A Safety and Dose Ranging Study of Idursulfase (Intrathecal) Administration Via an Intrathecal Drug Delivery Device in Pediatric Patients With Hunter Syndrome Who Have Central Nervous System Involvement and Are Receiving Treatment With Elaprase®
Completed
Shire
Phase 1/Phase 2
2009-11-18
Elaprase (idursulfase), a large molecular protein, is not expected to cross the blood brain
barrier at therapeutic levels when administered intravenously. A new formulation of
idursulfase, idursulfase-IT, that differs from that of the intravenous (IV) formulation,
Elaprase, has been developed to be suitable for delivery into the cerebrospinal fluid (CSF)
via intrathecal administration.
This Phase I/II study is designed to obtain necessary safety and exposure data, as well as
secondary and exploratory outcome measures, to be interpreted and used in the design of
subsequent clinical trials.
Extension Study of HGT-HIT-045 Evaluating Long-Term Safety and Clinical Outcomes of Idursulfase-IT in Conjunction With Elaprase in Pediatric Participants With Hunter Syndrome and Cognitive Impairment
Active, not recruiting
Shire
Phase 1/Phase 2
2010-08-01
This extension study of HGT-HIT-045 is designed to collect long-term safety data in pediatric
participants with Hunter syndrome and cognitive impairment who are receiving intrathecal (IT)
idursulfase-IT and intravenous (IV) Elaprase enzyme replacement therapy.
A Study to Test the Possibility of Cross Reaction Induced by the Idursulfase Drug to GSK2788723
Completed
GlaxoSmithKline
2012-04-09
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
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