UCAR T-cell Therapy Targeting CD19/BCMA in Patients With r/r Autoimmune Hemolytic Anemia

Launched by ZHEJIANG UNIVERSITY · Apr 2, 2025

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ClinConnect Summary

This clinical trial is exploring a new treatment called UCAR T-cell therapy for patients with a condition known as relapsed or refractory autoimmune hemolytic anemia (AIHA). This condition happens when the immune system mistakenly destroys red blood cells, leading to anemia, which can cause fatigue and weakness. The trial will involve up to 18 participants who have not responded to at least three previous treatments. Researchers want to see how safe and effective the new CAR T-cell therapy is in these patients.

To participate, individuals must be at least 18 years old and have a specific type of cells (CD19 or BCMA) present in their blood. They should also have experienced ongoing symptoms of anemia despite prior treatments. The trial is not yet recruiting participants, but those who meet the criteria will be asked to sign an informed consent form and will need to follow up regularly during the study. It’s important to note that some people may not be eligible for the trial due to health issues or other medical conditions. If you're interested in learning more about this study and whether it might be right for you or a loved one, please reach out to the research team for further information.

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Eligibility criteria

• Inclusion Criteria:
• • 1. Age ≥ 18 years
• • 2. Flow cytometry detected positive B cell CD19 or BCMA in the patient's peripheral blood.
• • 3. 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) .
• • 4. 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.
• 5. Functional requirements for major organs are as follows:
• • 1. The bone marrow function needs to meet: a Neutrophil count ≥ 0.5× 10 \^ 9/L; b. Platelets ≥ 30 × 10 \^ 9/L.
• • 2. Liver function: ALT ≤ 3 × UL; AST ≤ 3×ULN.
• • 3. Renal function: creatinine clearance rate (CrCl) ≥ 30 ml/min (Cockcroft/Gault formula).
• • 6. ECOG ≤ 2
• • 7. 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.
• • 8. Willing to participate in this clinical study, sign an informed consent form, have good compliance, and cooperate with follow-up.
• Exclusion Criteria:
• • 1. Subjects with a history of severe drug allergies or allergic tendencies.
• • 2. Presence or suspicion of uncontrolled or treatment-required fungal, bacterial, viral, or other infections.
• • 3. 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).
• • 4. Subjects with insufficient cardiac function.
• • 5. Subjects with congenital immunoglobulin deficiencies.
• • 6. History of malignancy within five years.
• • 7. Subjects with end-stage renal failure.
• • 8. Subjects who are positive for hepatitis B surface antigen (HBsAg) or hepatitis B core antibody (HBcAb) with peripheral blood HBV DNA titer higher than the upper limit of detection; 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.
• • 9. Subjects with psychiatric disorders and severe cognitive impairments.
• • 10. Subjects who have used immunosuppressive agents or biologics with therapeutic effects on the disease within five half-life before enrollment.
• • 11. Pregnant women or women planning to conceive.
• • 12. Active infection, active rheumatic and immune disease, drug induced and diagnosed lymphoproliferative tumor associated secondary AIHA patients.
• • 13. Subjects that the investigator believes have other reasons that make them unsuitable for inclusion in this study.