Autoimmune Diseases Refractory to Standard TherapiesPhase 1NCT07105735
RN1201 Injection for Autoimmune Diseases Refractory to Standard Therapies
A Phase 1 study testing allogeneic CAR-T in people with Autoimmune Diseases Refractory to Standard Therapies, run by The First Affiliated Hospital with Nanjing Medical University at 1 site in China.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Autoimmune Diseases Refractory to Standard Therapies
- What is being tested?
- allogeneic CAR-T
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 18 participants
- Where
- 1 siteChina
- Study dates
- 15 Aug 2025to Dec 2027
- Phase
- Phase 1
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
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- 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.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site, 1 recruiting
- Recruiting
The First Affiliated Hospital with Nanjing Medical University
Nanjing, Jiangsu, 210029
Lei Fan, 086+025-68306124, fanlei@jsph.org.cn
Lei Fan
About this study
Official title: 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.
- Being tested
- allogeneic CAR-T
- Keywords
- Autoimmune Diseases, Refractory to Standard Therapies
- Medical subject headings
- Autoimmune Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
allogeneic CAR-TBiological
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.
| Study group | Role | What they receive |
|---|---|---|
| allogeneic CAR-T cell therapy | Experimental | RN1201 cells injection will be infused only once intravenously at day 0 Receives: Biological: allogeneic CAR-T |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: 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)
The responses will be assessed by predefined disease specific criteria.
Measured: 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
Measured: 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.
Measured: up to 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Aug 2025
- Study start15 Aug 2025
- Primary completionAug 2027 (estimated)
- Study completionDec 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Autoimmune Diseases Refractory to Standard TherapiesStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 18 participants (estimated)
Sponsor and investigators
- Lead sponsor
- The First Affiliated Hospital with Nanjing Medical University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Lei Fan, The First Affiliated Hospital with Nanjing Medical University
- Investigators
- Lei Fan, Principal investigator, The First Affiliated Hospital with Nanjing Medical University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07105735
- Sponsor's protocol ID
- RN1201-AID
- Status last verified
- Aug 2025
- First submitted
- 24 Jul 2025
- Last updated
- 1 Sep 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No