A Study of Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease Who Have Reached Therapeutic Goals With Enzyme Replacement Therapy (ENCORE)

• Status: Completed
• Trial end date: 2015-06-01
• 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 who had reached therapeutic goals with enzyme replacement therapy (ERT).

• 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 18 years old at the time of randomization

• The participant had a confirmed diagnosis of Gaucher disease type 1

• The participant had received treatment with ERT for at least 3 years. Within the 9 months prior to randomization, the participant had received a total monthly dose of 30 to 130 Units/kilogram for at least 6 months

• The participant had reached Gaucher disease therapeutic goals prior to randomization

• Female participants of childbearing potential must have 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 had a partial or total splenectomy within 3 years prior to randomization

• The participant had received substrate reduction therapies for Gaucher disease within 6 months prior to randomization

• The participant had Gaucher disease type 2 or 3 or was suspected of having Gaucher disease type 3

• The participant had any clinically significant disease, other than Gaucher disease, including cardiovascular, renal, hepatic, gastrointestinal (GI), pulmonary, neurologic, endocrine, metabolic (e.g. hypokalemia, hypomagnesemia), or psychiatric disease, other medical conditions, or serious intercurrent illnesses that may confound the study results or, in the opinion of the Investigator, may 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