A Phase 1 Dose Escalation and Cohort Expansion Study of the Safety and Efficacy of Allogeneic CRISPR-Cas9-Engineered T Cells (CTX130) in Subjects With Advanced, Relapsed or Refractory Renal Cell Carcinoma With Clear Cell Differentiation
Study of CTX130 for Advanced Kidney Cancer (COBALT-RCC) - TERMINATED
Plain English Summary
A Safety and Efficacy Study Evaluating CTX130 in Subjects With Relapsed or Refractory Renal Cell Carcinoma (COBALT-RCC) is a Phase 1 clinical trial sponsored by CRISPR Therapeutics AG studying Renal Cell Carcinoma. This study tested a new cell therapy called CTX130 for advanced kidney cancer that has not responded to other treatments. It was for adults with a specific type of kidney cancer (clear cell differentiation) that has returned or worsened after standard treatments. Participation involved receiving CTX130, a type of immunotherapy, and regular check-ups to monitor safety and effectiveness. Alternative treatments for this condition include other types of chemotherapy, targeted therapies, and immunotherapies. The trial aims to enroll 19 participants.
Official Summary
This is a single-arm, open-label, multicenter, Phase 1 study evaluating the safety and efficacy of CTX130 in subjects with relapsed or refractory renal cell carcinoma.
Who Can Participate
Here is what you need to know about eligibility for this trial. Adults aged 18 and older with a specific type of advanced kidney cancer that has progressed after standard treatment. Patients must have good general health and organ function (kidneys, liver, heart, lungs). Individuals who have previously received certain types of cell therapies or treatments targeting CD70 are not eligible. People with certain brain, heart, or lung conditions, active infections, or active autoimmune diseases may not be able to join. This trial is studying Renal Cell Carcinoma, so participants generally need a confirmed diagnosis.
What They're Measuring
The primary outcomes measured how often side effects occurred and how well the treatment worked in shrinking tumors, indicating the safety and potential benefit of CTX130. The specific primary outcome measures are: Part A (dose escalation): Incidence of adverse events (From CTX130 infusion up to 28 days post-infusion); Part B (cohort expansion): Objective response rate (From CTX130 infusion up to 60 months post-infusion]). 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 address a significant unmet need for patients with advanced kidney cancer who have exhausted standard treatment options, by exploring a novel gene-edited cell therapy. This research targets Renal Cell Carcinoma, where improved treatment options are needed.
Investor Insight
This trial, though terminated, explored CRISPR-Cas9 technology in cancer therapy, a rapidly advancing field with significant investment potential, though approval probability for this specific termina Phase 1 trials have approximately a 10% chance of eventually gaining FDA approval.
Is This Trial Right for Me?
Ask your doctor about the potential risks and benefits of CTX130, and what to expect during treatment. Be prepared for regular hospital visits for infusions and monitoring of your health and response to treatment. You will need to use effective contraception during and for a period after the treatment. The trial is being conducted at 7 sites. 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: NA
- Model: SEQUENTIAL
- Masking: NONE
- Enrollment: 19 participants
Interventions
- BIOLOGICAL: CTX130 — CTX130 CD70-directed T-cell immunotherapy comprised of allogeneic T cells genetically modified ex vivo using CRISPR-Cas9 gene editing components.
Primary Outcomes
- Part A (dose escalation): Incidence of adverse events (From CTX130 infusion up to 28 days post-infusion)
- Part B (cohort expansion): Objective response rate (From CTX130 infusion up to 60 months post-infusion])
Secondary Outcomes
- Progression Free Survival (From date of CTX130 infusion until date of disease progression or death due to any cause, assessed up to 60 months)
- Overall Survival (From date of CTX130 until date of death due to any cause, assessed up to 60 months)
Full Eligibility Criteria
Abbreviated Inclusion Criteria: 1. Age ≥18 years and body weight ≥42 kg. 2. Unresectable or metastatic RCC that has exploited standard of care treatment. 3. Karnofsky performance status (KPS) ≥80%. 4. Adequate renal, liver, cardiac, and pulmonary organ function. 5. Female subjects of childbearing potential and male subjects must agree to use acceptable method(s) of contraception from enrollment through at least 12 months after CTX130 infusion. Abbreviated Exclusion Criteria: 1. Prior treatment with any anti-CD70 targeting agents. 2. Prior treatment with any CAR T cells or any other modified T or natural killer (NK) cells. 3. History of certain central nervous system (CNS), cardiac or pulmonary conditions. 4. Active HIV, hepatitis B virus or hepatitis C virus infection. 5. Previous or concurrent malignancy, except treated with curative approach not requiring systemic therapy and in remission for \>12 months, or any other localized malignancy with low risk of developing into metastatic disease. 6. Primary immunodeficiency disorder or active autoimmune disease requiring steroids and/or other immunosuppressive therapy. 7. Prior solid organ transplantation or bone marrow transplant. 8. Pregnant or breastfeeding females.
Trial Locations
- Research Site 2, Duarte, California, United States
- Research Site 5, Hartford, Connecticut, United States
- Research Site 4, Houston, Texas, United States
- Research Site 3, Salt Lake City, Utah, United States
- Research Site 1, Melbourne, Victoria, Australia
- Research Site 6, Toronto, Ontario, Canada
- Research Site 7, Amsterdam, North Holland, Netherlands
Frequently Asked Questions
What is clinical trial NCT04438083?
NCT04438083 is a Phase 1 INTERVENTIONAL study titled "A Safety and Efficacy Study Evaluating CTX130 in Subjects With Relapsed or Refractory Renal Cell Carcinoma (COBALT-RCC)." It is currently terminated and is sponsored by CRISPR Therapeutics AG. The trial targets enrollment of 19 participants.
What conditions does NCT04438083 study?
This trial investigates treatments for Renal Cell Carcinoma. The primary condition under study is Renal Cell Carcinoma.
What treatments are being tested in NCT04438083?
The interventions being studied include: CTX130 (BIOLOGICAL). CTX130 CD70-directed T-cell immunotherapy comprised of allogeneic T cells genetically modified ex vivo using CRISPR-Cas9 gene editing components.
What does Phase 1 mean for NCT04438083?
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 NCT04438083?
This trial is currently "Terminated." It started on 2020-06-16. The estimated completion date is 2024-10-08.
Who is sponsoring NCT04438083?
NCT04438083 is sponsored by CRISPR Therapeutics AG. The sponsor is responsible for funding, designing, and overseeing the clinical trial.
How many people can participate in NCT04438083?
The trial aims to enroll 19 participants. The trial status is terminated.
How is NCT04438083 designed?
This is a interventional study, uses na allocation, follows a sequential design, employs none masking.
What are the primary outcomes being measured in NCT04438083?
The primary outcome measures are: Part A (dose escalation): Incidence of adverse events (From CTX130 infusion up to 28 days post-infusion); Part B (cohort expansion): Objective response rate (From CTX130 infusion up to 60 months post-infusion]). These are the main endpoints researchers use to determine whether the treatment is effective.
Where is NCT04438083 being conducted?
This trial is being conducted at 7 sites, including Duarte, California; Hartford, Connecticut; Houston, Texas; Salt Lake City, Utah and 3 more sites (United States, Australia, Canada).
Where can I find official information about NCT04438083?
The official record for NCT04438083 is available on ClinicalTrials.gov at https://clinicaltrials.gov/study/NCT04438083. This government database provides the most up-to-date and detailed information about the trial.
What is NCT04438083 testing in simple terms?
This study tested a new cell therapy called CTX130 for advanced kidney cancer that has not responded to other treatments. It was for adults with a specific type of kidney cancer (clear cell differentiation) that has returned or worsened after standard treatments.
Why is this trial significant?
This trial aimed to address a significant unmet need for patients with advanced kidney cancer who have exhausted standard treatment options, by exploring a novel gene-edited cell therapy.
What are the potential risks of participating in NCT04438083?
The main risks involve side effects from the cell therapy, which can range from mild to severe. Potential side effects may include reactions to the treatment, effects on organ function, or the development of other medical conditions. Specific risks related to cell therapies include cytokine release syndrome and neurological toxicities. As with any clinical trial, participants are closely monitored and can withdraw at any time.
Should I consider participating in NCT04438083?
Ask your doctor about the potential risks and benefits of CTX130, and what to expect during treatment. Be prepared for regular hospital visits for infusions and monitoring of your health and response to treatment. You will need to use effective contraception during and for a period after the treatment. Always discuss clinical trial participation with your healthcare provider to determine if it is appropriate for your specific situation.
What does NCT04438083 signal from an investment perspective?
This trial, though terminated, explored CRISPR-Cas9 technology in cancer therapy, a rapidly advancing field with significant investment potential, though approval probability for this specific termina 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 CTX130, a type of immunotherapy, and regular check-ups to monitor safety and effectiveness. 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.
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This analysis is AI-generated and does not constitute medical advice. Always consult your healthcare provider before making decisions about clinical trial participation.