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| ID | Type | Description | Link |
|---|---|---|---|
| 2008-005222-37 | EudraCT Number | ||
| EFC12813 | Other Identifier | Sanofi |
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This Phase 3 study was designed to confirm the efficacy and safety of eliglustat tartrate (Genz-112638) in participants with Gaucher disease Type 1.
Gaucher disease is characterized by lysosomal accumulation of glucosylceramide due to impaired glucosylceramide hydrolysis. Type 1 Gaucher disease, the most common form accounts for greater than (>) 90% of cases and does not involve the central nervous system (CNS). Typical manifestations of Type 1 Gaucher disease include splenomegaly, hepatomegaly, thrombocytopenia, anemia, skeletal pathology and decreased quality of life. The disease manifestations are caused by the accumulations of glucosylceramide (storage material) in Gaucher cells which have infiltrated the spleen and liver as well as other tissue. Eliglustat tartrate is a small molecule developed as an oral therapy which acts to specifically inhibit production of this storage material in Gaucher cells.
This study was designed to determine the efficacy, safety, and pharmacokinetics (PK) of eliglustat tartrate in adult participants (>16 years) with Gaucher disease Type 1. The study consisted of 2 periods: The Double-Blind Primary Analysis Period (PAP [Day 1 to Week 39]) and the Long Term Treatment Period (LTTP/Open-Label Period (post-Week 39 [Day 1 of the Open-Label Period] through study completion).
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Active | Experimental | Eliglustat |
|
| Placebo | Placebo Comparator | Placebo |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Eliglustat tartrate | Drug | PAP: Eliglustat tartrate (ET) capsule 50 mg orally on Day 1 followed by ET 50 mg capsule twice daily (BID) from Day 2 to Week 4, then either ET 50 mg capsule BID (participants with Genz-99067 [active moiety of ET in plasma] trough plasma concentration >=5 ng/mL) or ET 100 mg capsule BID (participants with Genz-99067 trough plasma concentration <5 ng/mL), up to Week 39. PK assessment at Week 2 used for dose adjustment after Week 4. LTTP: Participants of the eliglustat arm in PAP who completed PAP were included in LTTP and received ET capsule 50 mg BID orally from Day 1 (post Week 39) until Week 43 followed by ET 50 mg or 100 mg capsule BID up to Week 47, then ET 50 mg or 100 mg or 150 mg capsule BID up to Week 312. Dose adjustments at Week 43 and Week 47 were based on Genz-99067 trough plasma concentrations (if trough plasma concentration <5 ng/mL: next higher dose administered; if >=5 ng/mL: same dose continued) at Week 41 & Week 45, respectively. |
| Measure | Description | Time Frame |
|---|---|---|
| PAP: Percent Change From Baseline in Spleen Volume (in Multiples of Normal [MN]) at Week 39 of the Primary Analysis Period With Eliglustat Tartrate Treatment as Compared to Placebo | Percent change in spleen volume = ([spleen volume at Week 39 minus spleen volume at baseline] divided by [spleen volume at baseline]) multiplied by 100, where all volumes are in MN. | PAP Baseline (Day 1), Week 39 |
| Measure | Description | Time Frame |
|---|---|---|
| PAP: Hemoglobin Level | PAP Baseline (Day 1) | |
| PAP: Absolute Change From Baseline in Hemoglobin Level at Week 39 | Absolute change = hemoglobin level at Week 39 minus hemoglobin level at baseline. |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Medical Monitor | Genzyme, a Sanofi Company | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| UCSF MS Center | San Francisco | California | 94143 | United States | ||
| Yale University School of Medicine |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 20439622 | Background | Lukina E, Watman N, Arreguin EA, Banikazemi M, Dragosky M, Iastrebner M, Rosenbaum H, Phillips M, Pastores GM, Rosenthal DI, Kaper M, Singh T, Puga AC, Bonate PL, Peterschmitt MJ. A phase 2 study of eliglustat tartrate (Genz-112638), an oral substrate reduction therapy for Gaucher disease type 1. Blood. 2010 Aug 12;116(6):893-9. doi: 10.1182/blood-2010-03-273151. Epub 2010 May 3. | |
| 20713962 |
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The 40 participants who met inclusion criteria received placebo or Genz-112638 (eliglustat tartrate) during 39 weeks primary analysis period (PAP). After Week 39 of the PAP, all participants who remained in the study received eliglustat tartrate in the long-term treatment period (LTTP) for up to Week 312.
A total of 72 participants were screened between 5 November 2009 and 29 July 2011, of which 32 participants were screen failure. Overall 40 participants were enrolled and the study was conducted across 18 centers in 12 countries.
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| ID | Title | Description |
|---|---|---|
| FG000 | PAP: Eliglustat | Eliglustat tartrate capsule as a single 50 milligram (mg) dose on Day 1 followed by eliglustat tartrate 50 mg capsule twice daily (BID) from Day 2 to Week 4, and then either eliglustat tartrate 50 mg capsule BID (in participants who had a Genz-99067 [active moiety of eliglustat tartrate in plasma] trough plasma concentration greater than or equal to [>=] 5 nanogram per milliliter [ng/mL]) or eliglustat tartrate 100 mg capsule BID (in participants who had a Genz-99067 trough plasma concentration less than [<] 5 ng/mL), up to Week 39. The pharmacokinetic (PK) assessment at Week 2 was used for dose adjustment after Week 4. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| PAP (Up To Week 39) |
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| Placebo | Drug | PAP: Matching placebo capsule once daily on Day 1 followed by matching placebo capsule BID from Day 2 through Week 39. LTTP: Participants of the placebo arm in PAP who completed PAP were included in LTTP and received eliglustat tartrate from Day 1 (post Week 39) up to Week 312. Day 1 (post Week 39) was considered as baseline for LTTP. On Day 1, participants received eliglustat tartrate capsule 50 mg BID orally until Week 43 followed by eliglustat tartrate 50 mg or 100 mg capsule BID up to Week 47, then eliglustat tartrate 50 mg or 100 mg or 150 mg capsule BID up to Week 312. Dose adjustments at Week 43 and Week 47 were based on Genz-99067 trough plasma concentrations (if trough plasma concentration <5 ng/mL: next higher dose administered; if >=5 ng/mL: same dose continued) at Week 41 & Week 45, respectively. |
|
| PAP Baseline (Day 1), Week 39 |
| PAP: Percent Change From Baseline in Liver Volume (in MN) at Week 39 | Percent change in liver volume = ([liver volume at Week 39 minus liver volume at baseline] divided by [liver volume at baseline]) multiplied by 100, where all volumes are in MN. | PAP Baseline (Day 1), Week 39 |
| PAP: Percent Change From Baseline in Platelet Counts at Week 39 | Percent change in platelet count = ([platelet count at Week 39 minus platelet count at baseline] divided by [platelet count at baseline]) multiplied by 100. | PAP Baseline (Day 1), Week 39 |
| LTTP: Percent Change From Baseline in Spleen Volume (in MN) at Week 234 | Percent change in spleen volume = ([spleen volume at Week 234 minus spleen volume at baseline] divided by [spleen volume at baseline]) multiplied by 100, where all volumes are in MN. Baseline values for the original placebo participants refer to Day 1 of LTTP and baseline values for the original eliglustat participants refer to the Day 1 of PAP. | PAP Baseline for Eliglustat (Originally on Eliglustat) arm, LTTP Baseline for Eliglustat (Originally on Placebo) arm, Week 234 |
| LTTP: Absolute Change From Baseline in Hemoglobin Level at Week 234 | Baseline values for the original placebo participants refer to Day 1 of LTTP and baseline values for the original eliglustat participants refer to the Day 1 of PAP. | PAP Baseline for Eliglustat (Originally on Eliglustat) arm, LTTP Baseline for Eliglustat (Originally on Placebo) arm, Week 234 |
| LTTP: Percent Change From Baseline in Liver Volume (in MN) at Week 234 | Percent change in liver volume = ([liver volume at Week 234 minus liver volume at baseline] divided by [liver volume at baseline]) multiplied by 100, where all volumes are in MN. Baseline values for the original placebo participants refer to Day 1 of LTTP and baseline values for the original eliglustat participants refer to the Day 1 of PAP. | PAP Baseline for Eliglustat (Originally on Eliglustat) arm, LTTP Baseline for Eliglustat (Originally on Placebo) arm, Week 234 |
| LTTP: Percent Change From Baseline in Platelet Counts at Week 234 | Percent change in platelet count = ([platelet count at Week 234 minus platelet count at baseline] divided by [platelet count at baseline]) multiplied by 100. Baseline values for the original placebo participants refer to Day 1 of LTTP and baseline values for the original eliglustat participants refer to the Day 1 of PAP. | PAP Baseline for Eliglustat (Originally on Eliglustat) arm, LTTP Baseline for Eliglustat (Originally on Placebo) arm, Week 234 |
| New Haven |
| Connecticut |
| 06510 |
| United States |
| Emory University Medical Genetics | Decatur | Georgia | 30033 | United States |
| University of Kansas Medical Center, Division of Hematology/Oncology, Dept. of Medicine | Westwood | Kansas | 66160 | United States |
| New York University School of Medicine, Neurology Department | New York | New York | 10016 | United States |
| Mount Sinai School of Medicine | New York | New York | 10029 | United States |
| University hospital "Alexandrovska" Sofia | Sofia | 1431 | Bulgaria |
| Sir Mortimer B. Davis - Jewish General Hospital | Montreal, Quebec | H3T 1E2 | Canada |
| Mount Sinai Hospital and the Samuel Lunenfeld Research Institute | Toronto Ontario | M5G 1X5 | Canada |
| Hospital de San Jose | Bogotá | Colombia |
| Christian Medical College Hospital | Vellore | 632004 | India |
| Rabin Medical Center, Beilinson Hospital | Petach Tikvah | 49100 | Israel |
| Hôtel-Dieu de France University Hospital | Beirut | Lebanon |
| OCA Hospital | Monterrey, Nuevo Leon | Mexico |
| Hematology Research Center of Ministry of Healthcare of the Russian Federation | Moscow | 125167 | Russia |
| Institut za endokrinologiju | Belgrade | 11000 | Serbia |
| Hopital La-Rabta | Tunis | TN | 1007 | Tunisia |
| Royal Free Hospital | London | NW3 2QG | United Kingdom |
| Background |
| Lukina E, Watman N, Arreguin EA, Dragosky M, Iastrebner M, Rosenbaum H, Phillips M, Pastores GM, Kamath RS, Rosenthal DI, Kaper M, Singh T, Puga AC, Peterschmitt MJ. Improvement in hematological, visceral, and skeletal manifestations of Gaucher disease type 1 with oral eliglustat tartrate (Genz-112638) treatment: 2-year results of a phase 2 study. Blood. 2010 Nov 18;116(20):4095-8. doi: 10.1182/blood-2010-06-293902. Epub 2010 Aug 16. |
| 17509920 | Background | McEachern KA, Fung J, Komarnitsky S, Siegel CS, Chuang WL, Hutto E, Shayman JA, Grabowski GA, Aerts JM, Cheng SH, Copeland DP, Marshall J. A specific and potent inhibitor of glucosylceramide synthase for substrate inhibition therapy of Gaucher disease. Mol Genet Metab. 2007 Jul;91(3):259-67. doi: 10.1016/j.ymgme.2007.04.001. Epub 2007 May 16. |
| 20864621 | Background | Peterschmitt MJ, Burke A, Blankstein L, Smith SE, Puga AC, Kramer WG, Harris JA, Mathews D, Bonate PL. Safety, tolerability, and pharmacokinetics of eliglustat tartrate (Genz-112638) after single doses, multiple doses, and food in healthy volunteers. J Clin Pharmacol. 2011 May;51(5):695-705. doi: 10.1177/0091270010372387. Epub 2010 Sep 23. |
| 24816856 | Background | Kamath RS, Lukina E, Watman N, Dragosky M, Pastores GM, Arreguin EA, Rosenbaum H, Zimran A, Aguzzi R, Puga AC, Norfleet AM, Peterschmitt MJ, Rosenthal DI. Skeletal improvement in patients with Gaucher disease type 1: a phase 2 trial of oral eliglustat. Skeletal Radiol. 2014 Oct;43(10):1353-60. doi: 10.1007/s00256-014-1891-9. Epub 2014 May 10. |
| 24835462 | Background | Lukina E, Watman N, Dragosky M, Pastores GM, Arreguin EA, Rosenbaum H, Zimran A, Angell J, Ross L, Puga AC, Peterschmitt JM. Eliglustat, an investigational oral therapy for Gaucher disease type 1: Phase 2 trial results after 4 years of treatment. Blood Cells Mol Dis. 2014 Dec;53(4):274-6. doi: 10.1016/j.bcmd.2014.04.002. Epub 2014 May 15. |
| 25688781 | Result | Mistry PK, Lukina E, Ben Turkia H, Amato D, Baris H, Dasouki M, Ghosn M, Mehta A, Packman S, Pastores G, Petakov M, Assouline S, Balwani M, Danda S, Hadjiev E, Ortega A, Shankar S, Solano MH, Ross L, Angell J, Peterschmitt MJ. Effect of oral eliglustat on splenomegaly in patients with Gaucher disease type 1: the ENGAGE randomized clinical trial. JAMA. 2015 Feb 17;313(7):695-706. doi: 10.1001/jama.2015.459. |
| 36739645 | Derived | Peterschmitt MJ, Foster MC, Ji AJ, Zajdel MB, Cox GF. Plasma glucosylsphingosine correlations with baseline disease burden and response to eliglustat in two clinical trials of previously untreated adults with Gaucher disease type 1. Mol Genet Metab. 2023 Mar;138(3):107527. doi: 10.1016/j.ymgme.2023.107527. Epub 2023 Jan 25. |
| 34161616 | Derived | Mistry PK, Lukina E, Ben Turkia H, Shankar SP, Baris Feldman H, Ghosn M, Mehta A, Packman S, Lau H, Petakov M, Assouline S, Balwani M, Danda S, Hadjiev E, Ortega A, Foster MC, Gaemers SJM, Peterschmitt MJ. Clinical outcomes after 4.5 years of eliglustat therapy for Gaucher disease type 1: Phase 3 ENGAGE trial final results. Am J Hematol. 2021 Sep 1;96(9):1156-1165. doi: 10.1002/ajh.26276. Epub 2021 Jul 11. |
| FG001 | PAP: Placebo | Matching placebo capsule once daily on Day 1 followed by matching placebo capsule BID from Day 2 through Week 39. |
| FG002 | LTTP: Eliglustat (Originally on Eliglustat) | Participants of the eliglustat arm in PAP who completed PAP were included in LTTP and received eliglustat tartrate capsule 50 mg BID orally from Day 1 (post Week 39) until Week 43 followed by eliglustat tartrate 50 mg or 100 mg capsule BID up to Week 47, then eliglustat tartrate 50 mg or 100 mg or 150 mg capsule BID up to Week 312. Dose adjustments at Week 43 and Week 47 were based on Genz-99067 trough plasma concentrations (if trough plasma concentration <5 ng/mL: next higher dose administered; if >=5 ng/mL: same dose continued) at Week 41 & Week 45, respectively. |
| FG003 | LTTP: Eliglustat (Originally on Placebo) | Participants of the placebo arm in PAP who completed PAP were included in LTTP and received eliglustat tartrate from Day 1 (post Week 39) up to Week 312. Day 1 (post Week 39) was considered as baseline of LTTP for this arm. On Day 1, participants received eliglustat tartrate capsule 50 mg BID orally until Week 43 followed by eliglustat tartrate 50 mg or 100 mg capsule BID up to Week 47, then eliglustat tartrate 50 mg or 100 mg or 150 mg capsule BID up to Week 312. Dose adjustments at Week 43 and Week 47 were based on Genz-99067 trough plasma concentrations (if trough plasma concentration <5 ng/mL: next higher dose administered; if >=5 ng/mL: same dose continued) at Week 41 & Week 45, respectively. |
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| NOT COMPLETED |
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| LTTP (Post-Week 39 up to Week 312) |
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Full analysis set (FAS) for PAP: included all participants who signed informed consent and received at least one dose of study drug (placebo or eliglustat).
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| ID | Title | Description |
|---|---|---|
| BG000 | PAP: Eliglustat | Eliglustat tartrate 50 mg capsule BID orally from Day 1 to Week 4, followed by eliglustat tartrate 50 mg or 100 mg capsule BID orally up to Week 39. |
| BG001 | PAP: Placebo | Matching placebo capsule once daily on Day 1 followed by matching placebo capsule BID from Day 2 through Week 39. |
| BG002 | Total | Total of all reporting groups |
| Units | Counts |
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| Participants |
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| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | years |
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| Gender | Count of Participants | Participants |
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| Race/Ethnicity, Customized | Number | participants |
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| Body Mass Index (BMI) | BMI was calculated as ([weight in kg] divided by [height in cm multiplied by 0.01]^2). | Mean | Standard Deviation | kilogram per square meter (kg/m^2) |
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| Weight | Mean | Standard Deviation | kilogram (kg) |
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| Height | Mean | Standard Deviation | centimeter (cm) |
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| 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 | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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| Primary | PAP: Percent Change From Baseline in Spleen Volume (in Multiples of Normal [MN]) at Week 39 of the Primary Analysis Period With Eliglustat Tartrate Treatment as Compared to Placebo | Percent change in spleen volume = ([spleen volume at Week 39 minus spleen volume at baseline] divided by [spleen volume at baseline]) multiplied by 100, where all volumes are in MN. | FAS for PAP included all participants who signed informed consent and received at least one dose of study drug (placebo or eliglustat). | Posted | Least Squares Mean | Standard Error | percent change | PAP Baseline (Day 1), Week 39 |
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| Secondary | PAP: Hemoglobin Level | FAS for PAP included all participants who signed informed consent and received at least one dose of study drug (placebo or eliglustat). | Posted | Mean | Standard Deviation | gram per deciliter (g/dL) | PAP Baseline (Day 1) |
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| Secondary | PAP: Absolute Change From Baseline in Hemoglobin Level at Week 39 | Absolute change = hemoglobin level at Week 39 minus hemoglobin level at baseline. | FAS for PAP included all participants who signed informed consent and received at least one dose of study drug (placebo or eliglustat). | Posted | Least Squares Mean | Standard Error | g/dL | PAP Baseline (Day 1), Week 39 |
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| Secondary | PAP: Percent Change From Baseline in Liver Volume (in MN) at Week 39 | Percent change in liver volume = ([liver volume at Week 39 minus liver volume at baseline] divided by [liver volume at baseline]) multiplied by 100, where all volumes are in MN. | FAS for PAP included all participants who signed informed consent and received at least one dose of study drug (placebo or eliglustat). | Posted | Least Squares Mean | Standard Error | percent change | PAP Baseline (Day 1), Week 39 |
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| Secondary | PAP: Percent Change From Baseline in Platelet Counts at Week 39 | Percent change in platelet count = ([platelet count at Week 39 minus platelet count at baseline] divided by [platelet count at baseline]) multiplied by 100. | FAS for PAP included all participants who signed informed consent and received at least one dose of study drug (placebo or eliglustat). | Posted | Least Squares Mean | Standard Error | percent change | PAP Baseline (Day 1), Week 39 |
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| Secondary | LTTP: Percent Change From Baseline in Spleen Volume (in MN) at Week 234 | Percent change in spleen volume = ([spleen volume at Week 234 minus spleen volume at baseline] divided by [spleen volume at baseline]) multiplied by 100, where all volumes are in MN. Baseline values for the original placebo participants refer to Day 1 of LTTP and baseline values for the original eliglustat participants refer to the Day 1 of PAP. | Intent-to-treat (ITT) population for LTTP included all participants who received at least 1 dose of eliglustat in LTTP period. Number of participants analyzed= participants evaluable for this outcome measure and had available data for baseline and Week 234 spleen volume assessment. | Posted | Mean | Standard Deviation | percent change | PAP Baseline for Eliglustat (Originally on Eliglustat) arm, LTTP Baseline for Eliglustat (Originally on Placebo) arm, Week 234 |
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| Secondary | LTTP: Absolute Change From Baseline in Hemoglobin Level at Week 234 | Baseline values for the original placebo participants refer to Day 1 of LTTP and baseline values for the original eliglustat participants refer to the Day 1 of PAP. | ITT population for LTTP included all participants who received at least 1 dose of eliglustat in LTTP period. Number of participants analyzed=participants evaluable for this outcome measure and had available data for baseline and Week 234 hemoglobin level assessment. | Posted | Mean | Standard Deviation | g/dL | PAP Baseline for Eliglustat (Originally on Eliglustat) arm, LTTP Baseline for Eliglustat (Originally on Placebo) arm, Week 234 |
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| Secondary | LTTP: Percent Change From Baseline in Liver Volume (in MN) at Week 234 | Percent change in liver volume = ([liver volume at Week 234 minus liver volume at baseline] divided by [liver volume at baseline]) multiplied by 100, where all volumes are in MN. Baseline values for the original placebo participants refer to Day 1 of LTTP and baseline values for the original eliglustat participants refer to the Day 1 of PAP. | ITT population for LTTP included all participants who received at least 1 dose of eliglustat in LTTP period. Number of participants analyzed=participants evaluable for this outcome measure and had available data for baseline and Week 234 liver volume assessment. | Posted | Mean | Standard Deviation | percent change | PAP Baseline for Eliglustat (Originally on Eliglustat) arm, LTTP Baseline for Eliglustat (Originally on Placebo) arm, Week 234 |
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| Secondary | LTTP: Percent Change From Baseline in Platelet Counts at Week 234 | Percent change in platelet count = ([platelet count at Week 234 minus platelet count at baseline] divided by [platelet count at baseline]) multiplied by 100. Baseline values for the original placebo participants refer to Day 1 of LTTP and baseline values for the original eliglustat participants refer to the Day 1 of PAP. | ITT population for LTTP included all participants who received at least 1 dose of eliglustat in LTTP period. Number of participants analyzed=participants evaluable for this outcome measure and had available data for baseline and Week 234 platelet count assessment. | Posted | Mean | Standard Deviation | percent change | PAP Baseline for Eliglustat (Originally on Eliglustat) arm, LTTP Baseline for Eliglustat (Originally on Placebo) arm, Week 234 |
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Adverse Events (AEs) were collected from the signature of the Informed Consent Form through the follow-up period (30-37 days after the last visit, which was Week 312).
Reported AEs are treatment-emergent that is AEs that developed/worsened during the 'on treatment period' (first dose of eliglustat to end of follow-up period).
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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 | PAP: Eliglustat tartrate capsule 50 mg orally on Day 1 followed by eliglustat tartrate 50 mg capsule BID from Day 2 to Week 4, then either eliglustat tartrate 50 mg capsule BID(participants with Genz-99067 trough plasma concentration>=5 ng/mL) or eliglustat tartrate 100 mg capsule BID(participants with Genz-99067 trough plasma concentration<5 ng/mL), up to Week 39. PK assessment at Week 2 used for dose adjustment after Week 4. LTTP: Participants of the eliglustat arm in PAP who completed PAP were included in LTTP & received eliglustat tartrate capsule 50 mg BID orally from Day 1(post Week 39) until Week 43 followed by eliglustat tartrate 50 mg or 100 mg capsule BID up to Week 47, then eliglustat tartrate 50 mg or 100 mg or 150 mg capsule BID up to Week 312. Dose adjustments at Week 43 & Week 47 were based on Genz-99067 trough plasma concentrations(if trough plasma concentration<5 ng/mL: next higher dose administered;if>=5 ng/mL: same dose continued) at Week 41 & Week 45, respectively. | 2 | 20 | 17 | 20 | ||
| EG001 | Placebo | PAP: Matching placebo capsule once daily on Day 1 followed by matching placebo capsule BID from Day 2 through Week 39. LTTP: Participants of the placebo arm in PAP who completed PAP were included in LTTP and received eliglustat tartrate from Day 1 (post Week 39) up to Week 312. Day 1 (post Week 39) was considered as baseline for LTTP. On Day 1, participants received eliglustat tartrate capsule 50 mg BID orally until Week 43 followed by eliglustat tartrate 50 mg or 100 mg capsule BID up to Week 47, then eliglustat tartrate 50 mg or 100 mg or 150 mg capsule BID up to Week 312. Dose adjustments at Week 43 and Week 47 were based on Genz-99067 trough plasma concentrations (if trough plasma concentration <5 ng/mL: next higher dose administered; if >=5 ng/mL: same dose continued) at Week 41 & Week 45, respectively. | 3 | 20 | 14 | 20 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Atrioventricular block | Cardiac disorders | MedDRA 18.0 | Systematic Assessment |
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| Atrioventricular block second degree | Cardiac disorders | MedDRA 18.0 | Systematic Assessment |
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| Ventricular tachycardia | Cardiac disorders | MedDRA 18.0 | Systematic Assessment |
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| Biliary colic | Hepatobiliary disorders | MedDRA 18.0 | Systematic Assessment |
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| Appendicitis | Infections and infestations | MedDRA 18.0 | Systematic Assessment |
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| Osteoarthritis | Musculoskeletal and connective tissue disorders | MedDRA 18.0 | Systematic Assessment |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Iron deficiency anaemia | Blood and lymphatic system disorders | MedDRA 18.0 | Systematic Assessment |
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| Palpitations | Cardiac disorders | MedDRA 18.0 | Systematic Assessment |
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| Eye irritation | Eye disorders | MedDRA 18.0 | Systematic Assessment |
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| Abdominal distension | Gastrointestinal disorders | MedDRA 18.0 | Systematic Assessment |
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| Abdominal pain | Gastrointestinal disorders | MedDRA 18.0 | Systematic Assessment |
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| Abdominal pain upper | Gastrointestinal disorders | MedDRA 18.0 | Systematic Assessment |
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| Diarrhoea | Gastrointestinal disorders | MedDRA 18.0 | Systematic Assessment |
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| Dry mouth | Gastrointestinal disorders | MedDRA 18.0 | Systematic Assessment |
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| Dyspepsia | Gastrointestinal disorders | MedDRA 18.0 | Systematic Assessment |
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| Gastritis | Gastrointestinal disorders | MedDRA 18.0 | Systematic Assessment |
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| Gastrooesophageal reflux disease | Gastrointestinal disorders | MedDRA 18.0 | Systematic Assessment |
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| Nausea | Gastrointestinal disorders | MedDRA 18.0 | Systematic Assessment |
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| Toothache | Gastrointestinal disorders | MedDRA 18.0 | Systematic Assessment |
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| Vomiting | Gastrointestinal disorders | MedDRA 18.0 | Systematic Assessment |
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| Asthenia | General disorders | MedDRA 18.0 | Systematic Assessment |
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| Fatigue | General disorders | MedDRA 18.0 | Systematic Assessment |
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| Oedema peripheral | General disorders | MedDRA 18.0 | Systematic Assessment |
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| Pyrexia | General disorders | MedDRA 18.0 | Systematic Assessment |
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| Seasonal allergy | Immune system disorders | MedDRA 18.0 | Systematic Assessment |
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| Bronchitis | Infections and infestations | MedDRA 18.0 | Systematic Assessment |
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| Gastroenteritis | Infections and infestations | MedDRA 18.0 | Systematic Assessment |
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| Hordeolum | Infections and infestations | MedDRA 18.0 | Systematic Assessment |
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| Nasopharyngitis | Infections and infestations | MedDRA 18.0 | Systematic Assessment |
| |
| Otitis media | Infections and infestations | MedDRA 18.0 | Systematic Assessment |
| |
| Sinusitis | Infections and infestations | MedDRA 18.0 | Systematic Assessment |
| |
| Tonsillitis | Infections and infestations | MedDRA 18.0 | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA 18.0 | Systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA 18.0 | Systematic Assessment |
| |
| Contusion | Injury, poisoning and procedural complications | MedDRA 18.0 | Systematic Assessment |
| |
| Ligament sprain | Injury, poisoning and procedural complications | MedDRA 18.0 | Systematic Assessment |
| |
| Thermal burn | Injury, poisoning and procedural complications | MedDRA 18.0 | Systematic Assessment |
| |
| Blood creatine phosphokinase increased | Investigations | MedDRA 18.0 | Systematic Assessment |
| |
| Bone density decreased | Investigations | MedDRA 18.0 | Systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA 18.0 | Systematic Assessment |
| |
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA 18.0 | Systematic Assessment |
| |
| Bone pain | Musculoskeletal and connective tissue disorders | MedDRA 18.0 | Systematic Assessment |
| |
| Joint stiffness | Musculoskeletal and connective tissue disorders | MedDRA 18.0 | Systematic Assessment |
| |
| Musculoskeletal pain | Musculoskeletal and connective tissue disorders | MedDRA 18.0 | Systematic Assessment |
| |
| Myalgia | Musculoskeletal and connective tissue disorders | MedDRA 18.0 | Systematic Assessment |
| |
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA 18.0 | Systematic Assessment |
| |
| Dizziness | Nervous system disorders | MedDRA 18.0 | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA 18.0 | Systematic Assessment |
| |
| Migraine | Nervous system disorders | MedDRA 18.0 | Systematic Assessment |
| |
| Anxiety | Psychiatric disorders | MedDRA 18.0 | Systematic Assessment |
| |
| Proteinuria | Renal and urinary disorders | MedDRA 18.0 | Systematic Assessment |
| |
| Dysmenorrhoea | Reproductive system and breast disorders | MedDRA 18.0 | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 18.0 | Systematic Assessment |
| |
| Epistaxis | Respiratory, thoracic and mediastinal disorders | MedDRA 18.0 | Systematic Assessment |
| |
| Nasal congestion | Respiratory, thoracic and mediastinal disorders | MedDRA 18.0 | Systematic Assessment |
| |
| Nasal obstruction | Respiratory, thoracic and mediastinal disorders | MedDRA 18.0 | Systematic Assessment |
| |
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA 18.0 | Systematic Assessment |
| |
| Rhinorrhoea | Respiratory, thoracic and mediastinal disorders | MedDRA 18.0 | Systematic Assessment |
| |
| Acne | Skin and subcutaneous tissue disorders | MedDRA 18.0 | Systematic Assessment |
| |
| Alopecia | Skin and subcutaneous tissue disorders | MedDRA 18.0 | Systematic Assessment |
| |
| Rash | Skin and subcutaneous tissue disorders | MedDRA 18.0 | Systematic Assessment |
| |
| Skin lesion | Skin and subcutaneous tissue disorders | MedDRA 18.0 | Systematic Assessment |
|
If no publication has occurred within 12 months of the completion of the study, the Investigator shall have the right to publish/present independently the results of the study. The Investigator shall provide the Sponsor with a copy of any such presentation/publication for comment at least 30 days before any presentation/submission for publication. If requested by the Sponsor, any presentation/submission shall be delayed up to 90 days, to allow the Sponsor to preserve its proprietary rights.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Trial Transparency Team | Sanofi | Contact-us@sanofi.com |
| ID | Term |
|---|---|
| D005776 | Gaucher Disease |
| ID | Term |
|---|---|
| D013106 | Sphingolipidoses |
| D020140 | Lysosomal Storage Diseases, Nervous System |
| D020739 | Brain Diseases, Metabolic, Inborn |
| D001928 | Brain Diseases, Metabolic |
| 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 |
Not provided
Not provided
| ID | Term |
|---|---|
| C522917 | eliglustat |
Not provided
Not provided
Not provided
| Pregnancy |
|
| Transitioned to commercial eliglustat |
|
| Male |
|
| Race: Asian |
|
| Ethnicity: Not Hispanic or Latino |
|
| Ethnicity: Hispanic or Latino |
|
|
|
|
|
Participants of the placebo arm in PAP who completed PAP were included in LTTP and received eliglustat tartrate from Day 1 (post Week 39) up to Week 312. Day 1 (post Week 39) was considered as baseline of LTTP for this arm. On Day 1, participants received eliglustat tartrate capsule 50 mg BID orally until Week 43 followed by eliglustat tartrate 50 mg or 100 mg capsule BID up to Week 47, then eliglustat tartrate 50 mg or 100 mg or 150 mg capsule BID up to Week 312. Dose adjustments at Week 43 and Week 47 were based on Genz-99067 trough plasma concentrations (if trough plasma concentration <5 ng/mL: next higher dose administered; if >=5 ng/mL: same dose continued) at Week 41 & Week 45, respectively. |
|
|
|
|
Participants of the placebo arm in PAP who completed PAP were included in LTTP and received eliglustat tartrate from Day 1 (post Week 39) up to Week 312. Day 1 (post Week 39) was considered as baseline of LTTP for this arm. On Day 1, participants received eliglustat tartrate capsule 50 mg BID orally until Week 43 followed by eliglustat tartrate 50 mg or 100 mg capsule BID up to Week 47, then eliglustat tartrate 50 mg or 100 mg or 150 mg capsule BID up to Week 312. Dose adjustments at Week 43 and Week 47 were based on Genz-99067 trough plasma concentrations (if trough plasma concentration <5 ng/mL: next higher dose administered; if >=5 ng/mL: same dose continued) at Week 41 & Week 45, respectively. |
|
|
Participants of the placebo arm in PAP who completed PAP were included in LTTP and received eliglustat tartrate from Day 1 (post Week 39) up to Week 312. Day 1 (post Week 39) was considered as baseline of LTTP for this arm. On Day 1, participants received eliglustat tartrate capsule 50 mg BID orally until Week 43 followed by eliglustat tartrate 50 mg or 100 mg capsule BID up to Week 47, then eliglustat tartrate 50 mg or 100 mg or 150 mg capsule BID up to Week 312. Dose adjustments at Week 43 and Week 47 were based on Genz-99067 trough plasma concentrations (if trough plasma concentration <5 ng/mL: next higher dose administered; if >=5 ng/mL: same dose continued) at Week 41 & Week 45, respectively. |
|
|