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

April 16, 2009
August 4, 2014
October 2008
February 2013   (final data collection date for primary outcome measure)
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.
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 ] [ Designated as safety issue: Yes ]
Complete list of historical versions of study NCT00882921 on ClinicalTrials.gov Archive Site
Change From Baseline in uGAG Levels to 109 Weeks [ Time Frame: Baseline to 109 Weeks ] [ Designated as safety issue: No ]
Urine GAG
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 ] [ Designated as safety issue: No ]
Not Provided
Not Provided
 
An Observational Study Evaluating Anti-Idursulfase Serum Antibody Response in Hunter Syndrome Patients
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

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.

Observational
Observational Model: Cohort
Time Perspective: Prospective
Not Provided
Retention:   Samples Without DNA
Description:

Blood and urine

Non-Probability Sample

Patients with Hunter syndrome

Hunter Syndrome
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
Elaprase
Idursulfase 0.5 mg/kg Weekly
Intervention: Biological: Idursulfase
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Completed
26
February 2013
February 2013   (final data collection date for primary outcome measure)

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.
Male
5 Years and older
No
Contact information is only displayed when the study is recruiting subjects
United Kingdom,   United States,   Brazil
 
NCT00882921
HGT-ELA-042
No
Shire
Shire
Not Provided
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.
Shire
July 2014

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP