CAR T-cell Therapy Targeting CD19 and BCMA in Patients with AIHA Who Have Failed ≥3 Lines of Therapy.

Launched by INSTITUTE OF HEMATOLOGY & BLOOD DISEASES HOSPITAL, CHINA · Dec 10, 2024

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ClinConnect Summary

This clinical trial is exploring a new treatment called CAR T-cell therapy for patients with a condition called Autoimmune Hemolytic Anemia (AIHA) who have not responded to at least three previous treatments. The therapy involves using specialized immune cells that target specific proteins (CD19 and BCMA) to help the body fight the disease more effectively. Researchers want to find out how safe and effective this therapy is for patients who have been struggling with AIHA, which causes the body to destroy its own red blood cells.

To participate in this study, individuals must be at least 18 years old and have a confirmed diagnosis of AIHA. They should have specific markers in their blood indicating they are eligible for this treatment. Additionally, patients need to be in relatively good health overall, with functioning major organs, and must agree to follow the study guidelines. Participants can expect close monitoring throughout the trial, and their involvement will help advance understanding of this treatment option for AIHA. If you or someone you know is considering joining, it’s important to discuss it with a healthcare provider to understand the potential benefits and risks.

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Eligibility criteria

• Inclusion Criteria:
• • Age ≥ 18 years
• • Flow cytometry detected positive B cell CD19 or BCMA in the patient's peripheral blood.
• • Patients diagnosed with AIHA, including warm antibody type, cold agglutinin disease, mixed type, and other types of AIHA, with diagnostic criteria referring to the "Chinese Adult Autoimmune Hemolytic Anemia Diagnosis and Treatment Guidelines (2023 Edition)"
• • The definition of recurrent/refractory AIHA that has received at least 3 failed lines of treatment is symptomatic anemia (hemoglobin\<100g/L) that persists after a routine treatment cycle of at least 6 months and is still ineffective or reappears after disease remission. The definition of conventional treatment: treatment with glucocorticoids and/or rituximab, as well as any 1-2 or more of the following immunomodulatory drugs: cyclophosphamide, azathioprine, mycophenolate mofetil, cyclosporine A, azathioprine, danazol, bendamustine, fludarabine, bortezomib, and biologics including daratumumab, BTK inhibitors, Syk inhibitors, and complement inhibitors.
• • ECOG ≤ 2
• * Functional requirements for major organs are as follows:
• • 1. . The bone marrow function needs to meet: a Neutrophil count ≥ 1.0 × 10 \^ 9/L; b. Platelets ≥ 30 × 10 \^ 9/L.
• • 2. Liver function: ALT ≤ 3 × UL; AST ≤ 3×ULN； Total bilirubin ≤ 2.0 × ULN (excluding Gilbert syndrome, total bilirubin ≤ 3.0 × ULN).
• • 3. Renal function: creatinine clearance rate (CrCl) ≥ 30 ml/min (Cockcroft/Gault formula, excluding acute CrCl decline caused by the disease itself).
• • Female subjects of childbearing potential and male subjects with partners of childbearing potential must use medically approved contraception or abstinence during the study treatment period and for at least 6 months after the end of the study treatment; Female subjects of childbearing potential must have a negative Human chorionic gonadotropin (HCG) test within 7 days before study enrollment and not be lactating.
• • Willing to participate in this clinical study, sign an informed consent form, have good compliance, and cooperate with follow-up.
• Exclusion Criteria:
• • Subjects with a history of severe drug allergies or allergic tendencies.
• • Presence or suspicion of uncontrolled or treatment-required fungal, bacterial, viral, or other infections.
• • Subjects with central nervous system diseases caused by autoimmune diseases or non-autoimmune diseases (including epilepsy, psychosis, organic brain syndrome, cerebral vascular accidents, encephalitis, central nervous system vasculitis).
• • Subjects with insufficient cardiac function
• • Subjects with congenital immunoglobulin deficiencies
• • History of malignancy within five years
• • Subjects with end-stage renal failure
• • Subjects who are positive for hepatitis B surface antigen (HBsAg) or hepatitis B core antibody (HBcAb) with peripheral blood HBV DNA \>ULN; subjects positive for hepatitis C virus (HCV) antibody and peripheral blood HCV RNA; individuals positive for human immunodeficiency virus (HIV) antibody; individuals positive for syphilis testing
• • Subjects with psychiatric disorders and severe cognitive impairments
• • Subjects who have used immunosuppressive agents or biologics with therapeutic effects on the disease within five half-life before enrollment
• • Pregnant women or women planning to conceive
• • Active infection, active rheumatic and immune disease, drug induced and diagnosed lymphoproliferative tumor associated secondary AIHA patients
• • Subjects that the investigator believes have other reasons that make them unsuitable for inclusion in this study.