In vivo CAR‑T treatment targeting BCMA, GPRC5D, DLL3, and FcRH5 for advanced tumors
A Phase I Study of the In Vivo CAR-T Platform for Treating Advanced Malignant Tumors Based on Target Screening
This trial will try an in-body CAR-T injection to treat adults with relapsed or refractory advanced blood cancers or solid tumors that express BCMA, GPRC5D, DLL3, or FcRH5.
Quick facts
| Phase | Early Phase 1 |
|---|---|
| Study type | Interventional |
| Enrollment | 50 (estimated) |
| Ages | 18 Years and up |
| Sex | All |
| Sponsor | Cancer Institute and Hospital, Chinese Academy of Medical Sciences Academic / other |
| Drugs / interventions | CAR-T |
| Locations | 1 site (Beijing, Beijing Municipality) |
| Trial ID | NCT07395479 on ClinicalTrials.gov |
What this trial studies
This single-arm, open-label, single-center, dose-escalation Phase I platform trial tests V001, an in vivo CAR‑T lentiviral injection, in cohorts defined by tumor type and target antigen. Patients with relapsed or refractory hematologic malignancies or solid tumors whose tumors express the chosen target (BCMA, GPRC5D, DLL3, or FcRH5) are enrolled into separate cohorts. Primary endpoints are safety and tolerability, with secondary and exploratory endpoints including preliminary anti-tumor activity, pharmacokinetics, and pharmacodynamics. The platform design allows multiple V001 constructs to be tested sequentially using dose-escalation to define recommended doses and early signals of activity.
Who should consider this trial
Good fit: Ideal candidates are adults with relapsed or refractory advanced hematologic or solid tumors that express the study target antigen, who meet performance status and organ-function requirements.
Not a fit: Patients whose tumors do not express the selected target, who have uncontrolled infections or active CNS metastases, or who have severe cardiac or pulmonary disease or recent anticancer treatments are unlikely to benefit or be eligible.
Why it matters
Potential benefit: If successful, this approach could provide a more accessible, in-body CAR-T option that produces targeted immune responses against relapsed or refractory tumors.
How similar studies have performed: Ex vivo CAR-T therapies have shown strong success in certain blood cancers, but in vivo CAR-T delivery is a newer, experimental approach with limited early human data.
Eligibility criteria
Show full inclusion / exclusion criteria
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.
Where this trial is running
Beijing, Beijing Municipality
- Cancer Hospital Chinese Academy of Medical Sciences 17 Panjiayuan Nanli, Chaoyang District — Beijing, Beijing Municipality, China (Recruiting)
Study contacts
- Study coordinator: Li Ning, M.D.
- Email: lining@cicams.ac.cn
- Phone: +86 01087788165
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.