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

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

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.

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.

Inclusion Criteria:

• 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.

Exclusion Criteria:

• 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.