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A Study to Test the Possibility of Cross Reaction Induced by the Idursulfase Drug to GSK2788723

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
GlaxoSmithKline
ClinicalTrials.gov Identifier:
NCT01602601
First received: May 17, 2012
Last updated: May 9, 2013
Last verified: October 2012
  Purpose

Study IDS116406 will be a non-interventional, phlebotomy study in Hunter Syndrome patients who are currently being treated with idursulfase, an enzyme replacement therapy, and in at least a single patient who is naïve to treatment, if possible to recruit. All patients enrolled into the study will have a single blood draw for the analysis of antibodies induced by this enzyme replacement therapy (idursulfase). Patient samples with positive responses to antibodies induced by idursulfase will be used to further evaluate whether the antibodies induced by idursulfase bind to GSK2788723 molecules in vitro and if these antibodies neutralize the bioactivity of GSK2788723 in vitro. Each subject will have a screening visit, which may occur at their regularly scheduled out-patient visit. If the patient consents to participate in the study, a blood sample (total volume of approximately 3mL) for immunogenicity analysis will be drawn before their current treatment infusion


Condition Intervention
Mucopolysaccharidosis II
Drug: Idursulfase
Drug: GSK2788723

Study Type: Observational
Study Design: Observational Model: Case-Only
Time Perspective: Cross-Sectional
Official Title: A Study to Test the Possibility of Cross Reaction of the Antibodies Induced by the ELAPRASE (R) to GSK2788723ELAPRASE is a Trade Mark Owned by a Third Party

Resource links provided by NLM:


Further study details as provided by GlaxoSmithKline:

Primary Outcome Measures:
  • Level and capability of binding and neutralizing antibodies induced by idursulfase after the administration of idursulfase [ Time Frame: 1 Day ] [ Designated as safety issue: No ]
    To assess the cross-reactivity of the antibodies induced by idursulfase to GSK2788723, in vitro


Biospecimen Retention:   Samples With DNA

blood samples will be collected and serum will be analyzed and retained


Enrollment: 10
Study Start Date: April 2012
Study Completion Date: April 2012
Primary Completion Date: April 2012 (Final data collection date for primary outcome measure)
Groups/Cohorts Assigned Interventions
Cohort 1
Patients will have a single blood draw for the analysis of antibodies induced by idursulfase. Samples will be used to further evaluate whether the antibodies induced by idursulfase bind to GSK2788723 molecules in vitro and if these antibodies neutralize the bioactivity of GSK2788723 in vitro.
Drug: Idursulfase
recombinant version of IDS produced from human fibroblast. idursulfase is approved for every week (EW) intravenous (IV) administration.
Other Name: recombinant IDS
Drug: GSK2788723
GSK2788723 is being developed by JCR and GSK for the treatment of Hunter syndrome
Other Name: GSK2788723

Detailed Description:

Study IDS116406 will be a non-interventional, phlebotomy study in Hunter Syndrome patients who are currently being treated with idursulfase, an enzyme replacement therapy, and in at least a single patient who is naïve to treatment, if possible to recruit. All patients enrolled into the study will have a single blood draw for the analysis of antibodies induced by this enzyme replacement therapy (idursulfase). Patient samples with positive responses to antibodies induced by idursulfase will be used to further evaluate whether the antibodies induced by idursulfase bind to GSK2788723 molecules in vitro and if these antibodies neutralize the bioactivity of GSK2788723 in vitro. Each subject will have a screening visit, which may occur at their regularly scheduled out-patient visit. If the patient consents to participate in the study, a blood sample (total volume of approximately 3mL) for immunogenicity analysis will be drawn before their current treatment infusion

  Eligibility

Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Sampling Method:   Probability Sample
Study Population

Approximately 11 subjects will be enrolled in the study.

Criteria

Inclusion Criteria:

A subject will be eligible for inclusion in this study only if patient meets criteria 1 and 2 or 1 and 3:

  • Diagnosed with Hunter syndrome
  • Patients with Hunter syndrome who are being treated with idursulfase
  • Patients with Hunter syndrome who are naïve of idursulfase treatment (if possible*) *This study will also attempt to recruit 1 patient who has never received idursulfase. If a naïve patient has not been identified within an agreed amount of time between GSK and the investigator, the study will concluded without this sample

Exclusion Criteria:

  • Subjects who are currently participating in another clinical trial are not permitted to be enrolled in this study.
  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: NCT01602601

Locations
Japan
GSK Investigational Site
Tokyo, Japan, 157-8535
Sponsors and Collaborators
GlaxoSmithKline
Investigators
Study Director: GSK Clinical Trials GlaxoSmithKline
  More Information

No publications provided

Responsible Party: GlaxoSmithKline
ClinicalTrials.gov Identifier: NCT01602601     History of Changes
Other Study ID Numbers: 116406
Study First Received: May 17, 2012
Last Updated: May 9, 2013
Health Authority: United States: Food and Drug Administration

Keywords provided by GlaxoSmithKline:
Hunter syndrome, immunogenicity, antibodies

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

ClinicalTrials.gov processed this record on November 25, 2014