A Phase 1/2, Open-Label, Multicenter, Dose Escalation and Cohort Expansion Study of the Safety and Efficacy of Anti-CD19 Allogeneic CRISPR-Cas9-Engineered T Cells (CTX112) in Subjects With Relapsed or Refractory B Cell Malignancies
New T-cell therapy for relapsed B-cell cancers tested in Phase 1/2 trial
Plain English Summary
A Safety and Efficacy Study Evaluating CTX112 in Subjects With Relapsed or Refractory B-Cell Malignancies is a Phase 2 clinical trial sponsored by CRISPR Therapeutics AG studying B-cell Lymphoma, Non-Hodgkin Lymphoma, B-cell Malignancy, Chronic Lymphocytic Leukemia (CLL)/Small Lymphocytic Lymphoma (SLL), Follicular Lymphoma, Mantle Cell Lymphoma, Marginal Zone Lymphoma, Large B-cell Lymphoma. This trial tests a new cell therapy called CTX112 for adults with B-cell cancers that have returned or not responded to previous treatments. It is for patients aged 18 and older with specific types of B-cell lymphomas and leukemias. Participation involves receiving CTX112 infusions and regular medical check-ups, including blood tests and scans. Alternative treatments may include chemotherapy, radiation, or other targeted therapies, depending on the specific cancer and prior treatments. The trial aims to enroll 120 participants.
Official Summary
This is an open-label, multicenter, Phase 1/2 study evaluating the safety and efficacy of CTX112™ 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 with a confirmed B-cell malignancy that has relapsed or is refractory. Patients must have good general health and adequate organ function (kidneys, liver, heart, lungs). Individuals with a history of certain central nervous system conditions, active infections, or recent cancer treatments may not be eligible. Women of childbearing potential and men must agree to use contraception during and after the study. This trial is studying B-cell Lymphoma, Non-Hodgkin Lymphoma, B-cell Malignancy, Chronic Lymphocytic Leukemia (CLL)/Small Lymphocytic Lymphoma (SLL), Follicular Lymphoma, Mantle Cell Lymphoma, Marginal Zone Lymphoma, Large B-cell Lymphoma, so participants generally need a confirmed diagnosis. The trial is currently accepting new participants.
What They're Measuring
The primary outcome measures how safe the treatment is by tracking side effects and how often it works by looking at tumor shrinkage or disappearance. The specific primary outcome measures are: Phase 1 (Dose Escalation): Incidence of adverse events, defined as dose-limiting toxicities (From CTX112 infusion up to 28 days post-infusion); Phase 2 (Cohort Expansion): Objective response rate (From CTX112 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 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-edited T-cell therapy, CTX112, 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 Lymphoma, Non-Hodgkin Lymphoma, B-cell Malignancy, Chronic Lymphocytic Leukemia (CLL)/Small Lymphocytic Lymphoma (SLL), Follicular Lymphoma, Mantle Cell Lymphoma, Marginal Zone Lymphoma, Large B-cell Lymphoma, where improved treatment options are needed.
Investor Insight
This trial signals investment in CRISPR-based cell therapies for blood cancers, a rapidly growing market with significant unmet needs and potential for high-value drug development. 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 CTX112, and how it compares to other treatment options. Participation involves regular clinic visits for infusions, blood draws, imaging scans, and monitoring for side effects. You will need to use effective contraception during the study and for a period afterward. This trial is currently recruiting participants. 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: 120 participants
Interventions
- BIOLOGICAL: CTX112 — CTX112 (CD19-directed T-cell immunotherapy comprised of allogeneic T cells genetically modified ex vivo using CRISPR-Cas9 gene editing components)
Primary Outcomes
- Phase 1 (Dose Escalation): Incidence of adverse events, defined as dose-limiting toxicities (From CTX112 infusion up to 28 days post-infusion)
- Phase 2 (Cohort Expansion): Objective response rate (From CTX112 infusion up to 60 months post-infusion)
Secondary Outcomes
- Duration of Response (From date of first objective response of complete response (CR)/partial response (PR) until date of disease progression or death due to any cause, assessed up to 60 months)
- Duration of Clinical Benefit (DOCB) (From date of first objective response of CR/PR until the relapse or death that followed the last response, assessed up to 60 months)
- Progression Free Survival (From date of CTX112 infusion until date of disease progression or death due to any cause, assessed up to 60 months)
- Overall Survival (From date of CTX112 infusion until date of death due to any cause, assessed up to 60 months)
Full Eligibility Criteria
Key Inclusion Criteria: 1. Age ≥18 years. 2. Refractory or relapsed B cell malignancy. 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 CTX112 infusion. Key Exclusion Criteria: 1. Prior allogeneic hematopoietic stem cell transplant (HSCT). 2. Active or 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 or requires IV anti-infectives. 5. Active HIV, hepatitis B virus or hepatitis C virus infection. 6. Previous or concurrent malignancy in the last 3 years (with the exception of non-melanoma skin cancer and other cancers deemed by the investigator and medical monitor to be of low likelihood for recurrence). 7. Concurrent systemic treatment with an anticancer biologic (e.g., monoclonal antibody) within 30 days prior to CTX112 infusion or with a nonbiological anticancer drug within 14 days prior to CTX112 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
- University of Kansas, Westwood, Kansas, United States
- Washington University, St Louis, Missouri, United States
- SCRI, San Antonio, Texas, United States
- University of Utah, Salt Lake City, Utah, United States
- Royal Prince Alfred, Camperdown, New South Wales, Australia
- Alfred Health, Melbourne, Victoria, Australia
- Sir Charles Gairdner Hospital, Nedlands, Western Australia, Australia
Frequently Asked Questions
What is clinical trial NCT05643742?
NCT05643742 is a Phase 2 INTERVENTIONAL study titled "A Safety and Efficacy Study Evaluating CTX112 in Subjects With Relapsed or Refractory B-Cell Malignancies." It is currently recruiting and is sponsored by CRISPR Therapeutics AG. The trial targets enrollment of 120 participants.
What conditions does NCT05643742 study?
This trial investigates treatments for B-cell Lymphoma, Non-Hodgkin Lymphoma, B-cell Malignancy, Chronic Lymphocytic Leukemia (CLL)/Small Lymphocytic Lymphoma (SLL), Follicular Lymphoma, Mantle Cell Lymphoma, Marginal Zone Lymphoma, Large B-cell Lymphoma. The primary condition under study is B-cell Lymphoma.
What treatments are being tested in NCT05643742?
The interventions being studied include: CTX112 (BIOLOGICAL). CTX112 (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 NCT05643742?
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 NCT05643742?
This trial is currently "Recruiting." It started on 2023-03-10. The estimated completion date is 2030-02.
Who is sponsoring NCT05643742?
NCT05643742 is sponsored by CRISPR Therapeutics AG. The sponsor is responsible for funding, designing, and overseeing the clinical trial.
How many people can participate in NCT05643742?
The trial aims to enroll 120 participants. The trial is currently recruiting and accepting new participants.
How is NCT05643742 designed?
This is a interventional study, uses na allocation, follows a sequential design, employs none masking.
What are the primary outcomes being measured in NCT05643742?
The primary outcome measures are: Phase 1 (Dose Escalation): Incidence of adverse events, defined as dose-limiting toxicities (From CTX112 infusion up to 28 days post-infusion); Phase 2 (Cohort Expansion): Objective response rate (From CTX112 infusion up to 60 months post-infusion). These are the main endpoints researchers use to determine whether the treatment is effective.
Where is NCT05643742 being conducted?
This trial is being conducted at 7 sites, including Westwood, Kansas; St Louis, Missouri; San Antonio, Texas; Salt Lake City, Utah and 3 more sites (United States, Australia).
Where can I find official information about NCT05643742?
The official record for NCT05643742 is available on ClinicalTrials.gov at https://clinicaltrials.gov/study/NCT05643742. This government database provides the most up-to-date and detailed information about the trial.
What is NCT05643742 testing in simple terms?
This trial tests a new cell therapy called CTX112 for adults with B-cell cancers that have returned or not responded to previous treatments. It is for patients aged 18 and older with specific types of B-cell lymphomas and leukemias.
Why is this trial significant?
This trial explores a novel gene-edited T-cell therapy, CTX112, 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 NCT05643742?
Common side effects may include fever, fatigue, nausea, and low blood cell counts. Serious risks can include cytokine release syndrome (a severe immune reaction), neurological side effects, and increased risk of infections. The treatment involves a complex cell therapy, and long-term effects are still being studied. As with any clinical trial, participants are closely monitored and can withdraw at any time.
Should I consider participating in NCT05643742?
Ask your doctor about the potential benefits and risks of CTX112, and how it compares to other treatment options. Participation involves regular clinic visits for infusions, blood draws, imaging 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 NCT05643742 signal from an investment perspective?
This trial signals investment in CRISPR-based cell therapies for blood cancers, a rapidly growing market with significant unmet needs and potential for high-value drug development. 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?
Participation involves receiving CTX112 infusions and regular medical check-ups, including blood tests and scans. 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.