Open-Label-Extension-of-Study-HGT-HIT-045

Clinical Trial Title	Evaluating Long-Term Safety and Clinical Outcomes of Intrathecal Idursulfase-IT Administered in Conjunction With Intravenous Elaprase in Pediatric Patients With Hunter Syndrome and Cognitive Impairment
Trial Status	Closed to Enrollment
Start Date	01/09/2012
Location	randall-childrens-hospital-at-legacy-emanuel
Trial Type	Genetic Diseases
Specific Condition	Hunter's Syndrome
Description	Elaprase, a large molecular protein, is not expected to cross the blood brain barrier when administered intravenously. A revised 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 extension study of HGT-HIT-045 is designed to collect long-term safety data in pediatric patients with Hunter syndrome and cognitive impairment who are receiving intrathecal idursulfase-IT and intravenous Elaprase enzyme replacement therapy.
Eligibility Criteria	Ages Eligible for Study: 3 Years to 18 Years Patient must have completed all study requirements and end of study assessments for study HGT-HIT-045 prior to enrolling in Study HGT-HIT-046 and must have no safety or medical issues that contraindicate participation. The patient's parent(s) or legally authorized guardian(s) 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. Consent of the patient's parent(s) or legally authorized guardian(s) and the patient's assent, as relevant, must be obtained. That patient has received and tolerated a minimum of 12 months of treatment with weekly IV infusions of Elaprase and has received 80% of the total planned infusions within the last 6 months.
IRB Number	10601
Notes	https://clinicaltrials.gov/ct2/show/NCT01506141 Study status is: Active, not recruiting: The study is ongoing, and participants are receiving an intervention or being examined, but potential participants are not currently being recruited or enrolled.
Principal Investigator	George Anadiotis, DO
Contact Name	Kristin Hickey
Contact Phone	(503) 413-5447
Contact Fax
Contact E-Mail	khickey@lhs.org