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®
|ClinicalTrials.gov Identifier: NCT00937794|
Recruitment Status : Completed
First Posted : July 13, 2009
Results First Posted : May 16, 2016
Last Update Posted : May 16, 2016
|Condition or disease||Intervention/treatment|
|Hunter Syndrome||Behavioral: Neurobehavioral testing Other: Visual and auditory assessments|
|Study Type :||Observational|
|Actual Enrollment :||33 participants|
|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 Treatment With Elaprase®|
|Study Start Date :||July 2009|
|Actual Primary Completion Date :||July 2011|
|Actual Study Completion Date :||July 2011|
This is a screening study designed to evaluate the behavioral, physical, and neurodevelopmental status in pediatric patients with Hunter syndrome who have early signs and symptoms of CNS involvement and who are currently receiving treatment with Elaprase.
Behavioral: Neurobehavioral testing
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.Other: Visual and auditory assessments
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.
- 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:
- Cognition: The Differential Ability Scale, Second Edition (DAS-II) or the Bayley Scales of Infant Development, Third Edition (BSID-III);
- Adaptive Behavior: The Scale of Independent Behavior-Revised (SIB-R);
- 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;
- 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.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00937794
|United States, North Carolina|
|University of North Carolina at Chapel Hill|
|Chapel Hill, North Carolina, United States, 27599|
|Birmingham Children's Hospital|
|Birmingham, United Kingdom, B46NH|
|Principal Investigator:||Joseph Muenzer, MD, PhD||University of North Carolina, Chapel Hill|