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| ID | Type | Description | Link |
|---|---|---|---|
| U1111-1166-6190 | Other Identifier | UTN |
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Primary Objective:
Evaluate long term skeletal response to eliglustat in adult participants who successfully completed one of the Phase 2 or Phase 3 eliglustat studies.
Secondary Objective:
Evaluate the safety of eliglustat (by serious adverse event continuous monitoring), the quality of life (Short Form-36 Health Survey [SF-36]) and biomarkers of Gaucher disease type 1 (GD1) (chitotriosidase, plasma glucosylceramide [GL-1] and lyso glucosylceramide [lyso-GL-1]) in adult participants who successfully completed one of the Phase 2 or Phase 3 studies.
Study duration for individual participants was to be of minimum 2 years and up to 4 years, or until commercial eliglustat was available to participants through reimbursement. If after 4 years since the beginning of the study eliglustat was not approved and available to participants in participating country, participants in this country might continue to receive study treatment until eliglustat was available to participants through the compassionate use (expanded access) program or was approved and available through reimbursement, whichever came first.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Eliglustat | Experimental | Participants who completed one of the Phase 2 (GZGD00304 [NCT00358150]) or Phase 3 studies (GZGD02507 [NCT00891202], GZGD02607 [NCT00943111], or GZGD03109 [NCT01074944]) were enrolled in this current (EFC13781) study. Participants who were cytochrome P450 (CYP) 2D6 intermediate metabolizer (IM), extensive metabolizer (EM) and ultra-rapid metabolizers (URM) received eliglustat 84 milligrams (mg) twice daily and participants who were CYP2D6 poor metabolizer (PM) received eliglustat 84 mg once daily, for duration of minimum 2 years (unless early discontinuation occurred) and up to 4 years, or until commercial eliglustat was available to participants through reimbursement or through the compassionate use (expanded access) program. |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Eliglustat, GZ385660 | Drug | Pharmaceutical form: capsule Route of administration: oral |
|
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants With Mobility Status Assessments at Study Baseline, Weeks 52, 104, 156 and 208 | Mobility, i.e., ability to walk was assessed as a part of Gaucher disease assessment in participants. In this outcome measure, number of participants with their different mobility status along with the use of mobility aids (unrestricted mobility, walks with difficulty, walks with orthopaedic aid, requires wheelchair, bedridden) at specified time points were reported. Baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry. | Study Baseline, Weeks 52, 104, 156 and 208 |
| Number of Participants With Bone Pain Levels During the Past 4 Weeks at Study Baseline, Weeks 52, 104, 156 and 208 | Bone pain was assessed as a part of Gaucher disease assessment in participants. Participants were categorized as none (no bone pain), very mild bone pain, mild bone pain, moderate bone pain, severe bone pain and extreme bone pain during the past 4 weeks at each specified visit. In this outcome measure, number of participants with different level of bone pain during the past 4 weeks at specified time points were reported. Baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry. | Study Baseline, Weeks 52, 104, 156 and 208 |
| Number of Participants With Bone Crisis at Study Baseline, Weeks 52, 104, 156 and 208 | Bone crisis was assessed as a part of Gaucher disease assessment in participants. Acute, excruciating episodic bone pain is characteristic of Gaucher bone crisis, which typically causes periosteal elevation, elevated white blood cell count, fever, or debilitation lasting several days or longer and requires treatment with immobilization of the affected area, and opioid analgesics. Participants were categorized as 0= no bone crisis, 1= 1 bone crisis, 2= 2 bone crisis and >=3 = more than 3 bone crisis during the assessment period. In this outcome measure, number of participants with different bone crises levels at specified time points were reported. Baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry. | Study Baseline, Weeks 52, 104, 156 and 208 |
| Measure | Description | Time Frame |
|---|---|---|
| Change From Current Study Baseline in Gaucher Disease Type 1 (GD1) Biomarker Levels: Chitotriosidase at Week 26, 52, 78, 104, 130, 156, 182, 208 and 234 | Chitotriosidase biomarker was assayed from plasma. Chitotriosidase biomarker levels for participants who were CYP2D6 non-Ultra Rapid Metabolizers (non-URM) was reported in this outcome measure. For this outcome measure, baseline refers to the study baseline, which was defined as status at study entry. |
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Inclusion criteria :
Exclusion criteria:
The above information was not intended to contain all considerations relevant to a participant's potential participation in a clinical trial.
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| Name | Affiliation | Role |
|---|---|---|
| Clinical Sciences & Operations | Sanofi | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Investigational Site Number 124002 | Montreal | H3T 1E2 | Canada | |||
| Investigational Site Number 643001 |
Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org.
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A total of 31 participants who completed one of the Phase 2 (GZGD00304 [NCT00358150]) or Phase 3 studies (GZGD02507 [ENGAGE; NCT00891202], GZGD02607 [ENCORE; NCT00943111], or GZGD03109 [EDGE; NCT01074944]) were enrolled in this current study (EFC13781) considered as an extension of these previous studies, and received treatment with eliglustat in the current (EFC13781) study.
The study was conducted at 4 active sites in 3 countries. A total of 33 participants were screened between 27 October 2015 and 07 June 2017, of which 2 participants were screen failures as the selection criteria were not met.
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| ID | Title | Description |
|---|---|---|
| FG000 | Eliglustat | Participants who completed one of the Phase 2 (GZGD00304 [NCT00358150]) or Phase 3 studies (GZGD02507 [NCT00891202], GZGD02607 [NCT00943111], or GZGD03109 [NCT01074944]) were enrolled in this current (EFC13781) study. Participants who were cytochrome P450 (CYP) 2D6 intermediate metabolizer (IM), extensive metabolizer (EM) and ultra-rapid metabolizers (URM) received eliglustat 84 milligrams (mg) twice daily and participants who were CYP2D6 poor metabolizer (PM) received eliglustat 84 mg once daily, for duration of minimum 2 years (unless early discontinuation occurred) and up to 4 years, or until commercial eliglustat was available to participants through reimbursement or through the compassionate use (expanded access) program. |
| Title | Milestones | Reasons Not Completed | ||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
|
Analysis was performed on the full analysis set (FAS) population defined as all participants enrolled who received at least 1 dose of eliglustat treatment in the current study.
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| ID | Title | Description |
|---|---|---|
| BG000 | Eliglustat | Participants who completed one of the Phase 2 (GZGD00304) or Phase 3 studies (GZGD02507, GZGD02607 or GZGD03109) were enrolled in this current (EFC13781) study. Participants who were CYP2D6 IM, EM and URM received eliglustat 84 mg twice daily and participants who were CYP2D6 PM received eliglustat 84 mg once daily, for duration of minimum 2 years (unless early discontinuation occurred) and up to 4 years, or until commercial eliglustat was available to participants through reimbursement or through the compassionate use (expanded access) program. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Age provided in this baseline measure defines the participant age at first dose of Eliglustat in the current study EFC13781 (in years). |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Number of Participants With Mobility Status Assessments at Study Baseline, Weeks 52, 104, 156 and 208 | Mobility, i.e., ability to walk was assessed as a part of Gaucher disease assessment in participants. In this outcome measure, number of participants with their different mobility status along with the use of mobility aids (unrestricted mobility, walks with difficulty, walks with orthopaedic aid, requires wheelchair, bedridden) at specified time points were reported. Baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry. | Analysis was performed on FAS population. Here, "number analyzed" = participants with available data for each specified category. Baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry. | Posted | Count of Participants | Participants | Study Baseline, Weeks 52, 104, 156 and 208 |
|
Time from the first administration of the IMP to the last administration of the IMP + 5 days (up to 4 years, or until commercial eliglustat was available to participants through reimbursement or through the compassionate use [expanded access] program)
Reported adverse events (AEs) are TEAEs i.e. AEs that developed, worsened, or became serious during the treatment-emergent period (time from the first administration of the IMP to the last administration of the IMP + 5 days). Analysis was performed on FAS population.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Eliglustat | Participants who completed one of the Phase 2 (GZGD00304) or Phase 3 studies (GZGD02507, GZGD02607 or GZGD03109) were enrolled in this current (EFC13781) study. Participants who were CYP2D6 IM, EM and URM received eliglustat 84 mg twice daily and participants who were CYP2D6 PM received eliglustat 84 mg once daily, for duration of minimum 2 years (unless early discontinuation occurred) and up to 4 years, or until commercial eliglustat was available to participants through reimbursement or through the compassionate use (expanded access) program. |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Pulmonary Tuberculosis | Infections and infestations | MedDRA 24.0 | Systematic Assessment |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Iron Deficiency Anaemia | Blood and lymphatic system disorders | MedDRA 24.0 | Systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Trial Transparency Team | Genzyme, a Sanofi Company | 800-633-1610 | 6# | Contact-US@sanofi.com |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Jul 31, 2017 | Jun 22, 2022 | Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Jul 12, 2021 | Jun 22, 2022 | SAP_001.pdf |
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| ID | Term |
|---|---|
| D005776 | Gaucher Disease |
| ID | Term |
|---|---|
| D013106 | Sphingolipidoses |
| D020140 | Lysosomal Storage Diseases, Nervous System |
| D020739 | Brain Diseases, Metabolic, Inborn |
| D001928 | Brain Diseases, Metabolic |
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| ID | Term |
|---|---|
| C522917 | eliglustat |
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| Change From Current Study Baseline in Total Bone Marrow Burden (BMB) Scores at Weeks 52, 104, 156 and 208 | Bone marrow burden (BMB) scores indicate the degree of bone marrow infiltration. BMB score was measured using MRI (magnetic resonance imaging (MRI), ranged from 0 (no abnormalities) to 8 points (severe disease) for the lumbar spine and from 0 (no abnormalities) to 8 points (severe disease) for the femurs. The total BMB score was calculated as the sum of scores for femur and lumbar spine regions which ranged from 0 (no abnormalities) to 16 (severe disease) points. A higher BMB score signified more severe bone marrow involvement. For this outcome measure, baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry. | Study Baseline, Weeks 52, 104, 156 and 208 |
| Change From Eliglustat Baseline in Total Bone Marrow Burden (BMB) Scores at Weeks 52, 104, 156 and 208 | Bone marrow burden (BMB) scores indicate the degree of bone marrow infiltration was measured using MRI, ranged from 0 (no abnormalities) to 8 points (severe disease) for the lumbar spine and from 0 (no abnormalities) to 8 points (severe disease) for the femurs. The total BMB score was calculated as the sum of scores for femur and lumbar spine regions which ranged from 0 (no abnormalities) to 16 (severe disease) points. A higher BMB score signified more severe bone marrow involvement. For this outcome measure, baseline refers to the eliglustat baseline, which was defined as participant's status at the time of first dose of eliglustat in the previous Phase 2 or Phase 3 study. | Eliglustat Baseline, Weeks 52, 104, 156 and 208 of the current study |
| Change From Current Study Baseline in Total Spine and Femur Bone Mineral Density (BMD) at Weeks 52, 104, 156 and 208 | Bone Mineral Density (BMD) measurements of the spine and bilateral femur were acquired by dual energy X-Ray absorptiometry (DXA) scan. Worst total femur at Baseline refers to the "worst" diseased left or right femur at Baseline. For this outcome measure, baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry. | Study Baseline, Weeks 52, 104, 156 and 208 |
| Change From Eliglustat Baseline in Total Spine and Femur Bone Mineral Density (BMD) at Weeks 52, 104, 156 and 208 | BMD measurements of the spine and bilateral femur were acquired by DXA scan. Worst total femur at Baseline refers to the "worst" diseased left or right femur at baseline. For this outcome measure, baseline refers to the eliglustat baseline, which was defined as participant's status at the time of first dose of eliglustat in the previous Phase 2 or Phase 3 study. | Eliglustat Baseline, Weeks 52, 104, 156 and 208 of the current study |
| Change From Current Study Baseline in Spine and Femur Total T-Scores for BMD at Weeks 52, 104, 156 and 208 | BMD measurements of the spine and bilateral femur were acquired by DXA scan. The T-score bone density categories were: normal (score >-1), osteopenia (score -2.5 to <=-1), and osteoporosis (score <= -2.5). Worst total femur at Baseline refers to the "worst" diseased left or right femur at baseline. For this outcome measure, baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry. | Study Baseline, Weeks 52, 104, 156 and 208 |
| Change From Eliglustat Baseline in Spine and Femur Total T-Scores for BMD at Weeks 52, 104, 156 and 208 | BMD measurements of the spine and bilateral femur were acquired by DXA scan. The T-score bone density categories were: normal (score >-1), osteopenia (score -2.5 to <=-1), and osteoporosis (score <= -2.5). Worst total femur at Baseline refers to the "worst" diseased left or right femur at baseline. For this outcome measure, baseline refers to the eliglustat baseline, which was defined as participant's status at the time of first dose of eliglustat in the previous Phase 2 or Phase 3 study. | Eliglustat Baseline, Weeks 52, 104, 156 and 208 of the current study |
| Change From Current Study Baseline in Spine and Femur Total Z-Scores for BMD at Weeks 52, 104, 156 and 208 | BMD measurements of the spine and bilateral femur were acquired by DXA scan. The Z-score bone density categories were: normal (score >-2) and below normal (score <=-2). Worst total femur at Baseline refers to the "worst" diseased left or right femur at baseline. For this outcome measure, baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry. | Study Baseline, Weeks 52, 104, 156 and 208 |
| Change From Eliglustat Baseline in Spine and Femur Total Z-Scores for BMD at Weeks 52, 104, 156 and 208 | BMD measurements of the spine and bilateral femur were acquired by DXA scan. The Z-score bone density categories are: normal (score >-2) and below normal (score <=-2). Worst total femur at Baseline refers to the "worst" diseased left or right femur at baseline. For this outcome measure, baseline refers to the eliglustat baseline, which was defined as participant's status at the time of first dose of eliglustat in the previous Phase 2 or Phase 3 study. | Eliglustat Baseline, Weeks 52, 104, 156 and 208 of the current study |
| Total Number of New or Worsening Osteonecrosis Events for Spine and Femur at Study Baseline, Week 52, 104, 156 and 208 | Osteonecrosis was assessed by bone MRI and X-Ray for spine and by MRI for femur. Total number of new or worsening osteonecrosis events among all the participants with corresponding assessment at specified time points were reported in this outcome measure. For this outcome measure, baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry. | Study Baseline, Weeks 52, 104, 156 and 208 |
| Total Number of New or Worsening Fracture Events for Spine and Femur at Study Baseline, Week 52, 104, 156 and 208 | Fracture was assessed by bone MRI and X-Ray for spine and by MRI for femur. Total number of new or worsening fracture events among all the participants with corresponding assessment at specified time points were reported in this outcome measure. For this outcome measure, baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry. | Study Baseline, Weeks 52, 104, 156 and 208 |
| Total Number of New or Worsening Infarcts Events for Spine and Femur at Study Baseline, Week 52, 104, 156 and 208 | Infarcts were assessed by bone MRI and X-Ray for spine and by MRI for femur. Total number of new or worsening infarcts events among all the participants with corresponding assessment at specified time points were reported in this outcome measure. For this outcome measure, baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry. | Study Baseline, Weeks 52, 104, 156 and 208 |
| Total Number of New or Worsening Lytic Lesions Events for Spine at Study Baseline, Week 104, and 208 | Lytic Lesions were assessed by bone X-Ray for spine. Total number of new or worsening lytic lesions events among all the participants with corresponding assessment at specified time points were reported in this outcome measure. For this outcome measure, baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry. | Study Baseline, Weeks 104, and 208 |
| Observed Annual Incidence Rate for Spine and Femur Osteonecrosis at Week 52, 104, 156 and 208 | Observed annual incidence rate was estimated using the total number of events divided by the total years of follow-up in each specified year (for all participants). Osteonecrosis was assessed by bone MRI and X-Ray for spine and by MRI for femur. | For 52 Weeks (i.e., 1 year),104 Weeks (i.e., 2 year), 156 Weeks (i.e., 3 year) and 208 Weeks (i.e., 4 years) |
| Observed Annual Incidence Rate for Spine and Femur Fracture at Week 52, 104, 156 and 208 | Observed annual incidence rate was estimated using the total number of events divided by the total years of follow-up in each specified year (for all participants). Fracture was assessed by bone MRI and X-Ray for spine and by MRI for femur. | For 52 Weeks (i.e., 1 year),104 Weeks (i.e., 2 year), 156 Weeks (i.e., 3 year) and 208 Weeks (i.e., 4 years) |
| Observed Annual Incidence Rate for Spine and Femur Infarcts at Week 52, 104, 156 and 208 | Observed annual incidence rate was estimated using the total number of events divided by the total years of follow-up in each specified year (for all participants). Infarcts were assessed by bone MRI and X-Ray for spine and by MRI for femur. | For 52 Weeks (i.e., 1 year),104 Weeks (i.e., 2 year), 156 Weeks (i.e., 3 year) and 208 Weeks (i.e., 4 years) |
| Observed Annual Incidence Rate for Spine Lytic Lesion at Week 104, and 208 | Observed annual incidence rate was estimated using the total number of events divided by the total years of follow-up in each specified year (for all participants). Lytic Lesions were assessed by bone X-Ray for spine. | For 104 Weeks (i.e., 2 year), and 208 Weeks (i.e., 4 years) |
| Change From Current Study Baseline in Bone Biomarker Level: Macrophage Inflammatory Protein 1 Beta (MIP-1β) at Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234 | MIP-1β considered a biomarker of active bone disease, was assayed from plasma. For this outcome measure, baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry. | Study Baseline, Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234 |
| Change From Eliglustat Baseline in Bone Biomarker Level: Macrophage Inflammatory Protein 1 Beta (MIP-1β) at Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234 | MIP-1β considered a biomarker of active bone disease, was assayed from plasma. For this outcome measure, baseline refers to the eliglustat baseline, which was defined as participant's status at the time of first dose of eliglustat in the previous Phase 2 or Phase 3 study. | Eliglustat Baseline, Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234 of the current study |
| Change From Current Study Baseline in Bone Biomarker Level: Procollagen 1 N- Terminal Propeptide (P1NP) at Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234 | P1NP, a marker of bone formation was assayed from plasma. For this outcome measure, baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry. | Study Baseline, Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234 |
| Change From Eliglustat Baseline in Bone Biomarker Level: Procollagen 1 N- Terminal Propeptide (P1NP) at Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234 | P1NP, a marker of bone formation was assayed from plasma. For this outcome measure, baseline refers to the eliglustat baseline, which was defined as participant's status at the time of first dose of eliglustat in the previous Phase 2 or Phase 3 study. | Eliglustat Baseline, Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234 of the current study |
| Change From Current Study Baseline in Bone Biomarker Level: Type 1 Collagen C-Telopeptides (CTx) at Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234 | CTx, a marker of bone resorption was assayed from plasma. For this outcome measure, baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry. | Study Baseline, Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234 |
| Change From Eliglustat Baseline in Bone Biomarker Level: Type 1 Collagen C-Telopeptides (CTx) at Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234 | CTx, a marker of bone resorption was assayed from plasma. For this outcome measure, baseline refers to the eliglustat baseline, which was defined as participant's status at the time of first dose of eliglustat in the previous Phase 2 or Phase 3 study. | Eliglustat Baseline, Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234 of the current study |
| Study Baseline, Week 26, 52, 78, 104, 130, 156, 182, 208 and 234 |
| Change From Eliglustat Baseline in Gaucher Disease Type 1 (GD1) Biomarker Levels: Chitotriosidase at Week 26, 52, 78, 104, 130, 156, 182, 208 and 234 | Chitotriosidase biomarker was assayed from plasma. Chitotriosidase biomarker levels for participants who were CYP2D6 non-URM was reported in this outcome measure. For this outcome measure, baseline refers to the eliglustat baseline, which was defined as participant's status at the time of first dose of eliglustat in the previous Phase 2 or Phase 3 study. Data for this outcome measure was planned to be collected and analyzed in reference to eliglustat baseline separately for Phase 2, ENGAGE and EDGE studies (GD treatment naïve participants) and from ENCORE study (participants switched from ERT). | Eliglustat Baseline, Week 26, 52, 78, 104, 130, 156, 182, 208 and 234 of the current study |
| Change From Current Study Baseline in GD1 Biomarker Levels: Glucosylceramide (GL-1) at Week 26, 52, 78, 104, 130, 156, 182, 208 and 234 | Glucosylceramide (GL-1) biomarker was assayed from plasma. GL-1 biomarker levels for participants who were CYP2D6 non-URM was reported in this outcome measure. For this outcome measure, baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry. | Study Baseline, Week 26, 52, 78, 104, 130, 156, 182, 208 and 234 |
| Change From Eliglustat Baseline in GD1 Biomarker Levels: Glucosylceramide (GL-1) at Week 26, 52, 78, 104, 130, 156, 182, 208 and 234 | Glucosylceramide (GL-1) biomarker was assayed from plasma. GL-1 biomarker levels for participants who were CYP2D6 non-URM was reported in this outcome measure. For this outcome measure, baseline refers to the eliglustat baseline, which was defined as participant's status at the time of first dose of eliglustat in the previous Phase 2 or Phase 3 study. Data for this outcome measure was planned to be collected and analyzed in reference to eliglustat baseline separately for participants from Phase 2, ENGAGE and EDGE studies (GD treatment naïve participants) and from ENCORE study (participants switched from ERT). | Eliglustat Baseline, Week 26, 52, 78, 104, 130, 156, 182, 208 and 234 of the current study |
| Change From Current Study Baseline in GD1 Biomarker Levels: Lyso Glucosylceramide (Lyso-GL-1) at Week 26, 52, 78, 104, 130, 156, 182, 208 and 234 | Lyso-GL-1 biomarker was assayed from plasma. Lyso-GL-1 biomarker levels for participants who were CYP2D6 non-URM was reported in this outcome measure. For this outcome measure, baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry. | Study Baseline, Week 26, 52, 78, 104, 130, 156, 182, 208 and 234 |
| Change From Eliglustat Baseline in GD1 Biomarker Levels: Lyso Glucosylceramide (Lyso-GL-1) at Week 26, 52, 78, 104, 130, 156, 182, 208 and 234 | Lyso-GL-1 biomarker was assayed from plasma. Lyso-GL-1 biomarker levels for participants who were CYP2D6 non-URM was reported in this outcome measure. For this outcome measure, baseline refers to the eliglustat baseline, which was defined as participant's status at the time of first dose of eliglustat in the previous Phase 2 or Phase 3 study. Data for this outcome measure was planned to be collected and analyzed in reference to eliglustat baseline separately for participants from Phase 2, ENGAGE and EDGE studies (GD treatment naïve participants) and from ENCORE study (participants switched from ERT). | Eliglustat Baseline, Week 26, 52, 78, 104, 130, 156, 182, 208 and 234 of the current study |
| Change From Current Study Baseline in Short Form-36 Health Survey (SF-36) Scores at Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234 | The 36-Item Short-Form Health Survey (SF-36) is standardized survey evaluating 8 aspects of functional health and well-being. Physical Component Summary (PCS) with 4 sub-scales: physical function, role limitations due to physical problems, bodily pain, and general health perception; and Mental Component Summary (MCS) with 4 sub-scales: vitality, social function, role limitations due to emotional problems, and mental health. Summations of item scores of the same sub-scale give the sub-scale scores, which are transformed into a range from 0 to 100; 0= worst and 100=best outcome. Both PCS and MCS range from 0 to 100 with higher scores indicating better physical and mental health. For this outcome measure, baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry. | Study Baseline, Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234 |
| Change From Eliglustat Baseline in SF-36 Scores at Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234 | SF-36 is a standardized survey evaluating 8 aspects of functional health and well-being. PCS with 4 sub-scales: physical function, role limitations due to physical problems, pain, and general health perception; and MCS with 4 sub-scales: vitality, social function, role limitations due to emotional problems, and mental health. Summations of item scores of same sub-scale give the sub-scale scores, which are transformed into range from 0 to 100; 0= worst, and 100=best outcome. Both PCS and MCS range from 0 to 100, higher scores indicating better physical and mental health. For this outcome measure, baseline refers to the eliglustat baseline, which was defined as participant's status at the time of first dose of eliglustat in the previous Phase 2 or Phase 3 study. | Eliglustat Baseline, Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234 of the current study |
| Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Treatment-emergent Serious Adverse Events (TESAEs) | An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug and did not necessarily had to have a causal relationship with the treatment. A serious adverse event (SAE) was any untoward medical occurrence that at any dose: resulted in death, was life-threatening, required inpatient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, was a congenital anomaly/birth defect, was a medically important event. TEAEs were defined as AEs that developed, worsened or became serious during the treatment-emergent period (time from the first administration of the investigational medicinal product (IMP) to the last administration of the IMP + 5 days). | From the first administration of the IMP to the last administration of the IMP + 5 days (up to 4 years, or until commercial eliglustat was available to participants through reimbursement or through the compassionate use [expanded access] program) |
| Moscow |
| 125167 |
| Russia |
| Investigational Site Number 643002 | Saint Petersburg | 197341 | Russia |
| Investigational Site Number 788001 | Tunis | 1007 | Tunisia |
| Mean |
| Standard Deviation |
| years |
|
| Sex: Female, Male | Count of Participants | Participants |
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| Race (NIH/OMB) | Count of Participants | Participants |
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| Time since first symptom of Gaucher to enrollment in EFC13781 | Mean | Standard Deviation | years |
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| Time since initial Gaucher diagnosis to enrollment in EFC13781 | Mean | Standard Deviation | years |
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| CYP2D6 metabolizer status | Data represents participants who were CYP2D6 intermediate metabolizer (IM), extensive metabolizer (EM), poor metabolizer (PM) and ultra-rapid metabolizers (URM) at the Baseline. | Count of Participants | Participants |
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Participants who completed one of the Phase 2 (GZGD00304) or Phase 3 studies (GZGD02507, GZGD02607 or GZGD03109) were enrolled in this current (EFC13781) study. Participants who were CYP2D6 IM, EM and URM received eliglustat 84 mg twice daily and participants who were CYP2D6 PM received eliglustat 84 mg once daily, for duration of minimum 2 years (unless early discontinuation occurred) and up to 4 years, or until commercial eliglustat was available to participants through reimbursement or through the compassionate use (expanded access) program.
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| Primary | Number of Participants With Bone Pain Levels During the Past 4 Weeks at Study Baseline, Weeks 52, 104, 156 and 208 | Bone pain was assessed as a part of Gaucher disease assessment in participants. Participants were categorized as none (no bone pain), very mild bone pain, mild bone pain, moderate bone pain, severe bone pain and extreme bone pain during the past 4 weeks at each specified visit. In this outcome measure, number of participants with different level of bone pain during the past 4 weeks at specified time points were reported. Baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry. | Analysis was performed on FAS population. Here, "number analyzed" = participants with available data for each specified category. Baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry. | Posted | Count of Participants | Participants | Study Baseline, Weeks 52, 104, 156 and 208 |
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| Primary | Number of Participants With Bone Crisis at Study Baseline, Weeks 52, 104, 156 and 208 | Bone crisis was assessed as a part of Gaucher disease assessment in participants. Acute, excruciating episodic bone pain is characteristic of Gaucher bone crisis, which typically causes periosteal elevation, elevated white blood cell count, fever, or debilitation lasting several days or longer and requires treatment with immobilization of the affected area, and opioid analgesics. Participants were categorized as 0= no bone crisis, 1= 1 bone crisis, 2= 2 bone crisis and >=3 = more than 3 bone crisis during the assessment period. In this outcome measure, number of participants with different bone crises levels at specified time points were reported. Baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry. | Analysis was performed on FAS population. Here, "number analyzed" = participants with available data for each specified category. Baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry. | Posted | Count of Participants | Participants | Study Baseline, Weeks 52, 104, 156 and 208 |
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| Primary | Change From Current Study Baseline in Total Bone Marrow Burden (BMB) Scores at Weeks 52, 104, 156 and 208 | Bone marrow burden (BMB) scores indicate the degree of bone marrow infiltration. BMB score was measured using MRI (magnetic resonance imaging (MRI), ranged from 0 (no abnormalities) to 8 points (severe disease) for the lumbar spine and from 0 (no abnormalities) to 8 points (severe disease) for the femurs. The total BMB score was calculated as the sum of scores for femur and lumbar spine regions which ranged from 0 (no abnormalities) to 16 (severe disease) points. A higher BMB score signified more severe bone marrow involvement. For this outcome measure, baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry. | Analysis was performed on FAS population. Here, "number analyzed" = participants with available data for each specified category. Baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry. | Posted | Mean | Standard Deviation | score on a scale | Study Baseline, Weeks 52, 104, 156 and 208 |
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| Primary | Change From Eliglustat Baseline in Total Bone Marrow Burden (BMB) Scores at Weeks 52, 104, 156 and 208 | Bone marrow burden (BMB) scores indicate the degree of bone marrow infiltration was measured using MRI, ranged from 0 (no abnormalities) to 8 points (severe disease) for the lumbar spine and from 0 (no abnormalities) to 8 points (severe disease) for the femurs. The total BMB score was calculated as the sum of scores for femur and lumbar spine regions which ranged from 0 (no abnormalities) to 16 (severe disease) points. A higher BMB score signified more severe bone marrow involvement. For this outcome measure, baseline refers to the eliglustat baseline, which was defined as participant's status at the time of first dose of eliglustat in the previous Phase 2 or Phase 3 study. | Analysis was performed on FAS population. Here, "number analyzed" = participants with available data for each specified category. Data for this outcome measure was planned to be collected and analyzed in reference to eliglustat baseline separately for participants from Phase 2, ENGAGE and EDGE studies (GD treatment naïve participants) and from ENCORE study (participants switched from Enzyme Replacement Therapy [ERT]). | Posted | Mean | Standard Deviation | score on a scale | Eliglustat Baseline, Weeks 52, 104, 156 and 208 of the current study |
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| Primary | Change From Current Study Baseline in Total Spine and Femur Bone Mineral Density (BMD) at Weeks 52, 104, 156 and 208 | Bone Mineral Density (BMD) measurements of the spine and bilateral femur were acquired by dual energy X-Ray absorptiometry (DXA) scan. Worst total femur at Baseline refers to the "worst" diseased left or right femur at Baseline. For this outcome measure, baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry. | Analysis was performed on FAS population. Here, "number analyzed" = participants with available data for each specified category. Baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry. | Posted | Mean | Standard Deviation | grams per centimeter square (g/cm^2) | Study Baseline, Weeks 52, 104, 156 and 208 |
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| Primary | Change From Eliglustat Baseline in Total Spine and Femur Bone Mineral Density (BMD) at Weeks 52, 104, 156 and 208 | BMD measurements of the spine and bilateral femur were acquired by DXA scan. Worst total femur at Baseline refers to the "worst" diseased left or right femur at baseline. For this outcome measure, baseline refers to the eliglustat baseline, which was defined as participant's status at the time of first dose of eliglustat in the previous Phase 2 or Phase 3 study. | Analysis was performed on FAS population. Here, "number analyzed" = participants with available data for each specified category. Data for this outcome measure was was planned to be collected and analyzed in reference to eliglustat baseline separately for participants from Phase 2, ENGAGE and EDGE studies (GD treatment naïve participants) and from ENCORE study (participants switched from ERT). | Posted | Mean | Standard Deviation | g/cm^2 | Eliglustat Baseline, Weeks 52, 104, 156 and 208 of the current study |
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| Primary | Change From Current Study Baseline in Spine and Femur Total T-Scores for BMD at Weeks 52, 104, 156 and 208 | BMD measurements of the spine and bilateral femur were acquired by DXA scan. The T-score bone density categories were: normal (score >-1), osteopenia (score -2.5 to <=-1), and osteoporosis (score <= -2.5). Worst total femur at Baseline refers to the "worst" diseased left or right femur at baseline. For this outcome measure, baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry. | Analysis was performed on FAS population. Here, "number analyzed" = participants with available data for each specified category. Baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry. | Posted | Mean | Standard Deviation | T-score | Study Baseline, Weeks 52, 104, 156 and 208 |
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| Primary | Change From Eliglustat Baseline in Spine and Femur Total T-Scores for BMD at Weeks 52, 104, 156 and 208 | BMD measurements of the spine and bilateral femur were acquired by DXA scan. The T-score bone density categories were: normal (score >-1), osteopenia (score -2.5 to <=-1), and osteoporosis (score <= -2.5). Worst total femur at Baseline refers to the "worst" diseased left or right femur at baseline. For this outcome measure, baseline refers to the eliglustat baseline, which was defined as participant's status at the time of first dose of eliglustat in the previous Phase 2 or Phase 3 study. | Analysis was performed on FAS population. Here, "number analyzed" = participants with available data for each specified category. Here, "0" signifies that none of the participants were available for assessment at the specified timepoint. Data for this outcome measure was planned to be collected and analyzed in reference to eliglustat baseline separately for participants from Phase 2, ENGAGE and EDGE studies (GD treatment naïve participants) and from ENCORE study (participants switched from ERT). | Posted | Mean | Standard Deviation | T-score | Eliglustat Baseline, Weeks 52, 104, 156 and 208 of the current study |
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| Primary | Change From Current Study Baseline in Spine and Femur Total Z-Scores for BMD at Weeks 52, 104, 156 and 208 | BMD measurements of the spine and bilateral femur were acquired by DXA scan. The Z-score bone density categories were: normal (score >-2) and below normal (score <=-2). Worst total femur at Baseline refers to the "worst" diseased left or right femur at baseline. For this outcome measure, baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry. | Analysis was performed on FAS population. Here, "number analyzed" = participants with available data for each specified category. Baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry. | Posted | Mean | Standard Deviation | Z-score | Study Baseline, Weeks 52, 104, 156 and 208 |
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| Primary | Change From Eliglustat Baseline in Spine and Femur Total Z-Scores for BMD at Weeks 52, 104, 156 and 208 | BMD measurements of the spine and bilateral femur were acquired by DXA scan. The Z-score bone density categories are: normal (score >-2) and below normal (score <=-2). Worst total femur at Baseline refers to the "worst" diseased left or right femur at baseline. For this outcome measure, baseline refers to the eliglustat baseline, which was defined as participant's status at the time of first dose of eliglustat in the previous Phase 2 or Phase 3 study. | Analysis was performed on FAS population. Here, "number analyzed" = participants with available data for each specified category. Data for this outcome measure was planned to be collected and analyzed in reference to eliglustat baseline separately for participants from Phase 2, ENGAGE and EDGE studies (GD treatment naïve participants) and from ENCORE study (participants switched from ERT). | Posted | Mean | Standard Deviation | Z-score | Eliglustat Baseline, Weeks 52, 104, 156 and 208 of the current study |
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| Primary | Total Number of New or Worsening Osteonecrosis Events for Spine and Femur at Study Baseline, Week 52, 104, 156 and 208 | Osteonecrosis was assessed by bone MRI and X-Ray for spine and by MRI for femur. Total number of new or worsening osteonecrosis events among all the participants with corresponding assessment at specified time points were reported in this outcome measure. For this outcome measure, baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry. | Analysis was performed on FAS population. Here, "number analyzed" = participants with available data for each specified category. Baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry. | Posted | Mean | Full Range | events | Study Baseline, Weeks 52, 104, 156 and 208 |
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| Primary | Total Number of New or Worsening Fracture Events for Spine and Femur at Study Baseline, Week 52, 104, 156 and 208 | Fracture was assessed by bone MRI and X-Ray for spine and by MRI for femur. Total number of new or worsening fracture events among all the participants with corresponding assessment at specified time points were reported in this outcome measure. For this outcome measure, baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry. | Analysis was performed on FAS population. Here, "number analyzed" = participants with available data for each specified category. Baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry. | Posted | Mean | Full Range | events | Study Baseline, Weeks 52, 104, 156 and 208 |
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| Primary | Total Number of New or Worsening Infarcts Events for Spine and Femur at Study Baseline, Week 52, 104, 156 and 208 | Infarcts were assessed by bone MRI and X-Ray for spine and by MRI for femur. Total number of new or worsening infarcts events among all the participants with corresponding assessment at specified time points were reported in this outcome measure. For this outcome measure, baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry. | Analysis was performed on FAS population. Here, "number analyzed" = participants with available data for each specified category. Baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry. | Posted | Mean | Full Range | events | Study Baseline, Weeks 52, 104, 156 and 208 |
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| Primary | Total Number of New or Worsening Lytic Lesions Events for Spine at Study Baseline, Week 104, and 208 | Lytic Lesions were assessed by bone X-Ray for spine. Total number of new or worsening lytic lesions events among all the participants with corresponding assessment at specified time points were reported in this outcome measure. For this outcome measure, baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry. | Analysis was performed on FAS population. Here, "number analyzed" = participants with available data for each specified category. Baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry. | Posted | Mean | Full Range | events | Study Baseline, Weeks 104, and 208 |
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| Primary | Observed Annual Incidence Rate for Spine and Femur Osteonecrosis at Week 52, 104, 156 and 208 | Observed annual incidence rate was estimated using the total number of events divided by the total years of follow-up in each specified year (for all participants). Osteonecrosis was assessed by bone MRI and X-Ray for spine and by MRI for femur. | Analysis was performed on FAS population. | Posted | Number | events per participant-year | For 52 Weeks (i.e., 1 year),104 Weeks (i.e., 2 year), 156 Weeks (i.e., 3 year) and 208 Weeks (i.e., 4 years) |
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| Primary | Observed Annual Incidence Rate for Spine and Femur Fracture at Week 52, 104, 156 and 208 | Observed annual incidence rate was estimated using the total number of events divided by the total years of follow-up in each specified year (for all participants). Fracture was assessed by bone MRI and X-Ray for spine and by MRI for femur. | Analysis was performed on FAS population. | Posted | Number | events per participant-year | For 52 Weeks (i.e., 1 year),104 Weeks (i.e., 2 year), 156 Weeks (i.e., 3 year) and 208 Weeks (i.e., 4 years) |
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| Primary | Observed Annual Incidence Rate for Spine and Femur Infarcts at Week 52, 104, 156 and 208 | Observed annual incidence rate was estimated using the total number of events divided by the total years of follow-up in each specified year (for all participants). Infarcts were assessed by bone MRI and X-Ray for spine and by MRI for femur. | Analysis was performed on FAS population. | Posted | Number | events per participant-year | For 52 Weeks (i.e., 1 year),104 Weeks (i.e., 2 year), 156 Weeks (i.e., 3 year) and 208 Weeks (i.e., 4 years) |
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| Primary | Observed Annual Incidence Rate for Spine Lytic Lesion at Week 104, and 208 | Observed annual incidence rate was estimated using the total number of events divided by the total years of follow-up in each specified year (for all participants). Lytic Lesions were assessed by bone X-Ray for spine. | Analysis was performed on FAS population. | Posted | Number | events per participant-year | For 104 Weeks (i.e., 2 year), and 208 Weeks (i.e., 4 years) |
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| Primary | Change From Current Study Baseline in Bone Biomarker Level: Macrophage Inflammatory Protein 1 Beta (MIP-1β) at Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234 | MIP-1β considered a biomarker of active bone disease, was assayed from plasma. For this outcome measure, baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry. | Analysis was performed on FAS population. Here, "number analyzed" = participants with available data for each specified category. Baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry. | Posted | Mean | Standard Deviation | picograms per milliliter (pg/mL) | Study Baseline, Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234 |
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| Primary | Change From Eliglustat Baseline in Bone Biomarker Level: Macrophage Inflammatory Protein 1 Beta (MIP-1β) at Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234 | MIP-1β considered a biomarker of active bone disease, was assayed from plasma. For this outcome measure, baseline refers to the eliglustat baseline, which was defined as participant's status at the time of first dose of eliglustat in the previous Phase 2 or Phase 3 study. | Analyzed on FAS population. Here, "number analyzed" = participants with available data for each specified category and "0" in number analyzed field signifies that none of participants had evaluable data at specified timepoint. Data for this outcome measure was planned to be collected and analyzed in reference to eliglustat baseline separately for participants from Phase 2, ENGAGE and EDGE studies (GD treatment naïve participants) and from ENCORE study (participants switched from ERT). | Posted | Mean | Standard Deviation | pg/mL | Eliglustat Baseline, Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234 of the current study |
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| Primary | Change From Current Study Baseline in Bone Biomarker Level: Procollagen 1 N- Terminal Propeptide (P1NP) at Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234 | P1NP, a marker of bone formation was assayed from plasma. For this outcome measure, baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry. | Analysis was performed on FAS population. Here, "number analyzed" = participants with available data for each specified category. Baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry. | Posted | Mean | Standard Deviation | micrograms per liter (mcg/L) | Study Baseline, Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234 |
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| Primary | Change From Eliglustat Baseline in Bone Biomarker Level: Procollagen 1 N- Terminal Propeptide (P1NP) at Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234 | P1NP, a marker of bone formation was assayed from plasma. For this outcome measure, baseline refers to the eliglustat baseline, which was defined as participant's status at the time of first dose of eliglustat in the previous Phase 2 or Phase 3 study. | Here, "0" signifies that data for this outcome measure (bone biomarker P1NP) was not collected and assessed in Phase 2 and Phase 3 studies, hence the biomarker level was not reported at specified timepoints. | Posted | Eliglustat Baseline, Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234 of the current study |
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| Primary | Change From Current Study Baseline in Bone Biomarker Level: Type 1 Collagen C-Telopeptides (CTx) at Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234 | CTx, a marker of bone resorption was assayed from plasma. For this outcome measure, baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry. | Analysis was performed on FAS population. Here, "number analyzed" = participants with available data for each specified category. Baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry. | Posted | Mean | Standard Deviation | mcg/L | Study Baseline, Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234 |
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| Primary | Change From Eliglustat Baseline in Bone Biomarker Level: Type 1 Collagen C-Telopeptides (CTx) at Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234 | CTx, a marker of bone resorption was assayed from plasma. For this outcome measure, baseline refers to the eliglustat baseline, which was defined as participant's status at the time of first dose of eliglustat in the previous Phase 2 or Phase 3 study. | Here, "0" signifies that data for this outcome measure (bone biomarker CTx) was not collected and assessed in Phase 2 and Phase 3 studies, hence the biomarker level was not reported at specified timepoints. | Posted | Eliglustat Baseline, Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234 of the current study |
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| Secondary | Change From Current Study Baseline in Gaucher Disease Type 1 (GD1) Biomarker Levels: Chitotriosidase at Week 26, 52, 78, 104, 130, 156, 182, 208 and 234 | Chitotriosidase biomarker was assayed from plasma. Chitotriosidase biomarker levels for participants who were CYP2D6 non-Ultra Rapid Metabolizers (non-URM) was reported in this outcome measure. For this outcome measure, baseline refers to the study baseline, which was defined as status at study entry. | Analysis was performed on FAS population. Here, "overall number of participants analyzed" = participants evaluable for this outcome measure and "number analyzed" = participants with available data for each specified category. Baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry. | Posted | Mean | Standard Deviation | nmol/hr/mL | Study Baseline, Week 26, 52, 78, 104, 130, 156, 182, 208 and 234 |
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| Secondary | Change From Eliglustat Baseline in Gaucher Disease Type 1 (GD1) Biomarker Levels: Chitotriosidase at Week 26, 52, 78, 104, 130, 156, 182, 208 and 234 | Chitotriosidase biomarker was assayed from plasma. Chitotriosidase biomarker levels for participants who were CYP2D6 non-URM was reported in this outcome measure. For this outcome measure, baseline refers to the eliglustat baseline, which was defined as participant's status at the time of first dose of eliglustat in the previous Phase 2 or Phase 3 study. Data for this outcome measure was planned to be collected and analyzed in reference to eliglustat baseline separately for Phase 2, ENGAGE and EDGE studies (GD treatment naïve participants) and from ENCORE study (participants switched from ERT). | Analysis was performed on FAS population. Here, "overall number of participants analyzed" = participants evaluable for this outcome measure and "number analyzed" = participants with available data for each specified category and "0" in number analyzed field signifies that none of the participants had evaluable data at specified timepoint. | Posted | Mean | Standard Deviation | nmol/hr/mL | Eliglustat Baseline, Week 26, 52, 78, 104, 130, 156, 182, 208 and 234 of the current study |
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| Secondary | Change From Current Study Baseline in GD1 Biomarker Levels: Glucosylceramide (GL-1) at Week 26, 52, 78, 104, 130, 156, 182, 208 and 234 | Glucosylceramide (GL-1) biomarker was assayed from plasma. GL-1 biomarker levels for participants who were CYP2D6 non-URM was reported in this outcome measure. For this outcome measure, baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry. | Analysis was performed on FAS population. Here, "overall number of participants analyzed" = participants evaluable for this outcome measure and "number analyzed" = participants with available data for each specified category. Baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry. | Posted | Mean | Standard Deviation | micrograms per milliliter (mcg/mL) | Study Baseline, Week 26, 52, 78, 104, 130, 156, 182, 208 and 234 |
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| Secondary | Change From Eliglustat Baseline in GD1 Biomarker Levels: Glucosylceramide (GL-1) at Week 26, 52, 78, 104, 130, 156, 182, 208 and 234 | Glucosylceramide (GL-1) biomarker was assayed from plasma. GL-1 biomarker levels for participants who were CYP2D6 non-URM was reported in this outcome measure. For this outcome measure, baseline refers to the eliglustat baseline, which was defined as participant's status at the time of first dose of eliglustat in the previous Phase 2 or Phase 3 study. Data for this outcome measure was planned to be collected and analyzed in reference to eliglustat baseline separately for participants from Phase 2, ENGAGE and EDGE studies (GD treatment naïve participants) and from ENCORE study (participants switched from ERT). | Analysis was performed on FAS population. Here, "overall number of participants analyzed" = participants evaluable for this outcome measure and "number analyzed" = participants with available data for each specified category and "0" signifies that none of the participants had evaluable data at the specified timepoint. | Posted | Mean | Standard Deviation | mcg/mL | Eliglustat Baseline, Week 26, 52, 78, 104, 130, 156, 182, 208 and 234 of the current study |
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| Secondary | Change From Current Study Baseline in GD1 Biomarker Levels: Lyso Glucosylceramide (Lyso-GL-1) at Week 26, 52, 78, 104, 130, 156, 182, 208 and 234 | Lyso-GL-1 biomarker was assayed from plasma. Lyso-GL-1 biomarker levels for participants who were CYP2D6 non-URM was reported in this outcome measure. For this outcome measure, baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry. | Analysis was performed on FAS population. Here, "overall number of participants analyzed" = participants evaluable for this outcome measure and "number analyzed" = participants with available data for each specified category. Baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry. | Posted | Mean | Standard Deviation | nanograms per milliliter (ng/mL) | Study Baseline, Week 26, 52, 78, 104, 130, 156, 182, 208 and 234 |
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| Secondary | Change From Eliglustat Baseline in GD1 Biomarker Levels: Lyso Glucosylceramide (Lyso-GL-1) at Week 26, 52, 78, 104, 130, 156, 182, 208 and 234 | Lyso-GL-1 biomarker was assayed from plasma. Lyso-GL-1 biomarker levels for participants who were CYP2D6 non-URM was reported in this outcome measure. For this outcome measure, baseline refers to the eliglustat baseline, which was defined as participant's status at the time of first dose of eliglustat in the previous Phase 2 or Phase 3 study. Data for this outcome measure was planned to be collected and analyzed in reference to eliglustat baseline separately for participants from Phase 2, ENGAGE and EDGE studies (GD treatment naïve participants) and from ENCORE study (participants switched from ERT). | Analysis was performed on FAS population. Here, "overall number of participants analyzed" = participants evaluable for this outcome measure and "number analyzed" = participants with available data for each specified category. Here, "0" in overall number analyzed field signifies that biomarker lyso-GL-1 was not assessed in Phase 3 studies, hence the biomarker levels at specified timepoint were not reported for any of the participants who were from previous Phase 3 studies. | Posted | Mean | Standard Deviation | ng/mL | Eliglustat Baseline, Week 26, 52, 78, 104, 130, 156, 182, 208 and 234 of the current study |
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| Secondary | Change From Current Study Baseline in Short Form-36 Health Survey (SF-36) Scores at Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234 | The 36-Item Short-Form Health Survey (SF-36) is standardized survey evaluating 8 aspects of functional health and well-being. Physical Component Summary (PCS) with 4 sub-scales: physical function, role limitations due to physical problems, bodily pain, and general health perception; and Mental Component Summary (MCS) with 4 sub-scales: vitality, social function, role limitations due to emotional problems, and mental health. Summations of item scores of the same sub-scale give the sub-scale scores, which are transformed into a range from 0 to 100; 0= worst and 100=best outcome. Both PCS and MCS range from 0 to 100 with higher scores indicating better physical and mental health. For this outcome measure, baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry. | Analysis was performed on FAS population. Here, "number analyzed" = participants with available data for each specified category. Baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry. | Posted | Mean | Standard Deviation | score on a scale | Study Baseline, Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234 |
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| Secondary | Change From Eliglustat Baseline in SF-36 Scores at Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234 | SF-36 is a standardized survey evaluating 8 aspects of functional health and well-being. PCS with 4 sub-scales: physical function, role limitations due to physical problems, pain, and general health perception; and MCS with 4 sub-scales: vitality, social function, role limitations due to emotional problems, and mental health. Summations of item scores of same sub-scale give the sub-scale scores, which are transformed into range from 0 to 100; 0= worst, and 100=best outcome. Both PCS and MCS range from 0 to 100, higher scores indicating better physical and mental health. For this outcome measure, baseline refers to the eliglustat baseline, which was defined as participant's status at the time of first dose of eliglustat in the previous Phase 2 or Phase 3 study. | Analysis was performed on full analysis population. Here, "number analyzed" = participants with available data for each specified category "0" signifies that none of the participants had evaluable data at the specified timepoint. Data for this outcome measure was planned to be collected and analyzed in reference to eliglustat baseline separately for participants from Phase 2, ENGAGE and EDGE studies (GD treatment naïve participants) and from ENCORE study (participants switched from ERT). | Posted | Mean | Standard Deviation | score on a scale | Eliglustat Baseline, Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234 of the current study |
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| Secondary | Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Treatment-emergent Serious Adverse Events (TESAEs) | An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug and did not necessarily had to have a causal relationship with the treatment. A serious adverse event (SAE) was any untoward medical occurrence that at any dose: resulted in death, was life-threatening, required inpatient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, was a congenital anomaly/birth defect, was a medically important event. TEAEs were defined as AEs that developed, worsened or became serious during the treatment-emergent period (time from the first administration of the investigational medicinal product (IMP) to the last administration of the IMP + 5 days). | Analysis was performed on FAS population. | Posted | Count of Participants | Participants | From the first administration of the IMP to the last administration of the IMP + 5 days (up to 4 years, or until commercial eliglustat was available to participants through reimbursement or through the compassionate use [expanded access] program) |
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| 0 |
| 31 |
| 4 |
| 31 |
| 12 |
| 31 |
| Meniscus Injury | Injury, poisoning and procedural complications | MedDRA 24.0 | Systematic Assessment |
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| Bone Pain | Musculoskeletal and connective tissue disorders | MedDRA 24.0 | Systematic Assessment |
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| Ovarian Cyst | Reproductive system and breast disorders | MedDRA 24.0 | Systematic Assessment |
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| Uterine Polyp | Reproductive system and breast disorders | MedDRA 24.0 | Systematic Assessment |
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| Toothache | Gastrointestinal disorders | MedDRA 24.0 | Systematic Assessment |
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| Human Chorionic Gonadotropin Increased | Investigations | MedDRA 24.0 | Systematic Assessment |
|
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA 24.0 | Systematic Assessment |
|
| Intervertebral Disc Protrusion | Musculoskeletal and connective tissue disorders | MedDRA 24.0 | Systematic Assessment |
|
| Osteopenia | Musculoskeletal and connective tissue disorders | MedDRA 24.0 | Systematic Assessment |
|
| Spinal Pain | Musculoskeletal and connective tissue disorders | MedDRA 24.0 | Systematic Assessment |
|
| Pregnancy | Pregnancy, puerperium and perinatal conditions | MedDRA 24.0 | Systematic Assessment |
|
The Sponsor supports publication of clinical trial results but may request that investigators temporarily delay or alter publications in order to protect proprietary information. The Sponsor may also require that the results of multicenter studies be published only in their entirety and not as individual site data.
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
| D008661 | Metabolism, Inborn Errors |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D008064 | Lipidoses |
| D008052 | Lipid Metabolism, Inborn Errors |
| D016464 | Lysosomal Storage Diseases |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
| D052439 | Lipid Metabolism Disorders |
|
| Mild: Study Baseline |
|
|
| Moderate: Study Baseline |
|
|
| Severe: Study Baseline |
|
|
| Extreme: Study Baseline |
|
|
| None: Week 52 |
|
|
| Very Mild: Week 52 |
|
|
| Mild: Week 52 |
|
|
| Moderate: Week 52 |
|
|
| Severe: Week 52 |
|
|
| Extreme: Week 52 |
|
|
| None: Week 104 |
|
|
| Very Mild: Week 104 |
|
|
| Mild: Week 104 |
|
|
| Moderate: Week 104 |
|
|
| Severe: Week 104 |
|
|
| Extreme: Week 104 |
|
|
| None: Week 156 |
|
|
| Very mild: Week 156 |
|
|
| Mild: Week 156 |
|
|
| Moderate: Week 156 |
|
|
| Severe: Week 156 |
|
|
| Extreme: Week 156 |
|
|
| None: Week 208 |
|
|
| Very Mild: Week 208 |
|
|
| Mild: Week 208 |
|
|
| Moderate: Week 208 |
|
|
| Severe: Week 208 |
|
|
| Extreme: Week 208 |
|
|
|
| Bone Crisis (2): Study Baseline |
|
|
| Bone Crisis (>=3): Study Baseline |
|
|
| Bone Crisis (0): Week 52 |
|
|
| Bone Crisis (1): Week 52 |
|
|
| Bone Crisis (2): Week 52 |
|
|
| Bone Crisis (>=3): Week 52 |
|
|
| Bone Crisis (0): Week 104 |
|
|
| Bone Crisis (1): Week 104 |
|
|
| Bone Crisis (2): Week 104 |
|
|
| Bone Crisis (>=3): Week 104 |
|
|
| Bone Crisis (0): Week 156 |
|
|
| Bone Crisis (1): Week 156 |
|
|
| Bone Crisis (2): Week 156 |
|
|
| Bone Crisis (>=3): Week 156 |
|
|
| Bone Crisis (0): Week 208 |
|
|
| Bone Crisis (1): Week 208 |
|
|
| Bone Crisis (2): Week 208 |
|
|
| Bone Crisis (>=3): Week 208 |
|
|
|
| Total BMB Score: Change at Week 52 |
|
|
| Total BMB Score: Week 104 |
|
|
| Total BMB Score: Change at Week 104 |
|
|
| Total BMB Score: Week 156 |
|
|
| Total BMB Score: Change at Week 156 |
|
|
| Total BMB Score: Week 208 |
|
|
| Total BMB Score: Change at Week 208 |
|
|
| Total BMB Score: Week 52 |
|
|
| Total BMB Score: Change at Week 52 |
|
|
| Total BMB Score: Week 104 |
|
|
| Total BMB Score: Change at Week 104 |
|
|
| Total BMB Score: Week 156 |
|
|
| Total BMB Score: Change at Week 156 |
|
|
| Total BMB Score: Week 208 |
|
|
| Total BMB Score: Change at Week 208 |
|
|
|
| Total Spine BMD: Change at Week 52 |
|
|
| Total Spine BMD: Week 104 |
|
|
| Total Spine BMD: Change at Week 104 |
|
|
| Total Spine BMD: Week 156 |
|
|
| Total Spine BMD: Change at Week 156 |
|
|
| Total Spine BMD: Week 208 |
|
|
| Total Spine BMD: Change at Week 208 |
|
|
| Worst Total Femur BMD: Study Baseline |
|
|
| Worst Total Femur BMD: Week 52 |
|
|
| Worst Total Femur BMD: Change at Week 52 |
|
|
| Worst Total Femur BMD: Week 104 |
|
|
| Worst Total Femur BMD: Change at Week 104 |
|
|
| Worst Total Femur BMD: Week 156 |
|
|
| Worst Total Femur BMD: Change at Week 156 |
|
|
| Worst Total Femur BMD: Week 208 |
|
|
| Worst Total Femur BMD: Change at Week 208 |
|
|
| Total Spine BMD: Week 52 |
|
|
| Total Spine BMD: Change at Week 52 |
|
|
| Total Spine BMD: Week 104 |
|
|
| Total Spine BMD: Change at Week 104 |
|
|
| Total Spine BMD: Week 156 |
|
|
| Total Spine BMD: Change at Week 156 |
|
|
| Total Spine BMD: Week 208 |
|
|
| Total Spine BMD: Change at Week 208 |
|
|
| Worst Total Femur BMD: Eliglustat Baseline |
|
|
| Worst Total Femur BMD: Week 52 |
|
|
| Worst Total Femur BMD: Change at Week 52 |
|
|
| Worst Total Femur BMD: Week 104 |
|
|
| Worst Total Femur BMD: Change at Week 104 |
|
|
| Worst Total Femur BMD: Week 156 |
|
|
| Worst Total Femur BMD: Change at Week 156 |
|
|
| Worst Total Femur BMD: Week 208 |
|
|
| Worst Total Femur BMD: Change at Week 208 |
|
|
|
| Total Spine T-score: Change at Week 52 |
|
|
| Total Spine T-score: Week 104 |
|
|
| Total Spine T-score: Change at Week 104 |
|
|
| Total Spine T-score: Week 156 |
|
|
| Total Spine T-score: Change at Week 156 |
|
|
| Total Spine T-score: Week 208 |
|
|
| Total Spine T-score: Change at Week 208 |
|
|
| Worst Total Femur T-score: Study Baseline |
|
|
| Worst Total Femur T-score: Week 52 |
|
|
| Worst Total Femur T-score: Change at Week 52 |
|
|
| Worst Total Femur T-score: Week 104 |
|
|
| Worst Total Femur T-score: Change at Week 104 |
|
|
| Worst Total Femur T-score: Week 156 |
|
|
| Worst Total Femur T-score: Change at Week 156 |
|
|
| Worst Total Femur T-score: Week 208 |
|
|
| Worst Total Femur T-score: Change at Week 208 |
|
|
| Total Spine T-score: Week 52 |
|
|
| Total Spine T-score: Change at Week 52 |
|
|
| Total Spine T-score: Week 104 |
|
|
| Total Spine T-score: Change at Week 104 |
|
|
| Total Spine T-score: Week 156 |
|
|
| Total Spine T-score: Change at Week 156 |
|
|
| Total Spine T-score: Week 208 |
|
|
| Total Spine T-score: Change at Week 208 |
|
|
| Worst Total Femur T-score: Eliglustat Baseline |
|
|
| Worst Total Femur T-score: Week 52 |
|
|
| Worst Total Femur T-score: Change at Week 52 |
|
|
| Worst Total Femur T-score: Week 104 |
|
|
| Worst Total Femur T-score: Change at Week 104 |
|
|
| Worst Total Femur T-score: Week 156 |
|
|
| Worst Total Femur T-score: Change at Week 156 |
|
|
| Worst Total Femur T-score: Week 208 |
|
|
| Worst Total Femur T-score: Change at Week 208 |
|
|
|
| Total Spine Z-score: Change at Week 52 |
|
|
| Total Spine Z-score: Week 104 |
|
|
| Total Spine Z-score: Change at Week 104 |
|
|
| Total Spine Z-score: Week 156 |
|
|
| Total Spine Z-score: Change at Week 156 |
|
|
| Total Spine Z-score: Week 208 |
|
|
| Total Spine Z-score: Change at Week 208 |
|
|
| Worst Total Femur Z-score: Study Baseline |
|
|
| Worst Total Femur Z-score: Week 52 |
|
|
| Worst Total Femur Z-score: Change at Week 52 |
|
|
| Worst Total Femur Z-score: Week 104 |
|
|
| Worst Total Femur Z-score: Change at Week 104 |
|
|
| Worst Total Femur Z-score: Week 156 |
|
|
| Worst Total Femur Z-score: Change at Week 156 |
|
|
| Worst Total Femur Z-score: Week 208 |
|
|
| Worst Total Femur Z-score: Change at Week 208 |
|
|
| Total Spine Z-score: Week 52 |
|
|
| Total Spine Z-score: Change at Week 52 |
|
|
| Total Spine Z-score: Week 104 |
|
|
| Total Spine Z-score: Change at Week 104 |
|
|
| Total Spine Z-score: Week 156 |
|
|
| Total Spine Z-score: Change at Week 156 |
|
|
| Total Spine Z-score: Week 208 |
|
|
| Total Spine Z-score: Change at Week 208 |
|
|
| Worst Total Femur Z-score: Eliglustat Baseline |
|
|
| Worst Total Femur Z-score: Week 52 |
|
|
| Worst Total Femur Z-score: Change at Week 52 |
|
|
| Worst Total Femur Z-score: Week 104 |
|
|
| Worst Total Femur Z-score: Change at Week 104 |
|
|
| Worst Total Femur Z-score: Week 156 |
|
|
| Worst Total Femur Z-score: Change at Week 156 |
|
|
| Worst Total Femur Z-score: Week 208 |
|
|
| Worst Total Femur Z-score: Change at Week 208 |
|
|
|
| Spine Osteonecrosis (MRI): Week 104 |
|
|
| Spine Osteonecrosis (MRI): Week 156 |
|
|
| Spine Osteonecrosis (MRI): Week 208 |
|
|
| Spine Osteonecrosis (X-ray): Study Baseline |
|
|
| Spine Osteonecrosis (X-ray): Week 104 |
|
|
| Spine Osteonecrosis (X-ray): Week 208 |
|
|
| Femur Osteonecrosis (MRI): Study Baseline |
|
|
| Femur Osteonecrosis (MRI): Week 52 |
|
|
| Femur Osteonecrosis (MRI): Week 104 |
|
|
| Femur Osteonecrosis (MRI): Week 156 |
|
|
| Femur Osteonecrosis (MRI): Week 208 |
|
|
|
| Spine Fracture (MRI): Week 104 |
|
|
| Spine Fracture (MRI): Week 156 |
|
|
| Spine Fracture (MRI): Week 208 |
|
|
| Spine Fracture (X-ray): Study Baseline |
|
|
| Spine Fracture (X-ray): Week 104 |
|
|
| Spine Fracture (X-ray): Week 208 |
|
|
| Femur Fracture (MRI): Study Baseline |
|
|
| Femur Fracture (MRI): Week 52 |
|
|
| Femur Fracture (MRI): Week 104 |
|
|
| Femur Fracture (MRI): Week 156 |
|
|
| Femur Fracture (MRI): Week 208 |
|
|
|
| Spine Infarcts (MRI): Week 104 |
|
|
| Spine Infarcts (MRI): Week 156 |
|
|
| Spine Infarcts (MRI): Week 208 |
|
|
| Spine Infarcts (X-ray): Study Baseline |
|
|
| Spine Infarcts (X-ray): Week 104 |
|
|
| Spine Infarcts (X-ray): Week 208 |
|
|
| Femur Infarcts (MRI): Study Baseline |
|
|
| Femur Infarcts (MRI): Week 52 |
|
|
| Femur Infarcts (MRI): Week 104 |
|
|
| Femur Infarcts (MRI): Week 156 |
|
|
| Femur Infarcts (MRI): Week 208 |
|
|
|
| Spine Lytic Lesions (X-ray): Week 208 |
|
|
| Title | Measurements |
|---|---|
|
| Spine Osteonecrosis (MRI): Week 208 |
|
| Spine Osteonecrosis (X-ray): Week 104 |
|
| Spine Osteonecrosis (X-ray): Week 208 |
|
| Femur Osteonecrosis (MRI): Week 52 |
|
| Femur Osteonecrosis (MRI): Week 104 |
|
| Femur Osteonecrosis (MRI): Week 156 |
|
| Femur Osteonecrosis (MRI): Week 208 |
|
| Title | Measurements |
|---|---|
|
| Spine Fracture (MRI): Week 208 |
|
| Spine Fracture (X-ray): Week 104 |
|
| Spine Fracture (X-ray): Week 208 |
|
| Femur Fracture (MRI): Week 52 |
|
| Femur Fracture (MRI): Week 104 |
|
| Femur Fracture (MRI): Week 156 |
|
| Femur Fracture (MRI): Week 208 |
|
| Title | Measurements |
|---|---|
|
| Spine Infarcts (MRI): Week 208 |
|
| Spine Infarcts (X-ray): Week 104 |
|
| Spine Infarcts (X-ray): Week 208 |
|
| Femur Infarcts (MRI): Week 52 |
|
| Femur Infarcts (MRI): Week 104 |
|
| Femur Infarcts (MRI): Week 156 |
|
| Femur Infarcts (MRI): Week 208 |
|
|
| Change at Week 52 |
|
|
| Change at Week 78 |
|
|
| Change at Week 104 |
|
|
| Change at Week 130 |
|
|
| Change at Week 156 |
|
|
| Change at Week 182 |
|
|
| Change at Week 208 |
|
|
| Change at Week 234 |
|
|
| Change at Week 26 |
|
|
| Change at Week 52 |
|
|
| Change at Week 78 |
|
|
| Change at Week 104 |
|
|
| Change at Week 130 |
|
|
| Change at Week 156 |
|
|
| Change at Week 182 |
|
|
| Change at Week 208 |
|
|
| Change at Week 234 |
|
|
|
| Change at Week 52 |
|
|
| Change at Week 78 |
|
|
| Change at Week 104 |
|
|
| Change at Week 130 |
|
|
| Change at Week 156 |
|
|
| Change at Week 182 |
|
|
| Change at Week 208 |
|
|
| Change at Week 234 |
|
|
|
| Change at Week 52 |
|
|
| Change at Week 78 |
|
|
| Change at Week 104 |
|
|
| Change at Week 130 |
|
|
| Change at Week 156 |
|
|
| Change at Week 182 |
|
|
| Change at Week 208 |
|
|
| Change at Week 234 |
|
|
|
| Change at Week 52 |
|
|
| Change at Week 78 |
|
|
| Change at Week 104 |
|
|
| Change at Week 130 |
|
|
| Change at Week 156 |
|
|
| Change at Week 182 |
|
|
| Change at Week 208 |
|
|
| Change at Week 234 |
|
|
| Change at Week 26 |
|
|
| Change at Week 52 |
|
|
| Change at Week 78 |
|
|
| Change at Week 104 |
|
|
| Change at Week 130 |
|
|
| Change at Week 156 |
|
|
| Change at Week 182 |
|
|
| Change at Week 208 |
|
|
| Change at Week 234 |
|
|
|
| Change at Week 52 |
|
|
| Change at Week 78 |
|
|
| Change at Week 104 |
|
|
| Change at Week 130 |
|
|
| Change at Week 156 |
|
|
| Change at Week 182 |
|
|
| Change at Week 208 |
|
|
| Change at Week 234 |
|
|
| Change at Week 26 |
|
|
| Change at Week 52 |
|
|
| Change at Week 78 |
|
|
| Change at Week 104 |
|
|
| Change at Week 130 |
|
|
| Change at Week 156 |
|
|
| Change at Week 182 |
|
|
| Change at Week 208 |
|
|
| Change at Week 234 |
|
|
|
| Change at Week 52 |
|
|
| Change at Week 78 |
|
|
| Change at Week 104 |
|
|
| Change at Week 130 |
|
|
| Change at Week 156 |
|
|
| Change at Week 182 |
|
|
| Change at Week 208 |
|
|
| Change at Week 234 |
|
|
| Change at Week 26 |
|
|
| Change at Week 52 |
|
|
| Change at Week 78 |
|
|
| Change at Week 104 |
|
|
| Change at Week 130 |
|
|
| Change at Week 156 |
|
|
| Change at Week 182 |
|
|
| Change at Week 208 |
|
|
| Change at Week 234 |
|
|
|
| PCS: Change at Week 26 |
|
|
| PCS: Week 52 |
|
|
| PCS: Change at Week 52 |
|
|
| PCS: Week 78 |
|
|
| PCS: Change at Week 78 |
|
|
| PCS: Week 104 |
|
|
| PCS: Change at Week 104 |
|
|
| PCS: Week 130 |
|
|
| PCS: Change at Week 130 |
|
|
| PCS: Week 156 |
|
|
| PCS: Change at Week 156 |
|
|
| PCS: Week 182 |
|
|
| PCS: Change at Week 182 |
|
|
| PCS: Week 208 |
|
|
| PCS: Change at Week 208 |
|
|
| PCS: Week 234 |
|
|
| PCS: Change at Week 234 |
|
|
| Study Baseline: MCS |
|
|
| MCS: Week 26 |
|
|
| MCS: Change at Week 26 |
|
|
| MCS: Week 52 |
|
|
| MCS: Change at Week 52 |
|
|
| MCS: Week 78 |
|
|
| MCS: Change at Week 78 |
|
|
| MCS: Week 104 |
|
|
| MCS: Change at Week 104 |
|
|
| MCS: Week 130 |
|
|
| MCS: Change at Week 130 |
|
|
| MCS: Week 156 |
|
|
| MCS: Change at Week 156 |
|
|
| MCS: Week 182 |
|
|
| MCS: Change at Week 182 |
|
|
| MCS: Week 208 |
|
|
| MCS: Change at Week 208 |
|
|
| MCS: Week 234 |
|
|
| MCS: Change at Week 234 |
|
|
| PCS: Week 26 |
|
|
| PCS: Change at Week 26 |
|
|
| PCS: Week 52 |
|
|
| PCS: Change at Week 52 |
|
|
| PCS: Week 78 |
|
|
| PCS: Change at Week 78 |
|
|
| PCS: Week 104 |
|
|
| PCS: Change at Week 104 |
|
|
| PCS: Week 130 |
|
|
| PCS: Change at Week 130 |
|
|
| PCS: Week 156 |
|
|
| PCS: Change at Week 156 |
|
|
| PCS: Week 182 |
|
|
| PCS: Change at Week 182 |
|
|
| PCS: Week 208 |
|
|
| PCS: Change at Week 208 |
|
|
| PCS: Week 234 |
|
|
| PCS: Change at Week 234 |
|
|
| Study Baseline: MCS |
|
|
| MCS: Week 26 |
|
|
| MCS: Change at Week 26 |
|
|
| MCS: Week 52 |
|
|
| MCS: Change at Week 52 |
|
|
| MCS: Week 78 |
|
|
| MCS: Change at Week 78 |
|
|
| MCS: Week 104 |
|
|
| MCS: Change at Week 104 |
|
|
| MCS: Week 130 |
|
|
| MCS: Change at Week 130 |
|
|
| MCS: Week 156 |
|
|
| MCS: Change at Week 156 |
|
|
| MCS: Week 182 |
|
|
| MCS: Change at Week 182 |
|
|
| MCS: Week 208 |
|
|
| MCS: Change at Week 208 |
|
|
| MCS: Week 234 |
|
|
| MCS: Change at Week 234 |
|
|