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

NCT07395479 | Recruiting Early Phase 1

• 1 other identifier: NCC5276
• Study Type: interventional
• Target: 50
• Locations: 1 country
• Sites: 1

Brief Summary

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.) in patients with advanced malignant tumors.

Conditions

Advanced Malignant Tumours

Keywords

in vivo CAR-T; DLL3; GPRC5D; BCMA

Outcome Measures

Primary Outcomes (3)

• 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 Outcomes (7)

• Objective Response Rate (ORR)

  At Day 28, Months 2, 3, 6, 9, 12, 18, 24 post-infusion

• Duration of Response (DOR)

  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

• Overall Survival (OS)

  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

  At multiple timepoints post-infusion up to Month 24

Other Outcomes (4)

• Cytokine levels

  At multiple timepoints post-infusion up to Month 24

• CRP

  At multiple timepoints post-infusion up to Month 24

• Ferritin

  At multiple timepoints post-infusion up to Month 24

Study Arms (3)

V001-BCMA

EXPERIMENTAL

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

V001-GPRC5D

EXPERIMENTAL

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

V001-DLL3

EXPERIMENTAL

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

Interventions

V001-BCMA GENETIC

An in vivo CAR-T drug targeting BCMA administered intravenously

V001-BCMA

V001-GPRC5D GENETIC

An in vivo CAR-T drug targeting GPRC5D administered intravenously

V001-GPRC5D

V001-DLL3 GENETIC

An in vivo CAR-T drug targeting DLL3 administered intravenously

V001-DLL3

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Cancer Institute and Hospital, Chinese Academy of Medical Sciences: lead

Study Sites (1)

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

Beijing, Beijing Municipality, 100021, China

RECRUITING

Study Officials

• Shuhang Wang, PhD

  Clinical Trial Center

  STUDY DIRECTOR

Central Study Contacts

Li Ning, M.D.

CONTACT

+86 01087788165; lining@cicams.ac.cn

Shuhang Wang, PhD

CONTACT

+86 01087788165; wangshuhang@cicams.ac.cn

Study Design

• Study Type: interventional
• Phase: early phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 10, 2026
• First Posted: February 9, 2026
• Study Start: November 21, 2025
• Primary Completion (Estimated): December 1, 2027
• Study Completion (Estimated): December 1, 2027
• Last Updated: February 9, 2026

Record last verified: 2026-02

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)