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.

Study ID



Shire Human Genetic Therapies Inc.

Local Principal Investigator

Simon Jones

Study Status

Active but not recruiting

Main Aims

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).

Inclusion Criteria
  • 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.
Exclusion Criteria

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:
    1. The patient has had, or may have, an allergic reaction to the materials of construction of the SOPH-A-PORT Mini S device
    2. 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
    3. The patient’s drug therapy requires substances known to be incompatible with the materials of construction
    4. The patient has a known or suspected local or general infection
    5. The patient is at risk of abnormal bleeding due to a medical condition or therapy
    6. The patient has one or more spinal abnormalities that could complicate safe implantation or fixation
    7. The patient has a functioning CSF shunt device
    8. The patient has shown an intolerance to an implanted device