A Multicenter Prospective Study of Iptacopan in the Treatment of Refractory/Relapsed Autoimmune Hemolytic Anemia (AIHA)

Обзор

To evaluate the efficacy and safety of ipecopam in the treatment of refractory/relapsed AIHA.

Вмешательства

• Препарат Iptacopan
  Iptacopan

Первичные конечные точки

• OR [Срок оценки: 3 months]
• CR [Срок оценки: 6 months]

Критерии участия

Критерии включения

• 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.

Критерии исключения

• 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.

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Нерандомизированное

Модель

Одна группа

Маскирование

Открытое

Основная цель

Лечение

Центры проведения

Китай · 1 центр

• Bing Han — Пекин

Идентификаторы

NCT: NCT06847607 · AIHA-Iptacopan

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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