Study of Velaglucerase Alfa Enzyme Replacement Therapy in Japanese Patients With Gaucher Disease

Recruitment Details

Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
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Pre-Assignment Details

Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
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Reporting Groups

	Description
VPRIV® (15-60 U/kg)	No text entered.

Participant Flow:   Overall Study

	VPRIV® (15-60 U/kg)
STARTED	6
COMPLETED	6
NOT COMPLETED	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® (15-60 U/kg)	No text entered.

Baseline Measures

	VPRIV® (15-60 U/kg)
Overall Participants Analyzed  [Units: Participants]	6
Age  [Units: Years] Mean (Standard Deviation)	20  (10.62)
Age, Customized  [Units: Participants]
<=18 years	4
Between 18 and 65 years	2
Gender  [Units: Participants]
Female	1
Male	5
Region of Enrollment  [Units: Participants]
JAPAN	6
Hemoglobin Concentration  [Units: (g/dL)] Mean (Standard Deviation)	13.77  (0.954)
Platelet Count  [Units: (x 10`{super 9}/L)] Mean (Standard Deviation)	182.9  (32.05)
Normalized liver volume  [Units: (% of Body Weight)] Mean (Standard Deviation)	1.92  (0.342)
Normalized spleen volume  [Units: (% of Body Weight)] Mean (Standard Deviation)	0.39  (0.067)
Plasma chitotriosidase  [Units: (nmol/mL/h)] Mean (Standard Deviation)	1243.7  (344.46)
CCL18 levels  [Units: (ng/mL)] Mean (Standard Deviation)	134.8  (132.26)

1.  Primary:

Number of Severe Adverse Events (SAE)   [ Time Frame: Baseline to week 51 ]

2.  Primary:

Number of Treatment Emergent Adverse Events (TEAE)   [ Time Frame: Baseline to week 51 ]

3.  Primary:

Development of Anti-velaglucerase Alfa Antibody   [ Time Frame: Baseline to week51 ]

4.  Primary:

Number of Infusion- Related Adverse Events   [ Time Frame: Baseline to week 51 ]

5.  Primary:

Number of Patients With Concomitant Medication   [ Time Frame: Baseline to week 51 ]

6.  Secondary:

Change From Baseline in Hemoglobin Concentration   [ Time Frame: Baseline to week 51 ]

7.  Secondary:

Change From Baseline in Platelet Count   [ Time Frame: Baseline to week 51 ]

8.  Secondary:

Change From Baseline in Liver Volume, Normalized to Body Weight   [ Time Frame: Baseline to week 51 ]

9.  Secondary:

Change From Baseline in Spleen Volume, Normalized to Body Weight   [ Time Frame: Baseline to week 51 ]

10.  Secondary:

Change From Baseline in Plasma Chitotriosidase Levels   [ Time Frame: Baseline to week 51 ]

11.  Secondary:

Change From Baseline in CCL18 Levels   [ Time Frame: Baseline to 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
All 6 enrolled patients received previous long-term treatment with the enzyme replacement therapy (ERT), imiglucerase. Key therapeutic parameters were expected to indicate stability after switching from imiglucerase.