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

A Study to Test the Possibility of Cross Reaction of the Antibodies Induced by the ELAPRASE (R) to GSK2788723 ELAPRASE is a Trade Mark Owned by a Third Party

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

Study Overview

• Status: Completed
• Conditions: Mucopolysaccharidosis II
• Intervention / Treatment: Drug: Idursulfase; Drug: 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

• Study Type: Observational
• Enrollment (Actual): 10

Contacts and Locations

Study Locations

• Japan
  • Tokyo, Japan, 157-8535
    • GSK Investigational Site

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

• Sampling Method: Probability Sample

Study Population

Approximately 11 subjects will be enrolled in the study.

Description

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.

Study Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
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 Names: recombinant IDS; Drug: GSK2788723; GSK2788723 is being developed by JCR and GSK for the treatment of Hunter syndrome

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Level and capability of binding and neutralizing antibodies induced by idursulfase after the administration of idursulfase	To assess the cross-reactivity of the antibodies induced by idursulfase to GSK2788723, in vitro	1 Day

Collaborators and Investigators

• Sponsor: GlaxoSmithKline

Study record dates

Study Major Dates

• Study Start (Actual): April 9, 2012
• Primary Completion (Actual): June 11, 2012
• Study Completion (Actual): June 11, 2012

Study Registration Dates

• First Submitted: May 17, 2012
• First Submitted That Met QC Criteria: May 17, 2012
• First Posted (Estimate): May 21, 2012

Study Record Updates

• Last Update Posted (Actual): September 25, 2017
• Last Update Submitted That Met QC Criteria: September 21, 2017
• Last Verified: September 1, 2017

More Information

Terms related to this study

• Keywords: Hunter syndrome, immunogenicity, antibodies
• Additional Relevant MeSH Terms: Metabolic Diseases; Nervous System Diseases; Neurologic Manifestations; Neurobehavioral Manifestations; Genetic Diseases, Inborn; Genetic Diseases, X-Linked; Connective Tissue Diseases; Carbohydrate Metabolism, Inborn Errors; Metabolism, Inborn Errors; Lysosomal Storage Diseases; Mucinoses; Mental Retardation, X-Linked; Intellectual Disability; Heredodegenerative Disorders, Nervous System; Mucopolysaccharidosis II; Mucopolysaccharidoses
• Other Study ID Numbers: 116406