This trial is active, not recruiting.

Condition gaucher disease, type 1
Treatments eliglustat tartrate, imiglucerase for injection
Phase phase 3
Sponsor Genzyme, a Sanofi Company
Start date October 2009
End date November 2012
Trial size 160 participants
Trial identifier NCT00943111, 2008-005223-28, GZGD02607


This Phase 3 study was designed to confirm the efficacy and safety of eliglustat tartrate (Genz-112638) in patients with Gaucher disease type 1 who have reached therapeutic goals with enzyme replacement therapy.

United States No locations recruiting
Other countries No locations recruiting

Study Design

Allocation randomized
Endpoint classification safety/efficacy study
Intervention model parallel assignment
Masking open label
Primary purpose treatment
eliglustat tartrate (Genz-112638)
eliglustat tartrate Genz-112638
Capsules: 50, 100, or 150 mg BID (twice daily)
(Active Comparator)
imiglucerase for injection Cerezyme
Intravenous (IV) Infusion, Varied Dose, Q2(bi-weekly)

Primary Outcomes

The percentage of patients who remain stable for 52 weeks (the primary analysis period)
time frame: 52 weeks

Secondary Outcomes

Absolute change in total T- and Z-scores for bone mineral density from baseline to week 52
time frame: 52 weeks
Absolute change in hemoglobin levels (g/dL) from baseline to week 52
time frame: 52 weeks
Percent change in platelet counts from baseline to week 52
time frame: 52 weeks
Percent change in spleen volume (in multiples of normal (MN)) from baseline to week 52
time frame: 52 weeks
Percent change in liver volume (in MN) from baseline to week 52
time frame: 52 weeks

Eligibility Criteria

Male or female participants at least 18 years old.

Inclusion Criteria: - The patient (and/or their parent/legal guardian) is willing and able to provide signed informed consent prior to any study-related procedures to be performed. - The patient is at least 18 years old at the time of randomization. - The patient has a confirmed diagnosis of Gaucher disease type 1. - The patient has received treatment with enzyme replacement therapy (ERT) for at least 3 years. Within the 9 months prior to randomization, the patient has received a total monthly dose of 30 to 130 U/kg for at least 6 months. - The patient has reached Gaucher disease therapeutic goals prior to randomization. - Female patients of childbearing potential must have a documented negative pregnancy test prior to dosing. In addition, all female patients of childbearing potential must use a medically accepted form of contraception throughout the study. Exclusion Criteria: - The patient has had a partial or total splenectomy within 3 years prior to randomization. - The patient has received substrate reduction therapies for Gaucher disease within 6 months prior to randomization. - The patient has Gaucher disease type 2 or 3 or is suspected of having Gaucher disease type 3. - The patient has 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 patient has tested positive for the human immunodeficiency virus (HIV) antibody, Hepatitis C antibody, or Hepatitis B surface antigen. - The patient has received an investigational product within 30 days prior to randomization. - The patient is pregnant or lactating.

Additional Information

Official title A Phase 3, Randomized, Multi-Center, Multi-National, Open-Label, Active Comparator Study to Evaluate the Efficacy and Safety of Genz-112638 in Patients With Gaucher Disease Type 1 Who Have Reached Therapeutic Goals With Enzyme Replacement Therapy
Description Gaucher disease is characterized by lysosomal accumulation of glucosylceramide due to impaired glucosylceramide hydrolysis. Gaucher disease type 1, which is the most common form, accounts for >90% of cases and does not involve the CNS. Typical manifestations of Gaucher disease type 1 include splenomegaly, hepatomegaly, thrombocytopenia, anemia, bone disease, and decreased quality of life. The disease manifestations are caused by the accumulation of glucosylceramide (storage material) in macrophages (called Gaucher cells) which have infiltrated the spleen and liver as well as other tissues. Eliglustat tartrate (Genz-112638) is a small molecule drug developed as an oral therapy which acts to specifically inhibit production of this storage material in Gaucher cells. This study is designed to determine the efficacy, safety, and pharmacokinetics (PK) of eliglustat tartrate (Genz-112638) in adult patients with Gaucher disease type 1 who have been stabilized on enzyme replacement therapy.
Trial information was received from ClinicalTrials.gov and was last updated in March 2014.
Information provided to ClinicalTrials.gov by Sanofi.