EUCTR2005-004732-42-IT进行中（未招募）不适用
A Phase 2, Open-Label, Multi-Center Study Evaluating the Efficacy, Safety and Pharmacokinetics of Genz-112638 in Gaucher Type 1 Patients - ND
试验速览
- 阶段
- 不适用
- 状态
- 进行中（未招募）
- 入组人数
- 20
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
- •1)The patient is willing and able to provide written informed consent prior to initiating any study-related procedures.
- •2)The patient has a diagnosis of Gaucher Type I disease and a documented deficiency of glucocerebrosidase activity by enzyme assay.
- •3)The patient has the following symptoms of Gaucher disease identified within 28 days of enrollment (at Screening):
- •a.At least one of the following laboratory abnormalities:
- •-Hemoglobin 8.0 to 10.0 g/dL if female, or 8.0 to 11.0 g/dL if male (the mean of 2 measurements from separate blood draws taken at least 24 hours apart during Screening).
- •- Platelet count 60,000 to 100,000/mm3, (the mean of 2 measurements from separate blood draws taken at least 24 hours apart during Screening).
- •b.Splenomegaly, by MRI or spiral computed tomography (CT), defined as spleen volume 10 x normal.
- •4)The patient consents to provide a blood sample to Genzyme for genotyping for Gaucher disease, chitotriosidase, and for genetic assessment of cytochrome P450 (e.g., 2D6 and other isoenzymes).
- •5)The patient is 18 to 65 years old at enrollment.
- •6)Male patients agree to use an appropriate barrier method of birth control from Screening through the 28-day study follow-up period (i.e., approximately 14 months).
- •7)Female patients of child-bearing potential must have a documented negative serum pregnancy test prior to dosing. In addition, all female patients of child-bearing potential must use a medically accepted method of contraception, from screening through the 28-day study follow-up period (i.e., approximately 14 months).
- •8)Patient weighs between 50 and 120 kg at enrollment.
- •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
- •1) The patient has had a partial or total splenectomy. 2) The patient has a hemoglobin level < 8.0 g/dL or platelet level < 60,000/mm3 (each calculated as the mean of 2 separate blood measurements taken at least 24 hours apart during Screening). 3) The patient received miglustat at any time prior to enrollment. 4) The patient received enzyme replacement therapy (ERT) or corticosteroids for Gaucher disease within 12 months prior to enrollment. 5) The patient received bisphosphonates for Gaucher disease within 3 months prior to enrollment. 6) The patient has documentation of infarcted areas of the spleen. 7) The patient has any evidence of neurologic (peripheral neuropathy, tremor, seizures, Parkinsonism or cognitive impairment) or pulmonary involvement (e.g., pulmonary hypertension). 8) The patient has documentation of new pathological bone involvement (osteonecrosis, pathological fractures, aseptic necrosis, lytic lesions) (as assessed by X-ray or MRI) or bone crisis in the 12 months prior to enrollment. 9) The patient is transfusion-dependent. 10) The patient has a documented etiology of anemia due to causes other than Gaucher disease (e.g., iron, vitamin B12, folate deficiency, or hemoglobinopathies). 11) The patient has ever had any radiation treatment. 12) The patient has documented prior bleeding varices or liver infarction. 13) The patient 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. 14) The patient has clinically significant electrocardiographic (ECG) or echocardiographic findings at the time of screening. 15) The patient has tested positive for human immunodeficiency virus (HIV) antibody, Hepatitis C antibody, or Hepatitis B surface antigen. 16) The patient has received an investigational product within 30 days prior to study enrollment. 17) The patient is scheduled for in-patient hospitalization, including elective surgery, during the study. 18) The patient has a history of cancer. 19) The patient is pregnant or lactating. 20) The patient has received any medication within 30 days prior to enrollment that may induce or inhibit CYP450 2D6, or cause QT interval prolongation (e.g., amiodarone, disopyramide, dofetilide, ibutilide, procainamide, quinidine, sotalol, thioridazine, etc. [Al-Khatib, 2003, JAMA])
研究者
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