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.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
50
An in vivo CAR-T drug targeting BCMA administered intravenously
An in vivo CAR-T drug targeting GPRC5D administered intravenously
An in vivo CAR-T drug targeting DLL3 administered intravenously
Cancer Hospital Chinese Academy of Medical Sciences 17 Panjiayuan Nanli, Chaoyang District
Beijing, Beijing Municipality, China
RECRUITINGIncidence of Dose-Limiting Toxicities (DLTs)
Incidence and characteristics of DLTs graded according to NCI CTCAE v5.0. The DLT observation period is 28 days post-infusion.
Time frame: Within 28 days after the first infusion
Maximum Tolerated Dose (MTD)
To determine the MTD of V001 Injection.
Time frame: During the dose-escalation phase (approximately 12 months)
Incidence of Adverse Events (AEs)
Incidence and severity of treatment-emergent adverse events (TEAEs) graded according to NCI CTCAE v5.0.
Time frame: From signing ICF until 24 months after the last infusion.
Objective Response Rate (ORR)
Best overall response rate assessed per indication-specific criteria.
Time frame: At Day 28, Months 2, 3, 6, 9, 12, 18, 24 post-infusion
Duration of Response (DOR)
Time from first achieving response to disease progression or death.
Time frame: 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)
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
Time frame: 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)
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
An in vivo CAR-T drug targeting FcRH5 administered intravenously
Time from infusion to death from any cause
Time frame: 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
Peak concentration of CAR-T cells in peripheral blood
Time frame: At multiple timepoints post-infusion up to Month 24
time to peak of CAR-T cells in peripheral blood
time to peak of CAR-T cells in peripheral blood
Time frame: At multiple timepoints post-infusion up to Month 24
AUC of CAR-T cells in peripheral blood
AUC of CAR-T cells in peripheral blood
Time frame: At multiple timepoints post-infusion up to Month 24