An extension to the safety, tolerability and preliminary efficacy study of Idursulfase-IT in patients with Hunter syndrome associated with learning disability

Conditions: Treatment of Hunter syndrome and cognitive impairment
MedDRA version: 16.1Level: PTClassification code 10056889Term: Mucopolysaccharidosis IISystem Organ Class: 10010331 - Congenital, familial and genetic disorders
Therapeutic area: Body processes [G] - Genetic Phenomena [G05]

Registration Number: EUCTR2011-000212-25-Outside-EU/EEA

Lead Sponsor: Shire HGT Inc

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: A

Sex: Male

Target Recruitment: 15

Inclusion Criteria

1 Patients must have completed all study requirements and EOS asessments 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.
Are the trial subjects under 18? yes
Number of subjects for this age range: 15
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

1 The patient is enrolled in another clinical study that involves clinical investigations or use of any investigational product (drug or device) other than the PORT-ACATH IDDD within 30 days prior to study enrollment or at any time during the study.
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.