AIHA - Cold Autoimmune Hemolytic Anemia, AIHA - Warm Autoimmune Hemolytic Anemia
Conditions
Brief summary
To evaluate the efficacy and safety of ipecopam in the treatment of refractory/relapsed AIHA.
Interventions
DRUGIptacopan
Iptacopan
Sponsors
Bing Han
Study design
Allocation
NON_RANDOMIZED
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| OR | 3 months | Overall response rate |
| CR | 6 months | complete response rate |
Countries
China
Contacts
Primary ContactBing Han Han
Outcome results
None listed