CTRI/2009/091/000695已完成未知
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
- 未知
- 状态
- 已完成
- 入组人数
- 100
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Observational
入排标准
入选标准
- •1) The patient (or their parent/legal guardian) is willing and able to provide written informed consent.
- •2) The patient has the following manifestations of Gaucher Type 1 disease identified prior to
- •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
排除标准
- •The patient has had a partial splenectomy within 36 months prior to screening or has had a total
- •splenectomy.
- •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., pulmonary
- •hypertension) 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 requires
- •immobilization of the affected area, narcotics for relief of pain, and may be accompanied by
- •periosteal elevation, increased white cell count, fever, or immobility of > 3 days) in the 12 months
- •prior 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 (sudden
- •cardiac death in 1st or 2nd degree relatives), cardiac medical history (including myocardial
- •infarction [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 cardiac
- •monitoring such as severe 1st degree atrioventricular (AV) block, any 2nd or 3rd degree AV
- •block, highly frequent or runs (3 or more) of atrial premature contractions (APCs) or premature
- •ventricular 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.
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