RN1201 Injection for Autoimmune Diseases Refractory to Standard Therapies

Официальное название

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

Обзор

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.

Вмешательства

• Биопрепарат 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.

Первичные конечные точки

• The incidence and severity of treatment-emergent adverse events (TEAEs) and dose-limiting toxicities (DLTs) [Срок оценки: DLTs: Within 28 days after CAR-T cell infusion; TEAEs: From infusion up to 12 months post-treatment.]

Вторичные конечные точки (3)

• Objective Response Rate (ORR), Disease control rate (DCR) [Срок оценки: Week 4, Month 3, Month 6 and Month 12]
• Pharmacokinetic (PK) of RN1201 [Срок оценки: up to 12 months]
• Pharmacodynamic (PD) of RN1201 [Срок оценки: up to 12 months]

Критерии участия

Критерии включения

• 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.

Критерии исключения

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.

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Не применимо

Модель

Одна группа

Маскирование

Открытое

Основная цель

Лечение

Центры проведения

Китай · 1 центр

• The First Affiliated Hospital with Nanjing Medical University — Нанкин

Идентификаторы

NCT: NCT07105735 · RN1201-AID

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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