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

NCT01602601 | Completed

• 1 other identifier: 116406
• Study Type: observational
• Target: 10
• Locations: 1 country
• Sites: 1

Brief Summary

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

Conditions

Mucopolysaccharidosis II

Keywords

Hunter syndrome, immunogenicity, antibodies

Outcome Measures

Primary Outcomes (1)

• 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

Study Arms (1)

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; Drug: GSK2788723

Interventions

Idursulfase DRUG

recombinant version of IDS produced from human fibroblast. idursulfase is approved for every week (EW) intravenous (IV) administration.

Also known as: recombinant IDS

Cohort 1

GSK2788723 DRUG

GSK2788723 is being developed by JCR and GSK for the treatment of Hunter syndrome

Cohort 1

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Probability Sample

Study Population

Approximately 11 subjects will be enrolled in the study.

You may qualify if:

• 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

You may not qualify if:

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

Sponsors & Collaborators

• GlaxoSmithKline: lead

Study Sites (1)

GSK Investigational Site

Tokyo, 157-8535, Japan

Location

Related Links

• Results for study 116406 can be found on the GSK Clinical Study Register.

Biospecimen

Retention: SAMPLES WITH DNA

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

MeSH Terms

Conditions

Mucopolysaccharidosis II

Interventions

idursulfase

Condition Hierarchy (Ancestors)

X-Linked Intellectual Disability; Intellectual Disability; Neurobehavioral Manifestations; Neurologic Manifestations; Nervous System Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Heredodegenerative Disorders, Nervous System; Mucopolysaccharidoses; Carbohydrate Metabolism, Inborn Errors; Metabolism, Inborn Errors; Lysosomal Storage Diseases; Mucinoses; Connective Tissue Diseases; Skin and Connective Tissue Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Officials

• GSK Clinical Trials

  GlaxoSmithKline

  STUDY DIRECTOR

Study Design

• Study Type: observational
• Observational Model: CASE ONLY
• Time Perspective: CROSS SECTIONAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 17, 2012
• First Posted: May 21, 2012
• Study Start: April 9, 2012
• Primary Completion: June 11, 2012
• Study Completion: June 11, 2012
• Last Updated: September 25, 2017

Record last verified: 2017-09

Locations

JP (1)