NCT06847607尚未招募不适用
A Multicenter Prospective Study of Iptacopan in the Treatment of Refractory/Relapsed Autoimmune Hemolytic Anemia (AIHA)
Bing Han1 个研究点 分布在 1 个国家目标入组 20 人开始时间: 2025年3月1日最近更新:
干预措施
相关药物
试验速览
- 阶段
- 不适用
- 状态
- 尚未招募
- 发起方
- 入组人数
- 20
- 试验地点
- 1
- 主要终点
- OR
研究概览
简要总结
To evaluate the efficacy and safety of ipecopam in the treatment of refractory/relapsed AIHA.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 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
- •Voluntarily signed an informed consent form (ICF);
- •Males or females aged 18 or older;
- •Physical status score [Eastern Cooperative Oncology Group (ECOG) score] ≤2;
- •Confirmed diagnosis of primary AIHA or secondary autoimmune disease (except rheumatoid arthritis and systemic lupus erythematosus) with underlying disease in a stable state;
- •Poor response to at least previous glucocorticoid therapy, including ineffective (defined as failure to achieve stabilization of Hb levels at 100 g/L or erythrocyte hematocrit <30% despite at least 4 weeks of treatment with previously recommended doses), or glucocorticoid-dependent (defined as maintenance of equal doses of prednisone exceeding 15 mg/d), or relapsed (defined as treatment that is effective and then again has an Hb of <100 g/L or an erythrocyte hematocrit of <30%), or otherwise contraindicated. 30%), or otherwise contraindicated or intolerant to glucocorticoid therapy;
- •Hemoglobin (Hb) <100 g/L before drug administration;
- •Positive direct anti-human globulin test (DAT) (IgA, IgM or IgG+, with or without C3+).
- •Combination of one anti-AIHA therapy [glucocorticoids only (≤15 mg prednisone equivalent), immunosuppressants (azathioprine, cyclosporine, and merti-macrolide only)] is permitted in this study, provided that the dose has been stable for at least 28 days prior to enrollment;
- •Laboratory tests meet the following criteria (no treatment for the abnormality of the index within 2 weeks prior to blood collection, or no long-acting G-CSF treatment within 2 weeks)
- •Neutrophil count >1.5×109/L and platelet >30×109/L;
- •ALT and AST ≤ 2 × ULN;
- •Serum creatinine concentration ≤ 2 × ULN and creatinine clearance ≥ 50mL/min;
- •No active infection; no pregnancy or lactation;
- •cAIHA patients presenting with skin cyanosis and thrombosis;
- •Written evidence of Neisseria meningitidis and Streptococcus pneumoniae vaccinations within 2 years or, if none, antibiotic prophylaxis until 2 weeks after completion of vaccination.
排除标准
- •Presence of secondary AIHA outside the inclusion criteria;
- •Hb <100 g/L due to non-AIHA factors; and
- •Infections requiring systemic therapy;
- •Those with a past history of malignancy (except cured basal cell carcinoma of the skin or cervical carcinoma in situ);
- •With history of vital organ transplantation or hematopoietic stem cell/bone marrow transplantation;
- •Those who have undergone splenectomy within 24 weeks prior to enrollment;
- •Those who have had major surgery within four weeks prior to enrollment or who require major elective surgery during the study period;
- •History of severe cardiovascular disease [e.g., class III/IV congestive heart failure, arrhythmia or angina requiring medication, unstable angina, coronary stenting, angioplasty or coronary artery bypass grafting, or corrected Q-T interval (QTcF) ≥ 90 mmHg]
- •Patients with medically uncontrolled hypertension (systolic blood pressure ≥140 mmHg or diastolic blood pressure ≥90 mmHg); or comorbid portal hypertension;
- •Patients with severe gastrointestinal disorders such as dysphagia, active gastric ulcers, etc., who are unable to take drugs orally or have impaired absorption of oral drugs;
- •Human immunodeficiency virus (HIV) infection
- •Uncontrolled or active HBV infection [Hepatitis B surface antigen (HBsAg) or Hepatitis B core antibody (HBcAb) positive patients, need to confirm Hepatitis B Virus Deoxyribonucleic Acid (HBV DNA) positive]; or Hepatitis C [patients with Hepatitis C Virus Ribonucleic Acid (HCV RNA) positive patients]; or cirrhosis of the liver;
- •Those who had received herbal treatment within one week prior to enrollment that interfered with the assessment of efficacy;
- •Patients with severe psychological or psychiatric abnormalities;
- •Alcohol or drug abusers;
- •Female patients who are pregnant or breastfeeding;
- •Patients who, in the opinion of the investigator, are not suitable for participation in this study.
研究组 & 干预措施
Iptacopan-AIHA
Experimental
Iptacopan-AIHA
干预措施: Iptacopan (Drug)
结局指标
主要结局
OR
时间窗: 3 months
Overall response rate
CR
时间窗: 6 months
complete response rate
次要结局
未报告次要终点
研究者
Bing Han
chief physician
Peking Union Medical College Hospital
研究点 (1)
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