Trial · NCT00891202
A Study of Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease (ENGAGE)
NCT00891202, Phase 3, Completed, Genzyme, a Sanofi Company; results first posted 2014-09-03
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. A Study of Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease (ENGAGE). Trials, record NCT00891202. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00891202
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Study of Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease (ENGAGE)
- NCT number
- NCT00891202
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 40 participants, actual
- Lead sponsor
- Genzyme, a Sanofi Company
- Conditions
- Gaucher Disease, Type 1
- Interventions
- Eliglustat tartrate, Placebo
- First posted
- 2009-05-01
- Last updated
- 2017-03-03
- Other registries' IDs
- eudract:2008-005222-37
- Results first posted
- 2014-09-03
Source ClinicalTrials.gov
Registered outcomes9
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | PAP: Percent Change From Baseline in Spleen Volume (in Multiples of Normal [MN]) at Week 39 of the Primary Analysis Period With Eliglustat Tartrate Treatment as Compared to Placebo | PAP Baseline (Day 1), Week 39 | Percent change in spleen volume = (\[spleen volume at Week 39 minus spleen volume at baseline\] divided by \[spleen volume at baseline\]) multiplied by 100, where all volumes are in MN. |
| Secondary | PAP: Hemoglobin Level | PAP Baseline (Day 1) | |
| Secondary | PAP: Absolute Change From Baseline in Hemoglobin Level at Week 39 | PAP Baseline (Day 1), Week 39 | Absolute change = hemoglobin level at Week 39 minus hemoglobin level at baseline. |
| Secondary | PAP: Percent Change From Baseline in Liver Volume (in MN) at Week 39 | PAP Baseline (Day 1), Week 39 | Percent change in liver volume = (\[liver volume at Week 39 minus liver volume at baseline\] divided by \[liver volume at baseline\]) multiplied by 100, where all volumes are in MN. |
| Secondary | PAP: Percent Change From Baseline in Platelet Counts at Week 39 | PAP Baseline (Day 1), Week 39 | Percent change in platelet count = (\[platelet count at Week 39 minus platelet count at baseline\] divided by \[platelet count at baseline\]) multiplied by 100. |
| Secondary | LTTP: Percent Change From Baseline in Spleen Volume (in MN) at Week 234 | PAP Baseline for Eliglustat (Originally on Eliglustat) arm, LTTP Baseline for Eliglustat (Originally on Placebo) arm, Week 234 | Percent change in spleen volume = (\[spleen volume at Week 234 minus spleen volume at baseline\] divided by \[spleen volume at baseline\]) multiplied by 100, where all volumes are in MN. Baseline values for the original placebo participants refer to Day 1 of LTTP and baseline values for the original eliglustat participants refer to the Day 1 of PAP. |
| Secondary | LTTP: Absolute Change From Baseline in Hemoglobin Level at Week 234 | PAP Baseline for Eliglustat (Originally on Eliglustat) arm, LTTP Baseline for Eliglustat (Originally on Placebo) arm, Week 234 | Baseline values for the original placebo participants refer to Day 1 of LTTP and baseline values for the original eliglustat participants refer to the Day 1 of PAP. |
| Secondary | LTTP: Percent Change From Baseline in Liver Volume (in MN) at Week 234 | PAP Baseline for Eliglustat (Originally on Eliglustat) arm, LTTP Baseline for Eliglustat (Originally on Placebo) arm, Week 234 | Percent change in liver volume = (\[liver volume at Week 234 minus liver volume at baseline\] divided by \[liver volume at baseline\]) multiplied by 100, where all volumes are in MN. Baseline values for the original placebo participants refer to Day 1 of LTTP and baseline values for the original eliglustat participants refer to the Day 1 of PAP. |
| Secondary | LTTP: Percent Change From Baseline in Platelet Counts at Week 234 | PAP Baseline for Eliglustat (Originally on Eliglustat) arm, LTTP Baseline for Eliglustat (Originally on Placebo) arm, Week 234 | Percent change in platelet count = (\[platelet count at Week 234 minus platelet count at baseline\] divided by \[platelet count at baseline\]) multiplied by 100. Baseline values for the original placebo participants refer to Day 1 of LTTP and baseline values for the original eliglustat participants refer to the Day 1 of PAP. |
Source ClinicalTrials.gov
Outcomes9
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | PAP: Percent Change From Baseline in Spleen Volume (in Multiples of Normal [MN]) at Week 39 of the Primary Analysis Period With Eliglustat Tartrate Treatment as Compared to Placebo | PAP Baseline (Day 1), Week 39 | FAS for PAP included all participants who signed informed consent and received at least one dose of study drug (placebo or eliglustat). | percent change | LEAST_SQUARES_MEAN |
| SECONDARY | PAP: Hemoglobin Level | PAP Baseline (Day 1) | FAS for PAP included all participants who signed informed consent and received at least one dose of study drug (placebo or eliglustat). | gram per deciliter (g/dL) | MEAN |
| SECONDARY | PAP: Absolute Change From Baseline in Hemoglobin Level at Week 39 | PAP Baseline (Day 1), Week 39 | FAS for PAP included all participants who signed informed consent and received at least one dose of study drug (placebo or eliglustat). | g/dL | LEAST_SQUARES_MEAN |
| SECONDARY | PAP: Percent Change From Baseline in Liver Volume (in MN) at Week 39 | PAP Baseline (Day 1), Week 39 | FAS for PAP included all participants who signed informed consent and received at least one dose of study drug (placebo or eliglustat). | percent change | LEAST_SQUARES_MEAN |
| SECONDARY | PAP: Percent Change From Baseline in Platelet Counts at Week 39 | PAP Baseline (Day 1), Week 39 | FAS for PAP included all participants who signed informed consent and received at least one dose of study drug (placebo or eliglustat). | percent change | LEAST_SQUARES_MEAN |
| SECONDARY | LTTP: Percent Change From Baseline in Spleen Volume (in MN) at Week 234 | PAP Baseline for Eliglustat (Originally on Eliglustat) arm, LTTP Baseline for Eliglustat (Originally on Placebo) arm, Week 234 | Intent-to-treat (ITT) population for LTTP included all participants who received at least 1 dose of eliglustat in LTTP period. Number of participants analyzed= participants evaluable for this outcome measure and had available data for baseline and Week 234 spleen volume assessment. | percent change | MEAN |
| SECONDARY | LTTP: Absolute Change From Baseline in Hemoglobin Level at Week 234 | PAP Baseline for Eliglustat (Originally on Eliglustat) arm, LTTP Baseline for Eliglustat (Originally on Placebo) arm, Week 234 | ITT population for LTTP included all participants who received at least 1 dose of eliglustat in LTTP period. Number of participants analyzed=participants evaluable for this outcome measure and had available data for baseline and Week 234 hemoglobin level assessment. | g/dL | MEAN |
| SECONDARY | LTTP: Percent Change From Baseline in Liver Volume (in MN) at Week 234 | PAP Baseline for Eliglustat (Originally on Eliglustat) arm, LTTP Baseline for Eliglustat (Originally on Placebo) arm, Week 234 | ITT population for LTTP included all participants who received at least 1 dose of eliglustat in LTTP period. Number of participants analyzed=participants evaluable for this outcome measure and had available data for baseline and Week 234 liver volume assessment. | percent change | MEAN |
| SECONDARY | LTTP: Percent Change From Baseline in Platelet Counts at Week 234 | PAP Baseline for Eliglustat (Originally on Eliglustat) arm, LTTP Baseline for Eliglustat (Originally on Placebo) arm, Week 234 | ITT population for LTTP included all participants who received at least 1 dose of eliglustat in LTTP period. Number of participants analyzed=participants evaluable for this outcome measure and had available data for baseline and Week 234 platelet count assessment. | percent change | MEAN |
Source ClinicalTrials.gov
Results by group18
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| PAP: Percent Change From Baseline in Spleen Volume (in Multiples of Normal [MN]) at Week 39 of the Primary Analysis Period With Eliglustat Tartrate Treatment as Compared to Placebo | PAP: Eliglustat | -27.77 | Standard Error 2.37 | percent change | |
| PAP: Percent Change From Baseline in Spleen Volume (in Multiples of Normal [MN]) at Week 39 of the Primary Analysis Period With Eliglustat Tartrate Treatment as Compared to Placebo | PAP: Placebo | 2.26 | Standard Error 2.37 | percent change | |
| PAP: Hemoglobin Level | PAP: Eliglustat | 12.05 | Standard Deviation 1.816 | gram per deciliter (g/dL) | |
| PAP: Hemoglobin Level | PAP: Placebo | 12.75 | Standard Deviation 1.629 | gram per deciliter (g/dL) | |
| PAP: Absolute Change From Baseline in Hemoglobin Level at Week 39 | PAP: Eliglustat | 0.69 | Standard Error 0.23 | g/dL | |
| PAP: Absolute Change From Baseline in Hemoglobin Level at Week 39 | PAP: Placebo | -0.54 | Standard Error 0.23 | g/dL | |
| PAP: Percent Change From Baseline in Liver Volume (in MN) at Week 39 | PAP: Eliglustat | -5.20 | Standard Error 1.64 | percent change | |
| PAP: Percent Change From Baseline in Liver Volume (in MN) at Week 39 | PAP: Placebo | 1.44 | Standard Error 1.64 | percent change | |
| PAP: Percent Change From Baseline in Platelet Counts at Week 39 | PAP: Eliglustat | 32.00 | Standard Error 5.95 | percent change | |
| PAP: Percent Change From Baseline in Platelet Counts at Week 39 | PAP: Placebo | -9.06 | Standard Error 5.95 | percent change |
1 to 10 of 18Source ClinicalTrials.gov
See all 18 results by groupParticipant flow24
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| PAP (Up To Week 39) | STARTED | PAP: Eliglustat | 20 | |
| PAP (Up To Week 39) | STARTED | PAP: Placebo | 20 | |
| PAP (Up To Week 39) | STARTED | LTTP: Eliglustat (Originally on Eliglustat) | 0 | |
| PAP (Up To Week 39) | STARTED | LTTP: Eliglustat (Originally on Placebo) | 0 | |
| PAP (Up To Week 39) | COMPLETED | PAP: Eliglustat | 19 | |
| PAP (Up To Week 39) | COMPLETED | PAP: Placebo | 20 | |
| PAP (Up To Week 39) | COMPLETED | LTTP: Eliglustat (Originally on Eliglustat) | 0 | |
| PAP (Up To Week 39) | COMPLETED | LTTP: Eliglustat (Originally on Placebo) | 0 | |
| PAP (Up To Week 39) | NOT COMPLETED | PAP: Eliglustat | 1 | |
| PAP (Up To Week 39) | NOT COMPLETED | PAP: Placebo | 0 |
1 to 10 of 24Source ClinicalTrials.gov
See all 24 participant flowAdverse events124
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 | Events |
|---|---|---|---|---|---|---|
| other | Iron deficiency anaemia | Blood and lymphatic system disorders | Eliglustat | 2 | 20 | |
| other | Iron deficiency anaemia | Blood and lymphatic system disorders | Placebo | 1 | 20 | |
| other | Palpitations | Cardiac disorders | Eliglustat | 0 | 20 | |
| other | Palpitations | Cardiac disorders | Placebo | 3 | 20 | |
| other | Eye irritation | Eye disorders | Eliglustat | 2 | 20 | |
| other | Eye irritation | Eye disorders | Placebo | 0 | 20 | |
| other | Abdominal distension | Gastrointestinal disorders | Eliglustat | 3 | 20 | |
| other | Abdominal distension | Gastrointestinal disorders | Placebo | 0 | 20 | |
| other | Abdominal pain | Gastrointestinal disorders | Eliglustat | 4 | 20 | |
| other | Abdominal pain | Gastrointestinal disorders | Placebo | 2 | 20 |
1 to 10 of 124Source ClinicalTrials.gov
See all 124 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Eliglustat | ||
| Total, all-cause mortality | Placebo | ||
| Total, other adverse events | Eliglustat | 17 | 20 |
| Total, other adverse events | Placebo | 14 | 20 |
| Total, serious adverse events | Eliglustat | 2 | 20 |
| Total, serious adverse events | Placebo | 3 | 20 |
Source ClinicalTrials.gov
Papers10
The papers the trial lists on ClinicalTrials.gov.
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 |
|---|
| Eliglustat vs Placebo: 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.
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/NCT00891202
GET https://conductscience.org/api/trials/v1/NCT00891202/flow?page=2