RN1201injection for Relapsed/Refractory CD19+/BCMA+ Hematologic Malignancies

Официальное название

An Exploratory Clinical Study on the Safety and Efficacy of Allogeneic CAR-T Cell (RN1201) for Relapsed/Refractory CD19+/BCMA+ Hematologic Malignancies

Обзор

This single-arm, dose-escalation exploratory trial evaluates the safety and efficacy of Allogeneic CAR-T (UCAR-T) cell therapy in patients with relapsed or refractory CD19+/BCMA+ hematologic malignancies, including those with minimal residual disease (MRD). Eligible patients will receive lymphodepletion followed by a single infusion of UCAR-T cells, either post-transplant or without transplantation depending on disease status. The trial assesses overall response and disease control rates, treatment-emergent adverse events, and in vivo behavior of UCAR-T cells.

Подробное описание

This single-arm, dose-escalation exploratory trial evaluates the safety and efficacy of Allogeneic CAR-T (UCAR-T) cell therapy in patients with relapsed or refractory CD19+/BCMA+ hematologic malignancies, including those with minimal residual disease (MRD). Eligible patients will receive lymphodepletion followed by a single infusion of UCAR-T cells, either post-transplant or without transplantation depending on disease status.Primary endpoints include treatment-emergent adverse events (TEAEs) and dose-limiting toxicities (DLTs). Secondary endpoints include objective response rate (ORR), disease control rate (DCR), pharmacokinetics, and pharmacodynamics of UCAR-T. This study aims to provide initial evidence for the safety and anti-tumor activity of UCAR-T in CD19+/BCMA+ hematologic malignancies.

Вмешательства

• Биопрепарат Allogeneic CAR-T
  Patients will receive lymphodepletion chemotherapy followed by a single intravenous infusion of Allogeneic CAR-T cells. In select cases, CAR-T infusion may be administered post-autologous hematopoietic stem cell transplantation (auto-HSCT)

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 (7)

• Objective Response Rate (ORR) [Срок оценки: Week 4, Month 3, Month 6 and Month 12]
• Disease control rate (DCR) [Срок оценки: Week 4, Month 3, Month 6 and Month 12]
• Progression-free survival (PFS) [Срок оценки: Week 4, Month 3, Month 6 and Month 12]
• Overall survival (OS) [Срок оценки: Week 4, Month 3, Month 6 and Month 12]
• Cmax of RN1201 [Срок оценки: Up to 12 months]
• Tmax of RN1201 [Срок оценки: Up to 12 months]
• Cytokines in the peripheral blood after RN1201 infusion [Срок оценки: Up to 12 months]

Критерии участия

Критерии включения

• Voluntary participation with signed informed consent.
• Pathologically confirmed CD19-positive and/or B-cell maturation antigen (BCMA)-positive hematologic malignancy according to the WHO 2017 classification, including but not limited to multiple myeloma, B-cell acute lymphoblastic leukemia (B-ALL), mature B-cell lymphomas, and plasmablastic lymphoma.
• Relapsed/refractory disease defined as failure to achieve complete remission after standard therapy, or relapse after an initial response during treatment or follow-up.
• Measurable disease required:

• For B-ALL: persistent minimal residual disease (MRD) positivity despite hematologic remission.
• For lymphoma: at least one measurable lesion ≥1.5 cm in longest diameter per IWG revised criteria.
• For multiple myeloma: positive immunofixation electrophoresis or presence of extramedullary disease.
• Age ≥18 years; both sexes eligible.
• Expected survival ≥12 weeks.
• Adequate organ function (exceptions for disease-related impairment are at the investigator's discretion):

• Total bilirubin <2× upper limit of normal (ULN); serum creatinine <ULN; ALT and AST <3× ULN.
• Absolute neutrophil count ≥0.5×10⁹/L; platelets ≥20×10⁹/L (no requirement if marrow involvement is documented).
• Eastern Cooperative Oncology Group (ECOG) performance status 0-3.
• Left ventricular ejection fraction (LVEF) ≥50%.

Критерии исключения

• Known hypersensitivity, allergy, intolerance, or contraindication to CD19/BCMA-UCAR-T or any study drugs (fludarabine, cyclophosphamide, tocilizumab).
• Genetic syndromes: Fanconi, Kostmann, Shwachman, or any documented bone-marrow failure syndrome.
• Active or uncontrolled infection requiring IV antibiotics; evidence of severe active infection.
• NYHA Class III or IV heart failure (unless clearly secondary to the underlying malignancy).
• Central Nervous System (CNS) disorders unrelated to the primary hematologic malignancy.
• Prior malignancy except adequately treated carcinoma in situ of skin, cervix, lung, or other non-active tumors.
• Significant bleeding diathesis (e.g., gastrointestinal (GI) bleeding, coagulopathy, hypersplenism).
• History of significant cardiac disease within the past 3 months that, in the investigator's judgment, renders the patient unable to tolerate study participation..
• Pregnancy, lactation, or planned pregnancy within 6 months.
• Any condition that, in the investigator's opinion, may increase 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: NCT07113496 · RN1201-Onc

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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