Autoimmune Hemolytic AnemiaPhase 1NCT06733610
CAR T-cell Therapy Targeting CD19 and BCMA in Patients with AIHA Who Have Failed ≥3 Lines of Therapy.
A Phase 1 study testing universal allogeneic anti-CD19/BCMA CAR T-cells in people with Autoimmune Hemolytic Anemia and CD19/BCMA CAR T-cells, run by Institute of Hematology & Blood Diseases Hospital, China at 1 site in China.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Autoimmune Hemolytic Anemia, CD19/BCMA CAR T-cells, Universal Allogeneic CAR T-cells
- What is being tested?
- universal allogeneic anti-CD19/BCMA CAR T-cells
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 15 participants
- Where
- 1 siteChina
- Study dates
- 5 Dec 2024to 5 Dec 2026
- Phase
- Phase 1
- 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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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:
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- . 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.
Who cannot join
- 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
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- 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.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site, 1 recruiting
- Recruiting
Regenerative Medicine Center and Red Blood Cell Disorders Center
Tianjin, Tianjin Municipality
Lele Zhang, PhD, 15811139278, zhanglele@ihcams.ac.cn
About this study
Official title: 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
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.
Read the detailed description
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.
- Being tested
- universal allogeneic anti-CD19/BCMA CAR T-cells
- Medical subject headings
- Anemia, Hemolytic, Autoimmune
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
universal allogeneic anti-CD19/BCMA CAR T-cellsBiological
A single injection of UCAR T-cells, referred to as universal allogeneic anti-CD19/BCMA CAR T-cells.
| Study group | Role | What they receive |
|---|---|---|
| UCAR T-cell group | Experimental | Universal allogeneic anti-CD19/BCMA CAR T-cells. Receives: Biological: universal allogeneic anti-CD19/BCMA CAR T-cells |
What is being measured
The primary outcome is the main question the study is designed to answer.
The number and severity of dose-limiting toxicity (DLT) events
DLT will be graded according to the NCI Common Terminology Criteria for Adverse Events (CTCAE) Version 5.0, and the ASTCT Consensus Grading for Cytokine Release Syndrome and Neurologic Toxicity Associated with Immune Effector Cells.
Measured: Within 28 Days After UCAR T-cell Infusion
The total number, incidence, and severity of AEs
Measured: Up to 12 Months After UCAR T-cell Infusion
Clinical response of AIHA who have failed ≥ 3 lines of therapy
Rates of CR, CRi, PR, ORR
Measured: Up to 24 Weeks After UCAR T-cell Infusion
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Dec 2024
- Study start5 Dec 2024
- Primary completion5 Feb 2026 (estimated)
- Study completion5 Dec 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for 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
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 15 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Institute of Hematology & Blood Diseases Hospital, China
- Sponsor type
- University, hospital or other
- Collaborators
- Xi'niao Biotech
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06733610
- Sponsor's protocol ID
- IIT2024096
- Status last verified
- Dec 2024
- First submitted
- 6 Dec 2024
- Last updated
- 13 Dec 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 13 Dec 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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
- ClinicalTrials.gov ID
- NCT06733610
- Acronym / sponsor ID
- IIT2024096
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 15 participants (estimated)
- Lead sponsor
- Institute of Hematology & Blood Diseases Hospital, ChinaUniversity, hospital or other
- Collaborators
- Xi'niao Biotech
- Study start
- 5 Dec 2024 (actual)
- Primary completion
- 5 Feb 2026 (estimated)
- Study completion
- 5 Dec 2026 (estimated)
- First posted
- 13 Dec 2024 (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 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.
Detailed description
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| UCAR T-cell group | Experimental | Biological: universal allogeneic anti-CD19/BCMA CAR T-cellsUniversal allogeneic anti-CD19/BCMA CAR T-cells. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (11 listed)
- 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
Key exclusion criteria (13 listed)
- 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
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 |
|---|---|---|
| universal allogeneic anti-CD19/BCMA CAR T-cellsA single injection of UCAR T-cells, referred to as universal allogeneic anti-CD19/BCMA CAR T-cells. | Biological | UCAR T-cell group |
Primary outcome
The number and severity of dose-limiting toxicity (DLT) events
Time frame: Within 28 Days After UCAR T-cell Infusion
DLT will be graded according to the NCI Common Terminology Criteria for Adverse Events (CTCAE) Version 5.0, and the ASTCT Consensus Grading for Cytokine Release Syndrome and Neurologic Toxicity Associated with Immune Effector Cells.
The total number, incidence, and severity of AEs
Time frame: Up to 12 Months After UCAR T-cell Infusion
Clinical response of AIHA who have failed ≥ 3 lines of therapy
Time frame: Up to 24 Weeks After UCAR T-cell Infusion
Rates of CR, CRi, PR, ORR
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 13 Dec 2024.
- Individual participant dataSharing planned: No
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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.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing universal allogeneic anti-CD19/BCMA CAR T-cells in people with Autoimmune Hemolytic Anemia and CD19/BCMA CAR T-cells, run by Institute of Hematology & Blood Diseases Hospital, China at 1 site in China.
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 11 inclusion and 13 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: looking for participants now, as of the sponsor's last update on 13 Dec 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
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 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.
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 Jun Shi, PhD (13752253515, shijun@ihcams.ac.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
The number and severity of dose-limiting toxicity (DLT) events (time frame: Within 28 Days After UCAR T-cell Infusion). The study lists 3 primary outcomes in all. 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 13 Dec 2024.