Safety and Efficacy of Cerezyme® Infusions Every 4 Weeks Versus Every 2 Weeks in Type 1 Gaucher Disease

This study has been completed.

Study NCT00364858 Information provided by Genzyme

First Received: August 15, 2006 Last Updated: September 3, 2009 History of Changes

Study Type:	Interventional
Study Design:	Randomized, Open Label, Uncontrolled, Parallel Assignment
Conditions:	Gaucher Disease, Type 1 Cerebroside Lipidosis Syndrome Glucocerebrosidase Deficiency Disease Glucosylceramide Beta-Glucosidase Deficiency Disease Gaucher Disease, Non-Neuronopathic Form
Intervention:	Drug: Cerezyme

Participant Flow

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Recruitment Details

Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
Eligible patients were randomized 2:1 to receive Cerezyme either once every 4 weeks (Q4) or once every 2 weeks (Q2) for 24 months. The studied period was from 14 December 2001 through 01 February 2007. There were 26 centers worldwide (18 United States, 1 Canada, 6 Europe, and 1 Brazil); 25 centers randomized patients to treatment.

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
Q2 Cerezyme	Patients receiving Cerezyme one infusion every 2 weeks (Q2).
Q4 Cerezyme	Patients receiving Cerezyme one infusion every 4 weeks(Q4).

Participant Flow: Overall Study

	Q2 Cerezyme	Q4 Cerezyme
STARTED	33	62
COMPLETED	26	40
NOT COMPLETED	7	22
Adverse Event	1	5
Withdrawal by Subject	1	1
Pregnancy	1	1
Clinical failure	2	13
Discontinuation at baseline	1	0
Non-compliant	1	0
Return to Q2 regimen	0	1
Clinical baseline issue	0	1

Baseline Characteristics

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Reporting Groups

	Description
Q2 Cerezyme	Patients receiving Cerezyme one infusion every 2 weeks (Q2).
Q4 Cerezyme	Patients receiving Cerezyme one infusion every 4 weeks(Q4).

Baseline Measures

	Q2 Cerezyme	Q4 Cerezyme	Total
Number of Participants [units: participants]	33	62	95
Age [units: years] Mean ± Standard Deviation	44.8 ± 17.40	47.8 ± 14.47	46.8 ± 15.53
Gender [units: participants]
Female	13	34	47
Male	20	28	48

Outcome Measures

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1. Primary:

Number of Participants With Clinical Success at Month 24/Discontinuation [ Month 24 (or at time of discontinuation) ]

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2. Secondary:

Mean Composite Scores of the SF-36 Health Survey at Baseline [ Baseline ]

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3. Secondary:

Mean Composite Scores of the SF-36 Health Survey at Month 24/Discontinuation. [ Month 24 (or at time of discontinuation) ]

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4. Secondary:

Mean Change From Baseline in Composite Scores of the SF-36 Health Survey at Month 24/Discontinuation [ Baseline and Month 24 (or at time of discontinuation) ]

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