Exploratory Study on mRNA Therapy Targeting CD19 for the Treatment of Refractory Autoimmune Diseases

Sponsor
Xinqiao Hospital of Chongqing
Study ID
NCT07362758
Phase
EARLY_PHASE1
Status
Not Yet Recruiting

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Conditions

  • Autoimmune Diseases

Eligibility Criteria

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

Interventions

  • in vivo CAR-T drug based on LNP-mRNA — DRUG
    in vivo CAR-T drug based on LNP-mRNA

Study Details

Autoimmune diseases, such as immune thrombocytopenia (ITP), immune hemolytic anemia (AIHA), systemic lupus erythematosus (SLE), lupus nephritis (LN), idiopathic inflammatory myopathy (IIM), ulcerative colitis (UC), and systemic sclerosis (SSc), are a type of chronic disabling disease characterized by the immune system mistakenly attacking the body itself, leading to tissue damage and organ dysfunction.Autoimmune hematological diseases, especially difficult to treat autoimmune diseases, are a type of disease that is difficult to treat and has a significant impact on patients' lives. Although there are various treatment methods currently available, there are still many limitations to autoimmune sexually transmitted diseases that aim for long-term remission, and further research and breakthroughs are urgently needed. 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. Lipid nanoparticles (LNP) are currently the most mature non viral delivery platform, capable of protecting mRNA from nuclease degradation, promoting intracellular uptake, and achieving efficient translation in vivo. The core of LNP-mRNA technology targeting CD19 is to encapsulate the mRNA encoding specific proteins (such as anti-CD19 related proteins) in lipid nanoparticles and deliver them to the body through intravenous or intramuscular injection.

Key Dates

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

Study Design

Enrollment
47 participants (estimated)
Allocation
NON_RANDOMIZED
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: in vivo CAR-T drug based on LNP-mRNA, Escalation dose
  • Experimental: in vivo CAR-T drug based on LNP-mRNA, Extended dose

Primary Outcome Measure

The incidence of dose limiting toxicity (DLT) [ Time Frame: Within 28 days after the initial treatment ]

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