Trial · NCT00943111
A Study of Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease Who Have Reached Therapeutic Goals With Enzyme Replacement Therapy (ENCORE)
NCT00943111, Phase 3, Completed, Genzyme, a Sanofi Company; results first posted 2014-09-04
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 Who Have Reached Therapeutic Goals With Enzyme Replacement Therapy (ENCORE). Trials, record NCT00943111. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00943111
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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 Who Have Reached Therapeutic Goals With Enzyme Replacement Therapy (ENCORE)
- NCT number
- NCT00943111
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 160 participants, actual
- Lead sponsor
- Genzyme, a Sanofi Company
- Conditions
- Gaucher Disease, Type 1
- Interventions
- Eliglustat tartrate, Imiglucerase
- First posted
- 2009-07-22
- Last updated
- 2016-11-25
- Other registries' IDs
- eudract:2008-005223-28
- Results first posted
- 2014-09-04
Source ClinicalTrials.gov
Registered outcomes17
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Percentage of Participants Who Remained Stable for 52 Weeks During the Primary Analysis Period | Baseline up to Week 52 | For a participant to be classified as stable, the participant must have remained stable in hematological parameters (hemoglobin levels and platelet counts) and organ volumes (spleen, when applicable, and liver volumes in multiples of normal \[MN\]). Stable hematological parameters were defined as hemoglobin level did not decrease more than (\>) 1.5 gram per deciliter (g/dL) from baseline and platelet count did not decrease \>25% from baseline. Stable organ volumes were defined as spleen volume (in MN) did not increase \>25% from baseline, if applicable, and liver volume (in MN) did not increase \>20% from baseline. |
| Primary | Percentage of Participants Who Remained Stable Annually for 4 Years During the LTTP | Week 52 up to week 208 | For a participant to be classified as stable, the participant must have remained stable in hematological parameters (hemoglobin levels and platelet counts) and organ volumes (spleen, when applicable, and liver volumes in MN). Stable hematological parameters were defined as hemoglobin level did not decrease \>1.5 g/dL from baseline and platelet count did not decrease \>25% from baseline. Stable organ volumes were defined as spleen volume (in MN) did not increase \>25% from baseline, if applicable, and liver volume did not increase \>20% from baseline. |
| Secondary | Total T-Scores for Bone Mineral Density | Baseline | Images of the spine and bilateral femur were obtained by dual energy X-Ray absorptiometry (DXA) to determine T-score for each bone area and total bone mineral density. T-score compares participant's bone density with that of healthy young participant. The T-score bone density categories are: normal (score greater than \[\>\]-1), osteopenia (score -2.5 to less than or equal to \[\<=\] -1), and osteoporosis (score \<= -2.5). |
| Secondary | Absolute Change From Baseline in Total T-Scores for Bone Mineral Density at Week 52 | Baseline, Week 52 | Images of the spine and bilateral femur were obtained by DXA to determine T-score for each bone area and total bone mineral density. T-score compares participant's bone density with that of healthy young participant. The T-score bone density categories are: normal (score \>-1), osteopenia (score -2.5 to \<=-1), and osteoporosis (score \<= -2.5). Absolute change = T-score at Week 52 minus T-score at baseline. |
| Secondary | Total Z-Scores for Bone Mineral Density | Baseline | Images of the spine and bilateral femur were obtained by DXA to determine Z-score for each bone area and total bone mineral density. The Z-score bone density categories are: normal (score \>-2) and below normal (score \<=-2). |
| Secondary | Absolute Change From Baseline in Total Z-Scores for Bone Mineral Density at Week 52 | Baseline, Week 52 | Images of the spine and bilateral femur were obtained by DXA to determine Z-score for each bone area and total bone mineral density. The Z-score bone density categories are: normal (score \>-2) and below normal (score \<=-2). Absolute change = Z-score at Week 52 minus Z-score at baseline. |
| Secondary | Hemoglobin Level | Baseline | |
| Secondary | Absolute Change From Baseline in Hemoglobin Levels at Week 52 | Baseline, Week 52 | Absolute change = hemoglobin level at Week 52 minus hemoglobin level at baseline. |
| Secondary | Percent Change From Baseline in Platelet Counts at Week 52 | Baseline, Week 52 | Percent change in platelet counts = (\[platelet count at Week 52 minus platelet count at baseline\] divided by \[platelet count at baseline\]) multiplied by 100. |
| Secondary | Percent Change From Baseline in Spleen Volume (MN) at Week 52 | Baseline, Week 52 | Percent change in spleen volume = (\[spleen volume at Week 52 minus spleen volume at baseline\] divided by \[spleen volume at baseline\]) multiplied by 100, where all volumes are in MN. |
1 to 10 of 17Source ClinicalTrials.gov
See all 17 registered outcomesOutcomes17
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Percentage of Participants Who Remained Stable for 52 Weeks During the Primary Analysis Period | Baseline up to Week 52 | Per protocol population for PAP included participants who were at least 80% compliant with treatment during PAP, had no major protocol deviations, and did not exhibit hematological decline as a result of medically determined etiologies other than Gaucher disease. | percentage of participants | NUMBER |
| PRIMARY | Percentage of Participants Who Remained Stable Annually for 4 Years During the LTTP | Week 52 up to week 208 | FAS population for LTTP: included all participants who received at least 1 dose of eliglustat in the extension study period. Number of participants analyzed=participants at risk at specified time-points. Here 'n' signifies number of participants with available data for specified time-points. | percentage of participants | NUMBER |
| SECONDARY | Total T-Scores for Bone Mineral Density | Baseline | Per protocol population for PAP. Number of participants analyzed = participants with baseline T-score assessment. Here, 'n' signifies participants with baseline T-score assessment for specified bone area. | T-score | MEAN |
| SECONDARY | Absolute Change From Baseline in Total T-Scores for Bone Mineral Density at Week 52 | Baseline, Week 52 | Per protocol population for PAP. Number of participants analyzed = participants with both baseline and Week 52 T-score assessment. Here, 'n' signifies participants with both baseline and Week 52 T-score assessment for specified bone area. Eliglustat participants switching to imiglucerase were excluded. | T-score | LEAST_SQUARES_MEAN |
| SECONDARY | Total Z-Scores for Bone Mineral Density | Baseline | Per protocol population for PAP. Number of participants analyzed = participants with baseline Z-score assessment. Here, 'n' signifies participants with baseline Z-score assessment for specified bone area. | Z-score | MEAN |
| SECONDARY | Absolute Change From Baseline in Total Z-Scores for Bone Mineral Density at Week 52 | Baseline, Week 52 | Per protocol population for PAP. Number of participants analyzed = participants with both baseline and Week 52 Z-score assessment. Here, 'n' signifies participants with both baseline and Week 52 Z-score assessment for specified bone area. Eliglustat participants switching to imiglucerase were excluded. | Z-score | LEAST_SQUARES_MEAN |
| SECONDARY | Hemoglobin Level | Baseline | Per protocol population for PAP. Number of participants analyzed = participants with baseline hemoglobin assessment. | gram per deciliter (g/dL) | MEAN |
| SECONDARY | Absolute Change From Baseline in Hemoglobin Levels at Week 52 | Baseline, Week 52 | Per protocol population for PAP. Number of participants analyzed = participants with both baseline and Week 52 hemoglobin assessment. Eliglustat participants switching to imiglucerase were excluded. | g/dL | LEAST_SQUARES_MEAN |
| SECONDARY | Percent Change From Baseline in Platelet Counts at Week 52 | Baseline, Week 52 | Per protocol population for PAP. Number of participants analyzed = participants with both baseline and Week 52 platelet assessment. Eliglustat participants switching to imiglucerase were excluded. | percent change | LEAST_SQUARES_MEAN |
| SECONDARY | Percent Change From Baseline in Spleen Volume (MN) at Week 52 | Baseline, Week 52 | Per protocol population for PAP. Number of participants analyzed = participants with both baseline and Week 52 spleen volume assessment. Eliglustat participants switching to imiglucerase were excluded. | percent change | LEAST_SQUARES_MEAN |
1 to 10 of 17Source ClinicalTrials.gov
See all 17 outcomesResults by group40
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Percentage of Participants Who Remained Stable for 52 Weeks During the Primary Analysis Period | Eliglustat: PAP | 84.8 | 95% Confidence Interval 76.2 to 91.3 | percentage of participants | |
| Percentage of Participants Who Remained Stable for 52 Weeks During the Primary Analysis Period | Imiglucerase: PAP | 93.6 | 95% Confidence Interval 82.5 to 98.7 | percentage of participants | |
| Percentage of Participants Who Remained Stable Annually for 4 Years During the LTTP | Eliglustat: LTTP | Year 1 (n=127) | 83.6 | percentage of participants | |
| Percentage of Participants Who Remained Stable Annually for 4 Years During the LTTP | Eliglustat: LTTP | Year 2 (n= 115) | 75.65 | percentage of participants | |
| Percentage of Participants Who Remained Stable Annually for 4 Years During the LTTP | Eliglustat: LTTP | Year 3 (n= 92) | 60.53 | percentage of participants | |
| Percentage of Participants Who Remained Stable Annually for 4 Years During the LTTP | Eliglustat: LTTP | Year 4 (n= 41) | 26.97 | percentage of participants | |
| Total T-Scores for Bone Mineral Density | Eliglustat: PAP | Lumbar Spine T-Score (n=81, 38) | -0.56 | Standard Deviation 1.309 | T-score |
| Total T-Scores for Bone Mineral Density | Imiglucerase: PAP | Lumbar Spine T-Score (n=81, 38) | -0.33 | Standard Deviation 1.169 | T-score |
| Total T-Scores for Bone Mineral Density | Eliglustat: PAP | Femur T-Score (n=80, 37) | -0.11 | Standard Deviation 1.080 | T-score |
| Total T-Scores for Bone Mineral Density | Imiglucerase: PAP | Femur T-Score (n=80, 37) | -0.47 | Standard Deviation 1.293 | T-score |
1 to 10 of 40Source ClinicalTrials.gov
See all 40 results by groupParticipant flow21
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| 52-Weeks Primary Analysis Period | STARTED | Eliglustat: PAP | 106 | |
| 52-Weeks Primary Analysis Period | STARTED | Imiglucerase: PAP | 54 | |
| 52-Weeks Primary Analysis Period | STARTED | Eliglustat: LTTP | 0 | |
| 52-Weeks Primary Analysis Period | Treated | Eliglustat: PAP | 106 | |
| 52-Weeks Primary Analysis Period | Treated | Imiglucerase: PAP | 53 | |
| 52-Weeks Primary Analysis Period | Treated | Eliglustat: LTTP | 0 | |
| 52-Weeks Primary Analysis Period | COMPLETED | Eliglustat: PAP | 104 | |
| 52-Weeks Primary Analysis Period | COMPLETED | Imiglucerase: PAP | 52 | |
| 52-Weeks Primary Analysis Period | COMPLETED | Eliglustat: LTTP | 0 | |
| 52-Weeks Primary Analysis Period | NOT COMPLETED | Eliglustat: PAP | 2 |
1 to 10 of 21Source ClinicalTrials.gov
See all 21 participant flowAdverse events150
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 | Palpitations | Cardiac disorders | Eliglustat | 8 | 106 | |
| other | Palpitations | Cardiac disorders | Imiglucerase | 3 | 53 | |
| other | Abdominal pain | Gastrointestinal disorders | Eliglustat | 14 | 106 | |
| other | Abdominal pain | Gastrointestinal disorders | Imiglucerase | 6 | 53 | |
| other | Abdominal pain lower | Gastrointestinal disorders | Eliglustat | 6 | 106 | |
| other | Abdominal pain lower | Gastrointestinal disorders | Imiglucerase | 0 | 53 | |
| other | Abdominal pain upper | Gastrointestinal disorders | Eliglustat | 26 | 106 | |
| other | Abdominal pain upper | Gastrointestinal disorders | Imiglucerase | 3 | 53 | |
| other | Constipation | Gastrointestinal disorders | Eliglustat | 10 | 106 | |
| other | Constipation | Gastrointestinal disorders | Imiglucerase | 6 | 53 |
1 to 10 of 150Source ClinicalTrials.gov
See all 150 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 | Imiglucerase | ||
| Total, other adverse events | Eliglustat | 91 | 106 |
| Total, other adverse events | Imiglucerase | 38 | 53 |
| Total, serious adverse events | Eliglustat | 18 | 106 |
| Total, serious adverse events | Imiglucerase | 7 | 53 |
Source ClinicalTrials.gov
Papers7
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 Imiglucerase: 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/NCT00943111
GET https://conductscience.org/api/trials/v1/NCT00943111/results?page=2