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

Completed · Phase 3

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 who had reached therapeutic goals with enzyme replacement therapy (ERT).

Key facts

Study ID

NCT00943111

Run by

Genzyme, a Sanofi Company

People needed

160

Starts

2009-09-01

Expected to finish

2015-06-01

Last updated by the study team

2016-11-25

Who can join

Age: 18 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 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

You may not qualify if…

• 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

Where it is running

• Tower Hematology Oncology Medical Group — Beverly Hills, California, United States
• UCSF MS Center — San Francisco, California, United States
• University of Colorado Health Science Center - Aurora — Aurora, Colorado, United States
• Yale University School of Medicine — New Haven, Connecticut, United States
• Northwest Oncology Hematology Associates PA — Coral Springs, Florida, United States
• Emory University Medical Genetics — Decatur, Georgia, United States
• Children's Memorial Hospital — Chicago, Illinois, United States
• University of Iowa Hospitals and Clinics — Iowa City, Iowa, United States
• Albany Medical Center — Albany, New York, United States
• North Shore University Medical Center — Manhasset, New York, United States
• Mount Sinai School of Medicine — New York, New York, United States
• New York University School of Medicine — New York, New York, United States
• Duke University Medical Center — Durham, North Carolina, United States
• Cincinnati Children's Hospital Medical Center — Cincinnati, Ohio, United States
• Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States
• University of Pittsburgh Medical Center — Pittsburgh, Pennsylvania, United States
• O and O Alpan LLC — Springfield, Virginia, United States
• Hospital General de Agudos J.M Ramos Mejia — Buenos Aires, Argentina
• Hospital General de Ninos Dr. Ricardo Gutierrez — Buenos Aires, Argentina
• Royal Perth Hospital — Perth, WA, Australia
• Hospital de Clinicas da Universidade Federal do Parana — Curitiba, Brazil
• Instituto de Estadual de Hematologia Arthur de Siqueria Cavalcanti — Rio de Janeiro, Brazil
• IGEIM — São Paulo, Brazil
• Mount Sinai Hospital and the Samuel Lunenfeld Research Institute — Toronto Ontario, Canada
• Abou El Reesh Children's University Hospital (El Mounira), Faculty of Medicine (Kasr Al-Aini), Cairo University Hospitals, El Mounira, Cairo, Egypt — Cairo, Egypt

Full record on ClinicalTrials.gov

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