EUCTR2015-001703-32-DE进行中（未招募）1 期
Phase III multicentric study evaluating the efficacy and safety of sirolimus in Vascular Anomalies that are refractory to standard care - VASE
Cliniques Universitaires Saint-Luc (CUSL)0 个研究点目标入组 250 人开始时间: 2016年5月31日最近更新:
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
- 250
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
- •- Inclusion is limited to children and adults from 3 months* to 70 years old:
- •* In Germany the inclusion is limited to adults only from 18 to 70 years old
- •- Patients with complex vascular anomalies that are refractory to standard care such as medical treatment, surgical resection and/or sclerotherapy/embolization (ineffective or accompanied by major complications)
- •- Infants with hemangioendothelioma with risk of Kasabach-Merritt phenomenon (very low platelet count (lower than 80 000/mm3)
- •- Except for infants (not in Germany) described just above, all patients must have adequate medullary function: Hemoglobine> 10,0 g/dl, neutrophils >1500/mm³ and platelets > 100.000/mm³
- •- Patients must have the following laboratory values:
- •o Total serum bilirubin = 1.5 x ULN (or totally bilirubin =3 x ULN with direct bilirubin = 1.5 x ULN in patients with well documented Gilbert Syndrome)
- •o Serum alanine aminotransferase (ALT) and aspartate aminotransferase (AST) = 3 x ULN (or <5.0 x ULN if hepatic metastases are present)
- •o Serum creatinine ?1.5 x ULN. If the serum creatinine is = 1.5 x ULN, then a 24-hour Creatinine Clearance must be conducted and the result must be = 60 mL/min.
- •- Karnofsky > 50
- •- Patients or legal relatives (no children in GERMANY) have to be able to sign the informed consent
- •- Negative urine pregnancy test in females with a childbearing potential. A woman is considered of childbearing potential (WOCBP), i.e. fertile, following menarche and until becoming post-menopausal unless permanently sterile. Permanent sterilisation methods include hysterectomy, bilateral salpingectomy and bilateral oophorectomy.
- •- Sexually active female patients (and female partners of male patients) must use adequate contraceptive measures while on study and for up to 8 weeks after ending treatment. Highly effective birth control methods include:
- •ocombined (estrogen and progestogen containing) hormonal contraception associated with inhibition of ovulation
- •oprogestogen-only hormonal contraception associated with inhibition of ovulation
- •ointrauterine device (IUD)
- •ointrauterine hormone-releasing system ( IUS)
- •obilateral tubal occlusion
- •ovasectomised partner
- •osexual abstinence (with regular verification by PI)
- •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) yes
- •F.1.3.1 Number of subjects for this age range 0
排除标准
- •- Any of the following concurrent severe and/or uncontrolled medical conditions, which could compromise participation in the study or interfere with the study results:
- •Impaired cardiac function or clinically significant cardiac diseases, including unstable angina pectoris, ventricular arrhythmia, valvular disease with documented compromise in cardiac function, myocardial infarction within the last 6 months, documented by persistent elevated cardiac enzymes or persistent regional wall abnormalities on assessment of LVEF function, history of documented congestive heart failure (New York Heart Association functional classification III-IV), documented cardiomyopathy, family history of congenital long or short QT, or known history of QT/QTc prolongation of Torsades de Pointes (TdP)
- •- Impairment of Gastro-Intestinal (GI) function or GI disease that may significantly alter the absorption of sirolimus (e.g., ulcerative diseases, uncontrolled nausea, vomiting, diarrhea = Grade 2, malabsorption syndrome, or small bowel resection)
- •- Known hypersensitivity to drugs or metabolites from similar classes as study treatment.
- •- Patient has other concurrent severe and /or uncontrolled medical condition that would,in the investigator’s judgment, contraindicated participation in the clinical study (e.g. acute or chronic pancreatitis, liver cirrhosis, active chronic hepatitis, severely impaired lung function with a spirometry = 50% of the normal predicted value and/or O2 saturation = 88% at rest, etc.)
- •- Immunocompromised patients, including known seropositivity for HIV
- •- Pregnant or lactating women
- •- Prior treatment with PI3K and/or mTOR inhibitors
研究者
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