Trial · NCT00376168

A Phase III Trial to Assess the Safety and Efficacy of Plant Cell Expressed GCD in Patients With Gaucher Disease

NCT00376168, Phase 3, Completed, Pfizer; results first posted 2012-07-26

3 sourcestrials-2026-11-12-0.5.0

Cite this record

ConductScience Library. A Phase III Trial to Assess the Safety and Efficacy of Plant Cell Expressed GCD in Patients With Gaucher Disease. Trials, record NCT00376168. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00376168

On this page

Trial

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

Title: A Phase III Trial to Assess the Safety and Efficacy of Plant Cell Expressed GCD in Patients With Gaucher Disease
NCT number: NCT00376168
Status: Completed
Phases: Phase 3
Enrollment: 32 participants, actual
Lead sponsor: Pfizer
Conditions: Gaucher Disease
Interventions: Plant cell expressed recombinant glucocerebrosidase (prGCD), Plant cell expressed recombinant glucocerebrosidase (prGCD)
First posted: 2006-09-14
Last updated: 2018-10-04
Results first posted: 2012-07-26

Source ClinicalTrials.gov

Registered outcomes5

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

List	Outcome	Time frame	Description
Primary	Change From Baseline in Spleen Volume Measured by MRI.	Baseline and 9 months	Calculated as percent change in spleen volume from Baseline to 9 months
Secondary	Change From Baseline in Liver Volume	Baseline and 9 months	Calculated as percent change in liver volume from Baseline to 9 months
Secondary	Change in Hemoglobin	Baseline and Month 9	Absolute change in Hemoglobin concentration from Baseline to Month 9
Secondary	Change in Platelet Count	Baseline and Month 9	Change in Platelet count from Baseline to Month 9
Other	Change in Chitotriosidase	Baseline and Month 9	Change in Chitotriosidase from Baseline to Month 9

Source ClinicalTrials.gov

Outcomes5

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

Type	Outcome	Time frame	Population	Units	Measured as
PRIMARY	Change From Baseline in Spleen Volume Measured by MRI.	Baseline and 9 months	Intent to treat	percentage of change	MEAN
SECONDARY	Change From Baseline in Liver Volume	Baseline and 9 months	Intent to treat	percentage of change from baseline	MEAN
SECONDARY	Change in Hemoglobin	Baseline and Month 9	Intent to treat	g/dL	MEAN
SECONDARY	Change in Platelet Count	Baseline and Month 9	Intent to treat	count/mm^3	MEAN
OTHER_PRE_SPECIFIED	Change in Chitotriosidase	Baseline and Month 9	Intent to treat	nmol/ml/hr	MEAN

Source ClinicalTrials.gov

Results by group10

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

Outcome	Group	Value	Spread	Units
Change From Baseline in Spleen Volume Measured by MRI.	ELELYSO 30 Units/kg	-26.91	Standard Deviation 7.79	percentage of change
Change From Baseline in Spleen Volume Measured by MRI.	ELELYSO 60 Units/kg	-38.01	Standard Deviation 9.38	percentage of change
Change From Baseline in Liver Volume	ELELYSO 30 Units/kg	-10.48	Standard Deviation 11.27	percentage of change from baseline
Change From Baseline in Liver Volume	ELELYSO 60 Units/kg	-11.11	Standard Deviation 6.68	percentage of change from baseline
Change in Hemoglobin	ELELYSO 30 Units/kg	1.6	Standard Deviation 1.4	g/dL
Change in Hemoglobin	ELELYSO 60 Units/kg	2.2	Standard Deviation 1.4	g/dL
Change in Platelet Count	ELELYSO 30 Units/kg	11,427	Standard Deviation 20,214	count/mm^3
Change in Platelet Count	ELELYSO 60 Units/kg	41,494	Standard Deviation 47,063	count/mm^3
Change in Chitotriosidase	ELELYSO 30 Units/kg	-14,548	Standard Deviation 8,025.8	nmol/ml/hr
Change in Chitotriosidase	ELELYSO 60 Units/kg	-12,538	Standard Deviation 14,489	nmol/ml/hr

Source ClinicalTrials.gov

Participant flow6

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

Period	Milestone	Group	Participants
Overall Study	STARTED	ELELYSO 30 Units/kg	16
Overall Study	STARTED	ELELYSO 60 Units/kg	16
Overall Study	COMPLETED	ELELYSO 30 Units/kg	14
Overall Study	COMPLETED	ELELYSO 60 Units/kg	15
Overall Study	NOT COMPLETED	ELELYSO 30 Units/kg	2
Overall Study	NOT COMPLETED	ELELYSO 60 Units/kg	1

Source ClinicalTrials.gov

Adverse events16

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	Abdominal pain	Gastrointestinal disorders	ELELYSO 30 Units/kg	1	16
other	Abdominal pain	Gastrointestinal disorders	ELELYSO 60 Units/kg	0	16
other	Feeling hot	General disorders	ELELYSO 30 Units/kg	1	16
other	Feeling hot	General disorders	ELELYSO 60 Units/kg	0	16
other	Hypersensitivity	Immune system disorders	ELELYSO 30 Units/kg	1	16
other	Hypersensitivity	Immune system disorders	ELELYSO 60 Units/kg	1	16
other	Arthralgia	Musculoskeletal and connective tissue disorders	ELELYSO 30 Units/kg	0	16
other	Arthralgia	Musculoskeletal and connective tissue disorders	ELELYSO 60 Units/kg	1	16
other	Muscle spasms	Musculoskeletal and connective tissue disorders	ELELYSO 30 Units/kg	1	16
other	Muscle spasms	Musculoskeletal and connective tissue disorders	ELELYSO 60 Units/kg	0	16

1 to 10 of 16Source ClinicalTrials.gov

See all 16 adverse events

Adverse event totals6

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	ELELYSO 30 Units/kg
Total, all-cause mortality	ELELYSO 60 Units/kg
Total, other adverse events	ELELYSO 30 Units/kg	6	16
Total, other adverse events	ELELYSO 60 Units/kg	4	16
Total, serious adverse events	ELELYSO 30 Units/kg	0	16
Total, serious adverse events	ELELYSO 60 Units/kg	0	16

Source ClinicalTrials.gov

Papers3

The papers the trial lists on ClinicalTrials.gov.

PMID	Listed as	Source
18729283	DERIVED	PubMed
21900191	DERIVED	PubMed
26053270	DERIVED	PubMed

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 comparisons1

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

Comparison
ELELYSO 30 Units/kg vs ELELYSO 60 Units/kg: serious adverse events

Source Trial comparisons

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/NCT00376168

GET https://conductscience.org/api/trials/v1/NCT00376168/registered

API reference For AI assistants