CTRI/2009/091/000689已完成3 期
A Phase 3 Randomized, Double-Blind, Placebo-Controlled, Multi-Center Study Confirming the Efficacy and Safety of Genz-112638 in Patients With Gaucher Disease Type 1
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 28
- 试验地点
- 4
- 主要终点
- The Primary objective is to compare the effects of Genz-112638 as compared to placebo in patients with Type 1 Gaucher Disease
研究概览
简要总结
This Phase 3, Study was designed to confirm the Efficacy and Safety of Genz-112638 in Patients with Gaucher Disease Type 1. Gaucher disease is characterised by lysosomal accumulation of glucosylceramide due to impaired glucosylceramide hydrolysis. Gaucher Disease Type 1, 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, skeletal pathology and decreased quality of life. The disease manifestations are caused by accumulation of glucosylceramide (storage material) in Gaucher cells which infiltrate the spleen and liver as well as other tissue. Genz-112638 is a small molecule developed as an oral therapy which acts to specifically inhibit production of this storage material. This study is designed to determine the efficacy, safety, and pharmacokinetics (PK) of Genz-112638 in adult patients (¡Ý 16 years) with Gaucher Disease type 1 Target Sample Size for India - 12 patients. We anticipate to enroll first patient from India, ,and globally, on 13 Oct 2009.
研究设计
- 研究类型
- Interventional
- 盲法
- Participant, Investigator and Outcome Assessor Blinded
入排标准
- 年龄范围
- 16.00 Year(s) 至 65.00 Year(s)（—）
- 性别
- All
入选标准
- •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 16 years old at the time of randomization.
- •The patient¡¯s Tanner Stage should be ¡Ý 4 prior to randomization.
- •The patient has a diagnosis of Gaucher disease type 1 confirmed by a documented deficiency of acid ¦Â-glucosidase activity by enzyme assay.
- •The patient has the following symptoms of Gaucher disease during the Screening period: A.
- •At least one of the following laboratory abnormalities:
- •Hemoglobin level of 8.0 to 11.0 g/dL if female or 8.0 to 12.0 g/dL if male (the mean of 2 measurements from separate blood samples collected at least 24 hours apart during Screening).
- •Platelet count of 50,000 to 130,000/mm3 (the mean of 2 measurements from separate blood samples collected at least 24 hours apart during Screening).
- •Splenomegaly (spleen volume of 6 to 30 MN).
- •The patient consents to provide a blood sample to Genzyme for genotyping for Gaucher disease, chitotriosidase, and CYP2D6 (to categorize the patient¡¯s predicted rate of metabolism), unless the patient¡¯s genotypes for Gaucher disease, chitotriosidase, and CYP2D6 are already available.
- •Female patients of childbearing potential must have a documented negative pregnancy test prior to randomization.
- •In addition, all female patients of childbearing potential must use a medically accepted form of contraception throughout the study (either a barrier method or hormonal contraceptive with ethinyl estradiol and norethindrone or similar active components).
- •The patient is willing to abstain from consumption of grapefruit or grapefruit juice for 72 hours prior to administration of the first dose of study medication and throughout the duration of the Double-Blind Primary Analysis Period.
排除标准
- •The patient has had a partial or total splenectomy.
- •The patient has received pharmacological chaperone or substrate reduction therapies for Gaucher disease within 6 months prior to randomization.
- •The patient has received enzyme replacement therapy for Gaucher disease within 9 months prior to randomization.
- •The patient has any evidence of neurologic (e.g., peripheral neuropathy, tremor, seizures, Parkinsonism, or cognitive impairment) or pulmonary involvement (e.g., pulmonary hypertension) as related to Gaucher disease.
- •The patient has current symptomatic bone disease such as bone pain attributable to osteonecrosis and/or pathologic fracture, or has had a bone crisis in the 12 months prior to randomization.
- •The patient is transfusion-dependent.
- •The patient has the following laboratory abnormalities during the Screening period: A.
- •Hemoglobin level 8 g/dL (the mean of 2 measurements from separate blood samples collected at least 24 hours apart during Screening).
- •Platelet count of 50,000/mm3 (the mean of 2 measurements from separate blood samples collected at least 24 hours apart during Screening).
- •The patient has documented anemia due to causes other than Gaucher disease that requires treatment not yet initiated or not yet stable under treatment for at least 3 months (e.g., iron, vitamin B-12, and/or folate deficiency) prior to randomization.
- •The patient has documented thalassemia minor or sickle cell trait with a platelet count of 50,000 or 130,000/mm
- •The patient has ever had any radiation treatment in the abdominal region.
- •The patient has documented prior esophageal varices or liver infarction or current liver enzymes (alanine aminotransferase [ALT]/ aspartate aminotransferase [AST]) or total bilirubin 2 times the upper limit of normal (ULN), unless the patient has a diagnosis of Gilbert Syndrome.
- •The patient has any clinically significant disease, other than Gaucher disease, including cardiovascular, renal, hepatic, GI, pulmonary, neurologic, endocrine, metabolic (e.g. hypokalemia, hypomagnesemia), or psychiatric disease, other medical conditions, or serious intercurrent illnesses that, in the opinion of the Investigator, may preclude participation in the study.
- •The patient is known to have any of the following: Clinically significant coronary artery disease including history of myocardial infarction [MI] or ongoing signs or symptoms consistent with cardiac ischemia or heart failure; or clinically significant arrhythmias or conduction defect such as 2nd or 3rd degree AV block, complete bundle branch block, prolonged QTc interval, or sustained ventricular tachycardia (VT).
- •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 scheduled for in-patient hospitalization, including elective surgery, during the study.
- •The patient is pregnant or lactating.
- •The patient has received any medication that may cause QTc interval prolongation within 30 days prior to randomization.
- •The patient has received (acute or chronic) treatment with a CYP3A4 or CYP2D6 inducer within 30 days prior to randomization.
- •The patient is not a CYP2D6 poor metabolizer, and has received any medication that is a strong inhibitor of CYP3A4 or CYP2D6 within 30 days prior to randomization, except where a patient has been receiving chronic treatment with either a strong inhibitor of CYP3A4 or a strong inhibitor of CYP2D6 (but not both medications) for at least 30 days prior to randomization and plans to continue on the same dosing regimen during the Double-Blind Primary Analysis Period.
- •The patient is a CYP2D6 poor metabolizer and has received (acute or chronic) treatment with a strong inhibitor of CYP3A4 within 30 days prior to randomization.
结局指标
主要结局
The Primary objective is to compare the effects of Genz-112638 as compared to placebo in patients with Type 1 Gaucher Disease
时间窗: Time Frame: 39 weeks
次要结局
- The secondary objectives for this study include an evaluation of safety and the effects on disease manifestations and disease specific bio-markers(Time Frame: 39 weeks)
研究者
研究点 (4)
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