Safety and Efficacy Study of RXIM002 in Severe, Relapsed or Refractory Autoimmune Diseases (RXIM002)

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 Phase 1, open-label study evaluates the safety, tolerability, and preliminary efficacy of RXIM002, a CD19-targeting circular RNA-mediated in-vivo CAR T-cell therapy, in adults with severe, relapsed, or refractory B cell-mediated autoimmune diseases.

Study Overview

• Status: Recruiting
• Conditions: Relapsed/Refractory B Cell-Mediated Autoimmune Diseases
• Intervention / Treatment: Biological: RXIM002 product

Detailed Description

This Phase 1, open-label, single-arm, dose-escalation study is designed to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, immunogenicity, and preliminary efficacy of RXIM002 in adults with severe, relapsed, or refractory B cell-mediated autoimmune diseases. Eligible participants will receive intravenous administration of RXIM002 and will be monitored for adverse events, laboratory parameters, and other safety outcomes. The study will also explore biological activity and potential clinical responses across the enrolled autoimmune conditions. Participants will be followed for a defined period after treatment to assess longer-term safety and durability of any observed effects. The study will include the following sequential phases: screening, treatment, and follow-up.

• Study Type: Interventional
• Enrollment (Estimated): 27
• Phase: Early Phase 1

Contacts and Locations

• Study Contact: Name: Qiongyi Hu; Phone Number: +86 021-50199835; Email: huqiongyi131@163.com

Study Locations

• China
  • Huangpu District
    • Shanghai, Huangpu District, China, No. 197, Ruijin 2nd Road
      • Recruiting
      • Ruijin Hospital
      • Contact: Hu Qiongyi Hu Qiongyi, Doctor; Phone Number: 086+15062338287; Email: huqiongyi131@163.com

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: RXIM002; Each study participant will be given 2 doses of RXIM002 at each dose level.	Biological: RXIM002 product; Prior to infusion of the RXIM002 product, subjects will receive pre-medication if needed

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Incidence and severity of Dose-Limiting Toxicities (DLTs)		28 days after RXIM002 first infusion (Day 1)
Incidence and severity of treatment-emergent adverse events (TEAEs)		52 weeks after RXIM002 first infusion (Day 1)
CAR positive cell Cmax	CAR positive T cells in peripheral blood after RXIM002 infusion, maximum concentration (Cmax).	24 weeks after RXIM002 first infusion (Day 1)
CAR positive cell Tmax	CAR positive T cells levels in peripheral blood after RXIM002 infusion, time to Cmax (Tmax).	24 weeks after RXIM002 first infusion (Day 1)
CAR positive cell AUC	CAR positive T cells levels in peripheral blood after RXIM002 infusion, area under the concentration-time curve (AUC) .	24 weeks after RXIM002 first infusion (Day 1)

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
LNP (Lipid Nanoparticle) Cmax	LNP in peripheral blood after RXIM002 infusion, maximum concentration (Cmax).	24 weeks after RXIM002 first infusion (Day 1)
LNP (Lipid Nanoparticle) Tmax	LNP levels in peripheral blood after RXIM002 infusion, time to Cmax (Tmax)	24 weeks after RXIM002 first infusion (Day 1)
LNP (Lipid Nanoparticle) AUC	LNP levels in peripheral blood after RXIM002 infusion, area under the concentration-time curve (AUC) .	24 weeks after RXIM002 first infusion (Day 1)
CircleRNA Cmax	CircleRNA in peripheral blood after RXIM002 infusion, maximum concentration (Cmax).	24 weeks after RXIM002 first infusion (Day 1)
CircleRNA Tmax	CircleRNA levels in peripheral blood after RXIM002 infusion, time to Cmax (Tmax).	24 weeks after RXIM002 first infusion (Day 1)
CircleRNA AUC	CircleRNA levels in peripheral blood after RXIM002 infusion, area under the concentration-time curve (AUC) .	24 weeks after RXIM002 first infusion (Day 1)
Systemic Lupus Erythematosus (SLE) disease activity: Systemic Lupus Erythematosus Disease Activity Index 2000 (SLEDAI-2K)	Change in Systemic Lupus Erythematosus Disease Activity Index 2000 (SLEDAI-2K) scores from baseline up to 52 weeks, a total score can fall between 0 and 105, with a higher score representing a more significant degree of disease activity.	52 weeks after RXIM002 first infusion (Day 1)
Systemic Sclerosis (SSc) disease activity :Modified rodnan skin score(mRSS)	Change in modified Rodnan skin score (mRSS) from baseline up to 52 weeks in SSc patients. The mRSS is used to assess skin thickness and scope of SSc patients ,with a total score can fall between 0 and 51, with a higher score representing a more significant degree of skin involvement.	52 weeks after RXIM002 first infusion (Day 1)
Idiopathic Inflammatory Myopathy (IIM) disease activity ：Manual muscle test-8	Change in manual muscle testing (MMT-8) score from baseline up to 52 weeks in IIM patients. This tool assesses muscle strength by testing 8 proximal, distal, and axial muscles ,assess unilateral(score range 0-80 points)handedness side, with a lower score representing a more significant degree of muscle involvement.	52 weeks after RXIM002 first infusion (Day 1)
Autoimmune Hemolytic Anemia (AIHA) disease activity: Overall Hb therapeutic response	At least one occurrence of Hb ≥ 100 g/L, concurrently with an increase of at least 20 g/L from baseline, and not attributable to red blood cell transfusion or other emergency therapeutic drugs	8 weeks after RXIM002 first infusion (Day 1)
Immune Thrombocytopenia (ITP) disease activity: Overall response of platelet increment.	The proportion of subjects whose platelet count recovered to ≥ 50 × 10^9/L and who did not require an increase in corticosteroid dose	8 weeks after RXIM002 first infusion (Day 1)
Lupus Nephritis (LN) disease activity: Proportion of subjects achieving Primary Efficacy Renal Response(PERR)	UPCR ≤ 0.7 g/g, with eGFR no more than 20% below the pre-lupus flare level or ≥ 60 mL/min/1.73 m², and no treatment failure event. Achieving Complete Renal Response (CRR)	52 weeks after RXIM002 first infusion (Day 1)
Membranous Nephropathy (MN) disease activity: Clinical remission rate	24-hour urinary protein quantification < 3.5 g/day with serum albumin > 30 g/L, and eGFR ≥ 85% of baseline value	52 weeks after RXIM002 first infusion (Day 1)

Collaborators and Investigators

• Sponsor: Ruijin Hospital
• Collaborators: RiboX Therapeutics Ltd.

Study record dates

Study Major Dates

• Study Start (Actual): December 24, 2025
• Primary Completion (Estimated): December 19, 2027
• Study Completion (Estimated): March 19, 2028

Study Registration Dates

• First Submitted: December 7, 2025
• First Submitted That Met QC Criteria: December 22, 2025
• First Posted (Actual): January 7, 2026

Study Record Updates

• Last Update Posted (Actual): January 23, 2026
• Last Update Submitted That Met QC Criteria: January 21, 2026
• Last Verified: December 1, 2025

More Information

Terms related to this study

• Keywords: Autoimmune Disease
• Additional Relevant MeSH Terms: Pathologic Processes; Disease Attributes; Immune System Diseases; Pathological Conditions, Signs and Symptoms; Recurrence; Autoimmune Diseases
• Other Study ID Numbers: RX-AID-CS101

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No