Relapsed/Refractory Warm Antibody Autoimmune Hemolytic AnemiaPhase 1/2NCT07324889
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
A Phase 1/2 study testing CD19/BCMA CAR-T in people with Relapsed/Refractory Warm Antibody Autoimmune Hemolytic Anemia, run by Yihao Wang.
Trial summary
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… Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Relapsed/Refractory Warm Antibody Autoimmune Hemolytic Anemia
- What is being tested?
- CD19/BCMA CAR-T
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 24 participants
- Where
- No sites listed yet
- Study dates
- 31 Jan 2026to 31 Oct 2027
- Phase
- Phase 1/2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
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- 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.
Who cannot join
- 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);
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- 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.
Trial locations and contacts
No sites are listed yet. Contact the study team or a site near you to ask about taking part.
About this study
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.
Read the detailed description
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.
- Being tested
- CD19/BCMA CAR-T
- Medical subject headings
- Recurrence
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CD19/BCMA CAR-TDrug
Eligible participants should receive preconditioning 5 to 3 days before CAR-T cell infusion. The recommended preconditioning regimen is fludarabine (30 mg/m²/day for 3 consecutive days) and cyclophosphamide (300 mg/m²/day for 3 consecutive days) (Flu/Cy). Thirty minutes before infusion, medications for preventing allergic reactions should be administered: 25 mg of promethazine hydrochloride or 12.5 mg of diphenhydramine, which can be given intramuscularly or orally. Adopting the two-stage optimal design proposed by Bryant and Day, it is planned to enroll 24 subjects with relapsed/refractory warm antibody autoimmune hemolytic anemia, with a selected dosage of 1×10⁶ CAR⁺ cells/kg.
| Study group | Role | What they receive |
|---|---|---|
| CD19/BCMA CAR-T Cell Therapy for Relapsed/Refractory Warm Antibody Autoimmune Hemolytic Anemia | Other | Not described Receives: Drug: CD19/BCMA CAR-T |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence and Severity of Adverse Events
Evaluate the number of cases, incidence rate, and severity of various adverse events after CD19/BCMA CAR-T infusion, mainly focusing on immunotherapy-related toxic reactions such as cytokine release syndrome (CRS), immune effector cell therapy-associated
Measured: Within 28 days after CAR-T infusion
Secondary outcomes (6)
Pharmacokinetics-AUC(0~28days)
Area under the curve of CAR T cells from time zero to Day 28
Measured: Day 7, Day10, Day 14, Day 28 after treatment
Overall response rate (ORR) evaluated by the investigators
Efficacy of CD19/BCMA CAR-T in the treatment of refractory autoimmune diseases, include: complete response (CR) rate and partial response (PR) rate (defined as a reduction of ≥50% in disease activity) at day28, month 3 and month 6 after treatment;
Measured: Day 28, Month 3, Month 6 after treatment
Changes in immunological indicators and hemolysis-related indicators after infusion.
Bilirubin, LDH, conjugated hemoglobin, free hemoglobin, Direct anti human globulin test (DAT) etc
Measured: Day 28, Month 2, Month 3, Month 6, Month 12, and Month 24 after treatment
Pharmacokinetics-Tmax
Time to peak transgene level
Measured: Day 7, Day10, Day 14, Day 28 after treatment
Pharmacokinetics-Tlast
The longest detectable CART copy number time (day)
Measured: Day7, Day10, Day14, Day28, Month2 , Month3 , Month6 , Month9, Month12 after treatment
Progression-free Survival (PFS)
Time from CAR-T infusion to first documentation of progressive disease (PD), or death due to any cause, whichever occurs first
Measured: Minimum of 1 years post CAR-T infusion
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Jan 2026
- Study start31 Jan 2026 (estimated)
- Primary completion31 Oct 2027 (estimated)
- Study completion31 Oct 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Relapsed/Refractory Warm Antibody Autoimmune Hemolytic AnemiaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 24 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Yihao Wang
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, Yihao Wang, Tianjin Medical University General Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07324889
- Sponsor's protocol ID
- IRB2025-YX-220-01
- Status last verified
- Oct 2025
- First submitted
- 23 Dec 2025
- Last updated
- 8 Jan 2026
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 8 Jan 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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
- ClinicalTrials.gov ID
- NCT07324889
- Acronym / sponsor ID
- IRB2025-YX-220-01
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 24 participants (estimated)
- Lead sponsor
- Yihao WangUniversity, hospital or other
- Study start
- 31 Jan 2026 (estimated)
- Primary completion
- 31 Oct 2027 (estimated)
- Study completion
- 31 Oct 2027 (estimated)
- First posted
- 8 Jan 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| CD19/BCMA CAR-T Cell Therapy for Relapsed/Refractory Warm Antibody Autoimmune Hemolytic Anemia | Other | Drug: CD19/BCMA CAR-T |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (17 listed)
- 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.
Key exclusion criteria (15 listed)
- 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:
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| CD19/BCMA CAR-TEligible participants should receive preconditioning 5 to 3 days before CAR-T cell infusion. The recommended preconditioning regimen is fludarabine (30 mg/m²/day for 3 consecutive days) and cyclophosphamide (300 mg/m²/day for 3 consecutive days) (Flu/Cy). Thirty minutes before infusion, medications for preventing allergic reactions should be administered: 25 mg of promethazine hydrochloride or 12.5 mg of diphenhydramine, which can be given intramuscularly or orally. Adopting the two-stage optimal design proposed by Bryant and Day, it is planned to enroll 24 subjects with relapsed/refractory warm antibody autoimmune hemolytic anemia, with a selected dosage of 1×10⁶ CAR⁺ cells/kg. | Drug | CD19/BCMA CAR-T Cell Therapy for Relapsed/Refractory Warm Antibody Autoimmune Hemolytic Anemia |
Primary outcome
Incidence and Severity of Adverse Events
Time frame: Within 28 days after CAR-T infusion
Evaluate the number of cases, incidence rate, and severity of various adverse events after CD19/BCMA CAR-T infusion, mainly focusing on immunotherapy-related toxic reactions such as cytokine release syndrome (CRS), immune effector cell therapy-associated
6 secondary outcomes
Pharmacokinetics-AUC(0~28days)
Time frame: Day 7, Day10, Day 14, Day 28 after treatment
Overall response rate (ORR) evaluated by the investigators
Time frame: Day 28, Month 3, Month 6 after treatment
Changes in immunological indicators and hemolysis-related indicators after infusion.
Time frame: Day 28, Month 2, Month 3, Month 6, Month 12, and Month 24 after treatment
Pharmacokinetics-Tmax
Time frame: Day 7, Day10, Day 14, Day 28 after treatment
Pharmacokinetics-Tlast
Time frame: Day7, Day10, Day14, Day28, Month2 , Month3 , Month6 , Month9, Month12 after treatment
Progression-free Survival (PFS)
Time frame: Minimum of 1 years post CAR-T infusion
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Jan 2026.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing CD19/BCMA CAR-T in people with Relapsed/Refractory Warm Antibody Autoimmune Hemolytic Anemia, run by Yihao Wang.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 17 inclusion and 15 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: opening soon, as of the sponsor's last update on 8 Jan 2026. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Yihao Wang (139 2038 6359, yhwtmu@126.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence and Severity of Adverse Events (time frame: Within 28 days after CAR-T infusion). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 8 Jan 2026.