RN1201 Injection for Autoimmune Diseases Refractory to Standard Therapies

An Exploratory Clinical Study on Allogeneic CAR-T Cell (RN1201) Injection for Autoimmune Diseases Refractory to Standard Therapies

This is a open-label, exploratory trial to evaluate the safety, feasibility, and preliminary efficacy of RN1201, an Allogeneic CAR-T cell therapy, in patients with autoimmune diseases refractory to standard treatment. Eligible patients with moderate to severe activity of diseases will receive a single infusion of RN1201 following lymphodepletion. Primary endpoints include dose-limiting toxicity and treatment-emergent adverse events. Secondary and exploratory endpoints assess clinical response and cell pharmacokinetics.

Study Overview

• Status: Not yet recruiting
• Conditions: Autoimmune Diseases Refractory to Standard Therapies
• Intervention / Treatment: Biological: allogeneic CAR-T

• Study Type: Interventional
• Enrollment (Estimated): 18
• Phase: Phase 1

Contacts and Locations

• Study Contact: Name: Lei Fan; Phone Number: 086+025-68306124; Email: fanlei@jsph.org.cn

Study Locations

• China
  • Jiangsu
    • Nanjing, Jiangsu, China, 210029
      • The First Affiliated Hospital with Nanjing Medical University
      • Principal Investigator: Lei Fan
      • Contact: Lei Fan; Phone Number: 086+025-68306124; Email: fanlei@jsph.org.cn
      • Sub-Investigator: Xiaoyan Qu

Participation Criteria

Eligibility Criteria

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

Description

Inclusion Criteria

1. Voluntary signed informed consent demonstrating understanding of the study and willingness/ability to comply with all trial procedures.
2. Age ≥18 years; both sexes eligible.
3. Documented diagnosis of an autoimmune disease for ≥6 months at screening, including but not limited to immune thrombocytopenia (ITP), autoimmune hemolytic anemia (AIHA), systemic lupus erythematosus (SLE), immune-mediated necrotizing myopathy (IMNM), neuromyelitis optica spectrum disorder (NMOSD), multiple sclerosis (MS), myasthenia gravis (MG), etc.
4. Standard-of-care therapy for ≥8 weeks before screening without achieving complete remission or adequate disease control, with stable dose for >2 weeks.
5. Subjects on corticosteroid monotherapy at screening must be receiving ≥7.5 mg/day prednisone (or equivalent).
6. Disease activity score meeting criteria for moderate-to-severe active disease.
7. Adequate bone-marrow reserve, coagulation, cardiopulmonary, hepatic, and renal function.
8. Agreement to use effective contraception for 24 months after study enrollment.

Exclusion Criteria

Subjects meeting any of the following cannot be enrolled:

1. Known hypersensitivity, allergy, intolerance, or contraindication to RN1201 or any study drug component (fludarabine, cyclophosphamide, tocilizumab) or history of severe allergic reactions.
2. Severe cardiovascular disease or organ failure.
3. Active or uncontrolled infection requiring IV antibiotics or evidence of severe active infection.
4. Significant bleeding tendency (e.g., Gastrointestinal bleeding, coagulopathy, hypersplenism).
5. Hepatitis C virus, HIV, or syphilis infection.
6. History of epilepsy or severe neurological disorders/pathology not attributable to autoimmune disease.
7. Malignancy within 2 years before screening, except adequately treated carcinoma in situ of skin, cervix, or lung or other non-active tumors.
8. Prior CAR-T therapy or other genetically modified T-cell therapy.
9. Prednisone (or equivalent) ≥100 mg/day for ≥14 days within 4 weeks before screening.
10. Pregnancy, lactation, or planned pregnancy within 2 years.
11. Any condition that, in the investigator's judgment, may increase subject risk or interfere with study results.

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: allogeneic CAR-T cell therapy; RN1201 cells injection will be infused only once intravenously at day 0	Biological: allogeneic CAR-T; Fludarabine injection (30 mg/m2，QD×3d) and cyclophosphamide injection (300 mg/m2，QD×3d)will be used to remove the lymphocyte before RN1201 infusion.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
The incidence and severity of treatment-emergent adverse events (TEAEs) and dose-limiting toxicities (DLTs)	TEAEs and DLTs will be graded according to Common Terminology Criteria for Adverse Events (CTCAE) v5.0 and American Society for Transplantation and Cellular Therapy (ASTCT) consensus criteria	DLTs: Within 28 days after CAR-T cell infusion; TEAEs: From infusion up to 12 months post-treatment.

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Objective Response Rate (ORR), Disease control rate (DCR)	The responses will be assessed by predefined disease specific criteria.	Week 4, Month 3, Month 6 and Month 12
Pharmacokinetic (PK) of RN1201	Levels of CAR-positive T cells in the blood and/or bone marrow	up to 12 months
Pharmacodynamic (PD) of RN1201	Levels of B cells and related immune markers (e.g., serum immunoglobulins, autoantibodies) in peripheral blood and/or bone marrow, as indicators of B cell activity and depletion.	up to 12 months

Collaborators and Investigators

• Sponsor: The First Affiliated Hospital with Nanjing Medical University
• Collaborators: Rui Therapeutics Co., Ltd; AlloRunning Therapeutics Co., Ltd.
• Investigators: Principal Investigator: Lei Fan, The First Affiliated Hospital with Nanjing Medical University

Study record dates

Study Major Dates

• Study Start (Estimated): August 1, 2025
• Primary Completion (Estimated): August 1, 2027
• Study Completion (Estimated): December 1, 2027

Study Registration Dates

• First Submitted: July 24, 2025
• First Submitted That Met QC Criteria: August 2, 2025
• First Posted (Actual): August 6, 2025

Study Record Updates

• Last Update Posted (Actual): August 6, 2025
• Last Update Submitted That Met QC Criteria: August 2, 2025
• Last Verified: August 1, 2025

More Information

Terms related to this study

• Keywords: Autoimmune Diseases; Refractory to Standard Therapies
• Additional Relevant MeSH Terms: Immune System Diseases; Autoimmune Diseases
• Other Study ID Numbers: RN1201-AID

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