ClinicalTrials.gov record NCT06920446
UCAR T-cell Therapy Targeting CD19/BCMA in Patients With r/r Autoimmune Hemolytic Anemia
A Clinical Study Evaluating the Safety and Preliminary Efficacy of Universal Allogeneic CAR T-cell Therapy Targeting CD19 and BCMA in Patients With Relapsed / Refractory Autoimmune Hemolytic Anemia
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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 early-phase, open-label study tests a single infusion of universal (allogeneic) anti-CD19/BCMA CAR T-cells given after a short lymphodepleting chemotherapy regimen. It is a dose-escalation study in up to 18 adults with relapsed or refractory autoimmune hemolytic anemia (AIHA) who have previously received multiple treatments. The trial will track safety, side effects, and clinical responses over time.
What it is studying. The study is testing the safety and preliminary effectiveness of a single intravenous dose of universal anti-CD19/BCMA CAR T-cells given after lymphodepleting therapy (fludarabine and cyclophosphamide). Investigators will record dose-limiting toxicities, other adverse events, and clinical response measures over follow-up periods specified in the registry.
What the researchers hope to learn. Scientific goals: evaluate safety (including dose-limiting toxicities and adverse events) and collect preliminary data on clinical responses to universal anti-CD19/BCMA CAR T-cells in relapsed/refractory AIHA. The study also uses a dose-escalation design to identify tolerable dosing.
What this study is about
The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.
This is an open label, single-site, dose-escalation study in up to 18 participants with relapsed or refractory Autoimmune hemolytic anemia. This study aims to evaluate the safety and efficacy of the treatment with universal CD19/BCMA CAR T-cells.
Interventions being studied
Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.
- Biological
- UCAR T-cell group
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.
- Functional requirements for major organs are as follows:
- The bone marrow function needs to meet: a Neutrophil count ≥ 0.5× 10 \^ 9/L; b. Platelets ≥ 30 × 10 \^ 9/L.
- Liver function: ALT ≤ 3 × UL; AST ≤ 3×ULN.
- Renal function: creatinine clearance rate (CrCl) ≥ 30 ml/min (Cockcroft/Gault formula).
- ECOG ≤ 2
- 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 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.
- 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 CD19/BCMA CAR T-cells in Relapsed or Refractory Autoimmune hemolytic anemia. Study intervention consists of a single infusion of universal 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 90 days 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 anti‑CD19/BCMA CAR T‑cells. They will be monitored closely for side effects with dose‑limiting toxicities assessed within 28 days and adverse events followed to 90 days; clinical response is assessed up to 24 months and an interim analysis is planned after the 90‑day visit. Participation involves regular clinic visits, physical exams and blood tests per the study schedule. Participants must sign informed consent and follow the study contraception and pregnancy‑testing requirements.
How the study is structured. An open-label, single-group, early-phase (dose-escalation) interventional study. Participants receive lymphodepleting chemotherapy followed by one IV infusion of universal anti-CD19/BCMA CAR T-cells. No masking; up to 18 participants; safety monitored closely with specified timepoints (DLTs assessed within 28 days, adverse events up to 90 days, clinical responses followed up to 24 months).
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
- Apr 3, 2025 (estimated)
- Primary completion
- April 2026 (estimated)
- Study completion
- November 2027 (estimated)
- Target enrollment
- 18 participants (estimated)
- Last updated on registry
- Apr 9, 2025
- 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
- Zhejiang University
- Principal investigator
- He Huang, Principal Investigator
Zhejiang University - Collaborators
- Shanghai Xiniao Biotech Co., Ltd.
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.
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.
- dose-escalation
- Groups of participants receive increasing doses of a treatment, one group after another, to find a dose that can be given safely.
- relapsed or 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.
- 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.
- 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.
- 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.
- 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.
- 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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