A Platform Study of In Vivo CAR-T for Treating Advanced Malignant Tumors Based on Target Screening

Официальное название

A Phase I Study of the In Vivo CAR-T Platform for Treating Advanced Malignant Tumors Based on Target Screening

Обзор

This is a single-arm, open-label, single-center, dose-escalation Phase I platform study designed to evaluate the safety, tolerability, preliminary efficacy, pharmacokinetics, and pharmacodynamics of an in vivo CAR-T therapy (V001 Injection, targeting BCMA, GPRC5D, DLL3,FcRH5, etc.) in patients with advanced malignant tumors.

Подробное описание

This is a single-arm, open-label, single-center, dose-escalation Phase I platform study designed to evaluate the safety, tolerability, preliminary efficacy, pharmacokinetics, and pharmacodynamics of an in vivo CAR-T therapy (V001 Injection, targeting BCMA, GPRC5D, DLL3, etc.) based on a lentiviral vector platform in patients with advanced malignant tumors (including hematological malignancies and solid tumors). The study employs a platform design, enrolling patients into different cohorts based on target and indication.

Вмешательства

• Генная терапия V001-BCMA
  An in vivo CAR-T drug targeting BCMA administered intravenously
• Генная терапия V001-GPRC5D
  An in vivo CAR-T drug targeting GPRC5D administered intravenously
• Генная терапия V001-DLL3
  An in vivo CAR-T drug targeting DLL3 administered intravenously
• Генная терапия V001-FcRH5
  An in vivo CAR-T drug targeting FcRH5 administered intravenously

Первичные конечные точки

• Incidence of Dose-Limiting Toxicities (DLTs) [Срок оценки: Within 28 days after the first infusion]
• Maximum Tolerated Dose (MTD) [Срок оценки: During the dose-escalation phase (approximately 12 months)]
• Incidence of Adverse Events (AEs) [Срок оценки: From signing ICF until 24 months after the last infusion.]

Вторичные конечные точки (7)

• Objective Response Rate (ORR) [Срок оценки: At Day 28, Months 2, 3, 6, 9, 12, 18, 24 post-infusion]
• Duration of Response (DOR) [Срок оценки: From date of the first response until the date of first documented progression or date of death from any cause, whichever came first, assessed up to 24 months]
• Progression-Free Survival (PFS) [Срок оценки: From date of infusion until the date of first documented progression or date of death from any cause, whichever came first, assessed up to 24 months]
• Overall Survival (OS) [Срок оценки: From the date of infusion until the date of death from any cause, assessed up to 24 months]
• Peak concentration of CAR-T cells in peripheral blood [Срок оценки: At multiple timepoints post-infusion up to Month 24]
• time to peak of CAR-T cells in peripheral blood [Срок оценки: At multiple timepoints post-infusion up to Month 24]
• AUC of CAR-T cells in peripheral blood [Срок оценки: At multiple timepoints post-infusion up to Month 24]

Критерии участия

Критерии включения

• Age ≥ 18 years.
• Histologically confirmed advanced hematological malignancies (e.g., multiple myeloma, lymphoma) or solid tumors (e.g., small cell lung cancer) that are relapsed or refractory.
• Tumor cells express the relevant target (e.g., BCMA, GPRC5D, DLL3) as required for the specific cohort.
• ECOG performance status 0-2 (hematological malignancies) or 0-1 (solid tumors) and life expectancy ≥ 3 months.
• Adequate organ function (e.g., creatinine clearance ≥45 mL/min, LVEF ≥45%).
• Patients of childbearing potential must agree to use effective contraception during the study and for 1 year after dosing.
• Signed informed consent form.

Критерии исключения

• Active, uncontrolled infection.
• Active central nervous system metastases or involvement.
• Prior anticancer therapy, radiotherapy, or investigational therapy within specified timeframes before the first study dose.
• Severe cardiac or pulmonary disease (e.g., NYHA Class III/IV heart failure), severe hepatic or renal impairment.
• Active Hepatitis B, Hepatitis C, HIV, or syphilis infection.
• Prior allogeneic hematopoietic stem cell transplantation (within specified window) or active graft-versus-host disease.
• Pregnancy or lactation.
• History of severe allergy to any components of the investigational product.
• Any other condition deemed by the investigator to 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

Нерандомизированное

Модель

Параллельные группы

Маскирование

Открытое

Основная цель

Лечение

Центры проведения

Китай · 1 центр

• Cancer Hospital Chinese Academy of Medical Sciences 17 Panjiayuan Nanli, Chaoyang District — Пекин

Идентификаторы

NCT: NCT07395479 · NCC5276

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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