RN1201 Injection for Autoimmune Diseases Refractory to Standard Therapies

NCT07105735 | Not Yet Recruiting Phase 1

• 1 other identifier: RN1201-AID
• Study Type: interventional
• Target: 18
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Autoimmune Diseases Refractory to Standard Therapies

Keywords

Autoimmune Diseases; Refractory to Standard Therapies

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (3)

• Objective Response Rate (ORR), Disease control rate (DCR)

  Week 4, Month 3, Month 6 and Month 12

• Pharmacokinetic (PK) of RN1201

  up to 12 months

• Pharmacodynamic (PD) of RN1201

  up to 12 months

Study Arms (1)

allogeneic CAR-T cell therapy

EXPERIMENTAL

RN1201 cells injection will be infused only once intravenously at day 0

Biological: allogeneic CAR-T

Interventions

allogeneic CAR-T BIOLOGICAL

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.

allogeneic CAR-T cell therapy

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Voluntary signed informed consent demonstrating understanding of the study and willingness/ability to comply with all trial procedures.
• Age ≥18 years; both sexes eligible.
• 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.
• Standard-of-care therapy for ≥8 weeks before screening without achieving complete remission or adequate disease control, with stable dose for \>2 weeks.
• Subjects on corticosteroid monotherapy at screening must be receiving ≥7.5 mg/day prednisone (or equivalent).
• Disease activity score meeting criteria for moderate-to-severe active disease.
• Adequate bone-marrow reserve, coagulation, cardiopulmonary, hepatic, and renal function.
• Agreement to use effective contraception for 24 months after study enrollment.

You may not qualify if:

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

Sponsors & Collaborators

• The First Affiliated Hospital with Nanjing Medical University: lead
• Rui Therapeutics Co., Ltd: collaborator
• Allorunning Therapeutics: collaborator

Study Sites (1)

The First Affiliated Hospital with Nanjing Medical University

Nanjing, Jiangsu, 210029, China

Location

MeSH Terms

Conditions

Autoimmune Diseases

Condition Hierarchy (Ancestors)

Immune System Diseases

Study Officials

• Lei Fan

  The First Affiliated Hospital with Nanjing Medical University

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Lei Fan

CONTACT

086+025-68306124; fanlei@jsph.org.cn

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Director of lymphoma center

Study Record Dates

• First Submitted: July 24, 2025
• First Posted: August 6, 2025
• Study Start: August 1, 2025
• Primary Completion (Estimated): August 1, 2027
• Study Completion (Estimated): December 1, 2027
• Last Updated: August 6, 2025

Record last verified: 2025-08

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)