A Phase 1, Open-Label, Multicenter, Dose Escalation and Cohort Expansion Study of the Safety and Efficacy of Anti-CD70 Allogeneic CRISPR-Cas9-Engineered T Cells (CTX130) in Subjects With Relapsed or Refractory T or B Cell Malignancies

Trial for advanced T and B cell cancers terminated, previously tested new gene-edited cell therapy.

NCT: NCT04502446 · Status: TERMINATED · Phase: Phase 1 · Sponsor: CRISPR Therapeutics AG · Started: 2020-07-31 · Est. Completion: 2024-08-30

Plain English Summary

A Safety and Efficacy Study Evaluating CTX130 in Subjects With Relapsed or Refractory T or B Cell Malignancies (COBALT-LYM) is a Phase 1 clinical trial sponsored by CRISPR Therapeutics AG studying T Cell Lymphoma. This trial tested a new type of cell therapy called CTX130, which uses gene editing (CRISPR-Cas9) to engineer a patient's own immune cells to fight cancer. It was designed for adults with T cell lymphoma or Diffuse Large B-Cell Lymphoma (DLBCL) that had returned or did not respond to previous treatments. Participation involved receiving CTX130 infusions, with regular check-ups to monitor for side effects and assess how well the treatment worked. Alternative treatments for these advanced blood cancers include chemotherapy, radiation, stem cell transplant, and other targeted therapies. The trial aims to enroll 49 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 T or B cell malignancies.

Who Can Participate

Here is what you need to know about eligibility for this trial. Adults aged 18 and older with a confirmed diagnosis of T cell lymphoma or Diffuse Large B-Cell Lymphoma (DLBCL). Patients must have had their cancer return or not respond to prior treatments. Individuals with good general health, including adequate kidney, liver, heart, and lung function, and a good performance status (able to carry out daily activities). People who have not had a prior stem cell transplant, have not received prior treatment targeting CD70, and do not have certain pre-existing heart, lung, or brain conditions. This trial is studying T Cell Lymphoma, so participants generally need a confirmed diagnosis.

What They're Measuring

The primary outcomes measured the safety of CTX130 and how well it controlled the cancer in the short term after treatment, helping doctors understand its initial effects. The specific primary outcome measures are: Part A (dose escalation) (From CTX130 infusion up to 28 days post-infusion); Part B (cohort expansion) (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 explore a novel gene-edited cell therapy for relapsed or refractory T and B cell malignancies, addressing a critical need for new treatment options in these aggressive blood cancer This research targets T Cell Lymphoma, where improved treatment options are needed.

Investor Insight

While this Phase 1 trial was terminated, the development of CRISPR-based cell therapies like CTX130 signals significant investment and innovation in the oncology market, with potential for future brea 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 CTX130, and how it compares to other available treatments for your condition. Be prepared for regular hospital visits for infusions and monitoring, which may include blood tests, imaging scans, and physical examinations. Understand that this is an experimental treatment, and its effectiveness and long-term effects are still being studied. The trial is being conducted at 10 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

Interventions

Primary Outcomes

Secondary Outcomes

Full Eligibility Criteria

Inclusion Criteria (abbreviated):

1. Age ≥18 years.
2. Confirmed diagnosis of a T cell malignancy or Diffuse Large B-Cell Lymphoma (DLBCL).
3. Eastern Cooperative Oncology Group (ECOG) performance status of 0-1.
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.

Exclusion Criteria (abbreviated):

1. Prior allogeneic stem cell transplant (SCT).
2. Prior treatment with any anti-CD70 targeting agents.
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.
8. Pregnant or breastfeeding females.

Trial Locations

Frequently Asked Questions

What is clinical trial NCT04502446?

NCT04502446 is a Phase 1 INTERVENTIONAL study titled "A Safety and Efficacy Study Evaluating CTX130 in Subjects With Relapsed or Refractory T or B Cell Malignancies (COBALT-LYM)." It is currently terminated and is sponsored by CRISPR Therapeutics AG. The trial targets enrollment of 49 participants.

What conditions does NCT04502446 study?

This trial investigates treatments for T Cell Lymphoma. The primary condition under study is T Cell Lymphoma.

What treatments are being tested in NCT04502446?

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 NCT04502446?

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 NCT04502446?

This trial is currently "Terminated." It started on 2020-07-31. The estimated completion date is 2024-08-30.

Who is sponsoring NCT04502446?

NCT04502446 is sponsored by CRISPR Therapeutics AG. The sponsor is responsible for funding, designing, and overseeing the clinical trial.

How many people can participate in NCT04502446?

The trial aims to enroll 49 participants. The trial status is terminated.

How is NCT04502446 designed?

This is a interventional study, uses na allocation, follows a sequential design, employs none masking.

What are the primary outcomes being measured in NCT04502446?

The primary outcome measures are: Part A (dose escalation) (From CTX130 infusion up to 28 days post-infusion); Part B (cohort expansion) (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 NCT04502446 being conducted?

This trial is being conducted at 10 sites, including Duarte, California; Stanford, California; New Haven, Connecticut; Miami, Florida and 6 more sites (United States, Australia, Canada).

Where can I find official information about NCT04502446?

The official record for NCT04502446 is available on ClinicalTrials.gov at https://clinicaltrials.gov/study/NCT04502446. This government database provides the most up-to-date and detailed information about the trial.

What is NCT04502446 testing in simple terms?

This trial tested a new type of cell therapy called CTX130, which uses gene editing (CRISPR-Cas9) to engineer a patient's own immune cells to fight cancer. It was designed for adults with T cell lymphoma or Diffuse Large B-Cell Lymphoma (DLBCL) that had returned or did not respond to previous treatments.

Why is this trial significant?

This trial aimed to explore a novel gene-edited cell therapy for relapsed or refractory T and B cell malignancies, addressing a critical need for new treatment options in these aggressive blood cancer

What are the potential risks of participating in NCT04502446?

Common side effects may include fatigue, fever, nausea, and low blood cell counts, which can increase the risk of infection. More serious risks could involve cytokine release syndrome (a severe immune reaction), neurological side effects, or organ damage. The treatment involves a modified immune cell therapy, and there's a risk of the immune system attacking the body's own tissues. As with any clinical trial, participants are closely monitored and can withdraw at any time.

Should I consider participating in NCT04502446?

Ask your doctor about the specific risks and benefits of CTX130, and how it compares to other available treatments for your condition. Be prepared for regular hospital visits for infusions and monitoring, which may include blood tests, imaging scans, and physical examinations. Understand that this is an experimental treatment, and its effectiveness and long-term effects are still being studied. Always discuss clinical trial participation with your healthcare provider to determine if it is appropriate for your specific situation.

What does NCT04502446 signal from an investment perspective?

While this Phase 1 trial was terminated, the development of CRISPR-based cell therapies like CTX130 signals significant investment and innovation in the oncology market, with potential for future brea 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 infusions, with regular check-ups to monitor for side effects and assess how well the treatment worked. 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.