A Safety and Efficacy Study Evaluating CTX131 in Adult Subjects With Relapsed/Refractory Hematologic Malignancies

NCT06492304 | Recruiting Phase 1 FDA Drug

• 1 other identifier: CRSP-ONC-008
• Study Type: interventional
• Target: 290
• Locations: 2 countries
• Sites: 7

Brief 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

Conditions

T Cell Lymphoma; B Cell Lymphoma; Acute Myeloid Leukemia

Keywords

CAR T; B Cell Lymphoma; T Cell Lymphoma; Allogeneic; Leukemia

Outcome Measures

Primary Outcomes (3)

• Phase 1 Part A (dose escalation) and Part B (dose optimization in selected disease types):

  For all cohorts: Incidence of Adverse events defined as dose-limiting toxicities

  From CTX131 infusion up to 28 days post-infusion

• Objective Response rate (ORR)

  Phase 2 (expansion of selected Phase 1 disease types)

  From CTX131 infusion up to 60 months post-infusion

• Composite Complete Remission (CRc)

  Phase 2 (expansion of selected Phase 1 disease types)

  From CTX131 infusion up to 60 months post-infusion

Study Arms (1)

CTX131

EXPERIMENTAL

Administered by IV infusion following lymphodepleting chemotherapy

Biological: CTX131

Interventions

CTX131 BIOLOGICAL

CTX131 (CD70-directed T-cell immunotherapy comprised of allogeneic T cells genetically modified ex vivo using CRISPR-Cas9 gene editing components

CTX131

Eligibility Criteria

• Age: 18 Years - 100 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• ≥18 years of age
• Eastern Cooperative Oncology Group (ECOG) performance status of 0-1 (ECOG status of 2 will be permitted for subjects with AML)
• 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.
• Adequate renal, liver, cardiac and pulmonary organ function
• 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

You may not qualify if:

• Prior treatment with anti-CD70 targeting agents
• Active CNS manifestation of underlying disease
• 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
• Uncontrolled bacterial, viral, or fungal infection
• Positive for HIV, or active hepatitis B virus or hepatitis C virus infection.
• 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.
• 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
• Primary immunodeficiency disorder or active autoimmune disease requiring steroids and/or other immunosuppressive therapy.
• 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
• Treatment with CD19-targeting CAR-T within 6 months prior to CTX131 infusion

Sponsors & Collaborators

• CRISPR Therapeutics: lead

Study Sites (7)

Research Site 6

Phoenix, Arizona, 85054, United States

RECRUITING

Research Site 5

Stanford, California, 94305, United States

RECRUITING

Research Site 3

Boston, Massachusetts, 02114, United States

RECRUITING

Research Site 4

New York, New York, 10065, United States

RECRUITING

Research Site 2

The Bronx, New York, 10467, United States

RECRUITING

Research Site 1

Houston, Texas, 77030, United States

RECRUITING

Research Site 7

East Melbourne, Victoria, 3002, Australia

RECRUITING

MeSH Terms

Conditions

Lymphoma, T-Cell; Lymphoma, B-Cell; Leukemia, Myeloid, Acute; Leukemia

Condition Hierarchy (Ancestors)

Lymphoma, Non-Hodgkin; Lymphoma; Neoplasms by Histologic Type; Neoplasms; Lymphoproliferative Disorders; Lymphatic Diseases; Hemic and Lymphatic Diseases; Immunoproliferative Disorders; Immune System Diseases; Leukemia, Myeloid; Hematologic Diseases

Study Officials

• Alissa Keegan, MD, PhD

  CRISPR Therapeutics

  STUDY DIRECTOR

Central Study Contacts

Clinical Trials

CONTACT

+1 877-214-4634; medicalaffairs@crisprtx.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 21, 2024
• First Posted: July 9, 2024
• Study Start: August 13, 2024
• Primary Completion (Estimated): May 1, 2030
• Study Completion (Estimated): November 1, 2030
• Last Updated: June 29, 2025

Record last verified: 2025-06

Data Sharing

• IPD Sharing: Will not share

Locations

US (6); AU (1)