UCAR T-cell Therapy Targeting CD19/BCMA in Patients With r/r Autoimmune Hemolytic Anemia

NCT06920446 | Not Yet Recruiting Early Phase 1

• 1 other identifier: QH-ZY-02
• Study Type: interventional
• Target: 18
• Locations: 0 countries
• Sites: N/A

Brief Summary

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.

Conditions

Relapsed / Refractory Autoimmune Hemolytic Anemia

Keywords

Universal Allogeneic CAR T-cells; CD19/BCMA CAR T-cells

Outcome Measures

Primary Outcomes (3)

• 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.

  Within 28 Days After UCAR T-cell Infusion

• The total number, incidence, and severity of AEs

  Up to 90 days After UCAR T-cell Infusion

• Clinical response

  Rates of CR, CRi, PR, ORR

  Up to 24 Months After UCAR T-cell Infusion

Study Arms (1)

anti-CD19/BCMA CAR T-cells

EXPERIMENTAL

UCAR T-cell group

Biological: UCAR T-cell group

Interventions

UCAR T-cell group BIOLOGICAL

A single injection of UCAR T-cells, referred to as universal allogeneic anti-CD19/BCMA CAR T-cells

anti-CD19/BCMA CAR T-cells

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Zhejiang University: lead
• Shanghai Xiniao Biotech Co., Ltd.: collaborator

MeSH Terms

Conditions

Anemia, Hemolytic, Autoimmune

Condition Hierarchy (Ancestors)

Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Autoimmune Diseases; Immune System Diseases

Study Design

• Study Type: interventional
• Phase: early phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Principal Investigator

Study Record Dates

• First Submitted: April 2, 2025
• First Posted: April 9, 2025
• Study Start: April 3, 2025
• Primary Completion: April 1, 2026
• Study Completion (Estimated): November 1, 2027
• Last Updated: April 9, 2025

Record last verified: 2025-04