EUCTR2008-005223-28-FR进行中（未招募）1 期
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 been Stabilized with Cerezyme - ENCORE
适应症
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 159
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional clinical trial of medicinal product
入排标准
- 性别
- All
入选标准
- •Patients must meet all of the following inclusion criteria in order to participate in this study:
- •1. The patient is willing and able to provide signed informed consent prior to any study-related procedures to be performed.
- •2. The patient is 18 to 65 years of age at the time of randomization.
- •3. The patient’s Tanner Stage should be = 4 prior to randomization.
- •4. The patient has a diagnosis of Gaucher disease type 1 confirmed by a documented deficiency of acid ß-glucosidase activity by enzyme assay.
- •5. The patient consents to provide a blood sample for genotyping for Gaucher disease (unless the patient’s Gaucher genotype is already available), chitotriosidase, and for genotyping of cytochrome P450 2D6 (CYP2D6) to categorize the patient’s predicted rate of metabolism.
- •6. The patient has received treatment with Cerezyme for at least 3 years and has received the equivalent of = 20 U/kg to = 60 U/kg (± 5 U/kg) q2w at a stable dose (and in a manner consistent with local labeling) for the last year prior to randomization with Genz-112638. Patients should not have missed more than 4 infusions within the last year prior to randomization.
- •7. The patient has clinically stable Gaucher disease prior to randomization. Stable Gaucher disease is defined as a patient with all of the following:
- •A. No bone crisis and free of symptomatic bone disease such as bone pain attributable to osteonecrosis and/or pathological fractures within the last year, and no documentation of acute pathological bone involvement by imaging (e.g., osteonecrosis, pathological fractures, etc.) as determined in review with a
- •central bone reviewer.
- •B. Mean hemoglobin level of = 11 g/dL if female and = 12 g/dL if male at the time of randomization and, in the 6 to 12 months prior to randomization, a median hemoglobin level of =11 g/dL if female or = 12 g/dL if male that has not varied more than ± 1 g/dL.
- •C. Mean platelet count = 100,000/mm3 at the time of randomization and, in the 6 to 12 months prior to randomization, a median platelet count = 100,000/mm3 with no single value < 85,000/mm3.
- •8. Spleen volume < 10 times Normal or total splenectomy (provided the splenectomy occurred > 3 years prior to randomization).
- •9. Liver volume < 1.5 times Normal.
- •10. Male patients agree to use a medically accepted method of contraception throughout the study.
- •11. 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 contraceptive with norethindrone and ethinyl estradiol).
- •12. The patient is willing to abstain from consumption of grapefruit or grapefruit juice for 72 hours prior to administration of the first dose of Genz-112638 and throughout the duration of the study.
- •Are the trial subjects under 18? no
- •Number of subjects for this age range:
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
排除标准
- •Patients will be excluded from participation in this study if they meet any of the following exclusion criteria:
- •1. The patient received pharmacological chaperones or miglustat within 6 months prior to randomization.
- •2. The patient has had a partial or total splenectomy within 3 years prior to randomization.
- •3. 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.
- •4. The patient is transfusion-dependent.
- •5. The patient has a documented deficiency of iron, vitamin B-12, or folate that requires treatment not yet initiated or not yet stable under treatment for at least 3 months prior to randomization.
- •6. The patient has documented prior esophageal varices or liver infarction or current liver enzymes (alanine transaminase [ALT]/aspartate aminotransferase [AST]) or Total Bilirubin > 2 times the upper limit of normal (ULN), unless the patient has a diagnosis of Gilbert Syndrome.
- •7. The patient has any clinically significant disease, other than Gaucher disease, including cardiovascular, renal, hepatic, gastrointestinal, 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.
- •8. 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 atrioventricular (AV) block, complete bundle branch block, prolonged QTc interval, or sustained ventricular
- •tachycardia (VT).
- •9. The patient has tested positive for the human immunodeficiency virus (HIV) antibody, Hepatitis C antibody, or Hepatitis B surface antigen.
- •10. The patient has received an investigational product within 30 days prior to randomization.
- •11. The patient is scheduled for in-patient hospitalization, including elective surgery, during the study.
- •12. The patient has a history of cancer, with the exception of basal cell carcinoma.
- •13. The patient is pregnant or lactating.
- •14. The patient has received any medication within 30 days prior to randomization that may induce or inhibit CYP2D6 or any medication that may cause QTc interval prolongation.
研究者
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