- ICH GCP
- US Clinical Trials Registry
- Klinisk forsøg 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
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.
Studieoversigt
Status
Intervention / Behandling
Detaljeret beskrivelse
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.
Undersøgelsestype
Tilmelding (Faktiske)
Fase
- Fase 1
Kontakter og lokationer
Studiesteder
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Tianjin Municipality
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Tianjin, Tianjin Municipality, Kina
- Regenerative Medicine Center and Red Blood Cell Disorders Center
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Deltagelseskriterier
Berettigelseskriterier
Aldre berettiget til at studere
Tager imod sunde frivillige
Køn, der er berettiget til at studere
Beskrivelse
Inclusion Criteria:
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Age ≥ 18 years
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Flow cytometry detected positive B cell CD19 or BCMA in the patient's peripheral blood.
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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)"
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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.
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ECOG ≤ 2
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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).
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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.
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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.
Studieplan
Hvordan er undersøgelsen tilrettelagt?
Design detaljer
- Primært formål: Behandling
- Tildeling: N/A
- Interventionel model: Enkelt gruppeopgave
- Maskning: Ingen (Åben etiket)
Våben og indgreb
Deltagergruppe / Arm |
Intervention / Behandling |
|---|---|
|
Eksperimentel: UCAR T-cell group
Universal allogeneic anti-CD19/BCMA CAR T-cells. |
A single injection of UCAR T-cells, referred to as universal allogeneic anti-CD19/BCMA CAR T-cells. |
Hvad måler undersøgelsen?
Primære resultatmål
Resultatmål |
Foranstaltningsbeskrivelse |
Tidsramme |
|---|---|---|
|
Det samlede antal, forekomst og sværhedsgrad af AE'er
Tidsramme: Op til 12 måneder efter UCAR T-celle infusion
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Op til 12 måneder efter UCAR T-celle infusion
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|
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The number and severity of dose-limiting toxicity (DLT) events
Tidsramme: Within 28 Days After UCAR T-cell Infusion
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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. |
Within 28 Days After UCAR T-cell Infusion
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Clinical response of AIHA who have failed ≥ 3 lines of therapy
Tidsramme: Up to 24 Weeks After UCAR T-cell Infusion
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Rates of CR, CRi, PR, ORR |
Up to 24 Weeks After UCAR T-cell Infusion
|
Samarbejdspartnere og efterforskere
Samarbejdspartnere
Datoer for undersøgelser
Studer store datoer
Studiestart (Faktiske)
Primær færdiggørelse (Anslået)
Studieafslutning (Anslået)
Datoer for studieregistrering
Først indsendt
Først indsendt, der opfyldte QC-kriterier
Først opslået (Faktiske)
Opdateringer af undersøgelsesjournaler
Sidste opdatering sendt (Faktiske)
Sidste opdatering indsendt, der opfyldte kvalitetskontrolkriterier
Sidst verificeret
Mere information
Begreber relateret til denne undersøgelse
Yderligere relevante MeSH-vilkår
Andre undersøgelses-id-numre
- IIT2024096
Plan for individuelle deltagerdata (IPD)
Planlægger du at dele individuelle deltagerdata (IPD)?
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