Trial Outcomes & Findings for A Study of Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease Who Have Reached Therapeutic Goals With Enzyme Replacement Therapy (ENCORE) (NCT NCT00943111)

NCT ID: NCT00943111

Last Updated: 2016-11-25

Results Overview

For a participant to be classified as stable, the participant must have remained stable in hematological parameters (hemoglobin levels and platelet counts) and organ volumes (spleen, when applicable, and liver volumes in multiples of normal \[MN\]). Stable hematological parameters were defined as hemoglobin level did not decrease more than (\>) 1.5 gram per deciliter (g/dL) from baseline and platelet count did not decrease \>25% from baseline. Stable organ volumes were defined as spleen volume (in MN) did not increase \>25% from baseline, if applicable, and liver volume (in MN) did not increase \>20% from baseline.

• Recruitment status: COMPLETED
• Study phase: PHASE3
• Target enrollment: 160 participants
• Primary outcome timeframe: Baseline up to Week 52
• Results posted on: 2016-11-25

Participant Flow

A total of 209 participants were screened, of which 46 participants were screen failure and 3 participants withdrew prior to randomization. A total of 160 participants were enrolled in this study.

All enrolled participants received eliglustat or imiglucerase in 52 week primary analysis period (PAP). After 52-weeks PAP, all participants who remained on-study, received eliglustat in the long-term treatment period (LTTP) for up to 5 years.

Participant milestones

Measure	Eliglustat: PAP Eliglustat tartrate (Genz-112638) capsule 50 milligram (mg) twice daily (BID) orally from Day 1 to Week 4 followed by eliglustat tartrate 50 mg or 100 mg capsule BID up to Week 8, and then eliglustat tartrate 50 mg or 100 mg or 150 mg capsule BID up to Week 52. The dose adjustments after Week 4 and Week 8 were based on Genz-99067 (active moiety of eliglustat tartrate in plasma) trough plasma concentrations. If Genz-99067 trough plasma concentration was less than \[\<\] 5 nanogram per milliliter \[ng/mL\] the next higher dose was administered whereas if the Genz-99067 trough plasma concentration was greater than or equal to \[\>=\] 5 ng/mL the same dose was continued. The pharmacokinetic (PK) assessment at Week 2 and Week 6 were used for dose adjustment after Week 4 and Week 8, respectively.	Imiglucerase: PAP Imiglucerase (Cerezyme®) intravenous infusion every other week (q2w) up to Week 52 in doses equivalent to participant's past enzyme replacement therapy (ERT) dose prior to any unanticipated treatment interruption, dose reduction, or regimen change.	Eliglustat: LTTP Participants from both the arms of PAP who completed PAP were included in this arm of LTTP. Participants originally randomized to eliglustat in PAP continued to receive eliglustat dose, based on their Genz 99067 plasma trough concentration at Week 6. Participants originally randomized to imiglucerase received eliglustat tartrate capsule 50 mg BID orally from Week 52+1 Day to Week 56 followed by eliglustat tartrate 50 mg or 100 mg capsule BID up to Week 60, and then eliglustat tartrate 50 mg or 100 mg or 150 mg capsule BID up to 5 years. The dose adjustments after Week 56 and Week 60 were based on Genz-99067 (active moiety of eliglustat tartrate in plasma) trough plasma concentrations. If Genz-99067 trough plasma concentration was \<5 ng/mL next higher dose was administered whereas if the Genz-99067 trough plasma concentration was \>=5 ng/mL the same dose was continued. PK assessment at Week 54 and Week 58 were used for dose adjustment after Week 56 and Week 60, respectively.
52-Weeks Primary Analysis Period STARTED	106	54	0
52-Weeks Primary Analysis Period Treated	106	53	0
52-Weeks Primary Analysis Period COMPLETED	104	52	0
52-Weeks Primary Analysis Period NOT COMPLETED	2	2	0
5 Years Long-term Treatment Period STARTED	0	0	152
5 Years Long-term Treatment Period COMPLETED	0	0	77
5 Years Long-term Treatment Period NOT COMPLETED	0	0	75

Reasons for withdrawal

Measure	Eliglustat: PAP Eliglustat tartrate (Genz-112638) capsule 50 milligram (mg) twice daily (BID) orally from Day 1 to Week 4 followed by eliglustat tartrate 50 mg or 100 mg capsule BID up to Week 8, and then eliglustat tartrate 50 mg or 100 mg or 150 mg capsule BID up to Week 52. The dose adjustments after Week 4 and Week 8 were based on Genz-99067 (active moiety of eliglustat tartrate in plasma) trough plasma concentrations. If Genz-99067 trough plasma concentration was less than \[\<\] 5 nanogram per milliliter \[ng/mL\] the next higher dose was administered whereas if the Genz-99067 trough plasma concentration was greater than or equal to \[\>=\] 5 ng/mL the same dose was continued. The pharmacokinetic (PK) assessment at Week 2 and Week 6 were used for dose adjustment after Week 4 and Week 8, respectively.	Imiglucerase: PAP Imiglucerase (Cerezyme®) intravenous infusion every other week (q2w) up to Week 52 in doses equivalent to participant's past enzyme replacement therapy (ERT) dose prior to any unanticipated treatment interruption, dose reduction, or regimen change.	Eliglustat: LTTP Participants from both the arms of PAP who completed PAP were included in this arm of LTTP. Participants originally randomized to eliglustat in PAP continued to receive eliglustat dose, based on their Genz 99067 plasma trough concentration at Week 6. Participants originally randomized to imiglucerase received eliglustat tartrate capsule 50 mg BID orally from Week 52+1 Day to Week 56 followed by eliglustat tartrate 50 mg or 100 mg capsule BID up to Week 60, and then eliglustat tartrate 50 mg or 100 mg or 150 mg capsule BID up to 5 years. The dose adjustments after Week 56 and Week 60 were based on Genz-99067 (active moiety of eliglustat tartrate in plasma) trough plasma concentrations. If Genz-99067 trough plasma concentration was \<5 ng/mL next higher dose was administered whereas if the Genz-99067 trough plasma concentration was \>=5 ng/mL the same dose was continued. PK assessment at Week 54 and Week 58 were used for dose adjustment after Week 56 and Week 60, respectively.
52-Weeks Primary Analysis Period Adverse Event	2	1	0
52-Weeks Primary Analysis Period Non-Compliance with Study Drug	0	1	0
5 Years Long-term Treatment Period Transition to commercial eliglustat	0	0	51
5 Years Long-term Treatment Period Adverse Event	0	0	9
5 Years Long-term Treatment Period Withdrawal by Subject	0	0	8
5 Years Long-term Treatment Period Pregnancy	0	0	4
5 Years Long-term Treatment Period Lost to Follow-up	0	0	1
5 Years Long-term Treatment Period Other than specified above	0	0	2

Baseline Characteristics

A Study of Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease Who Have Reached Therapeutic Goals With Enzyme Replacement Therapy (ENCORE)

Baseline characteristics by cohort

Measure	Eliglustat: PAP n=106 Participants Eliglustat tartrate 50 mg capsule BID orally from Day 1 to Week 4, followed by eliglustat tartrate 50, 100 or 150 mg capsule BID orally up to Week 52.	Imiglucerase: PAP n=53 Participants Imiglucerase intravenous infusion q2w up to Week 52 in doses equivalent to participant's past ERT dose prior to any unanticipated treatment interruption, dose reduction, or regimen change.	Total n=159 Participants Total of all reporting groups
Age, Continuous	37.6 years STANDARD_DEVIATION 14.17 • n=5 Participants	37.5 years STANDARD_DEVIATION 14.92 • n=7 Participants	37.5 years STANDARD_DEVIATION 14.37 • n=5 Participants
Sex: Female, Male Female	59 Participants n=5 Participants	28 Participants n=7 Participants	87 Participants n=5 Participants
Sex: Female, Male Male	47 Participants n=5 Participants	25 Participants n=7 Participants	72 Participants n=5 Participants
Race/Ethnicity, Customized Race: White	98 participants n=5 Participants	48 participants n=7 Participants	146 participants n=5 Participants
Race/Ethnicity, Customized Race: Black or African American	6 participants n=5 Participants	4 participants n=7 Participants	10 participants n=5 Participants
Race/Ethnicity, Customized Race: Asian	1 participants n=5 Participants	1 participants n=7 Participants	2 participants n=5 Participants
Race/Ethnicity, Customized Race: White/American Indian	1 participants n=5 Participants	0 participants n=7 Participants	1 participants n=5 Participants
Race/Ethnicity, Customized Ethnicity: Hispanic or Latino	42 participants n=5 Participants	19 participants n=7 Participants	61 participants n=5 Participants
Race/Ethnicity, Customized Ethnicity: Not Hispanic or Latino	64 participants n=5 Participants	34 participants n=7 Participants	98 participants n=5 Participants
Body Mass Index (BMI)	25.2 kilogram per square meter (kg/m^2) STANDARD_DEVIATION 5.24 • n=5 Participants	24.5 kilogram per square meter (kg/m^2) STANDARD_DEVIATION 4.51 • n=7 Participants	24.9 kilogram per square meter (kg/m^2) STANDARD_DEVIATION 5.01 • n=5 Participants
Weight	70.8 kilogram (kg) STANDARD_DEVIATION 16.82 • n=5 Participants	67.8 kilogram (kg) STANDARD_DEVIATION 14.44 • n=7 Participants	69.8 kilogram (kg) STANDARD_DEVIATION 16.08 • n=5 Participants
Height	167.6 centimeter (cm) STANDARD_DEVIATION 9.92 • n=5 Participants	166.2 centimeter (cm) STANDARD_DEVIATION 9.56 • n=7 Participants	167.1 centimeter (cm) STANDARD_DEVIATION 9.79 • n=5 Participants

PRIMARY outcome

Timeframe: Baseline up to Week 52

Population: Per protocol population for PAP included participants who were at least 80% compliant with treatment during PAP, had no major protocol deviations, and did not exhibit hematological decline as a result of medically determined etiologies other than Gaucher disease.

For a participant to be classified as stable, the participant must have remained stable in hematological parameters (hemoglobin levels and platelet counts) and organ volumes (spleen, when applicable, and liver volumes in multiples of normal [MN]). Stable hematological parameters were defined as hemoglobin level did not decrease more than (>) 1.5 gram per deciliter (g/dL) from baseline and platelet count did not decrease >25% from baseline. Stable organ volumes were defined as spleen volume (in MN) did not increase >25% from baseline, if applicable, and liver volume (in MN) did not increase >20% from baseline.

Outcome measures

Measure	Eliglustat: PAP n=99 Participants Eliglustat tartrate 50 mg capsule BID orally from Day 1 to Week 4, followed by eliglustat tartrate 50, 100 or 150 mg capsule BID orally up to Week 52.	Imiglucerase: PAP n=47 Participants Imiglucerase intravenous infusion q2w up to Week 52 in doses equivalent to participant's past ERT dose prior to any unanticipated treatment interruption, dose reduction, or regimen change.
Percentage of Participants Who Remained Stable for 52 Weeks During the Primary Analysis Period	84.8 percentage of participants Interval 76.2 to 91.3	93.6 percentage of participants Interval 82.5 to 98.7

PRIMARY outcome

Timeframe: Week 52 up to week 208

Population: FAS population for LTTP: included all participants who received at least 1 dose of eliglustat in the extension study period. Number of participants analyzed=participants at risk at specified time-points. Here 'n' signifies number of participants with available data for specified time-points.

For a participant to be classified as stable, the participant must have remained stable in hematological parameters (hemoglobin levels and platelet counts) and organ volumes (spleen, when applicable, and liver volumes in MN). Stable hematological parameters were defined as hemoglobin level did not decrease >1.5 g/dL from baseline and platelet count did not decrease >25% from baseline. Stable organ volumes were defined as spleen volume (in MN) did not increase >25% from baseline, if applicable, and liver volume did not increase >20% from baseline.

Outcome measures

Measure	Eliglustat: PAP n=152 Participants Eliglustat tartrate 50 mg capsule BID orally from Day 1 to Week 4, followed by eliglustat tartrate 50, 100 or 150 mg capsule BID orally up to Week 52.	Imiglucerase: PAP Imiglucerase intravenous infusion q2w up to Week 52 in doses equivalent to participant's past ERT dose prior to any unanticipated treatment interruption, dose reduction, or regimen change.
Percentage of Participants Who Remained Stable Annually for 4 Years During the LTTP Year 4 (n= 41)	26.97 percentage of participants	—
Percentage of Participants Who Remained Stable Annually for 4 Years During the LTTP Year 1 (n=127)	83.6 percentage of participants	—
Percentage of Participants Who Remained Stable Annually for 4 Years During the LTTP Year 2 (n= 115)	75.65 percentage of participants	—
Percentage of Participants Who Remained Stable Annually for 4 Years During the LTTP Year 3 (n= 92)	60.53 percentage of participants	—

SECONDARY outcome

Timeframe: Baseline

Population: Per protocol population for PAP. Number of participants analyzed = participants with baseline T-score assessment. Here, 'n' signifies participants with baseline T-score assessment for specified bone area.

Images of the spine and bilateral femur were obtained by dual energy X-Ray absorptiometry (DXA) to determine T-score for each bone area and total bone mineral density. T-score compares participant's bone density with that of healthy young participant. The T-score bone density categories are: normal (score greater than [>]-1), osteopenia (score -2.5 to less than or equal to [<=] -1), and osteoporosis (score <= -2.5).

Outcome measures

Measure	Eliglustat: PAP n=81 Participants Eliglustat tartrate 50 mg capsule BID orally from Day 1 to Week 4, followed by eliglustat tartrate 50, 100 or 150 mg capsule BID orally up to Week 52.	Imiglucerase: PAP n=38 Participants Imiglucerase intravenous infusion q2w up to Week 52 in doses equivalent to participant's past ERT dose prior to any unanticipated treatment interruption, dose reduction, or regimen change.
Total T-Scores for Bone Mineral Density Lumbar Spine T-Score (n=81, 38)	-0.56 T-score Standard Deviation 1.309	-0.33 T-score Standard Deviation 1.169
Total T-Scores for Bone Mineral Density Femur T-Score (n=80, 37)	-0.11 T-score Standard Deviation 1.080	-0.47 T-score Standard Deviation 1.293

SECONDARY outcome

Timeframe: Baseline, Week 52

Population: Per protocol population for PAP. Number of participants analyzed = participants with both baseline and Week 52 T-score assessment. Here, 'n' signifies participants with both baseline and Week 52 T-score assessment for specified bone area. Eliglustat participants switching to imiglucerase were excluded.

Images of the spine and bilateral femur were obtained by DXA to determine T-score for each bone area and total bone mineral density. T-score compares participant's bone density with that of healthy young participant. The T-score bone density categories are: normal (score >-1), osteopenia (score -2.5 to <=-1), and osteoporosis (score <= -2.5). Absolute change = T-score at Week 52 minus T-score at baseline.

Outcome measures

Measure	Eliglustat: PAP n=81 Participants Eliglustat tartrate 50 mg capsule BID orally from Day 1 to Week 4, followed by eliglustat tartrate 50, 100 or 150 mg capsule BID orally up to Week 52.	Imiglucerase: PAP n=38 Participants Imiglucerase intravenous infusion q2w up to Week 52 in doses equivalent to participant's past ERT dose prior to any unanticipated treatment interruption, dose reduction, or regimen change.
Absolute Change From Baseline in Total T-Scores for Bone Mineral Density at Week 52 Change in Lumbar Spine T-Score (n=81, 38)	0.04 T-score Standard Error 0.03	0.03 T-score Standard Error 0.05
Absolute Change From Baseline in Total T-Scores for Bone Mineral Density at Week 52 Change in Femur T-Score (n=80, 37)	0.00 T-score Standard Error 0.02	-0.03 T-score Standard Error 0.03

SECONDARY outcome

Timeframe: Baseline

Population: Per protocol population for PAP. Number of participants analyzed = participants with baseline Z-score assessment. Here, 'n' signifies participants with baseline Z-score assessment for specified bone area.

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).

Outcome measures

Measure	Eliglustat: PAP n=94 Participants Eliglustat tartrate 50 mg capsule BID orally from Day 1 to Week 4, followed by eliglustat tartrate 50, 100 or 150 mg capsule BID orally up to Week 52.	Imiglucerase: PAP n=45 Participants Imiglucerase intravenous infusion q2w up to Week 52 in doses equivalent to participant's past ERT dose prior to any unanticipated treatment interruption, dose reduction, or regimen change.
Total Z-Scores for Bone Mineral Density Lumbar Spine Z-Score (n=94, 45)	-0.35 Z-score Standard Deviation 1.260	-0.14 Z-score Standard Deviation 1.108
Total Z-Scores for Bone Mineral Density Femur Z-Score (n=93, 44)	0.09 Z-score Standard Deviation 1.020	-0.18 Z-score Standard Deviation 1.122

SECONDARY outcome

Timeframe: Baseline, Week 52

Population: Per protocol population for PAP. Number of participants analyzed = participants with both baseline and Week 52 Z-score assessment. Here, 'n' signifies participants with both baseline and Week 52 Z-score assessment for specified bone area. Eliglustat participants switching to imiglucerase were excluded.

Images of the spine and bilateral femur were obtained by DXA to determine Z-score for each bone area and total bone mineral density. The Z-score bone density categories are: normal (score >-2) and below normal (score <=-2). Absolute change = Z-score at Week 52 minus Z-score at baseline.

Outcome measures

Measure	Eliglustat: PAP n=94 Participants Eliglustat tartrate 50 mg capsule BID orally from Day 1 to Week 4, followed by eliglustat tartrate 50, 100 or 150 mg capsule BID orally up to Week 52.	Imiglucerase: PAP n=45 Participants Imiglucerase intravenous infusion q2w up to Week 52 in doses equivalent to participant's past ERT dose prior to any unanticipated treatment interruption, dose reduction, or regimen change.
Absolute Change From Baseline in Total Z-Scores for Bone Mineral Density at Week 52 Change in Lumbar Spine Z-Score (n=94, 45)	0.06 Z-score Standard Error 0.03	0.06 Z-score Standard Error 0.04
Absolute Change From Baseline in Total Z-Scores for Bone Mineral Density at Week 52 Change in Femur Z-Score (n=93, 44)	0.03 Z-score Standard Error 0.02	0.02 Z-score Standard Error 0.02

SECONDARY outcome

Timeframe: Baseline

Population: Per protocol population for PAP. Number of participants analyzed = participants with baseline hemoglobin assessment.

Outcome measures

Measure	Eliglustat: PAP n=98 Participants Eliglustat tartrate 50 mg capsule BID orally from Day 1 to Week 4, followed by eliglustat tartrate 50, 100 or 150 mg capsule BID orally up to Week 52.	Imiglucerase: PAP n=47 Participants Imiglucerase intravenous infusion q2w up to Week 52 in doses equivalent to participant's past ERT dose prior to any unanticipated treatment interruption, dose reduction, or regimen change.
Hemoglobin Level	13.592 gram per deciliter (g/dL) Standard Deviation 1.2467	13.797 gram per deciliter (g/dL) Standard Deviation 1.2234

SECONDARY outcome

Timeframe: Baseline, Week 52

Population: Per protocol population for PAP. Number of participants analyzed = participants with both baseline and Week 52 hemoglobin assessment. Eliglustat participants switching to imiglucerase were excluded.

Absolute change = hemoglobin level at Week 52 minus hemoglobin level at baseline.

Outcome measures

Measure	Eliglustat: PAP n=98 Participants Eliglustat tartrate 50 mg capsule BID orally from Day 1 to Week 4, followed by eliglustat tartrate 50, 100 or 150 mg capsule BID orally up to Week 52.	Imiglucerase: PAP n=47 Participants Imiglucerase intravenous infusion q2w up to Week 52 in doses equivalent to participant's past ERT dose prior to any unanticipated treatment interruption, dose reduction, or regimen change.
Absolute Change From Baseline in Hemoglobin Levels at Week 52	-0.22 g/dL Standard Error 0.07	0.05 g/dL Standard Error 0.10

SECONDARY outcome

Timeframe: Baseline, Week 52

Population: Per protocol population for PAP. Number of participants analyzed = participants with both baseline and Week 52 platelet assessment. Eliglustat participants switching to imiglucerase were excluded.

Percent change in platelet counts = ([platelet count at Week 52 minus platelet count at baseline] divided by [platelet count at baseline]) multiplied by 100.

Outcome measures

Measure	Eliglustat: PAP n=98 Participants Eliglustat tartrate 50 mg capsule BID orally from Day 1 to Week 4, followed by eliglustat tartrate 50, 100 or 150 mg capsule BID orally up to Week 52.	Imiglucerase: PAP n=47 Participants Imiglucerase intravenous infusion q2w up to Week 52 in doses equivalent to participant's past ERT dose prior to any unanticipated treatment interruption, dose reduction, or regimen change.
Percent Change From Baseline in Platelet Counts at Week 52	3.93 percent change Standard Error 1.71	2.63 percent change Standard Error 2.47

SECONDARY outcome

Timeframe: Baseline, Week 52

Population: Per protocol population for PAP. Number of participants analyzed = participants with both baseline and Week 52 spleen volume assessment. Eliglustat participants switching to imiglucerase were excluded.

Percent change in spleen volume = ([spleen volume at Week 52 minus spleen volume at baseline] divided by [spleen volume at baseline]) multiplied by 100, where all volumes are in MN.

Outcome measures

Measure	Eliglustat: PAP n=70 Participants Eliglustat tartrate 50 mg capsule BID orally from Day 1 to Week 4, followed by eliglustat tartrate 50, 100 or 150 mg capsule BID orally up to Week 52.	Imiglucerase: PAP n=39 Participants Imiglucerase intravenous infusion q2w up to Week 52 in doses equivalent to participant's past ERT dose prior to any unanticipated treatment interruption, dose reduction, or regimen change.
Percent Change From Baseline in Spleen Volume (MN) at Week 52	-6.05 percent change Standard Error 1.57	-3.22 percent change Standard Error 2.13

SECONDARY outcome

Timeframe: Baseline, Week 52

Population: Per protocol population for PAP. Number of participants analyzed = participants with both baseline and Week 52 liver volume assessment. Eliglustat participants switching to imiglucerase were excluded.

Percent change in liver volume = ([liver volume at Week 52 minus liver volume at baseline] divided by [liver volume at baseline]) multiplied by 100, where all volumes are in multiples of normal.

Outcome measures

Measure	Eliglustat: PAP n=98 Participants Eliglustat tartrate 50 mg capsule BID orally from Day 1 to Week 4, followed by eliglustat tartrate 50, 100 or 150 mg capsule BID orally up to Week 52.	Imiglucerase: PAP n=47 Participants Imiglucerase intravenous infusion q2w up to Week 52 in doses equivalent to participant's past ERT dose prior to any unanticipated treatment interruption, dose reduction, or regimen change.
Percent Change From Baseline in Liver Volume (in MN) at Week 52	1.99 percent change Standard Error 0.94	3.13 percent change Standard Error 1.36

SECONDARY outcome

Timeframe: Baseline, Week 208

Population: Number of participants analyzed = participants with both baseline and Week 208 T-score assessment.

Images of the spine and bilateral femur were obtained by DXA to determine T-score for each bone area and total bone mineral density. T-score compares participant's bone density with that of healthy young participant. The T-score bone density categories are: normal (score >-1), osteopenia (score -2.5 to <=-1), and osteoporosis (score <= -2.5). Absolute change = T-score at Week 208 minus T-score at baseline.

Outcome measures

Measure	Eliglustat: PAP n=36 Participants Eliglustat tartrate 50 mg capsule BID orally from Day 1 to Week 4, followed by eliglustat tartrate 50, 100 or 150 mg capsule BID orally up to Week 52.	Imiglucerase: PAP Imiglucerase intravenous infusion q2w up to Week 52 in doses equivalent to participant's past ERT dose prior to any unanticipated treatment interruption, dose reduction, or regimen change.
Absolute Change From Baseline in Total T-Scores for Bone Mineral Density at Week 208 Total Spine	0.22 T-Score Standard Deviation 0.405	—
Absolute Change From Baseline in Total T-Scores for Bone Mineral Density at Week 208 Total Femur	-0.03 T-Score Standard Deviation 0.345	—

SECONDARY outcome

Timeframe: Baseline, Week 208

Population: FAS population for LTTP. Number of participants analyzed = participants with both baseline and Week 208 Z-score assessment. Here, 'n' signifies participants with both baseline and Week 208 Z-score assessment for specified bone area.

Images of the spine and bilateral femur were obtained by DXA to determine Z-score for each bone area and total bone mineral density. The Z-score bone density categories are: normal (score >-2) and below normal (score <=-2). Absolute change = Z-score at Week 208 minus Z-score at baseline.

Outcome measures

Measure	Eliglustat: PAP n=42 Participants Eliglustat tartrate 50 mg capsule BID orally from Day 1 to Week 4, followed by eliglustat tartrate 50, 100 or 150 mg capsule BID orally up to Week 52.	Imiglucerase: PAP Imiglucerase intravenous infusion q2w up to Week 52 in doses equivalent to participant's past ERT dose prior to any unanticipated treatment interruption, dose reduction, or regimen change.
Absolute Change From Baseline in Total Z-Scores for Bone Mineral Density at Week 208 Total Spine	0.29 Z-score Standard Deviation 0.358	—
Absolute Change From Baseline in Total Z-Scores for Bone Mineral Density at Week 208 Total Femur	0.03 Z-score Standard Deviation 0.381	—

SECONDARY outcome

Timeframe: Baseline, Week 208

Population: FAS population for LTTP. Number of participants analyzed = participants with both baseline and Week 208 hemoglobin assessment.

Absolute change = hemoglobin level at Week 208 minus hemoglobin level at baseline.

Outcome measures

Measure	Eliglustat: PAP n=45 Participants Eliglustat tartrate 50 mg capsule BID orally from Day 1 to Week 4, followed by eliglustat tartrate 50, 100 or 150 mg capsule BID orally up to Week 52.	Imiglucerase: PAP Imiglucerase intravenous infusion q2w up to Week 52 in doses equivalent to participant's past ERT dose prior to any unanticipated treatment interruption, dose reduction, or regimen change.
Absolute Change From Baseline in Hemoglobin Levels at Week 208	0.297 g/dL Standard Deviation 0.7472	—

SECONDARY outcome

Timeframe: Baseline, Week 208

Population: FAS population for LTTP. Number of participants analyzed = participants with both baseline and Week 208 platelet assessment.

Percent change in platelet counts = ([platelet count at Week 208 minus platelet count at baseline] divided by [platelet count at baseline]) multiplied by 100.

Outcome measures

Measure	Eliglustat: PAP n=45 Participants Eliglustat tartrate 50 mg capsule BID orally from Day 1 to Week 4, followed by eliglustat tartrate 50, 100 or 150 mg capsule BID orally up to Week 52.	Imiglucerase: PAP Imiglucerase intravenous infusion q2w up to Week 52 in doses equivalent to participant's past ERT dose prior to any unanticipated treatment interruption, dose reduction, or regimen change.
Percent Change From Baseline in Platelet Counts at Week 208	6.990 percent change Standard Deviation 20.4382	—

SECONDARY outcome

Timeframe: Baseline, Week 208

Population: FAS population for LTTP. Number of participants analyzed = participants with both baseline and Week 208 spleen volume assessment.

Percent change in spleen volume = ([spleen volume at Week 208 minus spleen volume at baseline] divided by [spleen volume at baseline]) multiplied by 100, where all volumes are in MN.

Outcome measures

Measure	Eliglustat: PAP n=33 Participants Eliglustat tartrate 50 mg capsule BID orally from Day 1 to Week 4, followed by eliglustat tartrate 50, 100 or 150 mg capsule BID orally up to Week 52.	Imiglucerase: PAP Imiglucerase intravenous infusion q2w up to Week 52 in doses equivalent to participant's past ERT dose prior to any unanticipated treatment interruption, dose reduction, or regimen change.
Percent Change From Baseline in Spleen Volume (in MN) at Week 208	-14.768 percent change Standard Deviation 17.9435	—

SECONDARY outcome

Timeframe: Baseline, Week 208

Population: FAS population for LTTP. Number of participants analyzed = participants with both baseline and Week 208 liver volume assessment.

Percent change in liver volume = ([liver volume at Week 208 minus liver volume at baseline] divided by [liver volume at baseline]) multiplied by 100, where all volumes are in multiples of normal.

Outcome measures

Measure	Eliglustat: PAP n=46 Participants Eliglustat tartrate 50 mg capsule BID orally from Day 1 to Week 4, followed by eliglustat tartrate 50, 100 or 150 mg capsule BID orally up to Week 52.	Imiglucerase: PAP Imiglucerase intravenous infusion q2w up to Week 52 in doses equivalent to participant's past ERT dose prior to any unanticipated treatment interruption, dose reduction, or regimen change.
Percent Change From Baseline in Liver Volume (in MN) at Week 208	-2.345 percent change Standard Deviation 12.8795	—

Adverse Events

Eliglustat

Serious events: 18 serious events

Other events: 91 other events

Deaths: 0 deaths

Imiglucerase

Serious events: 7 serious events

Other events: 38 other events

Deaths: 0 deaths

Serious adverse events

Measure	Eliglustat n=106 participants at risk PAP: Eliglustat tartrate 50 mg capsule BID orally from Day 1 to Week 4, followed by eliglustat tartrate 50, 100 or 150 mg capsule BID orally up to Week 52. Dose adjustments after Week 4 and Week 8 were based on Genz-99067 (active moiety of eliglustat tartrate in plasma) trough plasma concentrations. LTTP: Participants originally randomized to eliglustat in PAP continued to receive eliglustat dose, based on their Genz 99067 plasma trough concentration at Week 6.	Imiglucerase n=53 participants at risk PAP: Imiglucerase (Cerezyme®) intravenous infusion q2w up to Week 52 in doses equivalent to participant's past ERT dose prior to any unanticipated treatment interruption, dose reduction, or regimen change. LTTP: Participants originally randomized to imiglucerase received eliglustat tartrate capsule 50 mg BID orally from Week 52+1 Day to Week 56 followed by eliglustat tartrate 50 mg or 100 mg capsule BID up to Week 60, and then eliglustat tartrate 50 mg or 100 mg or 150 mg capsule BID up to 5 years. The dose adjustments after Week 56 and Week 60 were based on Genz-99067 (active moiety of eliglustat tartrate in plasma) trough plasma concentrations.
Blood and lymphatic system disorders Polycythaemia	0.00% 0/106 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.	1.9% 1/53 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.
Cardiac disorders Acute myocardial infarction	0.00% 0/106 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.	1.9% 1/53 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.
Cardiac disorders Myocardial infarction	0.00% 0/106 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.	1.9% 1/53 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.
Gastrointestinal disorders Colitis ischaemic	0.94% 1/106 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.	0.00% 0/53 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.
Gastrointestinal disorders Diarrhoea	0.94% 1/106 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.	0.00% 0/53 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.
Gastrointestinal disorders Intestinal obstruction	0.94% 1/106 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.	0.00% 0/53 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.
General disorders Device malfunction	0.94% 1/106 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.	0.00% 0/53 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.
General disorders Pain	0.94% 1/106 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.	0.00% 0/53 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.
General disorders Pyrexia	0.94% 1/106 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.	0.00% 0/53 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.
Hepatobiliary disorders Biliary colic	0.94% 1/106 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.	0.00% 0/53 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.
Hepatobiliary disorders Cholecystitis	0.94% 1/106 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.	0.00% 0/53 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.
Infections and infestations Appendicitis	0.94% 1/106 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.	0.00% 0/53 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.
Infections and infestations Diverticulitis	0.94% 1/106 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.	0.00% 0/53 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.
Injury, poisoning and procedural complications Injury	0.00% 0/106 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.	1.9% 1/53 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.
Injury, poisoning and procedural complications Joint dislocation	0.94% 1/106 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.	0.00% 0/53 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Benign ovarian tumour	0.00% 0/106 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.	1.9% 1/53 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Metastatic renal cell carcinoma	0.00% 0/106 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.	1.9% 1/53 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Pancreatic carcinoma metastatic	0.00% 0/106 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.	1.9% 1/53 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Uterine leiomyoma	0.94% 1/106 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.	0.00% 0/53 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.
Nervous system disorders Neuropathy peripheral	0.94% 1/106 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.	0.00% 0/53 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.
Nervous system disorders Syncope	2.8% 3/106 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.	0.00% 0/53 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.
Respiratory, thoracic and mediastinal disorders Epistaxis	0.94% 1/106 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.	0.00% 0/53 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.
Respiratory, thoracic and mediastinal disorders Nasal septum deviation	0.94% 1/106 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.	0.00% 0/53 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.
Surgical and medical procedures Mammoplasty	0.94% 1/106 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.	0.00% 0/53 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.

Other adverse events

Measure	Eliglustat n=106 participants at risk PAP: Eliglustat tartrate 50 mg capsule BID orally from Day 1 to Week 4, followed by eliglustat tartrate 50, 100 or 150 mg capsule BID orally up to Week 52. Dose adjustments after Week 4 and Week 8 were based on Genz-99067 (active moiety of eliglustat tartrate in plasma) trough plasma concentrations. LTTP: Participants originally randomized to eliglustat in PAP continued to receive eliglustat dose, based on their Genz 99067 plasma trough concentration at Week 6.	Imiglucerase n=53 participants at risk PAP: Imiglucerase (Cerezyme®) intravenous infusion q2w up to Week 52 in doses equivalent to participant's past ERT dose prior to any unanticipated treatment interruption, dose reduction, or regimen change. LTTP: Participants originally randomized to imiglucerase received eliglustat tartrate capsule 50 mg BID orally from Week 52+1 Day to Week 56 followed by eliglustat tartrate 50 mg or 100 mg capsule BID up to Week 60, and then eliglustat tartrate 50 mg or 100 mg or 150 mg capsule BID up to 5 years. The dose adjustments after Week 56 and Week 60 were based on Genz-99067 (active moiety of eliglustat tartrate in plasma) trough plasma concentrations.
Cardiac disorders Palpitations	7.5% 8/106 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.	5.7% 3/53 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.
Gastrointestinal disorders Abdominal pain	13.2% 14/106 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.	11.3% 6/53 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.
Gastrointestinal disorders Abdominal pain lower	5.7% 6/106 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.	0.00% 0/53 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.
Gastrointestinal disorders Abdominal pain upper	24.5% 26/106 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.	5.7% 3/53 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.
Gastrointestinal disorders Constipation	9.4% 10/106 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.	11.3% 6/53 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.
Gastrointestinal disorders Diarrhoea	18.9% 20/106 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.	3.8% 2/53 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.
Gastrointestinal disorders Dyspepsia	14.2% 15/106 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.	7.5% 4/53 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.
Gastrointestinal disorders Gastritis	5.7% 6/106 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.	9.4% 5/53 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.
Gastrointestinal disorders Gastrooesophageal reflux disease	12.3% 13/106 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.	3.8% 2/53 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.
Gastrointestinal disorders Nausea	17.0% 18/106 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.	13.2% 7/53 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.
Gastrointestinal disorders Toothache	6.6% 7/106 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.	5.7% 3/53 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.
Gastrointestinal disorders Vomiting	9.4% 10/106 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.	0.00% 0/53 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.
General disorders Asthenia	9.4% 10/106 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.	1.9% 1/53 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.
General disorders Chest pain	8.5% 9/106 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.	1.9% 1/53 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.
General disorders Fatigue	18.9% 20/106 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.	13.2% 7/53 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.
General disorders Pain	5.7% 6/106 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.	5.7% 3/53 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.
General disorders Peripheral swelling	5.7% 6/106 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.	1.9% 1/53 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.
General disorders Pyrexia	1.9% 2/106 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.	5.7% 3/53 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.
Hepatobiliary disorders Hepatomegaly	4.7% 5/106 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.	5.7% 3/53 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.
Infections and infestations Bronchitis	7.5% 8/106 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.	3.8% 2/53 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.
Infections and infestations Gastroenteritis	10.4% 11/106 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.	3.8% 2/53 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.
Infections and infestations Influenza	18.9% 20/106 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.	11.3% 6/53 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.
Infections and infestations Nasopharyngitis	26.4% 28/106 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.	18.9% 10/53 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.
Infections and infestations Sinusitis	18.9% 20/106 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.	5.7% 3/53 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.
Infections and infestations Upper respiratory tract infection	19.8% 21/106 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.	13.2% 7/53 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.
Infections and infestations Urinary tract infection	15.1% 16/106 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.	5.7% 3/53 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.
Injury, poisoning and procedural complications Contusion	6.6% 7/106 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.	3.8% 2/53 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.
Injury, poisoning and procedural complications Laceration	6.6% 7/106 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.	1.9% 1/53 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.
Injury, poisoning and procedural complications Ligament sprain	7.5% 8/106 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.	0.00% 0/53 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.
Injury, poisoning and procedural complications Limb injury	5.7% 6/106 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.	0.00% 0/53 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.
Investigations Blood creatine phosphokinase increased	15.1% 16/106 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.	5.7% 3/53 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.
Investigations Mean cell volume abnormal	0.00% 0/106 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.	5.7% 3/53 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.
Investigations Nerve conduction studies abnormal	6.6% 7/106 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.	5.7% 3/53 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.
Musculoskeletal and connective tissue disorders Arthralgia	35.8% 38/106 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.	24.5% 13/53 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.
Musculoskeletal and connective tissue disorders Back pain	21.7% 23/106 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.	11.3% 6/53 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.
Musculoskeletal and connective tissue disorders Bone pain	8.5% 9/106 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.	5.7% 3/53 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.
Musculoskeletal and connective tissue disorders Muscle spasms	5.7% 6/106 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.	1.9% 1/53 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.
Musculoskeletal and connective tissue disorders Musculoskeletal pain	7.5% 8/106 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.	5.7% 3/53 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.
Musculoskeletal and connective tissue disorders Myalgia	5.7% 6/106 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.	3.8% 2/53 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.
Musculoskeletal and connective tissue disorders Pain in extremity	19.8% 21/106 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.	9.4% 5/53 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.
Nervous system disorders Dizziness	17.0% 18/106 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.	7.5% 4/53 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.
Nervous system disorders Headache	24.5% 26/106 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.	17.0% 9/53 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.
Nervous system disorders Hypoaesthesia	5.7% 6/106 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.	1.9% 1/53 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.
Nervous system disorders Paraesthesia	6.6% 7/106 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.	1.9% 1/53 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.
Psychiatric disorders Anxiety	9.4% 10/106 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.	1.9% 1/53 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.
Psychiatric disorders Depression	2.8% 3/106 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.	5.7% 3/53 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.
Respiratory, thoracic and mediastinal disorders Cough	10.4% 11/106 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.	3.8% 2/53 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.
Respiratory, thoracic and mediastinal disorders Epistaxis	6.6% 7/106 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.	1.9% 1/53 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.
Respiratory, thoracic and mediastinal disorders Oropharyngeal pain	6.6% 7/106 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.	5.7% 3/53 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.
Skin and subcutaneous tissue disorders Dry skin	4.7% 5/106 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.	7.5% 4/53 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.
Vascular disorders Hypertension	5.7% 6/106 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.	1.9% 1/53 • From signature of informed consent up to 30-37 days after the last dose of treatment (last dose = up to Week 104) Safety set included all participants who received at least 1 dose of study drug (Eliglustat or Imiglucerase). In the event a single participant experienced both serious and non-serious forms of same adverse events (AE), individual was included in numerator (number of participants affected) of each AE table.

Additional Information

Trial Transparency Team

Sanofi

Email: Contact-us@sanofi.com

Results disclosure agreements

• Principal investigator is a sponsor employee 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.
• Publication restrictions are in place

Restriction type: OTHER