A Safety and Efficacy Study Evaluating CTX131 in Adult Subjects With Relapsed or Refractory Solid Tumors

Sponsor

CRISPR Therapeutics AG (Industry)

Overall Status

Recruiting

CT.gov ID

NCT05795595

Collaborator

(none)

250

Enrollment

Location

Arm

Anticipated Duration (Months)

2.9

Patients Per Site Per Month

Study Details

Study Description

Brief Summary

This is an open-label, multicenter, Phase 1/2 study evaluating the safety and efficacy of CTX131™ in subjects with relapsed or refractory solid tumors.

Condition or Disease	Intervention/Treatment	Phase
Clear Cell Renal Cell Carcinoma Cervical Carcinoma Esophageal Carcinoma Pancreatic Adenocarcinoma Malignant Pleural Mesothelioma	Biological: CTX131	Phase 1/Phase 2

Detailed Description

An open-label, multi-center Phase 1/2 study of CTX131 in subjects with relapsed/refractory solid tumors. CTX131 is an is allogeneic CD70- directed chimeric antigen receptor (CAR) T cell immunotherapy comprised of allogeneic T cells that are genetically modified ex vivo using CRISPR-Cas9 (clustered regularly interspaced short palindromic repeats/ CRISPR associated protein 9) gene editing components (single guide RNA and Cas9 nuclease).

Study Design

Study Type:

Interventional

Anticipated Enrollment :

250 participants

Allocation:

N/A

Intervention Model:

Sequential Assignment

Masking:

None (Open Label)

Primary Purpose:

Treatment

Official Title:

A Phase 1/2, Open-label, Multicenter, Dose Escalation and Cohort Expansion Study of the Safety and Efficacy of Anti-CD70 Allogeneic CRISPR-Cas9-Engineered T Cells (CTX131) in Adult Subjects With Relapsed or Refractory Solid Tumors

Anticipated Study Start Date :

Mar 1, 2023

Anticipated Primary Completion Date :

Apr 1, 2030

Anticipated Study Completion Date :

May 1, 2030

Arms and Interventions

Arm	Intervention/Treatment
Experimental: CTX131 Administered by IV infusion following lymphodepleting chemotherapy.	Biological: CTX131 CTX131 (CD70-directed T-cell immunotherapy comprised of allogeneic T cells genetically modified ex vivo using CRISPR-Cas9 gene editing components)

Arm

Intervention/Treatment

Experimental: CTX131

Administered by IV infusion following lymphodepleting chemotherapy.

Biological: CTX131

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

Outcome Measures

Primary Outcome Measures

Phase 1 (Dose Escalation): Incidence of adverse events [From CTX131 infusion up to 28 days post-infusion]
Defined as dose-limiting toxicities
Phase 2 (Cohort Expansion): Objective response rate (ORR) [From CTX131 infusion up to 60 months post-infusion]
ORR based on Independent Review Committee (IRC) assessment, defined as the proportion of subjects who have achieved a best overall response of CR or PR according to appropriate response evaluation criteria for each disease histology

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

Key Inclusion Criteria:

Age ≥18 years.
Unresectable or metastatic, relapsed/refractory, clear cell renal cell carcinoma, cervical carcinoma, pancreatic adenocarcinoma, endometrial carcinoma, and malignant pleural mesothelioma.
Eastern Cooperative Oncology Group performance status 0 or 1.
Adequate renal, liver, cardiac and pulmonary organ function.
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 CTX131 infusion.

Key Exclusion Criteria:

Prior treatment with anti-CD70 targeting agents
History of certain central nervous system (CNS), cardiac or pulmonary conditions.
Presence of uncontrolled bacterial, viral, or fungal infection.
Active HIV, hepatitis B virus or hepatitis C virus infection.
Primary immunodeficiency disorder or active autoimmune disease requiring steroids and/or other immunosuppressive therapy.
Women who are pregnant or breastfeeding.