A Dose-escalation Phase I Trial of PD-1 Knockout Engineered T Cells for the Treatment of Muscle-invasive Bladder Cancer
PD-1 Knockout T Cells Trial for Advanced Bladder Cancer (Withdrawn)
Plain English Summary
PD-1 Knockout Engineered T Cells for Muscle-invasive Bladder Cancer is a Phase 1 clinical trial sponsored by Peking University studying Invasive Bladder Cancer Stage IV. This trial tested a new type of cell therapy called PD-1 knockout engineered T cells. It was for patients with advanced bladder cancer that had spread and didn't respond to standard treatments. Participation involved receiving the engineered T cells, along with other medications, and regular check-ups. This trial is no longer active, so alternative treatments would need to be discussed with a doctor.
Official Summary
This study will evaluate the safety of PD-1 knockout engineered T cells in treating metastatic advanced bladder cancer. Blood samples will also be collected for research purposes.
Who Can Participate
Here is what you need to know about eligibility for this trial. Patients with stage IV muscle-invasive bladder cancer that has spread and has measurable tumors. Individuals whose cancer has not responded to all previous standard treatments. Patients must have a good performance score (0-1) and a life expectancy of at least 6 months. Those with certain other medical conditions, active infections, or who cannot comply with the study requirements were excluded. This trial is studying Invasive Bladder Cancer Stage IV, so participants generally need a confirmed diagnosis.
What They're Measuring
The primary outcome measured the safety of the PD-1 knockout T cells by tracking any side effects or dose-limiting toxicities, helping to understand how well patients tolerated the treatment. The specific primary outcome measures are: Number of participants with Adverse Events and/or Dose Limiting Toxicities as a Measure of Safety and tolerability of dose of PD-1 Knockout T cells using Common Terminology Criteria for Adverse Events (CTCAE v4.0) in patients (Dose Escalation - Approximately 6 months). These endpoints are how researchers determine whether the treatment is effective and will form the basis of any future regulatory submissions.
About This Phase
This trial is in Phase 1, the first major stage of clinical testing. Phase 1 trials typically involve 20-100 participants and focus on safety, dosage levels, and side effects. The primary goal is not to test whether the treatment works but to establish that it is safe enough for further testing. About 70% of Phase 1 drugs advance to Phase 2. If successful, the treatment will proceed to Phase 2 efficacy testing.
Why This Trial Matters
This trial aimed to explore a novel cell therapy approach for a difficult-to-treat form of bladder cancer, addressing a gap in treatment options for patients with advanced disease. This research targets Invasive Bladder Cancer Stage IV, where improved treatment options are needed.
Investor Insight
While this specific trial was withdrawn, the development of engineered T cell therapies for solid tumors represents a significant area of investment and research in oncology, with potential for future Phase 1 trials have approximately a 10% chance of eventually gaining FDA approval.
Is This Trial Right for Me?
Ask your doctor about the specific risks and benefits of this type of cell therapy. Understand what the treatment schedule will be, including hospital visits and any required medications. Be prepared for regular blood tests and imaging scans to monitor your response and any side effects. The trial is being conducted at 1 site. Always discuss clinical trial participation with your healthcare provider before making any decisions. This information is for educational purposes only and is not medical advice.
AI-generated analysis for educational purposes only. This is not medical advice. Discuss clinical trial participation with your doctor. Data sourced from ClinicalTrials.gov.
Study Design
- Study Type: INTERVENTIONAL
- Allocation: NON_RANDOMIZED
- Model: PARALLEL
- Masking: NONE
Interventions
- BIOLOGICAL: PD-1 Knockout T Cells — PD-1 Knockout T Cells and PD-1 wild-type T Cells will be made by Cell Biotech Co., Ltd. 2x107/kg T cells will be used for test group and comparable group separately.
- DRUG: Cyclophosphamide — Cyclophosphamide at 20mg/kg single dose will be administered 3 days i.v. before cell infusion. Interleukin-2 (IL-2) will be given in the following 5 days, 720000 international unit（IU）/Kg/day (if tolerant).
- DRUG: IL-2 — Interleukin-2 (IL-2) will be given in the following 5 days after cell infusion, 720000 international unit（IU）/Kg/day (if tolerant).
Primary Outcomes
- Number of participants with Adverse Events and/or Dose Limiting Toxicities as a Measure of Safety and tolerability of dose of PD-1 Knockout T cells using Common Terminology Criteria for Adverse Events (CTCAE v4.0) in patients (Dose Escalation - Approximately 6 months)
Secondary Outcomes
- Response Rate:Response will be evaluated according to RECIST v1.1 (90 days)
- Progression free survival - PFS (From date of randomization until the date of first documented progression or date of death from any cause, whichever came first, assessed up to average 10 months)
- Overall Survival - OS (The time from randomization to death from any cause, assessed up to 2 years)
- Peripheral blood circulating tumor DNA (6 weeks)
- Temporal Interleukin-2 change in the peripheral blood (Baseline and 1 month and 3 months)
Full Eligibility Criteria
Inclusion Criteria: * Pathologically verified stage IV muscle-invasive bladder cancer with measurable lesions (On CT: longest diameter of tumoral lesion \>=10 mm, shorted diameter of lymph node \>=15 mm; measurable lesions should not have been irradiated) * Progressed after all standard treatment * Performance score: 0-1 * Expected life span: \>= 6 months * Toxicities from prior treatment has resolved. Washout period is 4 weeks for chemotherapy, and 2 weeks for targeted therapy * Major organs function normally * Women at pregnant ages should be under contraception * Willing and able to provide informed consent Exclusion Criteria: * Pathology is mixed type * Emergent treatment of tumor emergency is needed * Poor vasculature * Coagulopathy, or ongoing thrombolytics and/or anticoagulation * Blood-borne infectious disease, e.g. hepatitis B * History of mandatory custody because of psychosis or other psychological disease inappropriate for treatment deemed by treating physician * With other immune diseases, or chronic use of immunosuppressants or steroids * Compliance cannot be expected * Other conditions requiring exclusion deemed by physician
Trial Locations
- Department of Urology Peking University First Hospital, Beijing, Beijing Municipality, China
Frequently Asked Questions
What is clinical trial NCT02863913?
NCT02863913 is a Phase 1 INTERVENTIONAL study titled "PD-1 Knockout Engineered T Cells for Muscle-invasive Bladder Cancer." It is currently withdrawn and is sponsored by Peking University.
What conditions does NCT02863913 study?
This trial investigates treatments for Invasive Bladder Cancer Stage IV. The primary condition under study is Invasive Bladder Cancer Stage IV.
What treatments are being tested in NCT02863913?
The interventions being studied include: PD-1 Knockout T Cells (BIOLOGICAL), Cyclophosphamide (DRUG), IL-2 (DRUG). PD-1 Knockout T Cells and PD-1 wild-type T Cells will be made by Cell Biotech Co., Ltd. 2x107/kg T cells will be used for test group and comparable group separately.
What does Phase 1 mean for NCT02863913?
Phase 1 trials are the first stage of testing a new treatment in humans. They focus on safety, dosage, and side effects, usually involving 20-100 healthy volunteers or patients.
What is the current status of NCT02863913?
This trial is currently "Withdrawn." It started on 2016-09. The estimated completion date is 2019-09.
Who is sponsoring NCT02863913?
NCT02863913 is sponsored by Peking University. The sponsor is responsible for funding, designing, and overseeing the clinical trial.
How is NCT02863913 designed?
This is a interventional study, uses non_randomized allocation, follows a parallel design, employs none masking.
What are the primary outcomes being measured in NCT02863913?
The primary outcome measures are: Number of participants with Adverse Events and/or Dose Limiting Toxicities as a Measure of Safety and tolerability of dose of PD-1 Knockout T cells using Common Terminology Criteria for Adverse Events (CTCAE v4.0) in patients (Dose Escalation - Approximately 6 months). These are the main endpoints researchers use to determine whether the treatment is effective.
Where is NCT02863913 being conducted?
This trial is being conducted at 1 site, including Beijing, Beijing Municipality (China).
Where can I find official information about NCT02863913?
The official record for NCT02863913 is available on ClinicalTrials.gov at https://clinicaltrials.gov/study/NCT02863913. This government database provides the most up-to-date and detailed information about the trial.
What is NCT02863913 testing in simple terms?
This trial tested a new type of cell therapy called PD-1 knockout engineered T cells. It was for patients with advanced bladder cancer that had spread and didn't respond to standard treatments.
Why is this trial significant?
This trial aimed to explore a novel cell therapy approach for a difficult-to-treat form of bladder cancer, addressing a gap in treatment options for patients with advanced disease.
What are the potential risks of participating in NCT02863913?
Common side effects could include reactions related to the infusion of T cells, such as fever or flu-like symptoms. There is a risk of unexpected immune responses or toxicities that could affect major organs. The treatment might not be effective, and the cancer could continue to grow or spread. As with any clinical trial, participants are closely monitored and can withdraw at any time.
Should I consider participating in NCT02863913?
Ask your doctor about the specific risks and benefits of this type of cell therapy. Understand what the treatment schedule will be, including hospital visits and any required medications. Be prepared for regular blood tests and imaging scans to monitor your response and any side effects. Always discuss clinical trial participation with your healthcare provider to determine if it is appropriate for your specific situation.
What does NCT02863913 signal from an investment perspective?
While this specific trial was withdrawn, the development of engineered T cell therapies for solid tumors represents a significant area of investment and research in oncology, with potential for future This is a Phase 1 trial, which is in early development stages.
What happens if the treatment in this trial doesn't work?
Participation involved receiving the engineered T cells, along with other medications, and regular check-ups. Participants in clinical trials always have the right to withdraw and pursue alternative treatments. The study team will help transition patients to other available options.
Related Conditions
More Invasive Bladder Cancer Stage IV Trials
This analysis is AI-generated and does not constitute medical advice. Always consult your healthcare provider before making decisions about clinical trial participation.