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A Study of Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease (ENGAGE)

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ClinicalTrials.gov Identifier: NCT00891202
Recruitment Status : Completed
First Posted : May 1, 2009
Results First Posted : September 3, 2014
Last Update Posted : March 3, 2017
Sponsor:
Information provided by (Responsible Party):
Sanofi ( Genzyme, a Sanofi Company )

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Condition Gaucher Disease, Type 1
Interventions Drug: Eliglustat tartrate
Drug: Placebo
Enrollment 40

Recruitment Details 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.
Pre-assignment Details 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.
Arm/Group Title PAP: Eliglustat PAP: Placebo LTTP: Eliglustat (Originally on Eliglustat) LTTP: Eliglustat (Originally on Placebo)
Hide Arm/Group Description 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. Matching placebo capsule once daily on Day 1 followed by matching placebo capsule BID from Day 2 through Week 39. 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. 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.
Period Title: PAP (Up To Week 39)
Started 20 20 0 0
Completed 19 20 0 0
Not Completed 1 0 0 0
Reason Not Completed
Withdrawal by Subject             1             0             0             0
Period Title: LTTP (Post-Week 39 up to Week 312)
Started 0 0 19 20
Completed 0 0 12 15
Not Completed 0 0 7 5
Reason Not Completed
Withdrawal by Subject             0             0             4             0
Pregnancy             0             0             1             0
Transitioned to commercial eliglustat             0             0             2             5
Arm/Group Title PAP: Eliglustat PAP: Placebo Total
Hide Arm/Group Description 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. Matching placebo capsule once daily on Day 1 followed by matching placebo capsule BID from Day 2 through Week 39. Total of all reporting groups
Overall Number of Baseline Participants 20 20 40
Hide Baseline Analysis Population Description
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).
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 20 participants 20 participants 40 participants
31.6  (11.55) 32.1  (11.26) 31.8  (11.26)
Gender  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 20 participants 20 participants 40 participants
Female
12
  60.0%
8
  40.0%
20
  50.0%
Male
8
  40.0%
12
  60.0%
20
  50.0%
Race/Ethnicity, Customized  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 20 participants 20 participants 40 participants
Race: White 19 20 39
Race: Asian 1 0 1
Ethnicity: Not Hispanic or Latino 18 20 38
Ethnicity: Hispanic or Latino 2 0 2
Body Mass Index (BMI)   [1] 
Mean (Standard Deviation)
Unit of measure:  Kilogram per square meter (kg/m^2)
Number Analyzed 20 participants 20 participants 40 participants
23.3  (2.74) 23.4  (3.54) 23.4  (3.13)
[1]
Measure Description: BMI was calculated as ([weight in kg] divided by [height in cm multiplied by 0.01]^2).
Weight  
Mean (Standard Deviation)
Unit of measure:  Kilogram (kg)
Number Analyzed 20 participants 20 participants 40 participants
64.8  (11.74) 68.6  (17.17) 66.7  (14.65)
Height  
Mean (Standard Deviation)
Unit of measure:  Centimeter (cm)
Number Analyzed 20 participants 20 participants 40 participants
166.2  (9.91) 170.0  (12.02) 168.1  (11.05)
1.Primary Outcome
Title 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
Hide Description 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.
Time Frame PAP Baseline (Day 1), Week 39
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
FAS for PAP included all participants who signed informed consent and received at least one dose of study drug (placebo or eliglustat).
Arm/Group Title PAP: Eliglustat PAP: Placebo
Hide Arm/Group Description:
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.
Matching placebo capsule once daily on Day 1 followed by matching placebo capsule BID from Day 2 through Week 39.
Overall Number of Participants Analyzed 20 20
Least Squares Mean (Standard Error)
Unit of Measure: percent change
-27.77  (2.37) 2.26  (2.37)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection PAP: Eliglustat, PAP: Placebo
Comments Analysis was performed using analysis of covariance (ANCOVA) model fitted with treatment and baseline spleen severity (low spleen severity: spleen volume less than or equal to [<=] 20 multiples of normal spleen volume, high spleen severity: spleen volume greater than [>] 20 multiples of normal spleen volume).
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.0001
Comments [Not Specified]
Method ANCOVA
Comments [Not Specified]
Method of Estimation Estimation Parameter Least Squares Mean Difference
Estimated Value -30.03
Confidence Interval (2-Sided) 95%
-36.82 to -23.24
Parameter Dispersion
Type: Standard Error of the mean
Value: 3.35
Estimation Comments [Not Specified]
2.Secondary Outcome
Title PAP: Hemoglobin Level
Hide Description [Not Specified]
Time Frame PAP Baseline (Day 1)
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
FAS for PAP included all participants who signed informed consent and received at least one dose of study drug (placebo or eliglustat).
Arm/Group Title PAP: Eliglustat PAP: Placebo
Hide Arm/Group Description:
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.
Matching placebo capsule once daily on Day 1 followed by matching placebo capsule BID from Day 2 through Week 39.
Overall Number of Participants Analyzed 20 20
Mean (Standard Deviation)
Unit of Measure: gram per deciliter (g/dL)
12.05  (1.816) 12.75  (1.629)
3.Secondary Outcome
Title PAP: Absolute Change From Baseline in Hemoglobin Level at Week 39
Hide Description Absolute change = hemoglobin level at Week 39 minus hemoglobin level at baseline.
Time Frame PAP Baseline (Day 1), Week 39
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
FAS for PAP included all participants who signed informed consent and received at least one dose of study drug (placebo or eliglustat).
Arm/Group Title PAP: Eliglustat PAP: Placebo
Hide Arm/Group Description:
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.
Matching placebo capsule once daily on Day 1 followed by matching placebo capsule BID from Day 2 through Week 39.
Overall Number of Participants Analyzed 20 20
Least Squares Mean (Standard Error)
Unit of Measure: g/dL
0.69  (0.23) -0.54  (0.23)
4.Secondary Outcome
Title PAP: Percent Change From Baseline in Liver Volume (in MN) at Week 39
Hide Description 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.
Time Frame PAP Baseline (Day 1), Week 39
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
FAS for PAP included all participants who signed informed consent and received at least one dose of study drug (placebo or eliglustat).
Arm/Group Title PAP: Eliglustat PAP: Placebo
Hide Arm/Group Description:
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.
Matching placebo capsule once daily on Day 1 followed by matching placebo capsule BID from Day 2 through Week 39.
Overall Number of Participants Analyzed 20 20
Least Squares Mean (Standard Error)
Unit of Measure: percent change
-5.20  (1.64) 1.44  (1.64)
5.Secondary Outcome
Title PAP: Percent Change From Baseline in Platelet Counts at Week 39
Hide Description Percent change in platelet count = ([platelet count at Week 39 minus platelet count at baseline] divided by [platelet count at baseline]) multiplied by 100.
Time Frame PAP Baseline (Day 1), Week 39
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
FAS for PAP included all participants who signed informed consent and received at least one dose of study drug (placebo or eliglustat).
Arm/Group Title PAP: Eliglustat PAP: Placebo
Hide Arm/Group Description:
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.
Matching placebo capsule once daily on Day 1 followed by matching placebo capsule BID from Day 2 through Week 39.
Overall Number of Participants Analyzed 20 20
Least Squares Mean (Standard Error)
Unit of Measure: percent change
32.00  (5.95) -9.06  (5.95)
6.Secondary Outcome
Title LTTP: Percent Change From Baseline in Spleen Volume (in MN) at Week 234
Hide Description 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.
Time Frame PAP Baseline for Eliglustat (Originally on Eliglustat) arm, LTTP Baseline for Eliglustat (Originally on Placebo) arm, Week 234
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title LTTP: Eliglustat (Originally on Eliglustat) LTTP: Eliglustat (Originally on Placebo)
Hide Arm/Group Description:
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.
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.
Overall Number of Participants Analyzed 7 6
Mean (Standard Deviation)
Unit of Measure: percent change
-66.9  (8.45) -64.0  (6.43)
7.Secondary Outcome
Title LTTP: Absolute Change From Baseline in Hemoglobin Level at Week 234
Hide Description 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.
Time Frame PAP Baseline for Eliglustat (Originally on Eliglustat) arm, LTTP Baseline for Eliglustat (Originally on Placebo) arm, Week 234
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title LTTP: Eliglustat (Originally on Eliglustat) LTTP: Eliglustat (Originally on Placebo)
Hide Arm/Group Description:
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.
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.
Overall Number of Participants Analyzed 7 5
Mean (Standard Deviation)
Unit of Measure: g/dL
1.1  (0.65) 1.9  (1.88)
8.Secondary Outcome
Title LTTP: Percent Change From Baseline in Liver Volume (in MN) at Week 234
Hide Description 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.
Time Frame PAP Baseline for Eliglustat (Originally on Eliglustat) arm, LTTP Baseline for Eliglustat (Originally on Placebo) arm, Week 234
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title LTTP: Eliglustat (Originally on Eliglustat) LTTP: Eliglustat (Originally on Placebo)
Hide Arm/Group Description:
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.
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.
Overall Number of Participants Analyzed 7 6
Mean (Standard Deviation)
Unit of Measure: percent change
-24.3  (11.21) -22.4  (10.77)
9.Secondary Outcome
Title LTTP: Percent Change From Baseline in Platelet Counts at Week 234
Hide Description 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.
Time Frame PAP Baseline for Eliglustat (Originally on Eliglustat) arm, LTTP Baseline for Eliglustat (Originally on Placebo) arm, Week 234
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title LTTP: Eliglustat (Originally on Eliglustat) LTTP: Eliglustat (Originally on Placebo)
Hide Arm/Group Description:
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.
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.
Overall Number of Participants Analyzed 7 5
Mean (Standard Deviation)
Unit of Measure: percent change
77.3  (28.17) 100.1  (80.69)
Time Frame 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).
Adverse Event Reporting Description 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).
 
Arm/Group Title Eliglustat Placebo
Hide Arm/Group Description 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. 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.
All-Cause Mortality
Eliglustat Placebo
Affected / at Risk (%) Affected / at Risk (%)
Total   --/--   --/-- 
Show Serious Adverse Events Hide Serious Adverse Events
Eliglustat Placebo
Affected / at Risk (%) Affected / at Risk (%)
Total   2/20 (10.00%)   3/20 (15.00%) 
Cardiac disorders     
Atrioventricular block  1  1/20 (5.00%)  0/20 (0.00%) 
Atrioventricular block second degree  1  1/20 (5.00%)  0/20 (0.00%) 
Ventricular tachycardia  1  1/20 (5.00%)  0/20 (0.00%) 
Hepatobiliary disorders     
Biliary colic  1  0/20 (0.00%)  1/20 (5.00%) 
Infections and infestations     
Appendicitis  1  0/20 (0.00%)  2/20 (10.00%) 
Musculoskeletal and connective tissue disorders     
Osteoarthritis  1  1/20 (5.00%)  0/20 (0.00%) 
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA 18.0
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Eliglustat Placebo
Affected / at Risk (%) Affected / at Risk (%)
Total   17/20 (85.00%)   14/20 (70.00%) 
Blood and lymphatic system disorders     
Iron deficiency anaemia  1  2/20 (10.00%)  1/20 (5.00%) 
Cardiac disorders     
Palpitations  1  0/20 (0.00%)  3/20 (15.00%) 
Eye disorders     
Eye irritation  1  2/20 (10.00%)  0/20 (0.00%) 
Gastrointestinal disorders     
Abdominal distension  1  3/20 (15.00%)  0/20 (0.00%) 
Abdominal pain  1  4/20 (20.00%)  2/20 (10.00%) 
Abdominal pain upper  1  1/20 (5.00%)  4/20 (20.00%) 
Diarrhoea  1  5/20 (25.00%)  1/20 (5.00%) 
Dry mouth  1  0/20 (0.00%)  2/20 (10.00%) 
Dyspepsia  1  4/20 (20.00%)  2/20 (10.00%) 
Gastritis  1  2/20 (10.00%)  1/20 (5.00%) 
Gastrooesophageal reflux disease  1  1/20 (5.00%)  4/20 (20.00%) 
Nausea  1  3/20 (15.00%)  2/20 (10.00%) 
Toothache  1  2/20 (10.00%)  1/20 (5.00%) 
Vomiting  1  1/20 (5.00%)  2/20 (10.00%) 
General disorders     
Asthenia  1  3/20 (15.00%)  1/20 (5.00%) 
Fatigue  1  2/20 (10.00%)  2/20 (10.00%) 
Oedema peripheral  1  2/20 (10.00%)  0/20 (0.00%) 
Pyrexia  1  2/20 (10.00%)  2/20 (10.00%) 
Immune system disorders     
Seasonal allergy  1  1/20 (5.00%)  2/20 (10.00%) 
Infections and infestations     
Bronchitis  1  2/20 (10.00%)  1/20 (5.00%) 
Gastroenteritis  1  1/20 (5.00%)  3/20 (15.00%) 
Hordeolum  1  0/20 (0.00%)  2/20 (10.00%) 
Nasopharyngitis  1  5/20 (25.00%)  2/20 (10.00%) 
Otitis media  1  2/20 (10.00%)  0/20 (0.00%) 
Sinusitis  1  3/20 (15.00%)  2/20 (10.00%) 
Tonsillitis  1  2/20 (10.00%)  1/20 (5.00%) 
Upper respiratory tract infection  1  4/20 (20.00%)  3/20 (15.00%) 
Urinary tract infection  1  2/20 (10.00%)  1/20 (5.00%) 
Injury, poisoning and procedural complications     
Contusion  1  2/20 (10.00%)  0/20 (0.00%) 
Ligament sprain  1  0/20 (0.00%)  2/20 (10.00%) 
Thermal burn  1  0/20 (0.00%)  2/20 (10.00%) 
Investigations     
Blood creatine phosphokinase increased  1  1/20 (5.00%)  2/20 (10.00%) 
Bone density decreased  1  0/20 (0.00%)  2/20 (10.00%) 
Musculoskeletal and connective tissue disorders     
Arthralgia  1  11/20 (55.00%)  4/20 (20.00%) 
Back pain  1  5/20 (25.00%)  2/20 (10.00%) 
Bone pain  1  2/20 (10.00%)  2/20 (10.00%) 
Joint stiffness  1  2/20 (10.00%)  0/20 (0.00%) 
Musculoskeletal pain  1  1/20 (5.00%)  2/20 (10.00%) 
Myalgia  1  2/20 (10.00%)  0/20 (0.00%) 
Pain in extremity  1  4/20 (20.00%)  4/20 (20.00%) 
Nervous system disorders     
Dizziness  1  1/20 (5.00%)  3/20 (15.00%) 
Headache  1  11/20 (55.00%)  7/20 (35.00%) 
Migraine  1  3/20 (15.00%)  0/20 (0.00%) 
Psychiatric disorders     
Anxiety  1  2/20 (10.00%)  2/20 (10.00%) 
Renal and urinary disorders     
Proteinuria  1  2/20 (10.00%)  0/20 (0.00%) 
Reproductive system and breast disorders     
Dysmenorrhoea  1  2/20 (10.00%)  0/20 (0.00%) 
Respiratory, thoracic and mediastinal disorders     
Cough  1  2/20 (10.00%)  1/20 (5.00%) 
Epistaxis  1  2/20 (10.00%)  1/20 (5.00%) 
Nasal congestion  1  2/20 (10.00%)  0/20 (0.00%) 
Nasal obstruction  1  2/20 (10.00%)  0/20 (0.00%) 
Oropharyngeal pain  1  2/20 (10.00%)  1/20 (5.00%) 
Rhinorrhoea  1  2/20 (10.00%)  0/20 (0.00%) 
Skin and subcutaneous tissue disorders     
Acne  1  2/20 (10.00%)  1/20 (5.00%) 
Alopecia  1  2/20 (10.00%)  0/20 (0.00%) 
Rash  1  0/20 (0.00%)  3/20 (15.00%) 
Skin lesion  1  1/20 (5.00%)  2/20 (10.00%) 
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA 18.0
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
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.
Results Point of Contact
Name/Title: Trial Transparency Team
Organization: Sanofi
Other Publications:
Responsible Party: Sanofi ( Genzyme, a Sanofi Company )
ClinicalTrials.gov Identifier: NCT00891202     History of Changes
Other Study ID Numbers: GZGD02507
2008-005222-37 ( EudraCT Number )
EFC12813 ( Other Identifier: Sanofi )
First Submitted: April 30, 2009
First Posted: May 1, 2009
Results First Submitted: August 22, 2014
Results First Posted: September 3, 2014
Last Update Posted: March 3, 2017