An extension peadiatric study of Idursulfase-IT with Elaprase® in patients with Hunter Syndrome and early cognitive impairment

Recruitment & Eligibility

Status: Authorised-recruitment may be ongoing or finished

Sex: Male

Target Recruitment: 54

Inclusion Criteria

1. Subjects must have completed Visit Week 52 assessments in Study HGT-HIT-094
2. The subject’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 subject’s parent(s) or legally authorized guardian(s) and the subject’s consent/assent, as relevant, must be obtained
3. The subject has continued to receive Elaprase on a regular basis in Study HGT-HIT-094
Are the trial subjects under 18? yes
Number of subjects for this age range: 56
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 subject 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 subject has a known hypersensitivity to any of the components of idursulfase-IT
3. The subject has clinically relevant intracranial hypertension
4. The subject 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 subject 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 subject has a condition that is contraindicated as described in the SOPH-A-PORT Mini S IDDD Instructions for Use, including:
a. The subject has had, or may have, an allergic reaction to the materials of construction of the SOPH-A-PORT Mini S device
b. The subject’s body size is too small to support the size of the SOPH-A-PORT Mini S Access Port, as judged by the Investigator
c. The subject’s drug therapy requires substances known to be incompatible with the materials of construction
d. The subject has a known or suspected local or general infection
e. The subject is at risk of abnormal bleeding due to a medical condition or therapy
f. The subject has one or more spinal abnormalities that could complicate safe implantation or fixation
g. The subject has a functioning CSF shunt device
h. The subject has shown an intolerance to an implanted device

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method

Secondary Outcome Measures

Name	Time	Method

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