Fecal Microbiota Transplantation for Patients With Steroid-Resistant or Relapsed Immune Thrombocytopenia: A Randomized, Double-blind, Placebo-Controlled Phase 2 Trial
试验速览
- 阶段
- 2 期
- 状态
- 进行中（未招募）
- 入组人数
- 42
- 试验地点
- 1
- 主要终点
- Overall Response Rate (ORR) at Week 12 of Treatment
研究概览
简要总结
This is a randomized, double-blind, placebo-controlled phase 2 trial to evaluate the safety and efficacy in immune thrombocytopenia patients receiving fecal microbiota transplantation
详细描述
This is a prospective, randomized, double-blind, placebo-controlled design of 42 adult patients with steroid-resistantrelapse ITP in China. Patients are randomly assigned at a 2:1 ratio to receive fecal microbiota transplantation or placebo. Patients in the fecal microbiota transplantation group receive bowel preparation and standard fecal microbiota capsules for 6 days. Those in the placebo group receive bowel preparation and standard placebo capsules for 6 days. Treatment wil be discontinued if very severe or life-threateningadverse events developed or at the patients' request. The primary endpoint is the overall response rate (ORR) at week 12 of treatment, defined as the rate of patients who obtain a platelet count > 50,000/uL, and at least 30,000/uL increase of the baseline count.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
盲法说明
This is a prospective, randomized, double-blind, placebo-controlled trial.
入排标准
- 年龄范围
- 18 Years 至 70 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Aged 18 to 70 years, regardless of gender.
- •Patients diagnosed with immune thrombocytopenia (ITP) in line with the diagnostic criteria, and with a disease course of ≥ 12 months (chronic ITP).
- •Meeting the definitions of corticosteroid resistance or relapse (resistance: platelet count remains < 30×10⁹/L despite treatment with standard-dose corticosteroids for ≥ 4 weeks; relapse: platelet count drops again to < 30×10⁹/L after corticosteroid dose reduction or discontinuation).
- •Sustained platelet count < 30×10⁹/L during the screening period.
- •Subjects voluntarily participate in this study and sign a written informed consent form.
排除标准
- •Secondary thrombocytopenia caused by other factors, such as drug-induced etiology, infection-related conditions, or other autoimmune diseases (e.g., systemic lupus erythematosus).
- •Subjects positive for Helicobacter pylori (H. pylori) infection (rescreening is permitted only after completion of standard eradication therapy and a drug withdrawal period of at least 1 month).
- •Active infection or receipt of systemic antibiotic therapy within the past 4 weeks.
- •Severe cardiac, hepatic, or renal insufficiency (e.g., Child-Pugh Class C liver dysfunction, or estimated glomerular filtration rate < 30 mL/min/1.73 m²).
- •A history of malignant tumors or primary immunodeficiency diseases.
- •Pregnant or lactating women.
- •Previous splenectomy, rituximab therapy, or plasma exchange treatment within the past 3 months.
- •Other conditions deemed inappropriate for study participation by the investigators based on clinical judgment.
结局指标
主要结局
Overall Response Rate (ORR) at Week 12 of Treatment
时间窗: Week 12 (±7 days) after the initiation of treatment
the rate of patients who obtain a platelet count \> 50,000/uL, and at least 30,000/uL increase of the baseline count at week 12 (±7 days) after the initiation of treatment.
次要结局
- Bleeding events(27 months)
- Complete Response Rate (CR)(27 months)
- Duration of Sustained Response(27 months)
- Bleeding Score(27 months)
- Health-related quality of life (HRQOL)(27 months)
- Adverse events(27 months)
研究者
Xiao Hui Zhang
Vice president of Peking University Institute of Hematology
Peking University People's Hospital