Exploratory Study on mRNA Therapeutic Drug Targeting CD19 for the Treatment of Hematologic Malignancies

Обзор

Malignant hematological tumors mainly derived from adult B cells are mainly acute lymphoblastic leukemia (ALL) and non Hodgkin lymphoma (NHL). Overall, although existing therapies have significantly improved the survival rates of most patients, the treatment of relapsed/refractory patients still faces significant challenges. CD19 is one of the most clinically valuable targets for B-cell malignant hematological tumors. The advent of COVID-19 vaccine has brought LNP mRNA technology into the public's view. After years of development, it not only shines brilliantly in COVID-19 vaccine, but also is widely used in the treatment and exploration of cancer, rare diseases and other fields. Lipid nanoparticles (LNP) are currently the most mature non viral delivery platform, capable of protecting mRNA from nuclease degradation, promoting intracellular uptake, and achieving efficient translation in vivo. The core of LNP-mRNA technology targeting CD19 is to encapsulate the mRNA encoding specific proteins (such as anti-CD19 related proteins) in lipid nanoparticles and deliver them to the body through intravenous or intramuscular injection.

Вмешательства

• Препарат in vivo CAR-T drug based on LNP-mRNA
  in vivo CAR-T drug based on LNP-mRNA

Первичные конечные точки

• Dose limiting toxicity (DLT) and its incidence rate [Срок оценки: Within 28 days after the initial treatment]
• Maximum tolerated dose (MTD) or optimal biological dose (OBD) [Срок оценки: 3 months]

Вторичные конечные точки (3)

• Objective response rate (ORR) [Срок оценки: 1 month]
• Disease control rate (DCR) [Срок оценки: Through study completion, an average of 2 years]
• Progression free survival (PFS) [Срок оценки: From date of initial treatment until the date of first comfired progression or date of death from any cause, whichever came first, assessed up to 24 months]

Критерии участия

Критерии включения

• 1\. Age range of 18-70 years old, gender not limited;
• 2\. Expected survival time exceeds 12 weeks;
• 3\. B-cell lymphoma or lymphocytic leukemia diagnosed with CD19+, with no standard treatment options recommended according to guidelines
• 4\. There are assessable lesions (applicable only to lymphoma patients);
• 5\. The physical fitness status score of the Eastern Cancer Collaboration Group (ECOG) is 0 or 1;
• May involve other inclusion criteria

Критерии исключения

• 1\. Accompanied by other uncontrolled malignant tumors;
• 2\. Previously received chimeric antigen receptor therapy or other transgenic T cell therapy;
• 3\. Known history of HIV or hepatitis B (HBsAg positive and HBV DNA reaching the detection limit) or hepatitis C virus (anti HCV positive) infection;
• 4\. Participants with a history of CNS lymphoma, malignant cells in cerebrospinal fluid, or brain metastases;
• 5\. The researcher believes that there are any other factors that are not suitable for the study participants to enter this trial.

May involve other exclusion criteria

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

• Department of Hematology, Xinqiao Hospital — Чунцин

Идентификаторы

NCT: NCT07349849 · in vivo CAR-T of CD19

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

Открыть это исследование на ClinicalTrials.gov ↗