Trial · NCT02536755
Phase 3b Study to Evaluate Skeletal Response to Eliglustat in Adult Patients Who Completed Phase 2 or Phase 3 Studies
NCT02536755, Phase 3, Completed, Genzyme, a Sanofi Company; results first posted 2022-07-15
3 sourcestrials-2026-11-12-0.5.0
Download JSON
Cite this record
ConductScience Library. Phase 3b Study to Evaluate Skeletal Response to Eliglustat in Adult Patients Who Completed Phase 2 or Phase 3 Studies. Trials, record NCT02536755. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02536755
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Phase 3b Study to Evaluate Skeletal Response to Eliglustat in Adult Patients Who Completed Phase 2 or Phase 3 Studies
- NCT number
- NCT02536755
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 31 participants, actual
- Lead sponsor
- Genzyme, a Sanofi Company
- Conditions
- Gaucher Disease
- Interventions
- Eliglustat, GZ385660
- First posted
- 2015-09-01
- Last updated
- 2022-07-15
- Results first posted
- 2022-07-15
Source ClinicalTrials.gov
Registered outcomes34
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Number of Participants With Mobility Status Assessments at Study Baseline, Weeks 52, 104, 156 and 208 | Study Baseline, Weeks 52, 104, 156 and 208 | Mobility, i.e., ability to walk was assessed as a part of Gaucher disease assessment in participants. In this outcome measure, number of participants with their different mobility status along with the use of mobility aids (unrestricted mobility, walks with difficulty, walks with orthopaedic aid, requires wheelchair, bedridden) at specified time points were reported. Baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry. |
| Primary | Number of Participants With Bone Pain Levels During the Past 4 Weeks at Study Baseline, Weeks 52, 104, 156 and 208 | Study Baseline, Weeks 52, 104, 156 and 208 | Bone pain was assessed as a part of Gaucher disease assessment in participants. Participants were categorized as none (no bone pain), very mild bone pain, mild bone pain, moderate bone pain, severe bone pain and extreme bone pain during the past 4 weeks at each specified visit. In this outcome measure, number of participants with different level of bone pain during the past 4 weeks at specified time points were reported. Baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry. |
| Primary | Number of Participants With Bone Crisis at Study Baseline, Weeks 52, 104, 156 and 208 | Study Baseline, Weeks 52, 104, 156 and 208 | Bone crisis was assessed as a part of Gaucher disease assessment in participants. Acute, excruciating episodic bone pain is characteristic of Gaucher bone crisis, which typically causes periosteal elevation, elevated white blood cell count, fever, or debilitation lasting several days or longer and requires treatment with immobilization of the affected area, and opioid analgesics. Participants were categorized as 0= no bone crisis, 1= 1 bone crisis, 2= 2 bone crisis and \>=3 = more than 3 bone crisis during the assessment period. In this outcome measure, number of participants with different bone crises levels at specified time points were reported. Baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry. |
| Primary | Change From Current Study Baseline in Total Bone Marrow Burden (BMB) Scores at Weeks 52, 104, 156 and 208 | Study Baseline, Weeks 52, 104, 156 and 208 | Bone marrow burden (BMB) scores indicate the degree of bone marrow infiltration. BMB score was measured using MRI (magnetic resonance imaging (MRI), ranged from 0 (no abnormalities) to 8 points (severe disease) for the lumbar spine and from 0 (no abnormalities) to 8 points (severe disease) for the femurs. The total BMB score was calculated as the sum of scores for femur and lumbar spine regions which ranged from 0 (no abnormalities) to 16 (severe disease) points. A higher BMB score signified more severe bone marrow involvement. For this outcome measure, baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry. |
| Primary | Change From Eliglustat Baseline in Total Bone Marrow Burden (BMB) Scores at Weeks 52, 104, 156 and 208 | Eliglustat Baseline, Weeks 52, 104, 156 and 208 of the current study | Bone marrow burden (BMB) scores indicate the degree of bone marrow infiltration was measured using MRI, ranged from 0 (no abnormalities) to 8 points (severe disease) for the lumbar spine and from 0 (no abnormalities) to 8 points (severe disease) for the femurs. The total BMB score was calculated as the sum of scores for femur and lumbar spine regions which ranged from 0 (no abnormalities) to 16 (severe disease) points. A higher BMB score signified more severe bone marrow involvement. For this outcome measure, baseline refers to the eliglustat baseline, which was defined as participant's status at the time of first dose of eliglustat in the previous Phase 2 or Phase 3 study. |
| Primary | Change From Current Study Baseline in Total Spine and Femur Bone Mineral Density (BMD) at Weeks 52, 104, 156 and 208 | Study Baseline, Weeks 52, 104, 156 and 208 | Bone Mineral Density (BMD) measurements of the spine and bilateral femur were acquired by dual energy X-Ray absorptiometry (DXA) scan. Worst total femur at Baseline refers to the "worst" diseased left or right femur at Baseline. For this outcome measure, baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry. |
| Primary | Change From Eliglustat Baseline in Total Spine and Femur Bone Mineral Density (BMD) at Weeks 52, 104, 156 and 208 | Eliglustat Baseline, Weeks 52, 104, 156 and 208 of the current study | BMD measurements of the spine and bilateral femur were acquired by DXA scan. Worst total femur at Baseline refers to the "worst" diseased left or right femur at baseline. For this outcome measure, baseline refers to the eliglustat baseline, which was defined as participant's status at the time of first dose of eliglustat in the previous Phase 2 or Phase 3 study. |
| Primary | Change From Current Study Baseline in Spine and Femur Total T-Scores for BMD at Weeks 52, 104, 156 and 208 | Study Baseline, Weeks 52, 104, 156 and 208 | BMD measurements of the spine and bilateral femur were acquired by DXA scan. The T-score bone density categories were: normal (score \>-1), osteopenia (score -2.5 to \<=-1), and osteoporosis (score \<= -2.5). Worst total femur at Baseline refers to the "worst" diseased left or right femur at baseline. For this outcome measure, baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry. |
| Primary | Change From Eliglustat Baseline in Spine and Femur Total T-Scores for BMD at Weeks 52, 104, 156 and 208 | Eliglustat Baseline, Weeks 52, 104, 156 and 208 of the current study | BMD measurements of the spine and bilateral femur were acquired by DXA scan. The T-score bone density categories were: normal (score \>-1), osteopenia (score -2.5 to \<=-1), and osteoporosis (score \<= -2.5). Worst total femur at Baseline refers to the "worst" diseased left or right femur at baseline. For this outcome measure, baseline refers to the eliglustat baseline, which was defined as participant's status at the time of first dose of eliglustat in the previous Phase 2 or Phase 3 study. |
| Primary | Change From Current Study Baseline in Spine and Femur Total Z-Scores for BMD at Weeks 52, 104, 156 and 208 | Study Baseline, Weeks 52, 104, 156 and 208 | BMD measurements of the spine and bilateral femur were acquired by DXA scan. The Z-score bone density categories were: normal (score \>-2) and below normal (score \<=-2). Worst total femur at Baseline refers to the "worst" diseased left or right femur at baseline. For this outcome measure, baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry. |
1 to 10 of 34Source ClinicalTrials.gov
See all 34 registered outcomesOutcomes34
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 Mobility Status Assessments at Study Baseline, Weeks 52, 104, 156 and 208 | Study Baseline, Weeks 52, 104, 156 and 208 | Analysis was performed on FAS population. Here, number analyzed = participants with available data for each specified category. Baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Number of Participants With Bone Pain Levels During the Past 4 Weeks at Study Baseline, Weeks 52, 104, 156 and 208 | Study Baseline, Weeks 52, 104, 156 and 208 | Analysis was performed on FAS population. Here, number analyzed = participants with available data for each specified category. Baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Number of Participants With Bone Crisis at Study Baseline, Weeks 52, 104, 156 and 208 | Study Baseline, Weeks 52, 104, 156 and 208 | Analysis was performed on FAS population. Here, number analyzed = participants with available data for each specified category. Baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Change From Current Study Baseline in Total Bone Marrow Burden (BMB) Scores at Weeks 52, 104, 156 and 208 | Study Baseline, Weeks 52, 104, 156 and 208 | Analysis was performed on FAS population. Here, number analyzed = participants with available data for each specified category. Baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry. | score on a scale | MEAN |
| PRIMARY | Change From Eliglustat Baseline in Total Bone Marrow Burden (BMB) Scores at Weeks 52, 104, 156 and 208 | Eliglustat Baseline, Weeks 52, 104, 156 and 208 of the current study | Analysis was performed on FAS population. Here, number analyzed = participants with available data for each specified category. Data for this outcome measure was planned to be collected and analyzed in reference to eliglustat baseline separately for participants from Phase 2, ENGAGE and EDGE studies (GD treatment naïve participants) and from ENCORE study (participants switched from Enzyme Replacement Therapy \[ERT\]). | score on a scale | MEAN |
| PRIMARY | Change From Current Study Baseline in Total Spine and Femur Bone Mineral Density (BMD) at Weeks 52, 104, 156 and 208 | Study Baseline, Weeks 52, 104, 156 and 208 | Analysis was performed on FAS population. Here, number analyzed = participants with available data for each specified category. Baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry. | grams per centimeter square (g/cm^2) | MEAN |
| PRIMARY | Change From Eliglustat Baseline in Total Spine and Femur Bone Mineral Density (BMD) at Weeks 52, 104, 156 and 208 | Eliglustat Baseline, Weeks 52, 104, 156 and 208 of the current study | Analysis was performed on FAS population. Here, number analyzed = participants with available data for each specified category. Data for this outcome measure was was planned to be collected and analyzed in reference to eliglustat baseline separately for participants from Phase 2, ENGAGE and EDGE studies (GD treatment naïve participants) and from ENCORE study (participants switched from ERT). | g/cm^2 | MEAN |
| PRIMARY | Change From Current Study Baseline in Spine and Femur Total T-Scores for BMD at Weeks 52, 104, 156 and 208 | Study Baseline, Weeks 52, 104, 156 and 208 | Analysis was performed on FAS population. Here, number analyzed = participants with available data for each specified category. Baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry. | T-score | MEAN |
| PRIMARY | Change From Eliglustat Baseline in Spine and Femur Total T-Scores for BMD at Weeks 52, 104, 156 and 208 | Eliglustat Baseline, Weeks 52, 104, 156 and 208 of the current study | Analysis was performed on FAS population. Here, number analyzed = participants with available data for each specified category. Here, 0 signifies that none of the participants were available for assessment at the specified timepoint. Data for this outcome measure was planned to be collected and analyzed in reference to eliglustat baseline separately for participants from Phase 2, ENGAGE and EDGE studies (GD treatment naïve participants) and from ENCORE study (participants switched from ERT). | T-score | MEAN |
| PRIMARY | Change From Current Study Baseline in Spine and Femur Total Z-Scores for BMD at Weeks 52, 104, 156 and 208 | Study Baseline, Weeks 52, 104, 156 and 208 | Analysis was performed on FAS population. Here, number analyzed = participants with available data for each specified category. Baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry. | Z-score | MEAN |
1 to 10 of 34Source ClinicalTrials.gov
See all 34 outcomesResults by group576
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 Mobility Status Assessments at Study Baseline, Weeks 52, 104, 156 and 208 | Eliglustat | Unrestricted mobility: Study Baseline | 31 | Participants | |
| Number of Participants With Mobility Status Assessments at Study Baseline, Weeks 52, 104, 156 and 208 | Eliglustat | Walks with difficulty: Study Baseline | 0 | Participants | |
| Number of Participants With Mobility Status Assessments at Study Baseline, Weeks 52, 104, 156 and 208 | Eliglustat | Walks with orthopedic aid: Study Baseline | 0 | Participants | |
| Number of Participants With Mobility Status Assessments at Study Baseline, Weeks 52, 104, 156 and 208 | Eliglustat | Requires wheelchair: Study Baseline | 0 | Participants | |
| Number of Participants With Mobility Status Assessments at Study Baseline, Weeks 52, 104, 156 and 208 | Eliglustat | Bedridden: Study Baseline | 0 | Participants | |
| Number of Participants With Mobility Status Assessments at Study Baseline, Weeks 52, 104, 156 and 208 | Eliglustat | Unrestricted mobility: Week 52 | 31 | Participants | |
| Number of Participants With Mobility Status Assessments at Study Baseline, Weeks 52, 104, 156 and 208 | Eliglustat | Walks with difficulty: Week 52 | 0 | Participants | |
| Number of Participants With Mobility Status Assessments at Study Baseline, Weeks 52, 104, 156 and 208 | Eliglustat | Walks with orthopedic aid: Week 52 | 0 | Participants | |
| Number of Participants With Mobility Status Assessments at Study Baseline, Weeks 52, 104, 156 and 208 | Eliglustat | Requires wheelchair: Week 52 | 0 | Participants | |
| Number of Participants With Mobility Status Assessments at Study Baseline, Weeks 52, 104, 156 and 208 | Eliglustat | Bedridden: Week 52 | 0 | Participants |
1 to 10 of 576Source ClinicalTrials.gov
See all 576 results by groupParticipant flow3
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Eliglustat | 31 | |
| Overall Study | COMPLETED | Eliglustat | 27 | |
| Overall Study | NOT COMPLETED | Eliglustat | 4 |
Source ClinicalTrials.gov
Adverse events13
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 | 3 | 31 | 3 |
| other | Toothache | Gastrointestinal disorders | Eliglustat | 2 | 31 | 3 |
| other | Human Chorionic Gonadotropin Increased | Investigations | Eliglustat | 2 | 31 | 3 |
| other | Arthralgia | Musculoskeletal and connective tissue disorders | Eliglustat | 2 | 31 | 2 |
| other | Intervertebral Disc Protrusion | Musculoskeletal and connective tissue disorders | Eliglustat | 2 | 31 | 2 |
| other | Osteopenia | Musculoskeletal and connective tissue disorders | Eliglustat | 2 | 31 | 3 |
| other | Spinal Pain | Musculoskeletal and connective tissue disorders | Eliglustat | 2 | 31 | 2 |
| other | Pregnancy | Pregnancy, puerperium and perinatal conditions | Eliglustat | 4 | 31 | 5 |
| serious | Pulmonary Tuberculosis | Infections and infestations | Eliglustat | 1 | 31 | 1 |
| serious | Meniscus Injury | Injury, poisoning and procedural complications | Eliglustat | 1 | 31 | 1 |
1 to 10 of 13Source ClinicalTrials.gov
See all 13 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Eliglustat | 0 | 31 |
| Total, other adverse events | Eliglustat | 12 | 31 |
| Total, serious adverse events | Eliglustat | 4 | 31 |
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.
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/NCT02536755
GET https://conductscience.org/api/trials/v1/NCT02536755/registered?page=2