Exploratory Study on LNP-mRNA Drugs Targeting CD19/BCMA for the Treatment of Relapsed and Refractory Autoimmune Diseases

Sponsor
West China Hospital
Study ID
NCT07388212
Phase
EARLY_PHASE1
Status
Not Yet Recruiting

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Conditions

  • Autoimmune Diseases

Eligibility Criteria

Sex
ALL
Age
18 Years - 70 Years
Healthy Volunteers
Not accepted

Interventions

  • CD19/BCMA in vivo CAR-T — DRUG
    CD19/BCMA in vivo CAR-T based on LNP-mRNA

Study Details

Autoimmune diseases are a type of chronic disabling disease, characterized by the immune system incorrectly recognizing and attacking the body's own tissues, leading to tissue damage and organ dysfunction, seriously threatening life and health. Although there are various treatment methods currently available, there are still many limitations to immune related diseases that aim for long-term remission, and further research and breakthroughs are urgently needed. Targeting and clearing B cells is one of the core strategies in the treatment of autoimmune diseases. Its mechanism mainly involves clearing abnormally activated B cells, reducing the production of autoantibodies, and regulating immune disorders. The therapy targeting CD19 has become an important research and development direction due to its ability to clear a wider range of B cell lineages (including plasma cells), which may achieve deeper levels of B cell depletion than CD20 targeted therapy. In addition, BCMA is highly expressed specifically on plasma cells, especially long-lived plasma cells, and is a key target for clearing the source of antibody production. Therefore, the CD19/BCMA dual target therapy aims to achieve more complete coverage of the antibody production pathway by simultaneously targeting B cells (and precursors) and plasma cells, which is expected to further improve the treatment response rate and achieve deeper and more persistent immune reset. The advent of COVID-19 vaccine has brought LNP mRNA technology into the public's view. After years of development, it not only shines brilliantly in COVID-19 vaccine, but also is widely used in the treatment and exploration of cancer, rare diseases and other fields. The core of LNP mRNA technology targeting CD19/BCMA is to encapsulate the mRNA encoding specific proteins (such as anti-CD19/BCMA related proteins) in lipid nanoparticles and deliver them to the body through intravenous or intramuscular injection. This experimental drug is a messenger ribonucleic acid (mRNA) therapeutic drug based on the dual targets of CD19 and BCMA, formed by loading mRNA encoding CD19/BCMA receptor related proteins onto lipid nanoparticles (LNP) for injection.

Key Dates

First listed
Feb 5, 2026
Start date
Feb 23, 2026
Status verified
Jan 2026
Primary completion
Jan 1, 2028
Completion
Dec 31, 2028

Study Design

Enrollment
27 participants (estimated)
Allocation
NON_RANDOMIZED
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT

Arms

  • Experimental: CD19/BCMA in vivo CAR-T, Escalation doses
  • Experimental: CD19/BCMA in vivo CAR-T, Extended doses

Primary Outcome Measure

Dose limiting toxicity (DLT) and its incidence rate [ Time Frame: Within 28 days after the initial treatment ]

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