ClinicalTrials.gov record NCT07324889
An Open-label, Single-arm, Prospective, Multicenter, Phase I/II Clinical Study on the Safety and Efficacy of CD19/BCMA CAR-T Cell Therapy for Relapsed/Refractory Warm Antibody Autoimmune Hemolytic Anemia
An Open-label, Single-arm, Prospective, Multicenter, Phase I/II Clinical Study on the Safety and Efficacy of CD19/BCMA CAR-T Cell Therapy for Relapsed/Refractory Warm Antibody Autoimmune Hemolytic Anemia
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 is an open‑label, single‑arm Phase I/II study testing an infusion of CD19/BCMA CAR‑T cells after lymphodepletion. The study plans to enroll 24 people with relapsed or refractory warm antibody autoimmune hemolytic anemia using a two‑stage Bryant and Day design (8 participants first for safety, then 16 more to assess response). The planned CAR‑T dose is 1×10⁶ CAR⁺ cells/kg and participants receive preconditioning with fludarabine and cyclophosphamide before infusion.
What it is studying. The study is testing the safety, in‑body behavior (pharmacokinetics), and clinical activity of CD19/BCMA CAR‑T cell therapy in people with relapsed/refractory warm antibody autoimmune hemolytic anemia.
What the researchers hope to learn. Collect safety data on adverse events including immune‑related toxicities; characterize pharmacokinetics of the CAR‑T cells (AUC, Tmax, detectable duration); measure response rates (complete and partial responses) and changes in hemolysis and immune markers; estimate progression‑free survival.
What this study is about
The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.
This study is an open-label, single-arm, prospective, multicenter, phase I/II clinical trial. It adopts the two-stage optimal design proposed by Bryant and Day to investigate the efficacy, safety, and in vivo pharmacokinetic characteristics of CD19/BCMA CAR-T cell therapy in the treatment of relapsed/refractory warm antibody autoimmune hemolytic anemia.
Interventions being studied
Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.
- Drug
- CD19/BCMA CAR-T
Eligibility
The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.
Inclusion criteria
- Aged ≥ 18 years and ≤ 75 years.
- Meeting the criteria for relapsed/refractory warm antibody autoimmune hemolytic anemia (wAIHA), and the patient must have received treatment with at least one of rituximab or cyclophosphamide.
- Criteria for diagnosing relapsed/refractory warm antibody AIHA: It refers to warm antibody AIHA in which the patient has poor response to first-line and second-line or above standard treatments (e.g., glucocorticoids), or the disease recurs after effective treatment, or the patient requires continuous or repeated treatment to control the disease.
- Disease duration of more than 6 months, with persistent disease activity or progression despite receiving conventional treatment for ≥ 2 months, or recurrence of disease activity after disease remission. Definition of conventional treatment: Use of glucocorticoids plus at least one of the following immunomodulators: cyclophosphamide, cyclosporine, and biological agents (including rituximab, etc.).
- No systemic active infection (e.g., infectious pneumonia, pulmonary tuberculosis) within 2 weeks before leukapheresis.
- Expected survival time of more than 3 months from the date of signing the informed consent form.
- Peripheral blood routine meeting the following requirements simultaneously: absolute neutrophil count (ANC) ≥ 1000/μL; hemoglobin (HGB) ≥ 60 g/L; platelet count (PLT) ≥ 30,000/μL.
- Hepatic, renal, cardiopulmonary functions meeting the following requirements:
- Creatinine ≤ 1.5 × upper limit of normal (ULN);
- Left ventricular ejection fraction (LVEF) ≥ 50%;
- Oxygen saturation > 90%;
- Total bilirubin ≤ 4 × ULN;
- Alanine aminotransferase (ALT) and aspartate aminotransferase (AST) ≤ 2.5 × ULN.
- Studies in patients with an Eastern Cooperative Oncology Group (ECOG) performance status score of 0-2.
- Female study participants of childbearing potential (defined as all female participants who are physiologically capable of becoming pregnant) must agree to use highly effective contraceptive methods from at least 28 days before the start of lymphodepletion until 2 years after CAR-T cell infusion (including the period of study treatment with dose interruptions). Male participants whose partners are of childbearing potential must agree to use effective barrier contraceptive methods from the start of lymphodepletion until 2 years after CAR-T cell infusion, and must not donate semen or sperm throughout the study period.
- Female study participants of childbearing potential must have a negative result in serum beta-human chorionic gonadotropin (β-hCG) testing both at screening and within 48 hours before the first dose of lymphodepletion treatment.
- Participants must voluntarily agree to participate in this study and sign the informed consent form. Vulnerable populations may be included in the study. If a study participant is unable to read or sign the informed consent form due to incapacity or other reasons, their legal guardian must act as a proxy to go through the informed consent process and sign the form. If a study participant is unable to read the informed consent form (e.g., illiterate participants), a witness must be present to observe the informed consent process and sign the form.
Exclusion criteria
- Concomitant diagnosis of any type of tumor, which is deemed unsuitable for participation in this study by the investigator.
- A history of clinically significant central nervous system (CNS) diseases or pathological changes caused by non-autoimmune diseases prior to screening, including but not limited to: cerebrovascular accident, aneurysm, epilepsy, convulsions/seizures, aphasia, stroke, severe brain injury, dementia, Parkinson's disease, cerebellar disease, organic brain syndrome, or psychosis.
- A history of major organ transplantation (e.g., heart, lung, kidney, liver) or hematopoietic stem cell/bone marrow transplantation.
- Presence of IgA deficiency at screening (serum IgA level < 10 mg/dL).
- Presence of any of the following conditions at screening:
- Active hepatitis (hepatitis B virus deoxyribonucleic acid [HBV-DNA] or hepatitis C virus ribonucleic acid [HCV-RNA] test results above the lower limit of detection);
- Human immunodeficiency virus (HIV) infection, known acquired immunodeficiency syndrome (AIDS), or syphilis infection.
- A history of any of the following cardiovascular diseases within 6 months prior to screening: New York Heart Association (NYHA) Class III or IV heart failure, myocardial infarction, unstable angina pectoris, uncontrolled or symptomatic atrial arrhythmia, any ventricular arrhythmia, or other heart diseases with significant clinical significance.
- Having received any of the following treatments for autoimmune diseases:a) Use of therapeutic-dose corticosteroids (defined as prednisone or its equivalent > 20 mg/day) within 7 days before leukapheresis;b) Use of any other investigational drugs for autoimmune diseases within 4 weeks before leukapheresis, except for cases where the drug was ineffective or disease progressed during the investigational treatment, and at least 3 half-lives have passed before leukapheresis (enrollment is permitted in such cases);c) Previous receipt of CAR-T cell therapy or other genetically modified T cell therapies.
- A history of grade ≥ 2 bleeding within 30 days before screening, or long-term continuous treatment with anticoagulant drugs (e.g., warfarin, low-molecular-weight heparin, or factor Xa inhibitors, etc.).
- Having undergone plasma exchange, plasmapheresis, hemodialysis, or intravenous immunoglobulin (IVIG) administration within 14 days before leukapheresis.
- Use of any live vaccines for infectious diseases within 8 weeks before CAR-T cell infusion.
- Pregnant or lactating women.
- Known life-threatening allergic reactions, hypersensitivity reactions, or intolerance to the CAR-T cell preparation or its excipients (including DMSO).
- Poor compliance of the study participant as judged by the investigator, or unwillingness or inability to comply with other requirements of the study protocol.
Study description and what participation involves
The study's detailed description, as published on ClinicalTrials.gov.
Detailed description from the registry
With reference to the protocol by the team led by Georg Schett from Germany, a dosage of 1×10⁶ CAR⁺ cells per kilogram was selected, and it is planned to enroll 24 subjects with relapsed/refractory warm antibody autoimmune hemolytic anemia (wAIHA). The two-stage optimal design proposed by Bryant and Day was adopted: Stage 1: A total of 8 participants will be enrolled, with the core objective of evaluating the tolerance to treatment-related toxicity. By monitoring safety events (including grade ≥2 cytokine release syndrome (CRS), immune effector cell-associated neurotoxicity syndrome (ICANS), and grade 3-4 neutropenia lasting more than 28 days, etc.), it will be determined whether the number of such events is ≤ 5. Stage 2: A total of 16 participants will be enrolled, focusing on assessing treatment response, which requires that at least 4 participants achieve a definite therapeutic effect.
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 will undergo leukapheresis (blood cell collection) to make the CAR‑T product, receive short lymphodepleting chemotherapy (fludarabine + cyclophosphamide given about 5–3 days before infusion), then receive a CD19/BCMA CAR‑T cell infusion at the planned dose. Thirty minutes before infusion you will be given medication to help prevent allergic reactions. Expect frequent blood tests and safety monitoring for immune‑related toxicities and pharmacokinetic sampling at multiple timepoints, with follow-up visits and tests continuing up to about 24 months; contraception requirements described above must be followed during the study.
How the study is structured. Participants receive lymphodepletion (fludarabine + cyclophosphamide) then an infusion of CD19/BCMA CAR‑T cells at a set dose. The trial is open‑label and single‑arm, with a two‑stage plan: an initial group focused on treatment‑related toxicity, then a larger group to evaluate responses. Safety and CAR‑T levels are measured at multiple timepoints through 28 days and beyond.
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 registry says recruitment has not started. When is it expected to open, and can I register interest now?
- No sites are listed on the registry yet. Where will the study take place?
- 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
- Jan 31, 2026 (estimated)
- Primary completion
- Oct 31, 2027 (estimated)
- Study completion
- Oct 31, 2027 (estimated)
- Target enrollment
- 24 participants (estimated)
- Last updated on registry
- Jan 8, 2026
- 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
- Yihao Wang
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.
- Yihao Wang 📞 139 2038 6359 ✉ yhwtmu@126.com
Participating sites
Study locations have not been posted on ClinicalTrials.gov yet. This study is not yet recruiting, so sites may be added when recruitment begins. The central study contact above may be able to tell you about planned sites.
Terms used on this page · explanations by MedicalUpdates.ai
- open-label
- No masking is used, so the assigned intervention is not deliberately hidden from participants or study staff.
- single-arm
- All participants receive the same treatment or follow the same plan. There is no comparison group within the study.
- pharmacokinetic
- How the body absorbs, distributes, breaks down and removes a drug. It is studied by measuring drug levels or related substances at set times after a dose.
- 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.
- relapsed/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.
- 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.
- absolute 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.
- upper limit of normal
- 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.
- 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.
- 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.
- at screening
- The checks done before enrolment to see whether a person qualifies, such as tests, scans or a review of medical history.
- legal guardian
- A person who is legally allowed to make decisions, including consent to research, on behalf of someone who cannot consent for themselves.
- investigational
- A treatment that is being tested and has not been approved for this use. It may be approved for a different condition. Being studied does not mean it works or is available outside the study.
- half-lives
- 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.
- enrollment
- The number of participants a study plans to include, or has included. 'Estimated' means the planned number; 'actual' means the number who joined.
- 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.
- cytokine release syndrome
- A reaction in which the immune system releases a large amount of signalling proteins at once, causing fever and sometimes low blood pressure or breathing problems. It can occur after some immune therapies, such as CAR-T cells or bispecific antibodies.
- immune effector cell-associated neurotoxicity syndrome
- Nervous-system side effects, such as confusion or difficulty speaking, that can occur after some immune therapies. Usually temporary, but monitored closely.
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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