Gaucher Disease, Type 1, Cerebroside Lipidosis Syndrome, Glucocerebrosidase Deficiency Disease, Glucosylceramide Beta-Glucosidase Deficiency Disease, Gaucher Disease, Non-Neuronopathic Form
Conditions
Keywords
Type 1 Gaucher Disease, Glucocerebrosidase Deficiency Disease
Brief summary
Gaucher disease is a genetic disease that results in a deficiency of an enzyme acid beta-glucosidase, also known as glucocerebrosidase. This enzyme is needed to digest a substrate (lipid) called glucosylceramide and, to a lesser degree, glucosylsphingosine. In participants with Gaucher disease, the liver, spleen, bone marrow and brain show increases in lipid concentration, specifically in cells derived from the monocyte/macrophage system. Eliglustat tartrate (Genz-112638) is an oral drug that may regulate the Gaucher disease process by decreasing the synthesis of glucosylceramide. The primary objective of this study is to evaluate the efficacy, safety and pharmacokinetics (PK) of eliglustat tartrate, administered as an oral dose of either 50 milligram (mg) twice daily (BID) or 100 mg BID, to men and women with Gaucher disease Type 1 for 52 weeks.
Detailed description
This study consists of several phases: screening (-28 to -1 days), dose adjustment/treatment (Day 1 \[treatment baseline\] to Day 30), initial steady-state treatment (post-Day 30 through Week 52 post-baseline), a treatment interruption period (Week 52 through approximately Week 54), long-term steady-state treatment (approximately Week 54 through study completion), and safety follow-up (30 to 37 days after a participant withdraws from or completes the study). The Primary Analysis Period is from baseline through Week 52. The Extension Period is from Week 52 through study completion (that is, participant withdrawal, the study is terminated, eliglustat tartrate becomes commercially available, or where applicable, specific regulatory requirements have been met).
Interventions
Eliglustat (Genz-112638) capsule as single 50 mg dose on Day 1 then eliglustat 50 mg twice daily (BID) from Day 2 to Day 19, then either eliglustat 50 mg BID (if Genz-99067\[active moiety of eliglustat in plasma\] trough plasma concentration was greater than or equal to \[\>=\]5 nanogram per milliliter \[ng/mL\] on Day 10) or eliglustat 100 mg BID(if Genz-99067 trough plasma concentration was less than \[\<\] 5 ng/mL), from day 20 to Year 4. After primary completion date (Week 52) participants underwent treatment interruption period of approximately 2 weeks before continuing same treatment through study completion (Year 9). Participant receiving 100 mg BID could be considered for further dose increase to 150 mg BID at Week 24 if they met certain criteria (for example, had been on treatment for at least 24 months, had not reached therapeutic goals established for participants receiving Cerezyme, and if all other causes for lack of treatment effect had been evaluated and ruled out).
Sponsors
Genzyme, a Sanofi Company
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to 65 Years
Healthy volunteers
No
Inclusion criteria
* The participant had a diagnosis of Gaucher Type I disease and a documented deficiency of glucocerebrosidase activity by enzyme assay and was willing and able to provide written informed consent prior to initiating any study-related procedures; * The participant was 18 to 65 years old and weighed between 50 and 120 kilogram (kg) at enrollment; * The participant had the following symptoms of Gaucher disease identified within 28 days of enrollment (at screening); * Anemia - indicated by hemoglobin measurements taken during the screening phase (8 to 10 gram per deciliter (g/dL) if female, 8 to 11 g/dL if male); * Thrombocytopenia - indicated by platelet count measurements taken during the screening phase (60000 to 100000 per cubic millimeter); * Splenomegaly, as indicated by magnetic resonance imaging (MRI) or spiral computed tomography (CT) (\>= 10 multiples of normal); * Female participants of child-bearing potential must had a documented negative serum pregnancy test prior to dosing. Female participants agreed to use a reliable method of birth control throughout duration of trial.
Exclusion criteria
* Participant had a partial or total splenectomy or infarcted areas of the spleen; * Participant had documented prior bleeding varices or liver infarction; * Participant received miglustat within 12 months prior to study enrollment; * The participant had received an investigational product within 30 days prior to study enrollment; * Participant had neurologic or pulmonary involvement; * Participant had new pathological bone involvement or bone crisis in the 12 months prior to enrollment; * Participant was transfusion-dependent; * Participant had a documented etiology of anemia due to causes other than Gaucher disease; * The participant had cardiac functional and/or anatomical abnormalities, a history of cancer or tested positive for human immunodeficiency virus (HIV) antibody or Hepatitis; * Participant had a clinically significant disease, other than Gaucher disease, including cardiovascular, renal, hepatic, gastrointestinal, pulmonary, neurologic, endocrine, metabolic, or psychiatric disease, other medical conditions, or serious intercurrent illnesses that, in the opinion of the Investigator, might preclude participation in the study.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Percentage of Participants Demonstrating A Meaningful Clinical Response | Baseline, Year 1 | A meaningful clinical response was defined as an improvement in at least 2 of the 3 main efficacy parameters: a) an increase in hemoglobin of greater than or equal to (\>=) 0.5 gram/deciliter from baseline, b) an increase in platelets of \>=15 percent (%) from baseline, c) reduction in total spleen volume of \>= 15% from baseline. As hemoglobin, platelets, total spleen volume were abnormal at baseline, within each participant, only those parameters were used in the evaluation of meaningful clinical response which were abnormal at baseline. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Percent Change From Baseline in Liver Volume at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study (Up to Year 9) | Percent change in liver volume = (\[liver volume at specified time points minus liver volume at baseline\] divided by \[liver volume at baseline\]) multiplied by 100, where all volumes are in multiples of normal. |
| Absolute Change From Baseline in Hemoglobin at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study (Up to Year 9) | Absolute change = hemoglobin level at specified time points minus hemoglobin level at baseline. |
| Percent Change From Baseline in Platelet Count at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study (Up to Year 9) | Percent change in platelet count = (\[platelet count at specified time points minus platelet count at baseline\] divided by \[platelet count at baseline\]) multiplied by 100. |
| Percent Change From Baseline in Biomarker (Angiotensin Converting Enzyme) Level at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and End of Study (Up to Year 9) | — |
| Percent Change From Baseline in Biomarker (Tartrate-Resistant Acid Phosphatase [TRAP]) Level at Year 1 and Year 2 | Baseline, Year 1, Year 2 | — |
| Percent Change From Baseline in Biomarker Chemokine Ligand 18 (CCL18) Level at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and End of Study | Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and End of Study (Up to Year 9) | — |
| Percent Change From Baseline in Biomarker (Chitotriosidase) Level at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study (Up to Year 9) | — |
| Percent Change From Baseline in Spleen Volume at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study (Up to Year 9) | Percent change in spleen volume = (\[spleen volume at specified time points minus spleen volume at baseline\] divided by \[spleen volume at baseline\]) multiplied by 100, where all volumes are in multiples of normal. |
| Change From Baseline in Fatigue Severity Scale (FSS) Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study (Up to Year 9) | The FSS is an instrument consisting of 9 self-administered questions that measures the impact of severity of fatigue symptoms on everyday functioning, based on the recall over the past week. Score range for each question ranges from 1 (minimum) to 7 (maximum), where higher score indicates greater severity. FSS total score was calculated by averaging the results of all questions. Total FSS score ranges from 9 (minimum) to 63 (maximum), where higher scores indicates greater severity. |
| Number of Participants With Bone Pain Levels During the Past 4 Weeks at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study (Up to Year 9) | Bone pain was assessed as a part of Gaucher disease assessment in participants. Participants were categorized as none (no bone pain), very mild bone pain, mild bone pain and moderate bone pain. In this outcome, number of participants with different levels of bone pain at specified time points were reported. |
| Number of Participants With Mobility Status (MS) at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study (Up to Year 9) | Mobillity, i.e. ability to walk was assessed as a part of Gaucher disease assessment in participants.In this outcome, number of participants with their different mobility status (unrestricted mobility, walks with difficulty) at specified time points were reported. |
| Number of Participants With No Bone Crisis at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study (Up to Year 9) | Bone crisis was assessed as a part of Gaucher disease assessment in participants. Acute, excruciating episodic bone pain is characteristic of Gaucher bone crisis, which typically causes debilitation lasting several days or longer and requires treatment with immobilization, hydration, and opioid analgesics. Participants were categorized as 0= no bone crisis, 1= 1 bone crisis, and 2= 2 bone crises during the assessment period. In this outcome, number of participants with 0= no bone crises levels at specified time points were reported. |
| Bone Marrow Infiltration: Number of Participants With Improvement From Baseline in Dark Marrow at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and at End of Study (EOS) | Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and at End of Study (up to Year 9) | Bone marrow infiltration assessments were designed to evaluate improvements in dark marrow using MRI. Each MRI assessment was performed for both femurs and consisted of reviewing 6 different zones (the femoral head, greater trochanter, intertrochanteric region, shaft, distal metaphysis, and condyles). MRI images recorded dark marrow for each zone as either present or not present at baseline. In this outcome, number of participants (for whom dark marrow was present at baseline) with improvement from baseline in dark marrow at each specified time point were reported. |
| Lumbar Spine and Femur T-Scores for Bone Mineral Density (BMD) at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study (up to Year 9) | Images of the lumbar spine and femur were obtained by dual energy X-ray absorptiometry (DXA) to determine T-score for each bone area and total bone mineral density. T-scores compares participant's bone density with that of healthy young participant of same gender. The T-score bone density categories were: normal (score \>-1), osteopenia (score -2.5 to \<=-1), and osteoporosis (score \<= -2.5). |
| Lumbar Spine and Femur Z-Scores for BMD at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study (up to Year 9) | Images of the lumbar spine and femur were obtained by DXA to determine Z-score for each bone area and total bone mineral density. The Z-score bone density categories are: normal (score \>-2) and below normal (score \<=-2). |
| Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study | Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study (Up to Year 9) | The SF-36 questionnaire, version 2, investigates the participant's health-related quality of life (HRQL). It is a 36-item questionnaire measuring 8 domains (physical functioning, role-physical, bodily pain, general health, vitality, social functioning, role emotional, and mental health). Each domain score ranges from 0 (worst) to 100 (best), with higher scores reflecting best health-related quality of life. Two summary scale scores were computed from the 8 domain scores: the Physical Component Summary and the Mental Component Summary. Score range for both summary scale ranges from 0 (worst) to 100 (best), with higher scores reflecting best health-related quality of life. |
Countries
Argentina, Israel, Italy, Mexico, Russia, United States
Participant flow
Pre-assignment details
A total of 50 participants were screened of which 24 participants were screen failure. A total of 26 participants were enrolled in this study.
Participants by arm
| Arm | Count |
|---|---|
| Eliglustat Eliglustat (Genz-112638) capsule as single 50 mg dose on Day 1 then eliglustat 50 mg BID from Day 2 to Day 19, and then either eliglustat 50 mg BID (if Genz-99067 \[active moiety of eliglustat in plasma\] trough plasma concentration was greater than or equal to \[\>=\] 5 ng/mL on Day 10) or eliglustat 100 mg BID (if Genz-99067 trough plasma concentration was less than \[\<\] 5 ng/mL), from day 20 to Year 4. After primary completion date (Week 52) participants underwent treatment interruption period of approximately 2 weeks before continuing the same treatment up to Year 9. Participant receiving 100 mg BID could be considered for a further dose increase to 150 mg BID at Week 24 if they met certain criteria (for example, had been on treatment for at least 24 months, had not reached therapeutic goals established for participants receiving Cerezyme, and if all other causes for lack of treatment effect had been evaluated and ruled out). | 26 |
| Total | 26 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| Overall Study | Adverse Event | 3 |
| Overall Study | Undefined | 3 |
| Overall Study | Withdrawal by Subject | 1 |
Baseline characteristics
| Characteristic | Eliglustat |
|---|---|
| Age, Continuous | 34.47 years STANDARD_DEVIATION 12.96 |
| Body Mass Index (BMI) | 22.56 kilogram per square meter (kg/m^2) STANDARD_DEVIATION 3.529 |
| Gender Female | 16 Participants |
| Gender Male | 10 Participants |
| Height | 165.12 centimeter (cm) STANDARD_DEVIATION 9.747 |
| Hemoglobin | 11.10 gram per deciliter (g/dL) STANDARD_DEVIATION 1.674 |
| Platelet Count | 66.423 10^9 cells per liter STANDARD_DEVIATION 20.1413 |
| Race/Ethnicity, Customized Ashkenazi Jewish | 7 participants |
| Race/Ethnicity, Customized Hispanic | 3 participants |
| Race/Ethnicity, Customized Non-Jewish Caucasian | 16 participants |
| Spleen Volume | 20.04 Multiples of normal STANDARD_DEVIATION 12.798 |
| Weight | 61.47 kilogram (kg) STANDARD_DEVIATION 11.018 |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk | EG002 affected / at risk |
|---|---|---|---|
| deaths Total, all-cause mortality | — / — | — / — | — / — |
| other Total, other adverse events | 5 / 5 | 18 / 18 | 23 / 26 |
| serious Total, serious adverse events | 0 / 5 | 3 / 18 | 5 / 26 |
Outcome results
Primary
Percentage of Participants Demonstrating A Meaningful Clinical Response
A meaningful clinical response was defined as an improvement in at least 2 of the 3 main efficacy parameters: a) an increase in hemoglobin of greater than or equal to (\>=) 0.5 gram/deciliter from baseline, b) an increase in platelets of \>=15 percent (%) from baseline, c) reduction in total spleen volume of \>= 15% from baseline. As hemoglobin, platelets, total spleen volume were abnormal at baseline, within each participant, only those parameters were used in the evaluation of meaningful clinical response which were abnormal at baseline.
Time frame: Baseline, Year 1
Population: Full analysis set consists of all participants who signed informed consent and received at least one dose of eliglustat.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Eliglustat | Percentage of Participants Demonstrating A Meaningful Clinical Response | 77 percentage of participants |
Secondary
Absolute Change From Baseline in Hemoglobin at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study
Absolute change = hemoglobin level at specified time points minus hemoglobin level at baseline.
Time frame: Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study (Up to Year 9)
Population: Full analysis set consists of all participants who signed informed consent and received at least one dose of eliglustat. Here 'n' signifies number of participants with available data at specified time points.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Eliglustat | Absolute Change From Baseline in Hemoglobin at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Change at Year 1 (n=22) | 1.70 g/dL | Standard Deviation 1.274 |
| Eliglustat | Absolute Change From Baseline in Hemoglobin at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Change at Year 2 (n=20) | 2.13 g/dL | Standard Deviation 1.507 |
| Eliglustat | Absolute Change From Baseline in Hemoglobin at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Change at Year 3 (n=18) | 2.47 g/dL | Standard Deviation 1.406 |
| Eliglustat | Absolute Change From Baseline in Hemoglobin at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Change at Year 4 (n=19) | 2.27 g/dL | Standard Deviation 1.451 |
| Eliglustat | Absolute Change From Baseline in Hemoglobin at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Change at Year 5 (n=19) | 2.09 g/dL | Standard Deviation 1.746 |
| Eliglustat | Absolute Change From Baseline in Hemoglobin at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Change at Year 6 (n=18) | 2.01 g/dL | Standard Deviation 1.326 |
| Eliglustat | Absolute Change From Baseline in Hemoglobin at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Change at Year 7 (n=19) | 2.07 g/dL | Standard Deviation 1.485 |
| Eliglustat | Absolute Change From Baseline in Hemoglobin at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Change at Year 8 (n=16) | 2.08 g/dL | Standard Deviation 1.748 |
| Eliglustat | Absolute Change From Baseline in Hemoglobin at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Change at Year 9 (n=4) | 1.00 g/dL | Standard Deviation 1.48 |
| Eliglustat | Absolute Change From Baseline in Hemoglobin at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Change at End of Study (n=18) | 2.01 g/dL | Standard Deviation 1.785 |
Secondary
Bone Marrow Infiltration: Number of Participants With Improvement From Baseline in Dark Marrow at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and at End of Study (EOS)
Bone marrow infiltration assessments were designed to evaluate improvements in dark marrow using MRI. Each MRI assessment was performed for both femurs and consisted of reviewing 6 different zones (the femoral head, greater trochanter, intertrochanteric region, shaft, distal metaphysis, and condyles). MRI images recorded dark marrow for each zone as either present or not present at baseline. In this outcome, number of participants (for whom dark marrow was present at baseline) with improvement from baseline in dark marrow at each specified time point were reported.
Time frame: Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and at End of Study (up to Year 9)
Population: Full analysis set consists of all participants who signed informed consent and received at least one dose of eliglustat. Here 'n' signifies number of participants who were presented with dark marrow at baseline and had data available at specified time points.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Eliglustat | Bone Marrow Infiltration: Number of Participants With Improvement From Baseline in Dark Marrow at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and at End of Study (EOS) | Femoral Head: Improved: Year 1: (n=16) | 3 Participants |
| Eliglustat | Bone Marrow Infiltration: Number of Participants With Improvement From Baseline in Dark Marrow at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and at End of Study (EOS) | Femoral Head: Improved: Year 2: (n=14) | 2 Participants |
| Eliglustat | Bone Marrow Infiltration: Number of Participants With Improvement From Baseline in Dark Marrow at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and at End of Study (EOS) | Femoral Head: Improved: Year 3: (n=13) | 2 Participants |
| Eliglustat | Bone Marrow Infiltration: Number of Participants With Improvement From Baseline in Dark Marrow at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and at End of Study (EOS) | Femoral Head: Improved: Year 4: (n=13) | 2 Participants |
| Eliglustat | Bone Marrow Infiltration: Number of Participants With Improvement From Baseline in Dark Marrow at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and at End of Study (EOS) | Femoral Head: Improved: Year 5: (n=13) | 4 Participants |
| Eliglustat | Bone Marrow Infiltration: Number of Participants With Improvement From Baseline in Dark Marrow at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and at End of Study (EOS) | Femoral Head: Improved: Year 6: (n=12) | 5 Participants |
| Eliglustat | Bone Marrow Infiltration: Number of Participants With Improvement From Baseline in Dark Marrow at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and at End of Study (EOS) | Femoral Head: Improved: Year 7: (n=13) | 5 Participants |
| Eliglustat | Bone Marrow Infiltration: Number of Participants With Improvement From Baseline in Dark Marrow at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and at End of Study (EOS) | Femoral Head: Improved: Year 8: (n=13) | 5 Participants |
| Eliglustat | Bone Marrow Infiltration: Number of Participants With Improvement From Baseline in Dark Marrow at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and at End of Study (EOS) | Femoral Head: Improved: End of Study: (n=8) | 3 Participants |
| Eliglustat | Bone Marrow Infiltration: Number of Participants With Improvement From Baseline in Dark Marrow at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and at End of Study (EOS) | Greater Trochanter: Improved: Year 1 (n=8) | 3 Participants |
| Eliglustat | Bone Marrow Infiltration: Number of Participants With Improvement From Baseline in Dark Marrow at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and at End of Study (EOS) | Greater Trochanter: Improved: Year 2 (n=7) | 2 Participants |
| Eliglustat | Bone Marrow Infiltration: Number of Participants With Improvement From Baseline in Dark Marrow at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and at End of Study (EOS) | Greater Trochanter: Improved: Year 3 (n=6) | 3 Participants |
| Eliglustat | Bone Marrow Infiltration: Number of Participants With Improvement From Baseline in Dark Marrow at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and at End of Study (EOS) | Greater Trochanter: Improved: Year 4 (n=6) | 3 Participants |
| Eliglustat | Bone Marrow Infiltration: Number of Participants With Improvement From Baseline in Dark Marrow at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and at End of Study (EOS) | Greater Trochanter: Improved: Year 5 (n=7) | 3 Participants |
| Eliglustat | Bone Marrow Infiltration: Number of Participants With Improvement From Baseline in Dark Marrow at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and at End of Study (EOS) | Greater Trochanter: Improved: Year 6 (n=6) | 1 Participants |
| Eliglustat | Bone Marrow Infiltration: Number of Participants With Improvement From Baseline in Dark Marrow at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and at End of Study (EOS) | Greater Trochanter: Improved: Year 7 (n=7) | 1 Participants |
| Eliglustat | Bone Marrow Infiltration: Number of Participants With Improvement From Baseline in Dark Marrow at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and at End of Study (EOS) | Greater Trochanter: Improved: Year 8 (n=7) | 1 Participants |
| Eliglustat | Bone Marrow Infiltration: Number of Participants With Improvement From Baseline in Dark Marrow at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and at End of Study (EOS) | Greater Trochanter: Improved: End of Study (n=4) | 0 Participants |
| Eliglustat | Bone Marrow Infiltration: Number of Participants With Improvement From Baseline in Dark Marrow at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and at End of Study (EOS) | Intertrochanteric Regions: Improved: Year 1 (n=21) | 1 Participants |
| Eliglustat | Bone Marrow Infiltration: Number of Participants With Improvement From Baseline in Dark Marrow at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and at End of Study (EOS) | Intertrochanteric Regions: Improved: Year 2 (n=19) | 3 Participants |
| Eliglustat | Bone Marrow Infiltration: Number of Participants With Improvement From Baseline in Dark Marrow at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and at End of Study (EOS) | Intertrochanteric Regions: Improved: Year 3 (n=18) | 3 Participants |
| Eliglustat | Bone Marrow Infiltration: Number of Participants With Improvement From Baseline in Dark Marrow at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and at End of Study (EOS) | Intertrochanteric Regions: Improved: Year 4 (n=18) | 3 Participants |
| Eliglustat | Bone Marrow Infiltration: Number of Participants With Improvement From Baseline in Dark Marrow at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and at End of Study (EOS) | Intertrochanteric Regions: Improved: Year 5 (n=18) | 5 Participants |
| Eliglustat | Bone Marrow Infiltration: Number of Participants With Improvement From Baseline in Dark Marrow at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and at End of Study (EOS) | Intertrochanteric Regions: Improved: Year 6 (n=17) | 6 Participants |
| Eliglustat | Bone Marrow Infiltration: Number of Participants With Improvement From Baseline in Dark Marrow at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and at End of Study (EOS) | Intertrochanteric Regions: Improved: Year 7 (n=18) | 6 Participants |
| Eliglustat | Bone Marrow Infiltration: Number of Participants With Improvement From Baseline in Dark Marrow at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and at End of Study (EOS) | Intertrochanteric Regions: Improved: Year 8 (n=17) | 7 Participants |
| Eliglustat | Bone Marrow Infiltration: Number of Participants With Improvement From Baseline in Dark Marrow at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and at End of Study (EOS) | Intertrochanteric Regions: Improved: EOS (n=12) | 5 Participants |
| Eliglustat | Bone Marrow Infiltration: Number of Participants With Improvement From Baseline in Dark Marrow at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and at End of Study (EOS) | Shaft: Improved: Year 1 (n= 20) | 1 Participants |
| Eliglustat | Bone Marrow Infiltration: Number of Participants With Improvement From Baseline in Dark Marrow at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and at End of Study (EOS) | Shaft: Improved: Year 2 (n= 18) | 3 Participants |
| Eliglustat | Bone Marrow Infiltration: Number of Participants With Improvement From Baseline in Dark Marrow at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and at End of Study (EOS) | Shaft: Improved: Year 3 (n= 17) | 3 Participants |
| Eliglustat | Bone Marrow Infiltration: Number of Participants With Improvement From Baseline in Dark Marrow at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and at End of Study (EOS) | Shaft: Improved: Year 4 (n= 17) | 3 Participants |
| Eliglustat | Bone Marrow Infiltration: Number of Participants With Improvement From Baseline in Dark Marrow at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and at End of Study (EOS) | Shaft: Improved: Year 5 (n= 17) | 4 Participants |
| Eliglustat | Bone Marrow Infiltration: Number of Participants With Improvement From Baseline in Dark Marrow at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and at End of Study (EOS) | Shaft: Improved: Year 6 (n= 16) | 5 Participants |
| Eliglustat | Bone Marrow Infiltration: Number of Participants With Improvement From Baseline in Dark Marrow at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and at End of Study (EOS) | Shaft: Improved: Year 7 (n= 17) | 5 Participants |
| Eliglustat | Bone Marrow Infiltration: Number of Participants With Improvement From Baseline in Dark Marrow at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and at End of Study (EOS) | Shaft: Improved: Year 8 (n= 16) | 5 Participants |
| Eliglustat | Bone Marrow Infiltration: Number of Participants With Improvement From Baseline in Dark Marrow at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and at End of Study (EOS) | Shaft: Improved: End of Study (n=11) | 4 Participants |
| Eliglustat | Bone Marrow Infiltration: Number of Participants With Improvement From Baseline in Dark Marrow at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and at End of Study (EOS) | Distal Metaphysis: Improved: Year 1 (n=21) | 2 Participants |
| Eliglustat | Bone Marrow Infiltration: Number of Participants With Improvement From Baseline in Dark Marrow at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and at End of Study (EOS) | Distal Metaphysis: Improved: Year 2 (n=19) | 3 Participants |
| Eliglustat | Bone Marrow Infiltration: Number of Participants With Improvement From Baseline in Dark Marrow at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and at End of Study (EOS) | Distal Metaphysis: Improved: Year 3 (n=18) | 4 Participants |
| Eliglustat | Bone Marrow Infiltration: Number of Participants With Improvement From Baseline in Dark Marrow at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and at End of Study (EOS) | Distal Metaphysis: Improved: Year 4 (n=18) | 3 Participants |
| Eliglustat | Bone Marrow Infiltration: Number of Participants With Improvement From Baseline in Dark Marrow at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and at End of Study (EOS) | Distal Metaphysis: Improved: Year 5 (n=18) | 7 Participants |
| Eliglustat | Bone Marrow Infiltration: Number of Participants With Improvement From Baseline in Dark Marrow at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and at End of Study (EOS) | Distal Metaphysis: Improved: Year 6 (n=17) | 8 Participants |
| Eliglustat | Bone Marrow Infiltration: Number of Participants With Improvement From Baseline in Dark Marrow at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and at End of Study (EOS) | Distal Metaphysis: Improved: Year 7 (n=18) | 8 Participants |
| Eliglustat | Bone Marrow Infiltration: Number of Participants With Improvement From Baseline in Dark Marrow at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and at End of Study (EOS) | Distal Metaphysis: Improved: Year 8 (n=17) | 8 Participants |
| Eliglustat | Bone Marrow Infiltration: Number of Participants With Improvement From Baseline in Dark Marrow at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and at End of Study (EOS) | Distal Metaphysis: Improved: End of Study (n=12) | 6 Participants |
| Eliglustat | Bone Marrow Infiltration: Number of Participants With Improvement From Baseline in Dark Marrow at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and at End of Study (EOS) | Condyles: Improved: Year 1 (n=11) | 3 Participants |
| Eliglustat | Bone Marrow Infiltration: Number of Participants With Improvement From Baseline in Dark Marrow at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and at End of Study (EOS) | Condyles: Improved: Year 2 (n=9) | 1 Participants |
| Eliglustat | Bone Marrow Infiltration: Number of Participants With Improvement From Baseline in Dark Marrow at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and at End of Study (EOS) | Condyles: Improved: Year 3 (n=8) | 1 Participants |
| Eliglustat | Bone Marrow Infiltration: Number of Participants With Improvement From Baseline in Dark Marrow at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and at End of Study (EOS) | Condyles: Improved: Year 4 (n=8) | 1 Participants |
| Eliglustat | Bone Marrow Infiltration: Number of Participants With Improvement From Baseline in Dark Marrow at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and at End of Study (EOS) | Condyles: Improved: Year 7 (n=8) | 2 Participants |
| Eliglustat | Bone Marrow Infiltration: Number of Participants With Improvement From Baseline in Dark Marrow at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and at End of Study (EOS) | Condyles: Improved: Year 8 (n=8) | 2 Participants |
| Eliglustat | Bone Marrow Infiltration: Number of Participants With Improvement From Baseline in Dark Marrow at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and at End of Study (EOS) | Condyles: Improved: End of Study (n=5) | 2 Participants |
| Eliglustat | Bone Marrow Infiltration: Number of Participants With Improvement From Baseline in Dark Marrow at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and at End of Study (EOS) | Condyles: Improved: Year 5 (n=8) | 2 Participants |
| Eliglustat | Bone Marrow Infiltration: Number of Participants With Improvement From Baseline in Dark Marrow at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and at End of Study (EOS) | Condyles: Improved: Year 6 (n=7) | 2 Participants |
Secondary
Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study
The SF-36 questionnaire, version 2, investigates the participant's health-related quality of life (HRQL). It is a 36-item questionnaire measuring 8 domains (physical functioning, role-physical, bodily pain, general health, vitality, social functioning, role emotional, and mental health). Each domain score ranges from 0 (worst) to 100 (best), with higher scores reflecting best health-related quality of life. Two summary scale scores were computed from the 8 domain scores: the Physical Component Summary and the Mental Component Summary. Score range for both summary scale ranges from 0 (worst) to 100 (best), with higher scores reflecting best health-related quality of life.
Time frame: Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study (Up to Year 9)
Population: Full analysis set consists of all participants who signed informed consent and received at least one dose of eliglustat. Here 'n' signifies number of participants with available data at specified time points.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Eliglustat | Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study | Physical Functioning: Change at Year 1 (n=22) | 8.41 units on a scale | Standard Deviation 14.67 |
| Eliglustat | Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study | Physical Functioning: Change at Year 2 (n=20) | 9.50 units on a scale | Standard Deviation 17.837 |
| Eliglustat | Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study | Physical Functioning: Change at Year 3 (n=17) | 12.65 units on a scale | Standard Deviation 18.296 |
| Eliglustat | Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study | Physical Functioning: Change at Year 4 (n=19) | 11.32 units on a scale | Standard Deviation 16.231 |
| Eliglustat | Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study | Physical Functioning: Change at Year 5 (n=19) | 10.76 units on a scale | Standard Deviation 19.169 |
| Eliglustat | Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study | Physical Functioning: Change at Year 6 (n=19) | 12.63 units on a scale | Standard Deviation 17.589 |
| Eliglustat | Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study | Physical Functioning: Change at Year 7 (n=19) | 11.58 units on a scale | Standard Deviation 17.955 |
| Eliglustat | Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study | Physical Functioning: Change at Year 8 (n=16) | 11.56 units on a scale | Standard Deviation 17.485 |
| Eliglustat | Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study | Physical Functioning: Change at Year 9 (n=4) | 1.25 units on a scale | Standard Deviation 2.5 |
| Eliglustat | Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study | Physical Functioning:Change at End of Study (n=19) | 9.28 units on a scale | Standard Deviation 17.085 |
| Eliglustat | Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study | Role - Physical: Change at Year 1 (n=22) | 4.55 units on a scale | Standard Deviation 22.091 |
| Eliglustat | Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study | Role - Physical: Change at Year 2 (n=20) | 5.31 units on a scale | Standard Deviation 27.824 |
| Eliglustat | Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study | Role - Physical: Change at Year 3 (n=17) | 9.56 units on a scale | Standard Deviation 24.717 |
| Eliglustat | Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study | Role - Physical: Change at Year 4 (n=19) | 6.58 units on a scale | Standard Deviation 26.391 |
| Eliglustat | Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study | Role - Physical: Change at Year 5 (n=19) | 7.57 units on a scale | Standard Deviation 28.227 |
| Eliglustat | Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study | Role - Physical: Change at Year 6 (n=19) | 8.88 units on a scale | Standard Deviation 25.964 |
| Eliglustat | Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study | Role - Physical: Change at Year 7 (n=19) | 7.89 units on a scale | Standard Deviation 25.331 |
| Eliglustat | Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study | Role - Physical: Change at Year 8 (n=16) | 8.59 units on a scale | Standard Deviation 29.393 |
| Eliglustat | Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study | Role - Physical: Change at Year 9 (n=4) | -1.56 units on a scale | Standard Deviation 18.663 |
| Eliglustat | Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study | Role - Physical: Change at End of Study (n= 19) | 10.53 units on a scale | Standard Deviation 24.211 |
| Eliglustat | Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study | Bodily Pain: Change at Year 1 (n= 22) | -0.77 units on a scale | Standard Deviation 21.307 |
| Eliglustat | Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study | Bodily Pain: Change at Year 2 (n= 20) | 4.40 units on a scale | Standard Deviation 25.05 |
| Eliglustat | Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study | Bodily Pain: Change at Year 3 (n= 17) | 4.24 units on a scale | Standard Deviation 21.297 |
| Eliglustat | Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study | Bodily Pain: Change at Year 4 (n= 19) | -2.53 units on a scale | Standard Deviation 25.202 |
| Eliglustat | Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study | Bodily Pain: Change at Year 5 (n= 19) | 0.37 units on a scale | Standard Deviation 23.104 |
| Eliglustat | Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study | Bodily Pain: Change at Year 6 (n= 19) | -2.32 units on a scale | Standard Deviation 26.268 |
| Eliglustat | Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study | Bodily Pain: Change at Year 7 (n= 19) | 1.74 units on a scale | Standard Deviation 27.777 |
| Eliglustat | Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study | Bodily Pain: Change at Year 8 (n= 16) | 1.44 units on a scale | Standard Deviation 25.532 |
| Eliglustat | Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study | Bodily Pain: Change at Year 9 (n= 4) | 1.00 units on a scale | Standard Deviation 19.63 |
| Eliglustat | Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study | Bodily Pain: Change at End of Study (n= 19) | 6.11 units on a scale | Standard Deviation 29.603 |
| Eliglustat | Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study | General Health: Change at Year 1 (n= 22) | 10.36 units on a scale | Standard Deviation 19.822 |
| Eliglustat | Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study | General Health: Change at Year 2 (n= 20) | 11.30 units on a scale | Standard Deviation 24.262 |
| Eliglustat | Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study | General Health: Change at Year 3 (n= 17) | 16.94 units on a scale | Standard Deviation 23.485 |
| Eliglustat | Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study | General Health: Change at Year 4 (n= 19) | 15.53 units on a scale | Standard Deviation 23.136 |
| Eliglustat | Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study | General Health: Change at Year 5 (n= 19) | 13.58 units on a scale | Standard Deviation 23.71 |
| Eliglustat | Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study | General Health: Change at Year 6 (n= 19) | 12.83 units on a scale | Standard Deviation 23.443 |
| Eliglustat | Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study | General Health: Change at Year 7 (n= 19) | 15.89 units on a scale | Standard Deviation 23.278 |
| Eliglustat | Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study | General Health: Change at Year 8 (n= 16) | 18.19 units on a scale | Standard Deviation 20.14 |
| Eliglustat | Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study | General Health: Change at Year 9 (n= 4) | 11.25 units on a scale | Standard Deviation 11.087 |
| Eliglustat | Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study | General Health: Change at End of Study (n= 19) | 15.00 units on a scale | Standard Deviation 21.422 |
| Eliglustat | Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study | Vitality: Change at Year 1 (n= 22) | 5.68 units on a scale | Standard Deviation 17.242 |
| Eliglustat | Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study | Vitality: Change at Year 2 (n= 20) | 7.71 units on a scale | Standard Deviation 26.127 |
| Eliglustat | Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study | Vitality: Change at Year 3 (n= 17) | 14.71 units on a scale | Standard Deviation 21.188 |
| Eliglustat | Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study | Vitality: Change at Year 4 (n= 19) | 10.20 units on a scale | Standard Deviation 20.005 |
| Eliglustat | Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study | Vitality: Change at Year 5 (n= 19) | 13.82 units on a scale | Standard Deviation 23.623 |
| Eliglustat | Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study | Vitality: Change at Year 6 (n= 19) | 12.50 units on a scale | Standard Deviation 23.936 |
| Eliglustat | Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study | Vitality: Change at Year 7 (n= 19) | 7.57 units on a scale | Standard Deviation 22.782 |
| Eliglustat | Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study | Vitality: Change at Year 8 (n= 16) | 15.23 units on a scale | Standard Deviation 23.493 |
| Eliglustat | Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study | Vitality: Change at Year 9 (n= 4) | 3.13 units on a scale | Standard Deviation 10.825 |
| Eliglustat | Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study | Vitality: Change at End of Study (n= 19) | 15.13 units on a scale | Standard Deviation 21.278 |
| Eliglustat | Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study | Social Functioning: Change at Year 1 (n= 22) | -0.57 units on a scale | Standard Deviation 23.298 |
| Eliglustat | Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study | Social Functioning: Change at Year 2 (n= 20) | 10.00 units on a scale | Standard Deviation 26.47 |
| Eliglustat | Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study | Social Functioning: Change at Year 3 (n= 17) | 9.56 units on a scale | Standard Deviation 19.024 |
| Eliglustat | Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study | Social Functioning: Change at Year 4 (n= 19) | 3.29 units on a scale | Standard Deviation 25.291 |
| Eliglustat | Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study | Social Functioning: Change at Year 5 (n= 19) | 9.21 units on a scale | Standard Deviation 23.14 |
| Eliglustat | Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study | Social Functioning: Change at Year 6 (n= 19) | 9.21 units on a scale | Standard Deviation 26.628 |
| Eliglustat | Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study | Social Functioning: Change at Year 7 (n= 19) | 8.55 units on a scale | Standard Deviation 20.435 |
| Eliglustat | Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study | Social Functioning: Change at Year 8 (n= 16) | 11.72 units on a scale | Standard Deviation 24.778 |
| Eliglustat | Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study | Social Functioning: Change at Year 9 (n= 4) | 3.13 units on a scale | Standard Deviation 6.25 |
| Eliglustat | Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study | Social Functioning: Change at End of Study (n= 19) | 9.21 units on a scale | Standard Deviation 25.291 |
| Eliglustat | Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study | Role - Emotional: Change at Year 1 (n= 22) | -3.03 units on a scale | Standard Deviation 17.733 |
| Eliglustat | Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study | Role - Emotional: Change at Year 2 (n= 20) | 4.58 units on a scale | Standard Deviation 20.675 |
| Eliglustat | Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study | Role - Emotional: Change at Year 4 (n= 19) | 3.95 units on a scale | Standard Deviation 18.916 |
| Eliglustat | Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study | Role - Emotional: Change at Year 6 (n= 19) | 3.07 units on a scale | Standard Deviation 23.11 |
| Eliglustat | Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study | Role - Emotional: Change at Year 7 (n= 19) | 0.44 units on a scale | Standard Deviation 23.485 |
| Eliglustat | Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study | Role - Emotional: Change at Year 8 (n= 16) | 5.21 units on a scale | Standard Deviation 19.927 |
| Eliglustat | Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study | Role - Emotional: Change at Year 9 (n= 4) | -10.42 units on a scale | Standard Deviation 12.501 |
| Eliglustat | Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study | Role- Emotional: Change at End of study (n= 19) | 1.32 units on a scale | Standard Deviation 22.952 |
| Eliglustat | Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study | Mental Health: Change at Year 1 (n= 22) | 0.23 units on a scale | Standard Deviation 17.827 |
| Eliglustat | Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study | Mental Health: Change at Year 2 (n= 20) | 1.75 units on a scale | Standard Deviation 19.485 |
| Eliglustat | Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study | Mental Health: Change at Year 3 (n=17) | 7.65 units on a scale | Standard Deviation 16.117 |
| Eliglustat | Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study | Mental Health: Change at Year 4 (n= 19) | 4.21 units on a scale | Standard Deviation 19.809 |
| Eliglustat | Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study | Mental Health: Change at Year 5 (n= 19) | 6.84 units on a scale | Standard Deviation 19.945 |
| Eliglustat | Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study | Mental Health: Change at Year 6 (n= 19) | 5.53 units on a scale | Standard Deviation 22.785 |
| Eliglustat | Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study | Mental Health: Change at Year 7 (n= 19) | 6.05 units on a scale | Standard Deviation 21.186 |
| Eliglustat | Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study | Mental Health: Change at Year 8 (n= 16) | 7.66 units on a scale | Standard Deviation 19.989 |
| Eliglustat | Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study | Mental Health: Change at Year 9 (n= 4) | 3.75 units on a scale | Standard Deviation 4.787 |
| Eliglustat | Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study | Mental Health: Change at End of Study (n=19) | 8.68 units on a scale | Standard Deviation 20.196 |
| Eliglustat | Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study | Physical Component Summary:Change at Year 1(n= 22) | 3.58 units on a scale | Standard Deviation 6.263 |
| Eliglustat | Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study | Physical Component Summary:Change at Year 2(n= 20) | 3.81 units on a scale | Standard Deviation 7.968 |
| Eliglustat | Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study | Physical Component Summary:Change at Year 3(n= 17) | 5.17 units on a scale | Standard Deviation 7.044 |
| Eliglustat | Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study | Physical Component Summary:Change at Year 4(n= 19) | 3.69 units on a scale | Standard Deviation 8.195 |
| Eliglustat | Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study | Physical Component Summary:Change at Year 5(n= 19) | 3.75 units on a scale | Standard Deviation 8.43 |
| Eliglustat | Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study | Physical Component Summary:Change at Year 6(n= 19) | 3.88 units on a scale | Standard Deviation 6.038 |
| Eliglustat | Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study | Physical Component Summary:Change at Year 7(n= 19) | 4.57 units on a scale | Standard Deviation 7.892 |
| Eliglustat | Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study | Physical Component Summary:Change at Year 8(n= 16) | 4.36 units on a scale | Standard Deviation 6.946 |
| Eliglustat | Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study | Physical Component Summary: Change at Year 9 (n=4) | 1.98 units on a scale | Standard Deviation 5.029 |
| Eliglustat | Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study | Physical Component Summary: Change at EOS (n= 19) | 4.70 units on a scale | Standard Deviation 7.12 |
| Eliglustat | Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study | Mental Component Summary: Change at Year 1 (n= 22) | -1.03 units on a scale | Standard Deviation 8.703 |
| Eliglustat | Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study | Mental Component Summary: Change at Year 2 (n= 20) | 2.04 units on a scale | Standard Deviation 10.476 |
| Eliglustat | Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study | Mental Component Summary: Change at Year 3 (n= 17) | 3.45 units on a scale | Standard Deviation 7.881 |
| Eliglustat | Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study | Mental Component Summary: Change at Year 5 (n= 19) | 3.50 units on a scale | Standard Deviation 10.963 |
| Eliglustat | Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study | Mental Component Summary: Change at Year 6 (n= 19) | 3.03 units on a scale | Standard Deviation 12.535 |
| Eliglustat | Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study | Mental Component Summary: Change at Year 7 (n= 19) | 1.94 units on a scale | Standard Deviation 10.982 |
| Eliglustat | Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study | Mental Component Summary: Change at Year 8 (n= 16) | 4.59 units on a scale | Standard Deviation 10.106 |
| Eliglustat | Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study | Mental Component Summary: Change at Year 9 (n= 4) | -0.52 units on a scale | Standard Deviation 1.287 |
| Eliglustat | Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study | Mental Component Summary: Change at EOS (n=19) | 3.73 units on a scale | Standard Deviation 11.243 |
| Eliglustat | Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study | Role - Emotional: Change at Year 3 (n= 17) | 2.45 units on a scale | Standard Deviation 16.342 |
| Eliglustat | Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study | Role - Emotional: Change at Year 5 (n= 19) | 2.19 units on a scale | Standard Deviation 22.02 |
| Eliglustat | Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study | Mental Component Summary: Change at Year 4 (n= 19) | 2.11 units on a scale | Standard Deviation 10.013 |
Secondary
Change From Baseline in Fatigue Severity Scale (FSS) Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study
The FSS is an instrument consisting of 9 self-administered questions that measures the impact of severity of fatigue symptoms on everyday functioning, based on the recall over the past week. Score range for each question ranges from 1 (minimum) to 7 (maximum), where higher score indicates greater severity. FSS total score was calculated by averaging the results of all questions. Total FSS score ranges from 9 (minimum) to 63 (maximum), where higher scores indicates greater severity.
Time frame: Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study (Up to Year 9)
Population: Full analysis set consists of all participants who signed informed consent and received at least one dose of eliglustat. Here 'n' signifies number of participants with available data at specified time points.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Eliglustat | Change From Baseline in Fatigue Severity Scale (FSS) Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Change at Year 1 (n=17) | -0.56 units on a scale | Standard Deviation 1.169 |
| Eliglustat | Change From Baseline in Fatigue Severity Scale (FSS) Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Change at Year 2 (n=16) | -0.63 units on a scale | Standard Deviation 1.388 |
| Eliglustat | Change From Baseline in Fatigue Severity Scale (FSS) Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Change at Year 3 (n=14) | -1.37 units on a scale | Standard Deviation 1.983 |
| Eliglustat | Change From Baseline in Fatigue Severity Scale (FSS) Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Change at Year 4 (n=16) | -1.41 units on a scale | Standard Deviation 1.574 |
| Eliglustat | Change From Baseline in Fatigue Severity Scale (FSS) Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Change at Year 5 (n=16) | -1.26 units on a scale | Standard Deviation 1.418 |
| Eliglustat | Change From Baseline in Fatigue Severity Scale (FSS) Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Change at Year 6 (n=15) | -1.21 units on a scale | Standard Deviation 1.681 |
| Eliglustat | Change From Baseline in Fatigue Severity Scale (FSS) Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Change at Year 8 (n=13) | -1.22 units on a scale | Standard Deviation 1.489 |
| Eliglustat | Change From Baseline in Fatigue Severity Scale (FSS) Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Change at Year 9 (n=4) | -1.20 units on a scale | Standard Deviation 1.192 |
| Eliglustat | Change From Baseline in Fatigue Severity Scale (FSS) Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Change at End of Study (n=16) | -1.16 units on a scale | Standard Deviation 1.665 |
| Eliglustat | Change From Baseline in Fatigue Severity Scale (FSS) Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Change at Year 7 (n=16) | -0.97 units on a scale | Standard Deviation 1.429 |
Secondary
Lumbar Spine and Femur T-Scores for Bone Mineral Density (BMD) at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study
Images of the lumbar spine and femur were obtained by dual energy X-ray absorptiometry (DXA) to determine T-score for each bone area and total bone mineral density. T-scores compares participant's bone density with that of healthy young participant of same gender. The T-score bone density categories were: normal (score \>-1), osteopenia (score -2.5 to \<=-1), and osteoporosis (score \<= -2.5).
Time frame: Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study (up to Year 9)
Population: Full analysis set consists of all participants who signed informed consent and received at least one dose of eliglustat. Here 'n' signifies number of participants with available data at specified time points.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Eliglustat | Lumbar Spine and Femur T-Scores for Bone Mineral Density (BMD) at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Lumbar Spine T-Score: Baseline (n= 25) | -1.85 T-Score | Standard Deviation 1.094 |
| Eliglustat | Lumbar Spine and Femur T-Scores for Bone Mineral Density (BMD) at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Lumbar Spine T-Score: Year 1 (n= 20) | -1.43 T-Score | Standard Deviation 1.015 |
| Eliglustat | Lumbar Spine and Femur T-Scores for Bone Mineral Density (BMD) at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Lumbar Spine T-Score: Year 2 (n= 17) | -0.93 T-Score | Standard Deviation 1.268 |
| Eliglustat | Lumbar Spine and Femur T-Scores for Bone Mineral Density (BMD) at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Lumbar Spine T-Score: Year 3 (n= 15) | -1.09 T-Score | Standard Deviation 1.143 |
| Eliglustat | Lumbar Spine and Femur T-Scores for Bone Mineral Density (BMD) at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Lumbar Spine T-Score: Year 4 (n= 15) | -0.88 T-Score | Standard Deviation 1.258 |
| Eliglustat | Lumbar Spine and Femur T-Scores for Bone Mineral Density (BMD) at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Lumbar Spine T-Score: Year 5 (n= 15) | -0.79 T-Score | Standard Deviation 1.116 |
| Eliglustat | Lumbar Spine and Femur T-Scores for Bone Mineral Density (BMD) at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Lumbar Spine T-Score: Year 6 (n= 15) | -0.69 T-Score | Standard Deviation 1.313 |
| Eliglustat | Lumbar Spine and Femur T-Scores for Bone Mineral Density (BMD) at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Lumbar Spine T-Score: Year 7 (n= 15) | -0.59 T-Score | Standard Deviation 1.265 |
| Eliglustat | Lumbar Spine and Femur T-Scores for Bone Mineral Density (BMD) at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Lumbar Spine T-Score: Year 8 (n= 14) | -0.59 T-Score | Standard Deviation 1.294 |
| Eliglustat | Lumbar Spine and Femur T-Scores for Bone Mineral Density (BMD) at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Lumbar Spine T-Score: Year 9 (n= 4) | 0.00 T-Score | Standard Deviation 0.753 |
| Eliglustat | Lumbar Spine and Femur T-Scores for Bone Mineral Density (BMD) at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Lumbar Spine T-Score: End of Study (n= 6) | -0.35 T-Score | Standard Deviation 1.613 |
| Eliglustat | Lumbar Spine and Femur T-Scores for Bone Mineral Density (BMD) at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Femur T-Scores: Baseline (n= 23) | -0.47 T-Score | Standard Deviation 0.894 |
| Eliglustat | Lumbar Spine and Femur T-Scores for Bone Mineral Density (BMD) at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Femur T-Scores: Year 1 (n= 19) | -0.24 T-Score | Standard Deviation 0.954 |
| Eliglustat | Lumbar Spine and Femur T-Scores for Bone Mineral Density (BMD) at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Femur T-Scores: Year 2 (n= 15) | -0.09 T-Score | Standard Deviation 0.991 |
| Eliglustat | Lumbar Spine and Femur T-Scores for Bone Mineral Density (BMD) at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Femur T-Scores: Year 3 (n= 13) | 0.16 T-Score | Standard Deviation 0.987 |
| Eliglustat | Lumbar Spine and Femur T-Scores for Bone Mineral Density (BMD) at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Femur T-Scores: Year 4 (n= 13) | 0.13 T-Score | Standard Deviation 1.044 |
| Eliglustat | Lumbar Spine and Femur T-Scores for Bone Mineral Density (BMD) at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Femur T-Scores: Year 5 (n= 13) | 0.14 T-Score | Standard Deviation 1.016 |
| Eliglustat | Lumbar Spine and Femur T-Scores for Bone Mineral Density (BMD) at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Femur T-Scores: Year 6 (n= 13) | 0.19 T-Score | Standard Deviation 0.946 |
| Eliglustat | Lumbar Spine and Femur T-Scores for Bone Mineral Density (BMD) at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Femur T-Scores: Year 7 (n= 13) | 0.12 T-Score | Standard Deviation 1.021 |
| Eliglustat | Lumbar Spine and Femur T-Scores for Bone Mineral Density (BMD) at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Femur T-Scores: Year 8 (n= 12) | 0.33 T-Score | Standard Deviation 0.907 |
| Eliglustat | Lumbar Spine and Femur T-Scores for Bone Mineral Density (BMD) at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Femur T-Scores: Year 9 (n= 4) | 0.33 T-Score | Standard Deviation 0.618 |
| Eliglustat | Lumbar Spine and Femur T-Scores for Bone Mineral Density (BMD) at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Femur T-Scores: End of Study (n= 6) | 0.18 T-Score | Standard Deviation 1.278 |
Secondary
Lumbar Spine and Femur Z-Scores for BMD at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study
Images of the lumbar spine and femur were obtained by DXA to determine Z-score for each bone area and total bone mineral density. The Z-score bone density categories are: normal (score \>-2) and below normal (score \<=-2).
Time frame: Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study (up to Year 9)
Population: Full analysis set consists of all participants who signed informed consent and received at least one dose of eliglustat. Here 'n' signifies number of participants with available data at specified time points.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Eliglustat | Lumbar Spine and Femur Z-Scores for BMD at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Lumbar Spine Z-Score: Baseline (n= 25) | -1.49 Z-Score | Standard Deviation 1.055 |
| Eliglustat | Lumbar Spine and Femur Z-Scores for BMD at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Lumbar Spine Z-Score: Year 1 (n= 20) | -1.11 Z-Score | Standard Deviation 0.961 |
| Eliglustat | Lumbar Spine and Femur Z-Scores for BMD at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Lumbar Spine Z-Score: Year 2 (n= 17) | -0.64 Z-Score | Standard Deviation 0.999 |
| Eliglustat | Lumbar Spine and Femur Z-Scores for BMD at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Lumbar Spine Z-Score: Year 3 (n= 15) | -0.63 Z-Score | Standard Deviation 0.984 |
| Eliglustat | Lumbar Spine and Femur Z-Scores for BMD at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Lumbar Spine Z-Score: Year 4 (n= 15) | -0.48 Z-Score | Standard Deviation 1.073 |
| Eliglustat | Lumbar Spine and Femur Z-Scores for BMD at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Lumbar Spine Z-Score: Year 5 (n= 15) | -0.37 Z-Score | Standard Deviation 0.932 |
| Eliglustat | Lumbar Spine and Femur Z-Scores for BMD at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Lumbar Spine Z-Score: Year 6 (n= 15) | -0.27 Z-Score | Standard Deviation 1.1 |
| Eliglustat | Lumbar Spine and Femur Z-Scores for BMD at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Lumbar Spine Z-Score: Year 7 (n= 15) | -0.14 Z-Score | Standard Deviation 1.053 |
| Eliglustat | Lumbar Spine and Femur Z-Scores for BMD at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Lumbar Spine Z-Score: Year 8 (n= 14) | -0.29 Z-Score | Standard Deviation 1.088 |
| Eliglustat | Lumbar Spine and Femur Z-Scores for BMD at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Lumbar Spine Z-Score: Year 9 (n= 4) | -0.05 Z-Score | Standard Deviation 0.885 |
| Eliglustat | Lumbar Spine and Femur Z-Scores for BMD at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Lumbar Spine Z-Score: End of Study (n= 6) | 0.18 Z-Score | Standard Deviation 0.968 |
| Eliglustat | Lumbar Spine and Femur Z-Scores for BMD at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Femur Z-Scores: Baseline (n= 23) | -0.17 Z-Score | Standard Deviation 0.783 |
| Eliglustat | Lumbar Spine and Femur Z-Scores for BMD at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Femur Z-Scores: Year 1 (n= 19) | 0.04 Z-Score | Standard Deviation 0.813 |
| Eliglustat | Lumbar Spine and Femur Z-Scores for BMD at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Femur Z-Scores: Year 2 (n= 15) | 0.25 Z-Score | Standard Deviation 0.787 |
| Eliglustat | Lumbar Spine and Femur Z-Scores for BMD at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Femur Z-Scores: Year 3 (n= 13) | 0.52 Z-Score | Standard Deviation 0.704 |
| Eliglustat | Lumbar Spine and Femur Z-Scores for BMD at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Femur Z-Scores: Year 4 (n= 13) | 0.48 Z-Score | Standard Deviation 0.773 |
| Eliglustat | Lumbar Spine and Femur Z-Scores for BMD at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Femur Z-Scores: Year 5 (n= 13) | 0.49 Z-Score | Standard Deviation 0.741 |
| Eliglustat | Lumbar Spine and Femur Z-Scores for BMD at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Femur Z-Scores: Year 6 (n= 13) | 0.57 Z-Score | Standard Deviation 0.665 |
| Eliglustat | Lumbar Spine and Femur Z-Scores for BMD at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Femur Z-Scores: Year 7 (n= 13) | 0.52 Z-Score | Standard Deviation 0.683 |
| Eliglustat | Lumbar Spine and Femur Z-Scores for BMD at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Femur Z-Scores: Year 8 (n= 12) | 0.64 Z-Score | Standard Deviation 0.693 |
| Eliglustat | Lumbar Spine and Femur Z-Scores for BMD at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Femur Z-Scores: Year 9 (n= 4) | 0.38 Z-Score | Standard Deviation 0.793 |
| Eliglustat | Lumbar Spine and Femur Z-Scores for BMD at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Femur Z-Scores: End of Study (n= 6) | 0.73 Z-Score | Standard Deviation 0.766 |
Secondary
Number of Participants With Bone Pain Levels During the Past 4 Weeks at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study
Bone pain was assessed as a part of Gaucher disease assessment in participants. Participants were categorized as none (no bone pain), very mild bone pain, mild bone pain and moderate bone pain. In this outcome, number of participants with different levels of bone pain at specified time points were reported.
Time frame: Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study (Up to Year 9)
Population: Full analysis set consists of all participants who signed informed consent and received at least one dose of eliglustat. Here 'n' signifies number of participants with available data at specified time points.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Eliglustat | Number of Participants With Bone Pain Levels During the Past 4 Weeks at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | None: Baseline (n=26) | 23 participants |
| Eliglustat | Number of Participants With Bone Pain Levels During the Past 4 Weeks at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | None: Year 1 (n=22) | 17 participants |
| Eliglustat | Number of Participants With Bone Pain Levels During the Past 4 Weeks at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | None: Year 2 (n=20) | 17 participants |
| Eliglustat | Number of Participants With Bone Pain Levels During the Past 4 Weeks at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | None: Year 3 (n=19) | 14 participants |
| Eliglustat | Number of Participants With Bone Pain Levels During the Past 4 Weeks at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | None: Year 4 (n=19) | 14 participants |
| Eliglustat | Number of Participants With Bone Pain Levels During the Past 4 Weeks at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | None: Year 5 (n=19) | 16 participants |
| Eliglustat | Number of Participants With Bone Pain Levels During the Past 4 Weeks at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | None: Year 6 (n=19) | 17 participants |
| Eliglustat | Number of Participants With Bone Pain Levels During the Past 4 Weeks at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | None: Year 7 (n=19) | 15 participants |
| Eliglustat | Number of Participants With Bone Pain Levels During the Past 4 Weeks at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | None: Year 8 (n=16) | 14 participants |
| Eliglustat | Number of Participants With Bone Pain Levels During the Past 4 Weeks at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | None: Year 9 (n=4) | 4 participants |
| Eliglustat | Number of Participants With Bone Pain Levels During the Past 4 Weeks at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | None: End of Study (n=19) | 18 participants |
| Eliglustat | Number of Participants With Bone Pain Levels During the Past 4 Weeks at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Very Mild: Baseline (n=26) | 2 participants |
| Eliglustat | Number of Participants With Bone Pain Levels During the Past 4 Weeks at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Very Mild: Year 1 (n=22) | 4 participants |
| Eliglustat | Number of Participants With Bone Pain Levels During the Past 4 Weeks at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Very Mild: Year 2 (n=20) | 3 participants |
| Eliglustat | Number of Participants With Bone Pain Levels During the Past 4 Weeks at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Very Mild: Year 3 (n=19) | 3 participants |
| Eliglustat | Number of Participants With Bone Pain Levels During the Past 4 Weeks at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Very Mild: Year 4 (n=19) | 4 participants |
| Eliglustat | Number of Participants With Bone Pain Levels During the Past 4 Weeks at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Very Mild: Year 5 (n=19) | 1 participants |
| Eliglustat | Number of Participants With Bone Pain Levels During the Past 4 Weeks at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Very Mild: Year 6 (n=19) | 1 participants |
| Eliglustat | Number of Participants With Bone Pain Levels During the Past 4 Weeks at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Very Mild: Year 7 (n=19) | 3 participants |
| Eliglustat | Number of Participants With Bone Pain Levels During the Past 4 Weeks at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Very Mild: Year 8 (n=16) | 1 participants |
| Eliglustat | Number of Participants With Bone Pain Levels During the Past 4 Weeks at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Very Mild: Year 9 (n=4) | 0 participants |
| Eliglustat | Number of Participants With Bone Pain Levels During the Past 4 Weeks at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Very Mild: End of Study (n=19) | 1 participants |
| Eliglustat | Number of Participants With Bone Pain Levels During the Past 4 Weeks at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Mild: Baseline (n= 26) | 1 participants |
| Eliglustat | Number of Participants With Bone Pain Levels During the Past 4 Weeks at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Mild: Year 1 (n= 22) | 1 participants |
| Eliglustat | Number of Participants With Bone Pain Levels During the Past 4 Weeks at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Mild: Year 2 (n= 20) | 0 participants |
| Eliglustat | Number of Participants With Bone Pain Levels During the Past 4 Weeks at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Mild: Year 3 (n=19) | 2 participants |
| Eliglustat | Number of Participants With Bone Pain Levels During the Past 4 Weeks at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Mild: Year 4 (n=19) | 1 participants |
| Eliglustat | Number of Participants With Bone Pain Levels During the Past 4 Weeks at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Mild: Year 5 (n=19) | 2 participants |
| Eliglustat | Number of Participants With Bone Pain Levels During the Past 4 Weeks at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Mild: Year 6 (n=19) | 1 participants |
| Eliglustat | Number of Participants With Bone Pain Levels During the Past 4 Weeks at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Mild: Year 7 (n=19) | 1 participants |
| Eliglustat | Number of Participants With Bone Pain Levels During the Past 4 Weeks at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Mild: Year 8 (n=16) | 1 participants |
| Eliglustat | Number of Participants With Bone Pain Levels During the Past 4 Weeks at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Mild: Year 9 (n=4) | 0 participants |
| Eliglustat | Number of Participants With Bone Pain Levels During the Past 4 Weeks at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Mild: End of Study (n= 19) | 0 participants |
| Eliglustat | Number of Participants With Bone Pain Levels During the Past 4 Weeks at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Moderate: Baseline (n=26) | 0 participants |
| Eliglustat | Number of Participants With Bone Pain Levels During the Past 4 Weeks at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Moderate: Year 1 (n=22) | 0 participants |
| Eliglustat | Number of Participants With Bone Pain Levels During the Past 4 Weeks at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Moderate: Year 2 (n=20) | 0 participants |
| Eliglustat | Number of Participants With Bone Pain Levels During the Past 4 Weeks at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Moderate: Year 3 (n=19) | 0 participants |
| Eliglustat | Number of Participants With Bone Pain Levels During the Past 4 Weeks at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Moderate: Year 4 (n=19) | 0 participants |
| Eliglustat | Number of Participants With Bone Pain Levels During the Past 4 Weeks at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Moderate: Year 5 (n=19) | 0 participants |
| Eliglustat | Number of Participants With Bone Pain Levels During the Past 4 Weeks at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Moderate: Year 6 (n=19) | 0 participants |
| Eliglustat | Number of Participants With Bone Pain Levels During the Past 4 Weeks at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Moderate: Year 7 (n=19) | 0 participants |
| Eliglustat | Number of Participants With Bone Pain Levels During the Past 4 Weeks at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Moderate: Year 8 (n=16) | 0 participants |
| Eliglustat | Number of Participants With Bone Pain Levels During the Past 4 Weeks at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Moderate: Year 9 (n=4) | 0 participants |
| Eliglustat | Number of Participants With Bone Pain Levels During the Past 4 Weeks at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Moderate: End of Study (n=19) | 0 participants |
Secondary
Number of Participants With Mobility Status (MS) at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study
Mobillity, i.e. ability to walk was assessed as a part of Gaucher disease assessment in participants.In this outcome, number of participants with their different mobility status (unrestricted mobility, walks with difficulty) at specified time points were reported.
Time frame: Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study (Up to Year 9)
Population: Full analysis set consists of all participants who signed informed consent and received at least one dose of eliglustat. Here 'n' signifies number of participants with available data at specified time points.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Eliglustat | Number of Participants With Mobility Status (MS) at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | MS: Unrestricted Mobility: Baseline (n=26) | 24 participants |
| Eliglustat | Number of Participants With Mobility Status (MS) at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | MS: Unrestricted Mobility: Year 1 (n=22) | 22 participants |
| Eliglustat | Number of Participants With Mobility Status (MS) at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | MS: Unrestricted Mobility: Year 2 (n=20) | 20 participants |
| Eliglustat | Number of Participants With Mobility Status (MS) at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | MS: Unrestricted Mobility: Year 3 (n=19) | 19 participants |
| Eliglustat | Number of Participants With Mobility Status (MS) at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | MS: Unrestricted Mobility: Year 4 (n=19) | 19 participants |
| Eliglustat | Number of Participants With Mobility Status (MS) at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | MS: Unrestricted Mobility: Year 5 (n=19) | 19 participants |
| Eliglustat | Number of Participants With Mobility Status (MS) at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | MS: Unrestricted Mobility: Year 6 (n=19) | 19 participants |
| Eliglustat | Number of Participants With Mobility Status (MS) at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | MS: Unrestricted Mobility: Year 7 (n=19) | 19 participants |
| Eliglustat | Number of Participants With Mobility Status (MS) at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | MS: Unrestricted Mobility: Year 8 (n=16) | 16 participants |
| Eliglustat | Number of Participants With Mobility Status (MS) at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | MS: Unrestricted Mobility: Year 9 (n=4) | 4 participants |
| Eliglustat | Number of Participants With Mobility Status (MS) at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | MS: Unrestricted Mobility: End of Study (n=19) | 18 participants |
| Eliglustat | Number of Participants With Mobility Status (MS) at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | MS: Walks with Difficulty: Baseline (n=26) | 2 participants |
| Eliglustat | Number of Participants With Mobility Status (MS) at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | MS: Walks with Difficulty: Year 1 (n=22) | 0 participants |
| Eliglustat | Number of Participants With Mobility Status (MS) at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | MS: Walks with Difficulty: Year 2 (n=20) | 0 participants |
| Eliglustat | Number of Participants With Mobility Status (MS) at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | MS: Walks with Difficulty: Year 3 (n=19) | 0 participants |
| Eliglustat | Number of Participants With Mobility Status (MS) at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | MS: Walks with Difficulty: Year 4 (n=19) | 0 participants |
| Eliglustat | Number of Participants With Mobility Status (MS) at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | MS: Walks with Difficulty: Year 5 (n=19) | 0 participants |
| Eliglustat | Number of Participants With Mobility Status (MS) at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | MS: Walks with Difficulty: Year 6 (n=19) | 0 participants |
| Eliglustat | Number of Participants With Mobility Status (MS) at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | MS: Walks with Difficulty: Year 7 (n=19) | 0 participants |
| Eliglustat | Number of Participants With Mobility Status (MS) at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | MS: Walks with Difficulty: Year 8 (n=16) | 0 participants |
| Eliglustat | Number of Participants With Mobility Status (MS) at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | MS: Walks with Difficulty: End of Study (n=19) | 1 participants |
| Eliglustat | Number of Participants With Mobility Status (MS) at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | MS: Walks with Difficulty: Year 9 (n=4) | 0 participants |
Secondary
Number of Participants With No Bone Crisis at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study
Bone crisis was assessed as a part of Gaucher disease assessment in participants. Acute, excruciating episodic bone pain is characteristic of Gaucher bone crisis, which typically causes debilitation lasting several days or longer and requires treatment with immobilization, hydration, and opioid analgesics. Participants were categorized as 0= no bone crisis, 1= 1 bone crisis, and 2= 2 bone crises during the assessment period. In this outcome, number of participants with 0= no bone crises levels at specified time points were reported.
Time frame: Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study (Up to Year 9)
Population: Full analysis set consists of all participants who signed informed consent and received at least one dose of eliglustat. Here 'n' signifies number of participants with available data at specified time points.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Eliglustat | Number of Participants With No Bone Crisis at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Baseline (n=26) | 26 participants |
| Eliglustat | Number of Participants With No Bone Crisis at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Year 1 (n=22) | 22 participants |
| Eliglustat | Number of Participants With No Bone Crisis at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Year 2 (n=20) | 20 participants |
| Eliglustat | Number of Participants With No Bone Crisis at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Year 3 (n=19) | 19 participants |
| Eliglustat | Number of Participants With No Bone Crisis at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Year 4 (n=19) | 19 participants |
| Eliglustat | Number of Participants With No Bone Crisis at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Year 5 (n=19) | 19 participants |
| Eliglustat | Number of Participants With No Bone Crisis at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Year 6 (n=19) | 19 participants |
| Eliglustat | Number of Participants With No Bone Crisis at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Year 7 (n=19) | 19 participants |
| Eliglustat | Number of Participants With No Bone Crisis at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Year 8 (n=16) | 16 participants |
| Eliglustat | Number of Participants With No Bone Crisis at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Year 9 (n=4) | 4 participants |
| Eliglustat | Number of Participants With No Bone Crisis at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | End of Study (n=19) | 19 participants |
Secondary
Percent Change From Baseline in Biomarker (Angiotensin Converting Enzyme) Level at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study
Time frame: Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and End of Study (Up to Year 9)
Population: Full analysis set consists of all participants who signed informed consent and received at least one dose of eliglustat. Here 'n' signifies number of participants with available data at specified time point.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Eliglustat | Percent Change From Baseline in Biomarker (Angiotensin Converting Enzyme) Level at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Change at Year 1 (n=22) | -35.1 percent change | Standard Deviation 18.87 |
| Eliglustat | Percent Change From Baseline in Biomarker (Angiotensin Converting Enzyme) Level at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Change at Year 2 (n=20) | -53.5 percent change | Standard Deviation 21.05 |
| Eliglustat | Percent Change From Baseline in Biomarker (Angiotensin Converting Enzyme) Level at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Change at Year 3 (n=19) | -56.7 percent change | Standard Deviation 19.33 |
| Eliglustat | Percent Change From Baseline in Biomarker (Angiotensin Converting Enzyme) Level at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Change at Year 4 (n=18) | -60.7 percent change | Standard Deviation 18.44 |
| Eliglustat | Percent Change From Baseline in Biomarker (Angiotensin Converting Enzyme) Level at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Change at Year 5 (n=19) | -55.7 percent change | Standard Deviation 22.51 |
| Eliglustat | Percent Change From Baseline in Biomarker (Angiotensin Converting Enzyme) Level at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Change at Year 6 (n=17) | -61.5 percent change | Standard Deviation 24.73 |
| Eliglustat | Percent Change From Baseline in Biomarker (Angiotensin Converting Enzyme) Level at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Change at Year 7 (n=19) | -63.6 percent change | Standard Deviation 24.64 |
| Eliglustat | Percent Change From Baseline in Biomarker (Angiotensin Converting Enzyme) Level at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Change at Year 8 (n=16) | -59.2 percent change | Standard Deviation 21.77 |
| Eliglustat | Percent Change From Baseline in Biomarker (Angiotensin Converting Enzyme) Level at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Change at Year 9 (n=4) | -55.1 percent change | Standard Deviation 32.58 |
| Eliglustat | Percent Change From Baseline in Biomarker (Angiotensin Converting Enzyme) Level at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Change at End of Study (n=18) | -64.7 percent change | Standard Deviation 23.38 |
Secondary
Percent Change From Baseline in Biomarker Chemokine Ligand 18 (CCL18) Level at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and End of Study
Time frame: Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and End of Study (Up to Year 9)
Population: Full analysis set consists of all participants who signed informed consent and received at least one dose of eliglustat. Here 'n' signifies number of participants with available data at specified time points.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Eliglustat | Percent Change From Baseline in Biomarker Chemokine Ligand 18 (CCL18) Level at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and End of Study | Change at Year 1 (n=21) | -49.0 percent change | Standard Deviation 24.89 |
| Eliglustat | Percent Change From Baseline in Biomarker Chemokine Ligand 18 (CCL18) Level at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and End of Study | Change at Year 2 (n=15) | -72.1 percent change | Standard Deviation 19.11 |
| Eliglustat | Percent Change From Baseline in Biomarker Chemokine Ligand 18 (CCL18) Level at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and End of Study | Change at Year 3 (n=18) | -68.6 percent change | Standard Deviation 17.27 |
| Eliglustat | Percent Change From Baseline in Biomarker Chemokine Ligand 18 (CCL18) Level at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and End of Study | Change at Year 4 (n=18) | -80.2 percent change | Standard Deviation 12.71 |
| Eliglustat | Percent Change From Baseline in Biomarker Chemokine Ligand 18 (CCL18) Level at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and End of Study | Change at Year 5 (n=18) | -89.4 percent change | Standard Deviation 8.48 |
| Eliglustat | Percent Change From Baseline in Biomarker Chemokine Ligand 18 (CCL18) Level at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and End of Study | Change at Year 6 (n=18) | -82.7 percent change | Standard Deviation 11.98 |
| Eliglustat | Percent Change From Baseline in Biomarker Chemokine Ligand 18 (CCL18) Level at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and End of Study | Change at Year 7 (n=18) | -78.5 percent change | Standard Deviation 17.83 |
| Eliglustat | Percent Change From Baseline in Biomarker Chemokine Ligand 18 (CCL18) Level at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and End of Study | Change at Year 8 (n=16) | -79.8 percent change | Standard Deviation 16.19 |
| Eliglustat | Percent Change From Baseline in Biomarker Chemokine Ligand 18 (CCL18) Level at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and End of Study | Change at Year 9 (n=4) | -88.6 percent change | Standard Deviation 6.3 |
| Eliglustat | Percent Change From Baseline in Biomarker Chemokine Ligand 18 (CCL18) Level at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and End of Study | Change at End of Study (n=18) | -85.8 percent change | Standard Deviation 12.92 |
Secondary
Percent Change From Baseline in Biomarker (Chitotriosidase) Level at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study
Time frame: Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study (Up to Year 9)
Population: Full analysis set consists of all participants who signed informed consent and received at least one dose of eliglustat. Here 'n' signifies number of participants with available data at specified time points.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Eliglustat | Percent Change From Baseline in Biomarker (Chitotriosidase) Level at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Change at Year 1 (n=20) | -49.9 percent change | Standard Deviation 17.17 |
| Eliglustat | Percent Change From Baseline in Biomarker (Chitotriosidase) Level at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Change at Year 2 (n=18) | -73.5 percent change | Standard Deviation 14.93 |
| Eliglustat | Percent Change From Baseline in Biomarker (Chitotriosidase) Level at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Change at Year 3 (n=17) | -76.4 percent change | Standard Deviation 12.78 |
| Eliglustat | Percent Change From Baseline in Biomarker (Chitotriosidase) Level at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Change at Year 4 (n=17) | -79.2 percent change | Standard Deviation 16.18 |
| Eliglustat | Percent Change From Baseline in Biomarker (Chitotriosidase) Level at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Change at Year 5 (n=17) | -76.7 percent change | Standard Deviation 19.16 |
| Eliglustat | Percent Change From Baseline in Biomarker (Chitotriosidase) Level at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Change at Year 6 (n=17) | -74.1 percent change | Standard Deviation 26.97 |
| Eliglustat | Percent Change From Baseline in Biomarker (Chitotriosidase) Level at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Change at Year 7 (n=17) | -75.5 percent change | Standard Deviation 27.88 |
| Eliglustat | Percent Change From Baseline in Biomarker (Chitotriosidase) Level at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Change at End of Study (n=17) | -69.8 percent change | Standard Deviation 36.59 |
| Eliglustat | Percent Change From Baseline in Biomarker (Chitotriosidase) Level at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Change at Year 8 (n=14) | -72.5 percent change | Standard Deviation 29.2 |
| Eliglustat | Percent Change From Baseline in Biomarker (Chitotriosidase) Level at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Change at Year 9 (n=4) | -61.2 percent change | Standard Deviation 36.45 |
Secondary
Percent Change From Baseline in Biomarker (Tartrate-Resistant Acid Phosphatase [TRAP]) Level at Year 1 and Year 2
Time frame: Baseline, Year 1, Year 2
Population: Full analysis set consists of all participants who signed informed consent and received at least one dose of eliglustat. Here 'n' signifies number of participants with available data at specified time points. As per the change in planned analysis, TRAP was not assessed after Year 2.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Eliglustat | Percent Change From Baseline in Biomarker (Tartrate-Resistant Acid Phosphatase [TRAP]) Level at Year 1 and Year 2 | Change at Year 1 (n=22) | -37.0 percent change | Standard Deviation 11.64 |
| Eliglustat | Percent Change From Baseline in Biomarker (Tartrate-Resistant Acid Phosphatase [TRAP]) Level at Year 1 and Year 2 | Change at Year 2 (n= 10) | -52.5 percent change | Standard Deviation 10.61 |
Secondary
Percent Change From Baseline in Liver Volume at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study
Percent change in liver volume = (\[liver volume at specified time points minus liver volume at baseline\] divided by \[liver volume at baseline\]) multiplied by 100, where all volumes are in multiples of normal.
Time frame: Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study (Up to Year 9)
Population: Full analysis set consists of all participants who signed informed consent and received at least one dose of eliglustat. Here 'n' signifies number of participants with available data at specified time points.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Eliglustat | Percent Change From Baseline in Liver Volume at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Change at Year 1 (n=22) | -16.9 percent change | Standard Deviation 10.48 |
| Eliglustat | Percent Change From Baseline in Liver Volume at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Change at Year 2 (n=20) | -23.9 percent change | Standard Deviation 12.81 |
| Eliglustat | Percent Change From Baseline in Liver Volume at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Change at Year 3 (n=19) | -26.8 percent change | Standard Deviation 12.28 |
| Eliglustat | Percent Change From Baseline in Liver Volume at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Change at Year 4 (n=18) | -28.0 percent change | Standard Deviation 13.8 |
| Eliglustat | Percent Change From Baseline in Liver Volume at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Change at Year 5 (n=19) | -31.2 percent change | Standard Deviation 13.9 |
| Eliglustat | Percent Change From Baseline in Liver Volume at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Change at Year 6 (n=19) | -28.4 percent change | Standard Deviation 23.58 |
| Eliglustat | Percent Change From Baseline in Liver Volume at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Change at Year 7 (n=19) | -36.2 percent change | Standard Deviation 13.49 |
| Eliglustat | Percent Change From Baseline in Liver Volume at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Change at Year 8 (n=15) | -31.1 percent change | Standard Deviation 13.51 |
| Eliglustat | Percent Change From Baseline in Liver Volume at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Change at Year 9 (n=4) | -22.0 percent change | Standard Deviation 18.49 |
| Eliglustat | Percent Change From Baseline in Liver Volume at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Change at End of Study (n=19) | -36.4 percent change | Standard Deviation 14.91 |
Secondary
Percent Change From Baseline in Platelet Count at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study
Percent change in platelet count = (\[platelet count at specified time points minus platelet count at baseline\] divided by \[platelet count at baseline\]) multiplied by 100.
Time frame: Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study (Up to Year 9)
Population: Full analysis set consists of all participants who signed informed consent and received at least one dose of eliglustat. Here 'n' signifies number of participants with available data at specified time points.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Eliglustat | Percent Change From Baseline in Platelet Count at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Change at Year 1 (n=22) | 41.3 percent change | Standard Deviation 36.95 |
| Eliglustat | Percent Change From Baseline in Platelet Count at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Change at Year 2 (n=20) | 81.5 percent change | Standard Deviation 56.01 |
| Eliglustat | Percent Change From Baseline in Platelet Count at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Change at Year 3 (n=18) | 87.9 percent change | Standard Deviation 65.37 |
| Eliglustat | Percent Change From Baseline in Platelet Count at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Change at Year 4 (n=19) | 95.1 percent change | Standard Deviation 89.41 |
| Eliglustat | Percent Change From Baseline in Platelet Count at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Change at Year 5 (n=19) | 90.9 percent change | Standard Deviation 85.42 |
| Eliglustat | Percent Change From Baseline in Platelet Count at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Change at Year 6 (n=17) | 114.3 percent change | Standard Deviation 102.48 |
| Eliglustat | Percent Change From Baseline in Platelet Count at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Change at Year 7 (n=19) | 99.4 percent change | Standard Deviation 97.13 |
| Eliglustat | Percent Change From Baseline in Platelet Count at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Change at Year 8 (n=16) | 109.8 percent change | Standard Deviation 114.73 |
| Eliglustat | Percent Change From Baseline in Platelet Count at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Change at Year 9 (n=3) | 45.4 percent change | Standard Deviation 56.28 |
| Eliglustat | Percent Change From Baseline in Platelet Count at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Change at End of Study (n=18) | 117.5 percent change | Standard Deviation 116.91 |
Secondary
Percent Change From Baseline in Spleen Volume at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study
Percent change in spleen volume = (\[spleen volume at specified time points minus spleen volume at baseline\] divided by \[spleen volume at baseline\]) multiplied by 100, where all volumes are in multiples of normal.
Time frame: Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study (Up to Year 9)
Population: Full analysis set consists of all participants who signed informed consent and received at least one dose of eliglustat. Here 'n' signifies number of participants with available data at specified time points.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Eliglustat | Percent Change From Baseline in Spleen Volume at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Change at Year 1 (n=22) | -38.5 percent change | Standard Deviation 11.41 |
| Eliglustat | Percent Change From Baseline in Spleen Volume at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Change at Year 2 (n=20) | -52.4 percent change | Standard Deviation 10.73 |
| Eliglustat | Percent Change From Baseline in Spleen Volume at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Change at Year 3 (n=19) | -59.1 percent change | Standard Deviation 11.68 |
| Eliglustat | Percent Change From Baseline in Spleen Volume at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Change at Year 4 (n=18) | -62.5 percent change | Standard Deviation 11.63 |
| Eliglustat | Percent Change From Baseline in Spleen Volume at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Change at Year 5 (n=19) | -63.9 percent change | Standard Deviation 14.64 |
| Eliglustat | Percent Change From Baseline in Spleen Volume at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Change at Year 6 (n=19) | -66.2 percent change | Standard Deviation 15.72 |
| Eliglustat | Percent Change From Baseline in Spleen Volume at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Change at Year 7 (n=19) | -67.8 percent change | Standard Deviation 15.65 |
| Eliglustat | Percent Change From Baseline in Spleen Volume at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Change at Year 8 (n=15) | -67.9 percent change | Standard Deviation 17.11 |
| Eliglustat | Percent Change From Baseline in Spleen Volume at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Change at Year 9 (n=4) | -52.6 percent change | Standard Deviation 23.97 |
| Eliglustat | Percent Change From Baseline in Spleen Volume at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study | Change at End of Study (n=19) | -68.6 percent change | Standard Deviation 18.6 |