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

Launched by BING HAN · Feb 21, 2025

Keywords

ClinConnect Summary

This clinical trial is studying a new treatment called Iptacopan for patients with a condition known as autoimmune hemolytic anemia (AIHA), which means the body’s immune system mistakenly attacks and destroys its own red blood cells. This trial specifically focuses on patients whose condition has not improved with standard treatments or who have experienced a relapse. The goal is to see how effective and safe Iptacopan is for these individuals.

To be eligible for this trial, participants must be at least 18 years old, have a confirmed diagnosis of AIHA, and have low hemoglobin levels (less than 100 g/L). They should also have had poor results with previous treatments like glucocorticoids (steroid medications) or be dependent on them. Participants can expect regular check-ups and tests to monitor their health and response to the treatment. It’s important to note that certain individuals, such as those with specific infections, serious health conditions, or who are pregnant, will not be able to join the study. This trial is not yet recruiting, but it aims to provide new hope for patients struggling with this challenging condition.

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• • 1. Voluntarily signed an informed consent form (ICF);
• • 2. Males or females aged 18 or older;
• • 3. Physical status score \[Eastern Cooperative Oncology Group (ECOG) score\] ≤2;
• • 4. Confirmed diagnosis of primary AIHA or secondary autoimmune disease (except rheumatoid arthritis and systemic lupus erythematosus) with underlying disease in a stable state;
• • 5. 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;
• • 6. Hemoglobin (Hb) \<100 g/L before drug administration;
• • 7. Positive direct anti-human globulin test (DAT) (IgA, IgM or IgG+, with or without C3+).
• • 8. 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;
• • 9. 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)
• • 1. Neutrophil count \>1.5×109/L and platelet \>30×109/L;
• • 2. ALT and AST ≤ 2 × ULN;
• • 3. Serum creatinine concentration ≤ 2 × ULN and creatinine clearance ≥ 50mL/min;
• • 10. No active infection; no pregnancy or lactation;
• • 11. cAIHA patients presenting with skin cyanosis and thrombosis;
• • 12. Written evidence of Neisseria meningitidis and Streptococcus pneumoniae vaccinations within 2 years or, if none, antibiotic prophylaxis until 2 weeks after completion of vaccination.
• Exclusion Criteria:
• • 1. Presence of secondary AIHA outside the inclusion criteria;
• • 2. Hb \<100 g/L due to non-AIHA factors; and
• • 3. Infections requiring systemic therapy;
• • 4. Those with a past history of malignancy (except cured basal cell carcinoma of the skin or cervical carcinoma in situ);
• • 5. With history of vital organ transplantation or hematopoietic stem cell/bone marrow transplantation;
• • 6. Those who have undergone splenectomy within 24 weeks prior to enrollment;
• • 7. Those who have had major surgery within four weeks prior to enrollment or who require major elective surgery during the study period;
• • 8. History of severe cardiovascular disease \[e.g., class III/IV congestive heart failure, arrhythmia or angina requiring medication, unstable angina, coronary stenting, angioplasty or coronary artery bypass grafting, or corrected Q-T interval (QTcF) ≥ 90 mmHg\]
• • 9. Patients with medically uncontrolled hypertension (systolic blood pressure ≥140 mmHg or diastolic blood pressure ≥90 mmHg); or comorbid portal hypertension;
• • 10. 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;
• • 11. Human immunodeficiency virus (HIV) infection
• • 12. 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;
• • 13. Those who had received herbal treatment within one week prior to enrollment that interfered with the assessment of efficacy;
• • 14. Patients with severe psychological or psychiatric abnormalities;
• • 15. Alcohol or drug abusers;
• • 16. Female patients who are pregnant or breastfeeding;
• • 17. Patients who, in the opinion of the investigator, are not suitable for participation in this study.