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.
Age range
18 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
Number of Participants With Mobility Status Assessments at Study Baseline, Weeks 52, 104, 156 and 208
Timeframe: 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
Timeframe: Study Baseline, Weeks 52, 104, 156 and 208
Number of Participants With Bone Crisis at Study Baseline, Weeks 52, 104, 156 and 208
Timeframe: Study Baseline, Weeks 52, 104, 156 and 208
Change From Current Study Baseline in Total Bone Marrow Burden (BMB) Scores at Weeks 52, 104, 156 and 208
Timeframe: 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
Timeframe: 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
Timeframe: 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
Timeframe: 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
Timeframe: 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
Timeframe: 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
Timeframe: 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
Timeframe: 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
Timeframe: 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
Timeframe: 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
Timeframe: 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
Timeframe: Study Baseline, Weeks 104, and 208
Observed Annual Incidence Rate for Spine and Femur Osteonecrosis at Week 52, 104, 156 and 208
Timeframe: 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
Timeframe: 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
Timeframe: 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
Timeframe: 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
Timeframe: 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
Timeframe: 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
Timeframe: 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
Timeframe: 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
Timeframe: 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
Timeframe: Eliglustat Baseline, Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234 of the current study