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

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

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) ]

Measure Type	Primary
Measure Title	Number of Participants With Clinical Success at Month 24/Discontinuation
Measure Description	Patients are considered to be a clinical success if ALL of the following are met: The patient’s hemoglobin does not fall more than 1.25g/dL for women or 1.5 g/dL for men below the patient’s baseline value, platelet count does not fall more than 25% below the patient’s baseline value or does not fall below 80,000 mm3, liver and spleen volumes are not greater than 20% above the patient’s baseline value, no evidence of bone disease progression, including no incidence of pathologicfractures, medullary infarctions, lytic lesions or avascular necrosis and has had no bone crises during the study.
Time Frame	Month 24 (or at time of discontinuation)
Safety Issue	No

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.
Intent-to-Treat (ITT) Population. All patients who enrolled in the study and received AT LEAST ONE infusion were included in the ITT population.

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).

Measured Values

	Q2 Cerezyme	Q4 Cerezyme
Number of Participants Analyzed [units: participants]	26	57
Number of Participants With Clinical Success at Month 24/Discontinuation [units: patients]
Number of participants with Clinical Success	21	36
Proportion of participants with Clinical Success	0.808	0.632

Statistical Analysis 1 for Number of Participants With Clinical Success at Month 24/Discontinuation

Groups ^[1]	All groups
Agresti and Min ^[2]	-0.176
95% Confidence Interval	( -0.357 to 0.058 )

[1]	Additional details about the analysis, such as null hypothesis and power calculation:
	Difference in proportion of Clinical Success = (Proportion of participants with Clinical Success Q4 - Proportion of participants with Clinical Success Q2).
[2]	Other relevant estimation information:
	No text entered.

2. Secondary:

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

Measure Type	Secondary
Measure Title	Mean Composite Scores of the SF-36 Health Survey at Baseline
Measure Description	The mean composite scores (0 being worst and 100 being best) for both treatment groups at Baseline. Composite scores for both treatment groups approximated those of the general population at baseline and at Month 24.
Time Frame	Baseline
Safety Issue	No

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.
Quality of life was evaluated and measured by the SF-36 questionnaire.

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).

Measured Values

	Q2 Cerezyme	Q4 Cerezyme
Number of Participants Analyzed [units: participants]	33	61
Mean Composite Scores of the SF-36 Health Survey at Baseline [units: Units on a scale] Mean ± Standard Deviation
Composite Physical Health	49.8 ± 8.41	46.9 ± 10.26
Composite Mental Health	52.9 ± 7.6	53.0 ± 8.47

No statistical analysis provided for Mean Composite Scores of the SF-36 Health Survey at Baseline

3. Secondary:

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

Measure Type	Secondary
Measure Title	Mean Composite Scores of the SF-36 Health Survey at Month 24/Discontinuation.
Measure Description	The mean composite scores (0 being worst and 100 being best) for both treatment groups at Month 24/Discontinuation. The mean composite scores for both treatment groups approximated those of the general population at baseline and at Month 24.
Time Frame	Month 24 (or at time of discontinuation)
Safety Issue	No

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.
Quality of life was evaluated and measured by the SF-36 questionnaire.

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).

Measured Values

	Q2 Cerezyme	Q4 Cerezyme
Number of Participants Analyzed [units: participants]	25	53
Mean Composite Scores of the SF-36 Health Survey at Month 24/Discontinuation. [units: Units on a scale] Mean ± Standard Deviation
Composite Physical Health	49.1 ± 7.52	46.2 ± 12.39
Composite Mental Health	53.8 ± 8.47	52.6 ± 7.35

No statistical analysis provided for Mean Composite Scores of the SF-36 Health Survey at Month 24/Discontinuation.

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) ]

Measure Type	Secondary
Measure Title	Mean Change From Baseline in Composite Scores of the SF-36 Health Survey at Month 24/Discontinuation
Measure Description	The mean composite scores (0 being worst and 100 being best) for both treatment groups approximated those of the general population at baseline. Composite score - The overall composite scores were comprised of a standardized physical and mental component score.
Time Frame	Baseline and Month 24 (or at time of discontinuation)
Safety Issue	No

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.
Quality of life was evaluated and measured by the SF-36 questionnaire.

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).

Measured Values

	Q2 Cerezyme	Q4 Cerezyme
Number of Participants Analyzed [units: participants]	25	52
Mean Change From Baseline in Composite Scores of the SF-36 Health Survey at Month 24/Discontinuation [units: Units on a scale] Mean ± Standard Deviation
Composite Physical Health	-1.5 ± 7.40	-0.5 ± 7.33
Composite Mental Health	0.6 ± 6.48	-0.5 ± 7.34

No statistical analysis provided for Mean Change From Baseline in Composite Scores of the SF-36 Health Survey at Month 24/Discontinuation

Serious Adverse Events

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Other Adverse Events

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More Information

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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:

	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.
	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.
	Other disclosure agreement that restricts the right of the PI to discuss or publish trial results after the trial is completed. Restriction Description: In multi-site studies, PI can publish after Genzyme publishes or 18 months after study completion. PI gives Genzyme a draft 60 days before publication. Genzyme can ask that confidential information be removed, and can defer publication another 60 days upon notifying PI that it will file a patent application on inventions contained in the draft.

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
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Results Point of Contact:

Name/Title: Genzyme Medical Information
Organization: Genzyme Corporation
phone: 800-745-4447

No publications provided

Responsible Party:	Genzyme Coporation ( Medical Monitor )
Study ID Numbers:	CZ-011-01
Study First Received:	August 15, 2006
Results First Received:	May 28, 2009
Last Updated:	September 3, 2009
ClinicalTrials.gov Identifier:	NCT00364858 History of Changes
Health Authority:	United States: Food and Drug Administration