An extension study of intrathecal idursulfase in conjunction with Elaprase in Hunter syndrome
Mucopolysaccharidosis Type II (Hunter Syndrome)
Full Study Name
An Open Label Extension of Study HGT-HIT-094 Evaluating Long Term Safety and Clinical Outcomes of Intrathecal Idursulfase ministered in Conjunction with Elaprase® in Patients with Hunter Syndrome and Cognitive Impairment.
Shire Human Genetic Therapies Inc.
Local Principal Investigator
Active but not recruiting
The primary objective of this study is to evaluate long-term safety in patients with Hunter syndrome and cognitive impairment who are receiving intrathecal idursulfase-IT and intravenous (IV) Elaprase® enzyme replacement therapy (ERT).
- Patients must have completed Visit Week 52 assessments in Study HGT-HIT-094.
- The patient has continued to receive Elaprase on a regular basis in Study HGT-HIT-094.
The patient has experienced, in the opinion of the Investigator, a safety or medical issue that contraindicates treatment with idursulfase-IT, including, but not limited to, uncontrolled seizure disorder, bleeding disorder, and clinically relevant hypertension
- The patient has a known hypersensitivity to any of the components of idursulfase-IT
- The patient has clinically relevant intracranial hypertension
- The patient is enrolled in another clinical study, other than HGT-HIT-094, that involves clinical investigations or use of any investigational product (drug or [intrathecal/spinal] device) within 30 days prior to study enrollment or at any time during the study
- The patient has any known or suspected hypersensitivity to anesthesia or is thought to be at an unacceptably high risk for anesthesia due to compromised airways or other conditions.
- The patient has a condition that is contraindicated as described in the SOPH-A-PORT Mini S IDDD Instructions for Use, including:
- The patient has had, or may have, an allergic reaction to the materials of construction of the SOPH-A-PORT Mini S device
- The patient’s body size is too small to support the size of the SOPH-A-PORT Mini S Access Port, as judged by the Investigator
- The patient’s drug therapy requires substances known to be incompatible with the materials of construction
- The patient has a known or suspected local or general infection
- The patient is at risk of abnormal bleeding due to a medical condition or therapy
- The patient has one or more spinal abnormalities that could complicate safe implantation or fixation
- The patient has a functioning CSF shunt device
- The patient has shown an intolerance to an implanted device