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Trial record 45 of 103 for:    Gaucher Disease

An Open-Label Extension Study of GA-GCB ERT in Patients With Type 1 Gaucher Disease

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ClinicalTrials.gov Identifier: NCT00635427
Recruitment Status : Completed
First Posted : March 13, 2008
Results First Posted : January 28, 2014
Last Update Posted : May 18, 2015
Sponsor:
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: 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
The first participant was enrolled in the study on 13 March 2008 and the last participant completed study procedures on 28 December 2012.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
2 participants who did not have type 1 Gaucher disease were withdrawn from the intent-to-treat (ITT) set as per the statistical analysis plan and removed from the long-term efficacy analyses in this study, needed to support the interpretation of the long-term efficacy results. Hence, 93 of 95 participants were included in the HGT-GCB-044 ITT set.

Reporting Groups
  Description
VPRIV 60 U/kg (Parent Study VPRIV (45 U/kg) -TKT032) VPRIV 45 U/kg, IV, every other week (EOW) for 51 weeks in parent study TKT032 (NCT00430625) and switched to 60 U/kg in HGT-GCB-044
VPRIV 60 U/kg (Parent Study- VPRIV (60 U/kg)-TKT032) VPRIV 60 U/kg, IV, EOW for 51 weeks in parent study TKT032 (NCT00430625)
VPRIV 60 U/kg (Parent Study- VPRIV (60U/kg) HGT-GCB-039) VPRIV 60 U/kg, IV, EOW for 39 weeks in parent study HGT-GCB-039 (NCT00553631)
VPRIV 60 U/kg (Parent Study-imiglucerase(60 U/kg)HGT-GCB-039) imiglucerase 60 U/kg, IV, EOW for 39 weeks in parent study HGT-GCB-039 (NCT00553631)and switched 60 U/kg VPRIV in HGT-GCB-044
VPRIV 15-60 U/kg (Parent Study VPRIV(15-60 U/kg)TKT034) VPRIV 15- 60 U/kg, IV, EOW for 51 weeks in parent study TKT034 (NCT00478647) and continued in HGT-GCB-044 at the same dose as prescribed in TKT034

Participant Flow:   Overall Study
    VPRIV 60 U/kg (Parent Study VPRIV (45 U/kg) -TKT032)   VPRIV 60 U/kg (Parent Study- VPRIV (60 U/kg)-TKT032)   VPRIV 60 U/kg (Parent Study- VPRIV (60U/kg) HGT-GCB-039)   VPRIV 60 U/kg (Parent Study-imiglucerase(60 U/kg)HGT-GCB-039)   VPRIV 15-60 U/kg (Parent Study VPRIV(15-60 U/kg)TKT034)
STARTED   13 [1]   12 [2]   16   16   38 
COMPLETED   1   6   5   7   30 
NOT COMPLETED   12   6   11   9   8 
Refusal Of Required DiagnosticEvaluation                0                0                1                0                0 
Death                0                0                0                1                0 
Termination Of Study By Sponsor                12                6                10                6                6 
Withdrawal Of Consent                0                0                0                2                2 
[1] 12 participants were considered ITT population as 1 participant did not have type 1 Gaucher disease
[2] 11 participants were considered ITT population as 1 participant did not have type 1 Gaucher disease



  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.
Two participants who did not have type 1 Gaucher disease were withdrawn from the ITT study as per SAP definition and removed from the long-term efficacy analyses in this study, needed to support the interpretation of the long-term efficacy results. Therefore, 93 of 95 (97.9%) participants were included in the HGT-GCB-044 ITT population.

Reporting Groups
  Description
VPRIV 60 U/kg (Parent Study VPRIV (45 U/kg)-TKT032) VPRIV 45 U/kg, IV, EOW for 51 weeks in parent study TKT032 (NCT00430625) and switched to 60 U/kg in HGT-GCB-044.
VPRIV 60 U/kg (Parent Study VPRIV (60 U/kg)-TKT032) VPRIV 60 U/kg, IV, EOW for 51 weeks in parent study TKT032 (NCT00430625).
VPRIV 60 U/kg (Parent Study VPRIV (60U/kg) HGT-GCB-039) VPRIV 60 U/kg, IV, EOW for 39 weeks in parent study HGT-GCB-039 (NCT00553631).
VPRIV 60 U/kg (Parent Study-imiglucerase (60 U/kg) HGT-GCB-039 Imiglucerase 60 U/kg, IV, EOW for 39 weeks in parent study HGT-GCB-039 (NCT00553631) and switched 60 U/kg VPRIV in HGT-GCB-044
VPRIV 15-60 U/kg (Parent Study VPRIV (15-60 U/kg) TKT034) VPRIV 15- 60 U/kg, IV, EOW for 51 weeks in parent study TKT034 (NCT00478647). Participants continued to receive VPRIV at a dose of 15-60 U/kg throughout participation in HGT-GCB-044
Total Total of all reporting groups

Baseline Measures
   VPRIV 60 U/kg (Parent Study VPRIV (45 U/kg)-TKT032)   VPRIV 60 U/kg (Parent Study VPRIV (60 U/kg)-TKT032)   VPRIV 60 U/kg (Parent Study VPRIV (60U/kg) HGT-GCB-039)   VPRIV 60 U/kg (Parent Study-imiglucerase (60 U/kg) HGT-GCB-039   VPRIV 15-60 U/kg (Parent Study VPRIV (15-60 U/kg) TKT034)   Total 
Overall Participants Analyzed 
[Units: Participants]
 12   11   16   16   38   93 
Age [1] 
[Units: Years]
Mean (Standard Deviation)
 32.5  (16.75)   22  (11.08)   32.9  (16.14)   25  (17.33)   34.3  (17.94)   31.4  (17.15) 
[1] Age at the time the informed consent was obtained in the core study.
Age, Customized [1] 
[Units: Participants]
           
At least 18 years   2   3   3   5   9   22 
Between 18 and 65 years   10   8   13   11   26   68 
Greater than or equal to 65 years   0   0   0   0   3   3 
[1] Age at the time the informed consent was obtained in the core study.
Gender 
[Units: Participants]
           
Female   7   6   8   7   18   46 
Male   5   5   8   9   20   47 
Splenectomy status 
[Units: Participants]
           
Yes   0   0   9   10   3   22 
No   12   11   7   6   35   71 
Baseline hemoglobin concentration per treatment group [1] 
[Units: Gram per deciliter]
Mean (Full Range)
 10.68 
 (8.5 to 12.9) 
 10.68 
 (7.1 to 12.3) 
 11.56 
 (9.7 to 14.4) 
 10.58 
 (8.1 to 13.1) 
 13.82 
 (10.7 to 16.5) 
 12.09 
 (7.1 to 16.5) 
[1] Baseline was defined as data collected prior to the first dose in the core study (TKT032, TKT034, and HGT-GCB-039).
Baseline platelet counts per treatment group [1] 
[Units: x10^9/L]
Mean (Full Range)
 69.3 
 (13 to 146) 
 79.4 
 (47 to 139) 
 160.1 
 (44 to 310) 
 186.3 
 (63 to 430) 
 165.4 
 (29 to 399) 
 144.81 
 (13 to 430) 
[1] Baseline was defined as data collected prior to the first dose in the core study (TKT032, TKT034, and HGT-GCB-039).
Baseline liver volume per treatment group [1] 
[Units: Percent (%) body weight]
Mean (Full Range)
 1.64 
 (1.1 to 2.9) 
 1.63 
 (1 to 3.2) 
 1.59 
 (0.8 to 2.2) 
 1.68 
 (0.7 to 2.8) 
 0.82 
 (0.6 to 1.3) 
 1.302 
 (0.6 to 3.2) 
[1] Baseline was defined as data collected prior to the first dose in the core study (TKT032, TKT034, and HGT-GCB-039). Normal liver volume was defined as 2.5 percent of body weight.
Baseline Spleen volume per treatment group [1] 
[Units: Multiple of Normal]
Mean (Full Range)
 23.08 
 (4.8 to 65.1) 
 18.48 
 (5.7 to 36.9) 
 12.69 
 (7.2 to 31.6) 
 23.52 
 (3.1 to 44.4) 
 4.1 
 (1.2 to 15.8) 
 13.07 
 (1.2 to 65.1) 
[1] Baseline was defined as data collected prior to the first dose in the core study (TKT032, TKT034, and HT-GCB-039). Normal spleen volume is defined as 0.2 percentage of body weight.


  Outcome Measures

1.  Primary:   Overall Summary of Treatment Emergent Adverse Events   [ Time Frame: Baseline to termination of study ]

2.  Secondary:   Change From Baseline to 24 Months in Hemoglobin Concentration for Each Treatment Group   [ Time Frame: Baseline to 24 months ]

3.  Secondary:   Change From Baseline to 24 Months in Platelet Counts for Each Treatment Group   [ Time Frame: Baseline to 24 months ]

4.  Secondary:   Change From Baseline to 24 Months in Normalized Liver Volume for Each Treatment Group   [ Time Frame: Baseline to 24 months ]

5.  Secondary:   Percentage Change From Baseline to 24 Months in Normalized Spleen Volume for Each Treatment Group   [ Time Frame: Baseline to 24 months ]


  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: MedInfo
Organization: Shire
phone: +1-866-888-0660
e-mail: US_ShireHGT_Medicalinformation@shire.com


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

Responsible Party: Shire
ClinicalTrials.gov Identifier: NCT00635427     History of Changes
Other Study ID Numbers: HGT-GCB-044
2008-001965-27 ( EudraCT Number )
First Submitted: March 6, 2008
First Posted: March 13, 2008
Results First Submitted: December 11, 2013
Results First Posted: January 28, 2014
Last Update Posted: May 18, 2015