A Phase 1/2 Dose Evaluation and Cohort Expansion Study of the Safety and Efficacy of Anti-CD70 Allogeneic CRISPR-Cas9-Engineered T Cells (CTX131) in Adult Subjects With Relapsed/Refractory Hematologic Malignancies

New Gene-Edited Cell Therapy for Blood Cancers in Adults

NCT: NCT06492304 · Status: COMPLETED · Phase: Phase 2 · Sponsor: CRISPR Therapeutics · Started: 2024-08-13 · Est. Completion: 2026-03-11

Plain English Summary

A Safety and Efficacy Study Evaluating CTX131 in Adult Subjects With Relapsed/Refractory Hematologic Malignancies is a Phase 2 clinical trial sponsored by CRISPR Therapeutics studying T Cell Lymphoma, B Cell Lymphoma, Acute Myeloid Leukemia. This study tests a new cell therapy called CTX131, which uses a patient's own immune cells edited with CRISPR technology to fight cancer. It is for adults with specific types of relapsed or refractory (hard-to-treat) blood cancers, including T cell lymphoma, B cell lymphoma, and acute myeloid leukemia. Participation involves receiving the CTX131 infusion and regular check-ups to monitor for side effects and assess treatment effectiveness. Alternative treatments may include chemotherapy, radiation, stem cell transplant, or other targeted therapies, depending on the specific cancer type and prior treatments. The trial aims to enroll 12 participants.

Official Summary

This is an open label, multicenter, phase 1/2 dose evaluation and cohort expansion study evaluating the safety and efficacy of CTX131 in subjects with Relapsed/Refractory Hematologic Malignancies

Who Can Participate

Here is what you need to know about eligibility for this trial. Adults aged 18 and older with specific types of relapsed or refractory T cell lymphoma, B cell lymphoma, or acute myeloid leukemia. Patients must have a good general health status (ECOG 0-1, or 2 for AML) and adequate organ function (kidneys, liver, heart, lungs). Individuals who have previously been treated with anti-CD70 therapies, have active brain or central nervous system involvement of their cancer, or have uncontrolled infections cannot participate. Women of childbearing potential and men must agree to use effective contraception during and after the study. This trial is studying T Cell Lymphoma, B Cell Lymphoma, Acute Myeloid Leukemia, so participants generally need a confirmed diagnosis.

What They're Measuring

The primary outcomes measure how well the CTX131 therapy works by looking at the percentage of patients whose cancer shrinks or disappears, and how long this response lasts, indicating its potential t The specific primary outcome measures are: Phase 1 Part A (dose escalation) and Part B (dose optimization in selected disease types): (From CTX131 infusion up to 28 days post-infusion); Objective Response rate (ORR) (From CTX131 infusion up to 60 months post-infusion); Composite Complete Remission (CRc) (From CTX131 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-editing approach to harness the immune system against difficult-to-treat blood cancers, addressing a significant unmet need in patients who have exhausted standard tre Phase 2 success would typically lead to larger Phase 3 trials needed for regulatory approval. This research targets T Cell Lymphoma, B Cell Lymphoma, Acute Myeloid Leukemia, where improved treatment options are needed.

Investor Insight

This Phase 2 study of CTX131, a CRISPR-engineered cell therapy, signals a significant investment in the rapidly growing field of cell and gene therapies for hematologic malignancies, with potential fo 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 specific types of lymphoma or leukemia being studied and if your condition fits the trial criteria. Understand the potential risks and side effects of CTX131, including those related to the infusion and the CRISPR technology. Be prepared for regular hospital visits for infusions, blood tests, scans, and monitoring for side effects over an extended period. 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

Interventions

Primary Outcomes

Full Eligibility Criteria

Inclusion Criteria:

1. ≥18 years of age
2. Eastern Cooperative Oncology Group (ECOG) performance status of 0-1 (ECOG status of 2 will be permitted for subjects with AML)
3. Diagnosed with r/r T Cell Lymphoma (TCL), B Cell Lymphoma (BCL), or Acute Myeloid Leukemia (AML) T cell lymphoma, including Stage ≥IIB Mycosis fungoides (MF)/ Sézary syndrome (SS) after at least 2 prior systemic therapies Peripheral T cell lymphoma (PTCL) after at least 1 prior line of therapy (PTCL-note otherwise specified (NOS), PTCL-T follicular helper (TFH), Angioimmunoblastic T cell lymphoma (AITL), Adult T cell leukemia/lymphoma (ATLL) of leukemic, lymphomatous, and chronic unfavorable subtypes), (ALK)- ALCL after at least 1 prior line of therapy, ALK+ Anaplastic large cell lymphoma (ALCL) after at least 2 prior lines of therapy

   B cell lymphoma, including Diffuse large B cell lymphoma (DLBCL)-NOS, transformed marginal zone lymphoma(MZL), transformed FL, high-grade BCL with MYC and BCL2 and/or BCL6 rearrangements, Follicular lymphoma (FL) grade 3b, after at least 2 prior lines of therapy including an anti- CD20 monoclonal antibody and an anthracycline containing regimen Mantle cell lymphoma (MCL) after up to 5 prior lines of therapy which must include an anthracycline- or bendamustine-containing regimen, an anti- CD20 monoclonal antibody, and a BTK inhibitor

   Acute myeloid leukemia or AML/MDS per ELN criteria 2022 after at least 1 prior line of AML therapy. APL, BCR-ABL positive leukemia, and AML secondary to prior therapy or history of genetic syndrome associated with BM failure are excluded.
4. Adequate renal, liver, cardiac and pulmonary organ function
5. Females of childbearing potential and male subjects must agree to use an acceptable, highly effective method of contraception (as specified in the protocol) from enrollment through at least 12 months after last CTX131 infusion

Exclusion Criteria:

1. Prior treatment with anti-CD70 targeting agents
2. Active CNS manifestation of underlying disease
3. History or presence of clinically relevant CNS pathology such as seizure, stroke, severe brain injury, cerebellar disease, myelopathy, history of posterior reversible encephalopathy syndrome with prior therapy, or another condition that in opinion of investigator may increase CAR T-related toxicities
4. Uncontrolled bacterial, viral, or fungal infection
5. Positive for HIV, or active hepatitis B virus or hepatitis C virus infection.
6. Concurrent systemic treatment with an anticancer biologic (e.g., monoclonal antibody) within 30 days prior to CTX131 infusion or with a non-biological anticancer drug within 14 days prior to CTX131 infusion. Mogamulizumab treatment is prohibited 50 days prior to CTX131 infusion.
7. Diagnosis with another invasive malignancy in the last 5 years with the exception of non- melanoma skin cancer and malignancies deemed by the investigator and medical monitor to be of low likelihood for recurrence
8. Primary immunodeficiency disorder or active autoimmune disease requiring steroids and/or other immunosuppressive therapy.
9. Prior solid organ or allogeneic BM transplantation, except for AML cohorts if at least 3 months since allogeneic HSCT, not receiving immunosuppressive therapy or donor lymphocyte infusion post SCT in the 2 weeks prior to lymphodepletion, and have no clinically active GvHD
10. Treatment with CD19-targeting CAR-T within 6 months prior to CTX131 infusion

Trial Locations

Frequently Asked Questions

What is clinical trial NCT06492304?

NCT06492304 is a Phase 2 INTERVENTIONAL study titled "A Safety and Efficacy Study Evaluating CTX131 in Adult Subjects With Relapsed/Refractory Hematologic Malignancies." It is currently completed and is sponsored by CRISPR Therapeutics. The trial targets enrollment of 12 participants.

What conditions does NCT06492304 study?

This trial investigates treatments for T Cell Lymphoma, B Cell Lymphoma, Acute Myeloid Leukemia. The primary condition under study is T Cell Lymphoma.

What treatments are being tested in NCT06492304?

The interventions being studied include: CTX131 (BIOLOGICAL). CTX131 (CD70-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 NCT06492304?

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 NCT06492304?

This trial is currently "Completed." It started on 2024-08-13. The estimated completion date is 2026-03-11.

Who is sponsoring NCT06492304?

NCT06492304 is sponsored by CRISPR Therapeutics. The sponsor is responsible for funding, designing, and overseeing the clinical trial.

How many people can participate in NCT06492304?

The trial aims to enroll 12 participants. The trial status is completed.

How is NCT06492304 designed?

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

What are the primary outcomes being measured in NCT06492304?

The primary outcome measures are: Phase 1 Part A (dose escalation) and Part B (dose optimization in selected disease types): (From CTX131 infusion up to 28 days post-infusion); Objective Response rate (ORR) (From CTX131 infusion up to 60 months post-infusion); Composite Complete Remission (CRc) (From CTX131 infusion up to 60 months post-infusion). These are the main endpoints researchers use to determine whether the treatment is effective.

Where is NCT06492304 being conducted?

This trial is being conducted at 7 sites, including Phoenix, Arizona; Stanford, California; Boston, Massachusetts; New York, New York and 3 more sites (United States, Australia).

Where can I find official information about NCT06492304?

The official record for NCT06492304 is available on ClinicalTrials.gov at https://clinicaltrials.gov/study/NCT06492304. This government database provides the most up-to-date and detailed information about the trial.

What is NCT06492304 testing in simple terms?

This study tests a new cell therapy called CTX131, which uses a patient's own immune cells edited with CRISPR technology to fight cancer. It is for adults with specific types of relapsed or refractory (hard-to-treat) blood cancers, including T cell lymphoma, B cell lymphoma, and acute myeloid leukemia.

Why is this trial significant?

This trial explores a novel gene-editing approach to harness the immune system against difficult-to-treat blood cancers, addressing a significant unmet need in patients who have exhausted standard tre

What are the potential risks of participating in NCT06492304?

Common side effects may include fever, fatigue, nausea, and low blood cell counts, similar to other cell therapies. A serious potential risk is cytokine release syndrome (CRS), an inflammatory response that can cause flu-like symptoms, and neurological side effects. There is a risk of infection due to the impact on the immune system, requiring careful monitoring and management. As with any clinical trial, participants are closely monitored and can withdraw at any time.

Should I consider participating in NCT06492304?

Ask your doctor about the specific types of lymphoma or leukemia being studied and if your condition fits the trial criteria. Understand the potential risks and side effects of CTX131, including those related to the infusion and the CRISPR technology. Be prepared for regular hospital visits for infusions, blood tests, scans, and monitoring for side effects over an extended period. Always discuss clinical trial participation with your healthcare provider to determine if it is appropriate for your specific situation.

What does NCT06492304 signal from an investment perspective?

This Phase 2 study of CTX131, a CRISPR-engineered cell therapy, signals a significant investment in the rapidly growing field of cell and gene therapies for hematologic malignancies, with potential fo 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 the CTX131 infusion and regular check-ups to monitor for side effects and assess treatment 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.