EUCTR2013-001258-82-IT进行中（未招募）不适用
A phase II pilot clinical study of experimental research to evaluate thefunctional rescue of CFTR protein through proteostasis regulators - Proteostasis regulators effect in cystic fibrosis therapy
European Institute for Cystic Fibrosis Research (IERFC)0 个研究点开始时间: 2013年7月29日最近更新:
适应症
相关药物
试验速览
- 阶段
- 不适用
- 状态
- 进行中（未招募）
- 发起方
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
- •-Diagnosis of cystic fibrosis by sweat test with chloride level=60 mEq/l and confirmed by genetic analysis in order to search cystic fibrosis F508del-CFTR homozigotic mutation or F508del-CFTR combined mutations of class I and II
- •- males or females aged at least 6 years old.
- •- fertile females should accept a contraceptive method.
- •- FEV1 =40% as compared to expected value for age and sex.
- •- At least 2 sweat tests within the 3 months before Meeting 1 to
- •establish the interindividual variation of chloride level in the sweat.
- •-Informed agreement
- •- All subjects must be able to understand the aim of the study
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 60
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range 7
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
排除标准
- •- Treatment with glucocorticoids per os or via inhalationat Meeting 1 or within 4 weeks before meeting 1.
- •- Treatment with oxygen, over day or over night.
- •- Other experimental drugs.
- •- Hypersensitivity (local or general) to cysteamine or penicillamine.
- •- Modifications in the therapy with macrolides, ant-asmatic, mucolytic
- •drugs, Dornase alfa, and/or FANS, DHA within 28 days before Meeting 1.
- •However, ita can be accepted the use of some drugs (for example FANS).
- •- Lung/hepatic/other organ transplantation.
- •- Kidney or hepatic alterations at Meeting 1:
- •-AST, ALT > 5 fold normal value . -
- •Creatinine > 2 fold normal value (ULN).
- •- Pregnancy
- •- Nursing.
- •- No contraceptive method.
- •- Neurologic and psychiatric pathologies that, on the basis of the Researcher
- •experience, could interfere with protocol
研究者
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