CAR T-cell Therapy Targeting CD19 and BCMA in Patients With Relapse/Refractory Autoimmune Diseases

NCT06941129 | Recruiting Phase 1

• 1 other identifier: IIT2024107
• Study Type: interventional
• Target: 12
• Locations: 1 country
• Sites: 1

Brief Summary

CAR T-cell Therapy Targeting CD19 and BCMA in Patients With Relapse/Refractory Autoimmune Diseases

Conditions

Systemic Lupus Erythematosus; Systemic Sclerosis (SSc); Inflammatory Myopathy; ANCA-Associated Vasculitis (AAV); Connective Tissue Disease-Associated Thrombocytopenia; SLE-ITP

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

  The total number, incidence, and severity of AEs

  Up to 12 Months After UCAR T-cell Infusion

• Clinical response of R/R AIDs

  Clinical response of R/R AIDs

  Up to 24 Months After UCAR T-cell Infusion

Study Arms (1)

UCAR T-cell group

EXPERIMENTAL

Universal allogeneic anti-CD19/BCMA CAR T-cells. Biological/Vaccine: universal allogeneic anti-CD19/BCM

Biological: UCAR T-cell

Interventions

UCAR T-cell BIOLOGICAL

universal allogeneic anti-CD19/BCMA CAR T-cells

UCAR T-cell group

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 old (inclusive), regardless of gender.
• Positive expression of CD19 or BCMA on peripheral blood B cells confirmed by flow cytometry.
• Functional requirements for major organs are as follows（Except for abnormalities related to autoimmune disease activity）: 1) Bone marrow function must meet: A. Neutrophil count ≥ 1×109/L (no colony-stimulating factor treatment within 2 weeks before examination); B. Hemoglobin ≥ 60g/L; 2) Liver function: Alanine aminotransferase (ALT) ≤ 3×ULN (excluding ALT elevation due to inflammatory myopathy), aspartate aminotransferase (AST)≤3×Upper limit of normal (ULN) (excluding AST elevation due to inflammatory myopathy), TBIL≤2×ULN (or ≤ 3.0×ULN for subjects with Gilbert syndrome); 3) Renal function: creatinine clearance rate (CrCl) ≥ 30ml/minute (calculated by Cockcroft/Gault formula, acute CrCl decrease due to the target disease is excluded; LN is exluded).
• ECOG score 0-2.
• 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.
• Refractory/Relapsed Systemic Lupus Erythematosus:
• SLE meeting the 2019 the American College of Rheumatology (ACR) /European League Against Rheumatism (EULAR) and classification criteria.
• Disease activity score SLEDAI-2000 ≥ 8; or with significant organ involvement, such as lupus nephritis (histologically confirmed active nephritis of class III or IV, with or without class V, with an NIH activity index \> 2, evidence of increased chronicity index; urine protein-to-creatinine ratio \> 1.0 g/g, or 24-hour urinary protein \> 1.0 g).
• Definition of refractory or relapsing disease: lack of response after more than 6 months of conventional therapy, or recurrence of disease activity after remission. Conventional therapy is defined as treatment with glucocorticoids in combination with one or more of the following immunomodulatory agents: cyclophosphamide, antimalarial drugs, azathioprine, mycophenolate mofetil, methotrexate, leflunomide, tacrolimus, cyclosporine, as well as biologics such as rituximab, belimumab, and telitacicept.
• Refractory/Relapsed/Progressive Systemic Sclerosis:
• Scleroderma fulfilling the 2013 ACR classification criteria.
• Positive scleroderma-related antibodies.
• Presence of diffuse cutaneous sclerosis or active interstitial lung disease (high-resolution computed tomography (HRCT) showing ground-glass opacities).
• Definition of relapsed/refractory: Conventional treatment over 6 months remains ineffective, or disease recurrence after remission. Definition of conventional treatment: the use of glucocorticoids , and any one or more of the following immunomodulatory drugs: cyclophosphamide, antimalarials, azathioprine, mycophenolate mofetil, methotrexate, leflunomide, tacrolimus, cyclosporine, and biologics including belimumab, rituximab, and tocilizumab, etc.

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(LN is excluded).
• 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 participated in other clinical trials within the past 3 months prior to enrollment.
• Subjects who have received immunosuppressive agents or biologics with therapeutic effects for indications within 5 half-life prior to enrollment.
• Pregnant women or women planning to conceive.

Sponsors & Collaborators

• Institute of Hematology & Blood Diseases Hospital, China: lead
• Shanghai Xiniao Biotech Co., Ltd.: collaborator

Study Sites (1)

Institute of Hematology & Blood Diseases Hospital

Tianjin, China

RECRUITING

MeSH Terms

Conditions

Lupus Erythematosus, Systemic; Scleroderma, Systemic; Myositis; Anti-Neutrophil Cytoplasmic Antibody-Associated Vasculitis

Condition Hierarchy (Ancestors)

Connective Tissue Diseases; Skin and Connective Tissue Diseases; Autoimmune Diseases; Immune System Diseases; Skin Diseases; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Systemic Vasculitis; Vasculitis; Vascular Diseases; Cardiovascular Diseases; Skin Diseases, Vascular

Central Study Contacts

Ying Wang, PhD

CONTACT

86-22-23909278; wangying1@ihcams.ac.cn

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 16, 2025
• First Posted: April 23, 2025
• Study Start: February 11, 2025
• Primary Completion (Estimated): February 18, 2028
• Study Completion (Estimated): March 18, 2028
• Last Updated: September 29, 2025

Record last verified: 2025-02

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)