ClinicalTrials.gov record NCT06733610
CAR T-cell Therapy Targeting CD19 and BCMA in Patients with AIHA Who Have Failed ≥3 Lines of Therapy.
A Clinical Study Evaluating the Safety and Preliminary Efficacy of Universal Allogeneic CAR T-cell Therapy Targeting CD19 and BCMA in Patients with Autoimmune Hemolytic Anemia Have Failed ≥ 3 Lines of Therapy
AI summaryWhat this study is about, in plain languageShow ▾Hide ▴
AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.
This phase 1, investigator-initiated trial evaluates one intravenous infusion of universal allogeneic anti-CD19/BCMA CAR T-cells given after lymphodepleting chemotherapy (fludarabine and cyclophosphamide). It is for adults with autoimmune hemolytic anemia who have failed at least three prior lines of therapy. The study measures safety and early signs of clinical response, with an interim analysis at 12 weeks.
What it is studying. The study is testing the safety and early clinical activity of a single dose of universal allogeneic anti-CD19/BCMA CAR T-cells given by IV after lymphodepletion in people with autoimmune hemolytic anemia who have failed ≥3 prior treatments.
What the researchers hope to learn. To evaluate safety by recording dose-limiting toxicities within 28 days and overall adverse events up to 12 months; and to measure clinical response rates (CR, CRi, PR, ORR) up to 24 weeks after infusion.
What this study is about
The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.
This is an investigator-initiated trial to evaluate the safety and efficacy of universal allogeneic anti-CD19/BCMA CAR T-cells in AIHA who have failed ≥ 3 lines of therapy.
Interventions being studied
Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.
- Biological
- universal allogeneic anti-CD19/BCMA CAR T-cells
Eligibility
The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.
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:
- . The bone marrow function needs to meet: a Neutrophil count ≥ 1.0 × 10 \^ 9/L; b. Platelets ≥ 30 × 10 \^ 9/L.
- Liver function: ALT ≤ 3 × UL; AST ≤ 3×ULN； Total bilirubin ≤ 2.0 × ULN (excluding Gilbert syndrome, total bilirubin ≤ 3.0 × ULN).
- 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.
Study description and what participation involves
The study's detailed description, as published on ClinicalTrials.gov.
Detailed description from the registry
This is an investigator-initiated trial to evaluate the safety and efficacy of universal allogeneic anti-CD19/BCMA CAR T-cells in autoimmune hemolytic anemia who have failed ≥ 3 lines of therapy. Study intervention consists of a single infusion of universal allogeneic CAR T-cells administered intravenously after a lymphodepleting therapy regimen consisting of fludarabine and cyclophosphamide. Interim analysis will be performed when participants finish the visit 12 weeks after CAR T-cell infusion.
AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴
AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.
Participants receive lymphodepleting chemotherapy (fludarabine and cyclophosphamide) followed by a single intravenous infusion of universal allogeneic anti‑CD19/BCMA CAR T‑cells. They will have scheduled follow‑up visits for safety and response assessments, with an interim safety review after the 12‑week visit; response is assessed up to 24 weeks and adverse events up to 12 months. Participation requires blood tests, clinical monitoring, signing informed consent, and following the study's contraception and pregnancy‑testing rules; the study team provides the full visit schedule and monitoring details.
How the study is structured. Phase 1, interventional treatment study with a single treatment group and no masking. Participants receive lymphodepleting chemotherapy (fludarabine and cyclophosphamide) followed by one intravenous infusion of the investigational universal allogeneic anti-CD19/BCMA CAR T-cells. Safety monitoring includes early assessment for dose-limiting toxicities and adverse events, plus follow-up visits and assessments for clinical response up to planned time points.
Questions you could ask
These questions are suggestions for a conversation with the study team or your own clinician, not advice.
- This is an early-phase study, mainly about safety and dose. What is already known about this treatment?
- The listed sites are in China. Is any of them reachable for me, and does the study help with travel?
- Which of the eligibility criteria would need to be checked with tests before I could join?
More on autoimmune hemolytic anemia
Important dates
- Study start
- Dec 5, 2024
- Primary completion
- Feb 5, 2026 (estimated)
- Study completion
- Dec 5, 2026 (estimated)
- Target enrollment
- 15 participants (estimated)
- Last updated on registry
- Dec 13, 2024
- Data verified
- Oct 10, 2026
Sponsor and research team
The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.
- Lead sponsor
- Institute of Hematology & Blood Diseases Hospital, China
- Collaborators
- Xi'niao Biotech
Central study contact
If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.
- Jun Shi, PhD 📞 13752253515 ✉ shijun@ihcams.ac.cn
- Lele Zhang, PhD 📞 15811139278 ✉ zhanglele@ihcams.ac.cn
Participating sites (1)
- Regenerative Medicine Center and Red Blood Cell Disorders CenterRecruiting
Tianjin, Tianjin Municipality · China
- Lele Zhang, PhD · 15811139278 · zhanglele@ihcams.ac.cn
Terms used on this page · explanations by MedicalUpdates.ai
- CAR T
- A treatment in which a person's own immune cells (T cells) are collected, modified in a laboratory to recognise a target, and returned to the body.
- lines of therapy
- Treatment a person has already had for the condition. A 'line of therapy' is one course of treatment; studies often require, or limit, the number of prior lines.
- refractory
- A condition that has not responded to a treatment. 'Relapsed' means it responded and then came back; 'refractory' means it did not respond in the first place.
- glucocorticoids
- Steroid medicines, such as prednisone, that reduce inflammation. Studies often require a stable dose at entry or measure whether the study treatment allows the dose to be reduced.
- ECOG
- A scale from 0 to 5 that describes how a person's condition affects daily activity. 0 means fully active; 1 means restricted in strenuous activity; 2 means able to self-care but unable to work; higher numbers mean more limitation.
- Neutrophil count
- The number of neutrophils, a type of white blood cell that fights infection, in a blood sample. Studies often require it to be above a set level.
- ULN
- The highest value of a blood test that a laboratory considers normal. Criteria such as '≤ 3 × ULN' mean the result must be no more than three times that value.
- creatinine clearance
- A measure of kidney function, calculated from a blood creatinine level together with age, weight and sex.
- childbearing potential
- Studies often ask participants who could become pregnant, or could father a child, to use contraception during the study and for a period afterwards, because the effect of the treatment on a pregnancy is unknown.
- enrollment
- The number of participants a study plans to include, or has included. 'Estimated' means the planned number; 'actual' means the number who joined.
- informed consent form
- The process of explaining a study, including its risks and what participation involves, so that a person can decide freely whether to take part. Consent is usually documented in writing, and a participant can choose to leave the study later.
- follow-up
- The period after treatment or enrolment during which participants are observed or contacted, for example to record side effects or outcomes.
- immunosuppressive
- A medicine that reduces the activity of the immune system. Studies of autoimmune conditions often require a stable dose, a prior failure, or a washout of such medicines.
- half-life
- The time it takes for the amount of a drug in the body to fall by half. 'Five half-lives' is often used as the point at which a previous drug has essentially left the body.
- Inclusion Criteria
- The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
- Exclusion Criteria
- The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.
The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.
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