A Study of the Efficacy and Safety of Eliglustat Tartrate (Genz-112638) in Type 1 Gaucher Patients
| Status: | Active, not recruiting |
|---|---|
| Conditions: | Neurology, Endocrine, Metabolic |
| Therapuetic Areas: | Endocrinology, Neurology, Pharmacology / Toxicology |
| Healthy: | No |
| Age Range: | 18 - 65 |
| Updated: | 4/21/2016 |
| Start Date: | June 2006 |
| End Date: | December 2015 |
A Phase 2, Open-Label, Multi-Center Study Evaluating the Efficacy, Safety and Pharmacokinetics of Genz-112638 in Gaucher Type 1 Patients
Gaucher disease is a genetic disease that results in a deficiency of an enzyme acid
beta-glucosidase, also known as glucocerebrosidase. This enzyme is needed to digest a
substrate (lipid) called glucosylceramide and, to a lesser degree, glucosylsphingosine. In
participants with Gaucher disease, the liver, spleen, bone marrow and brain show increases
in lipid concentration, specifically in cells derived from the monocyte/macrophage system.
Eliglustat tartrate (Genz-112638) is an oral drug that may regulate the Gaucher disease
process by decreasing the synthesis of glucosylceramide. The primary objective of this study
is to evaluate the efficacy, safety and pharmacokinetics (PK) of eliglustat tartrate,
administered as an oral dose of either 50 milligram (mg) twice daily (BID) or 100 mg BID, to
men and women with Gaucher disease Type 1 for 52 weeks.
beta-glucosidase, also known as glucocerebrosidase. This enzyme is needed to digest a
substrate (lipid) called glucosylceramide and, to a lesser degree, glucosylsphingosine. In
participants with Gaucher disease, the liver, spleen, bone marrow and brain show increases
in lipid concentration, specifically in cells derived from the monocyte/macrophage system.
Eliglustat tartrate (Genz-112638) is an oral drug that may regulate the Gaucher disease
process by decreasing the synthesis of glucosylceramide. The primary objective of this study
is to evaluate the efficacy, safety and pharmacokinetics (PK) of eliglustat tartrate,
administered as an oral dose of either 50 milligram (mg) twice daily (BID) or 100 mg BID, to
men and women with Gaucher disease Type 1 for 52 weeks.
This study consists of several phases: screening (-28 to -1 days), dose adjustment/treatment
(Day 1 [treatment baseline] to Day 30), initial steady-state treatment (post-Day 30 through
Week 52 post-baseline), a treatment interruption period (Week 52 through approximately Week
54), long-term steady-state treatment (approximately Week 54 through study completion), and
safety follow-up (30 to 37 days after a participant withdraws from or completes the study).
The Primary Analysis Period is from baseline through Week 52. The Extension Period is from
Week 52 through study completion (that is, participant withdrawal, the study is terminated,
eliglustat tartrate becomes commercially available, or where applicable, specific regulatory
requirements have been met).
(Day 1 [treatment baseline] to Day 30), initial steady-state treatment (post-Day 30 through
Week 52 post-baseline), a treatment interruption period (Week 52 through approximately Week
54), long-term steady-state treatment (approximately Week 54 through study completion), and
safety follow-up (30 to 37 days after a participant withdraws from or completes the study).
The Primary Analysis Period is from baseline through Week 52. The Extension Period is from
Week 52 through study completion (that is, participant withdrawal, the study is terminated,
eliglustat tartrate becomes commercially available, or where applicable, specific regulatory
requirements have been met).
Inclusion Criteria:
- The participant has a diagnosis of Gaucher Type I disease and a documented deficiency
of glucocerebrosidase activity by enzyme assay and is willing and able to provide
written informed consent prior to initiating any study-related procedures
- The participant is 18 to 65 years old and weighs between 50 and 120 kilogram (kg) at
enrollment
- The participant has the following symptoms of Gaucher disease identified within 28
days of enrollment (at screening):
- Anemia - indicated by hemoglobin measurements taken during the screening phase
(8 to 10 gram per deciliter (g/dL) if female, 8 to 11 g/dL if male)
- Thrombocytopenia - indicated by platelet count measurements taken during the
screening phase (60000 to 100000 per cubic millimeter)
- Splenomegaly, as indicated by magnetic resonance imaging (MRI) or spiral
computed tomography (CT) (>= 10 multiples of normal)
- Female participants of child-bearing potential must have a documented negative serum
pregnancy test prior to dosing. Female participants agree to use a reliable method of
birth control throughout duration of trial
Exclusion Criteria:
- Participant has had a partial or total splenectomy or infarcted areas of the spleen
- Participant has documented prior bleeding varices or liver infarction
- Participant received miglustat within 12 months prior to study enrollment
- The participant has received an investigational product within 30 days prior to study
enrollment
- Participant has neurologic or pulmonary involvement
- Participant has new pathological bone involvement or bone crisis in the 12 months
prior to enrollment
- Participant is transfusion-dependent
- Participant has a documented etiology of anemia due to causes other than Gaucher
disease
- The participant has cardiac functional and/or anatomical abnormalities, a history of
cancer or tested positive for human immunodeficiency virus (HIV) antibody or
Hepatitis
- Participant has a clinically significant disease, other than Gaucher disease,
including cardiovascular, renal, hepatic, gastrointestinal, pulmonary, neurologic,
endocrine, metabolic, or psychiatric disease, other medical conditions, or serious
intercurrent illnesses that, in the opinion of the Investigator, may preclude
participation in the study
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