic Processes

ClinicalTrials.gov processed this record on September 16, 2014