CTRI/2009/091/000695已完成不适用
A screening protocol to characterize the disease status of Gaucher Type I patients for potential inclusion in a subsequent phase 3 clinical study.
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 100
- 试验地点
- 4
- 主要终点
- A gaucher enzyme assay will be performed to confirm deficiency of glucocerebrosidase activity if no historic data is available
研究概览
简要总结
Short description of the primary purpose of the protocol, including a brief statement of the study hypothesis.This will be a multi-center, multi-national screening study to assess the disease status of patients with Gaucher Type 1 disease utilizing criteria to be eligible for enrollment in a subsequent Phase 3 clinical study. The assessments performed in this study are standard of care for the evaluation of the symptoms of Gaucher Type 1 disease. No study drug will be administered in this screening study.Target sample size for India is 20 patients.The date of enrollment in India is 16 March 2009
研究设计
- 研究类型
- Observational
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 (or their parent/legal guardian) is willing and able to provide written informed consent.
- •The patient has the following manifestations of Gaucher Type 1 disease identified prior to screening: A.
- •Splenomegaly B.
- •Hepatomegaly C.
- •Anemia and/or thrombocytopenia 3) The patient is 16 to 65 years of age.
- •For patients <18 years of age, the patient?s Tanner stage should be ≥ 4.
排除标准
- •The patient has had a partial splenectomy within 36 months prior to screening or has had a totalsplenectomy.2) The patient has received miglustat within 3 months prior to screening.3) The patient has received enzyme replacement therapy within 9 months prior to screening.4) The patient is known to have any evidence of neurologic (e.g., peripheral neuropathy, tremor,seizures, Parkinsonism, or cognitive impairment) or pulmonary involvement (e.g., pulmonaryhypertension) as related to Gaucher Type 1 disease.5) The patient has documentation of new pathological bone involvement (e.g. osteonecrosis,pathological fractures, etc.) or has a bone crisis (pain with acute onset which requiresimmobilization of the affected area, narcotics for relief of pain, and may be accompanied byperiosteal elevation, increased white cell count, fever, or immobility of > 3 days) in the 12 monthsprior to screening.6) The patient is transfusion-dependent.7) The patient has ever had any radiation treatment.8) The patient is known to have prior esophageal varices or liver infarction.9) The patient is known to have a clinically significant disease, other than Gaucher Type 1 disease,including cardiovascular, renal, hepatic, gastrointestinal, pulmonary, neurologic, endocrine,metabolic, or psychiatric disease, other medical condition, or serious intercurrent illness.10) The patient is known to have any of the following: Clinically significant family history (suddencardiac death in 1st or 2nd degree relatives), cardiac medical history (including myocardialinfarction [MI] in the past year), or cardiac assessments/symptoms consistent with ischemia.11) The patient is known to have any of the following: Specific arrhythmias or findings on cardiacmonitoring such as severe 1st degree atrioventricular (AV) block, any 2nd or 3rd degree AVblock, highly frequent or runs (3 or more) of atrial premature contractions (APCs) or prematureventricular contractions (PVCs), complete left and right as well as partial left bundle branch block, or prolonged QT interval.12) The patient has previously tested positive for the human immunodeficiency virus (HIV) antibody,Hepatitis C antibody, or Hepatitis B surface antigen.13) The patient has received an investigational product within 30 days prior to screening.14) The patient has a history of cancer, with the exception of basal cell carcinoma.15) The patient is pregnant or lactating.
结局指标
主要结局
A gaucher enzyme assay will be performed to confirm deficiency of glucocerebrosidase activity if no historic data is available
时间窗: Approx. 1 week
次要结局
- Spleen and liver volumes will be assessed using spiral computed tomography (CT) scan or magnetic resonance imaging (MRI) (preferred method).(Approx. 1 week)
研究者
研究点 (4)
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