Study of Intrathecal Idursulfase-IT Administered in Conjunction With Elaprase® in Pediatric Patients With Hunter Syndrome and Early Cognitive Impairment

Inclusion Criteria

• •Patients must meet all of the following criteria to be considered eligible for randomization in the pivotal study:
• •1. The patient is male and is ≥3 and \<18 years of age at the time of informed consent.
• •2. The patient must have a documented diagnosis of MPS II.
• •3. The patient has evidence at Screening of Hunter syndrome-related cognitive impairment defined as follows:
• •A patient who is ≥3 and \<13 years of age must have one of the following criteria (3a OR 3b):
• •1. A GCA score ≥55 and ≤85 OR
• •2. If the patient has a GCA score at Screening \>85, there must be evidence of a decrease in GCA score of ≥10 points over 12 months from a previously documented test result in observational study HGT-HIT-090.
• •A patient who is ≥13 and \<18 years of age must have both of the following criteria (3c AND 3d):
• •3. A GCA score of ≥55 and ≤85. AND
• •4. There must be evidence of a decrease in GCA score of ≥10 points over 12 months from a previously documented
• •4. The patient has received and tolerated a minimum of 4 months of therapy with Elaprase during the period immediately prior to Screening.
• •5. The patient must have sufficient auditory capacity, with a hearing aid(s), if needed, in the Investigator's judgment to complete the required protocol testing and must be compliant with wearing the hearing aid(s), if needed, on scheduled testing days.
• •6. The patient's parent(s) or legally authorized guardian(s) must have voluntarily signed an Institutional Review Board/Independent Ethics Committee 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, if applicable, must be obtained prior to the start of any study procedures.
• •Patients must meet all of the following criteria to be considered eligible for enrollment in the separate substudy:
• •1. The patient is male and is \<3 years of age at the time of informed consent.
• •2. The patient must have a documented diagnosis of MPS II.
• •3. The patient has evidence at Screening of Hunter syndrome-related cognitive impairment
• •4. The patient has received and tolerated a minimum of 4 months of therapy with Elaprase during the period immediately prior to Screening.
• •5. The patient must have sufficient auditory capacity, with a hearing aid(s), if needed, in the Investigator's judgment to complete the required protocol testing and must be compliant with wearing the hearing aid(s), if needed, on scheduled testing days.
• •6. The patient's parent(s) or legally authorized guardian(s) must have voluntarily signed an Institutional Review Board/Independent Ethics Committee 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) must be obtained prior to the start of any study procedures.