| Primary | Percentage of Participants Who Remained Stable for 52 Weeks During the Primary Analysis Period | For a participant to be classified as stable, the participant must have remained stable in hematological parameters (hemoglobin levels and platelet counts) and organ volumes (spleen, when applicable, and liver volumes in multiples of normal [MN]). Stable hematological parameters were defined as hemoglobin level did not decrease more than (>) 1.5 gram per deciliter (g/dL) from baseline and platelet count did not decrease >25% from baseline. Stable organ volumes were defined as spleen volume (in MN) did not increase >25% from baseline, if applicable, and liver volume (in MN) did not increase >20% from baseline. | Per protocol population for PAP included participants who were at least 80% compliant with treatment during PAP, had no major protocol deviations, and did not exhibit hematological decline as a result of medically determined etiologies other than Gaucher disease. | Posted | | Number | 95% Confidence Interval | percentage of participants | | Baseline up to Week 52 | | | | ID | Title | Description |
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| OG000 | Eliglustat: PAP | Eliglustat tartrate 50 mg capsule BID orally from Day 1 to Week 4, followed by eliglustat tartrate 50, 100 or 150 mg capsule BID orally up to Week 52. | | OG001 | Imiglucerase: PAP | Imiglucerase intravenous infusion q2w up to Week 52 in doses equivalent to participant's past ERT dose prior to any unanticipated treatment interruption, dose reduction, or regimen change. |
| | | Title | Denominators | Categories |
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| | | Title | Measurements |
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| - OG00084.8(76.2 to 91.3)
- OG00193.6(82.5 to 98.7)
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| | Group IDs | Group Description | Statistical Method | Statistical Comment | P-Value | P-Value Comment | Parameter Type | Parameter Value | Dispersion Type | Dispersion Value | Confidence Interval Sides | Confidence Interval % | CI Lower Limit | CI Upper Limit | CI Lower Limit Comment | CI Upper Limit Comment | Estimate Comment | Tested Non-Inferiority | Non-Inferiority Type | Non-Inferiority Comment | Other Analysis Description |
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| | | | | | Difference in Percentage Stable | -8.8 | | | 2-Sided | 95 | -17.6 | 4.2 | | | | Yes | Non-Inferiority or Equivalence | The sample size for study was based on expected stability rates of 95% for the Imiglucerase group and 85% for the Eliglustat group, power of 85%, a one-sided significance level of 0.025, a non-inferiority margin of 25%, and a 20% non-evaluable/drop-out rate. Eliglustat was declared non-inferior to Imiglucerase if the lower-bound of the 95% confidence interval for the difference was within the non-inferiority margin of 25%. | |
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| Primary | Percentage of Participants Who Remained Stable Annually for 4 Years During the LTTP | For a participant to be classified as stable, the participant must have remained stable in hematological parameters (hemoglobin levels and platelet counts) and organ volumes (spleen, when applicable, and liver volumes in MN). Stable hematological parameters were defined as hemoglobin level did not decrease >1.5 g/dL from baseline and platelet count did not decrease >25% from baseline. Stable organ volumes were defined as spleen volume (in MN) did not increase >25% from baseline, if applicable, and liver volume did not increase >20% from baseline. | FAS population for LTTP: included all participants who received at least 1 dose of eliglustat in the extension study period. Number of participants analyzed=participants at risk at specified time-points. Here 'n' signifies number of participants with available data for specified time-points. | Posted | | Number | | percentage of participants | | Week 52 up to week 208 | | | | ID | Title | Description |
|---|
| OG000 | Eliglustat: LTTP | Participants from both the arms of PAP who completed PAP were included in this arm of LTTP. Participants originally randomized to eliglustat in PAP continued to receive eliglustat dose, based on their Genz 99067 plasma trough concentration at Week 6. Participants originally randomized to imiglucerase received eliglustat tartrate capsule 50 mg BID orally from Week 52+1 Day to Week 56 followed by eliglustat tartrate 50 mg or 100 mg capsule BID up to Week 60, and then eliglustat tartrate 50 mg or 100 mg or 150 mg capsule BID up to 5 years. The dose adjustments after Week 56 and Week 60 were based on Genz-99067 (active moiety of eliglustat tartrate in plasma) trough plasma concentrations. |
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| Secondary | Total T-Scores for Bone Mineral Density | Images of the spine and bilateral femur were obtained by dual energy X-Ray absorptiometry (DXA) to determine T-score for each bone area and total bone mineral density. T-score compares participant's bone density with that of healthy young participant. The T-score bone density categories are: normal (score greater than [>]-1), osteopenia (score -2.5 to less than or equal to [<=] -1), and osteoporosis (score <= -2.5). | Per protocol population for PAP. Number of participants analyzed = participants with baseline T-score assessment. Here, 'n' signifies participants with baseline T-score assessment for specified bone area. | Posted | | Mean | Standard Deviation | T-score | | Baseline | | | | ID | Title | Description |
|---|
| OG000 | Eliglustat: PAP | Eliglustat tartrate 50 mg capsule BID orally from Day 1 to Week 4, followed by eliglustat tartrate 50, 100 or 150 mg capsule BID orally up to Week 52. | | OG001 | Imiglucerase: PAP | Imiglucerase intravenous infusion q2w up to Week 52 in doses equivalent to participant's past ERT dose prior to any unanticipated treatment interruption, dose reduction, or regimen change. |
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| Secondary | Absolute Change From Baseline in Total T-Scores for Bone Mineral Density at Week 52 | Images of the spine and bilateral femur were obtained by DXA to determine T-score for each bone area and total bone mineral density. T-score compares participant's bone density with that of healthy young participant. The T-score bone density categories are: normal (score >-1), osteopenia (score -2.5 to <=-1), and osteoporosis (score <= -2.5). Absolute change = T-score at Week 52 minus T-score at baseline. | Per protocol population for PAP. Number of participants analyzed = participants with both baseline and Week 52 T-score assessment. Here, 'n' signifies participants with both baseline and Week 52 T-score assessment for specified bone area. Eliglustat participants switching to imiglucerase were excluded. | Posted | | Least Squares Mean | Standard Error | T-score | | Baseline, Week 52 | | | | ID | Title | Description |
|---|
| OG000 | Eliglustat: PAP | Eliglustat tartrate 50 mg capsule BID orally from Day 1 to Week 4, followed by eliglustat tartrate 50, 100 or 150 mg capsule BID orally up to Week 52. | | OG001 | Imiglucerase: PAP | Imiglucerase intravenous infusion q2w up to Week 52 in doses equivalent to participant's past ERT dose prior to any unanticipated treatment interruption, dose reduction, or regimen change. |
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| Secondary | Total Z-Scores for Bone Mineral Density | Images of the spine and bilateral femur were obtained by DXA to determine Z-score for each bone area and total bone mineral density. The Z-score bone density categories are: normal (score >-2) and below normal (score <=-2). | Per protocol population for PAP. Number of participants analyzed = participants with baseline Z-score assessment. Here, 'n' signifies participants with baseline Z-score assessment for specified bone area. | Posted | | Mean | Standard Deviation | Z-score | | Baseline | | | | ID | Title | Description |
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| OG000 | Eliglustat: PAP | Eliglustat tartrate 50 mg capsule BID orally from Day 1 to Week 4, followed by eliglustat tartrate 50, 100 or 150 mg capsule BID orally up to Week 52. | | OG001 | Imiglucerase: PAP | Imiglucerase intravenous infusion q2w up to Week 52 in doses equivalent to participant's past ERT dose prior to any unanticipated treatment interruption, dose reduction, or regimen change. |
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| Secondary | Absolute Change From Baseline in Total Z-Scores for Bone Mineral Density at Week 52 | Images of the spine and bilateral femur were obtained by DXA to determine Z-score for each bone area and total bone mineral density. The Z-score bone density categories are: normal (score >-2) and below normal (score <=-2). Absolute change = Z-score at Week 52 minus Z-score at baseline. | Per protocol population for PAP. Number of participants analyzed = participants with both baseline and Week 52 Z-score assessment. Here, 'n' signifies participants with both baseline and Week 52 Z-score assessment for specified bone area. Eliglustat participants switching to imiglucerase were excluded. | Posted | | Least Squares Mean | Standard Error | Z-score | | Baseline, Week 52 | | | | ID | Title | Description |
|---|
| OG000 | Eliglustat: PAP | Eliglustat tartrate 50 mg capsule BID orally from Day 1 to Week 4, followed by eliglustat tartrate 50, 100 or 150 mg capsule BID orally up to Week 52. | | OG001 | Imiglucerase: PAP | Imiglucerase intravenous infusion q2w up to Week 52 in doses equivalent to participant's past ERT dose prior to any unanticipated treatment interruption, dose reduction, or regimen change. |
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| Secondary | Hemoglobin Level | | Per protocol population for PAP. Number of participants analyzed = participants with baseline hemoglobin assessment. | Posted | | Mean | Standard Deviation | gram per deciliter (g/dL) | | Baseline | | | | ID | Title | Description |
|---|
| OG000 | Eliglustat: PAP | Eliglustat tartrate 50 mg capsule BID orally from Day 1 to Week 4, followed by eliglustat tartrate 50, 100 or 150 mg capsule BID orally up to Week 52. | | OG001 | Imiglucerase: PAP | Imiglucerase intravenous infusion q2w up to Week 52 in doses equivalent to participant's past ERT dose prior to any unanticipated treatment interruption, dose reduction, or regimen change. |
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| Secondary | Absolute Change From Baseline in Hemoglobin Levels at Week 52 | Absolute change = hemoglobin level at Week 52 minus hemoglobin level at baseline. | Per protocol population for PAP. Number of participants analyzed = participants with both baseline and Week 52 hemoglobin assessment. Eliglustat participants switching to imiglucerase were excluded. | Posted | | Least Squares Mean | Standard Error | g/dL | | Baseline, Week 52 | | | | ID | Title | Description |
|---|
| OG000 | Eliglustat: PAP | Eliglustat tartrate 50 mg capsule BID orally from Day 1 to Week 4, followed by eliglustat tartrate 50, 100 or 150 mg capsule BID orally up to Week 52. | | OG001 | Imiglucerase: PAP | Imiglucerase intravenous infusion q2w up to Week 52 in doses equivalent to participant's past ERT dose prior to any unanticipated treatment interruption, dose reduction, or regimen change. |
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| Secondary | Percent Change From Baseline in Platelet Counts at Week 52 | Percent change in platelet counts = ([platelet count at Week 52 minus platelet count at baseline] divided by [platelet count at baseline]) multiplied by 100. | Per protocol population for PAP. Number of participants analyzed = participants with both baseline and Week 52 platelet assessment. Eliglustat participants switching to imiglucerase were excluded. | Posted | | Least Squares Mean | Standard Error | percent change | | Baseline, Week 52 | | | | ID | Title | Description |
|---|
| OG000 | Eliglustat: PAP | Eliglustat tartrate 50 mg capsule BID orally from Day 1 to Week 4, followed by eliglustat tartrate 50, 100 or 150 mg capsule BID orally up to Week 52. | | OG001 | Imiglucerase: PAP | Imiglucerase intravenous infusion q2w up to Week 52 in doses equivalent to participant's past ERT dose prior to any unanticipated treatment interruption, dose reduction, or regimen change. |
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| Secondary | Percent Change From Baseline in Spleen Volume (MN) at Week 52 | Percent change in spleen volume = ([spleen volume at Week 52 minus spleen volume at baseline] divided by [spleen volume at baseline]) multiplied by 100, where all volumes are in MN. | Per protocol population for PAP. Number of participants analyzed = participants with both baseline and Week 52 spleen volume assessment. Eliglustat participants switching to imiglucerase were excluded. | Posted | | Least Squares Mean | Standard Error | percent change | | Baseline, Week 52 | | | | ID | Title | Description |
|---|
| OG000 | Eliglustat: PAP | Eliglustat tartrate 50 mg capsule BID orally from Day 1 to Week 4, followed by eliglustat tartrate 50, 100 or 150 mg capsule BID orally up to Week 52. | | OG001 | Imiglucerase: PAP | Imiglucerase intravenous infusion q2w up to Week 52 in doses equivalent to participant's past ERT dose prior to any unanticipated treatment interruption, dose reduction, or regimen change. |
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| Secondary | Percent Change From Baseline in Liver Volume (in MN) at Week 52 | Percent change in liver volume = ([liver volume at Week 52 minus liver volume at baseline] divided by [liver volume at baseline]) multiplied by 100, where all volumes are in multiples of normal. | Per protocol population for PAP. Number of participants analyzed = participants with both baseline and Week 52 liver volume assessment. Eliglustat participants switching to imiglucerase were excluded. | Posted | | Least Squares Mean | Standard Error | percent change | | Baseline, Week 52 | | | | ID | Title | Description |
|---|
| OG000 | Eliglustat: PAP | Eliglustat tartrate 50 mg capsule BID orally from Day 1 to Week 4, followed by eliglustat tartrate 50, 100 or 150 mg capsule BID orally up to Week 52. | | OG001 | Imiglucerase: PAP | Imiglucerase intravenous infusion q2w up to Week 52 in doses equivalent to participant's past ERT dose prior to any unanticipated treatment interruption, dose reduction, or regimen change. |
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| Secondary | Absolute Change From Baseline in Total T-Scores for Bone Mineral Density at Week 208 | Images of the spine and bilateral femur were obtained by DXA to determine T-score for each bone area and total bone mineral density. T-score compares participant's bone density with that of healthy young participant. The T-score bone density categories are: normal (score >-1), osteopenia (score -2.5 to <=-1), and osteoporosis (score <= -2.5). Absolute change = T-score at Week 208 minus T-score at baseline. | Number of participants analyzed = participants with both baseline and Week 208 T-score assessment. | Posted | | Mean | Standard Deviation | T-Score | | Baseline, Week 208 | | | | ID | Title | Description |
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| OG000 | Eliglustat: LTTP | Participants from both the arms of PAP who completed PAP were included in this arm of LTTP. Participants originally randomized to eliglustat in PAP continued to receive eliglustat dose, based on their Genz 99067 plasma trough concentration at Week 6. Participants originally randomized to imiglucerase received eliglustat tartrate capsule 50 mg BID orally from Week 52+1 Day to Week 56 followed by eliglustat tartrate 50 mg or 100 mg capsule BID up to Week 60, and then eliglustat tartrate 50 mg or 100 mg or 150 mg capsule BID up to 5 years. The dose adjustments after Week 56 and Week 60 were based on Genz-99067 (active moiety of eliglustat tartrate in plasma) trough plasma concentrations. |
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| Secondary | Absolute Change From Baseline in Total Z-Scores for Bone Mineral Density at Week 208 | Images of the spine and bilateral femur were obtained by DXA to determine Z-score for each bone area and total bone mineral density. The Z-score bone density categories are: normal (score >-2) and below normal (score <=-2). Absolute change = Z-score at Week 208 minus Z-score at baseline. | FAS population for LTTP. Number of participants analyzed = participants with both baseline and Week 208 Z-score assessment. Here, 'n' signifies participants with both baseline and Week 208 Z-score assessment for specified bone area. | Posted | | Mean | Standard Deviation | Z-score | | Baseline, Week 208 | | | | ID | Title | Description |
|---|
| OG000 | Eliglustat: LTTP | Participants from both the arms of PAP who completed PAP were included in this arm of LTTP. Participants originally randomized to eliglustat in PAP continued to receive eliglustat dose, based on their Genz 99067 plasma trough concentration at Week 6. Participants originally randomized to imiglucerase received eliglustat tartrate capsule 50 mg BID orally from Week 52+1 Day to Week 56 followed by eliglustat tartrate 50 mg or 100 mg capsule BID up to Week 60, and then eliglustat tartrate 50 mg or 100 mg or 150 mg capsule BID up to 5 years. The dose adjustments after Week 56 and Week 60 were based on Genz-99067 (active moiety of eliglustat tartrate in plasma) trough plasma concentrations. |
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| Secondary | Absolute Change From Baseline in Hemoglobin Levels at Week 208 | Absolute change = hemoglobin level at Week 208 minus hemoglobin level at baseline. | FAS population for LTTP. Number of participants analyzed = participants with both baseline and Week 208 hemoglobin assessment. | Posted | | Mean | Standard Deviation | g/dL | | Baseline, Week 208 | | | | ID | Title | Description |
|---|
| OG000 | Eliglustat: LTTP | Participants from both the arms of PAP who completed PAP were included in this arm of LTTP. Participants originally randomized to eliglustat in PAP continued to receive eliglustat dose, based on their Genz 99067 plasma trough concentration at Week 6. Participants originally randomized to imiglucerase received eliglustat tartrate capsule 50 mg BID orally from Week 52+1 Day to Week 56 followed by eliglustat tartrate 50 mg or 100 mg capsule BID up to Week 60, and then eliglustat tartrate 50 mg or 100 mg or 150 mg capsule BID up to 5 years. The dose adjustments after Week 56 and Week 60 were based on Genz-99067 (active moiety of eliglustat tartrate in plasma) trough plasma concentrations. |
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| Secondary | Percent Change From Baseline in Platelet Counts at Week 208 | Percent change in platelet counts = ([platelet count at Week 208 minus platelet count at baseline] divided by [platelet count at baseline]) multiplied by 100. | FAS population for LTTP. Number of participants analyzed = participants with both baseline and Week 208 platelet assessment. | Posted | | Mean | Standard Deviation | percent change | | Baseline, Week 208 | | | | ID | Title | Description |
|---|
| OG000 | Eliglustat: LTTP | Participants from both the arms of PAP who completed PAP were included in this arm of LTTP. Participants originally randomized to eliglustat in PAP continued to receive eliglustat dose, based on their Genz 99067 plasma trough concentration at Week 6. Participants originally randomized to imiglucerase received eliglustat tartrate capsule 50 mg BID orally from Week 52+1 Day to Week 56 followed by eliglustat tartrate 50 mg or 100 mg capsule BID up to Week 60, and then eliglustat tartrate 50 mg or 100 mg or 150 mg capsule BID up to 5 years. The dose adjustments after Week 56 and Week 60 were based on Genz-99067 (active moiety of eliglustat tartrate in plasma) trough plasma concentrations. |
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| Secondary | Percent Change From Baseline in Spleen Volume (in MN) at Week 208 | Percent change in spleen volume = ([spleen volume at Week 208 minus spleen volume at baseline] divided by [spleen volume at baseline]) multiplied by 100, where all volumes are in MN. | FAS population for LTTP. Number of participants analyzed = participants with both baseline and Week 208 spleen volume assessment. | Posted | | Mean | Standard Deviation | percent change | | Baseline, Week 208 | | | | ID | Title | Description |
|---|
| OG000 | Eliglustat: LTTP | Participants from both the arms of PAP who completed PAP were included in this arm of LTTP. Participants originally randomized to eliglustat in PAP continued to receive eliglustat dose, based on their Genz 99067 plasma trough concentration at Week 6. Participants originally randomized to imiglucerase received eliglustat tartrate capsule 50 mg BID orally from Week 52+1 Day to Week 56 followed by eliglustat tartrate 50 mg or 100 mg capsule BID up to Week 60, and then eliglustat tartrate 50 mg or 100 mg or 150 mg capsule BID up to 5 years. The dose adjustments after Week 56 and Week 60 were based on Genz-99067 (active moiety of eliglustat tartrate in plasma) trough plasma concentrations. |
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| Secondary | Percent Change From Baseline in Liver Volume (in MN) at Week 208 | Percent change in liver volume = ([liver volume at Week 208 minus liver volume at baseline] divided by [liver volume at baseline]) multiplied by 100, where all volumes are in multiples of normal. | FAS population for LTTP. Number of participants analyzed = participants with both baseline and Week 208 liver volume assessment. | Posted | | Mean | Standard Deviation | percent change | | Baseline, Week 208 | | | | ID | Title | Description |
|---|
| OG000 | Eliglustat: LTTP | Participants from both the arms of PAP who completed PAP were included in this arm of LTTP. Participants originally randomized to eliglustat in PAP continued to receive eliglustat dose, based on their Genz 99067 plasma trough concentration at Week 6. Participants originally randomized to imiglucerase received eliglustat tartrate capsule 50 mg BID orally from Week 52+1 Day to Week 56 followed by eliglustat tartrate 50 mg or 100 mg capsule BID up to Week 60, and then eliglustat tartrate 50 mg or 100 mg or 150 mg capsule BID up to 5 years. The dose adjustments after Week 56 and Week 60 were based on Genz-99067 (active moiety of eliglustat tartrate in plasma) trough plasma concentrations. |
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