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Trial record 48 of 102 for:    Gaucher Disease

A Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy in Gaucher Disease

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ClinicalTrials.gov Identifier: NCT00430625
Recruitment Status : Completed
First Posted : February 2, 2007
Results First Posted : September 10, 2010
Last Update Posted : March 17, 2014
Sponsor:
Information provided by (Responsible Party):
Shire

Study Type: Interventional
Study Design: Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Double (Participant, Investigator);   Primary Purpose: Treatment
Condition: Gaucher Disease, Type 1
Intervention: Biological: VPRIV ®,

  Participant Flow

Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
Type 1 Gaucher disease patients (pts) >2 years. The first patient (pt) consented to participate in the study on 11 January 2007 and the last patient enrolled on 04 April 2008.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
Gaucher disease-related anemia and at least 1 of the following: moderate splenomegaly, thrombocytopenia or palpable enlarged liver. Patients were not to have received any treatment for Gaucher disease within 30 months of study entry. Patients randomized to receive VPRIV®(45 or 60 U/kg)every other week by intravenous (IV) infusion.

Reporting Groups
  Description
VPRIV® (45 U/kg, IV, Every Other Week) velaglucerase alfa, Gene Activated® human glucocerebrosidase, GA-GCB
VPRIV® (60 U/kg, IV, Every Other Week) velaglucerase alfa, Gene Activated® human glucocerebrosidase, GA-GCB

Participant Flow:   Overall Study
    VPRIV® (45 U/kg, IV, Every Other Week)   VPRIV® (60 U/kg, IV, Every Other Week)
STARTED   13   12 
COMPLETED   13   12 
NOT COMPLETED   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
VPRIV® (45 U/kg, IV, Every Other Week) velaglucerase alfa, Gene Activated® human glucocerebrosidase, GA-GCB
VPRIV® (60 U/kg, IV, Every Other Week) velaglucerase alfa, Gene Activated® human glucocerebrosidase, GA-GCB
Total Total of all reporting groups

Baseline Measures
   VPRIV® (45 U/kg, IV, Every Other Week)   VPRIV® (60 U/kg, IV, Every Other Week)   Total 
Overall Participants Analyzed 
[Units: Participants]
 13   12   25 
Age 
[Units: Participants]
     
<=18 years   3   4   7 
Between 18 and 65 years   10   8   18 
>=65 years   0   0   0 
Age 
[Units: Years]
Median (Full Range)
 30.0 
 (6 to 62) 
 23.5 
 (4 to 42) 
 25 
 (4 to 62) 
Gender 
[Units: Participants]
     
Female   5   5   10 
Male   8   7   15 
Region of Enrollment 
[Units: Participants]
     
Paraguay   6   5   11 
Argentina   1   0   1 
Russian Federation   3   0   3 
Israel   1   6   7 
Tunisia   2   1   3 
Baseline Spleen volume [1] 
[Units: Percent of body weight]
Median (Full Range)
 2.9 
 (0.5 to 13.0) 
 2.8 
 (0.4 to 7.4) 
 2.9 
 (0.4 to 13.0) 
[1] Normal spleen volume is defined as 0.2 percentage of body weight.
Baseline hemoglobin concentration per treatment group [1] 
[Units: g/dL]
Median (Full Range)
 10.90 
 (8.45 to 12.85) 
 10.83 
 (7.05 to 12.25) 
 10.85 
 (7.05 to 12.85) 
[1] Modified baseline used for analysis (defined as average of screening and baseline values)
Baseline liver volume [1] 
[Units: Percent of body weight]
Median (Full Range)
 3.50 
 (2.3 to 7.2) 
 3.65 
 (2.4 to 8.0) 
 3.5 
 (2.3 to 8.0) 
[1] Normal liver volume is defined as 2.5 percent of body weight.
Baseline platelet counts per treatment group [1] 
[Units: (x10^9/L)]
Median (Full Range)
 58.00 
 (13 to 264) 
 66.75 
 (47 to 438) 
 65.5 
 (13 to 438) 
[1] Modified baseline used for analysis (defined as average of screening and baseline values)


  Outcome Measures

1.  Primary:   Change From Baseline to 12 Months in Hemoglobin Concentration for the 60 U/kg Treatment Group.   [ Time Frame: Week 53 ]

2.  Secondary:   Change From Baseline to 12 Months in Hemoglobin Concentration in 45 U/kg Treatment Group   [ Time Frame: Week 53 ]

3.  Secondary:   Change From Baseline to 12 Months in Platelet Counts for Each Treatment Group.   [ Time Frame: Week 53 ]

4.  Secondary:   Change From Baseline to 12 Months in Normalized Liver Volume (Percent Body Weight) for Each Treatment Group (Measured by Magnetic Resonance Imaging (MRI)   [ Time Frame: Week 51 ]

5.  Secondary:   Change From Baseline to 12 Months in Normalized Spleen Volume (Percent Body Weight) for Each Treatment Group (Measured by Magnetic Resonance Imaging (MRI))   [ Time Frame: Week 51 ]

6.  Secondary:   Percent Change From Baseline to 12 Months in Plasma Chitotriosidase for Each Treatment Group   [ Time Frame: Week 53 ]

7.  Secondary:   Percent Change From Baseline to 12 Months in Chemokine (C-C Motif) Ligand 18 (CCL18)   [ Time Frame: Week 53 ]


  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
Due to small sample sizes no conclusions could be drawn on Quality of life (QOL).


  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: Tiffany Crump
Organization: Shire Human Genetic Therapies (Shire HGT)
phone: 484-595-8850
e-mail: tcrump@shire.com


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

Responsible Party: Shire
ClinicalTrials.gov Identifier: NCT00430625     History of Changes
Other Study ID Numbers: TKT032
First Submitted: February 1, 2007
First Posted: February 2, 2007
Results First Submitted: June 3, 2010
Results First Posted: September 10, 2010
Last Update Posted: March 17, 2014