Status:
COMPLETED
A Study of the Efficacy and Safety of Eliglustat Tartrate (Genz-112638) in Type 1 Gaucher Patients
Lead Sponsor:
Genzyme, a Sanofi Company
Conditions:
Gaucher Disease, Type 1
Cerebroside Lipidosis Syndrome
Eligibility:
All Genders
18-65 years
Phase:
PHASE2
Brief Summary
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 gluco...
Detailed Description
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-...
Eligibility Criteria
Inclusion
- The participant had a diagnosis of Gaucher Type I disease and a documented deficiency of glucocerebrosidase activity by enzyme assay and was willing and able to provide written informed consent prior to initiating any study-related procedures;
- The participant was 18 to 65 years old and weighed between 50 and 120 kilogram (kg) at enrollment;
- The participant had 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 had a documented negative serum pregnancy test prior to dosing. Female participants agreed to use a reliable method of birth control throughout duration of trial.
Exclusion
- Participant had a partial or total splenectomy or infarcted areas of the spleen;
- Participant had documented prior bleeding varices or liver infarction;
- Participant received miglustat within 12 months prior to study enrollment;
- The participant had received an investigational product within 30 days prior to study enrollment;
- Participant had neurologic or pulmonary involvement;
- Participant had new pathological bone involvement or bone crisis in the 12 months prior to enrollment;
- Participant was transfusion-dependent;
- Participant had a documented etiology of anemia due to causes other than Gaucher disease;
- The participant had cardiac functional and/or anatomical abnormalities, a history of cancer or tested positive for human immunodeficiency virus (HIV) antibody or Hepatitis;
- Participant had 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, might preclude participation in the study.
Key Trial Info
Start Date :
June 1 2006
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
December 1 2015
Estimated Enrollment :
26 Patients enrolled
Trial Details
Trial ID
NCT00358150
Start Date
June 1 2006
End Date
December 1 2015
Last Update
February 15 2017
Active Locations (17)
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1
New York University
New York, New York, United States
2
New York, New York, United States
3
Aprillus Asistencia e Investigación
Buenos Aires, Argentina
4
Hospital de Oncologia Maria Curie
Buenos Aires, Argentina