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Summary

for males ages up to 18 years (full criteria)
at Oakland, California and other locations
study started
estimated completion:

Description

Summary

This extension study will allow participants that completed Study HGT-HIT-094 to continue receiving Elaprase treatment in conjunction with idursulfase IT or to continue receiving Elaprase treatment and begin concurrent IT treatment for those that did not received idursulfase IT treatment in Study HGT-HIT-094.

Official Title

An Open Label Extension of Study HGT-HIT-094 Evaluating Long Term Safety and Clinical Outcomes of Intrathecal Idursulfase Administered in Conjunction With Elaprase® in Patients With Hunter Syndrome and Cognitive Impairment

Keywords

Hunter Syndrome

Eligibility

You can join if…

Open to males ages up to 18 years

  1. Patients must have completed Visit Week 52 assessments in Study HGT-HIT-094
  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 continued to receive Elaprase on a regular basis in Study HGT-HIT-094

You CAN'T join if...

  1. 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
  2. The patient has a known hypersensitivity to any of the components of idursulfase-IT
  3. The patient has clinically relevant intracranial hypertension
  4. 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
  5. 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.
  6. 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

Locations

  • Instituto Nacional de Pediatría
    Coyoacan, Ciudad De México, 04530, Mexico
  • Ann & Robert H Lurie Children's Hospital of Chicago
    Chicago, Illinois, 60611, United States
  • The Hospital for Sick Children
    Toronto, Ontario, M5G 1X8, Canada
  • University of North Carolina
    Chapel Hill, North Carolina, 27514, United States
  • Royal Manchester Children's Hospital
    Manchester, M13 9WL, United Kingdom
  • Hôpital Femme Mère Enfants
    Bron, 69677, France
  • Hospital Infantil Universitario Niño Jesus
    Madrid, 28009, Spain
  • Women's and Children's Hospital
    Adelaide, 5006, Australia

Details

Status
accepting new patients by invitation only
Start Date
Completion Date
(estimated)
Sponsor
Shire
ID
NCT02412787
Phase
Phase 2/3
Study Type
Interventional
Last Updated
March 1, 2017