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

January 21, 2026 updated by: Ruijin Hospital

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

Status

Indicates the current recruitment status or the expanded access status.
Recruiting

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

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

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Estimated)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
27

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Early Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact

The name and contact information for the person who can answer enrollment questions for a clinical study. Each location where the study is being conducted may also have a specific contact, who may be better able to answer those questions.

Study Locations

  • China
    • Huangpu District
      • Shanghai, Huangpu District, China, No. 197, Ruijin 2nd Road
        • Recruiting
        • Ruijin Hospital
        • Contact:

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

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

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

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

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
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

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
Incidence and severity of Dose-Limiting Toxicities (DLTs)
Time Frame: 28 days after RXIM002 first infusion (Day 1)
28 days after RXIM002 first infusion (Day 1)
Incidence and severity of treatment-emergent adverse events (TEAEs)
Time Frame: 52 weeks after RXIM002 first infusion (Day 1)
52 weeks after RXIM002 first infusion (Day 1)
CAR positive cell Cmax
Time Frame: 24 weeks after RXIM002 first infusion (Day 1)
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
Time Frame: 24 weeks after RXIM002 first infusion (Day 1)
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
Time Frame: 24 weeks after RXIM002 first infusion (Day 1)
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

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
LNP (Lipid Nanoparticle) Cmax
Time Frame: 24 weeks after RXIM002 first infusion (Day 1)
LNP in peripheral blood after RXIM002 infusion, maximum concentration (Cmax).
24 weeks after RXIM002 first infusion (Day 1)
LNP (Lipid Nanoparticle) Tmax
Time Frame: 24 weeks after RXIM002 first infusion (Day 1)
LNP levels in peripheral blood after RXIM002 infusion, time to Cmax (Tmax)
24 weeks after RXIM002 first infusion (Day 1)
LNP (Lipid Nanoparticle) AUC
Time Frame: 24 weeks after RXIM002 first infusion (Day 1)
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
Time Frame: 24 weeks after RXIM002 first infusion (Day 1)
CircleRNA in peripheral blood after RXIM002 infusion, maximum concentration (Cmax).
24 weeks after RXIM002 first infusion (Day 1)
CircleRNA Tmax
Time Frame: 24 weeks after RXIM002 first infusion (Day 1)
CircleRNA levels in peripheral blood after RXIM002 infusion, time to Cmax (Tmax).
24 weeks after RXIM002 first infusion (Day 1)
CircleRNA AUC
Time Frame: 24 weeks after RXIM002 first infusion (Day 1)
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)
Time Frame: 52 weeks after RXIM002 first infusion (Day 1)
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)
Time Frame: 52 weeks after RXIM002 first infusion (Day 1)
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
Time Frame: 52 weeks after RXIM002 first infusion (Day 1)
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
Time Frame: 8 weeks after RXIM002 first infusion (Day 1)
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.
Time Frame: 8 weeks after RXIM002 first infusion (Day 1)
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)
Time Frame: 52 weeks after RXIM002 first infusion (Day 1)
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
Time Frame: 52 weeks after RXIM002 first infusion (Day 1)
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

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

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Ruijin Hospital

Collaborators

Collaborators

An organization other than the sponsor that provides support for a clinical study. This support may include activities related to funding, design, implementation, data analysis, or reporting.
RiboX Therapeutics Ltd.

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 (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
December 24, 2025

Primary Completion (Estimated)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
December 19, 2027

Study Completion (Estimated)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
March 19, 2028

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
December 7, 2025

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
December 22, 2025

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
January 7, 2026

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
January 23, 2026

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
January 21, 2026

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
December 1, 2025

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant 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

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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