NCT02402998已完成2 期
Eltrombopag as Second Line Therapy in Adult Patients With Primary Immune Thrombocytopenia (ESTIT Study) in an Attempt to Achieve Long-term Remission: a Single Arm Multicenter Phase II Clinical and Biological Study
适应症
干预措施
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 55
- 试验地点
- 14
- 主要终点
- Number of subjects who achieve long-term response (6 months sustained response) and able to taper down the dose.
研究概览
简要总结
A clinical study to evaluate the efficacy of a drug called eltrombopag in adult patients affected of primary immune thrombocytopenia as first treatment to address the disease.
详细描述
This is a prospective, multicenter phase II trial designed to evaluate the activity of Eltrombopag second line treatment in adult patients with primary Immune Thrombocytopenia (ITP) not responsive or in relapse after a full first line steroids treatment (prednisone or dexamethasone) ± Intravenous Immune Globulin (IVIG).
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosis of primary ITP;
- •Age ≥ 18 years;
- •Patients are in the new diagnosis or persistent phase of disease (i.e. within month 12 from diagnosis);
- •Patients not responsive or in relapse after a full course of steroid therapy (prednisone 1 mg/kg/d for at least 28 days or 3 cycles of dexamethasone 40 mg/day for consecutive 4 days, according to the GIMEMA ITP0207 trial) ± IVIG;
- •Patients have a platelet count < 10 x 109/L documented in a single blood cell count;
- •Patients have bleeding symptoms and a platelet count > 10 < 30 x 109/L documented in a single blood cell count;
- •Patients have no bleeding symptoms and a platelet count >10 < 30 x 109/L in at least 2 blood cell counts at 3 days interval in the week preceding the enrollment with the last count at the day of enrollment;
- •Patients have corticosteroids or IVIG dependence (the need for ongoing or repeated doses administration of corticosteroids or IVIG to maintain a platelet count ≥ 30 x 109/L and/or to avoid bleeding;
- •Written informed consent obtained from the subject;
- •Men with a female partner of childbearing potential must have either had a prior vasectomy or agree to use effective contraception from time of enrollment until 6 months after the last dose of study treatment;
- •Female subjects of non-childbearing potential may be enrolled in the study; for this study population, non-childbearing potential is defined as current tubal ligation, hysterectomy, ovariectomy or post-menopause; OR
- •Female subjects of childbearing potential may be enrolled in the study, if the subject has practiced adequate contraception for 30 days prior to start of Eltrombopag, has a negative pregnancy test within 14 days of first dose of Eltrombopag, and has agreed to continue adequate contraception during the entire treatment period and for 6 months after completion of the treatment.
排除标准
- •Diagnosis of secondary ITP. As far as patients with immune thrombocytopenia and antiphospholipid antibodies positivity, individuals without a previous thromboembolic event are excluded only if lupus anticoagulant (LAC) is associated with the presence of anticardiolipin (aCL) and aβ2-Glycoprotein I (aβ2GPI) antibodies;
- •Previous treatment with other anti-ITP second line therapies (i.e. Rituximab, Azathioprine, Cyclosporin-A or other); only patients with a previous full course of steroid (see inclusion criteria for definition) ± IVIG are admitted to the study;
- •Previous treatment with any TPO-R agonists;
- •Patients have life threatening bleeding complications;
- •Patients had deep venous thrombosis (DVT) or arterial thrombosis in the 3 months preceding the enrollment;
- •Patients are HIV, HCV, HBsAg positive;
- •Patients with hepatic impairment (i.e. mild, moderate or severe hepatic impairment (Child-Pugh score > 6);
- •Patients have a well established liver disease that represents a contraindication for the use of Eltrombopag;
- •Patients are unable to respect the 4-hour interval between Eltrombopag and other medications (e.g. antacids), calcium-rich foods (e.g. dairy products and calcium fortified juices), or supplements containing polyvalent cations such as iron, calcium, aluminium, magnesium, selenium, and zinc;
- •Patients are unable to stop medications that are known to cause a drug-drug interaction with Eltrombopag;
- •Subjects meeting any of the following criteria must not be enrolled in an Eltrombopag study:
- •Lactating female.
- •History of another malignancy. Exception: subjects who have been disease-free for 5 years, or subjects with a history of completely resected non-melanoma skin cancer or successfully treated in situ carcinoma are eligible.
- •Any serious and/or unstable pre-existing medical, psychiatric disorder, or other conditions that could interfere with subject's safety, obtaining informed consent or compliance with the study procedures.
- •Hormone replacement therapy. Subjects must discontinue hormone replacement therapy prior to study enrollment due to the potential for inhibition of Cytochrome P450 (CYP) enzymes that metabolize estrogens and progestins.
- •Administration of an investigational drug within 30 days or 5 half-lives, whichever is longer, preceding the first dose of study treatment.
- •Have a known immediate or delayed hypersensitivity reaction or idiosyncrasy to drugs chemically related to Eltrombopag or excipients that contraindicate their participation.
研究组 & 干预措施
Eltrombopag
Experimental
Eltrombopag 50 mg/daily.
干预措施: Eltrombopag (Drug)
结局指标
主要结局
Number of subjects who achieve long-term response (6 months sustained response) and able to taper down the dose.
时间窗: After one year from study treatment.
A patient will achieve this endpoint if at the end of the period of treatment he/she reaches to discontinue Eltrombopag and if, after discontinuation, has absence of bleeding and maintains a platelet count ≥ 30 x 109/L and at least a 2-fold increase from the baseline count in blood cell count performed in the 6 months of the period observation, during which no steroid, Eltrombopag, other anti TPO-R agonists and other anti-ITP medications are administered.
次要结局
- Number of bleeding events.(After one year from study treatment.)
- Number of months of response from eltrombopag discontinuation to the last follow-up.(After one year from study treatment.)
- Number of months of complete response from eltrombopag discontinuation to the last follow-up.(After one year from study treatment.)
研究者
研究点 (14)
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