A Safety and Efficacy Study Evaluating CTX130 in Subjects With Relapsed or Refractory Renal Cell Carcinoma (COBALT-RCC)

A Phase 1 Dose Escalation and Cohort Expansion Study of the Safety and Efficacy of Allogeneic CRISPR-Cas9-Engineered T Cells (CTX130) in Subjects With Advanced, Relapsed or Refractory Renal Cell Carcinoma With Clear Cell Differentiation

This is a single-arm, open-label, multicenter, Phase 1 study evaluating the safety and efficacy of CTX130 in subjects with relapsed or refractory renal cell carcinoma.

Study Overview

• Status: Terminated
• Conditions: Renal Cell Carcinoma
• Intervention / Treatment: Biological: CTX130

Detailed Description

The study may enroll approximately 107subjects in total.

• Study Type: Interventional
• Enrollment (Actual): 19
• Phase: Phase 1

Contacts and Locations

Study Locations

• Australia
  • Victoria
    • Melbourne, Victoria, Australia, 3000
      • Research Site 1
• Canada
  • Ontario
    • Toronto, Ontario, Canada, M5G 2M9
      • Research Site 6
• Netherlands
  • North Holland
    • Amsterdam, North Holland, Netherlands, 1066
      • Research Site 7
• United States
  • California
    • Duarte, California, United States, 91010
      • Research Site 2
  • Connecticut
    • Hartford, Connecticut, United States, 06520
      • Research Site 5
  • Texas
    • Houston, Texas, United States, 77030
      • Research Site 4
  • Utah
    • Salt Lake City, Utah, United States, 84112
      • Research Site 3

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Abbreviated Inclusion Criteria:

1. Age ≥18 years and body weight ≥42 kg.
2. Unresectable or metastatic RCC that has exploited standard of care treatment.
3. Karnofsky performance status (KPS) ≥80%.
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 CTX130 infusion.

Abbreviated Exclusion Criteria:

1. Prior treatment with any anti-CD70 targeting agents.
2. Prior treatment with any CAR T cells or any other modified T or natural killer (NK) cells.
3. History of certain central nervous system (CNS), cardiac or pulmonary conditions.
4. Active HIV, hepatitis B virus or hepatitis C virus infection.
5. Previous or concurrent malignancy, except treated with curative approach not requiring systemic therapy and in remission for >12 months, or any other localized malignancy with low risk of developing into metastatic disease.
6. Primary immunodeficiency disorder or active autoimmune disease requiring steroids and/or other immunosuppressive therapy.
7. Prior solid organ transplantation or bone marrow transplant.
8. Pregnant or breastfeeding females.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Sequential Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: CTX130; Administered by IV infusion following lymphodepleting chemotherapy.	Biological: CTX130; CTX130 CD70-directed T-cell immunotherapy comprised of allogeneic T cells genetically modified ex vivo using CRISPR-Cas9 gene editing components.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Part A (dose escalation): Incidence of adverse events	Adverse events defined as dose-limiting toxicities	From CTX130 infusion up to 28 days post-infusion
Part B (cohort expansion): Objective response rate	Objective response rate per Response Evaluation Criteria In Solid Tumors (RECIST) v1.1	From CTX130 infusion up to 60 months post-infusion]

Secondary Outcome Measures

Outcome Measure	Time Frame
Progression Free Survival	From date of CTX130 infusion until date of disease progression or death due to any cause, assessed up to 60 months
Overall Survival	From date of CTX130 until date of death due to any cause, assessed up to 60 months

Collaborators and Investigators

• Sponsor: CRISPR Therapeutics AG
• Investigators: Study Director: Alissa Keegan, MD, CRISPR Therapeutics

Study record dates

Study Major Dates

• Study Start (Actual): June 16, 2020
• Primary Completion (Actual): October 8, 2024
• Study Completion (Actual): October 8, 2024

Study Registration Dates

• First Submitted: June 16, 2020
• First Submitted That Met QC Criteria: June 16, 2020
• First Posted (Actual): June 18, 2020

Study Record Updates

• Last Update Posted (Actual): March 25, 2025
• Last Update Submitted That Met QC Criteria: January 7, 2025
• Last Verified: January 1, 2025

More Information

Terms related to this study

• Keywords: Allogeneic; Renal cell carcinoma; CAR T; CRISPR-Cas9; Renal cell carcinoma with clear cell differentiation
• Additional Relevant MeSH Terms: Urogenital Diseases; Urogenital Neoplasms; Neoplasms by Site; Neoplasms; Male Urogenital Diseases; Kidney Diseases; Urologic Diseases; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Neoplasms by Histologic Type; Neoplasms, Glandular and Epithelial; Adenocarcinoma; Urologic Neoplasms; Kidney Neoplasms; Carcinoma; Carcinoma, Renal Cell
• Other Study ID Numbers: CRSP-ONC-003

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No