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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®

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ClinicalTrials.gov Identifier: NCT00937794
Recruitment Status : Completed
First Posted : July 13, 2009
Results First Posted : May 16, 2016
Last Update Posted : May 16, 2016
Sponsor:
Information provided by (Responsible Party):
Shire

Tracking Information
First Submitted Date July 9, 2009
First Posted Date July 13, 2009
Results First Submitted Date December 18, 2015
Results First Posted Date May 16, 2016
Last Update Posted Date May 16, 2016
Study Start Date July 2009
Actual Primary Completion Date July 2011   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures
 (submitted: April 11, 2016)
  • 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 Measures
 (submitted: July 10, 2009)
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 ]
Change History Complete list of historical versions of study NCT00937794 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures Not Provided
Original Secondary Outcome Measures Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title 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®
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®
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 Description Not Provided
Study Type Observational
Study Design Observational Model: Cohort
Time Perspective: Prospective
Target Follow-Up Duration Not Provided
Biospecimen Not Provided
Sampling Method Non-Probability Sample
Study Population Initial patient eligibility will be based on patient age and gender. Patients must be receiving weekly IV Elaprase infusions to be eligible for enrollment.
Condition Hunter Syndrome
Intervention
  • 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.
Study Groups/Cohorts 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
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status Completed
Actual Enrollment
 (submitted: April 30, 2013)
33
Original Estimated Enrollment
 (submitted: July 10, 2009)
75
Actual Study Completion Date July 2011
Actual Primary Completion Date July 2011   (Final data collection date for primary outcome measure)
Eligibility Criteria

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.
Sex/Gender
Sexes Eligible for Study: Male
Ages 32 Months to 18 Years   (Child, Adult)
Accepts Healthy Volunteers No
Contacts Contact information is only displayed when the study is recruiting subjects
Listed Location Countries United Kingdom,   United States
Removed Location Countries  
 
Administrative Information
NCT Number NCT00937794
Other Study ID Numbers HGT-HIT-050
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement Not Provided
Responsible Party Shire
Study Sponsor Shire
Collaborators Not Provided
Investigators
Principal Investigator: Joseph Muenzer, MD, PhD University of North Carolina, Chapel Hill
PRS Account Shire
Verification Date December 2015