Clinical Trial: 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®

Study Status: Completed
Recruit Status: Completed
Study Type: Observational

Official Title: A Screening Study to Identify Pediatric Patients With Hunter Syndrome Who Demonstrate Evidence of Central Nervous System Involvement and Who Are Currently Receiving Treatm

Brief Summary: 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.

Detailed Summary:
Sponsor: Shire

Current Primary Outcome:

  • Number of Participants Who Were Screened For The Follow-On Study With an Investigational Agent [ Time Frame: 1 month ]

    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.
  • Number of Participants With a Score of at Least 90 on The General Conceptual Ability (GCA) Sub-Scale of The Differential Ability Scale (DAS) [ Time Frame: 1 month ]
    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.


Original Primary Outcome: To determine the neurodevelopmental status, including hearing ability, in a population of pediatric patients with Hunter syndrome who are receiving weekly infusions of Elaprase. [ Time Frame: 1 month ]

Current Secondary Outcome:

Original Secondary Outcome:

Information By: Shire

Dates:
Date Received: July 9, 2009
Date Started: July 2009
Date Completion:
Last Updated: April 11, 2016
Last Verified: December 2015