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

• Status: Completed
• Trial end date: 2016-01-01
• Target enrollment: 0
• Participant gender: All

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.

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Genzyme, a Sanofi Company
• Treatments: Eliglustat

Criteria

Inclusion Criteria:

• 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 Criteria:

• 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.