Trial · NCT01074944
A Study of Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease to Evaluate Once Daily Versus Twice Daily Dosing (EDGE)
NCT01074944, Phase 3, Completed, Genzyme, a Sanofi Company; results first posted 2016-12-02
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 to Evaluate Once Daily Versus Twice Daily Dosing (EDGE). Trials, record NCT01074944. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01074944
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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 to Evaluate Once Daily Versus Twice Daily Dosing (EDGE)
- NCT number
- NCT01074944
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 170 participants, actual
- Lead sponsor
- Genzyme, a Sanofi Company
- Conditions
- Gaucher Disease
- Interventions
- Eliglustat tartrate
- First posted
- 2010-02-24
- Last updated
- 2017-02-06
- Other registries' IDs
- eudract:2009-015811-42
- Results first posted
- 2016-12-02
Source ClinicalTrials.gov
Registered outcomes36
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | PAP: Percentage of Participants Who Remained Stable for 52 Weeks During the PAP | PAP Baseline up to the end of PAP (Week 52) | Participants were considered as stable if they met all of the following criteria: 1) no more than 2 bone crisis during PAP (with no more than 1 bone crisis during either the first 6 months or the later 6 months of the period), and were free of other clinically symptomatic bone disease during the entire 52-week PAP; 2) hemoglobin level not decreased \>1.5 g/dL from Baseline for PAP; 3) platelet count not decreased \>25% from Baseline for PAP; 4) spleen volume (in multiples of normal \[MN\]) did not increase \>25% from Baseline for PAP; 5) liver volume (in MN) did not increase \>20% from Baseline for PAP. Baseline for PAP was defined as the last assessment prior to randomization. |
| Secondary | PAP: Mean Hemoglobin (Hb) Level at Baseline, Weeks 26 and 52 | Baseline, Week 26, Week 52 | |
| Secondary | PAP: Mean Platelet Count at Baseline, Weeks 26, 52 | Baseline, Week 26, Week 52 | |
| Secondary | PAP: Mean Spleen Volume at Baseline, Weeks 26, 52 | Baseline, Week 26, Week 52 | |
| Secondary | PAP: Mean Liver Volume at Baseline, Weeks 26, 52 | Baseline, Week 26 and Week 52 | |
| Secondary | PAP: Mean Biomarker (Chitotriosidase) Value at Baseline, Weeks 26 and Week 52 | Baseline, Week 26, Week 52 | Chitotriosidase biomarker was assayed from plasma. |
| Secondary | PAP: Mean Biomarker (GL-1 on DBS) Value at Baseline, Week 26 and Week 52 | Baseline, Week 26 and week 52 | GL-1 on DBS biomarker was assayed from dried blood spot (DBS). |
| Secondary | PAP: Mean Biomarker Macrophage Inflammatory Protein-1 Beta (MIP1-beta) Value at Baseline, Weeks 26, 52 | Baseline, Week 26, Week 52 | MIP1-beta biomarker was assayed from plasma. |
| Secondary | PAP: Bone Mineral Density (BMD) at Baseline and Week 52 | Baseline, Week 52 | BMD measurements of the spine and bilateral femur were acquired by dual-energy x-ray absorptiometry (DXA) scan. |
| Secondary | PAP: Total T-Scores for BMD at Baseline and 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. The T-score bone density categories were: normal (score \>-1), osteopenia (score -2.5 to \<=-1), and osteoporosis (score \<= -2.5). |
1 to 10 of 36Source ClinicalTrials.gov
See all 36 registered outcomesOutcomes36
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | PAP: Percentage of Participants Who Remained Stable for 52 Weeks During the PAP | PAP Baseline up to the end of PAP (Week 52) | Analysis was performed on per protocol (PP) population which included all participants who were at least 80% compliant with investigational medicinal product (IMP) dosing during PAP, had all of the necessary Baseline and Week 52 assessments to evaluate the primary endpoint, and did not have major protocol deviations. | percentage of participants | NUMBER |
| SECONDARY | PAP: Mean Hemoglobin (Hb) Level at Baseline, Weeks 26 and 52 | Baseline, Week 26, Week 52 | PP population included all participants who were at least 80% compliant with IMP dosing during PAP, had all of the necessary Baseline and Week 52 assessments to evaluate the primary endpoint, and did not have major protocol deviations. Here 'n' signifies number of participants with available data at specified time points for each arm respectively. | g/dL | MEAN |
| SECONDARY | PAP: Mean Platelet Count at Baseline, Weeks 26, 52 | Baseline, Week 26, Week 52 | PP population included all participants who were at least 80% compliant with IMP dosing during PAP, had all of the necessary Baseline and Week 52 assessments to evaluate the primary endpoint, and did not have major protocol deviations. Here 'n' signifies number of participants with available data at specified time points for each arm respectively. | platelets*10^9 /L | MEAN |
| SECONDARY | PAP: Mean Spleen Volume at Baseline, Weeks 26, 52 | Baseline, Week 26, Week 52 | PP population included all participants who were at least 80% compliant with IMP dosing during PAP, had all of the necessary Baseline and Week 52 assessments to evaluate the primary endpoint, and did not have major protocol deviations. Here 'n' signifies number of participants with available data for specified category for each arm respectively. | MN | MEAN |
| SECONDARY | PAP: Mean Liver Volume at Baseline, Weeks 26, 52 | Baseline, Week 26 and Week 52 | PP population included all participants who were at least 80% compliant with IMP dosing during PAP, had all of the necessary Baseline and Week 52 assessments to evaluate the primary endpoint, and did not have major protocol deviations. | MN | MEAN |
| SECONDARY | PAP: Mean Biomarker (Chitotriosidase) Value at Baseline, Weeks 26 and Week 52 | Baseline, Week 26, Week 52 | PP population which all participants who were at least 80% compliant with IMP dosing during PAP, had all of the necessary Baseline and Week 52 assessments to evaluate the primary endpoint, and did not have major protocol deviations. Here, 'n' signifies number of participants with available data for specified category for each arm respectively. | nmol/hr/mL | MEAN |
| SECONDARY | PAP: Mean Biomarker (GL-1 on DBS) Value at Baseline, Week 26 and Week 52 | Baseline, Week 26 and week 52 | PP population included all participants who were at least 80% compliant with IMP dosing during PAP, had all of the necessary Baseline and Week 52 assessments to evaluate the primary endpoint, and did not have major protocol deviations. Here 'n' signifies number of participants with available data at specified time points for each arm respectively. | mcg/mL | MEAN |
| SECONDARY | PAP: Mean Biomarker Macrophage Inflammatory Protein-1 Beta (MIP1-beta) Value at Baseline, Weeks 26, 52 | Baseline, Week 26, Week 52 | PP population included all participants who were at least 80% compliant with dosing during PAP, had all of the necessary Baseline and Week 52 assessments to evaluate the primary endpoint, and did not have major protocol deviations. Here, 'n' signifies number of participants with available data for specified category for each arm respectively. | pg/mL | MEAN |
| SECONDARY | PAP: Bone Mineral Density (BMD) at Baseline and Week 52 | Baseline, Week 52 | PP population included all participants who were at least 80% compliant with IMP dosing during PAP, had all of the necessary Baseline and Week 52 assessments to evaluate the primary endpoint, and did not have major protocol deviations. Here, 'n' signifies number of participants with available data at specified time points for each arm respectively. | g/cm^2 | MEAN |
| SECONDARY | PAP: Total T-Scores for BMD at Baseline and Week 52 | Baseline, Week 52 | PP population included all participants who were at least 80% compliant with IMP dosing during PAP, had all of the necessary Baseline and Week 52 assessments to evaluate the primary endpoint, and did not have major protocol deviations. Here 'n' signifies number of participants with available data at specified time points for each arm respectively. | T-score | MEAN |
1 to 10 of 36Source ClinicalTrials.gov
See all 36 outcomesResults by group346
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| PAP: Percentage of Participants Who Remained Stable for 52 Weeks During the PAP | PAP, Eliglustat: Once Daily | 80.4 | 95% Confidence Interval 67.6 to 89.8 | percentage of participants | |
| PAP: Percentage of Participants Who Remained Stable for 52 Weeks During the PAP | PAP, Eliglustat: Twice Daily | 83.1 | 95% Confidence Interval 71.0 to 91.6 | percentage of participants | |
| PAP: Mean Hemoglobin (Hb) Level at Baseline, Weeks 26 and 52 | PAP, Eliglustat: Once Daily | Baseline (n=56, 59) | 13.641 | Standard Deviation 1.214 | g/dL |
| PAP: Mean Hemoglobin (Hb) Level at Baseline, Weeks 26 and 52 | PAP, Eliglustat: Twice Daily | Baseline (n=56, 59) | 13.691 | Standard Deviation 1.273 | g/dL |
| PAP: Mean Hemoglobin (Hb) Level at Baseline, Weeks 26 and 52 | PAP, Eliglustat: Once Daily | Week 26 (n=56, 57) | 13.677 | Standard Deviation 1.377 | g/dL |
| PAP: Mean Hemoglobin (Hb) Level at Baseline, Weeks 26 and 52 | PAP, Eliglustat: Twice Daily | Week 26 (n=56, 57) | 13.946 | Standard Deviation 1.509 | g/dL |
| PAP: Mean Hemoglobin (Hb) Level at Baseline, Weeks 26 and 52 | PAP, Eliglustat: Once Daily | Week 52 (n=56, 59) | 13.605 | Standard Deviation 1.432 | g/dL |
| PAP: Mean Hemoglobin (Hb) Level at Baseline, Weeks 26 and 52 | PAP, Eliglustat: Twice Daily | Week 52 (n=56, 59) | 13.824 | Standard Deviation 1.442 | g/dL |
| PAP: Mean Platelet Count at Baseline, Weeks 26, 52 | PAP, Eliglustat: Once Daily | Baseline (n=56, 59) | 204.01 | Standard Deviation 81.49 | platelets*10^9 /L |
| PAP: Mean Platelet Count at Baseline, Weeks 26, 52 | PAP, Eliglustat: Twice Daily | Baseline (n=56, 59) | 171.09 | Standard Deviation 63.50 | platelets*10^9 /L |
1 to 10 of 346Source ClinicalTrials.gov
See all 346 results by groupParticipant flow60
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Lead-in Period (up to 78 Weeks) | STARTED | LIP, Eliglustat | 170 | |
| Lead-in Period (up to 78 Weeks) | STARTED | PAP, Eliglustat: Once Daily | 0 | |
| Lead-in Period (up to 78 Weeks) | STARTED | PAP, Eliglustat: Twice Daily | 0 | |
| Lead-in Period (up to 78 Weeks) | STARTED | LTTP, Eliglustat | 0 | |
| Lead-in Period (up to 78 Weeks) | STARTED | ETP, Eliglustat | 0 | |
| Lead-in Period (up to 78 Weeks) | COMPLETED | LIP, Eliglustat | 157 | |
| Lead-in Period (up to 78 Weeks) | COMPLETED | PAP, Eliglustat: Once Daily | 0 | |
| Lead-in Period (up to 78 Weeks) | COMPLETED | PAP, Eliglustat: Twice Daily | 0 | |
| Lead-in Period (up to 78 Weeks) | COMPLETED | LTTP, Eliglustat | 0 | |
| Lead-in Period (up to 78 Weeks) | COMPLETED | ETP, Eliglustat | 0 |
1 to 10 of 60Source ClinicalTrials.gov
See all 60 participant flowAdverse events79
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 | Thrombocytopenia | Blood and lymphatic system disorders | Eliglustat | 9 | 170 | |
| other | Palpitations | Cardiac disorders | Eliglustat | 11 | 170 | |
| other | Abdominal distension | Gastrointestinal disorders | Eliglustat | 9 | 170 | |
| other | Abdominal pain | Gastrointestinal disorders | Eliglustat | 15 | 170 | |
| other | Abdominal pain upper | Gastrointestinal disorders | Eliglustat | 26 | 170 | |
| other | Constipation | Gastrointestinal disorders | Eliglustat | 16 | 170 | |
| other | Diarrhoea | Gastrointestinal disorders | Eliglustat | 24 | 170 | |
| other | Dyspepsia | Gastrointestinal disorders | Eliglustat | 19 | 170 | |
| other | Gastrooesophageal reflux disease | Gastrointestinal disorders | Eliglustat | 9 | 170 | |
| other | Nausea | Gastrointestinal disorders | Eliglustat | 18 | 170 |
1 to 10 of 79Source ClinicalTrials.gov
See all 79 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Eliglustat | ||
| Total, other adverse events | Eliglustat | 144 | 170 |
| Total, serious adverse events | Eliglustat | 40 | 170 |
Source ClinicalTrials.gov
Papers4
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 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.
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.
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GET https://conductscience.org/api/trials/v1/NCT01074944/registered?page=2