EUCTR2018-001942-33-IT进行中（未招募）1 期
A personalized medicine approach for beta-thalassemia transfusion dependent patients: testing SIROLIMUS in a first pilot clinical trial. - SIRTHALACLI
Rare Partners s.r.l. Impresa Sociale0 个研究点目标入组 20 人开始时间: 2018年8月3日最近更新:
适应症
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 发起方
- 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.Responsive to the in vitro (ErPCs) treatment with Sirolimus according to laboratory specific definition (= 20% increase of HbF in comparison with samples not treated with Sirolimus)
- •2.AST or ALT < 3x ULN (Upper Limit of Normal)
- •3.Documented Platelet count >150.000/ul
- •4.Fertile women using an adequate system of contraception
- •5.Patients willing to follow all the study requirements and perform all the study visits and to cooperate with the investigator
- •6.Selection criteria must still be fulfilled at the time of the inclusion visit
- •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 20
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
排除标准
- •1.Uncontrolled hypertension defined as systolic pressure (PA) = 140 mm Hg or diastolic pressure = 90 mm Hg.
- •2.Class 3 or higher heart failure (New York Heart Association, NYHA).
- •3.QTc> 450 msec on selection ECG.
- •4.White blood cell [WBC] count <3000 cells per µL and/or Granulocytes <1500/mm3
- •5.Total cholesterol > 240 mg/dl
- •6.Triglycerides > 200 mg/dl
- •7.Proteinuria with urinary protein >1g/24 hrs;
- •8.Any active infection requiring parenteral antibiotic therapy within 28 days prior to inclusion or oral antibiotics within 14 days prior to inclusion;
- •9.Enrolment into one other drug or device trial since selection;
- •10.Major surgery (including splenectomy) performed after the study selection visit .
- •11.Ongoing treatment with drugs and agents that could increase the concentration of Sirolimus in the blood including: ciclosporin, bromocriptine, cimetidine, cisapride, clotrimazole, danazol, diltiazem, fluconazole, protease inhibitors (eg for HIV and hepatitis C which require pharmacological treatment with ritonavir, indinavir, boceprevir and telaprevir), metoclopramide, nicardipine, troleandomycin, verapamil.
- •12.Ongoing treatment with drugs and agents that could reduce the concentration of Sirolimus including carbamazepine, phenobarbital, phenytoin, rifapentine, St. John's wort (Hypericum perforatum).
- •13.Cytotoxic agents, systemic corticosteroids, immunosuppressants or anticoagulant therapy such as warfarin or heparin within 28 days before inclusion (prophylactic aspirin up to 100 mg / day is allowed);
- •14.Macrolidic antibiotics (clarithromycin), ACE inhibitors.
- •15.Pregnant or lactating women.
- •16.Occurrence of an event which leads to the appearance of a non-selection criteria
研究者
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