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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

  Participant Flow

Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
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
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
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.

Reporting Groups
  Description
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.
PAP: Placebo Matching placebo capsule once daily on Day 1 followed by matching placebo capsule BID from Day 2 through Week 39.
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.
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.

Participant Flow for 2 periods

Period 1:   PAP (Up To Week 39)
    PAP: Eliglustat   PAP: Placebo   LTTP: Eliglustat (Originally on Eliglustat)   LTTP: Eliglustat (Originally on Placebo)
STARTED   20   20   0   0 
COMPLETED   19   20   0   0 
NOT COMPLETED   1   0   0   0 
Withdrawal by Subject                1                0                0                0 

Period 2:   LTTP (Post-Week 39 up to Week 312)
    PAP: Eliglustat   PAP: Placebo   LTTP: Eliglustat (Originally on Eliglustat)   LTTP: Eliglustat (Originally on Placebo)
STARTED   0   0   19   20 
COMPLETED   0   0   12   15 
NOT COMPLETED   0   0   7   5 
Withdrawal by Subject                0                0                4                0 
Pregnancy                0                0                1                0 
Transitioned to commercial eliglustat                0                0                2                5 



  Baseline Characteristics

Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
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).

Reporting Groups
  Description
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.
PAP: Placebo Matching placebo capsule once daily on Day 1 followed by matching placebo capsule BID from Day 2 through Week 39.
Total Total of all reporting groups

Baseline Measures
   PAP: Eliglustat   PAP: Placebo   Total 
Overall Participants Analyzed 
[Units: Participants]
 20   20   40 
Age 
[Units: Years]
Mean (Standard Deviation)
 31.6  (11.55)   32.1  (11.26)   31.8  (11.26) 
Gender 
[Units: Participants]
Count of Participants
     
Female      12  60.0%      8  40.0%      20  50.0% 
Male      8  40.0%      12  60.0%      20  50.0% 
Race/Ethnicity, Customized 
[Units: 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] 
[Units: Kilogram per square meter (kg/m^2)]
Mean (Standard Deviation)
 23.3  (2.74)   23.4  (3.54)   23.4  (3.13) 
Weight 
[Units: Kilogram (kg)]
Mean (Standard Deviation)
 64.8  (11.74)   68.6  (17.17)   66.7  (14.65) 
Height 
[Units: Centimeter (cm)]
Mean (Standard Deviation)
 166.2  (9.91)   170.0  (12.02)   168.1  (11.05) 


  Outcome Measures

1.  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   [ Time Frame: PAP Baseline (Day 1), Week 39 ]

2.  Secondary:   PAP: Hemoglobin Level   [ Time Frame: PAP Baseline (Day 1) ]

3.  Secondary:   PAP: Absolute Change From Baseline in Hemoglobin Level at Week 39   [ Time Frame: PAP Baseline (Day 1), Week 39 ]

4.  Secondary:   PAP: Percent Change From Baseline in Liver Volume (in MN) at Week 39   [ Time Frame: PAP Baseline (Day 1), Week 39 ]

5.  Secondary:   PAP: Percent Change From Baseline in Platelet Counts at Week 39   [ Time Frame: PAP Baseline (Day 1), Week 39 ]

6.  Secondary:   LTTP: Percent Change From Baseline in Spleen Volume (in MN) at Week 234   [ Time Frame: PAP Baseline for Eliglustat (Originally on Eliglustat) arm, LTTP Baseline for Eliglustat (Originally on Placebo) arm, Week 234 ]

7.  Secondary:   LTTP: Absolute Change From Baseline in Hemoglobin Level at Week 234   [ Time Frame: PAP Baseline for Eliglustat (Originally on Eliglustat) arm, LTTP Baseline for Eliglustat (Originally on Placebo) arm, Week 234 ]

8.  Secondary:   LTTP: Percent Change From Baseline in Liver Volume (in MN) at Week 234   [ Time Frame: PAP Baseline for Eliglustat (Originally on Eliglustat) arm, LTTP Baseline for Eliglustat (Originally on Placebo) arm, Week 234 ]

9.  Secondary:   LTTP: Percent Change From Baseline in Platelet Counts at Week 234   [ Time Frame: PAP Baseline for Eliglustat (Originally on Eliglustat) arm, LTTP Baseline for Eliglustat (Originally on Placebo) arm, Week 234 ]


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats

Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
No text entered.


  More Information

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.
The agreement is:
unchecked The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is less than or equal to 60 days. The sponsor cannot require changes to the communication and cannot extend the embargo.
unchecked The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is more than 60 days but less than or equal to 180 days. The sponsor cannot require changes to the communication and cannot extend the embargo.


Results Point of Contact:  
Name/Title: Trial Transparency Team
Organization: Sanofi
e-mail: Contact-us@sanofi.com


Publications of Results:
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