Trial Outcomes & Findings for A Study of the Efficacy and Safety of Eliglustat Tartrate (Genz-112638) in Type 1 Gaucher Patients (NCT NCT00358150)

Last Updated: 2017-02-15

Results Overview

A meaningful clinical response was defined as an improvement in at least 2 of the 3 main efficacy parameters: a) an increase in hemoglobin of greater than or equal to (\>=) 0.5 gram/deciliter from baseline, b) an increase in platelets of \>=15 percent (%) from baseline, c) reduction in total spleen volume of \>= 15% from baseline. As hemoglobin, platelets, total spleen volume were abnormal at baseline, within each participant, only those parameters were used in the evaluation of meaningful clinical response which were abnormal at baseline.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

26 participants

Primary outcome timeframe

Baseline, Year 1

Results posted on

2017-02-15

Participant Flow

A total of 50 participants were screened of which 24 participants were screen failure. A total of 26 participants were enrolled in this study.

Participant milestones

Participant milestones
Measure	Eliglustat Eliglustat (Genz-112638) capsule as single 50 milligram (mg) dose on Day 1 then eliglustat 50 mg twice daily (BID) from Day 2 to Day 19, and then either eliglustat 50 mg BID (if Genz-99067 \[active moiety of eliglustat in plasma\] trough plasma concentration was greater than or equal to \[\>=\] 5 nanogram per milliliter \[ng/mL\] on Day 10) or eliglustat 100 mg BID (if Genz-99067 trough plasma concentration was less than \[\<\] 5 ng/mL), from day 20 to Year 4. After primary completion date (Week 52) participants underwent treatment interruption period of approximately 2 weeks before continuing the same treatment up to Year 9. Participant receiving 100 mg BID could be considered for a further dose increase to 150 mg BID at Week 24 if they met certain criteria (for example, had been on treatment for at least 24 months, had not reached therapeutic goals established for participants receiving Cerezyme), and if all other causes for lack of treatment effect had been evaluated and ruled out).
Overall Study STARTED	26
Overall Study COMPLETED	19
Overall Study NOT COMPLETED	7

Reasons for withdrawal

Reasons for withdrawal
Measure	Eliglustat Eliglustat (Genz-112638) capsule as single 50 milligram (mg) dose on Day 1 then eliglustat 50 mg twice daily (BID) from Day 2 to Day 19, and then either eliglustat 50 mg BID (if Genz-99067 \[active moiety of eliglustat in plasma\] trough plasma concentration was greater than or equal to \[\>=\] 5 nanogram per milliliter \[ng/mL\] on Day 10) or eliglustat 100 mg BID (if Genz-99067 trough plasma concentration was less than \[\<\] 5 ng/mL), from day 20 to Year 4. After primary completion date (Week 52) participants underwent treatment interruption period of approximately 2 weeks before continuing the same treatment up to Year 9. Participant receiving 100 mg BID could be considered for a further dose increase to 150 mg BID at Week 24 if they met certain criteria (for example, had been on treatment for at least 24 months, had not reached therapeutic goals established for participants receiving Cerezyme), and if all other causes for lack of treatment effect had been evaluated and ruled out).
Overall Study Adverse Event	3
Overall Study Withdrawal by Subject	1
Overall Study Undefined	3

Baseline Characteristics

A Study of the Efficacy and Safety of Eliglustat Tartrate (Genz-112638) in Type 1 Gaucher Patients

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Eliglustat n=26 Participants Eliglustat (Genz-112638) capsule as single 50 mg dose on Day 1 then eliglustat 50 mg BID from Day 2 to Day 19, and then either eliglustat 50 mg BID (if Genz-99067 \[active moiety of eliglustat in plasma\] trough plasma concentration was greater than or equal to \[\>=\] 5 ng/mL on Day 10) or eliglustat 100 mg BID (if Genz-99067 trough plasma concentration was less than \[\<\] 5 ng/mL), from day 20 to Year 4. After primary completion date (Week 52) participants underwent treatment interruption period of approximately 2 weeks before continuing the same treatment up to Year 9. Participant receiving 100 mg BID could be considered for a further dose increase to 150 mg BID at Week 24 if they met certain criteria (for example, had been on treatment for at least 24 months, had not reached therapeutic goals established for participants receiving Cerezyme, and if all other causes for lack of treatment effect had been evaluated and ruled out).
Age, Continuous	34.47 years STANDARD_DEVIATION 12.960 • n=5 Participants
Gender Female	16 Participants n=5 Participants
Gender Male	10 Participants n=5 Participants
Race/Ethnicity, Customized Ashkenazi Jewish	7 participants n=5 Participants
Race/Ethnicity, Customized Non-Jewish Caucasian	16 participants n=5 Participants
Race/Ethnicity, Customized Hispanic	3 participants n=5 Participants
Body Mass Index (BMI)	22.56 kilogram per square meter (kg/m^2) STANDARD_DEVIATION 3.529 • n=5 Participants
Weight	61.47 kilogram (kg) STANDARD_DEVIATION 11.018 • n=5 Participants
Height	165.12 centimeter (cm) STANDARD_DEVIATION 9.747 • n=5 Participants
Hemoglobin	11.10 gram per deciliter (g/dL) STANDARD_DEVIATION 1.674 • n=5 Participants
Platelet Count	66.423 10^9 cells per liter STANDARD_DEVIATION 20.1413 • n=5 Participants
Spleen Volume	20.04 Multiples of normal STANDARD_DEVIATION 12.798 • n=5 Participants

PRIMARY outcome

Timeframe: Baseline, Year 1

Population: Full analysis set consists of all participants who signed informed consent and received at least one dose of eliglustat.

Outcome measures

Outcome measures
Measure	Eliglustat n=26 Participants Eliglustat (Genz-112638) capsule as single 50 mg dose on Day 1 then eliglustat 50 mg BID from Day 2 to Day 19, and then either eliglustat 50 mg BID (if Genz-99067 \[active moiety of eliglustat in plasma\] trough plasma concentration was greater than or equal to \[\>=\] 5 ng/mL on Day 10) or eliglustat 100 mg BID (if Genz-99067 trough plasma concentration was less than \[\<\] 5 ng/mL), from day 20 to Year 4. After primary completion date (Week 52) participants underwent treatment interruption period of approximately 2 weeks before continuing the same treatment up to Year 9. Participant receiving 100 mg BID could be considered for a further dose increase to 150 mg BID at Week 24 if they met certain criteria (for example, had been on treatment for at least 24 months, had not reached therapeutic goals established for participants receiving Cerezyme, and if all other causes for lack of treatment effect had been evaluated and ruled out).
Percentage of Participants Demonstrating A Meaningful Clinical Response	77 percentage of participants

SECONDARY outcome

Timeframe: Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study (Up to Year 9)

Population: Full analysis set consists of all participants who signed informed consent and received at least one dose of eliglustat. Here 'n' signifies number of participants with available data at specified time points.

Percent change in spleen volume = (\[spleen volume at specified time points minus spleen volume at baseline\] divided by \[spleen volume at baseline\]) multiplied by 100, where all volumes are in multiples of normal.

Outcome measures

Outcome measures
Measure	Eliglustat n=26 Participants Eliglustat (Genz-112638) capsule as single 50 mg dose on Day 1 then eliglustat 50 mg BID from Day 2 to Day 19, and then either eliglustat 50 mg BID (if Genz-99067 \[active moiety of eliglustat in plasma\] trough plasma concentration was greater than or equal to \[\>=\] 5 ng/mL on Day 10) or eliglustat 100 mg BID (if Genz-99067 trough plasma concentration was less than \[\<\] 5 ng/mL), from day 20 to Year 4. After primary completion date (Week 52) participants underwent treatment interruption period of approximately 2 weeks before continuing the same treatment up to Year 9. Participant receiving 100 mg BID could be considered for a further dose increase to 150 mg BID at Week 24 if they met certain criteria (for example, had been on treatment for at least 24 months, had not reached therapeutic goals established for participants receiving Cerezyme, and if all other causes for lack of treatment effect had been evaluated and ruled out).
Percent Change From Baseline in Spleen Volume at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Change at Year 6 (n=19)	-66.2 percent change Standard Deviation 15.72
Percent Change From Baseline in Spleen Volume at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Change at Year 7 (n=19)	-67.8 percent change Standard Deviation 15.65
Percent Change From Baseline in Spleen Volume at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Change at Year 1 (n=22)	-38.5 percent change Standard Deviation 11.41
Percent Change From Baseline in Spleen Volume at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Change at Year 2 (n=20)	-52.4 percent change Standard Deviation 10.73
Percent Change From Baseline in Spleen Volume at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Change at Year 3 (n=19)	-59.1 percent change Standard Deviation 11.68
Percent Change From Baseline in Spleen Volume at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Change at Year 4 (n=18)	-62.5 percent change Standard Deviation 11.63
Percent Change From Baseline in Spleen Volume at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Change at Year 5 (n=19)	-63.9 percent change Standard Deviation 14.64
Percent Change From Baseline in Spleen Volume at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Change at Year 8 (n=15)	-67.9 percent change Standard Deviation 17.11
Percent Change From Baseline in Spleen Volume at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Change at Year 9 (n=4)	-52.6 percent change Standard Deviation 23.97
Percent Change From Baseline in Spleen Volume at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Change at End of Study (n=19)	-68.6 percent change Standard Deviation 18.60

SECONDARY outcome

Timeframe: Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study (Up to Year 9)

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

Outcome measures

Outcome measures
Measure	Eliglustat n=26 Participants Eliglustat (Genz-112638) capsule as single 50 mg dose on Day 1 then eliglustat 50 mg BID from Day 2 to Day 19, and then either eliglustat 50 mg BID (if Genz-99067 \[active moiety of eliglustat in plasma\] trough plasma concentration was greater than or equal to \[\>=\] 5 ng/mL on Day 10) or eliglustat 100 mg BID (if Genz-99067 trough plasma concentration was less than \[\<\] 5 ng/mL), from day 20 to Year 4. After primary completion date (Week 52) participants underwent treatment interruption period of approximately 2 weeks before continuing the same treatment up to Year 9. Participant receiving 100 mg BID could be considered for a further dose increase to 150 mg BID at Week 24 if they met certain criteria (for example, had been on treatment for at least 24 months, had not reached therapeutic goals established for participants receiving Cerezyme, and if all other causes for lack of treatment effect had been evaluated and ruled out).
Percent Change From Baseline in Liver Volume at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Change at Year 6 (n=19)	-28.4 percent change Standard Deviation 23.58
Percent Change From Baseline in Liver Volume at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Change at Year 7 (n=19)	-36.2 percent change Standard Deviation 13.49
Percent Change From Baseline in Liver Volume at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Change at Year 1 (n=22)	-16.9 percent change Standard Deviation 10.48
Percent Change From Baseline in Liver Volume at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Change at Year 2 (n=20)	-23.9 percent change Standard Deviation 12.81
Percent Change From Baseline in Liver Volume at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Change at Year 3 (n=19)	-26.8 percent change Standard Deviation 12.28
Percent Change From Baseline in Liver Volume at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Change at Year 4 (n=18)	-28.0 percent change Standard Deviation 13.80
Percent Change From Baseline in Liver Volume at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Change at Year 5 (n=19)	-31.2 percent change Standard Deviation 13.90
Percent Change From Baseline in Liver Volume at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Change at Year 8 (n=15)	-31.1 percent change Standard Deviation 13.51
Percent Change From Baseline in Liver Volume at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Change at Year 9 (n=4)	-22.0 percent change Standard Deviation 18.49
Percent Change From Baseline in Liver Volume at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Change at End of Study (n=19)	-36.4 percent change Standard Deviation 14.91

SECONDARY outcome

Timeframe: Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study (Up to Year 9)

Absolute change = hemoglobin level at specified time points minus hemoglobin level at baseline.

Outcome measures

Outcome measures
Measure	Eliglustat n=26 Participants Eliglustat (Genz-112638) capsule as single 50 mg dose on Day 1 then eliglustat 50 mg BID from Day 2 to Day 19, and then either eliglustat 50 mg BID (if Genz-99067 \[active moiety of eliglustat in plasma\] trough plasma concentration was greater than or equal to \[\>=\] 5 ng/mL on Day 10) or eliglustat 100 mg BID (if Genz-99067 trough plasma concentration was less than \[\<\] 5 ng/mL), from day 20 to Year 4. After primary completion date (Week 52) participants underwent treatment interruption period of approximately 2 weeks before continuing the same treatment up to Year 9. Participant receiving 100 mg BID could be considered for a further dose increase to 150 mg BID at Week 24 if they met certain criteria (for example, had been on treatment for at least 24 months, had not reached therapeutic goals established for participants receiving Cerezyme, and if all other causes for lack of treatment effect had been evaluated and ruled out).
Absolute Change From Baseline in Hemoglobin at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Change at Year 8 (n=16)	2.08 g/dL Standard Deviation 1.748
Absolute Change From Baseline in Hemoglobin at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Change at Year 9 (n=4)	1.00 g/dL Standard Deviation 1.480
Absolute Change From Baseline in Hemoglobin at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Change at Year 1 (n=22)	1.70 g/dL Standard Deviation 1.274
Absolute Change From Baseline in Hemoglobin at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Change at Year 2 (n=20)	2.13 g/dL Standard Deviation 1.507
Absolute Change From Baseline in Hemoglobin at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Change at Year 3 (n=18)	2.47 g/dL Standard Deviation 1.406
Absolute Change From Baseline in Hemoglobin at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Change at Year 4 (n=19)	2.27 g/dL Standard Deviation 1.451
Absolute Change From Baseline in Hemoglobin at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Change at Year 5 (n=19)	2.09 g/dL Standard Deviation 1.746
Absolute Change From Baseline in Hemoglobin at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Change at Year 6 (n=18)	2.01 g/dL Standard Deviation 1.326
Absolute Change From Baseline in Hemoglobin at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Change at Year 7 (n=19)	2.07 g/dL Standard Deviation 1.485
Absolute Change From Baseline in Hemoglobin at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Change at End of Study (n=18)	2.01 g/dL Standard Deviation 1.785

SECONDARY outcome

Timeframe: Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study (Up to Year 9)

Percent change in platelet count = (\[platelet count at specified time points minus platelet count at baseline\] divided by \[platelet count at baseline\]) multiplied by 100.

Outcome measures

Outcome measures
Measure	Eliglustat n=26 Participants Eliglustat (Genz-112638) capsule as single 50 mg dose on Day 1 then eliglustat 50 mg BID from Day 2 to Day 19, and then either eliglustat 50 mg BID (if Genz-99067 \[active moiety of eliglustat in plasma\] trough plasma concentration was greater than or equal to \[\>=\] 5 ng/mL on Day 10) or eliglustat 100 mg BID (if Genz-99067 trough plasma concentration was less than \[\<\] 5 ng/mL), from day 20 to Year 4. After primary completion date (Week 52) participants underwent treatment interruption period of approximately 2 weeks before continuing the same treatment up to Year 9. Participant receiving 100 mg BID could be considered for a further dose increase to 150 mg BID at Week 24 if they met certain criteria (for example, had been on treatment for at least 24 months, had not reached therapeutic goals established for participants receiving Cerezyme, and if all other causes for lack of treatment effect had been evaluated and ruled out).
Percent Change From Baseline in Platelet Count at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Change at Year 1 (n=22)	41.3 percent change Standard Deviation 36.95
Percent Change From Baseline in Platelet Count at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Change at Year 2 (n=20)	81.5 percent change Standard Deviation 56.01
Percent Change From Baseline in Platelet Count at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Change at Year 3 (n=18)	87.9 percent change Standard Deviation 65.37
Percent Change From Baseline in Platelet Count at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Change at Year 4 (n=19)	95.1 percent change Standard Deviation 89.41
Percent Change From Baseline in Platelet Count at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Change at Year 5 (n=19)	90.9 percent change Standard Deviation 85.42
Percent Change From Baseline in Platelet Count at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Change at Year 6 (n=17)	114.3 percent change Standard Deviation 102.48
Percent Change From Baseline in Platelet Count at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Change at Year 7 (n=19)	99.4 percent change Standard Deviation 97.13
Percent Change From Baseline in Platelet Count at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Change at Year 8 (n=16)	109.8 percent change Standard Deviation 114.73
Percent Change From Baseline in Platelet Count at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Change at Year 9 (n=3)	45.4 percent change Standard Deviation 56.28
Percent Change From Baseline in Platelet Count at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Change at End of Study (n=18)	117.5 percent change Standard Deviation 116.91

SECONDARY outcome

Timeframe: Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and End of Study (Up to Year 9)

Outcome measures

Outcome measures
Measure	Eliglustat n=26 Participants Eliglustat (Genz-112638) capsule as single 50 mg dose on Day 1 then eliglustat 50 mg BID from Day 2 to Day 19, and then either eliglustat 50 mg BID (if Genz-99067 \[active moiety of eliglustat in plasma\] trough plasma concentration was greater than or equal to \[\>=\] 5 ng/mL on Day 10) or eliglustat 100 mg BID (if Genz-99067 trough plasma concentration was less than \[\<\] 5 ng/mL), from day 20 to Year 4. After primary completion date (Week 52) participants underwent treatment interruption period of approximately 2 weeks before continuing the same treatment up to Year 9. Participant receiving 100 mg BID could be considered for a further dose increase to 150 mg BID at Week 24 if they met certain criteria (for example, had been on treatment for at least 24 months, had not reached therapeutic goals established for participants receiving Cerezyme, and if all other causes for lack of treatment effect had been evaluated and ruled out).
Percent Change From Baseline in Biomarker (Angiotensin Converting Enzyme) Level at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Change at Year 6 (n=17)	-61.5 percent change Standard Deviation 24.73
Percent Change From Baseline in Biomarker (Angiotensin Converting Enzyme) Level at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Change at Year 7 (n=19)	-63.6 percent change Standard Deviation 24.64
Percent Change From Baseline in Biomarker (Angiotensin Converting Enzyme) Level at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Change at Year 8 (n=16)	-59.2 percent change Standard Deviation 21.77
Percent Change From Baseline in Biomarker (Angiotensin Converting Enzyme) Level at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Change at Year 1 (n=22)	-35.1 percent change Standard Deviation 18.87
Percent Change From Baseline in Biomarker (Angiotensin Converting Enzyme) Level at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Change at Year 2 (n=20)	-53.5 percent change Standard Deviation 21.05
Percent Change From Baseline in Biomarker (Angiotensin Converting Enzyme) Level at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Change at Year 3 (n=19)	-56.7 percent change Standard Deviation 19.33
Percent Change From Baseline in Biomarker (Angiotensin Converting Enzyme) Level at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Change at Year 4 (n=18)	-60.7 percent change Standard Deviation 18.44
Percent Change From Baseline in Biomarker (Angiotensin Converting Enzyme) Level at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Change at Year 5 (n=19)	-55.7 percent change Standard Deviation 22.51
Percent Change From Baseline in Biomarker (Angiotensin Converting Enzyme) Level at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Change at Year 9 (n=4)	-55.1 percent change Standard Deviation 32.58
Percent Change From Baseline in Biomarker (Angiotensin Converting Enzyme) Level at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Change at End of Study (n=18)	-64.7 percent change Standard Deviation 23.38

SECONDARY outcome

Timeframe: Baseline, Year 1, Year 2

Outcome measures

Outcome measures
Measure	Eliglustat n=26 Participants Eliglustat (Genz-112638) capsule as single 50 mg dose on Day 1 then eliglustat 50 mg BID from Day 2 to Day 19, and then either eliglustat 50 mg BID (if Genz-99067 \[active moiety of eliglustat in plasma\] trough plasma concentration was greater than or equal to \[\>=\] 5 ng/mL on Day 10) or eliglustat 100 mg BID (if Genz-99067 trough plasma concentration was less than \[\<\] 5 ng/mL), from day 20 to Year 4. After primary completion date (Week 52) participants underwent treatment interruption period of approximately 2 weeks before continuing the same treatment up to Year 9. Participant receiving 100 mg BID could be considered for a further dose increase to 150 mg BID at Week 24 if they met certain criteria (for example, had been on treatment for at least 24 months, had not reached therapeutic goals established for participants receiving Cerezyme, and if all other causes for lack of treatment effect had been evaluated and ruled out).
Percent Change From Baseline in Biomarker (Tartrate-Resistant Acid Phosphatase [TRAP]) Level at Year 1 and Year 2 Change at Year 2 (n= 10)	-52.5 percent change Standard Deviation 10.61
Percent Change From Baseline in Biomarker (Tartrate-Resistant Acid Phosphatase [TRAP]) Level at Year 1 and Year 2 Change at Year 1 (n=22)	-37.0 percent change Standard Deviation 11.64

SECONDARY outcome

Timeframe: Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and End of Study (Up to Year 9)

Outcome measures

Outcome measures
Measure	Eliglustat n=26 Participants Eliglustat (Genz-112638) capsule as single 50 mg dose on Day 1 then eliglustat 50 mg BID from Day 2 to Day 19, and then either eliglustat 50 mg BID (if Genz-99067 \[active moiety of eliglustat in plasma\] trough plasma concentration was greater than or equal to \[\>=\] 5 ng/mL on Day 10) or eliglustat 100 mg BID (if Genz-99067 trough plasma concentration was less than \[\<\] 5 ng/mL), from day 20 to Year 4. After primary completion date (Week 52) participants underwent treatment interruption period of approximately 2 weeks before continuing the same treatment up to Year 9. Participant receiving 100 mg BID could be considered for a further dose increase to 150 mg BID at Week 24 if they met certain criteria (for example, had been on treatment for at least 24 months, had not reached therapeutic goals established for participants receiving Cerezyme, and if all other causes for lack of treatment effect had been evaluated and ruled out).
Percent Change From Baseline in Biomarker Chemokine Ligand 18 (CCL18) Level at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and End of Study Change at Year 1 (n=21)	-49.0 percent change Standard Deviation 24.89
Percent Change From Baseline in Biomarker Chemokine Ligand 18 (CCL18) Level at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and End of Study Change at Year 2 (n=15)	-72.1 percent change Standard Deviation 19.11
Percent Change From Baseline in Biomarker Chemokine Ligand 18 (CCL18) Level at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and End of Study Change at Year 3 (n=18)	-68.6 percent change Standard Deviation 17.27
Percent Change From Baseline in Biomarker Chemokine Ligand 18 (CCL18) Level at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and End of Study Change at Year 4 (n=18)	-80.2 percent change Standard Deviation 12.71
Percent Change From Baseline in Biomarker Chemokine Ligand 18 (CCL18) Level at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and End of Study Change at Year 5 (n=18)	-89.4 percent change Standard Deviation 8.48
Percent Change From Baseline in Biomarker Chemokine Ligand 18 (CCL18) Level at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and End of Study Change at Year 6 (n=18)	-82.7 percent change Standard Deviation 11.98
Percent Change From Baseline in Biomarker Chemokine Ligand 18 (CCL18) Level at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and End of Study Change at Year 7 (n=18)	-78.5 percent change Standard Deviation 17.83
Percent Change From Baseline in Biomarker Chemokine Ligand 18 (CCL18) Level at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and End of Study Change at Year 8 (n=16)	-79.8 percent change Standard Deviation 16.19
Percent Change From Baseline in Biomarker Chemokine Ligand 18 (CCL18) Level at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and End of Study Change at Year 9 (n=4)	-88.6 percent change Standard Deviation 6.30
Percent Change From Baseline in Biomarker Chemokine Ligand 18 (CCL18) Level at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and End of Study Change at End of Study (n=18)	-85.8 percent change Standard Deviation 12.92

SECONDARY outcome

Timeframe: Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study (Up to Year 9)

Outcome measures

Outcome measures
Measure	Eliglustat n=26 Participants Eliglustat (Genz-112638) capsule as single 50 mg dose on Day 1 then eliglustat 50 mg BID from Day 2 to Day 19, and then either eliglustat 50 mg BID (if Genz-99067 \[active moiety of eliglustat in plasma\] trough plasma concentration was greater than or equal to \[\>=\] 5 ng/mL on Day 10) or eliglustat 100 mg BID (if Genz-99067 trough plasma concentration was less than \[\<\] 5 ng/mL), from day 20 to Year 4. After primary completion date (Week 52) participants underwent treatment interruption period of approximately 2 weeks before continuing the same treatment up to Year 9. Participant receiving 100 mg BID could be considered for a further dose increase to 150 mg BID at Week 24 if they met certain criteria (for example, had been on treatment for at least 24 months, had not reached therapeutic goals established for participants receiving Cerezyme, and if all other causes for lack of treatment effect had been evaluated and ruled out).
Percent Change From Baseline in Biomarker (Chitotriosidase) Level at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Change at Year 1 (n=20)	-49.9 percent change Standard Deviation 17.17
Percent Change From Baseline in Biomarker (Chitotriosidase) Level at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Change at Year 4 (n=17)	-79.2 percent change Standard Deviation 16.18
Percent Change From Baseline in Biomarker (Chitotriosidase) Level at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Change at Year 5 (n=17)	-76.7 percent change Standard Deviation 19.16
Percent Change From Baseline in Biomarker (Chitotriosidase) Level at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Change at Year 6 (n=17)	-74.1 percent change Standard Deviation 26.97
Percent Change From Baseline in Biomarker (Chitotriosidase) Level at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Change at Year 2 (n=18)	-73.5 percent change Standard Deviation 14.93
Percent Change From Baseline in Biomarker (Chitotriosidase) Level at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Change at Year 3 (n=17)	-76.4 percent change Standard Deviation 12.78
Percent Change From Baseline in Biomarker (Chitotriosidase) Level at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Change at Year 7 (n=17)	-75.5 percent change Standard Deviation 27.88
Percent Change From Baseline in Biomarker (Chitotriosidase) Level at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Change at Year 8 (n=14)	-72.5 percent change Standard Deviation 29.20
Percent Change From Baseline in Biomarker (Chitotriosidase) Level at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Change at Year 9 (n=4)	-61.2 percent change Standard Deviation 36.45
Percent Change From Baseline in Biomarker (Chitotriosidase) Level at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Change at End of Study (n=17)	-69.8 percent change Standard Deviation 36.59

SECONDARY outcome

Timeframe: Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study (Up to Year 9)

The SF-36 questionnaire, version 2, investigates the participant's health-related quality of life (HRQL). It is a 36-item questionnaire measuring 8 domains (physical functioning, role-physical, bodily pain, general health, vitality, social functioning, role emotional, and mental health). Each domain score ranges from 0 (worst) to 100 (best), with higher scores reflecting best health-related quality of life. Two summary scale scores were computed from the 8 domain scores: the Physical Component Summary and the Mental Component Summary. Score range for both summary scale ranges from 0 (worst) to 100 (best), with higher scores reflecting best health-related quality of life.

Outcome measures

Outcome measures
Measure	Eliglustat n=26 Participants Eliglustat (Genz-112638) capsule as single 50 mg dose on Day 1 then eliglustat 50 mg BID from Day 2 to Day 19, and then either eliglustat 50 mg BID (if Genz-99067 \[active moiety of eliglustat in plasma\] trough plasma concentration was greater than or equal to \[\>=\] 5 ng/mL on Day 10) or eliglustat 100 mg BID (if Genz-99067 trough plasma concentration was less than \[\<\] 5 ng/mL), from day 20 to Year 4. After primary completion date (Week 52) participants underwent treatment interruption period of approximately 2 weeks before continuing the same treatment up to Year 9. Participant receiving 100 mg BID could be considered for a further dose increase to 150 mg BID at Week 24 if they met certain criteria (for example, had been on treatment for at least 24 months, had not reached therapeutic goals established for participants receiving Cerezyme, and if all other causes for lack of treatment effect had been evaluated and ruled out).
Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study General Health: Change at Year 6 (n= 19)	12.83 units on a scale Standard Deviation 23.443
Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study Mental Health: Change at Year 6 (n= 19)	5.53 units on a scale Standard Deviation 22.785
Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study Physical Functioning: Change at Year 1 (n=22)	8.41 units on a scale Standard Deviation 14.670
Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study Physical Functioning: Change at Year 2 (n=20)	9.50 units on a scale Standard Deviation 17.837
Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study Physical Functioning: Change at Year 3 (n=17)	12.65 units on a scale Standard Deviation 18.296
Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study Physical Functioning: Change at Year 4 (n=19)	11.32 units on a scale Standard Deviation 16.231
Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study Physical Functioning: Change at Year 5 (n=19)	10.76 units on a scale Standard Deviation 19.169
Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study Physical Functioning: Change at Year 6 (n=19)	12.63 units on a scale Standard Deviation 17.589
Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study Physical Functioning: Change at Year 7 (n=19)	11.58 units on a scale Standard Deviation 17.955
Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study Physical Functioning: Change at Year 8 (n=16)	11.56 units on a scale Standard Deviation 17.485
Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study Physical Functioning: Change at Year 9 (n=4)	1.25 units on a scale Standard Deviation 2.500
Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study Physical Functioning:Change at End of Study (n=19)	9.28 units on a scale Standard Deviation 17.085
Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study Role - Physical: Change at Year 1 (n=22)	4.55 units on a scale Standard Deviation 22.091
Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study Role - Physical: Change at Year 2 (n=20)	5.31 units on a scale Standard Deviation 27.824
Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study Role - Physical: Change at Year 3 (n=17)	9.56 units on a scale Standard Deviation 24.717
Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study Role - Physical: Change at Year 4 (n=19)	6.58 units on a scale Standard Deviation 26.391
Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study Role - Physical: Change at Year 5 (n=19)	7.57 units on a scale Standard Deviation 28.227
Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study Role - Physical: Change at Year 6 (n=19)	8.88 units on a scale Standard Deviation 25.964
Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study Role - Physical: Change at Year 7 (n=19)	7.89 units on a scale Standard Deviation 25.331
Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study Role - Physical: Change at Year 8 (n=16)	8.59 units on a scale Standard Deviation 29.393
Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study Role - Physical: Change at Year 9 (n=4)	-1.56 units on a scale Standard Deviation 18.663
Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study Role - Physical: Change at End of Study (n= 19)	10.53 units on a scale Standard Deviation 24.211
Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study Bodily Pain: Change at Year 1 (n= 22)	-0.77 units on a scale Standard Deviation 21.307
Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study Bodily Pain: Change at Year 2 (n= 20)	4.40 units on a scale Standard Deviation 25.050
Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study Bodily Pain: Change at Year 3 (n= 17)	4.24 units on a scale Standard Deviation 21.297
Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study Bodily Pain: Change at Year 4 (n= 19)	-2.53 units on a scale Standard Deviation 25.202
Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study Bodily Pain: Change at Year 5 (n= 19)	0.37 units on a scale Standard Deviation 23.104
Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study Bodily Pain: Change at Year 6 (n= 19)	-2.32 units on a scale Standard Deviation 26.268
Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study Bodily Pain: Change at Year 7 (n= 19)	1.74 units on a scale Standard Deviation 27.777
Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study Bodily Pain: Change at Year 8 (n= 16)	1.44 units on a scale Standard Deviation 25.532
Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study Bodily Pain: Change at Year 9 (n= 4)	1.00 units on a scale Standard Deviation 19.630
Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study Bodily Pain: Change at End of Study (n= 19)	6.11 units on a scale Standard Deviation 29.603
Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study General Health: Change at Year 1 (n= 22)	10.36 units on a scale Standard Deviation 19.822
Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study General Health: Change at Year 2 (n= 20)	11.30 units on a scale Standard Deviation 24.262
Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study General Health: Change at Year 3 (n= 17)	16.94 units on a scale Standard Deviation 23.485
Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study General Health: Change at Year 4 (n= 19)	15.53 units on a scale Standard Deviation 23.136
Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study General Health: Change at Year 5 (n= 19)	13.58 units on a scale Standard Deviation 23.710
Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study General Health: Change at Year 7 (n= 19)	15.89 units on a scale Standard Deviation 23.278
Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study General Health: Change at Year 8 (n= 16)	18.19 units on a scale Standard Deviation 20.140
Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study General Health: Change at Year 9 (n= 4)	11.25 units on a scale Standard Deviation 11.087
Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study General Health: Change at End of Study (n= 19)	15.00 units on a scale Standard Deviation 21.422
Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study Vitality: Change at Year 1 (n= 22)	5.68 units on a scale Standard Deviation 17.242
Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study Vitality: Change at Year 2 (n= 20)	7.71 units on a scale Standard Deviation 26.127
Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study Vitality: Change at Year 3 (n= 17)	14.71 units on a scale Standard Deviation 21.188
Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study Vitality: Change at Year 4 (n= 19)	10.20 units on a scale Standard Deviation 20.005
Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study Vitality: Change at Year 5 (n= 19)	13.82 units on a scale Standard Deviation 23.623
Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study Vitality: Change at Year 6 (n= 19)	12.50 units on a scale Standard Deviation 23.936
Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study Vitality: Change at Year 7 (n= 19)	7.57 units on a scale Standard Deviation 22.782
Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study Vitality: Change at Year 8 (n= 16)	15.23 units on a scale Standard Deviation 23.493
Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study Vitality: Change at Year 9 (n= 4)	3.13 units on a scale Standard Deviation 10.825
Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study Vitality: Change at End of Study (n= 19)	15.13 units on a scale Standard Deviation 21.278
Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study Social Functioning: Change at Year 1 (n= 22)	-0.57 units on a scale Standard Deviation 23.298
Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study Social Functioning: Change at Year 2 (n= 20)	10.00 units on a scale Standard Deviation 26.470
Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study Social Functioning: Change at Year 3 (n= 17)	9.56 units on a scale Standard Deviation 19.024
Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study Social Functioning: Change at Year 4 (n= 19)	3.29 units on a scale Standard Deviation 25.291
Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study Social Functioning: Change at Year 5 (n= 19)	9.21 units on a scale Standard Deviation 23.140
Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study Social Functioning: Change at Year 6 (n= 19)	9.21 units on a scale Standard Deviation 26.628
Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study Social Functioning: Change at Year 7 (n= 19)	8.55 units on a scale Standard Deviation 20.435
Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study Social Functioning: Change at Year 8 (n= 16)	11.72 units on a scale Standard Deviation 24.778
Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study Social Functioning: Change at Year 9 (n= 4)	3.13 units on a scale Standard Deviation 6.250
Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study Social Functioning: Change at End of Study (n= 19)	9.21 units on a scale Standard Deviation 25.291
Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study Role - Emotional: Change at Year 1 (n= 22)	-3.03 units on a scale Standard Deviation 17.733
Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study Role - Emotional: Change at Year 2 (n= 20)	4.58 units on a scale Standard Deviation 20.675
Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study Role - Emotional: Change at Year 3 (n= 17)	2.45 units on a scale Standard Deviation 16.342
Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study Role - Emotional: Change at Year 4 (n= 19)	3.95 units on a scale Standard Deviation 18.916
Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study Role - Emotional: Change at Year 5 (n= 19)	2.19 units on a scale Standard Deviation 22.020
Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study Role - Emotional: Change at Year 6 (n= 19)	3.07 units on a scale Standard Deviation 23.110
Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study Role - Emotional: Change at Year 7 (n= 19)	0.44 units on a scale Standard Deviation 23.485
Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study Role - Emotional: Change at Year 8 (n= 16)	5.21 units on a scale Standard Deviation 19.927
Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study Role - Emotional: Change at Year 9 (n= 4)	-10.42 units on a scale Standard Deviation 12.501
Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study Role- Emotional: Change at End of study (n= 19)	1.32 units on a scale Standard Deviation 22.952
Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study Mental Health: Change at Year 1 (n= 22)	0.23 units on a scale Standard Deviation 17.827
Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study Mental Health: Change at Year 2 (n= 20)	1.75 units on a scale Standard Deviation 19.485
Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study Mental Health: Change at Year 3 (n=17)	7.65 units on a scale Standard Deviation 16.117
Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study Mental Health: Change at Year 4 (n= 19)	4.21 units on a scale Standard Deviation 19.809
Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study Mental Health: Change at Year 5 (n= 19)	6.84 units on a scale Standard Deviation 19.945
Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study Mental Health: Change at Year 7 (n= 19)	6.05 units on a scale Standard Deviation 21.186
Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study Mental Health: Change at Year 8 (n= 16)	7.66 units on a scale Standard Deviation 19.989
Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study Mental Health: Change at Year 9 (n= 4)	3.75 units on a scale Standard Deviation 4.787
Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study Mental Health: Change at End of Study (n=19)	8.68 units on a scale Standard Deviation 20.196
Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study Physical Component Summary:Change at Year 1(n= 22)	3.58 units on a scale Standard Deviation 6.263
Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study Physical Component Summary:Change at Year 2(n= 20)	3.81 units on a scale Standard Deviation 7.968
Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study Physical Component Summary:Change at Year 3(n= 17)	5.17 units on a scale Standard Deviation 7.044
Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study Physical Component Summary:Change at Year 4(n= 19)	3.69 units on a scale Standard Deviation 8.195
Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study Physical Component Summary:Change at Year 5(n= 19)	3.75 units on a scale Standard Deviation 8.430
Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study Physical Component Summary:Change at Year 6(n= 19)	3.88 units on a scale Standard Deviation 6.038
Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study Physical Component Summary:Change at Year 7(n= 19)	4.57 units on a scale Standard Deviation 7.892
Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study Physical Component Summary:Change at Year 8(n= 16)	4.36 units on a scale Standard Deviation 6.946
Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study Physical Component Summary: Change at Year 9 (n=4)	1.98 units on a scale Standard Deviation 5.029
Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study Physical Component Summary: Change at EOS (n= 19)	4.70 units on a scale Standard Deviation 7.120
Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study Mental Component Summary: Change at Year 1 (n= 22)	-1.03 units on a scale Standard Deviation 8.703
Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study Mental Component Summary: Change at Year 2 (n= 20)	2.04 units on a scale Standard Deviation 10.476
Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study Mental Component Summary: Change at Year 3 (n= 17)	3.45 units on a scale Standard Deviation 7.881
Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study Mental Component Summary: Change at Year 4 (n= 19)	2.11 units on a scale Standard Deviation 10.013
Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study Mental Component Summary: Change at Year 5 (n= 19)	3.50 units on a scale Standard Deviation 10.963
Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study Mental Component Summary: Change at Year 6 (n= 19)	3.03 units on a scale Standard Deviation 12.535
Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study Mental Component Summary: Change at Year 7 (n= 19)	1.94 units on a scale Standard Deviation 10.982
Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study Mental Component Summary: Change at Year 8 (n= 16)	4.59 units on a scale Standard Deviation 10.106
Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study Mental Component Summary: Change at Year 9 (n= 4)	-0.52 units on a scale Standard Deviation 1.287
Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study Mental Component Summary: Change at EOS (n=19)	3.73 units on a scale Standard Deviation 11.243

SECONDARY outcome

Timeframe: Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study (Up to Year 9)

The FSS is an instrument consisting of 9 self-administered questions that measures the impact of severity of fatigue symptoms on everyday functioning, based on the recall over the past week. Score range for each question ranges from 1 (minimum) to 7 (maximum), where higher score indicates greater severity. FSS total score was calculated by averaging the results of all questions. Total FSS score ranges from 9 (minimum) to 63 (maximum), where higher scores indicates greater severity.

Outcome measures

Outcome measures
Measure	Eliglustat n=26 Participants Eliglustat (Genz-112638) capsule as single 50 mg dose on Day 1 then eliglustat 50 mg BID from Day 2 to Day 19, and then either eliglustat 50 mg BID (if Genz-99067 \[active moiety of eliglustat in plasma\] trough plasma concentration was greater than or equal to \[\>=\] 5 ng/mL on Day 10) or eliglustat 100 mg BID (if Genz-99067 trough plasma concentration was less than \[\<\] 5 ng/mL), from day 20 to Year 4. After primary completion date (Week 52) participants underwent treatment interruption period of approximately 2 weeks before continuing the same treatment up to Year 9. Participant receiving 100 mg BID could be considered for a further dose increase to 150 mg BID at Week 24 if they met certain criteria (for example, had been on treatment for at least 24 months, had not reached therapeutic goals established for participants receiving Cerezyme, and if all other causes for lack of treatment effect had been evaluated and ruled out).
Change From Baseline in Fatigue Severity Scale (FSS) Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Change at Year 1 (n=17)	-0.56 units on a scale Standard Deviation 1.169
Change From Baseline in Fatigue Severity Scale (FSS) Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Change at Year 2 (n=16)	-0.63 units on a scale Standard Deviation 1.388
Change From Baseline in Fatigue Severity Scale (FSS) Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Change at Year 3 (n=14)	-1.37 units on a scale Standard Deviation 1.983
Change From Baseline in Fatigue Severity Scale (FSS) Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Change at Year 4 (n=16)	-1.41 units on a scale Standard Deviation 1.574
Change From Baseline in Fatigue Severity Scale (FSS) Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Change at Year 5 (n=16)	-1.26 units on a scale Standard Deviation 1.418
Change From Baseline in Fatigue Severity Scale (FSS) Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Change at Year 6 (n=15)	-1.21 units on a scale Standard Deviation 1.681
Change From Baseline in Fatigue Severity Scale (FSS) Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Change at Year 7 (n=16)	-0.97 units on a scale Standard Deviation 1.429
Change From Baseline in Fatigue Severity Scale (FSS) Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Change at Year 8 (n=13)	-1.22 units on a scale Standard Deviation 1.489
Change From Baseline in Fatigue Severity Scale (FSS) Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Change at Year 9 (n=4)	-1.20 units on a scale Standard Deviation 1.192
Change From Baseline in Fatigue Severity Scale (FSS) Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Change at End of Study (n=16)	-1.16 units on a scale Standard Deviation 1.665

SECONDARY outcome

Timeframe: Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study (Up to Year 9)

Bone pain was assessed as a part of Gaucher disease assessment in participants. Participants were categorized as none (no bone pain), very mild bone pain, mild bone pain and moderate bone pain. In this outcome, number of participants with different levels of bone pain at specified time points were reported.

Outcome measures

Outcome measures
Measure	Eliglustat n=26 Participants Eliglustat (Genz-112638) capsule as single 50 mg dose on Day 1 then eliglustat 50 mg BID from Day 2 to Day 19, and then either eliglustat 50 mg BID (if Genz-99067 \[active moiety of eliglustat in plasma\] trough plasma concentration was greater than or equal to \[\>=\] 5 ng/mL on Day 10) or eliglustat 100 mg BID (if Genz-99067 trough plasma concentration was less than \[\<\] 5 ng/mL), from day 20 to Year 4. After primary completion date (Week 52) participants underwent treatment interruption period of approximately 2 weeks before continuing the same treatment up to Year 9. Participant receiving 100 mg BID could be considered for a further dose increase to 150 mg BID at Week 24 if they met certain criteria (for example, had been on treatment for at least 24 months, had not reached therapeutic goals established for participants receiving Cerezyme, and if all other causes for lack of treatment effect had been evaluated and ruled out).
Number of Participants With Bone Pain Levels During the Past 4 Weeks at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Moderate: Year 9 (n=4)	0 participants
Number of Participants With Bone Pain Levels During the Past 4 Weeks at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study None: Baseline (n=26)	23 participants
Number of Participants With Bone Pain Levels During the Past 4 Weeks at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study None: Year 1 (n=22)	17 participants
Number of Participants With Bone Pain Levels During the Past 4 Weeks at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study None: Year 2 (n=20)	17 participants
Number of Participants With Bone Pain Levels During the Past 4 Weeks at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study None: Year 3 (n=19)	14 participants
Number of Participants With Bone Pain Levels During the Past 4 Weeks at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study None: Year 4 (n=19)	14 participants
Number of Participants With Bone Pain Levels During the Past 4 Weeks at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study None: Year 5 (n=19)	16 participants
Number of Participants With Bone Pain Levels During the Past 4 Weeks at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study None: Year 6 (n=19)	17 participants
Number of Participants With Bone Pain Levels During the Past 4 Weeks at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study None: Year 7 (n=19)	15 participants
Number of Participants With Bone Pain Levels During the Past 4 Weeks at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study None: Year 8 (n=16)	14 participants
Number of Participants With Bone Pain Levels During the Past 4 Weeks at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study None: Year 9 (n=4)	4 participants
Number of Participants With Bone Pain Levels During the Past 4 Weeks at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study None: End of Study (n=19)	18 participants
Number of Participants With Bone Pain Levels During the Past 4 Weeks at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Very Mild: Baseline (n=26)	2 participants
Number of Participants With Bone Pain Levels During the Past 4 Weeks at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Very Mild: Year 1 (n=22)	4 participants
Number of Participants With Bone Pain Levels During the Past 4 Weeks at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Very Mild: Year 2 (n=20)	3 participants
Number of Participants With Bone Pain Levels During the Past 4 Weeks at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Very Mild: Year 3 (n=19)	3 participants
Number of Participants With Bone Pain Levels During the Past 4 Weeks at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Very Mild: Year 4 (n=19)	4 participants
Number of Participants With Bone Pain Levels During the Past 4 Weeks at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Very Mild: Year 5 (n=19)	1 participants
Number of Participants With Bone Pain Levels During the Past 4 Weeks at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Very Mild: Year 6 (n=19)	1 participants
Number of Participants With Bone Pain Levels During the Past 4 Weeks at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Very Mild: Year 7 (n=19)	3 participants
Number of Participants With Bone Pain Levels During the Past 4 Weeks at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Very Mild: Year 8 (n=16)	1 participants
Number of Participants With Bone Pain Levels During the Past 4 Weeks at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Very Mild: Year 9 (n=4)	0 participants
Number of Participants With Bone Pain Levels During the Past 4 Weeks at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Very Mild: End of Study (n=19)	1 participants
Number of Participants With Bone Pain Levels During the Past 4 Weeks at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Mild: Baseline (n= 26)	1 participants
Number of Participants With Bone Pain Levels During the Past 4 Weeks at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Mild: Year 1 (n= 22)	1 participants
Number of Participants With Bone Pain Levels During the Past 4 Weeks at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Mild: Year 2 (n= 20)	0 participants
Number of Participants With Bone Pain Levels During the Past 4 Weeks at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Mild: Year 3 (n=19)	2 participants
Number of Participants With Bone Pain Levels During the Past 4 Weeks at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Mild: Year 4 (n=19)	1 participants
Number of Participants With Bone Pain Levels During the Past 4 Weeks at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Mild: Year 5 (n=19)	2 participants
Number of Participants With Bone Pain Levels During the Past 4 Weeks at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Mild: Year 6 (n=19)	1 participants
Number of Participants With Bone Pain Levels During the Past 4 Weeks at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Mild: Year 7 (n=19)	1 participants
Number of Participants With Bone Pain Levels During the Past 4 Weeks at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Mild: Year 8 (n=16)	1 participants
Number of Participants With Bone Pain Levels During the Past 4 Weeks at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Mild: Year 9 (n=4)	0 participants
Number of Participants With Bone Pain Levels During the Past 4 Weeks at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Mild: End of Study (n= 19)	0 participants
Number of Participants With Bone Pain Levels During the Past 4 Weeks at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Moderate: Baseline (n=26)	0 participants
Number of Participants With Bone Pain Levels During the Past 4 Weeks at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Moderate: Year 1 (n=22)	0 participants
Number of Participants With Bone Pain Levels During the Past 4 Weeks at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Moderate: Year 2 (n=20)	0 participants
Number of Participants With Bone Pain Levels During the Past 4 Weeks at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Moderate: Year 3 (n=19)	0 participants
Number of Participants With Bone Pain Levels During the Past 4 Weeks at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Moderate: Year 4 (n=19)	0 participants
Number of Participants With Bone Pain Levels During the Past 4 Weeks at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Moderate: Year 5 (n=19)	0 participants
Number of Participants With Bone Pain Levels During the Past 4 Weeks at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Moderate: Year 6 (n=19)	0 participants
Number of Participants With Bone Pain Levels During the Past 4 Weeks at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Moderate: Year 7 (n=19)	0 participants
Number of Participants With Bone Pain Levels During the Past 4 Weeks at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Moderate: Year 8 (n=16)	0 participants
Number of Participants With Bone Pain Levels During the Past 4 Weeks at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Moderate: End of Study (n=19)	0 participants

SECONDARY outcome

Timeframe: Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study (Up to Year 9)

Mobillity, i.e. ability to walk was assessed as a part of Gaucher disease assessment in participants.In this outcome, number of participants with their different mobility status (unrestricted mobility, walks with difficulty) at specified time points were reported.

Outcome measures

Outcome measures
Measure	Eliglustat n=26 Participants Eliglustat (Genz-112638) capsule as single 50 mg dose on Day 1 then eliglustat 50 mg BID from Day 2 to Day 19, and then either eliglustat 50 mg BID (if Genz-99067 \[active moiety of eliglustat in plasma\] trough plasma concentration was greater than or equal to \[\>=\] 5 ng/mL on Day 10) or eliglustat 100 mg BID (if Genz-99067 trough plasma concentration was less than \[\<\] 5 ng/mL), from day 20 to Year 4. After primary completion date (Week 52) participants underwent treatment interruption period of approximately 2 weeks before continuing the same treatment up to Year 9. Participant receiving 100 mg BID could be considered for a further dose increase to 150 mg BID at Week 24 if they met certain criteria (for example, had been on treatment for at least 24 months, had not reached therapeutic goals established for participants receiving Cerezyme, and if all other causes for lack of treatment effect had been evaluated and ruled out).
Number of Participants With Mobility Status (MS) at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study MS: Unrestricted Mobility: Baseline (n=26)	24 participants
Number of Participants With Mobility Status (MS) at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study MS: Unrestricted Mobility: Year 1 (n=22)	22 participants
Number of Participants With Mobility Status (MS) at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study MS: Unrestricted Mobility: Year 2 (n=20)	20 participants
Number of Participants With Mobility Status (MS) at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study MS: Unrestricted Mobility: Year 3 (n=19)	19 participants
Number of Participants With Mobility Status (MS) at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study MS: Unrestricted Mobility: Year 4 (n=19)	19 participants
Number of Participants With Mobility Status (MS) at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study MS: Unrestricted Mobility: Year 5 (n=19)	19 participants
Number of Participants With Mobility Status (MS) at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study MS: Unrestricted Mobility: Year 6 (n=19)	19 participants
Number of Participants With Mobility Status (MS) at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study MS: Unrestricted Mobility: Year 7 (n=19)	19 participants
Number of Participants With Mobility Status (MS) at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study MS: Unrestricted Mobility: Year 8 (n=16)	16 participants
Number of Participants With Mobility Status (MS) at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study MS: Unrestricted Mobility: Year 9 (n=4)	4 participants
Number of Participants With Mobility Status (MS) at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study MS: Unrestricted Mobility: End of Study (n=19)	18 participants
Number of Participants With Mobility Status (MS) at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study MS: Walks with Difficulty: Baseline (n=26)	2 participants
Number of Participants With Mobility Status (MS) at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study MS: Walks with Difficulty: Year 1 (n=22)	0 participants
Number of Participants With Mobility Status (MS) at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study MS: Walks with Difficulty: Year 2 (n=20)	0 participants
Number of Participants With Mobility Status (MS) at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study MS: Walks with Difficulty: Year 3 (n=19)	0 participants
Number of Participants With Mobility Status (MS) at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study MS: Walks with Difficulty: Year 4 (n=19)	0 participants
Number of Participants With Mobility Status (MS) at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study MS: Walks with Difficulty: Year 5 (n=19)	0 participants
Number of Participants With Mobility Status (MS) at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study MS: Walks with Difficulty: Year 6 (n=19)	0 participants
Number of Participants With Mobility Status (MS) at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study MS: Walks with Difficulty: Year 7 (n=19)	0 participants
Number of Participants With Mobility Status (MS) at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study MS: Walks with Difficulty: Year 8 (n=16)	0 participants
Number of Participants With Mobility Status (MS) at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study MS: Walks with Difficulty: Year 9 (n=4)	0 participants
Number of Participants With Mobility Status (MS) at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study MS: Walks with Difficulty: End of Study (n=19)	1 participants

SECONDARY outcome

Timeframe: Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study (Up to Year 9)

Bone crisis was assessed as a part of Gaucher disease assessment in participants. Acute, excruciating episodic bone pain is characteristic of Gaucher bone crisis, which typically causes debilitation lasting several days or longer and requires treatment with immobilization, hydration, and opioid analgesics. Participants were categorized as 0= no bone crisis, 1= 1 bone crisis, and 2= 2 bone crises during the assessment period. In this outcome, number of participants with 0= no bone crises levels at specified time points were reported.

Outcome measures

Outcome measures
Measure	Eliglustat n=26 Participants Eliglustat (Genz-112638) capsule as single 50 mg dose on Day 1 then eliglustat 50 mg BID from Day 2 to Day 19, and then either eliglustat 50 mg BID (if Genz-99067 \[active moiety of eliglustat in plasma\] trough plasma concentration was greater than or equal to \[\>=\] 5 ng/mL on Day 10) or eliglustat 100 mg BID (if Genz-99067 trough plasma concentration was less than \[\<\] 5 ng/mL), from day 20 to Year 4. After primary completion date (Week 52) participants underwent treatment interruption period of approximately 2 weeks before continuing the same treatment up to Year 9. Participant receiving 100 mg BID could be considered for a further dose increase to 150 mg BID at Week 24 if they met certain criteria (for example, had been on treatment for at least 24 months, had not reached therapeutic goals established for participants receiving Cerezyme, and if all other causes for lack of treatment effect had been evaluated and ruled out).
Number of Participants With No Bone Crisis at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Baseline (n=26)	26 participants
Number of Participants With No Bone Crisis at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Year 1 (n=22)	22 participants
Number of Participants With No Bone Crisis at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Year 2 (n=20)	20 participants
Number of Participants With No Bone Crisis at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Year 3 (n=19)	19 participants
Number of Participants With No Bone Crisis at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Year 4 (n=19)	19 participants
Number of Participants With No Bone Crisis at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Year 5 (n=19)	19 participants
Number of Participants With No Bone Crisis at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Year 6 (n=19)	19 participants
Number of Participants With No Bone Crisis at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Year 7 (n=19)	19 participants
Number of Participants With No Bone Crisis at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Year 8 (n=16)	16 participants
Number of Participants With No Bone Crisis at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Year 9 (n=4)	4 participants
Number of Participants With No Bone Crisis at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study End of Study (n=19)	19 participants

SECONDARY outcome

Timeframe: Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and at End of Study (up to Year 9)

Bone marrow infiltration assessments were designed to evaluate improvements in dark marrow using MRI. Each MRI assessment was performed for both femurs and consisted of reviewing 6 different zones (the femoral head, greater trochanter, intertrochanteric region, shaft, distal metaphysis, and condyles). MRI images recorded dark marrow for each zone as either present or not present at baseline. In this outcome, number of participants (for whom dark marrow was present at baseline) with improvement from baseline in dark marrow at each specified time point were reported.

Outcome measures

Outcome measures
Measure	Eliglustat n=26 Participants Eliglustat (Genz-112638) capsule as single 50 mg dose on Day 1 then eliglustat 50 mg BID from Day 2 to Day 19, and then either eliglustat 50 mg BID (if Genz-99067 \[active moiety of eliglustat in plasma\] trough plasma concentration was greater than or equal to \[\>=\] 5 ng/mL on Day 10) or eliglustat 100 mg BID (if Genz-99067 trough plasma concentration was less than \[\<\] 5 ng/mL), from day 20 to Year 4. After primary completion date (Week 52) participants underwent treatment interruption period of approximately 2 weeks before continuing the same treatment up to Year 9. Participant receiving 100 mg BID could be considered for a further dose increase to 150 mg BID at Week 24 if they met certain criteria (for example, had been on treatment for at least 24 months, had not reached therapeutic goals established for participants receiving Cerezyme, and if all other causes for lack of treatment effect had been evaluated and ruled out).
Bone Marrow Infiltration: Number of Participants With Improvement From Baseline in Dark Marrow at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and at End of Study (EOS) Intertrochanteric Regions: Improved: Year 2 (n=19)	3 Participants
Bone Marrow Infiltration: Number of Participants With Improvement From Baseline in Dark Marrow at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and at End of Study (EOS) Shaft: Improved: Year 7 (n= 17)	5 Participants
Bone Marrow Infiltration: Number of Participants With Improvement From Baseline in Dark Marrow at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and at End of Study (EOS) Distal Metaphysis: Improved: Year 3 (n=18)	4 Participants
Bone Marrow Infiltration: Number of Participants With Improvement From Baseline in Dark Marrow at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and at End of Study (EOS) Distal Metaphysis: Improved: Year 5 (n=18)	7 Participants
Bone Marrow Infiltration: Number of Participants With Improvement From Baseline in Dark Marrow at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and at End of Study (EOS) Distal Metaphysis: Improved: End of Study (n=12)	6 Participants
Bone Marrow Infiltration: Number of Participants With Improvement From Baseline in Dark Marrow at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and at End of Study (EOS) Condyles: Improved: Year 5 (n=8)	2 Participants
Bone Marrow Infiltration: Number of Participants With Improvement From Baseline in Dark Marrow at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and at End of Study (EOS) Femoral Head: Improved: Year 5: (n=13)	4 Participants
Bone Marrow Infiltration: Number of Participants With Improvement From Baseline in Dark Marrow at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and at End of Study (EOS) Femoral Head: Improved: Year 1: (n=16)	3 Participants
Bone Marrow Infiltration: Number of Participants With Improvement From Baseline in Dark Marrow at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and at End of Study (EOS) Femoral Head: Improved: Year 2: (n=14)	2 Participants
Bone Marrow Infiltration: Number of Participants With Improvement From Baseline in Dark Marrow at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and at End of Study (EOS) Femoral Head: Improved: Year 3: (n=13)	2 Participants
Bone Marrow Infiltration: Number of Participants With Improvement From Baseline in Dark Marrow at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and at End of Study (EOS) Femoral Head: Improved: Year 4: (n=13)	2 Participants
Bone Marrow Infiltration: Number of Participants With Improvement From Baseline in Dark Marrow at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and at End of Study (EOS) Femoral Head: Improved: Year 6: (n=12)	5 Participants
Bone Marrow Infiltration: Number of Participants With Improvement From Baseline in Dark Marrow at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and at End of Study (EOS) Femoral Head: Improved: Year 7: (n=13)	5 Participants
Bone Marrow Infiltration: Number of Participants With Improvement From Baseline in Dark Marrow at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and at End of Study (EOS) Shaft: Improved: Year 2 (n= 18)	3 Participants
Bone Marrow Infiltration: Number of Participants With Improvement From Baseline in Dark Marrow at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and at End of Study (EOS) Shaft: Improved: Year 3 (n= 17)	3 Participants
Bone Marrow Infiltration: Number of Participants With Improvement From Baseline in Dark Marrow at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and at End of Study (EOS) Femoral Head: Improved: Year 8: (n=13)	5 Participants
Bone Marrow Infiltration: Number of Participants With Improvement From Baseline in Dark Marrow at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and at End of Study (EOS) Femoral Head: Improved: End of Study: (n=8)	3 Participants
Bone Marrow Infiltration: Number of Participants With Improvement From Baseline in Dark Marrow at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and at End of Study (EOS) Greater Trochanter: Improved: Year 1 (n=8)	3 Participants
Bone Marrow Infiltration: Number of Participants With Improvement From Baseline in Dark Marrow at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and at End of Study (EOS) Greater Trochanter: Improved: Year 2 (n=7)	2 Participants
Bone Marrow Infiltration: Number of Participants With Improvement From Baseline in Dark Marrow at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and at End of Study (EOS) Greater Trochanter: Improved: Year 3 (n=6)	3 Participants
Bone Marrow Infiltration: Number of Participants With Improvement From Baseline in Dark Marrow at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and at End of Study (EOS) Greater Trochanter: Improved: Year 4 (n=6)	3 Participants
Bone Marrow Infiltration: Number of Participants With Improvement From Baseline in Dark Marrow at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and at End of Study (EOS) Greater Trochanter: Improved: Year 5 (n=7)	3 Participants
Bone Marrow Infiltration: Number of Participants With Improvement From Baseline in Dark Marrow at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and at End of Study (EOS) Greater Trochanter: Improved: Year 6 (n=6)	1 Participants
Bone Marrow Infiltration: Number of Participants With Improvement From Baseline in Dark Marrow at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and at End of Study (EOS) Greater Trochanter: Improved: Year 7 (n=7)	1 Participants
Bone Marrow Infiltration: Number of Participants With Improvement From Baseline in Dark Marrow at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and at End of Study (EOS) Greater Trochanter: Improved: Year 8 (n=7)	1 Participants
Bone Marrow Infiltration: Number of Participants With Improvement From Baseline in Dark Marrow at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and at End of Study (EOS) Greater Trochanter: Improved: End of Study (n=4)	0 Participants
Bone Marrow Infiltration: Number of Participants With Improvement From Baseline in Dark Marrow at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and at End of Study (EOS) Intertrochanteric Regions: Improved: Year 1 (n=21)	1 Participants
Bone Marrow Infiltration: Number of Participants With Improvement From Baseline in Dark Marrow at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and at End of Study (EOS) Intertrochanteric Regions: Improved: Year 3 (n=18)	3 Participants
Bone Marrow Infiltration: Number of Participants With Improvement From Baseline in Dark Marrow at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and at End of Study (EOS) Intertrochanteric Regions: Improved: Year 4 (n=18)	3 Participants
Bone Marrow Infiltration: Number of Participants With Improvement From Baseline in Dark Marrow at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and at End of Study (EOS) Intertrochanteric Regions: Improved: Year 5 (n=18)	5 Participants
Bone Marrow Infiltration: Number of Participants With Improvement From Baseline in Dark Marrow at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and at End of Study (EOS) Intertrochanteric Regions: Improved: Year 6 (n=17)	6 Participants
Bone Marrow Infiltration: Number of Participants With Improvement From Baseline in Dark Marrow at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and at End of Study (EOS) Intertrochanteric Regions: Improved: Year 7 (n=18)	6 Participants
Bone Marrow Infiltration: Number of Participants With Improvement From Baseline in Dark Marrow at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and at End of Study (EOS) Intertrochanteric Regions: Improved: Year 8 (n=17)	7 Participants
Bone Marrow Infiltration: Number of Participants With Improvement From Baseline in Dark Marrow at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and at End of Study (EOS) Intertrochanteric Regions: Improved: EOS (n=12)	5 Participants
Bone Marrow Infiltration: Number of Participants With Improvement From Baseline in Dark Marrow at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and at End of Study (EOS) Shaft: Improved: Year 1 (n= 20)	1 Participants
Bone Marrow Infiltration: Number of Participants With Improvement From Baseline in Dark Marrow at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and at End of Study (EOS) Shaft: Improved: Year 4 (n= 17)	3 Participants
Bone Marrow Infiltration: Number of Participants With Improvement From Baseline in Dark Marrow at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and at End of Study (EOS) Shaft: Improved: Year 5 (n= 17)	4 Participants
Bone Marrow Infiltration: Number of Participants With Improvement From Baseline in Dark Marrow at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and at End of Study (EOS) Shaft: Improved: Year 6 (n= 16)	5 Participants
Bone Marrow Infiltration: Number of Participants With Improvement From Baseline in Dark Marrow at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and at End of Study (EOS) Shaft: Improved: Year 8 (n= 16)	5 Participants
Bone Marrow Infiltration: Number of Participants With Improvement From Baseline in Dark Marrow at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and at End of Study (EOS) Shaft: Improved: End of Study (n=11)	4 Participants
Bone Marrow Infiltration: Number of Participants With Improvement From Baseline in Dark Marrow at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and at End of Study (EOS) Distal Metaphysis: Improved: Year 1 (n=21)	2 Participants
Bone Marrow Infiltration: Number of Participants With Improvement From Baseline in Dark Marrow at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and at End of Study (EOS) Distal Metaphysis: Improved: Year 2 (n=19)	3 Participants
Bone Marrow Infiltration: Number of Participants With Improvement From Baseline in Dark Marrow at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and at End of Study (EOS) Distal Metaphysis: Improved: Year 4 (n=18)	3 Participants
Bone Marrow Infiltration: Number of Participants With Improvement From Baseline in Dark Marrow at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and at End of Study (EOS) Distal Metaphysis: Improved: Year 6 (n=17)	8 Participants
Bone Marrow Infiltration: Number of Participants With Improvement From Baseline in Dark Marrow at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and at End of Study (EOS) Distal Metaphysis: Improved: Year 7 (n=18)	8 Participants
Bone Marrow Infiltration: Number of Participants With Improvement From Baseline in Dark Marrow at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and at End of Study (EOS) Distal Metaphysis: Improved: Year 8 (n=17)	8 Participants
Bone Marrow Infiltration: Number of Participants With Improvement From Baseline in Dark Marrow at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and at End of Study (EOS) Condyles: Improved: Year 1 (n=11)	3 Participants
Bone Marrow Infiltration: Number of Participants With Improvement From Baseline in Dark Marrow at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and at End of Study (EOS) Condyles: Improved: Year 2 (n=9)	1 Participants
Bone Marrow Infiltration: Number of Participants With Improvement From Baseline in Dark Marrow at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and at End of Study (EOS) Condyles: Improved: Year 3 (n=8)	1 Participants
Bone Marrow Infiltration: Number of Participants With Improvement From Baseline in Dark Marrow at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and at End of Study (EOS) Condyles: Improved: Year 4 (n=8)	1 Participants
Bone Marrow Infiltration: Number of Participants With Improvement From Baseline in Dark Marrow at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and at End of Study (EOS) Condyles: Improved: Year 6 (n=7)	2 Participants
Bone Marrow Infiltration: Number of Participants With Improvement From Baseline in Dark Marrow at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and at End of Study (EOS) Condyles: Improved: Year 7 (n=8)	2 Participants
Bone Marrow Infiltration: Number of Participants With Improvement From Baseline in Dark Marrow at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and at End of Study (EOS) Condyles: Improved: Year 8 (n=8)	2 Participants
Bone Marrow Infiltration: Number of Participants With Improvement From Baseline in Dark Marrow at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and at End of Study (EOS) Condyles: Improved: End of Study (n=5)	2 Participants

SECONDARY outcome

Timeframe: Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study (up to Year 9)

Images of the lumbar spine and femur were obtained by dual energy X-ray absorptiometry (DXA) to determine T-score for each bone area and total bone mineral density. T-scores compares participant's bone density with that of healthy young participant of same gender. The T-score bone density categories were: normal (score \>-1), osteopenia (score -2.5 to \<=-1), and osteoporosis (score \<= -2.5).

Outcome measures

Outcome measures
Measure	Eliglustat n=26 Participants Eliglustat (Genz-112638) capsule as single 50 mg dose on Day 1 then eliglustat 50 mg BID from Day 2 to Day 19, and then either eliglustat 50 mg BID (if Genz-99067 \[active moiety of eliglustat in plasma\] trough plasma concentration was greater than or equal to \[\>=\] 5 ng/mL on Day 10) or eliglustat 100 mg BID (if Genz-99067 trough plasma concentration was less than \[\<\] 5 ng/mL), from day 20 to Year 4. After primary completion date (Week 52) participants underwent treatment interruption period of approximately 2 weeks before continuing the same treatment up to Year 9. Participant receiving 100 mg BID could be considered for a further dose increase to 150 mg BID at Week 24 if they met certain criteria (for example, had been on treatment for at least 24 months, had not reached therapeutic goals established for participants receiving Cerezyme, and if all other causes for lack of treatment effect had been evaluated and ruled out).
Lumbar Spine and Femur T-Scores for Bone Mineral Density (BMD) at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Femur T-Scores: Year 1 (n= 19)	-0.24 T-Score Standard Deviation 0.954
Lumbar Spine and Femur T-Scores for Bone Mineral Density (BMD) at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Lumbar Spine T-Score: Baseline (n= 25)	-1.85 T-Score Standard Deviation 1.094
Lumbar Spine and Femur T-Scores for Bone Mineral Density (BMD) at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Lumbar Spine T-Score: Year 1 (n= 20)	-1.43 T-Score Standard Deviation 1.015
Lumbar Spine and Femur T-Scores for Bone Mineral Density (BMD) at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Lumbar Spine T-Score: Year 2 (n= 17)	-0.93 T-Score Standard Deviation 1.268
Lumbar Spine and Femur T-Scores for Bone Mineral Density (BMD) at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Lumbar Spine T-Score: Year 3 (n= 15)	-1.09 T-Score Standard Deviation 1.143
Lumbar Spine and Femur T-Scores for Bone Mineral Density (BMD) at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Lumbar Spine T-Score: Year 4 (n= 15)	-0.88 T-Score Standard Deviation 1.258
Lumbar Spine and Femur T-Scores for Bone Mineral Density (BMD) at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Lumbar Spine T-Score: Year 5 (n= 15)	-0.79 T-Score Standard Deviation 1.116
Lumbar Spine and Femur T-Scores for Bone Mineral Density (BMD) at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Lumbar Spine T-Score: Year 6 (n= 15)	-0.69 T-Score Standard Deviation 1.313
Lumbar Spine and Femur T-Scores for Bone Mineral Density (BMD) at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Lumbar Spine T-Score: Year 7 (n= 15)	-0.59 T-Score Standard Deviation 1.265
Lumbar Spine and Femur T-Scores for Bone Mineral Density (BMD) at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Lumbar Spine T-Score: Year 8 (n= 14)	-0.59 T-Score Standard Deviation 1.294
Lumbar Spine and Femur T-Scores for Bone Mineral Density (BMD) at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Lumbar Spine T-Score: Year 9 (n= 4)	0.00 T-Score Standard Deviation 0.753
Lumbar Spine and Femur T-Scores for Bone Mineral Density (BMD) at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Lumbar Spine T-Score: End of Study (n= 6)	-0.35 T-Score Standard Deviation 1.613
Lumbar Spine and Femur T-Scores for Bone Mineral Density (BMD) at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Femur T-Scores: Baseline (n= 23)	-0.47 T-Score Standard Deviation 0.894
Lumbar Spine and Femur T-Scores for Bone Mineral Density (BMD) at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Femur T-Scores: Year 2 (n= 15)	-0.09 T-Score Standard Deviation 0.991
Lumbar Spine and Femur T-Scores for Bone Mineral Density (BMD) at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Femur T-Scores: Year 3 (n= 13)	0.16 T-Score Standard Deviation 0.987
Lumbar Spine and Femur T-Scores for Bone Mineral Density (BMD) at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Femur T-Scores: Year 4 (n= 13)	0.13 T-Score Standard Deviation 1.044
Lumbar Spine and Femur T-Scores for Bone Mineral Density (BMD) at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Femur T-Scores: Year 5 (n= 13)	0.14 T-Score Standard Deviation 1.016
Lumbar Spine and Femur T-Scores for Bone Mineral Density (BMD) at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Femur T-Scores: Year 6 (n= 13)	0.19 T-Score Standard Deviation 0.946
Lumbar Spine and Femur T-Scores for Bone Mineral Density (BMD) at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Femur T-Scores: Year 7 (n= 13)	0.12 T-Score Standard Deviation 1.021
Lumbar Spine and Femur T-Scores for Bone Mineral Density (BMD) at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Femur T-Scores: Year 8 (n= 12)	0.33 T-Score Standard Deviation 0.907
Lumbar Spine and Femur T-Scores for Bone Mineral Density (BMD) at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Femur T-Scores: Year 9 (n= 4)	0.33 T-Score Standard Deviation 0.618
Lumbar Spine and Femur T-Scores for Bone Mineral Density (BMD) at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Femur T-Scores: End of Study (n= 6)	0.18 T-Score Standard Deviation 1.278

SECONDARY outcome

Timeframe: Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study (up to Year 9)

Images of the lumbar spine and 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

Outcome measures
Measure	Eliglustat n=26 Participants Eliglustat (Genz-112638) capsule as single 50 mg dose on Day 1 then eliglustat 50 mg BID from Day 2 to Day 19, and then either eliglustat 50 mg BID (if Genz-99067 \[active moiety of eliglustat in plasma\] trough plasma concentration was greater than or equal to \[\>=\] 5 ng/mL on Day 10) or eliglustat 100 mg BID (if Genz-99067 trough plasma concentration was less than \[\<\] 5 ng/mL), from day 20 to Year 4. After primary completion date (Week 52) participants underwent treatment interruption period of approximately 2 weeks before continuing the same treatment up to Year 9. Participant receiving 100 mg BID could be considered for a further dose increase to 150 mg BID at Week 24 if they met certain criteria (for example, had been on treatment for at least 24 months, had not reached therapeutic goals established for participants receiving Cerezyme, and if all other causes for lack of treatment effect had been evaluated and ruled out).
Lumbar Spine and Femur Z-Scores for BMD at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Lumbar Spine Z-Score: Year 3 (n= 15)	-0.63 Z-Score Standard Deviation 0.984
Lumbar Spine and Femur Z-Scores for BMD at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Lumbar Spine Z-Score: Year 4 (n= 15)	-0.48 Z-Score Standard Deviation 1.073
Lumbar Spine and Femur Z-Scores for BMD at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Lumbar Spine Z-Score: Year 7 (n= 15)	-0.14 Z-Score Standard Deviation 1.053
Lumbar Spine and Femur Z-Scores for BMD at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Lumbar Spine Z-Score: Baseline (n= 25)	-1.49 Z-Score Standard Deviation 1.055
Lumbar Spine and Femur Z-Scores for BMD at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Lumbar Spine Z-Score: Year 1 (n= 20)	-1.11 Z-Score Standard Deviation 0.961
Lumbar Spine and Femur Z-Scores for BMD at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Lumbar Spine Z-Score: Year 2 (n= 17)	-0.64 Z-Score Standard Deviation 0.999
Lumbar Spine and Femur Z-Scores for BMD at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Lumbar Spine Z-Score: Year 5 (n= 15)	-0.37 Z-Score Standard Deviation 0.932
Lumbar Spine and Femur Z-Scores for BMD at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Lumbar Spine Z-Score: Year 6 (n= 15)	-0.27 Z-Score Standard Deviation 1.100
Lumbar Spine and Femur Z-Scores for BMD at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Lumbar Spine Z-Score: Year 8 (n= 14)	-0.29 Z-Score Standard Deviation 1.088
Lumbar Spine and Femur Z-Scores for BMD at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Lumbar Spine Z-Score: Year 9 (n= 4)	-0.05 Z-Score Standard Deviation 0.885
Lumbar Spine and Femur Z-Scores for BMD at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Lumbar Spine Z-Score: End of Study (n= 6)	0.18 Z-Score Standard Deviation 0.968
Lumbar Spine and Femur Z-Scores for BMD at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Femur Z-Scores: Baseline (n= 23)	-0.17 Z-Score Standard Deviation 0.783
Lumbar Spine and Femur Z-Scores for BMD at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Femur Z-Scores: Year 1 (n= 19)	0.04 Z-Score Standard Deviation 0.813
Lumbar Spine and Femur Z-Scores for BMD at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Femur Z-Scores: Year 2 (n= 15)	0.25 Z-Score Standard Deviation 0.787
Lumbar Spine and Femur Z-Scores for BMD at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Femur Z-Scores: Year 3 (n= 13)	0.52 Z-Score Standard Deviation 0.704
Lumbar Spine and Femur Z-Scores for BMD at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Femur Z-Scores: Year 4 (n= 13)	0.48 Z-Score Standard Deviation 0.773
Lumbar Spine and Femur Z-Scores for BMD at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Femur Z-Scores: Year 5 (n= 13)	0.49 Z-Score Standard Deviation 0.741
Lumbar Spine and Femur Z-Scores for BMD at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Femur Z-Scores: Year 6 (n= 13)	0.57 Z-Score Standard Deviation 0.665
Lumbar Spine and Femur Z-Scores for BMD at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Femur Z-Scores: Year 7 (n= 13)	0.52 Z-Score Standard Deviation 0.683
Lumbar Spine and Femur Z-Scores for BMD at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Femur Z-Scores: Year 8 (n= 12)	0.64 Z-Score Standard Deviation 0.693
Lumbar Spine and Femur Z-Scores for BMD at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Femur Z-Scores: Year 9 (n= 4)	0.38 Z-Score Standard Deviation 0.793
Lumbar Spine and Femur Z-Scores for BMD at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study Femur Z-Scores: End of Study (n= 6)	0.73 Z-Score Standard Deviation 0.766

Adverse Events

Eliglustat 50 mg BID

Serious events: 0 serious events

Other events: 5 other events

Deaths: 0 deaths

Eliglustat 100 mg BID

Serious events: 3 serious events

Other events: 18 other events

Deaths: 0 deaths

Eliglustat

Serious events: 5 serious events

Other events: 23 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Eliglustat 50 mg BID n=5 participants at risk Participants who received eliglustat capsule as single 50 mg dose on Day 1 followed by eliglustat 50 mg BID from Day 2 to Year 9.	Eliglustat 100 mg BID n=18 participants at risk Participants who received eliglustat capsule as single 50 mg dose on Day 1 followed by eliglustat 50 mg capsule BID from Day 2 to Day 19 and then eliglustat 100 mg capsule BID from Day 20 to Year 9.	Eliglustat n=26 participants at risk Participants who received eliglustat capsule as a single 50 mg dose on Day 1 then eliglustat 50 mg BID from Day 2 to Day 19, and then either eliglustat 50 mg BID or eliglustat 100 mg BID from Day 20 to Year 9. Participants who discontinued prior to Day 20 dose had their dose group set to missing and are only included in the all participants group. The Eliglustat group contains all participants who were treated with Eliglustat, including 2 participants dosed with 50 mg QD and 1 participant dosed with 150 mg BID for majority of study. Participants who had dose adjustment will be presented in dose group they received for majority of study.
Cardiac disorders Ventricular tachycardia	0.00% 0/5 • Up to Year 9. Safety set: participants who signed informed consent, received at least 1 dose. Data reported as per dose groups (50/100 mg) as well as for all participants. In the event, 1 participant experienced both serious and non-serious forms of same AE, individual was included in numerator (number of participants affected) of each AE table.	0.00% 0/18 • Up to Year 9. Safety set: participants who signed informed consent, received at least 1 dose. Data reported as per dose groups (50/100 mg) as well as for all participants. In the event, 1 participant experienced both serious and non-serious forms of same AE, individual was included in numerator (number of participants affected) of each AE table.	3.8% 1/26 • Up to Year 9. Safety set: participants who signed informed consent, received at least 1 dose. Data reported as per dose groups (50/100 mg) as well as for all participants. In the event, 1 participant experienced both serious and non-serious forms of same AE, individual was included in numerator (number of participants affected) of each AE table.
Gastrointestinal disorders Inguinal hernia	0.00% 0/5 • Up to Year 9. Safety set: participants who signed informed consent, received at least 1 dose. Data reported as per dose groups (50/100 mg) as well as for all participants. In the event, 1 participant experienced both serious and non-serious forms of same AE, individual was included in numerator (number of participants affected) of each AE table.	5.6% 1/18 • Up to Year 9. Safety set: participants who signed informed consent, received at least 1 dose. Data reported as per dose groups (50/100 mg) as well as for all participants. In the event, 1 participant experienced both serious and non-serious forms of same AE, individual was included in numerator (number of participants affected) of each AE table.	3.8% 1/26 • Up to Year 9. Safety set: participants who signed informed consent, received at least 1 dose. Data reported as per dose groups (50/100 mg) as well as for all participants. In the event, 1 participant experienced both serious and non-serious forms of same AE, individual was included in numerator (number of participants affected) of each AE table.
Hepatobiliary disorders Cholecystitis	0.00% 0/5 • Up to Year 9. Safety set: participants who signed informed consent, received at least 1 dose. Data reported as per dose groups (50/100 mg) as well as for all participants. In the event, 1 participant experienced both serious and non-serious forms of same AE, individual was included in numerator (number of participants affected) of each AE table.	5.6% 1/18 • Up to Year 9. Safety set: participants who signed informed consent, received at least 1 dose. Data reported as per dose groups (50/100 mg) as well as for all participants. In the event, 1 participant experienced both serious and non-serious forms of same AE, individual was included in numerator (number of participants affected) of each AE table.	3.8% 1/26 • Up to Year 9. Safety set: participants who signed informed consent, received at least 1 dose. Data reported as per dose groups (50/100 mg) as well as for all participants. In the event, 1 participant experienced both serious and non-serious forms of same AE, individual was included in numerator (number of participants affected) of each AE table.
Injury, poisoning and procedural complications Exposure during pregnancy	0.00% 0/5 • Up to Year 9. Safety set: participants who signed informed consent, received at least 1 dose. Data reported as per dose groups (50/100 mg) as well as for all participants. In the event, 1 participant experienced both serious and non-serious forms of same AE, individual was included in numerator (number of participants affected) of each AE table.	11.1% 2/18 • Up to Year 9. Safety set: participants who signed informed consent, received at least 1 dose. Data reported as per dose groups (50/100 mg) as well as for all participants. In the event, 1 participant experienced both serious and non-serious forms of same AE, individual was included in numerator (number of participants affected) of each AE table.	7.7% 2/26 • Up to Year 9. Safety set: participants who signed informed consent, received at least 1 dose. Data reported as per dose groups (50/100 mg) as well as for all participants. In the event, 1 participant experienced both serious and non-serious forms of same AE, individual was included in numerator (number of participants affected) of each AE table.
Pregnancy, puerperium and perinatal conditions Abortion spontaneous	0.00% 0/5 • Up to Year 9. Safety set: participants who signed informed consent, received at least 1 dose. Data reported as per dose groups (50/100 mg) as well as for all participants. In the event, 1 participant experienced both serious and non-serious forms of same AE, individual was included in numerator (number of participants affected) of each AE table.	5.6% 1/18 • Up to Year 9. Safety set: participants who signed informed consent, received at least 1 dose. Data reported as per dose groups (50/100 mg) as well as for all participants. In the event, 1 participant experienced both serious and non-serious forms of same AE, individual was included in numerator (number of participants affected) of each AE table.	3.8% 1/26 • Up to Year 9. Safety set: participants who signed informed consent, received at least 1 dose. Data reported as per dose groups (50/100 mg) as well as for all participants. In the event, 1 participant experienced both serious and non-serious forms of same AE, individual was included in numerator (number of participants affected) of each AE table.
Reproductive system and breast disorders Ovarian cyst ruptured	0.00% 0/5 • Up to Year 9. Safety set: participants who signed informed consent, received at least 1 dose. Data reported as per dose groups (50/100 mg) as well as for all participants. In the event, 1 participant experienced both serious and non-serious forms of same AE, individual was included in numerator (number of participants affected) of each AE table.	0.00% 0/18 • Up to Year 9. Safety set: participants who signed informed consent, received at least 1 dose. Data reported as per dose groups (50/100 mg) as well as for all participants. In the event, 1 participant experienced both serious and non-serious forms of same AE, individual was included in numerator (number of participants affected) of each AE table.	3.8% 1/26 • Up to Year 9. Safety set: participants who signed informed consent, received at least 1 dose. Data reported as per dose groups (50/100 mg) as well as for all participants. In the event, 1 participant experienced both serious and non-serious forms of same AE, individual was included in numerator (number of participants affected) of each AE table.

Other adverse events

Additional Information

Trial Transparency Team

Sanofi

Email: [email protected]

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