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

January 30, 2026 updated by: Jiyan Liu, West China Hospital

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.

Study Overview

Status

Not yet recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Estimated)

27

Phase

  • Early Phase 1

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  • 1. Age range of 18-70 years old (including threshold), gender not limited;
  • 2. KPS score>60 points, life expectancy greater than 6 months;
  • 3. Male and female patients of appropriate age must use reliable methods of contraception before entering the trial, during the research process until 30 days after discontinuation of medication; Reliable contraceptive methods will be determined by the primary researchers or designated personnel;
  • 4. Those who can understand this experiment and have signed the informed consent form;
  • 5. Before screening (at baseline), corresponding conditions should be met;
  • 6. Disease related inclusion criteria (study participants must meet one of the following disease criteria): Indications for recurrent or refractory autoimmune diseases, including but not limited to systemic lupus erythematosus (SLE), etc.

Exclusion Criteria:

  • 1. Study participants who are allergic or hypersensitive to any component of the investigational drug, including those who are allergic to messenger RNA (mRNA) vaccines or other RNA LNP products.
  • 2. Merge any active infections that require antibiotic treatment and have not been controlled for at least one week prior to administration.
  • 3. Accompanied by other uncontrolled malignant tumors.
  • 4. History of cardiovascular disease within the first 6 months of screening: NYHA defined grade III or IV heart failure, myocardial infarction, unstable angina, uncontrolled or symptomatic atrial arrhythmias, any ventricular arrhythmias, or other clinically significant heart diseases.
  • 5. Positive for hepatitis B surface antigen (HBsAg); Or those who are positive for hepatitis B core antibody (HBcAb) and positive for hepatitis B virus (HBV) DNA or have a titer higher than the detection threshold; Individuals with positive hepatitis C virus (HCV) antibodies and HCV RNA positivity or titers above the detection threshold; Human immunodeficiency virus (HIV) antibody positive individuals; Cytomegalovirus (CMV) DNA positive or above the detection limit; Individuals who are positive for syphilis antigen or antibody.
  • 6. Have received attenuated live vaccine or protein subunit vaccine within 30 days before the first study medication.
  • 7. Women who are currently pregnant, breastfeeding, or planning to become pregnant.
  • 8. The researchers believe that there are other situations that are not suitable for participating in this clinical study.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Non-Randomized
  • Interventional Model: Sequential Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: CD19/BCMA in vivo CAR-T, Escalation doses
Drug: CD19/BCMA in vivo CAR-T
CD19/BCMA in vivo CAR-T based on LNP-mRNA
Experimental: CD19/BCMA in vivo CAR-T, Extended doses
Drug: CD19/BCMA in vivo CAR-T
CD19/BCMA in vivo CAR-T based on LNP-mRNA

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Dose limiting toxicity (DLT) and its incidence rate
Time Frame: Within 28 days after the initial treatment
Within 28 days after the initial treatment
Maxmum tolerated dose (MTD) or optimal biological dose (OBD)
Time Frame: Through study completion, an average of 2 years
Through study completion, an average of 2 years

Secondary Outcome Measures

Outcome Measure
Time Frame
Preliminary efficacy evaluation: Objective Response Rate（ORR）
Time Frame: Through study completion, an average of 2 years
Through study completion, an average of 2 years
Preliminary efficacy evaluation: Duration of Response (DoR)
Time Frame: Through study completion, an average of 2 years
Through study completion, an average of 2 years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

West China Hospital

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Estimated)

February 23, 2026

Primary Completion (Estimated)

January 1, 2028

Study Completion (Estimated)

December 31, 2028

Study Registration Dates

First Submitted

January 22, 2026

First Submitted That Met QC Criteria

January 30, 2026

First Posted (Actual)

February 5, 2026

Study Record Updates

Last Update Posted (Actual)

February 5, 2026

Last Update Submitted That Met QC Criteria

January 30, 2026

Last Verified

January 1, 2026

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • CD19/BCMA IIT for ADs

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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