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

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

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)

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 ]

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.