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

This study has been completed.
Information provided by (Responsible Party):
Sanofi ( Genzyme, a Sanofi Company ) Identifier:
First received: April 30, 2009
Last updated: March 25, 2016
Last verified: March 2016
This Phase 3 study is designed to confirm the efficacy and safety of eliglustat tartrate (Genz-112638) in participants with Gaucher disease Type 1.

Condition Intervention Phase
Gaucher Disease, Type 1
Drug: Eliglustat tartrate
Drug: Placebo
Phase 3

Study Type: Interventional
Study Design: Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multi-Center Study Confirming the Efficacy and Safety of Genz-112638 in Patients With Gaucher Disease Type 1 (ENGAGE)

Resource links provided by NLM:

Further study details as provided by Sanofi:

Primary Outcome Measures:
  • 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: Baseline, Week 39 ]
    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.

Secondary Outcome Measures:
  • Hemoglobin Level [ Time Frame: Baseline ]
  • Absolute Change From Baseline in Hemoglobin Level at Week 39 [ Time Frame: Baseline, Week 39 ]
    Absolute change = hemoglobin level at Week 39 minus hemoglobin level at baseline.

  • Percent Change From Baseline in Liver Volume (in MN) at Week 39 [ Time Frame: Baseline, 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.

  • Percent Change From Baseline in Platelet Counts at Week 39 [ Time Frame: Baseline, 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.

Enrollment: 40
Study Start Date: November 2009
Study Completion Date: January 2016
Primary Completion Date: July 2012 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Active
Eliglustat tartrate
Drug: Eliglustat tartrate
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.
Other Name: Genz-112638
Placebo Comparator: Placebo
Drug: Placebo
Matching placebo capsule once daily on Day 1 followed by matching placebo capsule BID from Day 2 through Week 39.

Detailed Description:

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 is designed to determine the efficacy, safety, and pharmacokinetics (PK) of eliglustat tartrate in adult participants (>16 years) with Gaucher disease Type 1. The study consists of 2 periods: The Double-Blind Primary Analysis Period (Day 1 to Week 39) and the Open-Label Period (post-Week 39 [Day 1 of the Open-Label Period] through study completion).


Ages Eligible for Study:   16 Years and older   (Child, Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • The participant (and/or their parent/legal guardian) is willing and able to provide signed informed consent prior to any study-related procedures to be performed
  • The participant is at least 16 years old at the time of randomization
  • The participant has a confirmed diagnosis of Gaucher disease Type 1
  • Female participants of childbearing potential must have a documented negative pregnancy test prior to dosing. In addition all female participants of childbearing potential must use a medically accepted form of contraception throughout the study

Exclusion Criteria:

  • The participant has had a partial or total splenectomy
  • The participant has received pharmacological chaperones or miglustat within 6 months prior to randomization
  • The participant has received enzyme replacement therapy within 9 months prior to randomization
  • The participant has Type 2 or 3 Gaucher disease or is suspected of having Type 3 Gaucher disease
  • The participant has any clinically significant disease, other than Gaucher disease, including cardiovascular, renal, hepatic, gastrointestinal (GI), pulmonary, neurologic, endocrine, metabolic, (for example, hypokalemia, hypomagnesemia), or psychiatric disease, other medical conditions, or serious intercurrent illness that may confound the study results, or, on the opinion of the investigator, may preclude participation in the study
  • The participant has tested positive for the human immunodeficiency virus (HIV) antibody, Hepatitis C antibody, or Hepatitis B surface antigen
  • The participant has received an investigational product within 30 days prior to randomization
  • The participant is pregnant or lactating
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT00891202

  Show 24 Study Locations
Sponsors and Collaborators
Genzyme, a Sanofi Company
Study Director: Medical Monitor Genzyme, a Sanofi Company
  More Information


Publications automatically indexed to this study by Identifier (NCT Number):
Responsible Party: Genzyme, a Sanofi Company Identifier: NCT00891202     History of Changes
Other Study ID Numbers: GZGD02507  2008-005222-37  EFC12813 
Study First Received: April 30, 2009
Results First Received: August 22, 2014
Last Updated: March 25, 2016

Keywords provided by Sanofi:
acid ß-glucosidase,
D-glucosyl-N-acylsphingosine glucohydrolase,
substrate reduction therapy

Additional relevant MeSH terms:
Gaucher Disease
Lysosomal Storage Diseases, Nervous System
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Lipid Metabolism, Inborn Errors
Lysosomal Storage Diseases
Metabolic Diseases
Lipid Metabolism Disorders
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action processed this record on February 24, 2017