Combination of Danazole With Berberine in the Treatment of ITP
试验速览
- 阶段
- 2 期
- 入组人数
- 55
- 试验地点
- 1
- 主要终点
- The Count of Participants That Achieved 6-month Sustained Response
研究概览
简要总结
A prospective, multicenter, open-label, Phase II, single arm, trial performed in 6 departments of hematology in China
详细描述
Immune thrombocytopenia (ITP) is an autoimmune disorder characterized by low platelet count and mucocutaneous bleeding. Approximately one-third of ITP patients fail to respond to first-line therapies. In addition, a certain amount of the patients relapse and require further therapy after one or more treatment strategies (e.g., thrombopoietin receptor agonists or rituximab). The optimal second-line treatment remains a challenge.Berberine (BBR), an isoquinoline alkaloid derived from plants, is widely used as a nonprescription drug to treat diarrhea. Our previous data demonstrated that gut microbiota dysbiosis may contribute to the development of corticosteroid-resistant ITP. BBR may correct corticosteroid-resistance by modulating the gut microbiota structure, thus being a novel potential second-line candidate to treat ITP. Importantly, the potential clinical benefits of BBR have already been evaluated in various studies using human subjects, and it has been shown to be safe. Danazol is an attenuated androgen that has successfully been used in the treatment of ITP. Considering the side-effects of a regular dose of danazol and that BBR and danazol share disparate mechanisms in the treatment of ITP, we hypothesized that the combination of these two agents might be a promising option to maximize efficacy while minimizing adverse effects. Therefore, we aimed to evaluate the long-term efficacy and safety of berberine plus danazol in patients with corticosteroid-resistant or relapsed ITP.
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 80 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Clinically confirmed immune thrombocytopenic purpura (ITP) newly diagnosed
- •Platelet count less than 30×109/L on two occasions or Platelets above 30×109/L combined with bleeding manifestation
- •Subject is ≥ 18 years and ≤80years
- •Subject has signed and dated written informed consent.
- •Fertile patients must use effective contraception during treatment and observational period
- •Negative pregnancy test
排除标准
- •Have an impaired renal function as indicated by a serum creatinine level > 2.0 mg/dL
- •Have an inadequate liver function as indicated by a total bilirubin level > 2.0 mg/dL and/or an aspartate aminotransaminase or alanine aminotransferase level > 3×upper limit of normal
- •Have a New York Heart Classification III or IV heart disease
- •Have a history of severe psychiatric disorder or are unable to comply with study and follow-up procedures
- •Have active hepatitis B or hepatitis C infection
- •Have a HIV infection
- •Have active infection requiring antibiotic therapy within 7 days prior to study entry
- •Are pregnant or lactating women, or plan to become pregnant or impregnated within 12 months of receiving study drug
- •Previous treatment with rituximab
- •Previous splenectomy
- •Had previous or concomitant malignant disease
- •Not willing to participate in the study.
- •Expected survival of < 2 years
- •Intolerant to murine antibodies
- •Immunosuppressive treatment within the last month
- •Connective tissue disease
- •Autoimmune hemolytic anemia
- •Patients currently involved in another clinical trial with evaluation of drug treatment
研究组 & 干预措施
Berberine plus danazol
Berberine plus danazol group
干预措施: Berberine plus danazol (Drug)
结局指标
主要结局
The Count of Participants That Achieved 6-month Sustained Response
时间窗: 6 month
6-month sustained response defined as platelet count of 30×10⁹/L or more and at least a doubling of baseline platelet count (partial response (PR)), or a platelet count of 100×10⁹/L or more and the absence of bleeding without rescue medication(complete response (CR))
次要结局
- the Count of Participants That Had Adverse Events(2 years)
- the Count of Participants That Achieved Initial Response(4 weeks)
- DOR(2 years)
研究者
Xiao Hui Zhang
Prof.
Peking University People's Hospital