A Phase 1/2 Dose Escalation and Cohort Expansion Study of the Safety and Efficacy of Allogeneic CRISPR-Cas9-Engineered T Cells (CTX110) in Subjects With Relapsed or Refractory B-Cell Malignancies (CARBON)

Study of CTX110 for Relapsed/Refractory B-Cell Cancers (CARBON)

NCT: NCT04035434 · Status: TERMINATED · Phase: Phase 2 · Sponsor: CRISPR Therapeutics AG · Started: 2019-07-22 · Est. Completion: 2024-10-04

Plain English Summary

A Safety and Efficacy Study Evaluating CTX110 in Subjects With Relapsed or Refractory B-Cell Malignancies (CARBON) is a Phase 2 clinical trial sponsored by CRISPR Therapeutics AG studying B-cell Malignancy, Non-Hodgkin Lymphoma, B-cell Lymphoma, Adult B Cell ALL. Tests a new gene-edited cell therapy (CTX110) to fight certain blood cancers. For adults with specific types of B-cell lymphomas or leukemias that have returned or not responded to treatment. Involves receiving an infusion of the therapy; patients will be closely monitored for side effects and effectiveness. Alternative treatments may include chemotherapy, radiation, stem cell transplant, or other targeted therapies. The trial aims to enroll 93 participants.

Official Summary

This is an open-label, multicenter, Phase 1/2 study evaluating the safety and efficacy of CTX110 in subjects with relapsed or refractory B-cell malignancies.

Who Can Participate

Here is what you need to know about eligibility for this trial. Adults aged 18 and older. Patients with non-Hodgkin lymphoma (NHL) who have had at least two prior treatments, or B-cell acute lymphoblastic leukemia (ALL) that is refractory or relapsed. Must have good general health, including adequate organ function (kidneys, liver, heart, lungs). Cannot have had a recent stem cell transplant (allogeneic) or certain other conditions like active infections or other cancers. This trial is studying B-cell Malignancy, Non-Hodgkin Lymphoma, B-cell Lymphoma, Adult B Cell ALL, so participants generally need a confirmed diagnosis.

What They're Measuring

The primary outcome measures how well the treatment works by looking at the percentage of patients who show a positive response, meaning their cancer shrinks or disappears. The specific primary outcome measures are: Dose Escalation Phase 1: Number of Participants With Dose-limiting Toxicities (DLT) in NHL and B Cell ALL Population (Up to 28 days); Dose Expansion Phase 1 and Phase 2: Percentage of Participants With Objective Response Rate in NHL Population (Up to 5 years). 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 2, which tests whether the treatment actually works against the target condition. Phase 2 trials involve 100-300 patients and continue to monitor safety while evaluating effectiveness. This phase often tests different dosages to find the optimal amount. About 33% of Phase 2 drugs advance to Phase 3. If successful, the treatment will move to large-scale Phase 3 trials needed for FDA approval.

Why This Trial Matters

This trial explores a novel gene-editing therapy (CTX110) to address the unmet need for effective treatments in patients with relapsed or refractory B-cell malignancies. Phase 2 success would typically lead to larger Phase 3 trials needed for regulatory approval. This research targets B-cell Malignancy, Non-Hodgkin Lymphoma, B-cell Lymphoma, Adult B Cell ALL, where improved treatment options are needed.

Investor Insight

This trial represents an investment in the rapidly growing field of cell and gene therapy for cancer, with CRISPR Therapeutics aiming to advance a potentially groundbreaking treatment for difficult-to Phase 2 trials have approximately a 15-20% chance of eventually gaining FDA approval.

Is This Trial Right for Me?

Ask your doctor about the potential benefits and risks of CTX110, and how it compares to other treatment options. Participation involves regular clinic visits for infusions, blood tests, scans, and monitoring for side effects. You will need to use effective contraception during the study and for a period afterward. The trial is being conducted at 20 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

Key Inclusion Criteria:

1. For NHL patients: Age ≥18 years. For B cell ALL patients: age ≥18 years to ≤70 years
2. Refractory or relapsed non-Hodgkin lymphoma, as evidenced by 2 or more lines of prior therapy, or histologically confirmed B cell ALL, refractory or relapsed.
3. Eastern Cooperative Oncology Group performance status 0 or 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 CTX110 infusion.

Key Exclusion Criteria:

1. For NHL patients: prior allogeneic HSCT. For B cell ALL patients: prior allogeneic HSCT within 6 months, and/or any evidence of GvHD.
2. History of central nervous system (CNS) involvement by malignancy
3. History of a seizure disorder, cerebrovascular ischemia/hemorrhage, dementia, cerebellar disease, or any autoimmune disease with CNS involvement.
4. Presence of bacterial, viral, or fungal infection that is uncontrolled.
5. Positive for HIV, or active hepatitis B virus or hepatitis C virus infection.
6. Previous or concurrent malignancy, except basal cell or squamous cell skin carcinoma, adequately resected and in situ carcinoma of cervix, or a previous malignancy that was completely resected and has been in remission for ≥5 years.
7. For NHL patients: Use of systemic anti-tumor therapy or investigational agent within 14 days or 5 half-lives, whichever is longer, of CTX110 infusion. For B cell ALL patients: Use of systemic antitumor therapy within 7 days of CTX110 infusion.
8. Primary immunodeficiency disorder or active autoimmune disease requiring steroids and/or other immunosuppressive therapy.
9. Women who are pregnant or breastfeeding.

Trial Locations

Frequently Asked Questions

What is clinical trial NCT04035434?

NCT04035434 is a Phase 2 INTERVENTIONAL study titled "A Safety and Efficacy Study Evaluating CTX110 in Subjects With Relapsed or Refractory B-Cell Malignancies (CARBON)." It is currently terminated and is sponsored by CRISPR Therapeutics AG. The trial targets enrollment of 93 participants.

What conditions does NCT04035434 study?

This trial investigates treatments for B-cell Malignancy, Non-Hodgkin Lymphoma, B-cell Lymphoma, Adult B Cell ALL. The primary condition under study is B-cell Malignancy.

What treatments are being tested in NCT04035434?

The interventions being studied include: CTX110 (BIOLOGICAL). CTX110 (CD19-directed T-cell immunotherapy comprised of allogeneic T cells genetically modified ex vivo using CRISPR-Cas9 gene editing components

What does Phase 2 mean for NCT04035434?

Phase 2 trials test whether the treatment works for the intended condition. They involve 100-300 patients and continue to evaluate safety while measuring effectiveness.

What is the current status of NCT04035434?

This trial is currently "Terminated." It started on 2019-07-22. The estimated completion date is 2024-10-04.

Who is sponsoring NCT04035434?

NCT04035434 is sponsored by CRISPR Therapeutics AG. The sponsor is responsible for funding, designing, and overseeing the clinical trial.

How many people can participate in NCT04035434?

The trial aims to enroll 93 participants. The trial status is terminated.

How is NCT04035434 designed?

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

What are the primary outcomes being measured in NCT04035434?

The primary outcome measures are: Dose Escalation Phase 1: Number of Participants With Dose-limiting Toxicities (DLT) in NHL and B Cell ALL Population (Up to 28 days); Dose Expansion Phase 1 and Phase 2: Percentage of Participants With Objective Response Rate in NHL Population (Up to 5 years). These are the main endpoints researchers use to determine whether the treatment is effective.

Where is NCT04035434 being conducted?

This trial is being conducted at 20 sites, including Los Angeles, California; San Francisco, California; Jacksonville, Florida; Atlanta, Georgia and 16 more sites (United States).

Where can I find official information about NCT04035434?

The official record for NCT04035434 is available on ClinicalTrials.gov at https://clinicaltrials.gov/study/NCT04035434. This government database provides the most up-to-date and detailed information about the trial.

What is NCT04035434 testing in simple terms?

Tests a new gene-edited cell therapy (CTX110) to fight certain blood cancers. For adults with specific types of B-cell lymphomas or leukemias that have returned or not responded to treatment.

Why is this trial significant?

This trial explores a novel gene-editing therapy (CTX110) to address the unmet need for effective treatments in patients with relapsed or refractory B-cell malignancies.

What are the potential risks of participating in NCT04035434?

Common side effects may include low blood cell counts, fever, fatigue, and infusion-related reactions. Serious risks can include cytokine release syndrome (a severe immune reaction) and neurotoxicity (effects on the nervous system). There is a risk that the treatment may not be effective or could cause new health problems. As with any clinical trial, participants are closely monitored and can withdraw at any time.

Should I consider participating in NCT04035434?

Ask your doctor about the potential benefits and risks of CTX110, and how it compares to other treatment options. Participation involves regular clinic visits for infusions, blood tests, scans, and monitoring for side effects. You will need to use effective contraception during the study and for a period afterward. Always discuss clinical trial participation with your healthcare provider to determine if it is appropriate for your specific situation.

What does NCT04035434 signal from an investment perspective?

This trial represents an investment in the rapidly growing field of cell and gene therapy for cancer, with CRISPR Therapeutics aiming to advance a potentially groundbreaking treatment for difficult-to This is a Phase 2 trial, which is focused on confirming efficacy before larger pivotal studies.

What happens if the treatment in this trial doesn't work?

Involves receiving an infusion of the therapy; patients will be closely monitored for side effects 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.