ClinicalTrials.gov
ClinicalTrials.gov Menu

A Study of Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease to Evaluate Once Daily Versus Twice Daily Dosing (EDGE)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT01074944
Recruitment Status : Completed
First Posted : February 24, 2010
Results First Posted : December 2, 2016
Last Update Posted : February 6, 2017
Sponsor:
Information provided by (Responsible Party):
Sanofi ( Genzyme, a Sanofi Company )

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Condition Gaucher Disease
Intervention Drug: Eliglustat tartrate
Enrollment 170

Recruitment Details The study was conducted at 45 centers in 17 countries between 1 June 2010 and 6 October 2015. A total of 219 participants were screened, out of which 170 entered into the lead in period (LIP). Remaining 48 participants were screen failures and 1 participant withdrew before entering into the LIP.
Pre-assignment Details Participant flow divided into 4 periods: LIP:to assess randomization criteria. Primary analysis period (PAP):to assess therapeutic efficacy at 2 dosing regimen in randomized participants. Long-term treatment period (LTTP): to assess long term efficacy. Extended treatment period (ETP):those who were non-randomized after LIP continued in this period.
Arm/Group Title LIP, Eliglustat PAP, Eliglustat: Once Daily PAP, Eliglustat: Twice Daily LTTP, Eliglustat ETP, Eliglustat
Hide Arm/Group Description All participants (except in Japan) received eliglustat 50 mg, twice daily (BID) on Day 1 titrated up to 100 mg BID (50 or 100 mg capsules) based on their individual pharmacokinetics (PK) data for up to 78 weeks. All participants in Japan received eliglustat 50 mg once only on Day 1 and then eliglustat 50 mg BID from Day 2 titrated up to 100 mg BID (50 or 100 mg capsules) based on their individual PK data for up to 78 weeks. All participants who were randomized after meeting all randomization criteria (defined as: no more than 1 bone crisis and was free of other clinically symptomatic bone disease [such as bone pain attributable to osteonecrosis and/or pathological fractures) during the first 6 months of the LIP; mean hemoglobin level of ≥11 g/dL [if female] and ≥12 g/dL [if male]; mean platelet count ≥100,000/mm^3; spleen volume ≤10 times of normal; liver volume ≤1.5 times of normal; had a dose of 50 mg BID or 100 mg BID of eliglustat for at least 4 months and a peak (2-hour) Genz-99067 plasma concentration of <50 ng/mL) after either 26, 52 or 78 weeks of LIP received eliglustat capsules at the total daily dose (TDD) of 100 mg or 200 mg (the TDD they were on before randomization) once daily (QD) from Day 1 up to Week 52 in PAP (50 or 100 mg capsules). All participants who were randomized after meeting all randomization criteria (defined as: no more than 1 bone crisis and was free of other clinically symptomatic bone disease [such as bone pain attributable to osteonecrosis and/or pathological fractures) during the first 6 months of the LIP; mean hemoglobin level of ≥11 g/dL [if female] and ≥12 g/dL [if male]; mean platelet count ≥100,000/mm^3; spleen volume ≤10 times of normal; liver volume ≤1.5 times of normal; had a dose of 50 mg BID or 100 mg BID of eliglustat for at least 4 months and a peak (2-hour) Genz-99067 plasma concentration of <50 ng/mL) after either 26, 52 or 78 weeks of LIP, received eliglustat at the TDD 50 mg BID or 100 mg BID (the TDD they were on before randomization) from Day 1 up to Week 52 in PAP (50 or 100 mg capsules). All participants who entered PAP continued their blinded randomized treatment for first 4 weeks. Participants who at Week 52 of PAP maintained their therapeutic goals (defined as: no more than 2 bone crisis during PAP [with no more than 1 bone crisis during either first 6 months or later 6 months of PAP] and is free of other clinically symptomatic bone disease during PAP; hemoglobin level not decreased by >1.5 g/dL from Baseline for PAP [defined as last available assessment prior to randomization]; platelet count not decreased by >25% from Baseline for PAP; spleen volume not increased by 25% from Baseline for PAP; liver volume not increased by >20% from Baseline for PAP), continued into the LTTP and received their TDD of eliglustat (100 mg or 200 mg) QD till the end of the study. The participants who did not maintain all their therapeutic goals continued into the LTTP and received their TDD of eliglustat (100 mg or 200 mg) BID till the end of the study. All participants who did not meet all the randomization criteria at Week 78 of the LIP continued in the ETP and received eliglustat capsules at the same dose as they were receiving at the end of LIP till the end of the study.
Period Title: Lead-in Period (up to 78 Weeks)
Started 170 [1] 0 0 0 0
Completed 157 0 0 0 0
Not Completed 13 0 0 0 0
Reason Not Completed
Adverse Event             2             0             0             0             0
Non-Compliance With Protocol             1             0             0             0             0
Pregnancy             4             0             0             0             0
Withdrawal by Subject             6             0             0             0             0
[1]
LIP conducted to assess randomization criteria of all participants in single reporting arm.
Period Title: Primary Analysis Period (up to 52 Weeks)
Started 0 65 [1] 66 [1] 0 0
Completed 0 54 60 0 0
Not Completed 0 11 6 0 0
Reason Not Completed
Adverse Event             0             2             3             0             0
Treatment Failure             0             6             1             0             0
Withdrawal by Subject             0             1             1             0             0
Pregnancy             0             1             0             0             0
Non-Compliant to Protocol             0             1             1             0             0
[1]
Participants who were randomized after LIP received treatment in PAP.
Period Title: Long Term Treatment
Started 0 0 0 121 [1] 0
Completed 0 0 0 95 0
Not Completed 0 0 0 26 0
Reason Not Completed
Adverse Event             0             0             0             3             0
Lost to Follow-up             0             0             0             2             0
Transitioned to Commercial Eliglustat             0             0             0             18             0
Entered in Period, But Not Treated             0             0             0             1             0
Withdrawal by Subject             0             0             0             2             0
[1]
Participants who completed PAP (114) including those who considered as treatment failure in PAP (7).
Period Title: Extended Treatment (up to 42 Months)
Started 0 0 0 0 25 [1]
Completed 0 0 0 0 20
Not Completed 0 0 0 0 5
Reason Not Completed
Pregnancy             0             0             0             0             2
Transitioned to Commercial Eliglustat             0             0             0             0             3
[1]
Participants who were failed to meet the randomization criteria during the lead-in period.
Arm/Group Title All Participants
Hide Arm/Group Description All participants who received treatment in LIP (eliglustat 50 mg BID on Day 1 titrated up to 100 mg BID [50 or 100 mg capsules] based on their individual PK data for up to 78 weeks [except for the participants in Japan]. Participants in Japan received eliglustat 50 mg once only on Day 1 and then eliglustat 50 mg BID from Day 2 titrated up to 100 mg BID [50 or 100 mg capsules] based on their individual PK data for up to 78 weeks) and assessed for randomization.
Overall Number of Baseline Participants 170
Hide Baseline Analysis Population Description
All treated (AT) analysis set included all participants who received at least 1 dose of eliglustat during LIP.
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 170 participants
37.7  (15.1)
Gender  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 170 participants
Female
81
  47.6%
Male
89
  52.4%
1.Primary Outcome
Title PAP: Percentage of Participants Who Remained Stable for 52 Weeks During the PAP
Hide Description 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.
Time Frame PAP Baseline up to the end of PAP (Week 52)
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title PAP, Eliglustat: Once Daily PAP, Eliglustat: Twice Daily
Hide Arm/Group Description:
All participants who were randomized after either 26, 52 or 78 weeks of LIP received eliglustat at the TDD of 100 mg or 200 mg (the TDD they were on before randomization) QD from Day 1 up to Week 52 in PAP (50 or 100 mg capsules).
All participants who were randomized after either 26, 52 or 78 weeks of LIP received eliglustat 50 mg BID or 100 mg BID (the TDD they were on before randomization) from Day 1 up to Week 52 in PAP (50 or 100 mg capsules).
Overall Number of Participants Analyzed 56 59
Measure Type: Number
Number (95% Confidence Interval)
Unit of Measure: percentage of participants
80.4
(67.6 to 89.8)
83.1
(71.0 to 91.6)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection PAP, Eliglustat: Once Daily, PAP, Eliglustat: Twice Daily
Comments [Not Specified]
Type of Statistical Test Non-Inferiority or Equivalence
Comments Eliglustat QD treatment was declared non-inferior to BID treatment if the lower bound of the 95% confidence interval (CI) for the difference was within the non-inferiority margin of -0.15 (or -15%).
Method of Estimation Estimation Parameter Difference in Percentage Stable
Estimated Value -2.7
Confidence Interval (2-Sided) 95%
-17.7 to 11.9
Estimation Comments [Not Specified]
2.Secondary Outcome
Title PAP: Mean Hemoglobin (Hb) Level at Baseline, Weeks 26 and 52
Hide Description [Not Specified]
Time Frame Baseline, Week 26, Week 52
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title PAP, Eliglustat: Once Daily PAP, Eliglustat: Twice Daily
Hide Arm/Group Description:
All participants who were randomized after either 26, 52 or 78 weeks of LIP received eliglustat at the TDD of 100 mg or 200 mg (the TDD they were on before randomization) QD from Day 1 up to Week 52 in PAP (50 or 100 mg capsules).
All participants who were randomized after either 26, 52 or 78 weeks of LIP received eliglustat 50 mg BID or 100 mg BID (the TDD they were on before randomization) from Day 1 up to Week 52 in PAP (50 or 100 mg capsules).
Overall Number of Participants Analyzed 56 59
Mean (Standard Deviation)
Unit of Measure: g/dL
Baseline (n=56, 59) 13.641  (1.214) 13.691  (1.273)
Week 26 (n=56, 57) 13.677  (1.377) 13.946  (1.509)
Week 52 (n=56, 59) 13.605  (1.432) 13.824  (1.442)
3.Secondary Outcome
Title PAP: Mean Platelet Count at Baseline, Weeks 26, 52
Hide Description [Not Specified]
Time Frame Baseline, Week 26, Week 52
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title PAP, Eliglustat: Once Daily PAP, Eliglustat: Twice Daily
Hide Arm/Group Description:
All participants who were randomized after either 26, 52 or 78 weeks of LIP received eliglustat at the TDD of 100 mg or 200 mg (the TDD they were on before randomization) QD from Day 1 up to Week 52 in PAP (50 or 100 mg capsules).
All participants who were randomized after either 26, 52 or 78 weeks of LIP received eliglustat 50 mg BID or 100 mg BID (the TDD they were on before randomization) from Day 1 up to Week 52 in PAP (50 or 100 mg capsules).
Overall Number of Participants Analyzed 56 59
Mean (Standard Deviation)
Unit of Measure: platelets*10^9 /L
Baseline (n=56, 59) 204.01  (81.49) 171.09  (63.50)
Week 26 (n=56, 57) 195.75  (66.65) 173.94  (65.61)
Week 52 (n=56, 59) 207.20  (80.62) 176.10  (62.01)
4.Secondary Outcome
Title PAP: Mean Spleen Volume at Baseline, Weeks 26, 52
Hide Description [Not Specified]
Time Frame Baseline, Week 26, Week 52
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title PAP, Eliglustat: Once Daily PAP, Eliglustat: Twice Daily
Hide Arm/Group Description:
All participants who were randomized after either 26, 52 or 78 weeks of LIP received eliglustat at the TDD of 100 mg or 200 mg (the TDD they were on before randomization) QD from Day 1 up to Week 52 in PAP (50 or 100 mg capsules).
All participants who were randomized after either 26, 52 or 78 weeks of LIP received eliglustat 50 mg BID or 100 mg BID (the TDD they were on before randomization) from Day 1 up to Week 52 in PAP (50 or 100 mg capsules).
Overall Number of Participants Analyzed 56 59
Mean (Standard Deviation)
Unit of Measure: MN
Baseline (n= 39, 45) 3.309  (1.465) 3.787  (1.623)
Week 26 (n= 39, 45) 3.066  (1.299) 3.504  (1.365)
Week 52 (n= 39, 45) 3.017  (1.381) 3.394  (1.305)
5.Secondary Outcome
Title PAP: Mean Liver Volume at Baseline, Weeks 26, 52
Hide Description [Not Specified]
Time Frame Baseline, Week 26 and Week 52
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title PAP, Eliglustat: Once Daily PAP, Eliglustat: Twice Daily
Hide Arm/Group Description:
All participants who were randomized after either 26, 52 or 78 weeks of LIP received eliglustat at the TDD of 100 mg or 200 mg (the TDD they were on before randomization) QD from Day 1 up to Week 52 in PAP (50 or 100 mg capsules).
All participants who were randomized after either 26, 52 or 78 weeks of LIP received eliglustat 50 mg BID or 100 mg BID (the TDD they were on before randomization) from Day 1 up to Week 52 in PAP (50 or 100 mg capsules).
Overall Number of Participants Analyzed 56 59
Mean (Standard Deviation)
Unit of Measure: MN
Baseline (n=56, 59) 0.981  (0.187) 1.040  (0.198)
Week 26 (n=56, 59) 0.987  (0.190) 1.024  (0.179)
Week 52 (n=56, 59) 0.970  (0.170) 1.009  (0.196)
6.Secondary Outcome
Title PAP: Mean Biomarker (Chitotriosidase) Value at Baseline, Weeks 26 and Week 52
Hide Description Chitotriosidase biomarker was assayed from plasma.
Time Frame Baseline, Week 26, Week 52
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title PAP, Eliglustat: Once Daily PAP, Eliglustat: Twice Daily
Hide Arm/Group Description:
All participants who were randomized after either 26, 52 or 78 weeks of LIP received eliglustat at the TDD of 100 mg or 200 mg (the TDD they were on before randomization) QD from Day 1 up to Week 52 in PAP (50 or 100 mg capsules).
All participants who were randomized after either 26, 52 or 78 weeks of LIP received eliglustat 50 mg BID or 100 mg BID (the TDD they were on before randomization) from Day 1 up to Week 52 in PAP (50 or 100 mg capsules).
Overall Number of Participants Analyzed 56 59
Mean (Standard Deviation)
Unit of Measure: nmol/hr/mL
Baseline (n=55, 59) 1523.7  (2556.6) 1554.9  (1895.0)
Week 26 (n=52, 54) 1279.6  (2328.1) 1242.0  (2012.6)
Week 52 (n=54, 55) 1076.6  (1855.8) 1170.1  (1683.3)
7.Secondary Outcome
Title PAP: Mean Biomarker (GL-1 on DBS) Value at Baseline, Week 26 and Week 52
Hide Description GL-1 on DBS biomarker was assayed from dried blood spot (DBS).
Time Frame Baseline, Week 26 and week 52
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title PAP, Eliglustat: Once Daily PAP, Eliglustat: Twice Daily
Hide Arm/Group Description:
All participants who were randomized after either 26, 52 or 78 weeks of LIP received eliglustat at the TDD of 100 mg or 200 mg (the TDD they were on before randomization) QD from Day 1 up to Week 52 in PAP (50 or 100 mg capsules).
All participants who were randomized after either 26, 52 or 78 weeks of LIP received eliglustat 50 mg BID or 100 mg BID (the TDD they were on before randomization) from Day 1 up to Week 52 in PAP (50 or 100 mg capsules).
Overall Number of Participants Analyzed 56 59
Mean (Standard Deviation)
Unit of Measure: mcg/mL
GL-1 on DBS: Baseline (n=54, 55) 2.257  (0.835) 2.425  (1.378)
GL-1 on DBS: week 26 (n=54, 54) 2.481  (1.037) 2.563  (1.100)
GL-1 on DBS: week 52 (n=53, 55) 2.853  (1.383) 2.707  (1.443)
8.Secondary Outcome
Title PAP: Mean Biomarker Macrophage Inflammatory Protein-1 Beta (MIP1-beta) Value at Baseline, Weeks 26, 52
Hide Description MIP1-beta biomarker was assayed from plasma.
Time Frame Baseline, Week 26, Week 52
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title PAP, Eliglustat: Once Daily PAP, Eliglustat: Twice Daily
Hide Arm/Group Description:
All participants who were randomized after either 26, 52 or 78 weeks of LIP received eliglustat at the TDD of 100 mg or 200 mg (the TDD they were on before randomization) QD from Day 1 up to Week 52 in PAP (50 or 100 mg capsules).
All participants who were randomized after either 26, 52 or 78 weeks of LIP received eliglustat 50 mg BID or 100 mg BID (the TDD they were on before randomization) from Day 1 up to Week 52 in PAP (50 or 100 mg capsules).
Overall Number of Participants Analyzed 56 59
Mean (Standard Deviation)
Unit of Measure: pg/mL
Baseline (n=54, 58) 77.7  (74.4) 118.8  (156.3)
Week 26 (n=52, 54) 74.5  (68.0) 121.0  (204.4)
Week 52 (n=54, 55) 81.3  (82.8) 117.9  (165.3)
9.Secondary Outcome
Title PAP: Bone Mineral Density (BMD) at Baseline and Week 52
Hide Description BMD measurements of the spine and bilateral femur were acquired by dual-energy x-ray absorptiometry (DXA) scan.
Time Frame Baseline, Week 52
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title PAP, Eliglustat: Once Daily PAP, Eliglustat: Twice Daily
Hide Arm/Group Description:
All participants who were randomized after either 26, 52 or 78 weeks of LIP received eliglustat at the TDD of 100 mg or 200 mg (the TDD they were on before randomization) QD from Day 1 up to Week 52 in PAP (50 or 100 mg capsules).
All participants who were randomized after either 26, 52 or 78 weeks of LIP received eliglustat 50 mg BID or 100 mg BID (the TDD they were on before randomization) from Day 1 up to Week 52 in PAP (50 or 100 mg capsules).
Overall Number of Participants Analyzed 56 59
Mean (Standard Deviation)
Unit of Measure: g/cm^2
Lumbar Spine: Baseline (n=51, 55) 1.073  (0.177) 1.081  (0.172)
Lumbar Spine: Week 52 (n=51, 55) 1.089  (0.183) 1.086  (0.177)
Left Femur: Baseline (n=48, 47) 0.979  (0.219) 1.000  (0.199)
Left Femur: Week 52 (n=48, 47) 0.972  (0.211) 0.990  (0.196)
Right Femur: Baseline (n=48, 47) 0.971  (0.217) 0.996  (0.184)
Right Femur: Week 52 (n=48, 47) 0.967  (0.213) 0.981  (0.177)
10.Secondary Outcome
Title PAP: Total T-Scores for BMD at Baseline and Week 52
Hide Description 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).
Time Frame Baseline, Week 52
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title PAP, Eliglustat: Once Daily PAP, Eliglustat: Twice Daily
Hide Arm/Group Description:
All participants who were randomized after either 26, 52 or 78 weeks of LIP received eliglustat at the TDD of 100 mg or 200 mg (the TDD they were on before randomization) QD from Day 1 up to Week 52 in PAP (50 or 100 mg capsules).
All participants who were randomized after either 26, 52 or 78 weeks of LIP received eliglustat 50 mg BID or 100 mg BID (the TDD they were on before randomization) from Day 1 up to Week 52 in PAP (50 or 100 mg capsules).
Overall Number of Participants Analyzed 56 59
Mean (Standard Deviation)
Unit of Measure: T-score
Lumbar Spine T-Score: Baseline (n=49, 52) -0.722  (1.415) -0.771  (1.217)
Lumbar Spine T-Score: Week 52 (n=49, 52) -0.580  (1.476) -0.717  (1.271)
Left Femur T-Score: Baseline (n=46, 44) -0.459  (1.385) -0.368  (1.347)
Left Femur T-Score: Week 52 (n=46, 44) -0.509  (1.342) -0.441  (1.326)
Right Femur T-score: Baseline (n=46, 44) -0.574  (1.327) -0.382  (1.282)
Right Femur T-score: Week 52 (n=46, 44) -0.607  (1.308) -0.530  (1.236)
11.Secondary Outcome
Title PAP: Total Z-scores for BMD at Baseline and Week 52
Hide Description 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).
Time Frame Baseline, Week 52
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
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
Arm/Group Title PAP, Eliglustat: Once Daily PAP, Eliglustat: Twice Daily
Hide Arm/Group Description:
All participants who were randomized after either 26, 52 or 78 weeks of LIP received eliglustat at the TDD of 100 mg or 200 mg (the TDD they were on before randomization) QD from Day 1 up to Week 52 in PAP (50 or 100 mg capsules).
All participants who were randomized after either 26, 52 or 78 weeks of LIP received eliglustat 50 mg BID or 100 mg BID (the TDD they were on before randomization) from Day 1 up to Week 52 in PAP (50 or 100 mg capsules).
Overall Number of Participants Analyzed 56 59
Mean (Standard Deviation)
Unit of Measure: Z-score
Lumbar Spine Z-scores: Baseline (n=51, 55) -0.492  (1.517) -0.609  (1.166)
Lumbar Spine Z-scores: Week 52 (n=51, 55) -0.324  (1.559) -0.555  (1.202)
Left Femur Z-scores: Baseline (n= 48, 47) -0.171  (1.316) -0.115  (1.294)
Left Femur Z-scores: Week 52 (n= 48, 47) -0.202  (1.250) -0.183  (1.274)
Right Femur Z-scores: Baseline (n= 48, 47) -0.235  (1.251) -0.140  (1.194)
Right Femur Z-scores: Week 52 (n= 48, 47) -0.248  (1.214) -0.260  (1.141)
12.Secondary Outcome
Title PAP: Number of Participants With Mobility Status Asessments (MS) at Baseline, Weeks 26, and 52.
Hide Description Mobility, i.e., ability to walk was assessed as a part of Gaucher disease assessment in participants. In this outcome, 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.
Time Frame Baseline, Week 26 and Week 52
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title PAP, Eliglustat: Once Daily PAP, Eliglustat: Twice Daily
Hide Arm/Group Description:
All participants who were randomized after either 26, 52 or 78 weeks of LIP received eliglustat at the TDD of 100 mg or 200 mg (the TDD they were on before randomization) QD from Day 1 up to Week 52 in PAP (50 or 100 mg capsules).
All participants who were randomized after either 26, 52 or 78 weeks of LIP received eliglustat 50 mg BID or 100 mg BID (the TDD they were on before randomization) from Day 1 up to Week 52 in PAP (50 or 100 mg capsules).
Overall Number of Participants Analyzed 56 59
Measure Type: Number
Unit of Measure: participants
MS, Unrestricted: Baseline (n=56, 59) 49 59
MS, Unrestricted: Week 26 (n=55, 57) 46 56
MS, Unrestricted: Week 52 (n=51, 58) 50 57
MS, Walks with Difficulty: Baseline (n=56, 59) 6 0
MS, Walks with Difficulty: Week 26 (n=55, 57) 8 1
Ms, Walks with Difficulty: Week 52 (n=51, 58) 5 1
MS, Walks with Orthopedic Aid: Baseline (n=56, 59) 1 0
MS, Walks with Orthopedic Aid: Week 26 (n=55, 57) 1 0
MS, Walks with Orthopedic Aid: Week 52 (n=51, 58) 0 0
MS, Required Wheelchair: Baseline (n=56, 59) 0 0
MS, Required Wheelchair: Week 26 (n=55, 57) 0 0
MS, Required Wheelchair: Week 52 (n=51, 58) 1 0
MS, Bedridden: Baseline (n=56, 59) 0 0
MS, Bedridden: Week 26 (n=55, 57) 0 0
MS, Bedridden: Week 52 (n=51, 58) 0 0
13.Secondary Outcome
Title PAP: Number of Participants With Bone Crises at Baseline, Weeks 26 and 52
Hide Description 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 debilitation lasting several days or longer and requires treatment with immobilization, hydration, and opioid analgesics. Participants were categorized as 0= no bone crisis, 1= 1 bone crisis, and 2= 2 bone crises during the assessment period. In this outcome, number of participants with different bone crises levels at specified time points were reported.
Time Frame Baseline, Week 26, and Week 52
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title PAP, Eliglustat: Once Daily PAP, Eliglustat: Twice Daily
Hide Arm/Group Description:
All participants who were randomized after either 26, 52 or 78 weeks of LIP received eliglustat at the TDD of 100 mg or 200 mg (the TDD they were on before randomization) QD from Day 1 up to Week 52 in PAP (50 or 100 mg capsules).
All participants who were randomized after either 26, 52 or 78 weeks of LIP received eliglustat 50 mg BID or 100 mg BID (the TDD they were on before randomization) from Day 1 up to Week 52 in PAP (50 or 100 mg capsules).
Overall Number of Participants Analyzed 56 59
Measure Type: Number
Unit of Measure: participants
Bone Crisis (0): Baseline (n=56, 59) 55 57
Bone Crisis (0): Week 26 (n=55,57) 54 56
Bone Crisis (0): Week 52 (n=56, 58) 56 57
Bone Crisis (1): Baseline (n=56, 59) 1 2
Bone Crisis (1): Week 26 (n=55, 57) 0 1
Bone Crisis (1): Week 52 (n=56, 58) 0 1
Bone Crisis (2): Baseline (n=56, 59) 0 0
Bone Crisis (2): week 26 (n=55, 57) 1 0
Bone Crisis (2): week 52 (n=56, 58) 0 0
14.Secondary Outcome
Title PAP: Number of Participants With Bone Pain Levels During the Past 4 Weeks at Baseline, Weeks 26 and 52
Hide Description 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. In this outcome, number of participants with different level of bone pain during the past 4 weeks at specified time points were reported.
Time Frame Baseline, Week 26, and Week 52
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title PAP, Eliglustat: Once Daily PAP, Eliglustat: Twice Daily
Hide Arm/Group Description:
All participants who were randomized after either 26, 52 or 78 weeks of LIP received eliglustat at the TDD of 100 mg or 200 mg (the TDD they were on before randomization) QD from Day 1 up to Week 52 in PAP (50 or 100 mg capsules).
All participants who were randomized after either 26, 52 or 78 weeks of LIP received eliglustat 50 mg BID or 100 mg BID (the TDD they were on before randomization) from Day 1 up to Week 52 in PAP (50 or 100 mg capsules).
Overall Number of Participants Analyzed 56 59
Measure Type: Number
Unit of Measure: participants
None: Baseline (n=56, 59) 42 49
None: Week 26 (n=55, 57) 42 49
None: Week 52 (n=56, 58) 41 45
Very Mild: Baseline (n=56, 59) 2 1
Very Mild: Week 26 (n=55, 57) 3 4
Very Mild: Week 52 (n=56, 58) 3 7
Mild: Baseline (n=56, 59) 7 3
Mild: Week 26 (n=55, 57) 5 3
Mild: Week 52 (n=56, 58) 6 2
Moderate: Baseline (n=56, 59) 4 5
Moderate: Week 26 (n=55, 57) 5 1
Moderate: Week 52 (56, 58) 6 2
Severe: Baseline (n=56, 59) 1 1
Severe: Week 26 (n=55, 57) 0 0
Severe: Week 52 (n=56, 58) 0 2
Extreme: Baseline (n=56, 59) 0 0
Extreme: Week 26 (n=55, 57) 0 0
Extreme: Week 52 (n=56, 58) 0 0
15.Secondary Outcome
Title PAP: Total Bone Marrow Burden Score (BMB) at Baseline and Week 52
Hide Description BMB Score was measured using magnetic resonance imaging (MRI), range 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 score was calculated as the sum of scores for femur and lumbar spine regions which ranged from 0 (no abnormalities) -16 (severe disease) points. A higher BMB score signified more severe bone marrow involvement.
Time Frame Baseline, Week 52
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title PAP, Eliglustat: Once Daily PAP, Eliglustat: Twice Daily
Hide Arm/Group Description:
All participants who were randomized after either 26, 52 or 78 weeks of LIP received eliglustat at the TDD of 100 mg or 200 mg (the TDD they were on before randomization) QD from Day 1 up to Week 52 in PAP (50 or 100 mg capsules).
All participants who were randomized after either 26, 52 or 78 weeks of LIP received eliglustat 50 mg BID or 100 mg BID (the TDD they were on before randomization) from Day 1 up to Week 52 in PAP (50 or 100 mg capsules).
Overall Number of Participants Analyzed 56 59
Mean (Standard Deviation)
Unit of Measure: BMB Score
BMB Score: Baseline (n=52, 49) 8.276  (2.891) 9.136  (2.784)
BMB Score: Week 52 (n=51, 48) 7.971  (2.689) 8.705  (2.633)
16.Secondary Outcome
Title LIP: Mean Hemoglobin (Hb) Level at Baseline, Weeks 26, 52 and 78
Hide Description [Not Specified]
Time Frame Baseline, Week 26, Week, 52, and Week 78
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Analysis was performed on all treated (AT) analysis set which included all participants who received at least 1 dose of eliglustat during lead in period. Here 'n' signifies number of participants with available data at specified time points.
Arm/Group Title LIP: Eliglustat
Hide Arm/Group Description:
All participants (except in Japan) received eliglustat 50 mg BID on Day 1 titrated up to 100 mg BID (50 or 100 mg capsules) based on their individual PK data for up to 78 weeks. All participants in Japan received eliglustat 50 mg once only on Day 1 and then eliglustat 50 mg BID from Day 2 titrated up to 100 mg BID (50 or 100 mg capsules) based on their individual PK data for up to 78 weeks.
Overall Number of Participants Analyzed 170
Mean (Standard Deviation)
Unit of Measure: g/dL
Baseline (n=170) 13.435  (1.560)
Week 26 (n=163) 13.443  (1.382)
Week 52 (n=74) 13.434  (1.497)
Week 78 (n=41) 13.329  (1.528)
17.Secondary Outcome
Title LIP: Mean Platelet Count at Baseline, Weeks 26, 52 and 78
Hide Description [Not Specified]
Time Frame Baseline, Week 26, Week 52, Week 78
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Analysis was performed on AT analysis set which included all participants who received at least 1 dose of eliglustat during LIP. Here ‘n’ signifies number of participants with available data at specified time points.
Arm/Group Title LIP: Eliglustat
Hide Arm/Group Description:
All participants (except in Japan) received eliglustat 50 mg BID on Day 1 titrated up to 100 mg BID (50 or 100 mg capsules) based on their individual PK data for up to 78 weeks. All participants in Japan received eliglustat 50 mg once only on Day 1 and then eliglustat 50 mg BID from Day 2 titrated up to 100 mg BID (50 or 100 mg capsules) based on their individual PK data for up to 78 weeks.
Overall Number of Participants Analyzed 170
Mean (Standard Deviation)
Unit of Measure: platelets*10^9 /L
Baseline (n=170) 178.653  (92.732)
Week 26 (n=163) 180.021  (85.426)
Week 52 (n=74) 176.378  (79.880)
Week 78 (n=41) 168.720  (74.117)
18.Secondary Outcome
Title LIP: Mean Liver Volume at Baseline, Weeks 26, 52 and 78
Hide Description [Not Specified]
Time Frame Baseline, Week 26, Week 52, Week 78
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Analysis was performed on AT participants which included all participants who received at least 1 dose of eliglustat during lead in period. Here, 'n' signifies number of participants with available data at specified time points.
Arm/Group Title LIP: Eliglustat
Hide Arm/Group Description:
All participants (except in Japan) received eliglustat 50 mg BID on Day 1 titrated up to 100 mg BID (50 or 100 mg capsules) based on their individual PK data for up to 78 weeks. All participants in Japan received eliglustat 50 mg once only on Day 1 and then eliglustat 50 mg BID from Day 2 titrated up to 100 mg BID (50 or 100 mg capsules) based on their individual PK data for up to 78 weeks.
Overall Number of Participants Analyzed 170
Mean (Standard Deviation)
Unit of Measure: MN
Baseline (n=170) 1.044  (0.243)
Week 26 (n=149) 1.040  (0.229)
Week 52 (n=68) 1.059  (0.242)
Week 78 (n=39) 1.062  (0.236)
19.Secondary Outcome
Title LIP: Mean Spleen Volume at Baseline, Weeks 26, 52 and 78
Hide Description [Not Specified]
Time Frame Baseline, Week 26, Week 52, Week 78
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Analysis was performed on AT participants which included all participants who received at least 1 dose of eliglustat during lead in period. Here 'n' signifies number of participants with available data at specified time points.
Arm/Group Title LIP: Eliglustat
Hide Arm/Group Description:
All participants (except in Japan) received eliglustat 50 mg BID on Day 1 titrated up to 100 mg BID (50 or 100 mg capsules) based on their individual PK data for up to 78 weeks. All participants in Japan received eliglustat 50 mg once only on Day 1 and then eliglustat 50 mg BID from Day 2 titrated up to 100 mg BID (50 or 100 mg capsules) based on their individual PK data for up to 78 weeks.
Overall Number of Participants Analyzed 170
Mean (Standard Deviation)
Unit of Measure: MN
Baseline (n=119) 4.448  (2.314)
Week 26 (n=106) 3.840  (1.801)
Week 52 (n=52) 4.094  (1.767)
Week 78 (n=30) 4.088  (2.089)
20.Secondary Outcome
Title LIP: Mean Biomarker (Chitotriosidase) Value at Baseline, Weeks 26, 52, and 78
Hide Description Chitotriosidase biomarker was assayed from plasma.
Time Frame Baseline, Week 26, Week 52 and Week 78
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Analysis was performed on AT analysis set which included all participants who received at least 1 dose of eliglustat during LIP. Here ‘n’ signifies number of participants with available data at specified time points.
Arm/Group Title LIP: Eliglustat
Hide Arm/Group Description:
All participants (except in Japan) received eliglustat 50 mg, twice daily (BID) on Day 1 titrated up to 100 mg BID (50 or 100 mg capsules) based on their individual PK data for up to 78 weeks. All participants in Japan received eliglustat 50 mg once only on Day 1 and then eliglustat 50 mg BID from Day 2 titrated up to 100 mg BID (50 or 100 mg capsules) based on their individual PK data for up to 78 weeks.
Overall Number of Participants Analyzed 170
Mean (Standard Deviation)
Unit of Measure: nmol/hr/mL
Chitotriosidase: Baseline (n=170) 2437.92  (3291.25)
Chitotriosidase: week 26 (n=157) 1802.93  (2529.29)
Chitotriosidase: week 52 (n=72) 1755.70  (2649.14)
Chitotriosidase: week 78 (n=41) 1677.02  (2718.75)
21.Secondary Outcome
Title LIP: Mean Biomarker (GL-1 on DBS) Value at Baseline, Week 26, Week 52, and Week 78
Hide Description GL-1 on DBS biomarker was assayed from dried blood spot.
Time Frame Baseline, Week 26, Week 52 and Week 78
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Analysis was performed on AT analysis set which included all participants who received at least 1 dose of eliglustat during LIP. Here ‘n’ signifies number of participants with available data at specified time points.
Arm/Group Title LIP: Eliglustat
Hide Arm/Group Description:
All participants (except in Japan) received eliglustat 50 mg, twice daily (BID) on Day 1 titrated up to 100 mg BID (50 or 100 mg capsules) based on their individual PK data for up to 78 weeks. All participants in Japan received eliglustat 50 mg once only on Day 1 and then eliglustat 50 mg BID from Day 2 titrated up to 100 mg BID (50 or 100 mg capsules) based on their individual PK data for up to 78 weeks.
Overall Number of Participants Analyzed 170
Mean (Standard Deviation)
Unit of Measure: mcg/mL
GL-1 on DBS: Baseline (n=159) 4.358  (2.155)
GL-1 on DBS: Week 26 (n=144) 2.340  (0.868)
GL-1 on DBS: Week 52 (n=68) 2.279  (0.730)
GL-1 on DBS: Week 78 (n=39) 2.495  (1.500)
22.Secondary Outcome
Title LIP: Mean Biomarker (MIP1-beta) Value at Baseline, Week 78
Hide Description MIP1-beta biomarker was assayed from plasma.
Time Frame Baseline and Week 78
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Analysis was performed on AT analysis set which included all participants who received at least 1 dose of eliglustat during LIP. Here ‘n’ signifies number of participants with available data at specified time points.
Arm/Group Title LIP: Eliglustat
Hide Arm/Group Description:
All participants (except in Japan) received eliglustat 50 mg, twice daily (BID) on Day 1 titrated up to 100 mg BID (50 or 100 mg capsules) based on their individual PK data for up to 78 weeks. All participants in Japan received eliglustat 50 mg once only on Day 1 and then eliglustat 50 mg BID from Day 2 titrated up to 100 mg BID (50 or 100 mg capsules) based on their individual PK data for up to 78 weeks.
Overall Number of Participants Analyzed 170
Mean (Standard Deviation)
Unit of Measure: pg/mL
MIP-1beta: Baseline (n=170) 142.433  (125.961)
MIP-1beta: Week 78 (n=41) 132.180  (189.454)
23.Secondary Outcome
Title LIP: Number of Participants With Mobility Status (MS) at Baseline, Weeks 26, 52 and 78
Hide Description Mobility, i.e., ability to walk was assessed as a part of Gaucher disease assessment in participants. In this outcome, 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.
Time Frame Baseline, Week 26, Week 52, Week 78
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Analysis was performed on AT analysis set which included all participants who received at least 1 dose of eliglustat during LIP.
Arm/Group Title LIP: Eliglustat
Hide Arm/Group Description:
All participants (except in Japan) received eliglustat 50 mg, twice daily (BID) on Day 1 titrated up to 100 mg BID (50 or 100 mg capsules) based on their individual PK data for up to 78 weeks. All participants in Japan received eliglustat 50 mg once only on Day 1 and then eliglustat 50 mg BID from Day 2 titrated up to 100 mg BID (50 or 100 mg capsules) based on their individual PK data for up to 78 weeks.
Overall Number of Participants Analyzed 170
Measure Type: Number
Unit of Measure: participants
MS,Unrestricted: Baseline (n=163) 146
MS,Unrestricted: Week 26 (n=161) 153
MS,Unrestricted: Week 52 (n=72) 70
MS,Unrestricted: Week 78 (n= 41) 39
MS, Walks With Difficulty: Baseline (n=163) 12
MS, Walks With Difficulty: Week 26 (n=161) 6
MS, Walks With Difficulty: Week 52 (n=72) 1
MS, Walks With Difficulty: Week 78 (n=41) 2
MS, Walks With Orthopedic Aid: Baseline (n=163) 3
MS, Walks With Orthopedic Aid: Week 26 (n=161) 1
MS, Walks With Orthopedic Aid: Week 52 (n=72) 0
MS, Walks With Orthopedic Aid: Week 78 (n=41) 0
MS, Required wheelchair: Baseline (n=163) 2
MS, Required wheelchair: Week 26 (n=161) 1
MS, Required wheelchair: Week 52 (n=72) 1
MS, Required wheelchair: Week 78 (n=41) 0
MS, Bedridden: Baseline (n=163) 0
MS, Bedridden: Week 26 (n=161) 0
MS, Bedridden: Week 52 (n=72) 0
MS, Bedridden: Week 78 (n=41) 0
24.Secondary Outcome
Title LIP: Number of Participants With Bone Crises Assessment at Baseline, Weeks 26, 52 and 78
Hide Description Bone crises was assessed as a part of Gaucher disease assessment in participants. Acute, excruciating episodic bone pain is characteristic of Gaucher bone crises, which typically causes debilitation lasting several days or longer and requires treatment with immobilization, hydration, and opioid analgesics. Participants were categorized as 0= no bone crisis, 1= 1 bone crisis, 2= 2 bone crises, 6= 6 bone crises, and 24= 24 bone crises during the assessment period. In this outcome, number of participants with different bone crises levels at specified time points were reported.
Time Frame Baseline, Week 26, Week 52, Week 78
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Analysis was performed on AT analysis set which included all participants who received at least 1 dose of eliglustat during LIP.
Arm/Group Title LIP: Eliglustat
Hide Arm/Group Description:
All participants (except in Japan) received eliglustat 50 mg, twice daily (BID) on Day 1 titrated up to 100 mg BID (50 or 100 mg capsules) based on their individual PK data for up to 78 weeks. All participants in Japan received eliglustat 50 mg once only on Day 1 and then eliglustat 50 mg BID from Day 2 titrated up to 100 mg BID (50 or 100 mg capsules) based on their individual PK data for up to 78 weeks.
Overall Number of Participants Analyzed 170
Measure Type: Number
Unit of Measure: participants
Bone Crisis (0): Baseline (n=162) 151
Bone Crisis (0): Week 26 (n=162) 159
Bone Crisis (0): Week 52 (n=72) 72
Bone Crisis (0): Week 78 (n=41) 41
Bone Crisis (1): Baseline (n=162) 8
Bone Crisis (1): Week 26 (n=162) 3
Bone Crisis (1): Week 52 (n=72) 0
Bone Crisis (1): Week 78 (n=41) 0
Bone Crisis (2): Baseline (n=162) 1
Bone Crisis (2): Week 26 (n=162) 0
Bone Crisis (2): Week 52 (n=72) 0
Bone Crisis (2): Week 78 (n=41) 0
Bone Crisis (6): Baseline (n=162) 1
Bone Crisis (6): Week 26 (n=162) 0
Bone Crisis (6): Week 52 (n=72) 0
Bone Crisis (6): Week 78 (n=41) 0
Bone Crisis (24): Baseline (n=162) 1
Bone Crisis (24): Week 26 (n=162) 0
Bone Crisis (24): Week 52 (n=72) 0
Bone Crisis (24): Week 78 (n=41) 0
25.Secondary Outcome
Title LIP: Number of Participants With Bone Pain Levels During the Past 4 Weeks at Baseline, Weeks 26, 52 and 78
Hide Description 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. In this outcome, number of participants with different type of bone pain during the past 4 weeks at specified time points were reported.
Time Frame Baseline, Week 26, Week 52, Week 78
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Analysis was performed on AT analysis set which included all participants who received at least 1 dose of eliglustat during LIP.
Arm/Group Title LIP: Eliglustat
Hide Arm/Group Description:
All participants (except in Japan) received eliglustat 50 mg, twice daily (BID) on Day 1 titrated up to 100 mg BID (50 or 100 mg capsules) based on their individual PK data for up to 78 weeks. All participants in Japan received eliglustat 50 mg once only on Day 1 and then eliglustat 50 mg BID from Day 2 titrated up to 100 mg BID (50 or 100 mg capsules) based on their individual PK data for up to 78 weeks.
Overall Number of Participants Analyzed 170
Measure Type: Number
Unit of Measure: participants
None: Baseline (n=163) 112
None: Week 26 (n=161) 125
None: Week 52 (n=72) 62
None: Week 78 (n=41) 39
Very Mild: Baseline (n=163) 17
Very Mild: Week 26 (n=161) 14
Very Mild: Week 52 (n=72) 4
Very Mild: Week 78 (n=41) 1
Mild: Baseline (n=163) 22
Mild: Week 26 (n=161) 10
Mild: Week 52 (n=72) 3
Mild: Week 78 (n=41) 0
Moderate: Baseline (n=163) 8
Moderate: Week 26 (n=161) 10
Moderate: Week 52 (n=72) 3
Moderate: Week 78 (n=41) 1
Severe: Baseline (n=163) 4
Severe: Week 26 (n=161) 2
Severe: Week 52 (n=72) 0
Severe: Week 78 (n=41) 0
Extreme: Baseline (n=163) 0
Extreme: Week 26 (n=161) 0
Extreme: Week 52 (n=72) 0
Extreme: Week 78 (n=41) 0
26.Secondary Outcome
Title LTTP: Percentage of Participants Who Maintained a Stable Bone Criterion ,Hemoglobin Level, Platelet Count, Liver Volume and Spleen Volume at 1 Year and 2 Years
Hide Description Participant were considered as stable if they met the following criteria: hemoglobin level did not decrease >1.5 g/dL from baseline for PAP, platelet count does not decrease >25% below Baseline for PAP, liver volume does not increase >20% above Baseline for PAP, spleen volume does not increase >25% above Baseline for PAP. Baseline for PAP was defined as last available assessment prior to randomization.
Time Frame 1 Year, 2 Years
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Analysis was performed on intent to treat (ITT) population which included all participants who received at least 1 dose of eliglustat after randomization. Here ‘n’ signifies number of participants with available data at specified time points.
Arm/Group Title LTTP: Eliglustat
Hide Arm/Group Description:
All participants who entered PAP continued their blinded randomized treatment for first 4 weeks. Participants who at Week 52 of PAP maintained their therapeutic goals (defined as: no more than 2 bone crisis during PAP [with no more than 1 bone crisis during either first 6 months or later 6 months of PAP] and is free of other clinically symptomatic bone disease during PAP; hemoglobin level not decreased by >1.5 g/dL from Baseline for PAP [defined as last available assessment prior to randomization]; platelet count not decreased by >25% from Baseline for PAP; spleen volume not increased by 25% from Baseline for PAP; liver volume not increased by >20% from Baseline for PAP), continued into the LTTP and received their TDD of eliglustat (100 mg or 200 mg) QD till the end of the study. The participants who did not maintain all their therapeutic goals continued into the LTTP and received their TDD of eliglustat (100 mg or 200 mg) BID till the end of the study.
Overall Number of Participants Analyzed 121
Measure Type: Number
Number (95% Confidence Interval)
Unit of Measure: percentage of participants
Bone Criterion Stable at 1 year (n=104)
92.3
(85.4 to 96.6)
Bone Criterion Stable at 2 years (n=32)
84.4
(67.2 to 94.7)
Hemoglobin Level Stable at 1 year (n=104)
92.3
(85.4 to 96.6)
Hemoglobin Level Stable at 2 years (n=32)
81.3
(63.6 to 92.8)
Platelet Count Stable at 1 year (n=104)
93.3
(86.6 to 97.3)
Platelet Count Stable at 2 years (n=32)
84.4
(67.2 to 94.7)
Liver Volume Stable at 1 year (n=103)
93.2
(86.5 to 97.2)
Liver Volume Stable at 2 years (n=31)
83.9
(66.3 to 94.5)
Spleen Volume Stable at 1 year (n=72)
95.8
(88.3 to 99.1)
Spleen Volume Stable at 2 years (n=20)
95.0
(75.1 to 99.9)
27.Secondary Outcome
Title LTTP: Number of Participants With Mobility Status (MS) at Baseline, 1 Year and 2 Years
Hide Description Mobility, i.e., ability to walk was assessed as a part of Gaucher disease assessment in participants. In this outcome, 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.
Time Frame Baseline, 1 year, and 2 years
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
All participants who received at least one dose of eliglustat during the LTTP.
Arm/Group Title LTTP: Eliglustat
Hide Arm/Group Description:
All participants who entered PAP continued their blinded randomized treatment for first 4 weeks. Participants who at Week 52 of PAP maintained their therapeutic goals (defined as: no more than 2 bone crisis during PAP [with no more than 1 bone crisis during either first 6 months or later 6 months of PAP] and is free of other clinically symptomatic bone disease during PAP; hemoglobin level not decreased by >1.5 g/dL from Baseline for PAP [defined as last available assessment prior to randomization]; platelet count not decreased by >25% from Baseline for PAP; spleen volume not increased by 25% from Baseline for PAP; liver volume not increased by >20% from Baseline for PAP), continued into the LTTP and received their TDD of eliglustat (100 mg or 200 mg) QD till the end of the study. The participants who did not maintain all their therapeutic goals continued into the LTTP and received their TDD of eliglustat (100 mg or 200 mg) BID till the end of the study.
Overall Number of Participants Analyzed 121
Measure Type: Number
Unit of Measure: participants
MS, Unrestricted: Baseline (n=120) 111
MS, Unrestricted: 1 year (n=104) 97
MS, Unrestricted: 2 years (n=32) 28
MS, Walks with Difficulty: Baseline (n=120) 7
MS, Walks with Difficulty: 1 year (n=104) 4
Ms, Walks with Difficulty: 2 years (n=32) 2
MS, Walks with Orthopedic Aid: Baseline (n=120) 0
MS, Walks with Orthopedic Aid: 1 year (n=104) 1
MS, Walks with Orthopedic Aid: 2 years (n=32) 0
MS, Required Wheelchair: Baseline (n=120) 2
MS, Required Wheelchair: 1 year (n=104) 2
MS, Required Wheelchair: 2 years (n=32) 2
MS, Bedridden: Baseline (n=120) 0
MS, Bedridden: 1 year (n=104) 0
MS, Bedridden: 2 years (n=32) 0
28.Secondary Outcome
Title LTTP: Number of Participants With Bone Crises Assessment at Baseline, 1 Year and 2 Years
Hide Description Bone crises was assessed as a part of Gaucher disease assessment in participants. Acute, excruciating episodic bone pain is characteristic of Gaucher bone crises, which typically causes debilitation lasting several days or longer and requires treatment with immobilization, hydration, and opioid analgesics. Participants were categorized as 0= no bone crises, 1= 1 bone crisis during the assessment period. In this outcome, number of participants with different bone crises levels at specified time points were reported.
Time Frame Baseline, 1 year and 2 years
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
All participants who received at least one dose of eliglustat during the LTTP.
Arm/Group Title LTTP: Eliglustat
Hide Arm/Group Description:
All participants who entered PAP continued their blinded randomized treatment for first 4 weeks. Participants who at Week 52 of PAP maintained their therapeutic goals (defined as: no more than 2 bone crisis during PAP [with no more than 1 bone crisis during either first 6 months or later 6 months of PAP] and is free of other clinically symptomatic bone disease during PAP; hemoglobin level not decreased by >1.5 g/dL from Baseline for PAP [defined as last available assessment prior to randomization]; platelet count not decreased by >25% from Baseline for PAP; spleen volume not increased by 25% from Baseline for PAP; liver volume not increased by >20% from Baseline for PAP), continued into the LTTP and received their TDD of eliglustat (100 mg or 200 mg) QD till the end of the study. The participants who did not maintain all their therapeutic goals continued into the LTTP and received their TDD of eliglustat (100 mg or 200 mg) BID till the end of the study.
Overall Number of Participants Analyzed 121
Measure Type: Number
Unit of Measure: participants
Bone Crisis (0): Baseline (n=120) 119
Bone Crisis (0): 1 year (n=104) 104
Bone Crisis (0): 2 years (n=32) 32
Bone Crisis (1): Baseline (n=120) 1
Bone Crisis (1): 1 year (n=104) 0
Bone Crisis (1): 2 years (n=32) 0
29.Secondary Outcome
Title LTTP: Number of Participants With Bone Pain Levels During the Past 4 Weeks at Baseline, 1 Year, and 2 Years
Hide Description 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. In this outcome, number of participants with different level of bone pain during the past 4 weeks at specified time points were reported.
Time Frame Baseline, 1 year and 2 years
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
All participants who received at least one dose of eliglustat during the LTTP.
Arm/Group Title LTTP: Eliglustat
Hide Arm/Group Description:
All participants who entered PAP continued their blinded randomized treatment for first 4 weeks. Participants who at Week 52 of PAP maintained their therapeutic goals (defined as: no more than 2 bone crisis during PAP [with no more than 1 bone crisis during either first 6 months or later 6 months of PAP] and is free of other clinically symptomatic bone disease during PAP; hemoglobin level not decreased by >1.5 g/dL from Baseline for PAP [defined as last available assessment prior to randomization]; platelet count not decreased by >25% from Baseline for PAP; spleen volume not increased by 25% from Baseline for PAP; liver volume not increased by >20% from Baseline for PAP), continued into the LTTP and received their TDD of eliglustat (100 mg or 200 mg) QD till the end of the study. The participants who did not maintain all their therapeutic goals continued into the LTTP and received their TDD of eliglustat (100 mg or 200 mg) BID till the end of the study.
Overall Number of Participants Analyzed 121
Measure Type: Number
Unit of Measure: participants
None: Baseline (n=120) 91
None: 1 year (n=104) 83
None: 2 years (n=32) 32
Very Mild: Baseline (n=120) 12
Very Mild: 1 year (n=104) 9
Very Mild: 2 years (n=32) 0
Mild: Baseline (n=120) 8
Mild: 1 year (n=104) 10
Mild: 2 years (n=32) 0
Moderate: Baseline (n=120) 7
Moderate: 1 year (n=104) 1
Moderate: 2 years (n=32) 0
Severe: Baseline (n=120) 2
Severe: 1 year (n=104) 0
Severe: 2 years (n=32) 0
Extreme: Baseline (n=120) 0
Extreme: 1 year (n=104) 1
Extreme: 2 years (n=32) 0
30.Secondary Outcome
Title LTTP: Bone Mineral Density (BMD) at Baseline, 1 Year, and 2 Years
Hide Description BMD measurements of the spine and bilateral femur were acquired by DXA scan.
Time Frame Baseline, 1 year, and 2 years
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
All participants who received at least one dose of eliglustat during the LTTP.
Arm/Group Title LTTP: Eliglustat
Hide Arm/Group Description:
All participants who entered PAP continued their blinded randomized treatment for first 4 weeks. Participants who at Week 52 of PAP maintained their therapeutic goals (defined as: no more than 2 bone crisis during PAP [with no more than 1 bone crisis during either first 6 months or later 6 months of PAP] and is free of other clinically symptomatic bone disease during PAP; hemoglobin level not decreased by >1.5 g/dL from Baseline for PAP [defined as last available assessment prior to randomization]; platelet count not decreased by >25% from Baseline for PAP; spleen volume not increased by 25% from Baseline for PAP; liver volume not increased by >20% from Baseline for PAP), continued into the LTTP and received their TDD of eliglustat (100 mg or 200 mg) QD till the end of the study. The participants who did not maintain all their therapeutic goals continued into the LTTP and received their TDD of eliglustat (100 mg or 200 mg) BID till the end of the study.
Overall Number of Participants Analyzed 121
Mean (Standard Deviation)
Unit of Measure: g/cm^2
Lumbar Spine: Baseline (n=113) 1.087  (0.182)
Lumbar Spine: 1 year (n=101) 1.083  (0.183)
Lumbar Spine: 2 years (n=26) 1.082  (0.190)
Left Femur: Baseline (n=107) 0.986  (0.205)
Left Femur: 1 year (n=95) 0.994  (0.226)
Left Femur: 2 years (n=22) 0.950  (0.220)
Right Femur: Baseline (n=103) 0.983  (0.201)
Right Femur: 1 year (n=91) 0.979  (0.202)
Right Femur: 2 years (n=21) 0.908  (0.131)
31.Secondary Outcome
Title LTTP: Total T-Scores for BMD at Baseline, 1 Year, and 2 Years
Hide Description 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).
Time Frame Baseline, 1 year, and 2 years
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
All participants who received at least one dose of eliglustat during the LTTP.
Arm/Group Title LTTP: Eliglustat
Hide Arm/Group Description:
All participants who entered PAP continued their blinded randomized treatment for first 4 weeks. Participants who at Week 52 of PAP maintained their therapeutic goals (defined as: no more than 2 bone crisis during PAP [with no more than 1 bone crisis during either first 6 months or later 6 months of PAP] and is free of other clinically symptomatic bone disease during PAP; hemoglobin level not decreased by >1.5 g/dL from Baseline for PAP [defined as last available assessment prior to randomization]; platelet count not decreased by >25% from Baseline for PAP; spleen volume not increased by 25% from Baseline for PAP; liver volume not increased by >20% from Baseline for PAP), continued into the LTTP and received their TDD of eliglustat (100 mg or 200 mg) QD till the end of the study. The participants who did not maintain all their therapeutic goals continued into the LTTP and received their TDD of eliglustat (100 mg or 200 mg) BID till the end of the study.
Overall Number of Participants Analyzed 121
Mean (Standard Deviation)
Unit of Measure: T-score
Lumbar Spine T-Score: Baseline (n=110) -0.674  (1.383)
Lumbar Spine T-Score: 1 year (n=98) -0.718  (1.394)
Lumbar Spine T-Score: 2 years (n=26) -0.750  (1.370)
Left Femur T-Score: Baseline (n=103) -0.421  (1.377)
Left Femur T-Score: 1 year (n=91) -0.382  (1.551)
Left Femur T-Score: 2 years (n=22) -0.682  (1.532)
Right Femur T-Score: Baseline (n=99) -0.461  (1.360)
Right Femur T-Score: 1 year (n=87) -0.500  (1.381)
Right Femur T-Score: 2 years (n=21) -1.005  (0.931)
32.Secondary Outcome
Title LTTP: Total Z-scores for BMD at Baseline, 1 Year, and 2 Years
Hide Description 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).
Time Frame Baseline, 1 year, and 2 years
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
All participants who received at least one dose of eliglustat during the LTTP.
Arm/Group Title LTTP: Eliglustat
Hide Arm/Group Description:
All participants who entered PAP continued their blinded randomized treatment for first 4 weeks. Participants who at Week 52 of PAP maintained their therapeutic goals (defined as: no more than 2 bone crisis during PAP [with no more than 1 bone crisis during either first 6 months or later 6 months of PAP] and is free of other clinically symptomatic bone disease during PAP; hemoglobin level not decreased by >1.5 g/dL from Baseline for PAP [defined as last available assessment prior to randomization]; platelet count not decreased by >25% from Baseline for PAP; spleen volume not increased by 25% from Baseline for PAP; liver volume not increased by >20% from Baseline for PAP), continued into the LTTP and received their TDD of eliglustat (100 mg or 200 mg) QD till the end of the study. The participants who did not maintain all their therapeutic goals continued into the LTTP and received their TDD of eliglustat (100 mg or 200 mg) BID till the end of the study.
Overall Number of Participants Analyzed 121
Mean (Standard Deviation)
Unit of Measure: Z-score
Lumbar Spine Z-Score: Baseline (n=113) -0.460  (1.376)
Lumbar Spine Z-Score: 1 year (n=101) -0.512  (1.368)
Lumbar Spine Z-Score: 2 years (n=26) -0.385  (1.316)
Left Femur Z-Score: Baseline (n=107) -0.164  (1.277)
Left Femur Z-Score: 1 year (n=95) -0.132  (1.459)
Left Femur Z-Score: 2 years (n=22) -0.264  (1.525)
Right Femur Z-Score: Baseline (n=103) -0.214  (1.227)
Right Femur Z-Score: 1 year (n=91) -0.262  (1.270)
Right Femur Z-Score: 2 years (n=21) -0.605  (0.957)
33.Secondary Outcome
Title LTTP: Total Bone Marrow Burden Score (BMB) at Baseline, 1 Year, and 2 Years
Hide Description BMB Score was measured using MRI, range 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 score was calculated as the sum of scores for femur and lumbar spine regions which ranged from 0 (no abnormalities) -16 (severe disease) points. A higher BMB score signified more severe bone marrow involvement.
Time Frame Baseline, 1 year, and 2 years
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
All participants who received at least one dose of eliglustat during the LTTP.
Arm/Group Title LTTP: Eliglustat
Hide Arm/Group Description:
All participants who entered PAP continued their blinded randomized treatment for first 4 weeks. Participants who at Week 52 of PAP maintained their therapeutic goals (defined as: no more than 2 bone crisis during PAP [with no more than 1 bone crisis during either first 6 months or later 6 months of PAP] and is free of other clinically symptomatic bone disease during PAP; hemoglobin level not decreased by >1.5 g/dL from Baseline for PAP [defined as last available assessment prior to randomization]; platelet count not decreased by >25% from Baseline for PAP; spleen volume not increased by 25% from Baseline for PAP; liver volume not increased by >20% from Baseline for PAP), continued into the LTTP and received their TDD of eliglustat (100 mg or 200 mg) QD till the end of the study. The participants who did not maintain all their therapeutic goals continued into the LTTP and received their TDD of eliglustat (100 mg or 200 mg) BID till the end of the study.
Overall Number of Participants Analyzed 121
Mean (Standard Deviation)
Unit of Measure: BMB Score
BMB Score: Baseline (n=115) 8.164  (2.646)
BMB Score: 1 year (n=26) 7.853  (2.497)
BMB Score: 2 years (n=17) 8.059  (1.918)
34.Secondary Outcome
Title LTTP: Mean Biomarker (Chitotriosidase) Value at Baseline, 1 Year, and 2 Years
Hide Description Chitotriosidase biomarker was assayed from plasma.
Time Frame Baseline, 1 year, and 2 years
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
All participants who received at least one dose of eliglustat during the LTTP.
Arm/Group Title LTTP: Eliglustat
Hide Arm/Group Description:
All participants who entered PAP continued their blinded randomized treatment for first 4 weeks. Participants who at Week 52 of PAP maintained their therapeutic goals (defined as: no more than 2 bone crisis during PAP [with no more than 1 bone crisis during either first 6 months or later 6 months of PAP] and is free of other clinically symptomatic bone disease during PAP; hemoglobin level not decreased by >1.5 g/dL from Baseline for PAP [defined as last available assessment prior to randomization]; platelet count not decreased by >25% from Baseline for PAP; spleen volume not increased by 25% from Baseline for PAP; liver volume not increased by >20% from Baseline for PAP), continued into the LTTP and received their TDD of eliglustat (100 mg or 200 mg) QD till the end of the study. The participants who did not maintain all their therapeutic goals continued into the LTTP and received their TDD of eliglustat (100 mg or 200 mg) BID till the end of the study.
Overall Number of Participants Analyzed 121
Mean (Standard Deviation)
Unit of Measure: nmol/hr/mL
Chitotriosidase: Baseline (n=118) 1188.983  (1857.521)
Chitotriosidase: 1 year (n=97)) 1221.753  (2072.446)
Chitotriosidase: 2 years (n=31) 598.161  (1463.603)
35.Secondary Outcome
Title LTTP: Mean Biomarker (GL-1 on DBS) Value at Baseline, 1 Year, and 2 Years
Hide Description GL-1 on DBS biomarker was assayed from dried blood spot.
Time Frame Baseline, 1 year, and 2 years
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
All participants who received at least one dose of eliglustat during the LTTP.
Arm/Group Title LTTP: Eliglustat
Hide Arm/Group Description:
All participants who entered PAP continued their blinded randomized treatment for first 4 weeks. Participants who at Week 52 of PAP maintained their therapeutic goals (defined as: no more than 2 bone crisis during PAP [with no more than 1 bone crisis during either first 6 months or later 6 months of PAP] and is free of other clinically symptomatic bone disease during PAP; hemoglobin level not decreased by >1.5 g/dL from Baseline for PAP [defined as last available assessment prior to randomization]; platelet count not decreased by >25% from Baseline for PAP; spleen volume not increased by 25% from Baseline for PAP; liver volume not increased by >20% from Baseline for PAP), continued into the LTTP and received their TDD of eliglustat (100 mg or 200 mg) QD till the end of the study. The participants who did not maintain all their therapeutic goals continued into the LTTP and received their TDD of eliglustat (100 mg or 200 mg) BID till the end of the study.
Overall Number of Participants Analyzed 121
Mean (Standard Deviation)
Unit of Measure: mcg/mL
GL-1 on DBS: Baseline (n=114) 2.725  (1.350)
GL-1 on DBS: 1 year (n=98) 2.500  (1.031)
GL-1 on DBS: 2 years (n=29) 2.238  (0.658)
36.Secondary Outcome
Title LTTP: Mean Biomarker (MIP1-beta) Value at Baseline, 1 Year, and 2 Years
Hide Description MIP1-beta biomarker was assayed from plasma.
Time Frame Baseline, 1 year, and 2 years
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
All participants who received at least one dose of eliglustat during the LTTP.
Arm/Group Title LTTP: Eliglustat
Hide Arm/Group Description:
All participants who entered PAP continued their blinded randomized treatment for first 4 weeks. Participants who at Week 52 of PAP maintained their therapeutic goals (defined as: no more than 2 bone crisis during PAP [with no more than 1 bone crisis during either first 6 months or later 6 months of PAP] and is free of other clinically symptomatic bone disease during PAP; hemoglobin level not decreased by >1.5 g/dL from Baseline for PAP [defined as last available assessment prior to randomization]; platelet count not decreased by >25% from Baseline for PAP; spleen volume not increased by 25% from Baseline for PAP; liver volume not increased by >20% from Baseline for PAP), continued into the LTTP and received their TDD of eliglustat (100 mg or 200 mg) QD till the end of the study. The participants who did not maintain all their therapeutic goals continued into the LTTP and received their TDD of eliglustat (100 mg or 200 mg) BID till the end of the study.
Overall Number of Participants Analyzed 121
Mean (Standard Deviation)
Unit of Measure: pg/mL
MIP-1beta: Baseline (n=114) 97.857  (125.857)
MIP-1beta: 1 year (n=94) 90.398  (90.549)
MIP-1beta: 2 years (n=31) 68.445  (64.774)
Time Frame [Not Specified]
Adverse Event Reporting Description Adverse events data was planned to be reported for overall population.
 
Arm/Group Title Eliglustat
Hide Arm/Group Description All participants who received eliglustat at the TDD of 100 mg or 200 mg in the LIP, PAP, LTTP or ETP.
All-Cause Mortality
Eliglustat
Affected / at Risk (%)
Total   --/-- 
Show Serious Adverse Events Hide Serious Adverse Events
Eliglustat
Affected / at Risk (%)
Total   40/170 (23.53%) 
Cardiac disorders   
Angina pectoris  1  2/170 (1.18%) 
Arrhythmia  1  1/170 (0.59%) 
Cardiac arrest  1  1/170 (0.59%) 
Gastrointestinal disorders   
Abdominal wall haematoma  1  1/170 (0.59%) 
Mallory-Weiss syndrome  1  1/170 (0.59%) 
General disorders   
Device dislocation  1  1/170 (0.59%) 
Disuse syndrome  1  1/170 (0.59%) 
Hernia  1  1/170 (0.59%) 
Impaired healing  1  1/170 (0.59%) 
Medical device pain  1  1/170 (0.59%) 
Hepatobiliary disorders   
Cholecystitis  1  1/170 (0.59%) 
Cholecystitis chronic  1  1/170 (0.59%) 
Infections and infestations   
Acute hepatitis B  1  1/170 (0.59%) 
Appendicitis  1  1/170 (0.59%) 
Hepatitis A  1  1/170 (0.59%) 
Staphylococcal sepsis  1  1/170 (0.59%) 
Injury, poisoning and procedural complications   
Fall  1  1/170 (0.59%) 
Femoral neck fracture  1  2/170 (1.18%) 
Femur fracture  1  1/170 (0.59%) 
Forearm fracture  1  1/170 (0.59%) 
Incisional hernia  1  1/170 (0.59%) 
Injury  1  1/170 (0.59%) 
Radius fracture  1  1/170 (0.59%) 
Spinal fracture  1  1/170 (0.59%) 
Investigations   
Hepatic enzyme increased  1  1/170 (0.59%) 
Musculoskeletal and connective tissue disorders   
Arthralgia  1  2/170 (1.18%) 
Arthritis  1  1/170 (0.59%) 
Joint range of motion decreased  1  1/170 (0.59%) 
Muscular weakness  1  1/170 (0.59%) 
Osteoarthritis  1  3/170 (1.76%) 
Osteonecrosis  1  1/170 (0.59%) 
Neoplasms benign, malignant and unspecified (incl cysts and polyps)   
Lipoma  1  1/170 (0.59%) 
Papillary thyroid cancer  1  1/170 (0.59%) 
Nervous system disorders   
Dizziness  1  1/170 (0.59%) 
Epilepsy  1  2/170 (1.18%) 
Ischaemic stroke  1  1/170 (0.59%) 
Presyncope  1  1/170 (0.59%) 
Seizure  1  1/170 (0.59%) 
Syncope  1  3/170 (1.76%) 
Pregnancy, puerperium and perinatal conditions   
Ectopic pregnancy  1  1/170 (0.59%) 
Psychiatric disorders   
Conversion disorder  1  1/170 (0.59%) 
Major depression  1  1/170 (0.59%) 
Renal and urinary disorders   
Calculus urinary  1  1/170 (0.59%) 
Reproductive system and breast disorders   
Uterine polyp  1  1/170 (0.59%) 
Vascular disorders   
Aortic aneurysm  1  1/170 (0.59%) 
Haemorrhage  1  1/170 (0.59%) 
Hypertension  1  2/170 (1.18%) 
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDra 18.0
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Eliglustat
Affected / at Risk (%)
Total   144/170 (84.71%) 
Blood and lymphatic system disorders   
Thrombocytopenia  1  9/170 (5.29%) 
Cardiac disorders   
Palpitations  1  11/170 (6.47%) 
Gastrointestinal disorders   
Abdominal distension  1  9/170 (5.29%) 
Abdominal pain  1  15/170 (8.82%) 
Abdominal pain upper  1  26/170 (15.29%) 
Constipation  1  16/170 (9.41%) 
Diarrhoea  1  24/170 (14.12%) 
Dyspepsia  1  19/170 (11.18%) 
Gastrooesophageal reflux disease  1  9/170 (5.29%) 
Nausea  1  18/170 (10.59%) 
Vomiting  1  14/170 (8.24%) 
General disorders   
Fatigue  1  17/170 (10.00%) 
Pyrexia  1  16/170 (9.41%) 
Hepatobiliary disorders   
Hepatomegaly  1  9/170 (5.29%) 
Infections and infestations   
Gastroenteritis  1  15/170 (8.82%) 
Influenza  1  23/170 (13.53%) 
Nasopharyngitis  1  52/170 (30.59%) 
Upper respiratory tract infection  1  22/170 (12.94%) 
Urinary tract infection  1  14/170 (8.24%) 
Viral infection  1  10/170 (5.88%) 
Musculoskeletal and connective tissue disorders   
Arthralgia  1  30/170 (17.65%) 
Back pain  1  29/170 (17.06%) 
Musculoskeletal pain  1  10/170 (5.88%) 
Myalgia  1  11/170 (6.47%) 
Pain in extremity  1  24/170 (14.12%) 
Nervous system disorders   
Dizziness  1  28/170 (16.47%) 
Headache  1  37/170 (21.76%) 
Psychiatric disorders   
Depression  1  10/170 (5.88%) 
Respiratory, thoracic and mediastinal disorders   
Cough  1  21/170 (12.35%) 
Epistaxis  1  13/170 (7.65%) 
Oropharyngeal pain  1  18/170 (10.59%) 
Vascular disorders   
Hypertension  1  9/170 (5.29%) 
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDra 18.0
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
If no publication has occurred within 12 months of the completion of the study, the Investigator shall have the right to publish/present independently the results of the study. The Investigator shall provide the Sponsor with a copy of any such presentation/publication for comment at least 30 days before any presentation/submission for publication. If requested by the Sponsor, any presentation/submission shall be delayed up to 90 days, to allow the Sponsor to preserve its proprietary rights.
Results Point of Contact
Name/Title: Trial Transparency Team
Organization: Sanofi
Responsible Party: Sanofi ( Genzyme, a Sanofi Company )
ClinicalTrials.gov Identifier: NCT01074944     History of Changes
Other Study ID Numbers: GZGD03109
2009-015811-42 ( EudraCT Number )
EFC12818 ( Other Identifier: Sanofi )
First Submitted: February 23, 2010
First Posted: February 24, 2010
Results First Submitted: October 7, 2016
Results First Posted: December 2, 2016
Last Update Posted: February 6, 2017