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

Completed · Phase 3 · Has a placebo group

Conditions studied: Gaucher Disease, Type 1

In brief

This Phase 3 study was designed to confirm the efficacy and safety of eliglustat tartrate (Genz-112638) in participants with Gaucher disease Type 1.

Key facts

Study ID

NCT00891202

Run by

Genzyme, a Sanofi Company

People needed

40

Starts

2009-11-01

Expected to finish

2016-01-01

Last updated by the study team

2017-03-03

Who can join

Age: 16 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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.

You may not qualify if…

• 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.

Where it is running

• UCSF MS Center — San Francisco, California, United States
• Yale University School of Medicine — New Haven, Connecticut, United States
• Emory University Medical Genetics — Decatur, Georgia, United States
• University of Kansas Medical Center, Division of Hematology/Oncology, Dept. of Medicine — Westwood, Kansas, United States
• New York University School of Medicine, Neurology Department — New York, New York, United States
• Mount Sinai School of Medicine — New York, New York, United States
• University hospital "Alexandrovska" Sofia — Sofia, Bulgaria
• Sir Mortimer B. Davis - Jewish General Hospital — Montreal, Quebec, Canada
• Mount Sinai Hospital and the Samuel Lunenfeld Research Institute — Toronto Ontario, Canada
• Hospital de San Jose — Bogotá, Colombia
• Christian Medical College Hospital — Vellore, India
• Rabin Medical Center, Beilinson Hospital — Petach Tikvah, 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, Russia
• Institut za endokrinologiju — Belgrade, Serbia
• Hopital La-Rabta — Tunis, TN, Tunisia
• Royal Free Hospital — London, United Kingdom

Full record on ClinicalTrials.gov

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