Working...
ClinicalTrials.gov
ClinicalTrials.gov Menu

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®

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
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

Study Type Observational
Study Design Observational Model: Cohort;   Time Perspective: Prospective
Condition Hunter Syndrome
Interventions Behavioral: Neurobehavioral testing
Other: Visual and auditory assessments
Enrollment 33
Recruitment Details This screening study evaluated patients for disease and neurodevelopmental status to determine their potential eligibility for investigational study HGT-HIT-045 (NCT00920647), and informed decisions regarding study design, rate of enrollment, and estimated dates of completion for subsequent investigational studies in Hunter syndrome.
Pre-assignment Details  
Arm/Group Title All Patients
Hide Arm/Group Description All patients who met the inclusion criteria and consented to participate in the study.
Period Title: Overall Study
Started 33
Completed 33
Not Completed 0
Arm/Group Title All Patients
Hide Arm/Group Description All patients who met the inclusion criteria and consented to participate in the study.
Overall Number of Baseline Participants 33
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 33 participants
<=18 years
33
 100.0%
Between 18 and 65 years
0
   0.0%
>=65 years
0
   0.0%
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 33 participants
6.42  (2.841)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 33 participants
Female
0
   0.0%
Male
33
 100.0%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 33 participants
United States 21
United Kingdom 12
1.Primary Outcome
Title Number of Participants Who Were Screened For The Follow-On Study With an Investigational Agent
Hide Description

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
Hide Outcome Measure Data
Hide Analysis Population Description
All enrolled patients, defined as patients who met the inclusion criteria and consented to participate in the study.
Arm/Group Title All Patients
Hide Arm/Group Description:
All patients who met the inclusion criteria and consented to participate in the study.
Overall Number of Participants Analyzed 33
Measure Type: Number
Unit of Measure: participants
17
2.Primary Outcome
Title Number of Participants With a Score of at Least 90 on The General Conceptual Ability (GCA) Sub-Scale of The Differential Ability Scale (DAS)
Hide Description 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
Hide Outcome Measure Data
Hide Analysis Population Description
All enrolled patients, defined as patients who met the inclusion criteria and consented to participate in the study.
Arm/Group Title All Patients
Hide Arm/Group Description:
All patients who met the inclusion criteria and consented to participate in the study.
Overall Number of Participants Analyzed 33
Measure Type: Number
Unit of Measure: participants
5
Time Frame [Not Specified]
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title All Patients
Hide Arm/Group Description All patients who met the inclusion criteria and consented to participate in the study.
All-Cause Mortality
All Patients
Affected / at Risk (%)
Total   --/-- 
Show Serious Adverse Events Hide Serious Adverse Events
All Patients
Affected / at Risk (%)
Total   0/33 (0.00%) 
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 1%
All Patients
Affected / at Risk (%)
Total   3/33 (9.09%) 
Gastrointestinal disorders   
Vomiting  1  1/33 (3.03%) 
General disorders   
Pyrexia  1  1/33 (3.03%) 
Investigations   
Blood pressure systolic increased  1  1/33 (3.03%) 
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA (12.1)
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Shire’s agreements with investigators vary. All agreements provide Shire the right to embargo communications regarding trial results prior to public release for a period ≤180 days from the time submitted to Shire for review. Shire does not prohibit publication, but can require the removal of confidential information (excluding trial results) and can request postponement of a single-center publication until after disclosure of the trial’s multi-center publication.
Results Point of Contact
Name/Title: Study Physician
Organization: Shire Development LLC
Phone: +1 866 842 5335
Responsible Party: Shire
ClinicalTrials.gov Identifier: NCT00937794     History of Changes
Other Study ID Numbers: HGT-HIT-050
First Submitted: July 9, 2009
First Posted: July 13, 2009
Results First Submitted: December 18, 2015
Results First Posted: May 16, 2016
Last Update Posted: May 16, 2016