RXIM002 for severe, relapsed or refractory B cell–mediated autoimmune diseases

A Clinical Study to Evaluate the Safety, Tolerability and Preliminary Efficacy of the CD19-Targeting Circular RNA Product RXIM002 in Patients With Relapsed or Refractory B Cell-Mediated Autoimmune Diseases

This trial tests RXIM002, a one-time IV in‑body CD19‑targeting CAR T therapy delivered by circular RNA, in adults 18–65 with severe, relapsed or refractory B cell–mediated autoimmune diseases to see if it is safe and can reduce disease activity.

Quick facts

• Phase: Early Phase 1
• Study type: Interventional
• Enrollment: 27 (estimated)
• Ages: 18 Years to 65 Years
• Sex: All
• Sponsor: Ruijin Hospital (Academic / other)
• Drugs / interventions: CAR T
• Locations: 1 site (Shanghai, Huangpu District)
• Trial ID: NCT07322718 on ClinicalTrials.gov

What this trial studies

This Phase 1, open‑label, single‑arm, dose‑escalation study gives adults with severe, relapsed or refractory B cell–mediated autoimmune diseases intravenous RXIM002 and follows them for safety and biological response. Participants will be monitored for adverse events, laboratory measures, pharmacokinetics, pharmacodynamics, and immunogenicity, and investigators will look for preliminary signs of clinical improvement. The protocol includes screening, treatment, and follow‑up periods to capture both short‑term safety and longer‑term durability of any effects. Dosing is escalated in sequential cohorts to determine tolerability and an appropriate dose range.

Who should consider this trial

Good fit: Adults aged 18–65 with severe, relapsed or refractory CD19‑positive B cell–mediated autoimmune diseases who meet organ‑function criteria and can attend the study site are the intended candidates.

Not a fit: Patients whose disease is not driven by CD19‑positive B cells, those with significant organ dysfunction or uncontrolled comorbidities, or those unable to travel to the site are unlikely to benefit from this intervention.

Why it matters

Potential benefit: If successful, RXIM002 could deplete pathogenic B cells and produce durable remission, reducing the need for ongoing immunosuppressive therapy.

How similar studies have performed: Ex‑vivo CD19 CAR T therapies have shown promise in some autoimmune cases, but an in‑vivo circular RNA‑mediated CAR T approach like RXIM002 is novel and largely untested in humans.

Eligibility criteria

Inclusion Criteria:

* Subjects voluntary agreement to provide written informed consent.
* Aged 18 to 65 years, either sex.
* Adequate organ function meeting screening criteria.
* Positive test for cluster of differentiation antigen 19 (CD19).

Systemic Lupus Erythematosus (SLE) and Lupus Nephritis (LN):

* Have been diagnosed with SLE or LN before screening.
* Presence of anti-dsDNA, anti-histone, anti-chromatin, anti-Ro (anti-SS-A), anti-La (anti-SS-B), antinuclear antibody (ANA), or anti-Sm antibodies at screening.
* Active disease at screening.
* Fulfill relapsed/refractory SLE or LN conditions

Lupus Nephritis (LN) :

* Kidney biopsy result indicating LN
* Evidence of LN disease activity

Systemic Sclerosis (SSc):

* Have been diagnosed with SSc before screening.
* Antinuclear Antibody (ANA) positive at screening or prior to screening. AND, evidence of SSc disease activity.
* Fulfill relapsed/refractory SSc conditions.

Immune Thrombocytopenia (ITP):

* Have been diagnosed with refractory ITP before screening.
* Platelet count \<50×10⁹/L at screening.

Idiopathic Inflammatory Myopathy (IIM):

* Have been diagnosed with IIM before screening.
* Presence of at least 1 myositis specific (MSA), associated antibody (MAA), or ANA at screening or prior to screening.
* Evidence of IIM disease activity.
* Fulfill relapsed/refractory IIM conditions.

Membranous Nephropathy (MN):

* Have been diagnosed with MN before screening.
* Active MN patients meeting screening criteria.
* Fulfill relapsed/refractory MN conditions.

Autoimmune Hemolytic Anemia (AIHA):

* Have been diagnosed with AIHA before screening.
* Active AIHA patients meeting screening criteria.
* Fulfill relapsed/refractory AIHA conditions.

Exclusion Criteria:

* Active infections such as hepatitis and tuberculosis.
* Other autoimmune diseases.
* Serious underlying diseases such as active malignancies, uncontrolled diabetes.
* Female subjects who were pregnant, breastfeeding.
* Any uncontrolled psychiatric disorders (e.g., schizophrenia, bipolar disorder, eating disorders, major depression or anxiety disorder), as declared by the participant or reported in the medical records.

Where this trial is running

Shanghai, Huangpu District

• Ruijin Hospital — Shanghai, Huangpu District, China (Recruiting)

Study contacts

• Study coordinator: Qiongyi Hu

Conditions Relapsed/Refractory B Cell-Mediated Autoimmune Diseases; Autoimmune Disease