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RN1201 Injection for Autoimmune Diseases Refractory to Standard Therapies

ConditionAutoimmune Diseases Refractory to Standard Therapies
Trial statusRecruiting
Trial phasePhase 1
Trial typeInterventional
Biological sexAll
Age18+
SponsorThe First Affiliated Hospital with Nanjing Medical University

About this trial

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.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

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.

Disqualifiers

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).

Trial design

Design model

Single group

Treatments tested in this trial

  • allogeneic CAR-T

    Biological/Vaccine

    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.

Treatment groups

18 Participants
are divided into 1 treatment group
Group A: allogeneic CAR-T cell therapyExperimental treatment 1 intervention

Trial outcomes

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

Time frame
DLTs: Within 28 days after CAR-T cell infusion; TEAEs: From infusion up to 12 months post-treatment.

Secondary outcomes

1

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

The responses will be assessed by predefined disease specific criteria.

Time frame
Week 4, Month 3, Month 6 and Month 12
2

Pharmacokinetic (PK) of RN1201

Levels of CAR-positive T cells in the blood and/or bone marrow

Time frame
up to 12 months
3

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.

Time frame
up to 12 months

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

The First Affiliated Hospital with Nanjing Medical University

Lead sponsor

Source ClinicalTrials.gov
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