Trial · NCT00364858

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

NCT00364858, Phase 4, Completed, Genzyme, a Sanofi Company; results first posted 2009-09-02

3 sourcestrials-2026-11-12-0.5.0

Cite this record

ConductScience Library. Safety and Efficacy of Cerezyme® Infusions Every 4 Weeks Versus Every 2 Weeks in Type 1 Gaucher Disease. Trials, record NCT00364858. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00364858

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Trial

As registered on ClinicalTrials.gov, in the graph's capture of the registry.

Title: Safety and Efficacy of Cerezyme® Infusions Every 4 Weeks Versus Every 2 Weeks in Type 1 Gaucher Disease
NCT number: NCT00364858
Status: Completed
Phases: Phase 4
Enrollment: 95 participants, actual
Lead sponsor: Genzyme, a Sanofi Company
Conditions: Gaucher Disease, Type 1, Cerebroside Lipidosis Syndrome, Glucocerebrosidase Deficiency Disease, Glucosylceramide Beta-Glucosidase Deficiency Disease, Gaucher Disease, Non-Neuronopathic Form
Interventions: Cerezyme
First posted: 2006-08-16
Last updated: 2015-04-03
Results first posted: 2009-09-02

Source ClinicalTrials.gov

Registered outcomes4

Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.

List	Outcome	Time frame	Description
Primary	Number of Participants With Clinical Success at Month 24/Discontinuation	Month 24 (or at time of discontinuation)	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 pathologic fractures, medullary infarctions, lytic lesions or avascular necrosis and has had no bone crises during the study.
Secondary	Mean Composite Scores of the SF-36 Health Survey at Baseline	Baseline	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.
Secondary	Mean Composite Scores of the SF-36 Health Survey at Month 24/Discontinuation.	Month 24 (or at time of discontinuation)	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.
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)	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.

Source ClinicalTrials.gov

Outcomes4

Each outcome the trial posted results for, in ClinicalTrials.gov's words.

Type	Outcome	Time frame	Population	Units	Measured as
PRIMARY	Number of Participants With Clinical Success at Month 24/Discontinuation	Month 24 (or at time of discontinuation)	Intent-to-Treat (ITT) Population. All patients who enrolled in the study and received AT LEAST ONE infusion were included in the ITT population.	patients	NUMBER
SECONDARY	Mean Composite Scores of the SF-36 Health Survey at Baseline	Baseline	Quality of life was evaluated and measured by the SF-36 questionnaire.	Units on a scale	MEAN
SECONDARY	Mean Composite Scores of the SF-36 Health Survey at Month 24/Discontinuation.	Month 24 (or at time of discontinuation)	Quality of life was evaluated and measured by the SF-36 questionnaire.	Units on a scale	MEAN
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)	Quality of life was evaluated and measured by the SF-36 questionnaire.	Units on a scale	MEAN

Source ClinicalTrials.gov

Results by group16

Each measurement the trial posted, group by group, in the words and numbers it posted.

Outcome	Group	Category	Value	Spread	Units
Number of Participants With Clinical Success at Month 24/Discontinuation	Q2 Cerezyme	Number of participants with Clinical Success	21		patients
Number of Participants With Clinical Success at Month 24/Discontinuation	Q4 Cerezyme	Number of participants with Clinical Success	36		patients
Number of Participants With Clinical Success at Month 24/Discontinuation	Q2 Cerezyme	Proportion of participants with Clinical Success	0.808		patients
Number of Participants With Clinical Success at Month 24/Discontinuation	Q4 Cerezyme	Proportion of participants with Clinical Success	0.632		patients
Mean Composite Scores of the SF-36 Health Survey at Baseline	Q2 Cerezyme	Composite Physical Health	49.8	Standard Deviation 8.41	Units on a scale
Mean Composite Scores of the SF-36 Health Survey at Baseline	Q4 Cerezyme	Composite Physical Health	46.9	Standard Deviation 10.26	Units on a scale
Mean Composite Scores of the SF-36 Health Survey at Baseline	Q2 Cerezyme	Composite Mental Health	52.9	Standard Deviation 7.6	Units on a scale
Mean Composite Scores of the SF-36 Health Survey at Baseline	Q4 Cerezyme	Composite Mental Health	53.0	Standard Deviation 8.47	Units on a scale
Mean Composite Scores of the SF-36 Health Survey at Month 24/Discontinuation.	Q2 Cerezyme	Composite Physical Health	49.1	Standard Deviation 7.52	Units on a scale
Mean Composite Scores of the SF-36 Health Survey at Month 24/Discontinuation.	Q4 Cerezyme	Composite Physical Health	46.2	Standard Deviation 12.39	Units on a scale

1 to 10 of 16Source ClinicalTrials.gov

See all 16 results by group

Participant flow6

How many started, completed or left each group, period by period.

Period	Milestone	Group	Participants
Overall Study	STARTED	Q2 Cerezyme	33
Overall Study	STARTED	Q4 Cerezyme	62
Overall Study	COMPLETED	Q2 Cerezyme	26
Overall Study	COMPLETED	Q4 Cerezyme	40
Overall Study	NOT COMPLETED	Q2 Cerezyme	7
Overall Study	NOT COMPLETED	Q4 Cerezyme	22

Source ClinicalTrials.gov

Adverse events531

Each adverse event the trial posted, group by group: the participants affected out of those at risk.

Type	Event	Organ system	Group	Affected	At risk
other	Anaemia	Blood and lymphatic system disorders	Q2 Cerezyme	0	33
other	Anaemia	Blood and lymphatic system disorders	Q4 Cerezyme	1	62
other	Anaemia	Blood and lymphatic system disorders	Total	1	95
other	Splenic infarction	Blood and lymphatic system disorders	Q2 Cerezyme	0	33
other	Splenic infarction	Blood and lymphatic system disorders	Q4 Cerezyme	1	62
other	Splenic infarction	Blood and lymphatic system disorders	Total	1	95
other	Splenomegaly	Blood and lymphatic system disorders	Q2 Cerezyme	0	33
other	Splenomegaly	Blood and lymphatic system disorders	Q4 Cerezyme	2	62
other	Splenomegaly	Blood and lymphatic system disorders	Total	2	95
other	Thrombocytopenia	Blood and lymphatic system disorders	Q2 Cerezyme	0	33

1 to 10 of 531Source ClinicalTrials.gov

See all 531 adverse events

Adverse event totals9

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	Q2 Cerezyme
Total, all-cause mortality	Q4 Cerezyme
Total, all-cause mortality	Total
Total, other adverse events	Q2 Cerezyme	20	33
Total, other adverse events	Q4 Cerezyme	51	62
Total, other adverse events	Total	71	95
Total, serious adverse events	Q2 Cerezyme	7	33
Total, serious adverse events	Q4 Cerezyme	4	62
Total, serious adverse events	Total	11	95

Source ClinicalTrials.gov

Papers0

The papers the trial lists on ClinicalTrials.gov.

None in this release.

Grants0

The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.

None in this release.

Trial comparisons0

Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.

None in this release.

Tests0

The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.

None in this release.

Data dates

The day each source was copied for this release.

ClinicalTrials.gov: 2026-11-12; ClinicalTrials.gov
Results, from AACT: 2026-03-23; AACT
NIH grant links: 2026-11-13; NIH RePORTER

Sources 3

ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
ClinicalTrials.govNo license stated
NIH RePORTER's clinical studies and project filesPublic domain

Each source's terms

ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
ClinicalTrials.govnone stated
NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)

ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.

For software

Read this trial from software.

The record and each of its lists are JSON, twenty rows a page. No key.

GET https://conductscience.org/api/trials/v1/NCT00364858

GET https://conductscience.org/api/trials/v1/NCT00364858/events?page=2

API reference For AI assistants