UCAR T-cell Therapy Targeting CD19/BCMA in Relapsed/Refractory Autoimmune Hemolytic Anemia

A Clinical Study of CD19/BCMA-Targeted Universal Allogeneic CAR-T Cell Therapy in Relapsed/Refractory Autoimmune Hemolytic Anima: Evaluating Safety and Preliminary Efficacy

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

Study Overview

• Status: Recruiting
• Conditions: AIHA - Warm Autoimmune Hemolytic Anemia; UCART
• Intervention / Treatment: Biological: QT-219C Cell Injection

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 Type: Interventional
• Enrollment (Estimated): 15
• Phase: Early Phase 1

Contacts and Locations

• Study Contact: Name: Zhimin Zhai, PhD; Phone Number: +86-0551-65997091; Email: zzzm889@163.com

Study Locations

• China
  • Hefei, China
    • Recruiting
    • The Second Hospital of Anhui Medical University
    • Contact: Zhimin Zhai, PhD; Phone Number: +86-0551-65997091; Email: zzzm889@163.com

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• 1. Age ≥ 10 years, regardless of sex;
• 2. Flow cytometry-confirmed CD19 or BCMA positivity on B cells in peripheral blood or bone marrow;
• 3. 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)";
• 4. 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;

• 5. Functional requirements for major organs are as follows:

  1. The bone marrow function needs to meet: a Neutrophil count ≥ 1.0

     × 10 ^ 9/L; b. Platelets ≥ 30 × 10 ^ 9/L.

  2. Liver function: ALT ≤ 3 × UL; AST ≤ 3×ULN# Total bilirubin ≤ 2.0 × ULN (excluding Gilbert syndrome, total bilirubin ≤ 3.0 × ULN).
  3. Renal function: creatinine clearance rate (CrCl) ≥ 30 ml/min (Cockcroft/Gault formula, excluding acute CrCl decline caused by the disease itself).
• 6. ECOG ≤ 2;
• 7. 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;
• 8. Willing to participate in this clinical study, sign an informed consent form, have good compliance, and cooperate with follow-up.

Exclusion Criteria:

• 1. Subjects with a history of severe drug allergies or allergic tendencies;
• 2. Presence or suspicion of uncontrolled or treatment-required fungal, bacterial, viral, or other infections;
• 3. History of recurrent infections (e.g., ≥3 episodes of active infection requiring medical intervention within 6 months prior to enrollment);
• 4. History of cytomegalovirus (CMV), Epstein-Barr virus (EBV), or fungal infections within 3 months prior to screening, or history of recurrent CMV, EBV, or fungal infections;
• 5. Receipt of any vaccination within 12 weeks prior to enrollment, or participation in a vaccine clinical trial within 12 weeks prior to enrollment;
• 6. Subjects with insufficient cardiac function;
• 7. Moderate to severe congestive heart failure (New York Heart Association [NYHA] Class III-IV);
• 8. Subjects with congenital immunoglobulin deficiencies;
• 9. History of malignancy within the past 5 years (except for non-melanoma skin cancer, completely resected Stage I tumor with low risk of recurrence, treated clinically localized prostate cancer, biopsy-proven cervical carcinoma in situ or squamous intraepithelial lesion on smear, and stable papillary or follicular thyroid cancer);
• 10. 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;
• 11. History of organ transplantation, including but not limited to bone marrow or hematopoietic stem cell transplantation;
• 12. Severe, progressive, uncontrolled disease of the cardiovascular, cerebrovascular, hepatic, renal, pulmonary, gastrointestinal, hematologic, endocrine, or nervous system;
• 13.Psychiatric disorder or severe cognitive impairment;
• 14. Pregnant women or women planning to conceive
• 15. Subjects that the investigator believes have other reasons that make them unsuitable for inclusion in this study

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: QT-219C Cell Injection; Universal allogeneic anti-CD19/BCMA CAR T-cells	Biological: QT-219C Cell Injection; A single injection of UCAR T-cells, referred to as universal allogeneic anti-CD19/BCMA CAR T-cells.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Clinical response of AIHA who have failed ≥ 3 lines of therapy	Rates of CR, CRi, PR, ORR	Up to 24 Weeks After UCAR T-cell Infusion
Incidence of Dose-Limiting Toxicities (DLTs)	The number, frequency, and severity of DLTs experienced by subjects after the first infusion of QT-219C. DLTs are defined by NCI-CTCAE 5.0 and ASTCT consensus for CRS and neurotoxicity	Day 0 to Day 28 post-infusion
Incidence of Adverse Events (AEs)	Evaluation of the number, frequency, and severity of all adverse events, including Treatment-Emergent Adverse Events (TEAEs), Treatment-Related Adverse Events (TRAEs), and Serious Adverse Events (SAEs).	Up to 12 Months After UCAR T-cell Infusion

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Tmax of CAR-T cells [PK parameter]	The time of amplified UCAR-T cells in peripheral blood to reach the maximum concentration (Tmax).	Within 28 Days After UCAR T-cell Infusion
Cmax of CAR-T cells [PK parameter]	The peak plasma concentration (Cmax) of amplified UCAR-T cells in peripheral blood after infusion	Within 28 Days After UCAR T-cell Infusion
AUC 0-28d of UCAR-T cells [PK parameter]	The area under the plasma concentration-time curve from 0 to 28 days after infusion (AUC0-28d)	Within 28 Days After UCAR T-cell Infusion

Collaborators and Investigators

• Sponsor: The Second Hospital of Anhui Medical University
• Investigators: Principal Investigator: Zhai, The Second Hospital of Anhui Medical University

Study record dates

Study Major Dates

• Study Start (Estimated): April 1, 2026
• Primary Completion (Estimated): December 31, 2029
• Study Completion (Estimated): December 31, 2030

Study Registration Dates

• First Submitted: April 8, 2026
• First Submitted That Met QC Criteria: April 8, 2026
• First Posted (Actual): April 15, 2026

Study Record Updates

• Last Update Posted (Actual): April 15, 2026
• Last Update Submitted That Met QC Criteria: April 8, 2026
• Last Verified: April 1, 2026

More Information

Terms related to this study

• Keywords: AIHA; UCART
• Other Study ID Numbers: QH-AY-02

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No