Phase 3b Study to Evaluate Skeletal Response to Eliglustat in Adult Patients Who Completed Phase 2 or Phase 3 Studies

Open Label Interventional Multicenter Phase 3b Study to Evaluate Skeletal Response to Eliglustat in Adult Patients Who Successfully Completed the Phase 2 or Phase 3 Studies

Status

Completed

Phases

Phase 3

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT02536755

Acronym

EXOSKEL

Enrollment

Registered

2015-09-01

Start date

2015-10-27

Completion date

2021-06-24

Last updated

2022-07-15

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Gaucher Disease

Brief summary

Primary Objective: Evaluate long term skeletal response to eliglustat in adult participants who successfully completed one of the Phase 2 or Phase 3 eliglustat studies. Secondary Objective: Evaluate the safety of eliglustat (by serious adverse event continuous monitoring), the quality of life (Short Form-36 Health Survey \[SF-36\]) and biomarkers of Gaucher disease type 1 (GD1) (chitotriosidase, plasma glucosylceramide \[GL-1\] and lyso glucosylceramide \[lyso-GL-1\]) in adult participants who successfully completed one of the Phase 2 or Phase 3 studies.

Detailed description

Study duration for individual participants was to be of minimum 2 years and up to 4 years, or until commercial eliglustat was available to participants through reimbursement. If after 4 years since the beginning of the study eliglustat was not approved and available to participants in participating country, participants in this country might continue to receive study treatment until eliglustat was available to participants through the compassionate use (expanded access) program or was approved and available through reimbursement, whichever came first.

Interventions

DRUGEliglustat, GZ385660

Pharmaceutical form: capsule Route of administration: oral

Study design

Allocation

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Masking

NONE

Eligibility

Sex/Gender

ALL

Age

18 Years to No maximum

Healthy volunteers

Inclusion criteria

: * The participant must have successfully completed the Phase 2 (GZGD00304) or a Phase 3 study (GZGD02507, GZGD02607 or GZGD03109). Successful completion was defined as participants enrolled in one of the above mentioned studies who received eliglustat through the end of the study and completed the end-of-study visit without having discontinued or been withdrawn prematurely. * The participant was willing and able to provide signed informed consent prior to any protocol-required procedures being performed. * Female participants of childbearing potential must have had a documented negative pregnancy test prior to enrollment and while they were receiving eliglustat treatment. * Female participants of childbearing potential must have been willing to practice true abstinence in line with their preferred and usual lifestyle, or used a medically accepted form of contraception (either a barrier method, such as condom or diaphragm + spermicide, or a non-barrier method such as oral, injected, or implanted hormonal methods, or an intra-uterine device or system) while receiving eliglustat.

Exclusion criteria

* The participant was unwilling to comply with the requirements of the protocol. * The participant had received an investigational product (other than eliglustat) within 30 days prior to enrollment. * The participant had received miglustat within the 6 months prior to enrollment. * The participant had documented prior esophageal varices or liver infarction or current liver enzymes (alanine transaminase, aspartate aminotransferase) or total bilirubin greater than (\>)2 times the upper limit of normal, unless the participant had a diagnosis of Gilbert Syndrome. * The participant had any clinically significant disease, other than Gaucher disease, including cardiovascular, renal, hepatic, gastrointestinal, pulmonary, neurologic, endocrine, metabolic (eg, hypokalemia, hypomagnesemia), or psychiatric disease, other medical conditions, or serious intercurrent illnesses that might preclude participation in the study. * The participant was known to have any of the following: cardiac disease (congestive heart failure, recent acute myocardial infarction, bradycardia, heart block, ventricular arrhythmia), long QT syndrome, or current treatment with Class IA or Class III antiarrhythmic medicinal products. * The participant had tested positive for the human immunodeficiency virus antibody, hepatitis C antibody, or hepatitis B surface antigen. * The participant had a history of cancer within 6 months of enrolment, with the exception of basal cell carcinoma. * Participant was a CYP2D6 IM, EM or URM and was taking a strong or moderate CYP2D6 inhibitor concomitantly with a strong or moderate CYP3A inhibitor. * Participant was a CYP2D6 PM having taken a strong CYP3A inhibitor within 2 weeks prior to enrolment. * If a female participant of childbearing potential had a positive pregnancy test (blood β-human chorionic gonadotropin \[β-HCG\]) or was breastfeeding prior to first dosing of eliglustat in this study, the participant could not enroll in the study at that time, but might have been rescreened after the end of the pregnancy, and/or when she was no longer breast feeding, provided rescreening took place before the end of the enrollment period. * Women of childbearing potential who were unwilling or unable to be tested for pregnancy. The above information was not intended to contain all considerations relevant to a participant's potential participation in a clinical trial.

Design outcomes

Primary

Measure	Time frame	Description
Change From Eliglustat Baseline in Bone Biomarker Level: Type 1 Collagen C-Telopeptides (CTx) at Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234	Eliglustat Baseline, Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234 of the current study	CTx, a marker of bone resorption was assayed from plasma. For this outcome measure, baseline refers to the eliglustat baseline, which was defined as participant's status at the time of first dose of eliglustat in the previous Phase 2 or Phase 3 study.
Observed Annual Incidence Rate for Spine and Femur Osteonecrosis at Week 52, 104, 156 and 208	For 52 Weeks (i.e., 1 year),104 Weeks (i.e., 2 year), 156 Weeks (i.e., 3 year) and 208 Weeks (i.e., 4 years)	Observed annual incidence rate was estimated using the total number of events divided by the total years of follow-up in each specified year (for all participants). Osteonecrosis was assessed by bone MRI and X-Ray for spine and by MRI for femur.
Observed Annual Incidence Rate for Spine and Femur Fracture at Week 52, 104, 156 and 208	For 52 Weeks (i.e., 1 year),104 Weeks (i.e., 2 year), 156 Weeks (i.e., 3 year) and 208 Weeks (i.e., 4 years)	Observed annual incidence rate was estimated using the total number of events divided by the total years of follow-up in each specified year (for all participants). Fracture was assessed by bone MRI and X-Ray for spine and by MRI for femur.
Observed Annual Incidence Rate for Spine and Femur Infarcts at Week 52, 104, 156 and 208	For 52 Weeks (i.e., 1 year),104 Weeks (i.e., 2 year), 156 Weeks (i.e., 3 year) and 208 Weeks (i.e., 4 years)	Observed annual incidence rate was estimated using the total number of events divided by the total years of follow-up in each specified year (for all participants). Infarcts were assessed by bone MRI and X-Ray for spine and by MRI for femur.
Observed Annual Incidence Rate for Spine Lytic Lesion at Week 104, and 208	For 104 Weeks (i.e., 2 year), and 208 Weeks (i.e., 4 years)	Observed annual incidence rate was estimated using the total number of events divided by the total years of follow-up in each specified year (for all participants). Lytic Lesions were assessed by bone X-Ray for spine.
Change From Current Study Baseline in Bone Biomarker Level: Macrophage Inflammatory Protein 1 Beta (MIP-1β) at Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234	Study Baseline, Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234	MIP-1β considered a biomarker of active bone disease, was assayed from plasma. For this outcome measure, baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry.
Change From Eliglustat Baseline in Bone Biomarker Level: Macrophage Inflammatory Protein 1 Beta (MIP-1β) at Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234	Eliglustat Baseline, Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234 of the current study	MIP-1β considered a biomarker of active bone disease, was assayed from plasma. For this outcome measure, baseline refers to the eliglustat baseline, which was defined as participant's status at the time of first dose of eliglustat in the previous Phase 2 or Phase 3 study.
Change From Current Study Baseline in Bone Biomarker Level: Procollagen 1 N- Terminal Propeptide (P1NP) at Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234	Study Baseline, Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234	P1NP, a marker of bone formation was assayed from plasma. For this outcome measure, baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry.
Change From Eliglustat Baseline in Bone Biomarker Level: Procollagen 1 N- Terminal Propeptide (P1NP) at Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234	Eliglustat Baseline, Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234 of the current study	P1NP, a marker of bone formation was assayed from plasma. For this outcome measure, baseline refers to the eliglustat baseline, which was defined as participant's status at the time of first dose of eliglustat in the previous Phase 2 or Phase 3 study.
Change From Current Study Baseline in Bone Biomarker Level: Type 1 Collagen C-Telopeptides (CTx) at Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234	Study Baseline, Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234	CTx, a marker of bone resorption was assayed from plasma. For this outcome measure, baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry.
Number of Participants With Mobility Status Assessments at Study Baseline, Weeks 52, 104, 156 and 208	Study Baseline, Weeks 52, 104, 156 and 208	Mobility, i.e., ability to walk was assessed as a part of Gaucher disease assessment in participants. In this outcome measure, number of participants with their different mobility status along with the use of mobility aids (unrestricted mobility, walks with difficulty, walks with orthopaedic aid, requires wheelchair, bedridden) at specified time points were reported. Baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry.
Number of Participants With Bone Pain Levels During the Past 4 Weeks at Study Baseline, Weeks 52, 104, 156 and 208	Study Baseline, Weeks 52, 104, 156 and 208	Bone pain was assessed as a part of Gaucher disease assessment in participants. Participants were categorized as none (no bone pain), very mild bone pain, mild bone pain, moderate bone pain, severe bone pain and extreme bone pain during the past 4 weeks at each specified visit. In this outcome measure, number of participants with different level of bone pain during the past 4 weeks at specified time points were reported. Baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry.
Number of Participants With Bone Crisis at Study Baseline, Weeks 52, 104, 156 and 208	Study Baseline, Weeks 52, 104, 156 and 208	Bone crisis was assessed as a part of Gaucher disease assessment in participants. Acute, excruciating episodic bone pain is characteristic of Gaucher bone crisis, which typically causes periosteal elevation, elevated white blood cell count, fever, or debilitation lasting several days or longer and requires treatment with immobilization of the affected area, and opioid analgesics. Participants were categorized as 0= no bone crisis, 1= 1 bone crisis, 2= 2 bone crisis and \>=3 = more than 3 bone crisis during the assessment period. In this outcome measure, number of participants with different bone crises levels at specified time points were reported. Baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry.
Change From Current Study Baseline in Total Bone Marrow Burden (BMB) Scores at Weeks 52, 104, 156 and 208	Study Baseline, Weeks 52, 104, 156 and 208	Bone marrow burden (BMB) scores indicate the degree of bone marrow infiltration. BMB score was measured using MRI (magnetic resonance imaging (MRI), ranged from 0 (no abnormalities) to 8 points (severe disease) for the lumbar spine and from 0 (no abnormalities) to 8 points (severe disease) for the femurs. The total BMB score was calculated as the sum of scores for femur and lumbar spine regions which ranged from 0 (no abnormalities) to 16 (severe disease) points. A higher BMB score signified more severe bone marrow involvement. For this outcome measure, baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry.
Change From Eliglustat Baseline in Total Bone Marrow Burden (BMB) Scores at Weeks 52, 104, 156 and 208	Eliglustat Baseline, Weeks 52, 104, 156 and 208 of the current study	Bone marrow burden (BMB) scores indicate the degree of bone marrow infiltration was measured using MRI, ranged from 0 (no abnormalities) to 8 points (severe disease) for the lumbar spine and from 0 (no abnormalities) to 8 points (severe disease) for the femurs. The total BMB score was calculated as the sum of scores for femur and lumbar spine regions which ranged from 0 (no abnormalities) to 16 (severe disease) points. A higher BMB score signified more severe bone marrow involvement. For this outcome measure, baseline refers to the eliglustat baseline, which was defined as participant's status at the time of first dose of eliglustat in the previous Phase 2 or Phase 3 study.
Change From Current Study Baseline in Total Spine and Femur Bone Mineral Density (BMD) at Weeks 52, 104, 156 and 208	Study Baseline, Weeks 52, 104, 156 and 208	Bone Mineral Density (BMD) measurements of the spine and bilateral femur were acquired by dual energy X-Ray absorptiometry (DXA) scan. Worst total femur at Baseline refers to the worst diseased left or right femur at Baseline. For this outcome measure, baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry.
Change From Eliglustat Baseline in Total Spine and Femur Bone Mineral Density (BMD) at Weeks 52, 104, 156 and 208	Eliglustat Baseline, Weeks 52, 104, 156 and 208 of the current study	BMD measurements of the spine and bilateral femur were acquired by DXA scan. Worst total femur at Baseline refers to the worst diseased left or right femur at baseline. For this outcome measure, baseline refers to the eliglustat baseline, which was defined as participant's status at the time of first dose of eliglustat in the previous Phase 2 or Phase 3 study.
Change From Current Study Baseline in Spine and Femur Total T-Scores for BMD at Weeks 52, 104, 156 and 208	Study Baseline, Weeks 52, 104, 156 and 208	BMD measurements of the spine and bilateral femur were acquired by DXA scan. The T-score bone density categories were: normal (score \>-1), osteopenia (score -2.5 to \<=-1), and osteoporosis (score \<= -2.5). Worst total femur at Baseline refers to the worst diseased left or right femur at baseline. For this outcome measure, baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry.
Change From Eliglustat Baseline in Spine and Femur Total T-Scores for BMD at Weeks 52, 104, 156 and 208	Eliglustat Baseline, Weeks 52, 104, 156 and 208 of the current study	BMD measurements of the spine and bilateral femur were acquired by DXA scan. The T-score bone density categories were: normal (score \>-1), osteopenia (score -2.5 to \<=-1), and osteoporosis (score \<= -2.5). Worst total femur at Baseline refers to the worst diseased left or right femur at baseline. For this outcome measure, baseline refers to the eliglustat baseline, which was defined as participant's status at the time of first dose of eliglustat in the previous Phase 2 or Phase 3 study.
Change From Current Study Baseline in Spine and Femur Total Z-Scores for BMD at Weeks 52, 104, 156 and 208	Study Baseline, Weeks 52, 104, 156 and 208	BMD measurements of the spine and bilateral femur were acquired by DXA scan. The Z-score bone density categories were: normal (score \>-2) and below normal (score \<=-2). Worst total femur at Baseline refers to the worst diseased left or right femur at baseline. For this outcome measure, baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry.
Change From Eliglustat Baseline in Spine and Femur Total Z-Scores for BMD at Weeks 52, 104, 156 and 208	Eliglustat Baseline, Weeks 52, 104, 156 and 208 of the current study	BMD measurements of the spine and bilateral femur were acquired by DXA scan. The Z-score bone density categories are: normal (score \>-2) and below normal (score \<=-2). Worst total femur at Baseline refers to the worst diseased left or right femur at baseline. For this outcome measure, baseline refers to the eliglustat baseline, which was defined as participant's status at the time of first dose of eliglustat in the previous Phase 2 or Phase 3 study.
Total Number of New or Worsening Osteonecrosis Events for Spine and Femur at Study Baseline, Week 52, 104, 156 and 208	Study Baseline, Weeks 52, 104, 156 and 208	Osteonecrosis was assessed by bone MRI and X-Ray for spine and by MRI for femur. Total number of new or worsening osteonecrosis events among all the participants with corresponding assessment at specified time points were reported in this outcome measure. For this outcome measure, baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry.
Total Number of New or Worsening Fracture Events for Spine and Femur at Study Baseline, Week 52, 104, 156 and 208	Study Baseline, Weeks 52, 104, 156 and 208	Fracture was assessed by bone MRI and X-Ray for spine and by MRI for femur. Total number of new or worsening fracture events among all the participants with corresponding assessment at specified time points were reported in this outcome measure. For this outcome measure, baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry.
Total Number of New or Worsening Infarcts Events for Spine and Femur at Study Baseline, Week 52, 104, 156 and 208	Study Baseline, Weeks 52, 104, 156 and 208	Infarcts were assessed by bone MRI and X-Ray for spine and by MRI for femur. Total number of new or worsening infarcts events among all the participants with corresponding assessment at specified time points were reported in this outcome measure. For this outcome measure, baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry.
Total Number of New or Worsening Lytic Lesions Events for Spine at Study Baseline, Week 104, and 208	Study Baseline, Weeks 104, and 208	Lytic Lesions were assessed by bone X-Ray for spine. Total number of new or worsening lytic lesions events among all the participants with corresponding assessment at specified time points were reported in this outcome measure. For this outcome measure, baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry.

Secondary

Measure	Time frame	Description
Change From Eliglustat Baseline in Gaucher Disease Type 1 (GD1) Biomarker Levels: Chitotriosidase at Week 26, 52, 78, 104, 130, 156, 182, 208 and 234	Eliglustat Baseline, Week 26, 52, 78, 104, 130, 156, 182, 208 and 234 of the current study	Chitotriosidase biomarker was assayed from plasma. Chitotriosidase biomarker levels for participants who were CYP2D6 non-URM was reported in this outcome measure. For this outcome measure, baseline refers to the eliglustat baseline, which was defined as participant's status at the time of first dose of eliglustat in the previous Phase 2 or Phase 3 study. Data for this outcome measure was planned to be collected and analyzed in reference to eliglustat baseline separately for Phase 2, ENGAGE and EDGE studies (GD treatment naïve participants) and from ENCORE study (participants switched from ERT).
Change From Current Study Baseline in GD1 Biomarker Levels: Glucosylceramide (GL-1) at Week 26, 52, 78, 104, 130, 156, 182, 208 and 234	Study Baseline, Week 26, 52, 78, 104, 130, 156, 182, 208 and 234	Glucosylceramide (GL-1) biomarker was assayed from plasma. GL-1 biomarker levels for participants who were CYP2D6 non-URM was reported in this outcome measure. For this outcome measure, baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry.
Change From Eliglustat Baseline in GD1 Biomarker Levels: Glucosylceramide (GL-1) at Week 26, 52, 78, 104, 130, 156, 182, 208 and 234	Eliglustat Baseline, Week 26, 52, 78, 104, 130, 156, 182, 208 and 234 of the current study	Glucosylceramide (GL-1) biomarker was assayed from plasma. GL-1 biomarker levels for participants who were CYP2D6 non-URM was reported in this outcome measure. For this outcome measure, baseline refers to the eliglustat baseline, which was defined as participant's status at the time of first dose of eliglustat in the previous Phase 2 or Phase 3 study. Data for this outcome measure was planned to be collected and analyzed in reference to eliglustat baseline separately for participants from Phase 2, ENGAGE and EDGE studies (GD treatment naïve participants) and from ENCORE study (participants switched from ERT).
Change From Current Study Baseline in GD1 Biomarker Levels: Lyso Glucosylceramide (Lyso-GL-1) at Week 26, 52, 78, 104, 130, 156, 182, 208 and 234	Study Baseline, Week 26, 52, 78, 104, 130, 156, 182, 208 and 234	Lyso-GL-1 biomarker was assayed from plasma. Lyso-GL-1 biomarker levels for participants who were CYP2D6 non-URM was reported in this outcome measure. For this outcome measure, baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry.
Change From Eliglustat Baseline in GD1 Biomarker Levels: Lyso Glucosylceramide (Lyso-GL-1) at Week 26, 52, 78, 104, 130, 156, 182, 208 and 234	Eliglustat Baseline, Week 26, 52, 78, 104, 130, 156, 182, 208 and 234 of the current study	Lyso-GL-1 biomarker was assayed from plasma. Lyso-GL-1 biomarker levels for participants who were CYP2D6 non-URM was reported in this outcome measure. For this outcome measure, baseline refers to the eliglustat baseline, which was defined as participant's status at the time of first dose of eliglustat in the previous Phase 2 or Phase 3 study. Data for this outcome measure was planned to be collected and analyzed in reference to eliglustat baseline separately for participants from Phase 2, ENGAGE and EDGE studies (GD treatment naïve participants) and from ENCORE study (participants switched from ERT).
Change From Current Study Baseline in Short Form-36 Health Survey (SF-36) Scores at Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234	Study Baseline, Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234	The 36-Item Short-Form Health Survey (SF-36) is standardized survey evaluating 8 aspects of functional health and well-being. Physical Component Summary (PCS) with 4 sub-scales: physical function, role limitations due to physical problems, bodily pain, and general health perception; and Mental Component Summary (MCS) with 4 sub-scales: vitality, social function, role limitations due to emotional problems, and mental health. Summations of item scores of the same sub-scale give the sub-scale scores, which are transformed into a range from 0 to 100; 0= worst and 100=best outcome. Both PCS and MCS range from 0 to 100 with higher scores indicating better physical and mental health. For this outcome measure, baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry.
Change From Eliglustat Baseline in SF-36 Scores at Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234	Eliglustat Baseline, Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234 of the current study	SF-36 is a standardized survey evaluating 8 aspects of functional health and well-being. PCS with 4 sub-scales: physical function, role limitations due to physical problems, pain, and general health perception; and MCS with 4 sub-scales: vitality, social function, role limitations due to emotional problems, and mental health. Summations of item scores of same sub-scale give the sub-scale scores, which are transformed into range from 0 to 100; 0= worst, and 100=best outcome. Both PCS and MCS range from 0 to 100, higher scores indicating better physical and mental health. For this outcome measure, baseline refers to the eliglustat baseline, which was defined as participant's status at the time of first dose of eliglustat in the previous Phase 2 or Phase 3 study.
Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Treatment-emergent Serious Adverse Events (TESAEs)	From the first administration of the IMP to the last administration of the IMP + 5 days (up to 4 years, or until commercial eliglustat was available to participants through reimbursement or through the compassionate use [expanded access] program)	An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug and did not necessarily had to have a causal relationship with the treatment. A serious adverse event (SAE) was any untoward medical occurrence that at any dose: resulted in death, was life-threatening, required inpatient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, was a congenital anomaly/birth defect, was a medically important event. TEAEs were defined as AEs that developed, worsened or became serious during the treatment-emergent period (time from the first administration of the investigational medicinal product (IMP) to the last administration of the IMP + 5 days).
Change From Current Study Baseline in Gaucher Disease Type 1 (GD1) Biomarker Levels: Chitotriosidase at Week 26, 52, 78, 104, 130, 156, 182, 208 and 234	Study Baseline, Week 26, 52, 78, 104, 130, 156, 182, 208 and 234	Chitotriosidase biomarker was assayed from plasma. Chitotriosidase biomarker levels for participants who were CYP2D6 non-Ultra Rapid Metabolizers (non-URM) was reported in this outcome measure. For this outcome measure, baseline refers to the study baseline, which was defined as status at study entry.

Countries

Canada, Russia, Tunisia

Participant flow

Recruitment details

The study was conducted at 4 active sites in 3 countries. A total of 33 participants were screened between 27 October 2015 and 07 June 2017, of which 2 participants were screen failures as the selection criteria were not met.

Pre-assignment details

A total of 31 participants who completed one of the Phase 2 (GZGD00304 \[NCT00358150\]) or Phase 3 studies (GZGD02507 \[ENGAGE; NCT00891202\], GZGD02607 \[ENCORE; NCT00943111\], or GZGD03109 \[EDGE; NCT01074944\]) were enrolled in this current study (EFC13781) considered as an extension of these previous studies, and received treatment with eliglustat in the current (EFC13781) study.

Participants by arm

Arm	Count
Eliglustat Participants who completed one of the Phase 2 (GZGD00304) or Phase 3 studies (GZGD02507, GZGD02607 or GZGD03109) were enrolled in this current (EFC13781) study. Participants who were CYP2D6 IM, EM and URM received eliglustat 84 mg twice daily and participants who were CYP2D6 PM received eliglustat 84 mg once daily, for duration of minimum 2 years (unless early discontinuation occurred) and up to 4 years, or until commercial eliglustat was available to participants through reimbursement or through the compassionate use (expanded access) program.	31
Total	31

Arm

Count

Eliglustat

Participants who completed one of the Phase 2 (GZGD00304) or Phase 3 studies (GZGD02507, GZGD02607 or GZGD03109) were enrolled in this current (EFC13781) study. Participants who were CYP2D6 IM, EM and URM received eliglustat 84 mg twice daily and participants who were CYP2D6 PM received eliglustat 84 mg once daily, for duration of minimum 2 years (unless early discontinuation occurred) and up to 4 years, or until commercial eliglustat was available to participants through reimbursement or through the compassionate use (expanded access) program.

Total

Withdrawals & dropouts

Period	Reason	FG000
Overall Study	Adverse Event	4

Baseline characteristics

Characteristic	Eliglustat
Age, Continuous	36.8 years STANDARD_DEVIATION 10.3
CYP2D6 metabolizer status CYP2D6 EM	28 Participants
CYP2D6 metabolizer status CYP2D6 IM	1 Participants
CYP2D6 metabolizer status CYP2D6 PM	1 Participants
CYP2D6 metabolizer status CYP2D6 URM	1 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants
Race (NIH/OMB) Asian	0 Participants
Race (NIH/OMB) Black or African American	0 Participants
Race (NIH/OMB) More than one race	0 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants
Race (NIH/OMB) White	31 Participants
Sex: Female, Male Female	17 Participants
Sex: Female, Male Male	14 Participants
Time since first symptom of Gaucher to enrollment in EFC13781	22.6 years STANDARD_DEVIATION 11
Time since initial Gaucher diagnosis to enrollment in EFC13781	15.8 years STANDARD_DEVIATION 9.2

Adverse events

Event type	EG000 affected / at risk
deaths Total, all-cause mortality	0 / 31
other Total, other adverse events	12 / 31
serious Total, serious adverse events	4 / 31

Outcome results

Primary

Change From Current Study Baseline in Bone Biomarker Level: Macrophage Inflammatory Protein 1 Beta (MIP-1β) at Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234

MIP-1β considered a biomarker of active bone disease, was assayed from plasma. For this outcome measure, baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry.

Time frame: Study Baseline, Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234

Population: Analysis was performed on FAS population. Here, number analyzed = participants with available data for each specified category. Baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry.

Arm	Measure	Group	Value (MEAN)	Dispersion
Eliglustat	Change From Current Study Baseline in Bone Biomarker Level: Macrophage Inflammatory Protein 1 Beta (MIP-1β) at Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234	Study Baseline	175.8296 picograms per milliliter (pg/mL)	Standard Deviation 114.6313
Eliglustat	Change From Current Study Baseline in Bone Biomarker Level: Macrophage Inflammatory Protein 1 Beta (MIP-1β) at Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234	Change at Week 130	897.1680 picograms per milliliter (pg/mL)	Standard Deviation 4660.2892
Eliglustat	Change From Current Study Baseline in Bone Biomarker Level: Macrophage Inflammatory Protein 1 Beta (MIP-1β) at Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234	Change at Week 156	1008.8400 picograms per milliliter (pg/mL)	Standard Deviation 5150.4257
Eliglustat	Change From Current Study Baseline in Bone Biomarker Level: Macrophage Inflammatory Protein 1 Beta (MIP-1β) at Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234	Change at Week 182	1007.9880 picograms per milliliter (pg/mL)	Standard Deviation 5177.6248
Eliglustat	Change From Current Study Baseline in Bone Biomarker Level: Macrophage Inflammatory Protein 1 Beta (MIP-1β) at Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234	Change at Week 208	1381.6652 picograms per milliliter (pg/mL)	Standard Deviation 6751.1152
Eliglustat	Change From Current Study Baseline in Bone Biomarker Level: Macrophage Inflammatory Protein 1 Beta (MIP-1β) at Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234	Change at Week 234	6721.9000 picograms per milliliter (pg/mL)	Standard Deviation 20005.3684
Eliglustat	Change From Current Study Baseline in Bone Biomarker Level: Macrophage Inflammatory Protein 1 Beta (MIP-1β) at Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234	Change at Week 26	-36.0593 picograms per milliliter (pg/mL)	Standard Deviation 78.2488
Eliglustat	Change From Current Study Baseline in Bone Biomarker Level: Macrophage Inflammatory Protein 1 Beta (MIP-1β) at Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234	Change at Week 52	-31.4692 picograms per milliliter (pg/mL)	Standard Deviation 97.6425
Eliglustat	Change From Current Study Baseline in Bone Biomarker Level: Macrophage Inflammatory Protein 1 Beta (MIP-1β) at Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234	Change at Week 78	-16.6818 picograms per milliliter (pg/mL)	Standard Deviation 93.2333
Eliglustat	Change From Current Study Baseline in Bone Biomarker Level: Macrophage Inflammatory Protein 1 Beta (MIP-1β) at Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234	Change at Week 104	-33.0217 picograms per milliliter (pg/mL)	Standard Deviation 77.0603

Primary

Change From Current Study Baseline in Bone Biomarker Level: Procollagen 1 N- Terminal Propeptide (P1NP) at Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234

P1NP, a marker of bone formation was assayed from plasma. For this outcome measure, baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry.

Time frame: Study Baseline, Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234

Arm	Measure	Group	Value (MEAN)	Dispersion
Eliglustat	Change From Current Study Baseline in Bone Biomarker Level: Procollagen 1 N- Terminal Propeptide (P1NP) at Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234	Study Baseline	44.3960 micrograms per liter (mcg/L)	Standard Deviation 15.7898
Eliglustat	Change From Current Study Baseline in Bone Biomarker Level: Procollagen 1 N- Terminal Propeptide (P1NP) at Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234	Change at Week 26	-0.6947 micrograms per liter (mcg/L)	Standard Deviation 9.8577
Eliglustat	Change From Current Study Baseline in Bone Biomarker Level: Procollagen 1 N- Terminal Propeptide (P1NP) at Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234	Change at Week 52	2.0917 micrograms per liter (mcg/L)	Standard Deviation 12.4906
Eliglustat	Change From Current Study Baseline in Bone Biomarker Level: Procollagen 1 N- Terminal Propeptide (P1NP) at Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234	Change at Week 78	3.6133 micrograms per liter (mcg/L)	Standard Deviation 16.548
Eliglustat	Change From Current Study Baseline in Bone Biomarker Level: Procollagen 1 N- Terminal Propeptide (P1NP) at Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234	Change at Week 104	1.6300 micrograms per liter (mcg/L)	Standard Deviation 12.2829
Eliglustat	Change From Current Study Baseline in Bone Biomarker Level: Procollagen 1 N- Terminal Propeptide (P1NP) at Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234	Change at Week 130	0.1675 micrograms per liter (mcg/L)	Standard Deviation 14.7345
Eliglustat	Change From Current Study Baseline in Bone Biomarker Level: Procollagen 1 N- Terminal Propeptide (P1NP) at Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234	Change at Week 156	-1.9043 micrograms per liter (mcg/L)	Standard Deviation 11.5887
Eliglustat	Change From Current Study Baseline in Bone Biomarker Level: Procollagen 1 N- Terminal Propeptide (P1NP) at Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234	Change at Week 182	1.6632 micrograms per liter (mcg/L)	Standard Deviation 12.1623
Eliglustat	Change From Current Study Baseline in Bone Biomarker Level: Procollagen 1 N- Terminal Propeptide (P1NP) at Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234	Change at Week 208	0.1354 micrograms per liter (mcg/L)	Standard Deviation 14.6972
Eliglustat	Change From Current Study Baseline in Bone Biomarker Level: Procollagen 1 N- Terminal Propeptide (P1NP) at Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234	Change at Week 234	3.4467 micrograms per liter (mcg/L)	Standard Deviation 12.1002

Primary

Change From Current Study Baseline in Bone Biomarker Level: Type 1 Collagen C-Telopeptides (CTx) at Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234

CTx, a marker of bone resorption was assayed from plasma. For this outcome measure, baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry.

Time frame: Study Baseline, Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234

Arm	Measure	Group	Value (MEAN)	Dispersion
Eliglustat	Change From Current Study Baseline in Bone Biomarker Level: Type 1 Collagen C-Telopeptides (CTx) at Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234	Study Baseline	0.4540 mcg/L	Standard Deviation 0.1827
Eliglustat	Change From Current Study Baseline in Bone Biomarker Level: Type 1 Collagen C-Telopeptides (CTx) at Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234	Change at Week 26	-0.0363 mcg/L	Standard Deviation 0.1515
Eliglustat	Change From Current Study Baseline in Bone Biomarker Level: Type 1 Collagen C-Telopeptides (CTx) at Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234	Change at Week 52	-0.0034 mcg/L	Standard Deviation 0.1309
Eliglustat	Change From Current Study Baseline in Bone Biomarker Level: Type 1 Collagen C-Telopeptides (CTx) at Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234	Change at Week 78	-0.0041 mcg/L	Standard Deviation 0.1696
Eliglustat	Change From Current Study Baseline in Bone Biomarker Level: Type 1 Collagen C-Telopeptides (CTx) at Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234	Change at Week 104	0.0000 mcg/L	Standard Deviation 0.166
Eliglustat	Change From Current Study Baseline in Bone Biomarker Level: Type 1 Collagen C-Telopeptides (CTx) at Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234	Change at Week 130	-0.0426 mcg/L	Standard Deviation 0.142
Eliglustat	Change From Current Study Baseline in Bone Biomarker Level: Type 1 Collagen C-Telopeptides (CTx) at Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234	Change at Week 156	-0.0738 mcg/L	Standard Deviation 0.1294
Eliglustat	Change From Current Study Baseline in Bone Biomarker Level: Type 1 Collagen C-Telopeptides (CTx) at Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234	Change at Week 182	0.0011 mcg/L	Standard Deviation 0.2439
Eliglustat	Change From Current Study Baseline in Bone Biomarker Level: Type 1 Collagen C-Telopeptides (CTx) at Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234	Change at Week 208	-0.0176 mcg/L	Standard Deviation 0.1812
Eliglustat	Change From Current Study Baseline in Bone Biomarker Level: Type 1 Collagen C-Telopeptides (CTx) at Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234	Change at Week 234	0.1200 mcg/L	Standard Deviation 0.215

Primary

Change From Current Study Baseline in Spine and Femur Total T-Scores for BMD at Weeks 52, 104, 156 and 208

BMD measurements of the spine and bilateral femur were acquired by DXA scan. The T-score bone density categories were: normal (score \>-1), osteopenia (score -2.5 to \<=-1), and osteoporosis (score \<= -2.5). Worst total femur at Baseline refers to the worst diseased left or right femur at baseline. For this outcome measure, baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry.

Time frame: Study Baseline, Weeks 52, 104, 156 and 208

Arm	Measure	Group	Value (MEAN)	Dispersion
Eliglustat	Change From Current Study Baseline in Spine and Femur Total T-Scores for BMD at Weeks 52, 104, 156 and 208	Total Spine T-score: Study Baseline	-0.4413 T-score	Standard Deviation 0.9782
Eliglustat	Change From Current Study Baseline in Spine and Femur Total T-Scores for BMD at Weeks 52, 104, 156 and 208	Total Spine T-score: Week 52	-0.4362 T-score	Standard Deviation 1.0155
Eliglustat	Change From Current Study Baseline in Spine and Femur Total T-Scores for BMD at Weeks 52, 104, 156 and 208	Total Spine T-score: Change at Week 52	-0.0250 T-score	Standard Deviation 0.2356
Eliglustat	Change From Current Study Baseline in Spine and Femur Total T-Scores for BMD at Weeks 52, 104, 156 and 208	Total Spine T-score: Week 104	-0.5566 T-score	Standard Deviation 0.9522
Eliglustat	Change From Current Study Baseline in Spine and Femur Total T-Scores for BMD at Weeks 52, 104, 156 and 208	Total Spine T-score: Change at Week 104	-0.0479 T-score	Standard Deviation 0.2751
Eliglustat	Change From Current Study Baseline in Spine and Femur Total T-Scores for BMD at Weeks 52, 104, 156 and 208	Total Spine T-score: Week 156	-0.5152 T-score	Standard Deviation 0.9075
Eliglustat	Change From Current Study Baseline in Spine and Femur Total T-Scores for BMD at Weeks 52, 104, 156 and 208	Total Spine T-score: Change at Week 156	-0.0066 T-score	Standard Deviation 0.4013
Eliglustat	Change From Current Study Baseline in Spine and Femur Total T-Scores for BMD at Weeks 52, 104, 156 and 208	Total Spine T-score: Week 208	-0.6238 T-score	Standard Deviation 0.9015
Eliglustat	Change From Current Study Baseline in Spine and Femur Total T-Scores for BMD at Weeks 52, 104, 156 and 208	Total Spine T-score: Change at Week 208	-0.1012 T-score	Standard Deviation 0.458
Eliglustat	Change From Current Study Baseline in Spine and Femur Total T-Scores for BMD at Weeks 52, 104, 156 and 208	Worst Total Femur T-score: Study Baseline	0.0119 T-score	Standard Deviation 1.0001
Eliglustat	Change From Current Study Baseline in Spine and Femur Total T-Scores for BMD at Weeks 52, 104, 156 and 208	Worst Total Femur T-score: Week 52	-0.0119 T-score	Standard Deviation 1.0104
Eliglustat	Change From Current Study Baseline in Spine and Femur Total T-Scores for BMD at Weeks 52, 104, 156 and 208	Worst Total Femur T-score: Change at Week 52	0.0273 T-score	Standard Deviation 0.1481
Eliglustat	Change From Current Study Baseline in Spine and Femur Total T-Scores for BMD at Weeks 52, 104, 156 and 208	Worst Total Femur T-score: Week 104	-0.1345 T-score	Standard Deviation 0.8867
Eliglustat	Change From Current Study Baseline in Spine and Femur Total T-Scores for BMD at Weeks 52, 104, 156 and 208	Worst Total Femur T-score: Change at Week 104	-0.0245 T-score	Standard Deviation 0.167
Eliglustat	Change From Current Study Baseline in Spine and Femur Total T-Scores for BMD at Weeks 52, 104, 156 and 208	Worst Total Femur T-score: Week 156	-0.1032 T-score	Standard Deviation 0.9087
Eliglustat	Change From Current Study Baseline in Spine and Femur Total T-Scores for BMD at Weeks 52, 104, 156 and 208	Worst Total Femur T-score: Change at Week 156	0.0179 T-score	Standard Deviation 0.2704
Eliglustat	Change From Current Study Baseline in Spine and Femur Total T-Scores for BMD at Weeks 52, 104, 156 and 208	Worst Total Femur T-score: Week 208	-0.1196 T-score	Standard Deviation 0.9594
Eliglustat	Change From Current Study Baseline in Spine and Femur Total T-Scores for BMD at Weeks 52, 104, 156 and 208	Worst Total Femur T-score: Change at Week 208	-0.0073 T-score	Standard Deviation 0.4318

Primary

Change From Current Study Baseline in Spine and Femur Total Z-Scores for BMD at Weeks 52, 104, 156 and 208

BMD measurements of the spine and bilateral femur were acquired by DXA scan. The Z-score bone density categories were: normal (score \>-2) and below normal (score \<=-2). Worst total femur at Baseline refers to the worst diseased left or right femur at baseline. For this outcome measure, baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry.

Time frame: Study Baseline, Weeks 52, 104, 156 and 208

Arm	Measure	Group	Value (MEAN)	Dispersion
Eliglustat	Change From Current Study Baseline in Spine and Femur Total Z-Scores for BMD at Weeks 52, 104, 156 and 208	Worst Total Femur Z-score: Study Baseline	0.1935 Z-score	Standard Deviation 0.9392
Eliglustat	Change From Current Study Baseline in Spine and Femur Total Z-Scores for BMD at Weeks 52, 104, 156 and 208	Worst Total Femur Z-score: Week 52	0.2196 Z-score	Standard Deviation 0.9469
Eliglustat	Change From Current Study Baseline in Spine and Femur Total Z-Scores for BMD at Weeks 52, 104, 156 and 208	Total Spine Z-score: Study Baseline	-0.3687 Z-score	Standard Deviation 0.8529
Eliglustat	Change From Current Study Baseline in Spine and Femur Total Z-Scores for BMD at Weeks 52, 104, 156 and 208	Total Spine Z-score: Week 52	-0.3338 Z-score	Standard Deviation 0.8597
Eliglustat	Change From Current Study Baseline in Spine and Femur Total Z-Scores for BMD at Weeks 52, 104, 156 and 208	Total Spine Z-score: Change at Week 52	-0.0092 Z-score	Standard Deviation 0.2405
Eliglustat	Change From Current Study Baseline in Spine and Femur Total Z-Scores for BMD at Weeks 52, 104, 156 and 208	Total Spine Z-score: Week 104	-0.4555 Z-score	Standard Deviation 0.8522
Eliglustat	Change From Current Study Baseline in Spine and Femur Total Z-Scores for BMD at Weeks 52, 104, 156 and 208	Total Spine Z-score: Change at Week 104	-0.0245 Z-score	Standard Deviation 0.2945
Eliglustat	Change From Current Study Baseline in Spine and Femur Total Z-Scores for BMD at Weeks 52, 104, 156 and 208	Total Spine Z-score: Week 156	-0.4034 Z-score	Standard Deviation 0.8464
Eliglustat	Change From Current Study Baseline in Spine and Femur Total Z-Scores for BMD at Weeks 52, 104, 156 and 208	Total Spine Z-score: Change at Week 156	0.0276 Z-score	Standard Deviation 0.4275
Eliglustat	Change From Current Study Baseline in Spine and Femur Total Z-Scores for BMD at Weeks 52, 104, 156 and 208	Total Spine Z-score: Week 208	-0.5035 Z-score	Standard Deviation 0.8289
Eliglustat	Change From Current Study Baseline in Spine and Femur Total Z-Scores for BMD at Weeks 52, 104, 156 and 208	Total Spine Z-score: Change at Week 208	-0.0519 Z-score	Standard Deviation 0.4759
Eliglustat	Change From Current Study Baseline in Spine and Femur Total Z-Scores for BMD at Weeks 52, 104, 156 and 208	Worst Total Femur Z-score: Change at Week 52	0.0504 Z-score	Standard Deviation 0.1495
Eliglustat	Change From Current Study Baseline in Spine and Femur Total Z-Scores for BMD at Weeks 52, 104, 156 and 208	Worst Total Femur Z-score: Week 104	0.0921 Z-score	Standard Deviation 0.8288
Eliglustat	Change From Current Study Baseline in Spine and Femur Total Z-Scores for BMD at Weeks 52, 104, 156 and 208	Worst Total Femur Z-score: Change at Week 104	0.0114 Z-score	Standard Deviation 0.1663
Eliglustat	Change From Current Study Baseline in Spine and Femur Total Z-Scores for BMD at Weeks 52, 104, 156 and 208	Worst Total Femur Z-score: Week 156	0.1521 Z-score	Standard Deviation 0.85
Eliglustat	Change From Current Study Baseline in Spine and Femur Total Z-Scores for BMD at Weeks 52, 104, 156 and 208	Worst Total Femur Z-score: Change at Week 156	0.0757 Z-score	Standard Deviation 0.2705
Eliglustat	Change From Current Study Baseline in Spine and Femur Total Z-Scores for BMD at Weeks 52, 104, 156 and 208	Worst Total Femur Z-score: Week 208	0.1408 Z-score	Standard Deviation 0.9077
Eliglustat	Change From Current Study Baseline in Spine and Femur Total Z-Scores for BMD at Weeks 52, 104, 156 and 208	Worst Total Femur Z-score: Change at Week 208	0.0746 Z-score	Standard Deviation 0.4267

Primary

Change From Current Study Baseline in Total Bone Marrow Burden (BMB) Scores at Weeks 52, 104, 156 and 208

Bone marrow burden (BMB) scores indicate the degree of bone marrow infiltration. BMB score was measured using MRI (magnetic resonance imaging (MRI), ranged from 0 (no abnormalities) to 8 points (severe disease) for the lumbar spine and from 0 (no abnormalities) to 8 points (severe disease) for the femurs. The total BMB score was calculated as the sum of scores for femur and lumbar spine regions which ranged from 0 (no abnormalities) to 16 (severe disease) points. A higher BMB score signified more severe bone marrow involvement. For this outcome measure, baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry.

Time frame: Study Baseline, Weeks 52, 104, 156 and 208

Arm	Measure	Group	Value (MEAN)	Dispersion
Eliglustat	Change From Current Study Baseline in Total Bone Marrow Burden (BMB) Scores at Weeks 52, 104, 156 and 208	Total BMB Score: Study Baseline	8.6667 score on a scale	Standard Deviation 3.386
Eliglustat	Change From Current Study Baseline in Total Bone Marrow Burden (BMB) Scores at Weeks 52, 104, 156 and 208	Total BMB Score: Week 52	8.5057 score on a scale	Standard Deviation 2.6482
Eliglustat	Change From Current Study Baseline in Total Bone Marrow Burden (BMB) Scores at Weeks 52, 104, 156 and 208	Total BMB Score: Change at Week 52	-0.0256 score on a scale	Standard Deviation 1.6969
Eliglustat	Change From Current Study Baseline in Total Bone Marrow Burden (BMB) Scores at Weeks 52, 104, 156 and 208	Total BMB Score: Week 104	9.1111 score on a scale	Standard Deviation 2.9627
Eliglustat	Change From Current Study Baseline in Total Bone Marrow Burden (BMB) Scores at Weeks 52, 104, 156 and 208	Total BMB Score: Change at Week 104	1.2639 score on a scale	Standard Deviation 2.0897
Eliglustat	Change From Current Study Baseline in Total Bone Marrow Burden (BMB) Scores at Weeks 52, 104, 156 and 208	Total BMB Score: Week 156	8.7821 score on a scale	Standard Deviation 2.6178
Eliglustat	Change From Current Study Baseline in Total Bone Marrow Burden (BMB) Scores at Weeks 52, 104, 156 and 208	Total BMB Score: Change at Week 156	0.7101 score on a scale	Standard Deviation 1.8404
Eliglustat	Change From Current Study Baseline in Total Bone Marrow Burden (BMB) Scores at Weeks 52, 104, 156 and 208	Total BMB Score: Week 208	8.9067 score on a scale	Standard Deviation 2.8844
Eliglustat	Change From Current Study Baseline in Total Bone Marrow Burden (BMB) Scores at Weeks 52, 104, 156 and 208	Total BMB Score: Change at Week 208	0.8939 score on a scale	Standard Deviation 2.4023

Primary

Change From Current Study Baseline in Total Spine and Femur Bone Mineral Density (BMD) at Weeks 52, 104, 156 and 208

Bone Mineral Density (BMD) measurements of the spine and bilateral femur were acquired by dual energy X-Ray absorptiometry (DXA) scan. Worst total femur at Baseline refers to the worst diseased left or right femur at Baseline. For this outcome measure, baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry.

Time frame: Study Baseline, Weeks 52, 104, 156 and 208

Arm	Measure	Group	Value (MEAN)	Dispersion
Eliglustat	Change From Current Study Baseline in Total Spine and Femur Bone Mineral Density (BMD) at Weeks 52, 104, 156 and 208	Total Spine BMD: Study Baseline	1.1449 grams per centimeter square (g/cm^2)	Standard Deviation 0.1236
Eliglustat	Change From Current Study Baseline in Total Spine and Femur Bone Mineral Density (BMD) at Weeks 52, 104, 156 and 208	Total Spine BMD: Week 52	1.1476 grams per centimeter square (g/cm^2)	Standard Deviation 0.1255
Eliglustat	Change From Current Study Baseline in Total Spine and Femur Bone Mineral Density (BMD) at Weeks 52, 104, 156 and 208	Total Spine BMD: Change at Week 52	-0.0029 grams per centimeter square (g/cm^2)	Standard Deviation 0.0282
Eliglustat	Change From Current Study Baseline in Total Spine and Femur Bone Mineral Density (BMD) at Weeks 52, 104, 156 and 208	Total Spine BMD: Week 104	1.1326 grams per centimeter square (g/cm^2)	Standard Deviation 0.1217
Eliglustat	Change From Current Study Baseline in Total Spine and Femur Bone Mineral Density (BMD) at Weeks 52, 104, 156 and 208	Total Spine BMD: Change at Week 104	-0.0055 grams per centimeter square (g/cm^2)	Standard Deviation 0.0333
Eliglustat	Change From Current Study Baseline in Total Spine and Femur Bone Mineral Density (BMD) at Weeks 52, 104, 156 and 208	Total Spine BMD: Week 156	1.1373 grams per centimeter square (g/cm^2)	Standard Deviation 0.1173
Eliglustat	Change From Current Study Baseline in Total Spine and Femur Bone Mineral Density (BMD) at Weeks 52, 104, 156 and 208	Total Spine BMD: Change at Week 156	-0.0007 grams per centimeter square (g/cm^2)	Standard Deviation 0.0484
Eliglustat	Change From Current Study Baseline in Total Spine and Femur Bone Mineral Density (BMD) at Weeks 52, 104, 156 and 208	Total Spine BMD: Week 208	1.1236 grams per centimeter square (g/cm^2)	Standard Deviation 0.1146
Eliglustat	Change From Current Study Baseline in Total Spine and Femur Bone Mineral Density (BMD) at Weeks 52, 104, 156 and 208	Total Spine BMD: Change at Week 208	-0.0120 grams per centimeter square (g/cm^2)	Standard Deviation 0.055
Eliglustat	Change From Current Study Baseline in Total Spine and Femur Bone Mineral Density (BMD) at Weeks 52, 104, 156 and 208	Worst Total Femur BMD: Study Baseline	1.0567 grams per centimeter square (g/cm^2)	Standard Deviation 0.1449
Eliglustat	Change From Current Study Baseline in Total Spine and Femur Bone Mineral Density (BMD) at Weeks 52, 104, 156 and 208	Worst Total Femur BMD: Week 52	1.0538 grams per centimeter square (g/cm^2)	Standard Deviation 0.1423
Eliglustat	Change From Current Study Baseline in Total Spine and Femur Bone Mineral Density (BMD) at Weeks 52, 104, 156 and 208	Worst Total Femur BMD: Change at Week 52	0.0035 grams per centimeter square (g/cm^2)	Standard Deviation 0.0188
Eliglustat	Change From Current Study Baseline in Total Spine and Femur Bone Mineral Density (BMD) at Weeks 52, 104, 156 and 208	Worst Total Femur BMD: Week 104	1.0416 grams per centimeter square (g/cm^2)	Standard Deviation 0.1394
Eliglustat	Change From Current Study Baseline in Total Spine and Femur Bone Mineral Density (BMD) at Weeks 52, 104, 156 and 208	Worst Total Femur BMD: Change at Week 104	-0.0030 grams per centimeter square (g/cm^2)	Standard Deviation 0.0222
Eliglustat	Change From Current Study Baseline in Total Spine and Femur Bone Mineral Density (BMD) at Weeks 52, 104, 156 and 208	Worst Total Femur BMD: Week 156	1.0437 grams per centimeter square (g/cm^2)	Standard Deviation 0.1411
Eliglustat	Change From Current Study Baseline in Total Spine and Femur Bone Mineral Density (BMD) at Weeks 52, 104, 156 and 208	Worst Total Femur BMD: Change at Week 156	0.0021 grams per centimeter square (g/cm^2)	Standard Deviation 0.0364
Eliglustat	Change From Current Study Baseline in Total Spine and Femur Bone Mineral Density (BMD) at Weeks 52, 104, 156 and 208	Worst Total Femur BMD: Week 208	1.0412 grams per centimeter square (g/cm^2)	Standard Deviation 0.1465
Eliglustat	Change From Current Study Baseline in Total Spine and Femur Bone Mineral Density (BMD) at Weeks 52, 104, 156 and 208	Worst Total Femur BMD: Change at Week 208	-0.0015 grams per centimeter square (g/cm^2)	Standard Deviation 0.056

Primary

Change From Eliglustat Baseline in Bone Biomarker Level: Macrophage Inflammatory Protein 1 Beta (MIP-1β) at Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234

MIP-1β considered a biomarker of active bone disease, was assayed from plasma. For this outcome measure, baseline refers to the eliglustat baseline, which was defined as participant's status at the time of first dose of eliglustat in the previous Phase 2 or Phase 3 study.

Time frame: Eliglustat Baseline, Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234 of the current study

Population: Analyzed on FAS population. Here, number analyzed = participants with available data for each specified category and 0 in number analyzed field signifies that none of participants had evaluable data at specified timepoint. Data for this outcome measure was planned to be collected and analyzed in reference to eliglustat baseline separately for participants from Phase 2, ENGAGE and EDGE studies (GD treatment naïve participants) and from ENCORE study (participants switched from ERT).

Arm	Measure	Group	Value (MEAN)	Dispersion
Eliglustat	Change From Eliglustat Baseline in Bone Biomarker Level: Macrophage Inflammatory Protein 1 Beta (MIP-1β) at Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234	Eliglustat Baseline	265.3944 pg/mL	Standard Deviation 149.3311
Eliglustat	Change From Eliglustat Baseline in Bone Biomarker Level: Macrophage Inflammatory Protein 1 Beta (MIP-1β) at Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234	Change at Week 26	-136.2167 pg/mL	Standard Deviation 135.6276
Eliglustat	Change From Eliglustat Baseline in Bone Biomarker Level: Macrophage Inflammatory Protein 1 Beta (MIP-1β) at Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234	Change at Week 52	-153.7647 pg/mL	Standard Deviation 127.0446
Eliglustat	Change From Eliglustat Baseline in Bone Biomarker Level: Macrophage Inflammatory Protein 1 Beta (MIP-1β) at Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234	Change at Week 78	-151.1857 pg/mL	Standard Deviation 118.798
Eliglustat	Change From Eliglustat Baseline in Bone Biomarker Level: Macrophage Inflammatory Protein 1 Beta (MIP-1β) at Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234	Change at Week 104	-152.8875 pg/mL	Standard Deviation 128.7985
Eliglustat	Change From Eliglustat Baseline in Bone Biomarker Level: Macrophage Inflammatory Protein 1 Beta (MIP-1β) at Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234	Change at Week 130	-159.4750 pg/mL	Standard Deviation 149.3802
Eliglustat	Change From Eliglustat Baseline in Bone Biomarker Level: Macrophage Inflammatory Protein 1 Beta (MIP-1β) at Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234	Change at Week 156	-141.7750 pg/mL	Standard Deviation 129.3039
Eliglustat	Change From Eliglustat Baseline in Bone Biomarker Level: Macrophage Inflammatory Protein 1 Beta (MIP-1β) at Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234	Change at Week 182	-145.6750 pg/mL	Standard Deviation 138.9781
Eliglustat	Change From Eliglustat Baseline in Bone Biomarker Level: Macrophage Inflammatory Protein 1 Beta (MIP-1β) at Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234	Change at Week 208	-149.8933 pg/mL	Standard Deviation 153.1632
Eliglustat	Change From Eliglustat Baseline in Bone Biomarker Level: Macrophage Inflammatory Protein 1 Beta (MIP-1β) at Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234	Change at Week 234	-222.6857 pg/mL	Standard Deviation 64.4851
Eliglustat: Participants From ENCORE	Change From Eliglustat Baseline in Bone Biomarker Level: Macrophage Inflammatory Protein 1 Beta (MIP-1β) at Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234	Eliglustat Baseline	103.7750 pg/mL	Standard Deviation 32.0705
Eliglustat: Participants From ENCORE	Change From Eliglustat Baseline in Bone Biomarker Level: Macrophage Inflammatory Protein 1 Beta (MIP-1β) at Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234	Change at Week 208	67.2500 pg/mL	Standard Deviation 75.0664
Eliglustat: Participants From ENCORE	Change From Eliglustat Baseline in Bone Biomarker Level: Macrophage Inflammatory Protein 1 Beta (MIP-1β) at Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234	Change at Week 26	66.7750 pg/mL	Standard Deviation 70.3377
Eliglustat: Participants From ENCORE	Change From Eliglustat Baseline in Bone Biomarker Level: Macrophage Inflammatory Protein 1 Beta (MIP-1β) at Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234	Change at Week 130	53.6250 pg/mL	Standard Deviation 86.1059
Eliglustat: Participants From ENCORE	Change From Eliglustat Baseline in Bone Biomarker Level: Macrophage Inflammatory Protein 1 Beta (MIP-1β) at Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234	Change at Week 52	79.0750 pg/mL	Standard Deviation 115.7302
Eliglustat: Participants From ENCORE	Change From Eliglustat Baseline in Bone Biomarker Level: Macrophage Inflammatory Protein 1 Beta (MIP-1β) at Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234	Change at Week 182	46.6500 pg/mL	Standard Deviation 64.9619
Eliglustat: Participants From ENCORE	Change From Eliglustat Baseline in Bone Biomarker Level: Macrophage Inflammatory Protein 1 Beta (MIP-1β) at Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234	Change at Week 78	37.7000 pg/mL	Standard Deviation 79.6721
Eliglustat: Participants From ENCORE	Change From Eliglustat Baseline in Bone Biomarker Level: Macrophage Inflammatory Protein 1 Beta (MIP-1β) at Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234	Change at Week 156	68.9000 pg/mL	Standard Deviation 101.0239
Eliglustat: Participants From ENCORE	Change From Eliglustat Baseline in Bone Biomarker Level: Macrophage Inflammatory Protein 1 Beta (MIP-1β) at Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234	Change at Week 104	74.6750 pg/mL	Standard Deviation 81.1326

Primary

Change From Eliglustat Baseline in Bone Biomarker Level: Procollagen 1 N- Terminal Propeptide (P1NP) at Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234

P1NP, a marker of bone formation was assayed from plasma. For this outcome measure, baseline refers to the eliglustat baseline, which was defined as participant's status at the time of first dose of eliglustat in the previous Phase 2 or Phase 3 study.

Time frame: Eliglustat Baseline, Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234 of the current study

Population: Here, 0 signifies that data for this outcome measure (bone biomarker P1NP) was not collected and assessed in Phase 2 and Phase 3 studies, hence the biomarker level was not reported at specified timepoints.

Primary

Change From Eliglustat Baseline in Bone Biomarker Level: Type 1 Collagen C-Telopeptides (CTx) at Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234

CTx, a marker of bone resorption was assayed from plasma. For this outcome measure, baseline refers to the eliglustat baseline, which was defined as participant's status at the time of first dose of eliglustat in the previous Phase 2 or Phase 3 study.

Time frame: Eliglustat Baseline, Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234 of the current study

Population: Here, 0 signifies that data for this outcome measure (bone biomarker CTx) was not collected and assessed in Phase 2 and Phase 3 studies, hence the biomarker level was not reported at specified timepoints.

Primary

Change From Eliglustat Baseline in Spine and Femur Total T-Scores for BMD at Weeks 52, 104, 156 and 208

BMD measurements of the spine and bilateral femur were acquired by DXA scan. The T-score bone density categories were: normal (score \>-1), osteopenia (score -2.5 to \<=-1), and osteoporosis (score \<= -2.5). Worst total femur at Baseline refers to the worst diseased left or right femur at baseline. For this outcome measure, baseline refers to the eliglustat baseline, which was defined as participant's status at the time of first dose of eliglustat in the previous Phase 2 or Phase 3 study.

Time frame: Eliglustat Baseline, Weeks 52, 104, 156 and 208 of the current study

Population: Analysis was performed on FAS population. Here, number analyzed = participants with available data for each specified category. Here, 0 signifies that none of the participants were available for assessment at the specified timepoint. Data for this outcome measure was planned to be collected and analyzed in reference to eliglustat baseline separately for participants from Phase 2, ENGAGE and EDGE studies (GD treatment naïve participants) and from ENCORE study (participants switched from ERT).

Arm	Measure	Group	Value (MEAN)	Dispersion
Eliglustat	Change From Eliglustat Baseline in Spine and Femur Total T-Scores for BMD at Weeks 52, 104, 156 and 208	Total Spine T-score: Eliglustat Baseline	-0.9381 T-score	Standard Deviation 1.0443
Eliglustat	Change From Eliglustat Baseline in Spine and Femur Total T-Scores for BMD at Weeks 52, 104, 156 and 208	Total Spine T-score: Week 52	-0.4063 T-score	Standard Deviation 1.053
Eliglustat	Change From Eliglustat Baseline in Spine and Femur Total T-Scores for BMD at Weeks 52, 104, 156 and 208	Total Spine T-score: Change at Week 52	0.5126 T-score	Standard Deviation 0.5622
Eliglustat	Change From Eliglustat Baseline in Spine and Femur Total T-Scores for BMD at Weeks 52, 104, 156 and 208	Total Spine T-score: Week 104	-0.5808 T-score	Standard Deviation 0.9512
Eliglustat	Change From Eliglustat Baseline in Spine and Femur Total T-Scores for BMD at Weeks 52, 104, 156 and 208	Total Spine T-score: Change at Week 104	0.5215 T-score	Standard Deviation 0.5229
Eliglustat	Change From Eliglustat Baseline in Spine and Femur Total T-Scores for BMD at Weeks 52, 104, 156 and 208	Total Spine T-score: Week 156	-0.5172 T-score	Standard Deviation 0.8875
Eliglustat	Change From Eliglustat Baseline in Spine and Femur Total T-Scores for BMD at Weeks 52, 104, 156 and 208	Total Spine T-score: Change at Week 156	0.6150 T-score	Standard Deviation 0.6
Eliglustat	Change From Eliglustat Baseline in Spine and Femur Total T-Scores for BMD at Weeks 52, 104, 156 and 208	Total Spine T-score: Week 208	-0.6570 T-score	Standard Deviation 0.8823
Eliglustat	Change From Eliglustat Baseline in Spine and Femur Total T-Scores for BMD at Weeks 52, 104, 156 and 208	Total Spine T-score: Change at Week 208	0.5544 T-score	Standard Deviation 0.6867
Eliglustat	Change From Eliglustat Baseline in Spine and Femur Total T-Scores for BMD at Weeks 52, 104, 156 and 208	Worst Total Femur T-score: Eliglustat Baseline	-0.0190 T-score	Standard Deviation 1.0939
Eliglustat	Change From Eliglustat Baseline in Spine and Femur Total T-Scores for BMD at Weeks 52, 104, 156 and 208	Worst Total Femur T-score: Week 52	0.1142 T-score	Standard Deviation 1.1458
Eliglustat	Change From Eliglustat Baseline in Spine and Femur Total T-Scores for BMD at Weeks 52, 104, 156 and 208	Worst Total Femur T-score: Change at Week 52	0.0616 T-score	Standard Deviation 0.3755
Eliglustat	Change From Eliglustat Baseline in Spine and Femur Total T-Scores for BMD at Weeks 52, 104, 156 and 208	Worst Total Femur T-score: Week 104	-0.0950 T-score	Standard Deviation 0.9778
Eliglustat	Change From Eliglustat Baseline in Spine and Femur Total T-Scores for BMD at Weeks 52, 104, 156 and 208	Worst Total Femur T-score: Change at Week 104	0.0400 T-score	Standard Deviation 0.3661
Eliglustat	Change From Eliglustat Baseline in Spine and Femur Total T-Scores for BMD at Weeks 52, 104, 156 and 208	Worst Total Femur T-score: Week 156	-0.0615 T-score	Standard Deviation 1
Eliglustat	Change From Eliglustat Baseline in Spine and Femur Total T-Scores for BMD at Weeks 52, 104, 156 and 208	Worst Total Femur T-score: Change at Week 156	0.0735 T-score	Standard Deviation 0.4602
Eliglustat	Change From Eliglustat Baseline in Spine and Femur Total T-Scores for BMD at Weeks 52, 104, 156 and 208	Worst Total Femur T-score: Week 208	-0.0867 T-score	Standard Deviation 1.0293
Eliglustat	Change From Eliglustat Baseline in Spine and Femur Total T-Scores for BMD at Weeks 52, 104, 156 and 208	Worst Total Femur T-score: Change at Week 208	0.1022 T-score	Standard Deviation 0.4355
Eliglustat: Participants From ENCORE	Change From Eliglustat Baseline in Spine and Femur Total T-Scores for BMD at Weeks 52, 104, 156 and 208	Total Spine T-score: Eliglustat Baseline	-0.3000 T-score	Standard Deviation 0.8485
Eliglustat: Participants From ENCORE	Change From Eliglustat Baseline in Spine and Femur Total T-Scores for BMD at Weeks 52, 104, 156 and 208	Worst Total Femur T-score: Eliglustat Baseline	1.1000 T-score	Standard Deviation 0.9899
Eliglustat: Participants From ENCORE	Change From Eliglustat Baseline in Spine and Femur Total T-Scores for BMD at Weeks 52, 104, 156 and 208	Total Spine T-score: Week 52	-0.7950 T-score	Standard Deviation 0.0354
Eliglustat: Participants From ENCORE	Change From Eliglustat Baseline in Spine and Femur Total T-Scores for BMD at Weeks 52, 104, 156 and 208	Worst Total Femur T-score: Change at Week 208	-0.4500 T-score	—
Eliglustat: Participants From ENCORE	Change From Eliglustat Baseline in Spine and Femur Total T-Scores for BMD at Weeks 52, 104, 156 and 208	Worst Total Femur T-score: Week 52	0.3800 T-score	—
Eliglustat: Participants From ENCORE	Change From Eliglustat Baseline in Spine and Femur Total T-Scores for BMD at Weeks 52, 104, 156 and 208	Total Spine T-score: Week 104	-0.4050 T-score	Standard Deviation 1.0898
Eliglustat: Participants From ENCORE	Change From Eliglustat Baseline in Spine and Femur Total T-Scores for BMD at Weeks 52, 104, 156 and 208	Worst Total Femur T-score: Week 156	0.9050 T-score	Standard Deviation 0.7283
Eliglustat: Participants From ENCORE	Change From Eliglustat Baseline in Spine and Femur Total T-Scores for BMD at Weeks 52, 104, 156 and 208	Total Spine T-score: Change at Week 104	0.5200 T-score	Standard Deviation 0.5657
Eliglustat: Participants From ENCORE	Change From Eliglustat Baseline in Spine and Femur Total T-Scores for BMD at Weeks 52, 104, 156 and 208	Worst Total Femur T-score: Change at Week 52	-0.0200 T-score	—
Eliglustat: Participants From ENCORE	Change From Eliglustat Baseline in Spine and Femur Total T-Scores for BMD at Weeks 52, 104, 156 and 208	Total Spine T-score: Week 156	-0.5025 T-score	Standard Deviation 1.1771
Eliglustat: Participants From ENCORE	Change From Eliglustat Baseline in Spine and Femur Total T-Scores for BMD at Weeks 52, 104, 156 and 208	Worst Total Femur T-score: Week 208	1.3500 T-score	—
Eliglustat: Participants From ENCORE	Change From Eliglustat Baseline in Spine and Femur Total T-Scores for BMD at Weeks 52, 104, 156 and 208	Total Spine T-score: Change at Week 156	0.4300 T-score	Standard Deviation 0.7495
Eliglustat: Participants From ENCORE	Change From Eliglustat Baseline in Spine and Femur Total T-Scores for BMD at Weeks 52, 104, 156 and 208	Worst Total Femur T-score: Week 104	0.8200 T-score	Standard Deviation 0.6647
Eliglustat: Participants From ENCORE	Change From Eliglustat Baseline in Spine and Femur Total T-Scores for BMD at Weeks 52, 104, 156 and 208	Total Spine T-score: Week 208	-0.3700 T-score	Standard Deviation 1.2194
Eliglustat: Participants From ENCORE	Change From Eliglustat Baseline in Spine and Femur Total T-Scores for BMD at Weeks 52, 104, 156 and 208	Worst Total Femur T-score: Change at Week 156	-0.1950 T-score	Standard Deviation 0.2616
Eliglustat: Participants From ENCORE	Change From Eliglustat Baseline in Spine and Femur Total T-Scores for BMD at Weeks 52, 104, 156 and 208	Total Spine T-score: Change at Week 208	0.7300 T-score	—
Eliglustat: Participants From ENCORE	Change From Eliglustat Baseline in Spine and Femur Total T-Scores for BMD at Weeks 52, 104, 156 and 208	Worst Total Femur T-score: Change at Week 104	-0.2800 T-score	Standard Deviation 0.3253

Primary

Change From Eliglustat Baseline in Spine and Femur Total Z-Scores for BMD at Weeks 52, 104, 156 and 208

BMD measurements of the spine and bilateral femur were acquired by DXA scan. The Z-score bone density categories are: normal (score \>-2) and below normal (score \<=-2). Worst total femur at Baseline refers to the worst diseased left or right femur at baseline. For this outcome measure, baseline refers to the eliglustat baseline, which was defined as participant's status at the time of first dose of eliglustat in the previous Phase 2 or Phase 3 study.

Time frame: Eliglustat Baseline, Weeks 52, 104, 156 and 208 of the current study

Population: Analysis was performed on FAS population. Here, number analyzed = participants with available data for each specified category. Data for this outcome measure was planned to be collected and analyzed in reference to eliglustat baseline separately for participants from Phase 2, ENGAGE and EDGE studies (GD treatment naïve participants) and from ENCORE study (participants switched from ERT).

Arm	Measure	Group	Value (MEAN)	Dispersion
Eliglustat	Change From Eliglustat Baseline in Spine and Femur Total Z-Scores for BMD at Weeks 52, 104, 156 and 208	Total Spine Z-score: Change at Week 104	0.5186 Z-score	Standard Deviation 0.4578
Eliglustat	Change From Eliglustat Baseline in Spine and Femur Total Z-Scores for BMD at Weeks 52, 104, 156 and 208	Worst Total Femur Z-score: Eliglustat Baseline	0.0826 Z-score	Standard Deviation 1.0373
Eliglustat	Change From Eliglustat Baseline in Spine and Femur Total Z-Scores for BMD at Weeks 52, 104, 156 and 208	Total Spine Z-score: Change at Week 52	0.5030 Z-score	Standard Deviation 0.423
Eliglustat	Change From Eliglustat Baseline in Spine and Femur Total Z-Scores for BMD at Weeks 52, 104, 156 and 208	Worst Total Femur Z-score: Week 52	0.3475 Z-score	Standard Deviation 1.0593
Eliglustat	Change From Eliglustat Baseline in Spine and Femur Total Z-Scores for BMD at Weeks 52, 104, 156 and 208	Total Spine Z-score: Week 156	-0.3876 Z-score	Standard Deviation 0.8129
Eliglustat	Change From Eliglustat Baseline in Spine and Femur Total Z-Scores for BMD at Weeks 52, 104, 156 and 208	Worst Total Femur Z-score: Change at Week 52	0.1825 Z-score	Standard Deviation 0.3842
Eliglustat	Change From Eliglustat Baseline in Spine and Femur Total Z-Scores for BMD at Weeks 52, 104, 156 and 208	Total Spine Z-score: Week 52	-0.2954 Z-score	Standard Deviation 0.8851
Eliglustat	Change From Eliglustat Baseline in Spine and Femur Total Z-Scores for BMD at Weeks 52, 104, 156 and 208	Worst Total Femur Z-score: Week 104	0.1429 Z-score	Standard Deviation 0.9361
Eliglustat	Change From Eliglustat Baseline in Spine and Femur Total Z-Scores for BMD at Weeks 52, 104, 156 and 208	Total Spine Z-score: Change at Week 156	0.6276 Z-score	Standard Deviation 0.5671
Eliglustat	Change From Eliglustat Baseline in Spine and Femur Total Z-Scores for BMD at Weeks 52, 104, 156 and 208	Worst Total Femur Z-score: Change at Week 104	0.1571 Z-score	Standard Deviation 0.3503
Eliglustat	Change From Eliglustat Baseline in Spine and Femur Total Z-Scores for BMD at Weeks 52, 104, 156 and 208	Total Spine Z-score: Week 104	-0.4636 Z-score	Standard Deviation 0.8357
Eliglustat	Change From Eliglustat Baseline in Spine and Femur Total Z-Scores for BMD at Weeks 52, 104, 156 and 208	Worst Total Femur Z-score: Week 156	0.1876 Z-score	Standard Deviation 0.9632
Eliglustat	Change From Eliglustat Baseline in Spine and Femur Total Z-Scores for BMD at Weeks 52, 104, 156 and 208	Total Spine Z-score: Week 208	-0.5209 Z-score	Standard Deviation 0.8017
Eliglustat	Change From Eliglustat Baseline in Spine and Femur Total Z-Scores for BMD at Weeks 52, 104, 156 and 208	Worst Total Femur Z-score: Change at Week 156	0.2019 Z-score	Standard Deviation 0.4407
Eliglustat	Change From Eliglustat Baseline in Spine and Femur Total Z-Scores for BMD at Weeks 52, 104, 156 and 208	Total Spine Z-score: Eliglustat Baseline	-0.8826 Z-score	Standard Deviation 0.9824
Eliglustat	Change From Eliglustat Baseline in Spine and Femur Total Z-Scores for BMD at Weeks 52, 104, 156 and 208	Worst Total Femur Z-score: Week 208	0.1758 Z-score	Standard Deviation 0.983
Eliglustat	Change From Eliglustat Baseline in Spine and Femur Total Z-Scores for BMD at Weeks 52, 104, 156 and 208	Total Spine Z-score: Change at Week 208	0.5716 Z-score	Standard Deviation 0.6454
Eliglustat	Change From Eliglustat Baseline in Spine and Femur Total Z-Scores for BMD at Weeks 52, 104, 156 and 208	Worst Total Femur Z-score: Change at Week 208	0.2495 Z-score	Standard Deviation 0.4439
Eliglustat: Participants From ENCORE	Change From Eliglustat Baseline in Spine and Femur Total Z-Scores for BMD at Weeks 52, 104, 156 and 208	Worst Total Femur Z-score: Change at Week 208	-0.2633 Z-score	Standard Deviation 0.4652
Eliglustat: Participants From ENCORE	Change From Eliglustat Baseline in Spine and Femur Total Z-Scores for BMD at Weeks 52, 104, 156 and 208	Total Spine Z-score: Eliglustat Baseline	-0.5750 Z-score	Standard Deviation 0.6185
Eliglustat: Participants From ENCORE	Change From Eliglustat Baseline in Spine and Femur Total Z-Scores for BMD at Weeks 52, 104, 156 and 208	Total Spine Z-score: Week 52	-0.7950 Z-score	Standard Deviation 0.0354
Eliglustat: Participants From ENCORE	Change From Eliglustat Baseline in Spine and Femur Total Z-Scores for BMD at Weeks 52, 104, 156 and 208	Total Spine Z-score: Change at Week 52	0.0550 Z-score	Standard Deviation 0.3889
Eliglustat: Participants From ENCORE	Change From Eliglustat Baseline in Spine and Femur Total Z-Scores for BMD at Weeks 52, 104, 156 and 208	Total Spine Z-score: Week 104	-0.4050 Z-score	Standard Deviation 1.0898
Eliglustat: Participants From ENCORE	Change From Eliglustat Baseline in Spine and Femur Total Z-Scores for BMD at Weeks 52, 104, 156 and 208	Total Spine Z-score: Change at Week 104	0.1700 Z-score	Standard Deviation 0.5553
Eliglustat: Participants From ENCORE	Change From Eliglustat Baseline in Spine and Femur Total Z-Scores for BMD at Weeks 52, 104, 156 and 208	Total Spine Z-score: Week 156	-0.5025 Z-score	Standard Deviation 1.1771
Eliglustat: Participants From ENCORE	Change From Eliglustat Baseline in Spine and Femur Total Z-Scores for BMD at Weeks 52, 104, 156 and 208	Total Spine Z-score: Change at Week 156	0.0725 Z-score	Standard Deviation 0.6233
Eliglustat: Participants From ENCORE	Change From Eliglustat Baseline in Spine and Femur Total Z-Scores for BMD at Weeks 52, 104, 156 and 208	Total Spine Z-score: Week 208	-0.3700 Z-score	Standard Deviation 1.2194
Eliglustat: Participants From ENCORE	Change From Eliglustat Baseline in Spine and Femur Total Z-Scores for BMD at Weeks 52, 104, 156 and 208	Total Spine Z-score: Change at Week 208	0.0967 Z-score	Standard Deviation 0.5615
Eliglustat: Participants From ENCORE	Change From Eliglustat Baseline in Spine and Femur Total Z-Scores for BMD at Weeks 52, 104, 156 and 208	Worst Total Femur Z-score: Eliglustat Baseline	0.5000 Z-score	Standard Deviation 0.8287
Eliglustat: Participants From ENCORE	Change From Eliglustat Baseline in Spine and Femur Total Z-Scores for BMD at Weeks 52, 104, 156 and 208	Worst Total Femur Z-score: Week 52	0.1100 Z-score	Standard Deviation 0.5071
Eliglustat: Participants From ENCORE	Change From Eliglustat Baseline in Spine and Femur Total Z-Scores for BMD at Weeks 52, 104, 156 and 208	Worst Total Femur Z-score: Change at Week 52	0.0100 Z-score	Standard Deviation 0.453
Eliglustat: Participants From ENCORE	Change From Eliglustat Baseline in Spine and Femur Total Z-Scores for BMD at Weeks 52, 104, 156 and 208	Worst Total Femur Z-score: Week 104	0.3375 Z-score	Standard Deviation 0.7968
Eliglustat: Participants From ENCORE	Change From Eliglustat Baseline in Spine and Femur Total Z-Scores for BMD at Weeks 52, 104, 156 and 208	Worst Total Femur Z-score: Change at Week 104	-0.1625 Z-score	Standard Deviation 0.3728
Eliglustat: Participants From ENCORE	Change From Eliglustat Baseline in Spine and Femur Total Z-Scores for BMD at Weeks 52, 104, 156 and 208	Worst Total Femur Z-score: Week 156	0.3975 Z-score	Standard Deviation 0.8695
Eliglustat: Participants From ENCORE	Change From Eliglustat Baseline in Spine and Femur Total Z-Scores for BMD at Weeks 52, 104, 156 and 208	Worst Total Femur Z-score: Change at Week 156	-0.1025 Z-score	Standard Deviation 0.418
Eliglustat: Participants From ENCORE	Change From Eliglustat Baseline in Spine and Femur Total Z-Scores for BMD at Weeks 52, 104, 156 and 208	Worst Total Femur Z-score: Week 208	0.2700 Z-score	Standard Deviation 1.0789

Primary

Change From Eliglustat Baseline in Total Bone Marrow Burden (BMB) Scores at Weeks 52, 104, 156 and 208

Bone marrow burden (BMB) scores indicate the degree of bone marrow infiltration was measured using MRI, ranged from 0 (no abnormalities) to 8 points (severe disease) for the lumbar spine and from 0 (no abnormalities) to 8 points (severe disease) for the femurs. The total BMB score was calculated as the sum of scores for femur and lumbar spine regions which ranged from 0 (no abnormalities) to 16 (severe disease) points. A higher BMB score signified more severe bone marrow involvement. For this outcome measure, baseline refers to the eliglustat baseline, which was defined as participant's status at the time of first dose of eliglustat in the previous Phase 2 or Phase 3 study.

Time frame: Eliglustat Baseline, Weeks 52, 104, 156 and 208 of the current study

Arm	Measure	Group	Value (MEAN)	Dispersion
Eliglustat	Change From Eliglustat Baseline in Total Bone Marrow Burden (BMB) Scores at Weeks 52, 104, 156 and 208	Total BMB Score: Week 52	8.4533 score on a scale	Standard Deviation 2.7502
Eliglustat	Change From Eliglustat Baseline in Total Bone Marrow Burden (BMB) Scores at Weeks 52, 104, 156 and 208	Total BMB Score: Week 156	8.6250 score on a scale	Standard Deviation 2.66
Eliglustat	Change From Eliglustat Baseline in Total Bone Marrow Burden (BMB) Scores at Weeks 52, 104, 156 and 208	Total BMB Score: Week 104	8.9420 score on a scale	Standard Deviation 3.1537
Eliglustat	Change From Eliglustat Baseline in Total Bone Marrow Burden (BMB) Scores at Weeks 52, 104, 156 and 208	Total BMB Score: Change at Week 156	-1.2424 score on a scale	Standard Deviation 2.3098
Eliglustat	Change From Eliglustat Baseline in Total Bone Marrow Burden (BMB) Scores at Weeks 52, 104, 156 and 208	Total BMB Score: Change at Week 52	-1.7179 score on a scale	Standard Deviation 2.0765
Eliglustat	Change From Eliglustat Baseline in Total Bone Marrow Burden (BMB) Scores at Weeks 52, 104, 156 and 208	Total BMB Score: Week 208	8.7879 score on a scale	Standard Deviation 3.0334
Eliglustat	Change From Eliglustat Baseline in Total Bone Marrow Burden (BMB) Scores at Weeks 52, 104, 156 and 208	Total BMB Score: Change at Week 104	-0.1818 score on a scale	Standard Deviation 2.0459
Eliglustat	Change From Eliglustat Baseline in Total Bone Marrow Burden (BMB) Scores at Weeks 52, 104, 156 and 208	Total BMB Score: Change at Week 208	-1.0606 score on a scale	Standard Deviation 2.7641
Eliglustat	Change From Eliglustat Baseline in Total Bone Marrow Burden (BMB) Scores at Weeks 52, 104, 156 and 208	Total BMB Score: Eliglustat Baseline	10.4286 score on a scale	Standard Deviation 2.8146
Eliglustat: Participants From ENCORE	Change From Eliglustat Baseline in Total Bone Marrow Burden (BMB) Scores at Weeks 52, 104, 156 and 208	Total BMB Score: Change at Week 208	3.3333 score on a scale	Standard Deviation 3.383
Eliglustat: Participants From ENCORE	Change From Eliglustat Baseline in Total Bone Marrow Burden (BMB) Scores at Weeks 52, 104, 156 and 208	Total BMB Score: Eliglustat Baseline	6.8333 score on a scale	Standard Deviation 2.4114
Eliglustat: Participants From ENCORE	Change From Eliglustat Baseline in Total Bone Marrow Burden (BMB) Scores at Weeks 52, 104, 156 and 208	Total BMB Score: Week 52	8.8333 score on a scale	Standard Deviation 2.1858
Eliglustat: Participants From ENCORE	Change From Eliglustat Baseline in Total Bone Marrow Burden (BMB) Scores at Weeks 52, 104, 156 and 208	Total BMB Score: Change at Week 52	2.0000 score on a scale	Standard Deviation 1.2472
Eliglustat: Participants From ENCORE	Change From Eliglustat Baseline in Total Bone Marrow Burden (BMB) Scores at Weeks 52, 104, 156 and 208	Total BMB Score: Week 104	10.0833 score on a scale	Standard Deviation 1.2874
Eliglustat: Participants From ENCORE	Change From Eliglustat Baseline in Total Bone Marrow Burden (BMB) Scores at Weeks 52, 104, 156 and 208	Total BMB Score: Change at Week 104	3.2500 score on a scale	Standard Deviation 2.658
Eliglustat: Participants From ENCORE	Change From Eliglustat Baseline in Total Bone Marrow Burden (BMB) Scores at Weeks 52, 104, 156 and 208	Total BMB Score: Week 156	10.6667 score on a scale	Standard Deviation 0.9428
Eliglustat: Participants From ENCORE	Change From Eliglustat Baseline in Total Bone Marrow Burden (BMB) Scores at Weeks 52, 104, 156 and 208	Total BMB Score: Change at Week 156	3.3333 score on a scale	Standard Deviation 2.357
Eliglustat: Participants From ENCORE	Change From Eliglustat Baseline in Total Bone Marrow Burden (BMB) Scores at Weeks 52, 104, 156 and 208	Total BMB Score: Week 208	9.7778 score on a scale	Standard Deviation 1.3878

Primary

Change From Eliglustat Baseline in Total Spine and Femur Bone Mineral Density (BMD) at Weeks 52, 104, 156 and 208

BMD measurements of the spine and bilateral femur were acquired by DXA scan. Worst total femur at Baseline refers to the worst diseased left or right femur at baseline. For this outcome measure, baseline refers to the eliglustat baseline, which was defined as participant's status at the time of first dose of eliglustat in the previous Phase 2 or Phase 3 study.

Time frame: Eliglustat Baseline, Weeks 52, 104, 156 and 208 of the current study

Population: Analysis was performed on FAS population. Here, number analyzed = participants with available data for each specified category. Data for this outcome measure was was planned to be collected and analyzed in reference to eliglustat baseline separately for participants from Phase 2, ENGAGE and EDGE studies (GD treatment naïve participants) and from ENCORE study (participants switched from ERT).

Arm	Measure	Group	Value (MEAN)	Dispersion
Eliglustat	Change From Eliglustat Baseline in Total Spine and Femur Bone Mineral Density (BMD) at Weeks 52, 104, 156 and 208	Total Spine BMD: Eliglustat Baseline	1.0757 g/cm^2	Standard Deviation 0.1322
Eliglustat	Change From Eliglustat Baseline in Total Spine and Femur Bone Mineral Density (BMD) at Weeks 52, 104, 156 and 208	Total Spine BMD: Week 52	1.1495 g/cm^2	Standard Deviation 0.1307
Eliglustat	Change From Eliglustat Baseline in Total Spine and Femur Bone Mineral Density (BMD) at Weeks 52, 104, 156 and 208	Total Spine BMD: Change at Week 52	0.0582 g/cm^2	Standard Deviation 0.0656
Eliglustat	Change From Eliglustat Baseline in Total Spine and Femur Bone Mineral Density (BMD) at Weeks 52, 104, 156 and 208	Total Spine BMD: Week 104	1.1280 g/cm^2	Standard Deviation 0.1216
Eliglustat	Change From Eliglustat Baseline in Total Spine and Femur Bone Mineral Density (BMD) at Weeks 52, 104, 156 and 208	Total Spine BMD: Change at Week 104	0.0600 g/cm^2	Standard Deviation 0.0633
Eliglustat	Change From Eliglustat Baseline in Total Spine and Femur Bone Mineral Density (BMD) at Weeks 52, 104, 156 and 208	Total Spine BMD: Week 156	1.1354 g/cm^2	Standard Deviation 0.1153
Eliglustat	Change From Eliglustat Baseline in Total Spine and Femur Bone Mineral Density (BMD) at Weeks 52, 104, 156 and 208	Total Spine BMD: Change at Week 156	0.0712 g/cm^2	Standard Deviation 0.0718
Eliglustat	Change From Eliglustat Baseline in Total Spine and Femur Bone Mineral Density (BMD) at Weeks 52, 104, 156 and 208	Total Spine BMD: Week 208	1.1168 g/cm^2	Standard Deviation 0.112
Eliglustat	Change From Eliglustat Baseline in Total Spine and Femur Bone Mineral Density (BMD) at Weeks 52, 104, 156 and 208	Total Spine BMD: Change at Week 208	0.0616 g/cm^2	Standard Deviation 0.0799
Eliglustat	Change From Eliglustat Baseline in Total Spine and Femur Bone Mineral Density (BMD) at Weeks 52, 104, 156 and 208	Worst Total Femur BMD: Eliglustat Baseline	1.0330 g/cm^2	Standard Deviation 0.155
Eliglustat	Change From Eliglustat Baseline in Total Spine and Femur Bone Mineral Density (BMD) at Weeks 52, 104, 156 and 208	Worst Total Femur BMD: Week 52	1.0643 g/cm^2	Standard Deviation 0.1565
Eliglustat	Change From Eliglustat Baseline in Total Spine and Femur Bone Mineral Density (BMD) at Weeks 52, 104, 156 and 208	Worst Total Femur BMD: Change at Week 52	0.0169 g/cm^2	Standard Deviation 0.0495
Eliglustat	Change From Eliglustat Baseline in Total Spine and Femur Bone Mineral Density (BMD) at Weeks 52, 104, 156 and 208	Worst Total Femur BMD: Week 104	1.0346 g/cm^2	Standard Deviation 0.1485
Eliglustat	Change From Eliglustat Baseline in Total Spine and Femur Bone Mineral Density (BMD) at Weeks 52, 104, 156 and 208	Worst Total Femur BMD: Change at Week 104	0.0118 g/cm^2	Standard Deviation 0.046
Eliglustat	Change From Eliglustat Baseline in Total Spine and Femur Bone Mineral Density (BMD) at Weeks 52, 104, 156 and 208	Worst Total Femur BMD: Week 156	1.0380 g/cm^2	Standard Deviation 0.1515
Eliglustat	Change From Eliglustat Baseline in Total Spine and Femur Bone Mineral Density (BMD) at Weeks 52, 104, 156 and 208	Worst Total Femur BMD: Change at Week 156	0.0152 g/cm^2	Standard Deviation 0.0586
Eliglustat	Change From Eliglustat Baseline in Total Spine and Femur Bone Mineral Density (BMD) at Weeks 52, 104, 156 and 208	Worst Total Femur BMD: Week 208	1.0304 g/cm^2	Standard Deviation 0.1547
Eliglustat	Change From Eliglustat Baseline in Total Spine and Femur Bone Mineral Density (BMD) at Weeks 52, 104, 156 and 208	Worst Total Femur BMD: Change at Week 208	0.0198 g/cm^2	Standard Deviation 0.0598
Eliglustat: Participants From ENCORE	Change From Eliglustat Baseline in Total Spine and Femur Bone Mineral Density (BMD) at Weeks 52, 104, 156 and 208	Worst Total Femur BMD: Change at Week 104	-0.0340 g/cm^2	Standard Deviation 0.0551
Eliglustat: Participants From ENCORE	Change From Eliglustat Baseline in Total Spine and Femur Bone Mineral Density (BMD) at Weeks 52, 104, 156 and 208	Total Spine BMD: Eliglustat Baseline	1.1350 g/cm^2	Standard Deviation 0.0896
Eliglustat: Participants From ENCORE	Change From Eliglustat Baseline in Total Spine and Femur Bone Mineral Density (BMD) at Weeks 52, 104, 156 and 208	Worst Total Femur BMD: Eliglustat Baseline	1.1600 g/cm^2	Standard Deviation 0.1356
Eliglustat: Participants From ENCORE	Change From Eliglustat Baseline in Total Spine and Femur Bone Mineral Density (BMD) at Weeks 52, 104, 156 and 208	Total Spine BMD: Week 52	1.1245 g/cm^2	Standard Deviation 0.0035
Eliglustat: Participants From ENCORE	Change From Eliglustat Baseline in Total Spine and Femur Bone Mineral Density (BMD) at Weeks 52, 104, 156 and 208	Worst Total Femur BMD: Change at Week 208	-0.0503 g/cm^2	Standard Deviation 0.0715
Eliglustat: Participants From ENCORE	Change From Eliglustat Baseline in Total Spine and Femur Bone Mineral Density (BMD) at Weeks 52, 104, 156 and 208	Total Spine BMD: Change at Week 52	0.0195 g/cm^2	Standard Deviation 0.053
Eliglustat: Participants From ENCORE	Change From Eliglustat Baseline in Total Spine and Femur Bone Mineral Density (BMD) at Weeks 52, 104, 156 and 208	Worst Total Femur BMD: Week 52	1.0853 g/cm^2	Standard Deviation 0.0634
Eliglustat: Participants From ENCORE	Change From Eliglustat Baseline in Total Spine and Femur Bone Mineral Density (BMD) at Weeks 52, 104, 156 and 208	Total Spine BMD: Week 104	1.1615 g/cm^2	Standard Deviation 0.1371
Eliglustat: Participants From ENCORE	Change From Eliglustat Baseline in Total Spine and Femur Bone Mineral Density (BMD) at Weeks 52, 104, 156 and 208	Worst Total Femur BMD: Week 156	1.1328 g/cm^2	Standard Deviation 0.1358
Eliglustat: Participants From ENCORE	Change From Eliglustat Baseline in Total Spine and Femur Bone Mineral Density (BMD) at Weeks 52, 104, 156 and 208	Total Spine BMD: Change at Week 104	0.0265 g/cm^2	Standard Deviation 0.0617
Eliglustat: Participants From ENCORE	Change From Eliglustat Baseline in Total Spine and Femur Bone Mineral Density (BMD) at Weeks 52, 104, 156 and 208	Worst Total Femur BMD: Change at Week 52	-0.0080 g/cm^2	Standard Deviation 0.0686
Eliglustat: Participants From ENCORE	Change From Eliglustat Baseline in Total Spine and Femur Bone Mineral Density (BMD) at Weeks 52, 104, 156 and 208	Total Spine BMD: Week 156	1.1498 g/cm^2	Standard Deviation 0.148
Eliglustat: Participants From ENCORE	Change From Eliglustat Baseline in Total Spine and Femur Bone Mineral Density (BMD) at Weeks 52, 104, 156 and 208	Worst Total Femur BMD: Week 208	1.1430 g/cm^2	Standard Deviation 0.1572
Eliglustat: Participants From ENCORE	Change From Eliglustat Baseline in Total Spine and Femur Bone Mineral Density (BMD) at Weeks 52, 104, 156 and 208	Total Spine BMD: Change at Week 156	0.0148 g/cm^2	Standard Deviation 0.0692
Eliglustat: Participants From ENCORE	Change From Eliglustat Baseline in Total Spine and Femur Bone Mineral Density (BMD) at Weeks 52, 104, 156 and 208	Worst Total Femur BMD: Week 104	1.1260 g/cm^2	Standard Deviation 0.1253
Eliglustat: Participants From ENCORE	Change From Eliglustat Baseline in Total Spine and Femur Bone Mineral Density (BMD) at Weeks 52, 104, 156 and 208	Total Spine BMD: Week 208	1.1757 g/cm^2	Standard Deviation 0.1466
Eliglustat: Participants From ENCORE	Change From Eliglustat Baseline in Total Spine and Femur Bone Mineral Density (BMD) at Weeks 52, 104, 156 and 208	Worst Total Femur BMD: Change at Week 156	-0.0273 g/cm^2	Standard Deviation 0.0614
Eliglustat: Participants From ENCORE	Change From Eliglustat Baseline in Total Spine and Femur Bone Mineral Density (BMD) at Weeks 52, 104, 156 and 208	Total Spine BMD: Change at Week 208	0.0190 g/cm^2	Standard Deviation 0.0596

Primary

Number of Participants With Bone Crisis at Study Baseline, Weeks 52, 104, 156 and 208

Bone crisis was assessed as a part of Gaucher disease assessment in participants. Acute, excruciating episodic bone pain is characteristic of Gaucher bone crisis, which typically causes periosteal elevation, elevated white blood cell count, fever, or debilitation lasting several days or longer and requires treatment with immobilization of the affected area, and opioid analgesics. Participants were categorized as 0= no bone crisis, 1= 1 bone crisis, 2= 2 bone crisis and \>=3 = more than 3 bone crisis during the assessment period. In this outcome measure, number of participants with different bone crises levels at specified time points were reported. Baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry.

Time frame: Study Baseline, Weeks 52, 104, 156 and 208

Arm	Measure	Group	Value (COUNT_OF_PARTICIPANTS)
Eliglustat	Number of Participants With Bone Crisis at Study Baseline, Weeks 52, 104, 156 and 208	Bone Crisis (0): Study Baseline	31 Participants
Eliglustat	Number of Participants With Bone Crisis at Study Baseline, Weeks 52, 104, 156 and 208	Bone Crisis (1): Study Baseline	0 Participants
Eliglustat	Number of Participants With Bone Crisis at Study Baseline, Weeks 52, 104, 156 and 208	Bone Crisis (2): Study Baseline	0 Participants
Eliglustat	Number of Participants With Bone Crisis at Study Baseline, Weeks 52, 104, 156 and 208	Bone Crisis (>=3): Study Baseline	0 Participants
Eliglustat	Number of Participants With Bone Crisis at Study Baseline, Weeks 52, 104, 156 and 208	Bone Crisis (0): Week 52	31 Participants
Eliglustat	Number of Participants With Bone Crisis at Study Baseline, Weeks 52, 104, 156 and 208	Bone Crisis (1): Week 52	0 Participants
Eliglustat	Number of Participants With Bone Crisis at Study Baseline, Weeks 52, 104, 156 and 208	Bone Crisis (2): Week 52	0 Participants
Eliglustat	Number of Participants With Bone Crisis at Study Baseline, Weeks 52, 104, 156 and 208	Bone Crisis (>=3): Week 52	0 Participants
Eliglustat	Number of Participants With Bone Crisis at Study Baseline, Weeks 52, 104, 156 and 208	Bone Crisis (0): Week 104	29 Participants
Eliglustat	Number of Participants With Bone Crisis at Study Baseline, Weeks 52, 104, 156 and 208	Bone Crisis (1): Week 104	0 Participants
Eliglustat	Number of Participants With Bone Crisis at Study Baseline, Weeks 52, 104, 156 and 208	Bone Crisis (2): Week 104	0 Participants
Eliglustat	Number of Participants With Bone Crisis at Study Baseline, Weeks 52, 104, 156 and 208	Bone Crisis (>=3): Week 104	0 Participants
Eliglustat	Number of Participants With Bone Crisis at Study Baseline, Weeks 52, 104, 156 and 208	Bone Crisis (0): Week 156	28 Participants
Eliglustat	Number of Participants With Bone Crisis at Study Baseline, Weeks 52, 104, 156 and 208	Bone Crisis (1): Week 156	1 Participants
Eliglustat	Number of Participants With Bone Crisis at Study Baseline, Weeks 52, 104, 156 and 208	Bone Crisis (2): Week 156	0 Participants
Eliglustat	Number of Participants With Bone Crisis at Study Baseline, Weeks 52, 104, 156 and 208	Bone Crisis (>=3): Week 156	0 Participants
Eliglustat	Number of Participants With Bone Crisis at Study Baseline, Weeks 52, 104, 156 and 208	Bone Crisis (0): Week 208	29 Participants
Eliglustat	Number of Participants With Bone Crisis at Study Baseline, Weeks 52, 104, 156 and 208	Bone Crisis (1): Week 208	0 Participants
Eliglustat	Number of Participants With Bone Crisis at Study Baseline, Weeks 52, 104, 156 and 208	Bone Crisis (2): Week 208	0 Participants
Eliglustat	Number of Participants With Bone Crisis at Study Baseline, Weeks 52, 104, 156 and 208	Bone Crisis (>=3): Week 208	0 Participants

Primary

Number of Participants With Bone Pain Levels During the Past 4 Weeks at Study Baseline, Weeks 52, 104, 156 and 208

Bone pain was assessed as a part of Gaucher disease assessment in participants. Participants were categorized as none (no bone pain), very mild bone pain, mild bone pain, moderate bone pain, severe bone pain and extreme bone pain during the past 4 weeks at each specified visit. In this outcome measure, number of participants with different level of bone pain during the past 4 weeks at specified time points were reported. Baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry.

Time frame: Study Baseline, Weeks 52, 104, 156 and 208

Arm	Measure	Group	Value (COUNT_OF_PARTICIPANTS)
Eliglustat	Number of Participants With Bone Pain Levels During the Past 4 Weeks at Study Baseline, Weeks 52, 104, 156 and 208	None: Study Baseline	28 Participants
Eliglustat	Number of Participants With Bone Pain Levels During the Past 4 Weeks at Study Baseline, Weeks 52, 104, 156 and 208	Very Mild: Study Baseline	1 Participants
Eliglustat	Number of Participants With Bone Pain Levels During the Past 4 Weeks at Study Baseline, Weeks 52, 104, 156 and 208	Mild: Study Baseline	1 Participants
Eliglustat	Number of Participants With Bone Pain Levels During the Past 4 Weeks at Study Baseline, Weeks 52, 104, 156 and 208	Moderate: Study Baseline	1 Participants
Eliglustat	Number of Participants With Bone Pain Levels During the Past 4 Weeks at Study Baseline, Weeks 52, 104, 156 and 208	Severe: Study Baseline	0 Participants
Eliglustat	Number of Participants With Bone Pain Levels During the Past 4 Weeks at Study Baseline, Weeks 52, 104, 156 and 208	Extreme: Study Baseline	0 Participants
Eliglustat	Number of Participants With Bone Pain Levels During the Past 4 Weeks at Study Baseline, Weeks 52, 104, 156 and 208	None: Week 52	29 Participants
Eliglustat	Number of Participants With Bone Pain Levels During the Past 4 Weeks at Study Baseline, Weeks 52, 104, 156 and 208	Very Mild: Week 52	1 Participants
Eliglustat	Number of Participants With Bone Pain Levels During the Past 4 Weeks at Study Baseline, Weeks 52, 104, 156 and 208	Mild: Week 52	0 Participants
Eliglustat	Number of Participants With Bone Pain Levels During the Past 4 Weeks at Study Baseline, Weeks 52, 104, 156 and 208	Moderate: Week 52	1 Participants
Eliglustat	Number of Participants With Bone Pain Levels During the Past 4 Weeks at Study Baseline, Weeks 52, 104, 156 and 208	Severe: Week 52	0 Participants
Eliglustat	Number of Participants With Bone Pain Levels During the Past 4 Weeks at Study Baseline, Weeks 52, 104, 156 and 208	Extreme: Week 52	0 Participants
Eliglustat	Number of Participants With Bone Pain Levels During the Past 4 Weeks at Study Baseline, Weeks 52, 104, 156 and 208	None: Week 104	28 Participants
Eliglustat	Number of Participants With Bone Pain Levels During the Past 4 Weeks at Study Baseline, Weeks 52, 104, 156 and 208	Very Mild: Week 104	1 Participants
Eliglustat	Number of Participants With Bone Pain Levels During the Past 4 Weeks at Study Baseline, Weeks 52, 104, 156 and 208	Mild: Week 104	0 Participants
Eliglustat	Number of Participants With Bone Pain Levels During the Past 4 Weeks at Study Baseline, Weeks 52, 104, 156 and 208	Moderate: Week 104	0 Participants
Eliglustat	Number of Participants With Bone Pain Levels During the Past 4 Weeks at Study Baseline, Weeks 52, 104, 156 and 208	Severe: Week 104	0 Participants
Eliglustat	Number of Participants With Bone Pain Levels During the Past 4 Weeks at Study Baseline, Weeks 52, 104, 156 and 208	Extreme: Week 104	0 Participants
Eliglustat	Number of Participants With Bone Pain Levels During the Past 4 Weeks at Study Baseline, Weeks 52, 104, 156 and 208	None: Week 156	29 Participants
Eliglustat	Number of Participants With Bone Pain Levels During the Past 4 Weeks at Study Baseline, Weeks 52, 104, 156 and 208	Very mild: Week 156	0 Participants
Eliglustat	Number of Participants With Bone Pain Levels During the Past 4 Weeks at Study Baseline, Weeks 52, 104, 156 and 208	Mild: Week 156	0 Participants
Eliglustat	Number of Participants With Bone Pain Levels During the Past 4 Weeks at Study Baseline, Weeks 52, 104, 156 and 208	Moderate: Week 156	0 Participants
Eliglustat	Number of Participants With Bone Pain Levels During the Past 4 Weeks at Study Baseline, Weeks 52, 104, 156 and 208	Severe: Week 156	0 Participants
Eliglustat	Number of Participants With Bone Pain Levels During the Past 4 Weeks at Study Baseline, Weeks 52, 104, 156 and 208	Extreme: Week 156	0 Participants
Eliglustat	Number of Participants With Bone Pain Levels During the Past 4 Weeks at Study Baseline, Weeks 52, 104, 156 and 208	None: Week 208	28 Participants
Eliglustat	Number of Participants With Bone Pain Levels During the Past 4 Weeks at Study Baseline, Weeks 52, 104, 156 and 208	Very Mild: Week 208	1 Participants
Eliglustat	Number of Participants With Bone Pain Levels During the Past 4 Weeks at Study Baseline, Weeks 52, 104, 156 and 208	Mild: Week 208	0 Participants
Eliglustat	Number of Participants With Bone Pain Levels During the Past 4 Weeks at Study Baseline, Weeks 52, 104, 156 and 208	Moderate: Week 208	0 Participants
Eliglustat	Number of Participants With Bone Pain Levels During the Past 4 Weeks at Study Baseline, Weeks 52, 104, 156 and 208	Severe: Week 208	0 Participants
Eliglustat	Number of Participants With Bone Pain Levels During the Past 4 Weeks at Study Baseline, Weeks 52, 104, 156 and 208	Extreme: Week 208	0 Participants

Primary

Number of Participants With Mobility Status Assessments at Study Baseline, Weeks 52, 104, 156 and 208

Mobility, i.e., ability to walk was assessed as a part of Gaucher disease assessment in participants. In this outcome measure, number of participants with their different mobility status along with the use of mobility aids (unrestricted mobility, walks with difficulty, walks with orthopaedic aid, requires wheelchair, bedridden) at specified time points were reported. Baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry.

Time frame: Study Baseline, Weeks 52, 104, 156 and 208

Arm	Measure	Group	Value (COUNT_OF_PARTICIPANTS)
Eliglustat	Number of Participants With Mobility Status Assessments at Study Baseline, Weeks 52, 104, 156 and 208	Unrestricted mobility: Study Baseline	31 Participants
Eliglustat	Number of Participants With Mobility Status Assessments at Study Baseline, Weeks 52, 104, 156 and 208	Walks with difficulty: Study Baseline	0 Participants
Eliglustat	Number of Participants With Mobility Status Assessments at Study Baseline, Weeks 52, 104, 156 and 208	Walks with orthopedic aid: Study Baseline	0 Participants
Eliglustat	Number of Participants With Mobility Status Assessments at Study Baseline, Weeks 52, 104, 156 and 208	Requires wheelchair: Study Baseline	0 Participants
Eliglustat	Number of Participants With Mobility Status Assessments at Study Baseline, Weeks 52, 104, 156 and 208	Bedridden: Study Baseline	0 Participants
Eliglustat	Number of Participants With Mobility Status Assessments at Study Baseline, Weeks 52, 104, 156 and 208	Unrestricted mobility: Week 52	31 Participants
Eliglustat	Number of Participants With Mobility Status Assessments at Study Baseline, Weeks 52, 104, 156 and 208	Walks with difficulty: Week 52	0 Participants
Eliglustat	Number of Participants With Mobility Status Assessments at Study Baseline, Weeks 52, 104, 156 and 208	Walks with orthopedic aid: Week 52	0 Participants
Eliglustat	Number of Participants With Mobility Status Assessments at Study Baseline, Weeks 52, 104, 156 and 208	Requires wheelchair: Week 52	0 Participants
Eliglustat	Number of Participants With Mobility Status Assessments at Study Baseline, Weeks 52, 104, 156 and 208	Bedridden: Week 52	0 Participants
Eliglustat	Number of Participants With Mobility Status Assessments at Study Baseline, Weeks 52, 104, 156 and 208	Unrestricted mobility: Week 104	28 Participants
Eliglustat	Number of Participants With Mobility Status Assessments at Study Baseline, Weeks 52, 104, 156 and 208	Walks with Difficulty: Week 104	1 Participants
Eliglustat	Number of Participants With Mobility Status Assessments at Study Baseline, Weeks 52, 104, 156 and 208	Walks with orthopedic aid: Week 104	0 Participants
Eliglustat	Number of Participants With Mobility Status Assessments at Study Baseline, Weeks 52, 104, 156 and 208	Requires wheelchair: Week 104	0 Participants
Eliglustat	Number of Participants With Mobility Status Assessments at Study Baseline, Weeks 52, 104, 156 and 208	Bedridden: Week 104	0 Participants
Eliglustat	Number of Participants With Mobility Status Assessments at Study Baseline, Weeks 52, 104, 156 and 208	Unrestricted mobility: Week 156	29 Participants
Eliglustat	Number of Participants With Mobility Status Assessments at Study Baseline, Weeks 52, 104, 156 and 208	Walks with difficulty: Week 156	0 Participants
Eliglustat	Number of Participants With Mobility Status Assessments at Study Baseline, Weeks 52, 104, 156 and 208	Walks with orthopedic aid: Week 156	0 Participants
Eliglustat	Number of Participants With Mobility Status Assessments at Study Baseline, Weeks 52, 104, 156 and 208	Requires wheelchair: Week 156	0 Participants
Eliglustat	Number of Participants With Mobility Status Assessments at Study Baseline, Weeks 52, 104, 156 and 208	Bedridden: Week 156	0 Participants
Eliglustat	Number of Participants With Mobility Status Assessments at Study Baseline, Weeks 52, 104, 156 and 208	Unrestricted mobility: Week 208	29 Participants
Eliglustat	Number of Participants With Mobility Status Assessments at Study Baseline, Weeks 52, 104, 156 and 208	Walks with difficulty: Week 208	0 Participants
Eliglustat	Number of Participants With Mobility Status Assessments at Study Baseline, Weeks 52, 104, 156 and 208	Walks with orthopedic aid: Week 208	0 Participants
Eliglustat	Number of Participants With Mobility Status Assessments at Study Baseline, Weeks 52, 104, 156 and 208	Requires wheelchair: Week 208	0 Participants
Eliglustat	Number of Participants With Mobility Status Assessments at Study Baseline, Weeks 52, 104, 156 and 208	Bedridden: Week 208	0 Participants

Primary

Observed Annual Incidence Rate for Spine and Femur Fracture at Week 52, 104, 156 and 208

Observed annual incidence rate was estimated using the total number of events divided by the total years of follow-up in each specified year (for all participants). Fracture was assessed by bone MRI and X-Ray for spine and by MRI for femur.

Time frame: For 52 Weeks (i.e., 1 year),104 Weeks (i.e., 2 year), 156 Weeks (i.e., 3 year) and 208 Weeks (i.e., 4 years)

Population: Analysis was performed on FAS population.

Arm	Measure	Group	Value (NUMBER)
Eliglustat	Observed Annual Incidence Rate for Spine and Femur Fracture at Week 52, 104, 156 and 208	Spine Fracture (MRI): Week 52	0.1350 events per participant-year
Eliglustat	Observed Annual Incidence Rate for Spine and Femur Fracture at Week 52, 104, 156 and 208	Spine Fracture (MRI): Week 104	0.0366 events per participant-year
Eliglustat	Observed Annual Incidence Rate for Spine and Femur Fracture at Week 52, 104, 156 and 208	Spine Fracture(MRI): Week 156	0 events per participant-year
Eliglustat	Observed Annual Incidence Rate for Spine and Femur Fracture at Week 52, 104, 156 and 208	Spine Fracture (MRI): Week 208	0.0104 events per participant-year
Eliglustat	Observed Annual Incidence Rate for Spine and Femur Fracture at Week 52, 104, 156 and 208	Spine Fracture (X-ray): Week 104	0.0369 events per participant-year
Eliglustat	Observed Annual Incidence Rate for Spine and Femur Fracture at Week 52, 104, 156 and 208	Spine Fracture (X-ray): Week 208	0 events per participant-year
Eliglustat	Observed Annual Incidence Rate for Spine and Femur Fracture at Week 52, 104, 156 and 208	Femur Fracture (MRI): Week 52	0 events per participant-year
Eliglustat	Observed Annual Incidence Rate for Spine and Femur Fracture at Week 52, 104, 156 and 208	Femur Fracture (MRI): Week 104	0 events per participant-year
Eliglustat	Observed Annual Incidence Rate for Spine and Femur Fracture at Week 52, 104, 156 and 208	Femur Fracture (MRI): Week 156	0 events per participant-year
Eliglustat	Observed Annual Incidence Rate for Spine and Femur Fracture at Week 52, 104, 156 and 208	Femur Fracture (MRI): Week 208	0 events per participant-year

Primary

Observed Annual Incidence Rate for Spine and Femur Infarcts at Week 52, 104, 156 and 208

Observed annual incidence rate was estimated using the total number of events divided by the total years of follow-up in each specified year (for all participants). Infarcts were assessed by bone MRI and X-Ray for spine and by MRI for femur.

Time frame: For 52 Weeks (i.e., 1 year),104 Weeks (i.e., 2 year), 156 Weeks (i.e., 3 year) and 208 Weeks (i.e., 4 years)

Population: Analysis was performed on FAS population.

Arm	Measure	Group	Value (NUMBER)
Eliglustat	Observed Annual Incidence Rate for Spine and Femur Infarcts at Week 52, 104, 156 and 208	Femur Infarcts (MRI): Week 104	0.2470 events per participant-year
Eliglustat	Observed Annual Incidence Rate for Spine and Femur Infarcts at Week 52, 104, 156 and 208	Femur Infarcts (MRI): Week 156	0.1664 events per participant-year
Eliglustat	Observed Annual Incidence Rate for Spine and Femur Infarcts at Week 52, 104, 156 and 208	Spine Infarcts (MRI): Week 52	0 events per participant-year
Eliglustat	Observed Annual Incidence Rate for Spine and Femur Infarcts at Week 52, 104, 156 and 208	Spine Infarcts (MRI): Week 104	0 events per participant-year
Eliglustat	Observed Annual Incidence Rate for Spine and Femur Infarcts at Week 52, 104, 156 and 208	Spine Infarcts (MRI): Week 156	0 events per participant-year
Eliglustat	Observed Annual Incidence Rate for Spine and Femur Infarcts at Week 52, 104, 156 and 208	Spine Infarcts (MRI): Week 208	0 events per participant-year
Eliglustat	Observed Annual Incidence Rate for Spine and Femur Infarcts at Week 52, 104, 156 and 208	Spine Infarcts (X-ray): Week 104	0 events per participant-year
Eliglustat	Observed Annual Incidence Rate for Spine and Femur Infarcts at Week 52, 104, 156 and 208	Spine Infarcts (X-ray): Week 208	0 events per participant-year
Eliglustat	Observed Annual Incidence Rate for Spine and Femur Infarcts at Week 52, 104, 156 and 208	Femur Infarcts (MRI): Week 52	0.6752 events per participant-year
Eliglustat	Observed Annual Incidence Rate for Spine and Femur Infarcts at Week 52, 104, 156 and 208	Femur Infarcts (MRI): Week 208	0.1145 events per participant-year

Primary

Observed Annual Incidence Rate for Spine and Femur Osteonecrosis at Week 52, 104, 156 and 208

Observed annual incidence rate was estimated using the total number of events divided by the total years of follow-up in each specified year (for all participants). Osteonecrosis was assessed by bone MRI and X-Ray for spine and by MRI for femur.

Time frame: For 52 Weeks (i.e., 1 year),104 Weeks (i.e., 2 year), 156 Weeks (i.e., 3 year) and 208 Weeks (i.e., 4 years)

Population: Analysis was performed on FAS population.

Arm	Measure	Group	Value (NUMBER)
Eliglustat	Observed Annual Incidence Rate for Spine and Femur Osteonecrosis at Week 52, 104, 156 and 208	Spine Osteonecrosis (MRI): Week 52	0 events per participant-year
Eliglustat	Observed Annual Incidence Rate for Spine and Femur Osteonecrosis at Week 52, 104, 156 and 208	Spine Osteonecrosis (MRI): Week 104	0 events per participant-year
Eliglustat	Observed Annual Incidence Rate for Spine and Femur Osteonecrosis at Week 52, 104, 156 and 208	Spine Osteonecrosis (MRI): Week 156	0 events per participant-year
Eliglustat	Observed Annual Incidence Rate for Spine and Femur Osteonecrosis at Week 52, 104, 156 and 208	Spine Osteonecrosis (MRI): Week 208	0 events per participant-year
Eliglustat	Observed Annual Incidence Rate for Spine and Femur Osteonecrosis at Week 52, 104, 156 and 208	Spine Osteonecrosis (X-ray): Week 104	0 events per participant-year
Eliglustat	Observed Annual Incidence Rate for Spine and Femur Osteonecrosis at Week 52, 104, 156 and 208	Spine Osteonecrosis (X-ray): Week 208	0 events per participant-year
Eliglustat	Observed Annual Incidence Rate for Spine and Femur Osteonecrosis at Week 52, 104, 156 and 208	Femur Osteonecrosis (MRI): Week 52	0.1013 events per participant-year
Eliglustat	Observed Annual Incidence Rate for Spine and Femur Osteonecrosis at Week 52, 104, 156 and 208	Femur Osteonecrosis (MRI): Week 104	0.0353 events per participant-year
Eliglustat	Observed Annual Incidence Rate for Spine and Femur Osteonecrosis at Week 52, 104, 156 and 208	Femur Osteonecrosis (MRI): Week 156	0.0384 events per participant-year
Eliglustat	Observed Annual Incidence Rate for Spine and Femur Osteonecrosis at Week 52, 104, 156 and 208	Femur Osteonecrosis (MRI): Week 208	0.0104 events per participant-year

Primary

Observed Annual Incidence Rate for Spine Lytic Lesion at Week 104, and 208

Observed annual incidence rate was estimated using the total number of events divided by the total years of follow-up in each specified year (for all participants). Lytic Lesions were assessed by bone X-Ray for spine.

Time frame: For 104 Weeks (i.e., 2 year), and 208 Weeks (i.e., 4 years)

Population: Analysis was performed on FAS population.

Arm	Measure	Group	Value (NUMBER)
Eliglustat	Observed Annual Incidence Rate for Spine Lytic Lesion at Week 104, and 208	Spine Lytic Lesions (X-ray): Week 208	0 events per participant-year
Eliglustat	Observed Annual Incidence Rate for Spine Lytic Lesion at Week 104, and 208	Spine Lytic Lesions (X-ray): Week 104	0 events per participant-year

Primary

Total Number of New or Worsening Fracture Events for Spine and Femur at Study Baseline, Week 52, 104, 156 and 208

Fracture was assessed by bone MRI and X-Ray for spine and by MRI for femur. Total number of new or worsening fracture events among all the participants with corresponding assessment at specified time points were reported in this outcome measure. For this outcome measure, baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry.

Time frame: Study Baseline, Weeks 52, 104, 156 and 208

Arm	Measure	Group	Value (MEAN)
Eliglustat	Total Number of New or Worsening Fracture Events for Spine and Femur at Study Baseline, Week 52, 104, 156 and 208	Spine Fracture (MRI): Study Baseline	0.0000 events
Eliglustat	Total Number of New or Worsening Fracture Events for Spine and Femur at Study Baseline, Week 52, 104, 156 and 208	Spine Fracture (MRI): Week 52	0.0690 events
Eliglustat	Total Number of New or Worsening Fracture Events for Spine and Femur at Study Baseline, Week 52, 104, 156 and 208	Spine Fracture (MRI): Week 104	0.0000 events
Eliglustat	Total Number of New or Worsening Fracture Events for Spine and Femur at Study Baseline, Week 52, 104, 156 and 208	Spine Fracture (MRI): Week 156	0.0000 events
Eliglustat	Total Number of New or Worsening Fracture Events for Spine and Femur at Study Baseline, Week 52, 104, 156 and 208	Spine Fracture (MRI): Week 208	0.0417 events
Eliglustat	Total Number of New or Worsening Fracture Events for Spine and Femur at Study Baseline, Week 52, 104, 156 and 208	Spine Fracture (X-ray): Study Baseline	0.0000 events
Eliglustat	Total Number of New or Worsening Fracture Events for Spine and Femur at Study Baseline, Week 52, 104, 156 and 208	Spine Fracture (X-ray): Week 104	0.0741 events
Eliglustat	Total Number of New or Worsening Fracture Events for Spine and Femur at Study Baseline, Week 52, 104, 156 and 208	Spine Fracture (X-ray): Week 208	0.0000 events
Eliglustat	Total Number of New or Worsening Fracture Events for Spine and Femur at Study Baseline, Week 52, 104, 156 and 208	Femur Fracture (MRI): Study Baseline	0.0000 events
Eliglustat	Total Number of New or Worsening Fracture Events for Spine and Femur at Study Baseline, Week 52, 104, 156 and 208	Femur Fracture (MRI): Week 52	0.0000 events
Eliglustat	Total Number of New or Worsening Fracture Events for Spine and Femur at Study Baseline, Week 52, 104, 156 and 208	Femur Fracture (MRI): Week 104	0.0000 events
Eliglustat	Total Number of New or Worsening Fracture Events for Spine and Femur at Study Baseline, Week 52, 104, 156 and 208	Femur Fracture (MRI): Week 156	0.0000 events
Eliglustat	Total Number of New or Worsening Fracture Events for Spine and Femur at Study Baseline, Week 52, 104, 156 and 208	Femur Fracture (MRI): Week 208	0.0000 events

Primary

Total Number of New or Worsening Infarcts Events for Spine and Femur at Study Baseline, Week 52, 104, 156 and 208

Infarcts were assessed by bone MRI and X-Ray for spine and by MRI for femur. Total number of new or worsening infarcts events among all the participants with corresponding assessment at specified time points were reported in this outcome measure. For this outcome measure, baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry.

Time frame: Study Baseline, Weeks 52, 104, 156 and 208

Arm	Measure	Group	Value (MEAN)
Eliglustat	Total Number of New or Worsening Infarcts Events for Spine and Femur at Study Baseline, Week 52, 104, 156 and 208	Spine Infarcts (MRI): Study Baseline	0.0000 events
Eliglustat	Total Number of New or Worsening Infarcts Events for Spine and Femur at Study Baseline, Week 52, 104, 156 and 208	Spine Infarcts (MRI): Week 52	0.0000 events
Eliglustat	Total Number of New or Worsening Infarcts Events for Spine and Femur at Study Baseline, Week 52, 104, 156 and 208	Spine Infarcts (MRI): Week 104	0.0000 events
Eliglustat	Total Number of New or Worsening Infarcts Events for Spine and Femur at Study Baseline, Week 52, 104, 156 and 208	Spine Infarcts (MRI): Week 156	0.0000 events
Eliglustat	Total Number of New or Worsening Infarcts Events for Spine and Femur at Study Baseline, Week 52, 104, 156 and 208	Spine Infarcts (MRI): Week 208	0.0000 events
Eliglustat	Total Number of New or Worsening Infarcts Events for Spine and Femur at Study Baseline, Week 52, 104, 156 and 208	Spine Infarcts (X-ray): Study Baseline	0.0000 events
Eliglustat	Total Number of New or Worsening Infarcts Events for Spine and Femur at Study Baseline, Week 52, 104, 156 and 208	Spine Infarcts (X-ray): Week 104	0.0000 events
Eliglustat	Total Number of New or Worsening Infarcts Events for Spine and Femur at Study Baseline, Week 52, 104, 156 and 208	Spine Infarcts (X-ray): Week 208	0.0000 events
Eliglustat	Total Number of New or Worsening Infarcts Events for Spine and Femur at Study Baseline, Week 52, 104, 156 and 208	Femur Infarcts (MRI): Study Baseline	0.6000 events
Eliglustat	Total Number of New or Worsening Infarcts Events for Spine and Femur at Study Baseline, Week 52, 104, 156 and 208	Femur Infarcts (MRI): Week 52	0.2069 events
Eliglustat	Total Number of New or Worsening Infarcts Events for Spine and Femur at Study Baseline, Week 52, 104, 156 and 208	Femur Infarcts (MRI): Week 104	0.0000 events
Eliglustat	Total Number of New or Worsening Infarcts Events for Spine and Femur at Study Baseline, Week 52, 104, 156 and 208	Femur Infarcts (MRI): Week 156	0.0000 events
Eliglustat	Total Number of New or Worsening Infarcts Events for Spine and Femur at Study Baseline, Week 52, 104, 156 and 208	Femur Infarcts (MRI): Week 208	0.0417 events

Primary

Total Number of New or Worsening Lytic Lesions Events for Spine at Study Baseline, Week 104, and 208

Lytic Lesions were assessed by bone X-Ray for spine. Total number of new or worsening lytic lesions events among all the participants with corresponding assessment at specified time points were reported in this outcome measure. For this outcome measure, baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry.

Time frame: Study Baseline, Weeks 104, and 208

Arm	Measure	Group	Value (MEAN)
Eliglustat	Total Number of New or Worsening Lytic Lesions Events for Spine at Study Baseline, Week 104, and 208	Spine Lytic Lesions (X-ray): Study Baseline	0.0385 events
Eliglustat	Total Number of New or Worsening Lytic Lesions Events for Spine at Study Baseline, Week 104, and 208	Spine Lytic Lesions (X-ray): Week 104	0.0000 events
Eliglustat	Total Number of New or Worsening Lytic Lesions Events for Spine at Study Baseline, Week 104, and 208	Spine Lytic Lesions (X-ray): Week 208	0.0000 events

Primary

Total Number of New or Worsening Osteonecrosis Events for Spine and Femur at Study Baseline, Week 52, 104, 156 and 208

Osteonecrosis was assessed by bone MRI and X-Ray for spine and by MRI for femur. Total number of new or worsening osteonecrosis events among all the participants with corresponding assessment at specified time points were reported in this outcome measure. For this outcome measure, baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry.

Time frame: Study Baseline, Weeks 52, 104, 156 and 208

Arm	Measure	Group	Value (MEAN)
Eliglustat	Total Number of New or Worsening Osteonecrosis Events for Spine and Femur at Study Baseline, Week 52, 104, 156 and 208	Spine Osteonecrosis (MRI): Study Baseline	0.0000 events
Eliglustat	Total Number of New or Worsening Osteonecrosis Events for Spine and Femur at Study Baseline, Week 52, 104, 156 and 208	Spine Osteonecrosis (MRI): Week 52	0.0000 events
Eliglustat	Total Number of New or Worsening Osteonecrosis Events for Spine and Femur at Study Baseline, Week 52, 104, 156 and 208	Spine Osteonecrosis (MRI): Week 104	0.0000 events
Eliglustat	Total Number of New or Worsening Osteonecrosis Events for Spine and Femur at Study Baseline, Week 52, 104, 156 and 208	Spine Osteonecrosis (MRI): Week 156	0.0000 events
Eliglustat	Total Number of New or Worsening Osteonecrosis Events for Spine and Femur at Study Baseline, Week 52, 104, 156 and 208	Spine Osteonecrosis (MRI): Week 208	0.0000 events
Eliglustat	Total Number of New or Worsening Osteonecrosis Events for Spine and Femur at Study Baseline, Week 52, 104, 156 and 208	Spine Osteonecrosis (X-ray): Study Baseline	0.0000 events
Eliglustat	Total Number of New or Worsening Osteonecrosis Events for Spine and Femur at Study Baseline, Week 52, 104, 156 and 208	Spine Osteonecrosis (X-ray): Week 104	0.0000 events
Eliglustat	Total Number of New or Worsening Osteonecrosis Events for Spine and Femur at Study Baseline, Week 52, 104, 156 and 208	Spine Osteonecrosis (X-ray): Week 208	0.0000 events
Eliglustat	Total Number of New or Worsening Osteonecrosis Events for Spine and Femur at Study Baseline, Week 52, 104, 156 and 208	Femur Osteonecrosis (MRI): Study Baseline	0.0690 events
Eliglustat	Total Number of New or Worsening Osteonecrosis Events for Spine and Femur at Study Baseline, Week 52, 104, 156 and 208	Femur Osteonecrosis (MRI): Week 52	0.0345 events
Eliglustat	Total Number of New or Worsening Osteonecrosis Events for Spine and Femur at Study Baseline, Week 52, 104, 156 and 208	Femur Osteonecrosis (MRI): Week 104	0.0000 events
Eliglustat	Total Number of New or Worsening Osteonecrosis Events for Spine and Femur at Study Baseline, Week 52, 104, 156 and 208	Femur Osteonecrosis (MRI): Week 156	0.0385 events
Eliglustat	Total Number of New or Worsening Osteonecrosis Events for Spine and Femur at Study Baseline, Week 52, 104, 156 and 208	Femur Osteonecrosis (MRI): Week 208	0.0000 events

Secondary

Change From Current Study Baseline in Gaucher Disease Type 1 (GD1) Biomarker Levels: Chitotriosidase at Week 26, 52, 78, 104, 130, 156, 182, 208 and 234

Chitotriosidase biomarker was assayed from plasma. Chitotriosidase biomarker levels for participants who were CYP2D6 non-Ultra Rapid Metabolizers (non-URM) was reported in this outcome measure. For this outcome measure, baseline refers to the study baseline, which was defined as status at study entry.

Time frame: Study Baseline, Week 26, 52, 78, 104, 130, 156, 182, 208 and 234

Population: Analysis was performed on FAS population. Here, overall number of participants analyzed = participants evaluable for this outcome measure and number analyzed = participants with available data for each specified category. Baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry.

Arm	Measure	Group	Value (MEAN)	Dispersion
Eliglustat	Change From Current Study Baseline in Gaucher Disease Type 1 (GD1) Biomarker Levels: Chitotriosidase at Week 26, 52, 78, 104, 130, 156, 182, 208 and 234	Study Baseline	4039.3333 nmol/hr/mL	Standard Deviation 4441.8462
Eliglustat	Change From Current Study Baseline in Gaucher Disease Type 1 (GD1) Biomarker Levels: Chitotriosidase at Week 26, 52, 78, 104, 130, 156, 182, 208 and 234	Change at Week 26	-411.2333 nmol/hr/mL	Standard Deviation 2486.4359
Eliglustat	Change From Current Study Baseline in Gaucher Disease Type 1 (GD1) Biomarker Levels: Chitotriosidase at Week 26, 52, 78, 104, 130, 156, 182, 208 and 234	Change at Week 52	-308.6897 nmol/hr/mL	Standard Deviation 2843.4606
Eliglustat	Change From Current Study Baseline in Gaucher Disease Type 1 (GD1) Biomarker Levels: Chitotriosidase at Week 26, 52, 78, 104, 130, 156, 182, 208 and 234	Change at Week 78	-492.4286 nmol/hr/mL	Standard Deviation 3850.9447
Eliglustat	Change From Current Study Baseline in Gaucher Disease Type 1 (GD1) Biomarker Levels: Chitotriosidase at Week 26, 52, 78, 104, 130, 156, 182, 208 and 234	Change at Week 104	-709.2143 nmol/hr/mL	Standard Deviation 3274.5604
Eliglustat	Change From Current Study Baseline in Gaucher Disease Type 1 (GD1) Biomarker Levels: Chitotriosidase at Week 26, 52, 78, 104, 130, 156, 182, 208 and 234	Change at Week 130	-712.4643 nmol/hr/mL	Standard Deviation 3366.5521
Eliglustat	Change From Current Study Baseline in Gaucher Disease Type 1 (GD1) Biomarker Levels: Chitotriosidase at Week 26, 52, 78, 104, 130, 156, 182, 208 and 234	Change at Week 156	-367.9286 nmol/hr/mL	Standard Deviation 3615.7654
Eliglustat	Change From Current Study Baseline in Gaucher Disease Type 1 (GD1) Biomarker Levels: Chitotriosidase at Week 26, 52, 78, 104, 130, 156, 182, 208 and 234	Change at Week 182	-582.1429 nmol/hr/mL	Standard Deviation 3386.1815
Eliglustat	Change From Current Study Baseline in Gaucher Disease Type 1 (GD1) Biomarker Levels: Chitotriosidase at Week 26, 52, 78, 104, 130, 156, 182, 208 and 234	Change at Week 208	-334.1538 nmol/hr/mL	Standard Deviation 3523.866
Eliglustat	Change From Current Study Baseline in Gaucher Disease Type 1 (GD1) Biomarker Levels: Chitotriosidase at Week 26, 52, 78, 104, 130, 156, 182, 208 and 234	Change at Week 234	411.7692 nmol/hr/mL	Standard Deviation 1880.7128

Secondary

Change From Current Study Baseline in GD1 Biomarker Levels: Glucosylceramide (GL-1) at Week 26, 52, 78, 104, 130, 156, 182, 208 and 234

Time frame: Study Baseline, Week 26, 52, 78, 104, 130, 156, 182, 208 and 234

Arm	Measure	Group	Value (MEAN)	Dispersion
Eliglustat	Change From Current Study Baseline in GD1 Biomarker Levels: Glucosylceramide (GL-1) at Week 26, 52, 78, 104, 130, 156, 182, 208 and 234	Study Baseline	5.3243 micrograms per milliliter (mcg/mL)	Standard Deviation 3.583
Eliglustat	Change From Current Study Baseline in GD1 Biomarker Levels: Glucosylceramide (GL-1) at Week 26, 52, 78, 104, 130, 156, 182, 208 and 234	Change at Week 26	-1.7770 micrograms per milliliter (mcg/mL)	Standard Deviation 3.8871
Eliglustat	Change From Current Study Baseline in GD1 Biomarker Levels: Glucosylceramide (GL-1) at Week 26, 52, 78, 104, 130, 156, 182, 208 and 234	Change at Week 52	-1.7002 micrograms per milliliter (mcg/mL)	Standard Deviation 4.6162
Eliglustat	Change From Current Study Baseline in GD1 Biomarker Levels: Glucosylceramide (GL-1) at Week 26, 52, 78, 104, 130, 156, 182, 208 and 234	Change at Week 78	-1.4000 micrograms per milliliter (mcg/mL)	Standard Deviation 4.1376
Eliglustat	Change From Current Study Baseline in GD1 Biomarker Levels: Glucosylceramide (GL-1) at Week 26, 52, 78, 104, 130, 156, 182, 208 and 234	Change at Week 104	-2.0657 micrograms per milliliter (mcg/mL)	Standard Deviation 3.5678
Eliglustat	Change From Current Study Baseline in GD1 Biomarker Levels: Glucosylceramide (GL-1) at Week 26, 52, 78, 104, 130, 156, 182, 208 and 234	Change at Week 130	-1.7405 micrograms per milliliter (mcg/mL)	Standard Deviation 3.5448
Eliglustat	Change From Current Study Baseline in GD1 Biomarker Levels: Glucosylceramide (GL-1) at Week 26, 52, 78, 104, 130, 156, 182, 208 and 234	Change at Week 156	-1.6073 micrograms per milliliter (mcg/mL)	Standard Deviation 3.3841
Eliglustat	Change From Current Study Baseline in GD1 Biomarker Levels: Glucosylceramide (GL-1) at Week 26, 52, 78, 104, 130, 156, 182, 208 and 234	Change at Week 182	-1.7304 micrograms per milliliter (mcg/mL)	Standard Deviation 3.814
Eliglustat	Change From Current Study Baseline in GD1 Biomarker Levels: Glucosylceramide (GL-1) at Week 26, 52, 78, 104, 130, 156, 182, 208 and 234	Change at Week 208	-2.0408 micrograms per milliliter (mcg/mL)	Standard Deviation 3.7987
Eliglustat	Change From Current Study Baseline in GD1 Biomarker Levels: Glucosylceramide (GL-1) at Week 26, 52, 78, 104, 130, 156, 182, 208 and 234	Change at Week 234	0.7691 micrograms per milliliter (mcg/mL)	Standard Deviation 2.257

Secondary

Change From Current Study Baseline in GD1 Biomarker Levels: Lyso Glucosylceramide (Lyso-GL-1) at Week 26, 52, 78, 104, 130, 156, 182, 208 and 234

Time frame: Study Baseline, Week 26, 52, 78, 104, 130, 156, 182, 208 and 234

Arm	Measure	Group	Value (MEAN)	Dispersion
Eliglustat	Change From Current Study Baseline in GD1 Biomarker Levels: Lyso Glucosylceramide (Lyso-GL-1) at Week 26, 52, 78, 104, 130, 156, 182, 208 and 234	Study Baseline	114.2429 nanograms per milliliter (ng/mL)	Standard Deviation 89.5197
Eliglustat	Change From Current Study Baseline in GD1 Biomarker Levels: Lyso Glucosylceramide (Lyso-GL-1) at Week 26, 52, 78, 104, 130, 156, 182, 208 and 234	Change at Week 26	-43.8238 nanograms per milliliter (ng/mL)	Standard Deviation 63.2152
Eliglustat	Change From Current Study Baseline in GD1 Biomarker Levels: Lyso Glucosylceramide (Lyso-GL-1) at Week 26, 52, 78, 104, 130, 156, 182, 208 and 234	Change at Week 52	-40.7500 nanograms per milliliter (ng/mL)	Standard Deviation 61.0894
Eliglustat	Change From Current Study Baseline in GD1 Biomarker Levels: Lyso Glucosylceramide (Lyso-GL-1) at Week 26, 52, 78, 104, 130, 156, 182, 208 and 234	Change at Week 78	-30.6711 nanograms per milliliter (ng/mL)	Standard Deviation 71.1072
Eliglustat	Change From Current Study Baseline in GD1 Biomarker Levels: Lyso Glucosylceramide (Lyso-GL-1) at Week 26, 52, 78, 104, 130, 156, 182, 208 and 234	Change at Week 104	-35.5721 nanograms per milliliter (ng/mL)	Standard Deviation 47.2414
Eliglustat	Change From Current Study Baseline in GD1 Biomarker Levels: Lyso Glucosylceramide (Lyso-GL-1) at Week 26, 52, 78, 104, 130, 156, 182, 208 and 234	Change at Week 130	-33.6195 nanograms per milliliter (ng/mL)	Standard Deviation 74.1656
Eliglustat	Change From Current Study Baseline in GD1 Biomarker Levels: Lyso Glucosylceramide (Lyso-GL-1) at Week 26, 52, 78, 104, 130, 156, 182, 208 and 234	Change at Week 156	-27.6795 nanograms per milliliter (ng/mL)	Standard Deviation 72.4469
Eliglustat	Change From Current Study Baseline in GD1 Biomarker Levels: Lyso Glucosylceramide (Lyso-GL-1) at Week 26, 52, 78, 104, 130, 156, 182, 208 and 234	Change at Week 182	-32.3611 nanograms per milliliter (ng/mL)	Standard Deviation 71.9446
Eliglustat	Change From Current Study Baseline in GD1 Biomarker Levels: Lyso Glucosylceramide (Lyso-GL-1) at Week 26, 52, 78, 104, 130, 156, 182, 208 and 234	Change at Week 208	-31.2882 nanograms per milliliter (ng/mL)	Standard Deviation 64.0608
Eliglustat	Change From Current Study Baseline in GD1 Biomarker Levels: Lyso Glucosylceramide (Lyso-GL-1) at Week 26, 52, 78, 104, 130, 156, 182, 208 and 234	Change at Week 234	-45.5100 nanograms per milliliter (ng/mL)	Standard Deviation 31.0448

Secondary

Change From Current Study Baseline in Short Form-36 Health Survey (SF-36) Scores at Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234

The 36-Item Short-Form Health Survey (SF-36) is standardized survey evaluating 8 aspects of functional health and well-being. Physical Component Summary (PCS) with 4 sub-scales: physical function, role limitations due to physical problems, bodily pain, and general health perception; and Mental Component Summary (MCS) with 4 sub-scales: vitality, social function, role limitations due to emotional problems, and mental health. Summations of item scores of the same sub-scale give the sub-scale scores, which are transformed into a range from 0 to 100; 0= worst and 100=best outcome. Both PCS and MCS range from 0 to 100 with higher scores indicating better physical and mental health. For this outcome measure, baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry.

Time frame: Study Baseline, Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234

Arm	Measure	Group	Value (MEAN)	Dispersion
Eliglustat	Change From Current Study Baseline in Short Form-36 Health Survey (SF-36) Scores at Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234	PCS: Week 26	53.3052 score on a scale	Standard Deviation 6.1692
Eliglustat	Change From Current Study Baseline in Short Form-36 Health Survey (SF-36) Scores at Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234	PCS: Change at Week 26	0.4258 score on a scale	Standard Deviation 3.3324
Eliglustat	Change From Current Study Baseline in Short Form-36 Health Survey (SF-36) Scores at Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234	PCS: Week 52	52.8240 score on a scale	Standard Deviation 6.0361
Eliglustat	Change From Current Study Baseline in Short Form-36 Health Survey (SF-36) Scores at Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234	PCS: Week 78	53.0769 score on a scale	Standard Deviation 5.737
Eliglustat	Change From Current Study Baseline in Short Form-36 Health Survey (SF-36) Scores at Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234	PCS: Week 104	53.1225 score on a scale	Standard Deviation 5.5751
Eliglustat	Change From Current Study Baseline in Short Form-36 Health Survey (SF-36) Scores at Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234	PCS: Change at Week 104	-0.4975 score on a scale	Standard Deviation 3.0048
Eliglustat	Change From Current Study Baseline in Short Form-36 Health Survey (SF-36) Scores at Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234	PCS: Change at Week 130	-1.3203 score on a scale	Standard Deviation 4.4392
Eliglustat	Change From Current Study Baseline in Short Form-36 Health Survey (SF-36) Scores at Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234	PCS: Week 156	51.6359 score on a scale	Standard Deviation 6.5758
Eliglustat	Change From Current Study Baseline in Short Form-36 Health Survey (SF-36) Scores at Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234	PCS: Change at Week 156	-1.9841 score on a scale	Standard Deviation 3.5507
Eliglustat	Change From Current Study Baseline in Short Form-36 Health Survey (SF-36) Scores at Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234	PCS: Week 182	52.4094 score on a scale	Standard Deviation 6.4415
Eliglustat	Change From Current Study Baseline in Short Form-36 Health Survey (SF-36) Scores at Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234	PCS: Change at Week 182	-1.2105 score on a scale	Standard Deviation 4.0492
Eliglustat	Change From Current Study Baseline in Short Form-36 Health Survey (SF-36) Scores at Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234	PCS: Week 208	52.2733 score on a scale	Standard Deviation 7.2695
Eliglustat	Change From Current Study Baseline in Short Form-36 Health Survey (SF-36) Scores at Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234	PCS: Change at Week 208	-1.3466 score on a scale	Standard Deviation 4.6688
Eliglustat	Change From Current Study Baseline in Short Form-36 Health Survey (SF-36) Scores at Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234	PCS: Week 234	52.5847 score on a scale	Standard Deviation 5.5468
Eliglustat	Change From Current Study Baseline in Short Form-36 Health Survey (SF-36) Scores at Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234	PCS: Change at Week 234	-1.0284 score on a scale	Standard Deviation 4.5981
Eliglustat	Change From Current Study Baseline in Short Form-36 Health Survey (SF-36) Scores at Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234	MCS: Change at Week 26	2.1274 score on a scale	Standard Deviation 5.8293
Eliglustat	Change From Current Study Baseline in Short Form-36 Health Survey (SF-36) Scores at Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234	MCS: Week 52	50.1502 score on a scale	Standard Deviation 11.2198
Eliglustat	Change From Current Study Baseline in Short Form-36 Health Survey (SF-36) Scores at Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234	MCS: Change at Week 52	0.5279 score on a scale	Standard Deviation 8.9388
Eliglustat	Change From Current Study Baseline in Short Form-36 Health Survey (SF-36) Scores at Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234	MCS: Week 78	51.0151 score on a scale	Standard Deviation 10.1117
Eliglustat	Change From Current Study Baseline in Short Form-36 Health Survey (SF-36) Scores at Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234	MCS: Change at Week 78	-0.2412 score on a scale	Standard Deviation 5.9877
Eliglustat	Change From Current Study Baseline in Short Form-36 Health Survey (SF-36) Scores at Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234	MCS: Week 130	51.6144 score on a scale	Standard Deviation 10.6866
Eliglustat	Change From Current Study Baseline in Short Form-36 Health Survey (SF-36) Scores at Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234	MCS: Change at Week 130	0.3582 score on a scale	Standard Deviation 5.717
Eliglustat	Change From Current Study Baseline in Short Form-36 Health Survey (SF-36) Scores at Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234	MCS: Week 182	52.1595 score on a scale	Standard Deviation 9.6141
Eliglustat	Change From Current Study Baseline in Short Form-36 Health Survey (SF-36) Scores at Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234	MCS: Week 208	49.2308 score on a scale	Standard Deviation 11.8064
Eliglustat	Change From Current Study Baseline in Short Form-36 Health Survey (SF-36) Scores at Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234	MCS: Change at Week 208	-2.0254 score on a scale	Standard Deviation 7.6197
Eliglustat	Change From Current Study Baseline in Short Form-36 Health Survey (SF-36) Scores at Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234	MCS: Week 234	47.7470 score on a scale	Standard Deviation 12.5183
Eliglustat	Change From Current Study Baseline in Short Form-36 Health Survey (SF-36) Scores at Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234	MCS: Change at Week 234	-3.9989 score on a scale	Standard Deviation 8.9392
Eliglustat	Change From Current Study Baseline in Short Form-36 Health Survey (SF-36) Scores at Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234	MCS: Change at Week 182	0.9033 score on a scale	Standard Deviation 6.0446
Eliglustat	Change From Current Study Baseline in Short Form-36 Health Survey (SF-36) Scores at Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234	Study Baseline: PCS	52.8795 score on a scale	Standard Deviation 6.5046
Eliglustat	Change From Current Study Baseline in Short Form-36 Health Survey (SF-36) Scores at Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234	PCS: Change at Week 52	-0.0554 score on a scale	Standard Deviation 3.8484
Eliglustat	Change From Current Study Baseline in Short Form-36 Health Survey (SF-36) Scores at Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234	PCS: Change at Week 78	-0.5431 score on a scale	Standard Deviation 3.9927
Eliglustat	Change From Current Study Baseline in Short Form-36 Health Survey (SF-36) Scores at Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234	PCS: Week 130	52.2996 score on a scale	Standard Deviation 6.6942
Eliglustat	Change From Current Study Baseline in Short Form-36 Health Survey (SF-36) Scores at Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234	Study Baseline: MCS	49.6223 score on a scale	Standard Deviation 11.236
Eliglustat	Change From Current Study Baseline in Short Form-36 Health Survey (SF-36) Scores at Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234	MCS: Week 26	51.7497 score on a scale	Standard Deviation 9.4775
Eliglustat	Change From Current Study Baseline in Short Form-36 Health Survey (SF-36) Scores at Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234	MCS: Week 104	50.0147 score on a scale	Standard Deviation 12.7162
Eliglustat	Change From Current Study Baseline in Short Form-36 Health Survey (SF-36) Scores at Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234	MCS: Change at Week 104	-1.2415 score on a scale	Standard Deviation 7.1779
Eliglustat	Change From Current Study Baseline in Short Form-36 Health Survey (SF-36) Scores at Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234	MCS: Week 156	51.5030 score on a scale	Standard Deviation 10.6347
Eliglustat	Change From Current Study Baseline in Short Form-36 Health Survey (SF-36) Scores at Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234	MCS: Change at Week 156	0.2468 score on a scale	Standard Deviation 7.086

Secondary

Change From Eliglustat Baseline in Gaucher Disease Type 1 (GD1) Biomarker Levels: Chitotriosidase at Week 26, 52, 78, 104, 130, 156, 182, 208 and 234

Chitotriosidase biomarker was assayed from plasma. Chitotriosidase biomarker levels for participants who were CYP2D6 non-URM was reported in this outcome measure. For this outcome measure, baseline refers to the eliglustat baseline, which was defined as participant's status at the time of first dose of eliglustat in the previous Phase 2 or Phase 3 study. Data for this outcome measure was planned to be collected and analyzed in reference to eliglustat baseline separately for Phase 2, ENGAGE and EDGE studies (GD treatment naïve participants) and from ENCORE study (participants switched from ERT).

Time frame: Eliglustat Baseline, Week 26, 52, 78, 104, 130, 156, 182, 208 and 234 of the current study

Population: Analysis was performed on FAS population. Here, overall number of participants analyzed = participants evaluable for this outcome measure and number analyzed = participants with available data for each specified category and 0 in number analyzed field signifies that none of the participants had evaluable data at specified timepoint.

Arm	Measure	Group	Value (MEAN)	Dispersion
Eliglustat	Change From Eliglustat Baseline in Gaucher Disease Type 1 (GD1) Biomarker Levels: Chitotriosidase at Week 26, 52, 78, 104, 130, 156, 182, 208 and 234	Eliglustat Baseline	9507.9630 nmol/hr/mL	Standard Deviation 7618.0282
Eliglustat	Change From Eliglustat Baseline in Gaucher Disease Type 1 (GD1) Biomarker Levels: Chitotriosidase at Week 26, 52, 78, 104, 130, 156, 182, 208 and 234	Change at Week 26	-6054.5185 nmol/hr/mL	Standard Deviation 6817.9692
Eliglustat	Change From Eliglustat Baseline in Gaucher Disease Type 1 (GD1) Biomarker Levels: Chitotriosidase at Week 26, 52, 78, 104, 130, 156, 182, 208 and 234	Change at Week 52	-6207.9615 nmol/hr/mL	Standard Deviation 7178.4162
Eliglustat	Change From Eliglustat Baseline in Gaucher Disease Type 1 (GD1) Biomarker Levels: Chitotriosidase at Week 26, 52, 78, 104, 130, 156, 182, 208 and 234	Change at Week 78	-6475.0800 nmol/hr/mL	Standard Deviation 8035.2136
Eliglustat	Change From Eliglustat Baseline in Gaucher Disease Type 1 (GD1) Biomarker Levels: Chitotriosidase at Week 26, 52, 78, 104, 130, 156, 182, 208 and 234	Change at Week 104	-6704.6000 nmol/hr/mL	Standard Deviation 7658.9433
Eliglustat	Change From Eliglustat Baseline in Gaucher Disease Type 1 (GD1) Biomarker Levels: Chitotriosidase at Week 26, 52, 78, 104, 130, 156, 182, 208 and 234	Change at Week 130	-6663.5200 nmol/hr/mL	Standard Deviation 7578.2222
Eliglustat	Change From Eliglustat Baseline in Gaucher Disease Type 1 (GD1) Biomarker Levels: Chitotriosidase at Week 26, 52, 78, 104, 130, 156, 182, 208 and 234	Change at Week 156	-6335.6000 nmol/hr/mL	Standard Deviation 7785.3273
Eliglustat	Change From Eliglustat Baseline in Gaucher Disease Type 1 (GD1) Biomarker Levels: Chitotriosidase at Week 26, 52, 78, 104, 130, 156, 182, 208 and 234	Change at Week 182	-6543.0000 nmol/hr/mL	Standard Deviation 7349.2169
Eliglustat	Change From Eliglustat Baseline in Gaucher Disease Type 1 (GD1) Biomarker Levels: Chitotriosidase at Week 26, 52, 78, 104, 130, 156, 182, 208 and 234	Change at Week 208	-6380.3913 nmol/hr/mL	Standard Deviation 7729.7877
Eliglustat	Change From Eliglustat Baseline in Gaucher Disease Type 1 (GD1) Biomarker Levels: Chitotriosidase at Week 26, 52, 78, 104, 130, 156, 182, 208 and 234	Change at Week 234	-7212.0769 nmol/hr/mL	Standard Deviation 7757.2285
Eliglustat: Participants From ENCORE	Change From Eliglustat Baseline in Gaucher Disease Type 1 (GD1) Biomarker Levels: Chitotriosidase at Week 26, 52, 78, 104, 130, 156, 182, 208 and 234	Eliglustat Baseline	2844.5000 nmol/hr/mL	Standard Deviation 338.7041
Eliglustat: Participants From ENCORE	Change From Eliglustat Baseline in Gaucher Disease Type 1 (GD1) Biomarker Levels: Chitotriosidase at Week 26, 52, 78, 104, 130, 156, 182, 208 and 234	Change at Week 208	-1160.5000 nmol/hr/mL	Standard Deviation 2560.4337
Eliglustat: Participants From ENCORE	Change From Eliglustat Baseline in Gaucher Disease Type 1 (GD1) Biomarker Levels: Chitotriosidase at Week 26, 52, 78, 104, 130, 156, 182, 208 and 234	Change at Week 26	-122.5000 nmol/hr/mL	Standard Deviation 2112.128
Eliglustat: Participants From ENCORE	Change From Eliglustat Baseline in Gaucher Disease Type 1 (GD1) Biomarker Levels: Chitotriosidase at Week 26, 52, 78, 104, 130, 156, 182, 208 and 234	Change at Week 130	-981.5000 nmol/hr/mL	Standard Deviation 2724.4824
Eliglustat: Participants From ENCORE	Change From Eliglustat Baseline in Gaucher Disease Type 1 (GD1) Biomarker Levels: Chitotriosidase at Week 26, 52, 78, 104, 130, 156, 182, 208 and 234	Change at Week 52	-405.5000 nmol/hr/mL	Standard Deviation 2581.6469
Eliglustat: Participants From ENCORE	Change From Eliglustat Baseline in Gaucher Disease Type 1 (GD1) Biomarker Levels: Chitotriosidase at Week 26, 52, 78, 104, 130, 156, 182, 208 and 234	Change at Week 182	-933.0000 nmol/hr/mL	Standard Deviation 2851.0545
Eliglustat: Participants From ENCORE	Change From Eliglustat Baseline in Gaucher Disease Type 1 (GD1) Biomarker Levels: Chitotriosidase at Week 26, 52, 78, 104, 130, 156, 182, 208 and 234	Change at Week 78	-1233.0000 nmol/hr/mL	Standard Deviation 2054.8523
Eliglustat: Participants From ENCORE	Change From Eliglustat Baseline in Gaucher Disease Type 1 (GD1) Biomarker Levels: Chitotriosidase at Week 26, 52, 78, 104, 130, 156, 182, 208 and 234	Change at Week 156	-1181.0000 nmol/hr/mL	Standard Deviation 2494.6727
Eliglustat: Participants From ENCORE	Change From Eliglustat Baseline in Gaucher Disease Type 1 (GD1) Biomarker Levels: Chitotriosidase at Week 26, 52, 78, 104, 130, 156, 182, 208 and 234	Change at Week 104	-1249.0000 nmol/hr/mL	Standard Deviation 2186.3742

Secondary

Change From Eliglustat Baseline in GD1 Biomarker Levels: Glucosylceramide (GL-1) at Week 26, 52, 78, 104, 130, 156, 182, 208 and 234

Glucosylceramide (GL-1) biomarker was assayed from plasma. GL-1 biomarker levels for participants who were CYP2D6 non-URM was reported in this outcome measure. For this outcome measure, baseline refers to the eliglustat baseline, which was defined as participant's status at the time of first dose of eliglustat in the previous Phase 2 or Phase 3 study. Data for this outcome measure was planned to be collected and analyzed in reference to eliglustat baseline separately for participants from Phase 2, ENGAGE and EDGE studies (GD treatment naïve participants) and from ENCORE study (participants switched from ERT).

Time frame: Eliglustat Baseline, Week 26, 52, 78, 104, 130, 156, 182, 208 and 234 of the current study

Population: Analysis was performed on FAS population. Here, overall number of participants analyzed = participants evaluable for this outcome measure and number analyzed = participants with available data for each specified category and 0 signifies that none of the participants had evaluable data at the specified timepoint.

Arm	Measure	Group	Value (MEAN)	Dispersion
Eliglustat	Change From Eliglustat Baseline in GD1 Biomarker Levels: Glucosylceramide (GL-1) at Week 26, 52, 78, 104, 130, 156, 182, 208 and 234	Eliglustat Baseline	11.8304 mcg/mL	Standard Deviation 3.5804
Eliglustat	Change From Eliglustat Baseline in GD1 Biomarker Levels: Glucosylceramide (GL-1) at Week 26, 52, 78, 104, 130, 156, 182, 208 and 234	Change at Week 26	-8.2095 mcg/mL	Standard Deviation 3.6233
Eliglustat	Change From Eliglustat Baseline in GD1 Biomarker Levels: Glucosylceramide (GL-1) at Week 26, 52, 78, 104, 130, 156, 182, 208 and 234	Change at Week 52	-8.0970 mcg/mL	Standard Deviation 4.0568
Eliglustat	Change From Eliglustat Baseline in GD1 Biomarker Levels: Glucosylceramide (GL-1) at Week 26, 52, 78, 104, 130, 156, 182, 208 and 234	Change at Week 78	-7.7498 mcg/mL	Standard Deviation 3.7788
Eliglustat	Change From Eliglustat Baseline in GD1 Biomarker Levels: Glucosylceramide (GL-1) at Week 26, 52, 78, 104, 130, 156, 182, 208 and 234	Change at Week 104	-8.6111 mcg/mL	Standard Deviation 3.7016
Eliglustat	Change From Eliglustat Baseline in GD1 Biomarker Levels: Glucosylceramide (GL-1) at Week 26, 52, 78, 104, 130, 156, 182, 208 and 234	Change at Week 130	-8.5354 mcg/mL	Standard Deviation 3.87
Eliglustat	Change From Eliglustat Baseline in GD1 Biomarker Levels: Glucosylceramide (GL-1) at Week 26, 52, 78, 104, 130, 156, 182, 208 and 234	Change at Week 156	-8.1772 mcg/mL	Standard Deviation 3.4306
Eliglustat	Change From Eliglustat Baseline in GD1 Biomarker Levels: Glucosylceramide (GL-1) at Week 26, 52, 78, 104, 130, 156, 182, 208 and 234	Change at Week 182	-8.0321 mcg/mL	Standard Deviation 3.6982
Eliglustat	Change From Eliglustat Baseline in GD1 Biomarker Levels: Glucosylceramide (GL-1) at Week 26, 52, 78, 104, 130, 156, 182, 208 and 234	Change at Week 208	-8.5976 mcg/mL	Standard Deviation 3.4497
Eliglustat	Change From Eliglustat Baseline in GD1 Biomarker Levels: Glucosylceramide (GL-1) at Week 26, 52, 78, 104, 130, 156, 182, 208 and 234	Change at Week 234	-7.9463 mcg/mL	Standard Deviation 3.6203
Eliglustat: Participants From ENCORE	Change From Eliglustat Baseline in GD1 Biomarker Levels: Glucosylceramide (GL-1) at Week 26, 52, 78, 104, 130, 156, 182, 208 and 234	Eliglustat Baseline	5.9667 mcg/mL	Standard Deviation 0.9713
Eliglustat: Participants From ENCORE	Change From Eliglustat Baseline in GD1 Biomarker Levels: Glucosylceramide (GL-1) at Week 26, 52, 78, 104, 130, 156, 182, 208 and 234	Change at Week 208	-4.2390 mcg/mL	Standard Deviation 2.2343
Eliglustat: Participants From ENCORE	Change From Eliglustat Baseline in GD1 Biomarker Levels: Glucosylceramide (GL-1) at Week 26, 52, 78, 104, 130, 156, 182, 208 and 234	Change at Week 26	-2.2470 mcg/mL	Standard Deviation 3.7756
Eliglustat: Participants From ENCORE	Change From Eliglustat Baseline in GD1 Biomarker Levels: Glucosylceramide (GL-1) at Week 26, 52, 78, 104, 130, 156, 182, 208 and 234	Change at Week 130	-3.2767 mcg/mL	Standard Deviation 1.8706
Eliglustat: Participants From ENCORE	Change From Eliglustat Baseline in GD1 Biomarker Levels: Glucosylceramide (GL-1) at Week 26, 52, 78, 104, 130, 156, 182, 208 and 234	Change at Week 52	-2.9880 mcg/mL	Standard Deviation 3.3407
Eliglustat: Participants From ENCORE	Change From Eliglustat Baseline in GD1 Biomarker Levels: Glucosylceramide (GL-1) at Week 26, 52, 78, 104, 130, 156, 182, 208 and 234	Change at Week 182	-3.9720 mcg/mL	Standard Deviation 2.0463
Eliglustat: Participants From ENCORE	Change From Eliglustat Baseline in GD1 Biomarker Levels: Glucosylceramide (GL-1) at Week 26, 52, 78, 104, 130, 156, 182, 208 and 234	Change at Week 78	-3.1150 mcg/mL	Standard Deviation 3.5522
Eliglustat: Participants From ENCORE	Change From Eliglustat Baseline in GD1 Biomarker Levels: Glucosylceramide (GL-1) at Week 26, 52, 78, 104, 130, 156, 182, 208 and 234	Change at Week 156	-1.8643 mcg/mL	Standard Deviation 5.671
Eliglustat: Participants From ENCORE	Change From Eliglustat Baseline in GD1 Biomarker Levels: Glucosylceramide (GL-1) at Week 26, 52, 78, 104, 130, 156, 182, 208 and 234	Change at Week 104	-3.6920 mcg/mL	Standard Deviation 2.8225

Secondary

Change From Eliglustat Baseline in GD1 Biomarker Levels: Lyso Glucosylceramide (Lyso-GL-1) at Week 26, 52, 78, 104, 130, 156, 182, 208 and 234

Lyso-GL-1 biomarker was assayed from plasma. Lyso-GL-1 biomarker levels for participants who were CYP2D6 non-URM was reported in this outcome measure. For this outcome measure, baseline refers to the eliglustat baseline, which was defined as participant's status at the time of first dose of eliglustat in the previous Phase 2 or Phase 3 study. Data for this outcome measure was planned to be collected and analyzed in reference to eliglustat baseline separately for participants from Phase 2, ENGAGE and EDGE studies (GD treatment naïve participants) and from ENCORE study (participants switched from ERT).

Time frame: Eliglustat Baseline, Week 26, 52, 78, 104, 130, 156, 182, 208 and 234 of the current study

Population: Analysis was performed on FAS population. Here, overall number of participants analyzed = participants evaluable for this outcome measure and number analyzed = participants with available data for each specified category. Here, 0 in overall number analyzed field signifies that biomarker lyso-GL-1 was not assessed in Phase 3 studies, hence the biomarker levels at specified timepoint were not reported for any of the participants who were from previous Phase 3 studies.

Arm	Measure	Group	Value (MEAN)	Dispersion
Eliglustat	Change From Eliglustat Baseline in GD1 Biomarker Levels: Lyso Glucosylceramide (Lyso-GL-1) at Week 26, 52, 78, 104, 130, 156, 182, 208 and 234	Change at Week 52	-537.1222 ng/mL	Standard Deviation 250.8135
Eliglustat	Change From Eliglustat Baseline in GD1 Biomarker Levels: Lyso Glucosylceramide (Lyso-GL-1) at Week 26, 52, 78, 104, 130, 156, 182, 208 and 234	Eliglustat Baseline	612.2222 ng/mL	Standard Deviation 256.6835
Eliglustat	Change From Eliglustat Baseline in GD1 Biomarker Levels: Lyso Glucosylceramide (Lyso-GL-1) at Week 26, 52, 78, 104, 130, 156, 182, 208 and 234	Change at Week 26	-548.3111 ng/mL	Standard Deviation 236.1365
Eliglustat	Change From Eliglustat Baseline in GD1 Biomarker Levels: Lyso Glucosylceramide (Lyso-GL-1) at Week 26, 52, 78, 104, 130, 156, 182, 208 and 234	Change at Week 78	-522.6833 ng/mL	Standard Deviation 248.3267
Eliglustat	Change From Eliglustat Baseline in GD1 Biomarker Levels: Lyso Glucosylceramide (Lyso-GL-1) at Week 26, 52, 78, 104, 130, 156, 182, 208 and 234	Change at Week 104	-516.3022 ng/mL	Standard Deviation 236.9183
Eliglustat	Change From Eliglustat Baseline in GD1 Biomarker Levels: Lyso Glucosylceramide (Lyso-GL-1) at Week 26, 52, 78, 104, 130, 156, 182, 208 and 234	Change at Week 130	-516.9889 ng/mL	Standard Deviation 238.9292
Eliglustat	Change From Eliglustat Baseline in GD1 Biomarker Levels: Lyso Glucosylceramide (Lyso-GL-1) at Week 26, 52, 78, 104, 130, 156, 182, 208 and 234	Change at Week 156	-511.6000 ng/mL	Standard Deviation 241.2484
Eliglustat	Change From Eliglustat Baseline in GD1 Biomarker Levels: Lyso Glucosylceramide (Lyso-GL-1) at Week 26, 52, 78, 104, 130, 156, 182, 208 and 234	Change at Week 182	-516.5111 ng/mL	Standard Deviation 244.4099
Eliglustat	Change From Eliglustat Baseline in GD1 Biomarker Levels: Lyso Glucosylceramide (Lyso-GL-1) at Week 26, 52, 78, 104, 130, 156, 182, 208 and 234	Change at Week 208	-495.3500 ng/mL	Standard Deviation 261.1088
Eliglustat	Change From Eliglustat Baseline in GD1 Biomarker Levels: Lyso Glucosylceramide (Lyso-GL-1) at Week 26, 52, 78, 104, 130, 156, 182, 208 and 234	Change at Week 234	-590.8100 ng/mL	Standard Deviation 220.5445

Secondary

Change From Eliglustat Baseline in SF-36 Scores at Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234

SF-36 is a standardized survey evaluating 8 aspects of functional health and well-being. PCS with 4 sub-scales: physical function, role limitations due to physical problems, pain, and general health perception; and MCS with 4 sub-scales: vitality, social function, role limitations due to emotional problems, and mental health. Summations of item scores of same sub-scale give the sub-scale scores, which are transformed into range from 0 to 100; 0= worst, and 100=best outcome. Both PCS and MCS range from 0 to 100, higher scores indicating better physical and mental health. For this outcome measure, baseline refers to the eliglustat baseline, which was defined as participant's status at the time of first dose of eliglustat in the previous Phase 2 or Phase 3 study.

Time frame: Eliglustat Baseline, Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234 of the current study

Population: Analysis was performed on full analysis population. Here, number analyzed = participants with available data for each specified category 0 signifies that none of the participants had evaluable data at the specified timepoint. Data for this outcome measure was planned to be collected and analyzed in reference to eliglustat baseline separately for participants from Phase 2, ENGAGE and EDGE studies (GD treatment naïve participants) and from ENCORE study (participants switched from ERT).

Arm	Measure	Group	Value (MEAN)	Dispersion
Eliglustat	Change From Eliglustat Baseline in SF-36 Scores at Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234	PCS: Week 234	52.5847 score on a scale	Standard Deviation 5.5468
Eliglustat	Change From Eliglustat Baseline in SF-36 Scores at Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234	PCS: Change at Week 104	9.0939 score on a scale	Standard Deviation 8.6935
Eliglustat	Change From Eliglustat Baseline in SF-36 Scores at Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234	PCS: Week 130	52.2561 score on a scale	Standard Deviation 6.0816
Eliglustat	Change From Eliglustat Baseline in SF-36 Scores at Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234	PCS: Change at Week 130	8.9364 score on a scale	Standard Deviation 9.5735
Eliglustat	Change From Eliglustat Baseline in SF-36 Scores at Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234	PCS: Change at Week 156	8.1580 score on a scale	Standard Deviation 9.4576
Eliglustat	Change From Eliglustat Baseline in SF-36 Scores at Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234	PCS: Change at Week 182	9.5587 score on a scale	Standard Deviation 9.0071
Eliglustat	Change From Eliglustat Baseline in SF-36 Scores at Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234	PCS: Change at Week 208	9.2886 score on a scale	Standard Deviation 9.1756
Eliglustat	Change From Eliglustat Baseline in SF-36 Scores at Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234	PCS: Change at Week 26	9.4479 score on a scale	Standard Deviation 9.3442
Eliglustat	Change From Eliglustat Baseline in SF-36 Scores at Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234	PCS: Change at Week 234	8.7252 score on a scale	Standard Deviation 7.217
Eliglustat	Change From Eliglustat Baseline in SF-36 Scores at Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234	Study Baseline: PCS	43.0578 score on a scale	Standard Deviation 6.0372
Eliglustat	Change From Eliglustat Baseline in SF-36 Scores at Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234	PCS: Week 26	53.0454 score on a scale	Standard Deviation 6.042
Eliglustat	Change From Eliglustat Baseline in SF-36 Scores at Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234	PCS: Week 52	52.8961 score on a scale	Standard Deviation 5.8443
Eliglustat	Change From Eliglustat Baseline in SF-36 Scores at Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234	PCS: Change at Week 52	9.0581 score on a scale	Standard Deviation 8.8189
Eliglustat	Change From Eliglustat Baseline in SF-36 Scores at Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234	PCS: Week 156	51.6309 score on a scale	Standard Deviation 6.1757
Eliglustat	Change From Eliglustat Baseline in SF-36 Scores at Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234	PCS: Week 182	52.5222 score on a scale	Standard Deviation 5.6686
Eliglustat	Change From Eliglustat Baseline in SF-36 Scores at Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234	PCS: Week 208	52.4226 score on a scale	Standard Deviation 5.9064
Eliglustat	Change From Eliglustat Baseline in SF-36 Scores at Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234	Study Baseline: MCS	41.8312 score on a scale	Standard Deviation 8.5951
Eliglustat	Change From Eliglustat Baseline in SF-36 Scores at Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234	MCS: Week 26	52.7415 score on a scale	Standard Deviation 7.3261
Eliglustat	Change From Eliglustat Baseline in SF-36 Scores at Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234	MCS: Change at Week 26	9.9441 score on a scale	Standard Deviation 8.2305
Eliglustat	Change From Eliglustat Baseline in SF-36 Scores at Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234	MCS: Week 52	50.5915 score on a scale	Standard Deviation 10.92
Eliglustat	Change From Eliglustat Baseline in SF-36 Scores at Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234	MCS: Change at Week 52	8.0060 score on a scale	Standard Deviation 8.7063
Eliglustat	Change From Eliglustat Baseline in SF-36 Scores at Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234	MCS: Week 78	51.1656 score on a scale	Standard Deviation 10.1361
Eliglustat	Change From Eliglustat Baseline in SF-36 Scores at Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234	MCS: Change at Week 78	6.2543 score on a scale	Standard Deviation 7.5038
Eliglustat	Change From Eliglustat Baseline in SF-36 Scores at Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234	MCS: Week 104	51.4510 score on a scale	Standard Deviation 10.2052
Eliglustat	Change From Eliglustat Baseline in SF-36 Scores at Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234	MCS: Change at Week 104	6.9764 score on a scale	Standard Deviation 6.7131
Eliglustat	Change From Eliglustat Baseline in SF-36 Scores at Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234	MCS: Week 130	52.5954 score on a scale	Standard Deviation 9.4948
Eliglustat	Change From Eliglustat Baseline in SF-36 Scores at Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234	MCS: Change at Week 130	7.7807 score on a scale	Standard Deviation 6.0885
Eliglustat	Change From Eliglustat Baseline in SF-36 Scores at Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234	MCS: Week 156	52.1408 score on a scale	Standard Deviation 9.3668
Eliglustat	Change From Eliglustat Baseline in SF-36 Scores at Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234	MCS: Change at Week 156	8.0714 score on a scale	Standard Deviation 5.6904
Eliglustat	Change From Eliglustat Baseline in SF-36 Scores at Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234	MCS: Week 182	52.8180 score on a scale	Standard Deviation 8.8447
Eliglustat	Change From Eliglustat Baseline in SF-36 Scores at Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234	MCS: Change at Week 182	8.5582 score on a scale	Standard Deviation 7.2613
Eliglustat	Change From Eliglustat Baseline in SF-36 Scores at Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234	MCS: Week 208	50.0376 score on a scale	Standard Deviation 10.3762
Eliglustat	Change From Eliglustat Baseline in SF-36 Scores at Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234	MCS: Change at Week 208	5.9449 score on a scale	Standard Deviation 7.3056
Eliglustat	Change From Eliglustat Baseline in SF-36 Scores at Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234	MCS: Week 234	47.7470 score on a scale	Standard Deviation 12.5183
Eliglustat	Change From Eliglustat Baseline in SF-36 Scores at Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234	MCS: Change at Week 234	4.0884 score on a scale	Standard Deviation 7.5215
Eliglustat	Change From Eliglustat Baseline in SF-36 Scores at Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234	PCS: Week 78	53.1474 score on a scale	Standard Deviation 5.2925
Eliglustat	Change From Eliglustat Baseline in SF-36 Scores at Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234	PCS: Change at Week 78	9.6365 score on a scale	Standard Deviation 9.2094
Eliglustat	Change From Eliglustat Baseline in SF-36 Scores at Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234	PCS: Week 104	52.8224 score on a scale	Standard Deviation 5.5216
Eliglustat: Participants From ENCORE	Change From Eliglustat Baseline in SF-36 Scores at Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234	PCS: Change at Week 26	12.2772 score on a scale	Standard Deviation 8.2025
Eliglustat: Participants From ENCORE	Change From Eliglustat Baseline in SF-36 Scores at Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234	PCS: Change at Week 52	9.5562 score on a scale	Standard Deviation 8.831
Eliglustat: Participants From ENCORE	Change From Eliglustat Baseline in SF-36 Scores at Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234	MCS: Week 104	41.0384 score on a scale	Standard Deviation 23.4822
Eliglustat: Participants From ENCORE	Change From Eliglustat Baseline in SF-36 Scores at Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234	PCS: Week 78	52.6363 score on a scale	Standard Deviation 9.1001
Eliglustat: Participants From ENCORE	Change From Eliglustat Baseline in SF-36 Scores at Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234	PCS: Week 208	51.3400 score on a scale	Standard Deviation 14.5875
Eliglustat: Participants From ENCORE	Change From Eliglustat Baseline in SF-36 Scores at Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234	PCS: Change at Week 78	9.8546 score on a scale	Standard Deviation 9.698
Eliglustat: Participants From ENCORE	Change From Eliglustat Baseline in SF-36 Scores at Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234	MCS: Change at Week 208	1.6924 score on a scale	Standard Deviation 14.9871
Eliglustat: Participants From ENCORE	Change From Eliglustat Baseline in SF-36 Scores at Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234	MCS: Change at Week 104	-1.4576 score on a scale	Standard Deviation 18.5758
Eliglustat: Participants From ENCORE	Change From Eliglustat Baseline in SF-36 Scores at Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234	MCS: Week 26	45.0552 score on a scale	Standard Deviation 19.0913
Eliglustat: Participants From ENCORE	Change From Eliglustat Baseline in SF-36 Scores at Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234	PCS: Week 130	52.5714 score on a scale	Standard Deviation 11.0568
Eliglustat: Participants From ENCORE	Change From Eliglustat Baseline in SF-36 Scores at Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234	MCS: Change at Week 156	5.0205 score on a scale	Standard Deviation 13.3734
Eliglustat: Participants From ENCORE	Change From Eliglustat Baseline in SF-36 Scores at Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234	PCS: Week 156	51.6671 score on a scale	Standard Deviation 9.9228
Eliglustat: Participants From ENCORE	Change From Eliglustat Baseline in SF-36 Scores at Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234	MCS: Change at Week 26	2.5592 score on a scale	Standard Deviation 14.2291
Eliglustat: Participants From ENCORE	Change From Eliglustat Baseline in SF-36 Scores at Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234	MCS: Week 130	45.4826 score on a scale	Standard Deviation 16.9277
Eliglustat: Participants From ENCORE	Change From Eliglustat Baseline in SF-36 Scores at Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234	PCS: Change at Week 182	8.9229 score on a scale	Standard Deviation 12.108
Eliglustat: Participants From ENCORE	Change From Eliglustat Baseline in SF-36 Scores at Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234	MCS: Week 52	47.1715 score on a scale	Standard Deviation 14.5528
Eliglustat: Participants From ENCORE	Change From Eliglustat Baseline in SF-36 Scores at Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234	PCS: Change at Week 208	8.5582 score on a scale	Standard Deviation 15.2331
Eliglustat: Participants From ENCORE	Change From Eliglustat Baseline in SF-36 Scores at Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234	MCS: Change at Week 182	5.5479 score on a scale	Standard Deviation 9.6766
Eliglustat: Participants From ENCORE	Change From Eliglustat Baseline in SF-36 Scores at Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234	MCS: Change at Week 52	4.6756 score on a scale	Standard Deviation 9.2744
Eliglustat: Participants From ENCORE	Change From Eliglustat Baseline in SF-36 Scores at Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234	Study Baseline: MCS	42.4960 score on a scale	Standard Deviation 7.0731
Eliglustat: Participants From ENCORE	Change From Eliglustat Baseline in SF-36 Scores at Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234	MCS: Change at Week 130	2.9866 score on a scale	Standard Deviation 13.2768
Eliglustat: Participants From ENCORE	Change From Eliglustat Baseline in SF-36 Scores at Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234	Study Baseline: PCS	42.7817 score on a scale	Standard Deviation 1.4815
Eliglustat: Participants From ENCORE	Change From Eliglustat Baseline in SF-36 Scores at Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234	MCS: Week 78	50.0740 score on a scale	Standard Deviation 11.4453
Eliglustat: Participants From ENCORE	Change From Eliglustat Baseline in SF-36 Scores at Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234	PCS: Week 26	55.0589 score on a scale	Standard Deviation 7.7136
Eliglustat: Participants From ENCORE	Change From Eliglustat Baseline in SF-36 Scores at Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234	MCS: Week 182	48.0439 score on a scale	Standard Deviation 14.5143
Eliglustat: Participants From ENCORE	Change From Eliglustat Baseline in SF-36 Scores at Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234	PCS: Week 52	52.3379 score on a scale	Standard Deviation 8.2436
Eliglustat: Participants From ENCORE	Change From Eliglustat Baseline in SF-36 Scores at Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234	MCS: Change at Week 78	7.5780 score on a scale	Standard Deviation 7.7376
Eliglustat: Participants From ENCORE	Change From Eliglustat Baseline in SF-36 Scores at Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234	PCS: Week 104	54.9983 score on a scale	Standard Deviation 6.3833
Eliglustat: Participants From ENCORE	Change From Eliglustat Baseline in SF-36 Scores at Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234	PCS: Change at Week 104	12.2166 score on a scale	Standard Deviation 7.0338
Eliglustat: Participants From ENCORE	Change From Eliglustat Baseline in SF-36 Scores at Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234	PCS: Change at Week 130	9.7897 score on a scale	Standard Deviation 11.8746
Eliglustat: Participants From ENCORE	Change From Eliglustat Baseline in SF-36 Scores at Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234	MCS: Week 156	47.5165 score on a scale	Standard Deviation 18.1409
Eliglustat: Participants From ENCORE	Change From Eliglustat Baseline in SF-36 Scores at Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234	PCS: Change at Week 156	8.8854 score on a scale	Standard Deviation 10.5054
Eliglustat: Participants From ENCORE	Change From Eliglustat Baseline in SF-36 Scores at Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234	MCS: Week 208	44.1884 score on a scale	Standard Deviation 20.0085
Eliglustat: Participants From ENCORE	Change From Eliglustat Baseline in SF-36 Scores at Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234	PCS: Week 182	51.7047 score on a scale	Standard Deviation 11.3772

Secondary

Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Treatment-emergent Serious Adverse Events (TESAEs)

An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug and did not necessarily had to have a causal relationship with the treatment. A serious adverse event (SAE) was any untoward medical occurrence that at any dose: resulted in death, was life-threatening, required inpatient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, was a congenital anomaly/birth defect, was a medically important event. TEAEs were defined as AEs that developed, worsened or became serious during the treatment-emergent period (time from the first administration of the investigational medicinal product (IMP) to the last administration of the IMP + 5 days).

Time frame: From the first administration of the IMP to the last administration of the IMP + 5 days (up to 4 years, or until commercial eliglustat was available to participants through reimbursement or through the compassionate use [expanded access] program)

Population: Analysis was performed on FAS population.

Arm	Measure	Group	Value (COUNT_OF_PARTICIPANTS)
Eliglustat	Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Treatment-emergent Serious Adverse Events (TESAEs)	TEAEs	19 Participants
Eliglustat	Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Treatment-emergent Serious Adverse Events (TESAEs)	TESAEs	4 Participants

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026

Phase 3b Study to Evaluate Skeletal Response to Eliglustat in Adult Patients Who Completed Phase 2 or Phase 3 Studies

Conditions

Brief summary

Detailed description

Related papers

Interventions

Sponsors

Study design

Eligibility

Inclusion criteria

Exclusion criteria

Design outcomes

Primary

Secondary

Countries

Participant flow

Recruitment details

Pre-assignment details

Participants by arm

Withdrawals & dropouts

Baseline characteristics

Adverse events

Outcome results

Change From Current Study Baseline in Bone Biomarker Level: Macrophage Inflammatory Protein 1 Beta (MIP-1β) at Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234

Change From Current Study Baseline in Bone Biomarker Level: Procollagen 1 N- Terminal Propeptide (P1NP) at Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234

Change From Current Study Baseline in Bone Biomarker Level: Type 1 Collagen C-Telopeptides (CTx) at Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234

Change From Current Study Baseline in Spine and Femur Total T-Scores for BMD at Weeks 52, 104, 156 and 208

Change From Current Study Baseline in Spine and Femur Total Z-Scores for BMD at Weeks 52, 104, 156 and 208

Change From Current Study Baseline in Total Bone Marrow Burden (BMB) Scores at Weeks 52, 104, 156 and 208

Change From Current Study Baseline in Total Spine and Femur Bone Mineral Density (BMD) at Weeks 52, 104, 156 and 208

Change From Eliglustat Baseline in Bone Biomarker Level: Macrophage Inflammatory Protein 1 Beta (MIP-1β) at Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234

Change From Eliglustat Baseline in Bone Biomarker Level: Procollagen 1 N- Terminal Propeptide (P1NP) at Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234

Change From Eliglustat Baseline in Bone Biomarker Level: Type 1 Collagen C-Telopeptides (CTx) at Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234

Change From Eliglustat Baseline in Spine and Femur Total T-Scores for BMD at Weeks 52, 104, 156 and 208

Change From Eliglustat Baseline in Spine and Femur Total Z-Scores for BMD at Weeks 52, 104, 156 and 208

Change From Eliglustat Baseline in Total Bone Marrow Burden (BMB) Scores at Weeks 52, 104, 156 and 208

Change From Eliglustat Baseline in Total Spine and Femur Bone Mineral Density (BMD) at Weeks 52, 104, 156 and 208

Number of Participants With Bone Crisis at Study Baseline, Weeks 52, 104, 156 and 208

Number of Participants With Bone Pain Levels During the Past 4 Weeks at Study Baseline, Weeks 52, 104, 156 and 208

Number of Participants With Mobility Status Assessments at Study Baseline, Weeks 52, 104, 156 and 208

Observed Annual Incidence Rate for Spine and Femur Fracture at Week 52, 104, 156 and 208

Observed Annual Incidence Rate for Spine and Femur Infarcts at Week 52, 104, 156 and 208

Observed Annual Incidence Rate for Spine and Femur Osteonecrosis at Week 52, 104, 156 and 208

Observed Annual Incidence Rate for Spine Lytic Lesion at Week 104, and 208

Total Number of New or Worsening Fracture Events for Spine and Femur at Study Baseline, Week 52, 104, 156 and 208

Total Number of New or Worsening Infarcts Events for Spine and Femur at Study Baseline, Week 52, 104, 156 and 208

Total Number of New or Worsening Lytic Lesions Events for Spine at Study Baseline, Week 104, and 208

Total Number of New or Worsening Osteonecrosis Events for Spine and Femur at Study Baseline, Week 52, 104, 156 and 208

Change From Current Study Baseline in Gaucher Disease Type 1 (GD1) Biomarker Levels: Chitotriosidase at Week 26, 52, 78, 104, 130, 156, 182, 208 and 234

Change From Current Study Baseline in GD1 Biomarker Levels: Glucosylceramide (GL-1) at Week 26, 52, 78, 104, 130, 156, 182, 208 and 234

Change From Current Study Baseline in GD1 Biomarker Levels: Lyso Glucosylceramide (Lyso-GL-1) at Week 26, 52, 78, 104, 130, 156, 182, 208 and 234

Change From Current Study Baseline in Short Form-36 Health Survey (SF-36) Scores at Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234

Change From Eliglustat Baseline in Gaucher Disease Type 1 (GD1) Biomarker Levels: Chitotriosidase at Week 26, 52, 78, 104, 130, 156, 182, 208 and 234

Change From Eliglustat Baseline in GD1 Biomarker Levels: Glucosylceramide (GL-1) at Week 26, 52, 78, 104, 130, 156, 182, 208 and 234

Change From Eliglustat Baseline in GD1 Biomarker Levels: Lyso Glucosylceramide (Lyso-GL-1) at Week 26, 52, 78, 104, 130, 156, 182, 208 and 234

Change From Eliglustat Baseline in SF-36 Scores at Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234

Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Treatment-emergent Serious Adverse Events (TESAEs)