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®

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Shire
ClinicalTrials.gov Identifier:
NCT00937794
First received: July 9, 2009
Last updated: April 11, 2016
Last verified: December 2015

July 9, 2009
April 11, 2016
July 2009
July 2011   (final data collection date for primary outcome measure)
  • Number of Participants Who Were Screened For The Follow-On Study With an Investigational Agent [ Time Frame: 1 month ] [ Designated as safety issue: No ]

    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 ] [ Designated as safety issue: No ]
    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.
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 ] [ Designated as safety issue: No ]
Complete list of historical versions of study NCT00937794 on ClinicalTrials.gov Archive Site
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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®
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®
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.
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Observational
Observational Model: Cohort
Time Perspective: Prospective
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Non-Probability Sample
Initial patient eligibility will be based on patient age and gender. Patients must be receiving weekly IV Elaprase infusions to be eligible for enrollment.
Hunter Syndrome
  • 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.
No treatment
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.
Interventions:
  • Behavioral: Neurobehavioral testing
  • Other: Visual and auditory assessments
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*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Completed
33
July 2011
July 2011   (final data collection date for primary outcome measure)

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.
Male
32 Months to 18 Years   (Child, Adult)
No
Contact information is only displayed when the study is recruiting subjects
United States,   United Kingdom
 
NCT00937794
HGT-HIT-050
No
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Shire
Shire
Not Provided
Principal Investigator: Joseph Muenzer, MD, PhD University of North Carolina, Chapel Hill
Shire
December 2015

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP