Trial Review

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been endorsed by the ANZCTR. Before participating in a study, talk to your health care provider and refer to this information for consumers
Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT06492304




Registration number
NCT06492304
Ethics application status
Date submitted
21/06/2024
Date registered
9/07/2024
Date last updated
29/06/2025

Titles & IDs
Public title
A Safety and Efficacy Study Evaluating CTX131 in Adult Subjects With Relapsed/Refractory Hematologic Malignancies
Scientific title
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
Secondary ID [1] 0 0
CRSP-ONC-008
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
T Cell Lymphoma 0 0
B Cell Lymphoma 0 0
Acute Myeloid Leukemia 0 0
Condition category
Condition code
Cancer 0 0 0 0
Lymphoma (non Hodgkin's lymphoma) - High grade lymphoma
Cancer 0 0 0 0
Lymphoma (non Hodgkin's lymphoma) - Low grade lymphoma
Cancer 0 0 0 0
Leukaemia - Acute leukaemia
Cancer 0 0 0 0
Leukaemia - Chronic leukaemia

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Other - CTX131

Experimental: CTX131 - Administered by IV infusion following lymphodepleting chemotherapy


Treatment: Other: CTX131
CTX131 (CD70-directed T-cell immunotherapy comprised of allogeneic T cells genetically modified ex vivo using CRISPR-Cas9 gene editing components
Intervention code [1] 0 0
Treatment: Other
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Phase 1 Part A (dose escalation) and Part B (dose optimization in selected disease types):
Timepoint [1] 0 0
From CTX131 infusion up to 28 days post-infusion
Primary outcome [2] 0 0
Objective Response rate (ORR)
Timepoint [2] 0 0
From CTX131 infusion up to 60 months post-infusion
Primary outcome [3] 0 0
Composite Complete Remission (CRc)
Timepoint [3] 0 0
From CTX131 infusion up to 60 months post-infusion

Eligibility
Key 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
Minimum age
18 Years
Maximum age
100 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key 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

Study design
Purpose of the study
Treatment
Allocation to intervention
Not applicable
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Other
Other design features
Phase
Phase 1
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
13/08/2024
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/11/2030
Actual
Sample size
Target
290
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
VIC
Recruitment hospital [1] 0 0
Research Site 7 - East Melbourne
Recruitment postcode(s) [1] 0 0
3002 - East Melbourne
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Arizona
Country [2] 0 0
United States of America
State/province [2] 0 0
California
Country [3] 0 0
United States of America
State/province [3] 0 0
Massachusetts
Country [4] 0 0
United States of America
State/province [4] 0 0
New York
Country [5] 0 0
United States of America
State/province [5] 0 0
Texas

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
CRISPR Therapeutics
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Alissa Keegan, MD, PhD
Address 0 0
CRISPR Therapeutics
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Clinical Trials
Address 0 0
Country 0 0
Phone 0 0
+1 877-214-4634
Fax 0 0
Email 0 0
[email protected]
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
No/undecided IPD sharing reason/comment


What supporting documents are/will be available?

No Supporting Document Provided


Results publications and other study-related documents

No documents have been uploaded by study researchers.

Results not provided in https://clinicaltrials.gov/study/NCT06492304