Screening Study to Identify Pediatric Patients With Hunter Syndrome Who Demonstrate Evidence of Central Nervous System (CNS) Involvement and Who Are Currently Receiving Treatment With Elaprase®

Shire33 enrolled

Overview

This study is being conducted to identify pediatric patients with Hunter syndrome who have neurodevelopmental disease characteristics, who are currently receiving treatment with Elaprase, and who may be suitable to participate in a clinical study with an investigational agent.

Study Type

OBSERVATIONAL

Enrollment

Conditions

Hunter Syndrome

Interventions

Neurobehavioral testingBEHAVIORAL

If the patient is found to be eligible after completion of a telephone interview, he will undergo further testing to assess his neurodevelopmental status using a standardized battery of neurobehavioral testing.

Visual and auditory assessmentsOTHER

If the patient is found to be eligible after completion of a telephone interview, he will undergo further testing to evaluate his visual and auditory function.

Eligibility

Sex: MALEMin age: 32 MonthsMax age: 18 Years

Medical Language ↔ Plain English

Inclusion Criteria: 1. The patient is male and is ≥3 and \<18 years of age 2. The patient is currently receiving weekly IV infusions of Elaprase. 3. The patient, patient's parent(s), or legally authorized guardian(s) has 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 with the patient. The guardians' consent and subject's assent, as relevant, must be obtained. Exclusion Criteria: 1. The patient has a CNS shunt. 2. The patient has received a hematopoietic stem cell transplant. 3. The patient is currently enrolled in a clinical trial. 4. The patient has a significant medical or psychiatric comorbidity(ies) that might affect study data or confound the integrity of study results.

Locations (2)

University of North Carolina at Chapel Hill

Chapel Hill, North Carolina, United States

Birmingham Children's Hospital

Birmingham, United Kingdom

Outcomes

Primary Outcomes

Number of Participants Who Were Screened For The Follow-On Study With an Investigational Agent

Standardized tests were used to identify patients who were receiving treatment with Elaprase, had cognitive impairment, and were suitable to participate in the follow-on clinical study (HGT-HIT-045). Assessments included: 1-Cognition: The Differential Ability Scale, Second Edition (DAS-II) or the Bayley Scales of Infant Development, Third Edition (BSID-III); 2-Adaptive Behavior: The Scale of Independent Behavior-Revised (SIB-R); 3-Executive Function: The Behavior Rating Inventory of Executive Function-Preschool version (BRIEF-P) for children or the Behavior Rating Inventory of Executive Function (BRIEF) for children less than or ≥6 years of age, respectively; 4-Motor: The Peabody Developmental Motor Scales-2 (PDMS-2) or the Bruininks-Oseretsky Test of Motor Proficiency, Second Edition (BOT-2) for children less than or ≥6 years of age, respectively.

Time frame: 1 month

Number of Participants With a Score of at Least 90 on The General Conceptual Ability (GCA) Sub-Scale of The Differential Ability Scale (DAS)

The GCA sub-scale of the DAS, Second Edition (DAS-II) was used to obtain a general measure of cognitive ability.The maximum score is 120, with a higher score indicating greater cognitive ability. A score of 100 is considered an average score.

Time frame: 1 month

Data from ClinicalTrials.gov

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