|Principal Investigator: DaveViskochil|
|Keywords: Hunter syndrome , MPS II||Department: Pediatric Genetics|
|IRB Number: 00046177||Co Investigator: NicolaLongo|
|Specialty: Pediatric Genetics|
|Sub Specialties: Medical Genetics|
- To evaluate the long-term effects of intrathecal idursulfase-IT, given in conjunction with Elaprase, on clinical parameters (eg, physiological assessments, neurocognitive assessments, neurologic function, and brain structure volumes)
- To evaluate the long-term effects of intrathecal idursulfase-IT, given in conjunction with Elaprase, on functional activities of daily living, as determined by the Scales of Independent Behavior-Revised (SIB-R)
- To explore potential relationships between biomarkers and CNS symptomatology
- To determine whether monthly idursulfase-IT administrations results in accumulation of idursulfase within the CSF compartment by measuring idursulfase levels in CSF immediately prior to idursulfase-IT administration
- To determine the safety and performance of the SOPH-A-PORT Mini S
Participant Selection Criteria for Local Site:
• Signed Consent with the main site, University of North Carolina, Chapel Hill.
• Hunter syndrome with CNS involvement, defined per main site inclusion criteria from prior study HGT-HIT-045.
Participant Selection Criteria for Main Site:
Patients must meet all of the following criteria to be considered eligible for enrollment:
1. Patients must have completed all study requirements and EOS 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.
2. 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.
3. The 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.
• None for local site
Patients who meet any of the following criteria are not eligible for participation in this study:
1. The patient has received treatment with any investigational drug (other than idursulfase-IT) or device within 30 days prior to study entry.
2. The patient is unable to comply with the protocol (eg, is unable to return for safety evaluations, or is otherwise unlikely to complete the study) as determined by the Investigator.
3. The patient has experienced an adverse reaction to study drug in Study HGT-HIT-045 that contraindicates further treatment with intrathecal idursulfase-IT.
4. The patient has a known hypersensitivity to any of the components of idursulfase-IT.
An additional exclusion criterion for patients who were previously untreated with intrathecal idursulfase-IT in Study HGT-HIT-045:
1. The patient has an opening CSF pressure upon lumbar puncture that exceeds 30.0 cm H2O.