CAR-T for Autoimmune Hemolytic Anemia Patients Who Have Failed Three or More Lines of Therapy

Launched by INSTITUTE OF HEMATOLOGY & BLOOD DISEASES HOSPITAL, CHINA · Jan 8, 2024

Keywords

ClinConnect Summary

This clinical trial is studying a new treatment called CAR-T cell therapy for patients with autoimmune hemolytic anemia (AIHA) who have not responded to at least three other treatments. AIHA is a condition where the body’s immune system mistakenly destroys its own red blood cells, leading to anemia, or low hemoglobin levels. The goal of the trial is to see if this innovative therapy can safely help patients who have not found relief from traditional medications.

To be eligible for this trial, participants need to be at least 12 years old and have a specific diagnosis of AIHA or related conditions, along with low hemoglobin levels. They must have already tried and failed three or more lines of treatment, such as steroids or other immune therapies. Participants will undergo monitoring throughout the trial to ensure their safety and health, and those who are able to conceive will need to follow certain precautions regarding pregnancy. This trial is currently recruiting participants, so if you or someone you know is struggling with AIHA and has exhausted treatment options, this may be an opportunity to consider.

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• • Subject and/or subject's legal personal representative fully understand and voluntarily sign informed consent forms.
• • Male or female age ≥ 12 years.
• • ECOG performance status ≤2.
• • Diagnosis of warm antibody hemolytic anemia (AIHA), cold AIHA, mixed AIHA or Evans syndrome.
• • Hemoglobin\<100g/L.
• • Failure or intolerance to at least 3 lines of therapy, including glucocorticoids, rituximab, cyclophosphamide, azathioprine, fludarabine, cyclosporine, mycophenolate mofetil, Bruton's tyrosine kinase inhibitors, splenectomy.
• • Laboratory tests of adequate organ function: Serum alanine aminotransferase (ALT) and aspartate aminotransferase (AST) ≤3×ULN; Creatinine clearance (CrCl) (Cockcroft-Gault formula) ≥40ml/min; Absolute neutrophil count (ANC) ≥1.0×10\^9/L (growth factors such as granulocyte colony-stimulating factor \[G-CSF\] were not received within 7 days before the screening period); Left ventricular ejection fraction (LVEF) ≥45%; Blood oxygen saturation (SpO2) ≥92%.
• • Subjects of childbearing potential will be required to follow contraception requirements from the time of enrollment until the end of the 6-month safety follow-up period. Female subjects of childbearing potential must have a negative Serum HCG test within 7 days before enrollment and not in the lactation period.
• Exclusion Criteria:
• • Clear diagnosis of lymphoproliferative tumor.
• • The platelet count in peripheral blood during the screening period is \<20×10\^9/L.
• • Have a history of severe drug allergy or allergic constitution.
• • Have a history of any of the following diseases: craniocerebral trauma, consciousness disorder, epilepsy, cerebrovascular ischemia, cerebrovascular, and hemorrhagic diseases within 6 months before enrollment.
• • Have any of the following serious cardiovascular diseases: myocardial infarction within 6 months before enrollment, cardiac angioplasty or stent implantation); Unstable angina; Severe cardiac arrhythmias; History of severe nonischemic cardiomyopathy; Congestive heart failure (New York Heart Association \[NYHA\] Class III or IV)).
• • Have malignant tumors within 5 years before enrollment, unless any of the following conditions: fully treated cervical carcinoma in situ, fully treated basal cell or squamous epithelial cell skin cancer, localized prostate cancer after radical mastectomy, breast ductal carcinoma in situ after radical mastectomy, Carcinoma in situ in other locations one year after radical resection, and these diseases have no evidence of relapse.
• • Subjects with any serious active fungal, bacterial, viral, tuberculosis or other infections, including active hepatitis B (defined as serum HBV-DNA ≥ 2000 IU/mL), active hepatitis C virus (Hepatitis C virus (HCV) infection, human immunodeficiency virus (HIV) antibody-positive or active syphilis patients, etc. Subjects whose HBV-DNA \< 2000 IU/mL can be included on the condition that they receive antiviral drugs and monitor the related indicators during the study.
• • Have mental illness and severe cognitive impairment.
• • Have a history of live attenuated vaccines within 4 weeks before enrollment.
• • Subjects considered to be ineligible for the study by the investigator for reasons other than the above.