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Trial record 19 of 31 for:    "Gaucher disease type 1"

Study of GA-GCB Enzyme Replacement Therapy in Type 1 Gaucher Disease Patients Previously Treated With Imiglucerase

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ClinicalTrials.gov Identifier: NCT00478647
Recruitment Status : Completed
First Posted : May 25, 2007
Results First Posted : September 2, 2010
Last Update Posted : April 9, 2015
Sponsor:
Collaborator:
Shire Human Genetic Therapies, Inc.
Information provided by (Responsible Party):
Shire

Study Type: Interventional
Study Design: Allocation: Non-Randomized;   Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition: Gaucher Disease, Type 1
Intervention: Biological: GA-GCB (velaglucerase 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
The first participant was enrolled on 25 July 2007 and the last participant completed on 26 June 2009. Participants received the same dose of velaglucerase alfa (GA-GCB) as their previous dose of imiglucerase (range- </= 60 Unit per kilogram (U/kg) - >/=15 U/kg) every other week via intravenous infusion.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
Participant at least 2 years old with documented diagnosis of type 1 Gaucher disease.Consistent treatment(every other week at a dose ≤/= 60 U/kg and ≥/= 15 U/kg) with imiglucerase for a minimum of 30 consecutive months;same dose during the 6 months prior to study enrollment.Minor dosing interval variance was allowed per standard clinical practice.

Reporting Groups
  Description
GA-GCB (Velaglucerase Alfa) 15-60 U/kg, every other week via intravenous infusion

Participant Flow:   Overall Study
    GA-GCB (Velaglucerase Alfa)
STARTED   40 
COMPLETED   38 [1] 
NOT COMPLETED   2 
Adverse Event                1 
Withdrawal by Subject                1 
[1] Intent-to-Treat (ITT)=participants who have received at least 1 full or partial dose of study drug.



  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
GA-GCB (Velaglucerase Alfa) 15-60 U/kg, every other week via intravenous infusion

Baseline Measures
   GA-GCB (Velaglucerase Alfa) 
Overall Participants Analyzed 
[Units: Participants]
 40 
Age 
[Units: Participants]
 
<=18 years   9 
Between 18 and 65 years   27 
>=65 years   4 
Age 
[Units: Years]
Mean (Standard Deviation)
 35.6  (18.37) 
Gender 
[Units: Participants]
 
Female   22 
Male   18 
Region of Enrollment 
[Units: Participants]
 
United States   22 
Spain   1 
Poland   5 
Israel   9 
United Kingdom   3 
Baseline hemoglobin concentration 
[Units: Gram per deciliter (g/dL)]
Median (Full Range)
 13.775 
 (10.40 to 16.45) 
Baseline liver volume 
[Units: Percent (%) body weight]
Median (Full Range)
 1.90 
 (1.4 to 3.9) 
Baseline platelet count 
[Units: 10^9 per liter (10^9/L)]
Median (Full Range)
 162.00 
 (29.0 to 399.0) 
Baseline spleen volume 
[Units: Percent (%) body weight]
Median (Full Range)
 0.50 
 (0.2 to 3.2) 


  Outcome Measures

1.  Primary:   Participants Who Experienced at Least One Adverse Event   [ Time Frame: Week 53 ]

2.  Secondary:   Change From Baseline to Week 53 in Hemoglobin Concentration   [ Time Frame: Week 53 ]

3.  Secondary:   Percent Change From Baseline to Week 53 in Platelet Count   [ Time Frame: Week 53 ]

4.  Secondary:   Percent Change From Baseline to Week 51 in Normalized Liver Volume   [ Time Frame: Week 51 ]

5.  Secondary:   Percent Change From Baseline to Week 51 in Normalized Spleen Volume   [ Time Frame: Week 51 ]


  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 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 HGT
phone: 484-595-8850
e-mail: tcrump@shire.com



Responsible Party: Shire
ClinicalTrials.gov Identifier: NCT00478647     History of Changes
Other Study ID Numbers: TKT034
2006-006304-11 ( EudraCT Number )
First Submitted: May 23, 2007
First Posted: May 25, 2007
Results First Submitted: August 4, 2010
Results First Posted: September 2, 2010
Last Update Posted: April 9, 2015