Status:
COMPLETED
A Study of Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease (ENGAGE)
Lead Sponsor:
Genzyme, a Sanofi Company
Conditions:
Gaucher Disease, Type 1
Eligibility:
All Genders
16+ years
Phase:
PHASE3
Brief Summary
This Phase 3 study was designed to confirm the efficacy and safety of eliglustat tartrate (Genz-112638) in participants with Gaucher disease Type 1.
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 (\>) ...
Eligibility Criteria
Inclusion
- The participant (and/or their parent/legal guardian) was willing and able to provide signed informed consent prior to any study-related procedures to be performed;
- The participant was at least 16 years old at the time of randomization;
- The participant had a confirmed diagnosis of Gaucher disease Type 1;
- Female participants of childbearing potential must had 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
- The participant has had a partial or total splenectomy;
- The participant had received pharmacological chaperones or miglustat within 6 months prior to randomization;
- The participant had received enzyme replacement therapy within 9 months prior to randomization;
- The participant had Type 2 or 3 Gaucher disease or was suspected of having Type 3 Gaucher disease;
- The participant had 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 might confound the study results, or, on the opinion of the investigator, might preclude participation in the study;
- The participant had tested positive for the human immunodeficiency virus (HIV) antibody, Hepatitis C antibody, or Hepatitis B surface antigen;
- The participant had received an investigational product within 30 days prior to randomization;
- The participant was pregnant or lactating.
Key Trial Info
Start Date :
November 1 2009
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
January 1 2016
Estimated Enrollment :
40 Patients enrolled
Trial Details
Trial ID
NCT00891202
Start Date
November 1 2009
End Date
January 1 2016
Last Update
March 3 2017
Active Locations (18)
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1
UCSF MS Center
San Francisco, California, United States, 94143
2
Yale University School of Medicine
New Haven, Connecticut, United States, 06510
3
Emory University Medical Genetics
Decatur, Georgia, United States, 30033
4
University of Kansas Medical Center, Division of Hematology/Oncology, Dept. of Medicine
Westwood, Kansas, United States, 66160