CAR-T therapy for patients with autoimmune hemolytic anemia who have not responded to multiple treatments
The Safety and Efficacy of ThisCART19A for Relapsed/Refractory Autoimmune Hemolytic Anemia Patients After Receiving Three or More Lines of Therapy.
This study is testing a new CAR-T therapy to see if it can help people with autoimmune hemolytic anemia who haven't responded to other treatments.
Quick facts
| Phase | Phase 1 |
|---|---|
| Study type | Interventional |
| Enrollment | 13 (estimated) |
| Ages | 12 Years and up |
| Sex | All |
| Sponsor | Institute of Hematology & Blood Diseases Hospital, China Academic / other |
| Drugs / interventions | rituximab, cyclophosphamide, fludarabine, CAR-T |
| Locations | 1 site (Tianjin, Tianjin) |
| Trial ID | NCT06212154 on ClinicalTrials.gov |
What this trial studies
This phase 1, single-arm, open-label study evaluates the safety and efficacy of ThisCART19A in patients with autoimmune hemolytic anemia who have failed three or more lines of therapy. Participants will receive ThisCART19A cell infusion after a preconditioning regimen and will be closely monitored for 28 days post-infusion. The study aims to assess the tolerability, pharmacokinetics, and pharmacodynamics of the treatment in this specific patient population.
Who should consider this trial
Good fit: Ideal candidates are patients aged 12 years and older with a diagnosis of autoimmune hemolytic anemia who have failed at least three prior lines of therapy.
Not a fit: Patients who have not been diagnosed with autoimmune hemolytic anemia or those who have not failed multiple lines of therapy may not benefit from this study.
Why it matters
Potential benefit: If successful, this treatment could provide a new therapeutic option for patients with autoimmune hemolytic anemia who have limited treatment alternatives.
How similar studies have performed: While CAR-T therapies have shown promise in hematological malignancies, this specific application for autoimmune hemolytic anemia is novel and has not been extensively tested in prior studies.
Eligibility criteria
Show full inclusion / exclusion 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.
Where this trial is running
Tianjin, Tianjin
- Institute of Hematology & Blood Diseases Hospital — Tianjin, Tianjin, China (Recruiting)
Study contacts
- Study coordinator: Jun Shi, PhD
- Email: shijun@ihcams.ac.cn
- Phone: 13752253515
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.