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Trial record 17 of 102 for:    Gaucher Disease

A Multicenter Extension Study of Taliglucerase Alfa in Pediatric Subjects With Gaucher Disease

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: NCT01411228
Recruitment Status : Completed
First Posted : August 8, 2011
Results First Posted : January 18, 2016
Last Update Posted : July 21, 2016
Sponsor:
Information provided by (Responsible Party):
Protalix

Study Type: Interventional
Study Design: Allocation: Non-Randomized;   Intervention Model: Parallel Assignment;   Masking: Triple (Participant, Care Provider, Investigator);   Primary Purpose: Treatment
Condition: Gaucher Disease
Intervention: Drug: Taliglucerase alfa

  Participant Flow

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
No text entered.

Reporting Groups
  Description
60 Units/kg Taliglucerase alfa: Taliglucerase alfa for infusion every two weeks for 24 months
30 Units/kg Taliglucerase alfa: Taliglucerase alfa for infusion every two weeks for 24 months
Switchover Taliglucerase alfa: Taliglucerase alfa for infusion every two weeks for 24 months

Participant Flow:   Overall Study
    60 Units/kg   30 Units/kg   Switchover
STARTED   5   5   5 
COMPLETED   4   5   4 
NOT COMPLETED   1   0   1 
Lost to Follow-up                0                0                1 
Withdrawal by Subject                1                0                0 



  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
60 Units/kg Taliglucerase alfa: Taliglucerase alfa for infusion every two weeks for 24 months
30 Units/kg Taliglucerase alfa: Taliglucerase alfa for infusion every two weeks for 24 months
Dose Adjusted Taliglucerase alfa: Dose increased from 30 Units/kg to 45 or 60 Units/kg
Total Total of all reporting groups

Baseline Measures
   60 Units/kg   30 Units/kg   Dose Adjusted   Total 
Overall Participants Analyzed 
[Units: Participants]
 5   5   5   15 
Age 
[Units: Years]
Mean (Standard Deviation)
 6.6  (3.1)   8.6  (3.8)   13.0  (4.1)   9.4  (4.4) 
Gender 
[Units: Participants]
       
Female   1   2   2   5 
Male   4   3   3   10 
Race (NIH/OMB) 
[Units: Participants]
       
American Indian or Alaska Native   0   0   0   0 
Asian   0   0   0   0 
Native Hawaiian or Other Pacific Islander   0   0   1   1 
Black or African American   0   0   0   0 
White   4   5   4   13 
More than one race   0   0   0   0 
Unknown or Not Reported   1   0   0   1 
Region of Enrollment 
[Units: Participants]
       
South Africa   2   1   0   3 
Israel   2   1   0   3 
Spain   0   0   2   2 
Serbia   0   0   2   2 
Paraguay   1   3   0   4 
Singapore   0   0   1   1 


  Outcome Measures

1.  Primary:   Hemoglobin   [ Time Frame: Baseline, months 9, 12 and 24 ]

2.  Secondary:   Chitotriosidase   [ Time Frame: Baseline, months 9, 12 and 24 ]

3.  Secondary:   Spleen Volume   [ Time Frame: Baseline, months 12 and 24 ]

4.  Secondary:   Platelet Count   [ Time Frame: Baseline, months 9, 12, 24 and 33-36 ]

5.  Secondary:   Liver Volume   [ Time Frame: Baseline, months 12 and 24 ]


  Serious Adverse Events


  Other Adverse Events


  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

Certain Agreements:  
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT 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.


Results Point of Contact:  
Name/Title: Glen Park
Organization: Target Health Inc.
phone: 212-681-2100
e-mail: gpark@targethealth.com


Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

Responsible Party: Protalix
ClinicalTrials.gov Identifier: NCT01411228     History of Changes
Other Study ID Numbers: PB-06-006
First Submitted: August 4, 2011
First Posted: August 8, 2011
Results First Submitted: November 6, 2015
Results First Posted: January 18, 2016
Last Update Posted: July 21, 2016