| NCT ID | Title | Phase | Status | Enrollment | Velocity | Design | Start | Completion | Last Updated | Sites | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT02536755 | Phase 3b Study to Evaluate Skeletal Response to Eliglustat in Adult Patients Who Completed Phase 2 or Phase 3 Studies | PHASE3 | COMPLETED | 31 | — | — | Oct 27, 2015 | Jun 24, 2021 | Jul 15, 2022 | 4 | Canada, Russia +1 |
| NCT01074944 | A Study of Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease to Evaluate Once Daily Versus Twice Daily Dosing (EDGE) | PHASE3 | COMPLETED | 170 | — | — | Jun 1, 2010 | Oct 1, 2015 | Feb 6, 2017 | 46 | United States, Australia +15 |
| NCT02536911 | A Study of the Effects of Hepatic Impairment on the Pharmacokinetics and Tolerability of Eliglustat Tartrate | PHASE1 | COMPLETED | 24 | — | — | Sep 1, 2015 | Dec 1, 2016 | Feb 10, 2017 | 2 | United States |
| NCT02536937 | A Study of the Effects of Renal Impairment on the Pharmacokinetics and Tolerability of Eliglustat Tartrate | PHASE1 | COMPLETED | 32 | — | — | Sep 1, 2015 | Jan 1, 2017 | Mar 8, 2017 | 3 | United States |
| NCT02422654 | Taste Evaluation of Different Liquid Formulations With Eliglustat | PHASE1 | COMPLETED | 8 | — | — | Apr 1, 2015 | May 1, 2015 | May 27, 2015 | 1 | United States |
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
Observed annual incidence rate was estimated using the total number of events divided by the total years of follow-up in each specified year (for all participants). Osteonecrosis was assessed by bone MRI and X-Ray for spine and by MRI for femur.
Observed annual incidence rate was estimated using the total number of events divided by the total years of follow-up in each specified year (for all participants). Fracture was assessed by bone MRI and X-Ray for spine and by MRI for femur.
Observed annual incidence rate was estimated using the total number of events divided by the total years of follow-up in each specified year (for all participants). Infarcts were assessed by bone MRI and X-Ray for spine and by MRI for femur.
Observed annual incidence rate 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.
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.
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.
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.
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.
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.
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.
Participants were considered as stable if they met all of the following criteria: 1) no more than 2 bone crisis during PAP (with no more than 1 bone crisis during either the first 6 months or the later 6 months of the period), and were free of other clinically symptomatic bone disease during the entire 52-week PAP; 2) hemoglobin level not decreased \>1.5 g/dL from Baseline for PAP; 3) platelet count not decreased \>25% from Baseline for PAP; 4) spleen volume (in multiples of normal \[MN\]) did not increase \>25% from Baseline for PAP; 5) liver volume (in MN) did not increase \>20% from Baseline for PAP. Baseline for PAP was defined as the last assessment prior to randomization.
| Arm | Type | Description |
|---|---|---|
| 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. |
| Twice Daily (BID) Dose Regimen | EXPERIMENTAL | Patients will receive either 50 mg BID or 100 mg BID |
| Once Daily (QD) Dose Regimen | EXPERIMENTAL | Patients will receive either 100 mg QD or 200 mg QD |
| GZ385660 (healthy subjects) | EXPERIMENTAL | Single dose of eliglustat tartrate will be given under fed conditions |
| GZ385660 (subjects with mild hepatic impairment) | EXPERIMENTAL | Single dose of eliglustat tartrate will be given under fed conditions |
| GZ385660 (subjects with moderate hepatic impairment) | EXPERIMENTAL | Single dose of eliglustat tartrate will be given under fed conditions |
| GZ385660 (subjects with mild renal impairment) | EXPERIMENTAL | Single dose of eliglustat tartrate will be given under fed conditions |
| GZ385660 (subjects with moderate renal impairment) | EXPERIMENTAL | Single dose of eliglustat tartrate will be given under fed conditions |
| GZ385660 (subjects with severe renal impairment) | EXPERIMENTAL | Single dose of eliglustat tartrate will be given under fed conditions |
| Concentration 1 eliglustat in vehicle A | EXPERIMENTAL | Single dose of 5 mL solution held in the mouth for 15 seconds with swishing but with no ingestion |
| Concentration 1 eliglustat in vehicle B | EXPERIMENTAL | Single dose of 5 mL solution held in the mouth for 15 seconds with swishing but with no ingestion |
| Concentration 1 eliglustat in vehicle C | EXPERIMENTAL | Single dose of 5 mL solution held in the mouth for 15 seconds with swishing but with no ingestion |
| Concentration 1 eliglustat in vehicle D | EXPERIMENTAL | Single dose of 5 mL solution held in the mouth for 15 seconds with swishing but with no ingestion |
| Concentration 1 eliglustat in vehicle E | EXPERIMENTAL | Single dose of 5 mL solution held in the mouth for 15 seconds with swishing but with no ingestion |
| Concentration 2 eliglustat in vehicle A | EXPERIMENTAL | Single dose of 5 mL liquid formulation held in the mouth for 15 seconds with swishing but with no ingestion |
| Concentration 2 eliglustat in vehicle B | EXPERIMENTAL | Single dose of 5 mL liquid formulation held in the mouth for 15 seconds with swishing but with no ingestion |
| Concentration 2 eliglustat in vehicle C | EXPERIMENTAL | Single dose of 5 mL liquid formulation held in the mouth for 15 seconds with swishing but with no ingestion |
| Concentration 2 eliglustat in vehicle D | EXPERIMENTAL | Single dose of 5 mL liquid formulation held in the mouth for 15 seconds with swishing but with no ingestion |
| Concentration 2 eliglustat in vehicle E | EXPERIMENTAL | Single dose of 5 mL liquid formulation held in the mouth for 15 seconds with swishing but with no ingestion |
| Concentration 3 eliglustat in vehicle A | EXPERIMENTAL | Single dose of 5 mL liquid formulation held in the mouth for 15 seconds with swishing but with no ingestion |
| Concentration 3 eliglustat in vehicle B | EXPERIMENTAL | Single dose of 5 mL liquid formulation held in the mouth for 15 seconds with swishing but with no ingestion |
| Concentration 3 eliglustat in vehicle C | EXPERIMENTAL | Single dose of 5 mL liquid formulation held in the mouth for 15 seconds with swishing but with no ingestion |
| Concentration 3 eliglustat in vehicle D | EXPERIMENTAL | Single dose of 5 mL liquid formulation held in the mouth for 15 seconds with swishing but with no ingestion |
| Concentration 3 eliglustat in vehicle E | EXPERIMENTAL | Single dose of 5 mL liquid formulation held in the mouth for 15 seconds with swishing but with no ingestion |
| Name | Type | Description |
|---|---|---|
| Eliglustat, GZ385660 | DRUG | Pharmaceutical form: capsule Route of administration: oral |
| Eliglustat tartrate | DRUG | Oral Capsule in 50 mg or 100 mg dosages |
| eliglustat | DRUG | Pharmaceutical form: capsule Route of administration: oral |
Inclusion criteria : * The participant must have successfully completed the Phase 2 (GZGD00304) or a Phase 3 study (GZGD02507, GZGD02607 or GZGD03109). Successful completion was defined as participants enrolled in one of the above mentioned studies who received eliglustat through the end of the stu...