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.

Study Overview

• Status: Recruiting
• Conditions: Advanced Malignant Tumours
• Intervention / Treatment: Genetic: V001-BCMA; Genetic: V001-GPRC5D; Genetic: V001-DLL3; Genetic: V001-FcRH5

Detailed Description

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
• Enrollment (Estimated): 50
• Phase: Early Phase 1

Contacts and Locations

• Study Contact: Name: Li Ning, M.D.; Phone Number: +86 01087788165; Email: lining@cicams.ac.cn
• Study Contact Backup: Name: Shuhang Wang, PhD; Phone Number: +86 01087788165; Email: wangshuhang@cicams.ac.cn

Study Locations

• China
  • Beijing Municipality
    • Beijing, Beijing Municipality, China, 100021
      • Recruiting
      • Cancer Hospital Chinese Academy of Medical Sciences 17 Panjiayuan Nanli, Chaoyang District
      • Contact: Ning Li; Phone Number: +86010-87788165; Email: lining@cicams.ac.cn

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Non-Randomized
• Interventional Model: Parallel Assignment
• Masking: None (Open Label)

Number of Arms

4

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: V001-BCMA; Intravenous administration of V001-BCMA as a single agent for patients with B-cell-related hematologic malignancies. Dose cohorts: 1x10^8 TU、2x10^8 TU、≤4x10^8 TU and ≤8x10^8 TU.	Genetic: V001-BCMA; An in vivo CAR-T drug targeting BCMA administered intravenously
Experimental: V001-GPRC5D; Intravenous administration of V001-GPRC5D as a single agent for patients with B-cell-related hematologic malignancies. Dose cohorts: 1x10^8 TU、2x10^8 TU、≤4x10^8 TU and ≤8x10^8 TU.	Genetic: V001-GPRC5D; An in vivo CAR-T drug targeting GPRC5D administered intravenously
Experimental: V001-DLL3; Intravenous administration of V001-BCMA as a single agent for patients with small cell lung cancer. Dose cohorts: 1x10^8 TU、2x10^8 TU、≤4x10^8 TU and ≤8x10^8 TU.	Genetic: V001-DLL3; An in vivo CAR-T drug targeting DLL3 administered intravenously
Experimental: V001-FcRH5; Intravenous administration of V001-FcRH5 as a single agent for patients with B-cell-related hematologic malignancies. Dose cohorts: 1x10^8 TU、2x10^8 TU、≤4x10^8 TU and ≤8x10^8 TU.	Genetic: V001-FcRH5; An in vivo CAR-T drug targeting FcRH5 administered intravenously

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Incidence 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.	Within 28 days after the first infusion
Maximum Tolerated Dose (MTD)	To determine the MTD of V001 Injection.	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.	From signing ICF until 24 months after the last infusion.

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Objective Response Rate (ORR)	Best overall response rate assessed per indication-specific criteria.	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.	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	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)	Time from infusion to death from any cause	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	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	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	At multiple timepoints post-infusion up to Month 24

Other Outcome Measures

Outcome Measure	Measure Description	Time Frame
Cytokine levels	Pharmacodynamic Biomarkers	At multiple timepoints post-infusion up to Month 24
CRP	Pharmacodynamic Biomarkers	At multiple timepoints post-infusion up to Month 24
Ferritin	Pharmacodynamic Biomarkers	At multiple timepoints post-infusion up to Month 24
Immunogenicity	Incidence of anti-drug antibodies.	Baseline, Day 28, Months 3, 6, 9, 12

Collaborators and Investigators

• Sponsor: Cancer Institute and Hospital, Chinese Academy of Medical Sciences
• Investigators: Study Director: Shuhang Wang, PhD, Clinical Trial Center

Study record dates

Study Major Dates

• Study Start (Actual): November 21, 2025
• Primary Completion (Estimated): December 1, 2027
• Study Completion (Estimated): December 1, 2027

Study Registration Dates

• First Submitted: January 10, 2026
• First Submitted That Met QC Criteria: February 1, 2026
• First Posted (Actual): February 9, 2026

Study Record Updates

• Last Update Posted (Actual): May 13, 2026
• Last Update Submitted That Met QC Criteria: May 9, 2026
• Last Verified: February 1, 2026

More Information

Terms related to this study

• Keywords: BCMA; DLL3; GPRC5D; in vivo CAR-T; FcRH5
• Other Study ID Numbers: NCC5276

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No