In Vivo CAR-T (V001 Injection) Platform Study for Advanced Cancers
A first-in-human Phase 1 platform study at the Cancer Hospital, Chinese Academy of Medical Sciences in Beijing, evaluating an in vivo CAR-T therapy (V001 Injection) across BCMA, GPRC5D, DLL3 and FcRH5 cohorts for adults with relapsed or refractory advanced malignancies. CancerFax can help international patients understand whether this trial may be relevant and coordinate with the CAMS team.
About This Clinical Trial
Patients with advanced multiple myeloma, certain lymphomas and small cell lung cancer often run out of effective standard options after relapse or refractoriness. CAR-T cell therapy has reshaped outcomes for some of these patients, but conventional CAR-T requires apheresis, weeks of manufacturing in a specialised facility, lymphodepleting chemotherapy and inpatient monitoring. This is logistically demanding, expensive and not always feasible for patients whose disease is moving quickly or who live far from a cell therapy centre.
This trial, NCT07395479, studies V001 Injection, an investigational in vivo CAR-T therapy delivered as an injection. Instead of collecting T cells, engineering them in a lab and giving them back, the V001 platform uses a lentiviral vector designed to engineer CAR-T cells directly inside the body. It is a Phase 1, single-arm, open-label, single-centre, dose-escalation platform study at the Cancer Hospital, Chinese Academy of Medical Sciences in Beijing. The platform design enrolls separate cohorts based on the tumour target: BCMA for multiple myeloma and B-cell malignancies, GPRC5D for myeloma, DLL3 for small cell lung cancer, and FcRH5 for B-cell lymphoid cancers.
If in vivo CAR-T can be made to work safely, it could simplify access to CAR-T therapy across more hospitals, shorten the time from decision to treatment, and remove the apheresis and manufacturing steps that delay or block many patients today. This study is one of the first formally registered in vivo CAR-T platform trials in China, and is being run by a leading national cancer centre with significant CAR-T and cellular therapy experience.
Primary endpoints focus on safety: incidence of dose-limiting toxicities within 28 days, the maximum tolerated dose, and overall adverse event profile. Secondary endpoints look at preliminary anti-tumour activity (objective response rate, duration of response, progression-free survival, overall survival) and at how the engineered CAR-T cells behave in the body (pharmacokinetics in peripheral blood).
Whether V001 Injection can safely generate functional CAR-T cells inside the body across multiple tumour targets, what dose level is tolerated, and whether early signals of anti-tumour activity are seen in advanced relapsed or refractory cancers.
Trial at a Glance
A quick summary of the V001 in vivo CAR-T trial. This is for orientation only and is not a confirmation of eligibility.
Final eligibility is determined only by the trial investigators after reviewing complete medical records.
Treatment Being Studied
V001 Injection is an investigational in vivo CAR-T therapy. Rather than collecting a patient's T cells, engineering them outside the body and infusing them back, V001 uses a lentiviral vector platform designed to engineer CAR-T cells directly inside the patient.
The trial uses a platform design — different cohorts of patients receive a version of V001 directed at a different tumour target, depending on what their cancer expresses. Targets currently studied include BCMA (multiple myeloma and B-cell malignancies), GPRC5D (multiple myeloma), DLL3 (small cell lung cancer) and FcRH5 (B-cell lymphoid cancers).
How in vivo CAR-T therapy works (in simple terms)
In conventional CAR-T, doctors remove T cells from the patient, send them to a lab, genetically modify them to attack a specific tumour protein, grow large numbers of these cells and infuse them back. The in vivo approach studied in this trial aims to skip the lab step. A specially designed lentiviral vector is given as an injection. The vector is engineered to find T cells inside the body and insert the CAR (chimeric antigen receptor) genetic instructions directly into them. The patient's own T cells then become CAR-T cells in the body, recognise tumour cells carrying the chosen target and attack them. Because this is the first study of V001 in humans, the trial is starting with very careful dose escalation and intensive safety monitoring.
Full diagnostic review, performance status assessment, organ function tests, and confirmation that the tumour expresses the target relevant to the cohort (BCMA, GPRC5D, DLL3 or FcRH5).
Imaging (PET-CT, CT or MRI as appropriate), bone marrow studies for myeloma or lymphoma, infection screening, cardiac and pulmonary assessment to confirm fitness for cell therapy.
The trial team explains the protocol, known risks (including CRS and neurological toxicity seen with CAR-T therapies), unknown long-term risks of in vivo CAR-T, alternatives, and the right to withdraw.
Patients are enrolled at a specific dose level of V001 Injection based on where the dose-escalation cohort currently is. Cohorts move up only after safety is confirmed.
V001 is administered as an injection per protocol. The patient remains in hospital for close monitoring during the dose-limiting toxicity (DLT) window of 28 days.
Vital signs, cytokine levels, CRP, ferritin, peripheral blood CAR-T cell levels, immunogenicity and response assessments are tracked at Day 28, Months 2, 3, 6, 9, 12, 18 and 24 post-infusion.
V001 Injection has not been approved by any regulatory authority. In vivo CAR-T is a new approach and long-term safety is not yet known. Patients should only consider this trial under expert oncology guidance.
Who This Trial May Be For
This trial is being studied in adults with advanced cancers that have come back or stopped responding to standard treatment, where the tumour expresses one of the targets the platform is built against. Final fit can only be confirmed by the trial team after detailed review.
Histologically confirmed advanced hematologic malignancy (such as multiple myeloma or lymphoma) or solid tumour (such as small cell lung cancer) that has relapsed after, or is refractory to, standard therapy.
Tumour cells must express the relevant target for the cohort — BCMA, GPRC5D, DLL3 or FcRH5. Recent biomarker or flow cytometry reports are usually required.
Open to patients aged 18 years and above. No upper age limit is specified in the registry, but overall fitness for cell therapy must be confirmed.
ECOG performance status 0 to 2 for hematologic malignancies, or 0 to 1 for solid tumours. Life expectancy of at least 3 months.
Reasonable kidney function (creatinine clearance ≥45 mL/min), cardiac function (LVEF ≥45%) and no severe hepatic or pulmonary disease. Active infections, HBV, HCV, HIV or syphilis exclude participation.
Patients must be able to travel to the CAMS Cancer Hospital in Beijing, complete inpatient monitoring around the infusion period and return for protocol-mandated follow-up visits.
Eligibility Criteria
Eligibility is determined by the trial investigators after detailed review. Meeting some of the criteria below does not guarantee enrollment, and the trial protocol may include additional details not captured in the public registry.
check_circleInclusion Criteria — May Be Eligible
- ✓Age ≥ 18 years
- ✓Histologically confirmed advanced hematologic malignancies (such as multiple myeloma or lymphoma) or solid tumours (such as small cell lung cancer) that are relapsed or refractory
- ✓Tumour cells express the relevant target (BCMA, GPRC5D, DLL3 or FcRH5) as required for the specific cohort
- ✓ECOG performance status 0–2 for hematologic malignancies, or 0–1 for solid tumours, and life expectancy ≥ 3 months
- ✓Adequate organ function, including creatinine clearance ≥45 mL/min and 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
cancelExclusion Criteria — May Not Be Eligible
- ×Active, uncontrolled infection
- ×Active central nervous system metastases or CNS involvement
- ×Prior anticancer therapy, radiotherapy or investigational therapy within the protocol-specified washout window before the first study dose
- ×Severe cardiac or pulmonary disease (such as NYHA Class III or IV heart failure) or severe hepatic or renal impairment
- ×Active Hepatitis B, Hepatitis C, HIV or syphilis infection
- ×Prior allogeneic hematopoietic stem cell transplantation within the protocol-specified window, or active graft-versus-host disease
- ×Pregnancy or lactation
- ×History of severe allergy to any component of the investigational product
- ×Any other condition deemed by the investigator to increase risk or interfere with study results
Criteria here are illustrative. The trial protocol has its own detailed list. CancerFax can help organize records for review, but only the trial center can confirm participation.
Medical Records and Tests Needed for Review
A complete and well-organised set of medical records makes the trial team's review faster and more accurate. CancerFax can help compile, translate and structure these documents on the patient's behalf.
How the Trial Process May Work
In vivo CAR-T trials involve careful screening, intensive in-hospital monitoring after the V001 injection, and structured long-term follow-up. The steps below summarise the typical patient journey for this protocol.
Medical records are reviewed remotely to assess whether the diagnosis, target expression and prior treatment history may broadly align with one of the V001 cohorts. CancerFax can help compile and translate this dossier.
The CAMS trial team or its representatives provide an initial view on whether the case is worth a formal screening visit, and which cohort may be most relevant.
On-site review at the Cancer Hospital, Chinese Academy of Medical Sciences. Confirmatory tests may include repeat imaging, biomarker reconfirmation, organ function tests, infection screening and cardiac/pulmonary workup.
The investigators explain the study in detail, including known and unknown risks of in vivo CAR-T. Patients who consent are assigned to a cohort and dose level per the platform design.
V001 Injection is administered per protocol. Patients are monitored as inpatients during the 28-day dose-limiting toxicity window for cytokine release syndrome, neurological events, infection and other adverse events.
Response assessments and pharmacokinetic / pharmacodynamic studies at Day 28, Months 2, 3, 6, 9, 12, 18 and 24 post-infusion. Adverse event monitoring continues for up to 24 months after the last infusion.
Potential Benefits
For carefully selected patients whose disease has limited remaining standard options, participation in a CAR-T platform trial may offer specific benefits, alongside meaningful uncertainties. Realistic expectations are essential.
V001 is one of the first registered in vivo CAR-T programmes in China. For patients who cannot easily access conventional CAR-T due to logistics, manufacturing time or cost, this represents a different pathway, although still investigational.
The trial is run at the Cancer Hospital of the Chinese Academy of Medical Sciences in Beijing, a leading national cancer institution with significant experience in advanced therapies, hematologic malignancies and complex solid tumours.
Cohorts targeting BCMA, GPRC5D, DLL3 and FcRH5 mean that several different patient groups (multiple myeloma, certain B-cell lymphomas, small cell lung cancer) can be considered under one structured study.
Phase 1 cell therapy trials include very close clinical and laboratory monitoring, especially during the 28-day DLT window. This level of observation is typically not available outside of trial settings.
Patients are not enrolled into a generic protocol but into a cohort defined by what their tumour actually expresses. This is consistent with modern precision oncology principles.
Data from this trial will help define whether in vivo CAR-T can become a viable, scalable approach. Patients who participate contribute to that knowledge regardless of individual response.
This is a first-in-human Phase 1 study. The primary aim is to learn about safety and the right dose. Anti-tumour response is a secondary endpoint, and individual benefit cannot be promised.
Risks and Side Effects
All CAR-T therapies, including in vivo approaches like V001, carry recognised and potential risks that need to be weighed carefully with the treating oncologist and the trial team.
Fever, low blood pressure, low oxygen and other inflammatory signs caused by CAR-T cell activation against the tumour. CRS is well-described in CAR-T therapy and can range from mild to life-threatening; supportive treatments such as tocilizumab and intensive care are sometimes needed.
Confusion, tremor, language difficulty, seizures or other neurological symptoms can occur after CAR-T cell activity. These are monitored closely during the inpatient observation period.
Drops in white cells, red cells and platelets can occur, raising the risk of serious infections and bleeding. Frequent blood tests and supportive care are part of the protocol.
CAR-T cells can attack normal cells that share the target (for example, healthy plasma cells in BCMA-targeted therapy). This can cause specific organ or tissue toxicity depending on the cohort.
Unlike conventional ex vivo CAR-T, in vivo CAR-T using lentiviral delivery is in very early human study. Long-term effects on the immune system, insertional events and other unknown risks cannot be ruled out and require lifelong follow-up.
International patients must factor in travel to Beijing, prolonged stay during inpatient monitoring, costs not covered by the trial, and the possibility that screening will not lead to enrollment or that the cancer may progress during the process.
Time, travel and cost burden, no guaranteed benefit, possible disease progression during screening, and possible early trial discontinuation — these factors should be weighed carefully with the family and treating team.
Trial Location and Hospital Information
The trial is currently being conducted at a single centre — the Cancer Hospital, Chinese Academy of Medical Sciences, 17 Panjiayuan Nanli, Chaoyang District, Beijing, China. Patients accepted into the study will need to travel to Beijing for screening, treatment and protocol-mandated follow-up.
International patients typically need a medical visa for China, fully translated medical records (Chinese and English), remote case review before travel, accommodation arrangements in Beijing during the inpatient monitoring period, and ongoing coordination with the CAMS trial team. CancerFax can help compile and translate records, coordinate remote review, support visa documentation requests and assist with logistics in Beijing.
Costs, Trial Coverage, and Patient Expenses
Costs in cell therapy trials vary by sponsor, country and whether the patient is local or international. The table below describes typical coverage patterns for early-phase cell therapy studies and should be confirmed in writing by the trial centre before any travel commitment.
CancerFax helps understand expected cost categories and what is covered. Final confirmation must come from the CAMS trial centre in writing. ⚠VERIFY: exact coverage scope for V001 — not specified in public registry.
Standard Treatment vs Clinical Trial
For patients with advanced relapsed or refractory disease, the choice between standard treatment and a clinical trial like this in vivo CAR-T study is significant. The table below highlights how the two approaches typically differ.
How CancerFax Helps Patients Explore This Trial
CancerFax is a specialist cancer patient-navigation and advanced cancer treatment access platform. For complex options like in vivo CAR-T at a national cancer centre in Beijing, we help patients and families navigate the gap between a confusing medical situation and a structured, expert-reviewed pathway.
We compile and structure pathology, biomarker reports, imaging, treatment history and discharge summaries so that the CAMS team can review the case quickly and accurately.
We help map the case against the V001 cohorts and trial criteria, and highlight which target (BCMA, GPRC5D, DLL3, FcRH5) may be most relevant for further review by the trial team.
We coordinate communication with the trial site at the Cancer Hospital, Chinese Academy of Medical Sciences, share documents in appropriate formats and relay clarifications back to the patient and family.
We assist with documentation needed for medical visa applications to China, planning the trip to Beijing, accommodation near the hospital and logistics during the inpatient monitoring period.
If V001 is not the right fit, we help explore other CAR-T, CAR-NK, TIL, bispecific antibody or precision oncology options in China, India and other oncology centres.
From the first medical summary to follow-up after treatment, CancerFax provides a single point of contact for the family, the local treating oncologist and the international trial team.
CancerFax does not guarantee trial enrollment, treatment response, or outcome. Our role is to help patients access accurate information and appropriate pathways.
Questions to Ask Before Considering This Trial
These questions are designed to help patients and families have a clear, informed conversation with their oncologist and with the trial team before deciding to pursue the V001 trial.
Frequently Asked Questions
Want to Know Whether This Trial May Be Relevant?
If you or a family member has advanced multiple myeloma, lymphoma, small cell lung cancer or another advanced cancer that has relapsed or stopped responding to standard treatment, CancerFax can review the medical records and help understand whether the V001 in vivo CAR-T trial at CAMS Beijing is worth exploring further.
The information on this page is for educational and patient-navigation purposes only. It does not replace medical advice, diagnosis, or treatment from a qualified physician. Clinical trial eligibility, enrollment, treatment decisions, and costs are determined only by the trial investigators, hospital, sponsor, and applicable regulations. CancerFax helps patients and families understand options and coordinate case review where appropriate, but does not guarantee trial acceptance, treatment response, or clinical outcome. All clinical decisions must be made in consultation with a qualified, licensed physician with access to the patient's complete medical information.
Explore Related CancerFax Pages
© CancerFax · Specialist cancer access and patient-navigation platform. CancerFax is not a medical institution, hospital, or clinical trial sponsor. Trial details may change; always confirm current eligibility, status, and costs directly with the trial center.