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Efficacy and Safety Study of Velaglucerase Alfa in Children and Adolescents With Type 3 Gaucher Disease

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Shire
ClinicalTrials.gov Identifier:
NCT01685216
First received: September 10, 2012
Last updated: October 1, 2015
Last verified: October 2015
History of Changes
Results First Received: October 1, 2015  
Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment;   Masking: Open Label;   Primary Purpose: Treatment
Condition: Gaucher Disease, Type 3
Intervention: Biological: velaglucerase alfa

  Participant Flow
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Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
No text entered.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
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Reporting Groups
  Description
Velaglucerase Alfa Participants received an intravenous (IV) infusion of velaglucerase alfa at 60 U/kg, every other week for 1 year, then were followed for 1 month.

Participant Flow:   Overall Study
    Velaglucerase Alfa
STARTED   7 
COMPLETED   6 
NOT COMPLETED   1 
Did not receive study drug                1 



  Baseline Characteristics
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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.
The Intent-to-Treat population, defined as all participants who received at least 1 study drug infusion (full or partial).

Reporting Groups
  Description
Velaglucerase Alfa Participants received an intravenous (IV) infusion of velaglucerase alfa at 60 U/kg, every other week for 1 year, then were followed for 1 month.

Baseline Measures
   Velaglucerase Alfa 
Overall Participants Analyzed 
[Units: Participants]
 		 6 
Age 
[Units: Years]
Mean (Standard Deviation) 		 5.17  (4.446) 
Age, Customized 
[Units: Participants]
 		 
2 to 4 years   4 
5 to 17 years   2 
Gender 
[Units: Participants]
 		 
Female   1 
Male   5 


  Outcome Measures
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1.  Primary:   Change From Baseline to 12 Months (Week 53) in Hemoglobin Concentration   [ Time Frame: Baseline, Week 53 or end of study ]
  Show Outcome Measure 1

2.  Secondary:   Change From Baseline to 12 Months (Week 53) in Platelet Count   [ Time Frame: Baseline, Week 53 ]
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3.  Secondary:   Percent Change From Baseline to 12 Months (Week 51) in Normalized Liver Volume Measured Using Magnetic Resonance Imaging (MRI)   [ Time Frame: Baseline, Week 51 or end of study ]
  Show Outcome Measure 3

4.  Secondary:   Percent Change From Baseline to 12 Months (Week 51) in Normalized Spleen Volume Measured Using Magnetic Resonance Imaging (MRI)   [ Time Frame: Baseline, Week 51 ]
  Show Outcome Measure 4

5.  Secondary:   Number of Participants With Abnormal Neurological Status During The Study   [ Time Frame: Baseline, Weeks 13, 25, 37, and 53 or end of study ]
  Show Outcome Measure 5

6.  Secondary:   Number of Participants Who Experienced a Treatment-Emergent Adverse Event   [ Time Frame: 57 weeks ]
  Show Outcome Measure 6

7.  Secondary:   Number of Participants Who Developed Anti-Velaglucerase Alfa Antibodies During The Study   [ Time Frame: Baseline, Weeks 13, 25, 37 and 53 ]
  Show Outcome Measure 7


  Serious Adverse Events
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  Other Adverse Events
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  Limitations and Caveats
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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
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  More Information
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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: NCT01685216     History of Changes
Other Study ID Numbers: HGT-GCB-068
Study First Received: September 10, 2012
Results First Received: October 1, 2015
Last Updated: October 1, 2015