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

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

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.
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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)

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 ]

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).