An Exploratory Clinical Study on the Safety and Efficacy of RN1201 Injection in the Treatment of Antibody-Mediated Diseases
This single-arm, open-label exploratory trial aims to evaluate the safety, feasibility, and preliminary efficacy of RN1201 Injection in patients with antibody-mediated diseases, including refractory immune-mediated platelet transfusion refractoriness (PTR) and relapsed or refractory immune thrombocytopenia (ITP). Patients will receive RN1201 cells infusion following lymphodepletion. The study will assess safety, response rates, B-cell depletion, and immune reconstitution. Exploratory analyses will examine in vivo persistence and activity of RN1201.
‣ Inclusion: - specific for Refractory immune-mediated PTR:
• Aged 16-65 years
• Diagnosed with refractory immune-mediated PTR
• Resistant to at least 3 standard therapies
• Able to understand the study and consent
• Projected survival time exceeding three months
• Left Ventricular Ejection Fraction (LVEF) ≥0.5 (as measured by echocardiogram)
• Creatinine \<1.6 mg/dL
• Aspartate Aminotransferase (AST) \< three times the upper limit of normal
• Total bilirubin \<2.0 mg/dL
⁃ Karnofsky Performance Status (KPS) score ≥60.
‣ Inclusion: -specific for Relapsed or Refractory Immune Thrombocytopenia
• Written informed consent obtained;
• Male or female patients aged 18 years or older on the day of informed consent signing;
• Left Ventricular Ejection Fraction (LVEF) ≥50% with no pericardial effusion;
• As assessed by the investigator, systemic treatment drugs (excluding supportive and symptomatic treatment, and prednisone at a daily dose of ≤10 mg or equivalent) can be discontinued prior to lymphodepletion preconditioning.
• Historically diagnosed with primary immune thrombocytopenia (ITP) (based on the 2019 International Working Group for ITP and the American Society of Hematology (ASH));
• At least two consecutive blood routine examinations showing reduced platelet count, with no significant abnormalities in blood cell morphology on peripheral blood smear microscopy;
• At the screening visit, the subject has no splenomegaly;
• Bone marrow examination: the bone marrow cytology of ITP patients is characterized by increased or normal megakaryocytes with maturation disorders (investigators may assess whether to accept previous bone marrow examination reports, and if previous reports are used, they must be kept as copies in the study documents);
• The subject is a refractory ITP patient who has not responded to first-line treatment drugs, second-line thrombopoiesis-stimulating agents, and rituximab therapy, or who has undergone ineffective splenectomy or postoperative recurrence, and after re-evaluation of the diagnosis, is still confirmed to have ITP;
⁃ Relapsed ITP is defined as a decrease in platelet count to below 30×10⁹/L after an initial response to treatment, or to less than twice the baseline level, or the reappearance of bleeding symptoms.
⁃ The subject has received at least four weeks of the most recent treatment (non-biological background therapy, antimalarial monotherapy, antimalarial combined with oral glucocorticoids (OCS) and/or immunosuppressants, or combined therapy with OCS and/or immunosuppressants).