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
Results First Received: December 18, 2015  
Study Type: Observational
Study Design: Observational Model: Cohort;   Time Perspective: Prospective
Condition: Hunter Syndrome
Interventions: Behavioral: Neurobehavioral testing
Other: Visual and auditory assessments

  Participant Flow
  Hide Participant Flow

Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
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
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
No text entered.

Reporting Groups
  Description
All Patients All patients who met the inclusion criteria and consented to participate in the study.

Participant Flow:   Overall Study
    All Patients  
STARTED     33  
COMPLETED     33  
NOT COMPLETED     0  



  Baseline Characteristics
  Hide Baseline Characteristics

Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
No text entered.

Reporting Groups
  Description
All Patients All patients who met the inclusion criteria and consented to participate in the study.

Baseline Measures
    All Patients  
Number of Participants  
[units: participants]
  33  
Age  
[units: participants]
 
<=18 years     33  
Between 18 and 65 years     0  
>=65 years     0  
Age  
[units: years]
Mean (Standard Deviation)
  6.42  (2.841)  
Gender  
[units: participants]
 
Female     0  
Male     33  
Region of Enrollment  
[units: participants]
 
United States     21  
United Kingdom     12  



  Outcome Measures
  Show All Outcome Measures

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

2.  Primary:   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 ]


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats
  Hide Limitations and Caveats

Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
No text entered.


  More Information
  Hide More Information

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.
The agreement is:
unchecked The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is less than or equal to 60 days. The sponsor cannot require changes to the communication and cannot extend the embargo.
unchecked The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is more than 60 days but less than or equal to 180 days. The sponsor cannot require changes to the communication and cannot extend the embargo.


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
Study First Received: July 9, 2009
Results First Received: December 18, 2015
Last Updated: April 11, 2016
Health Authority: United States: Food and Drug Administration