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

Sponsor

CRISPR Therapeutics AG (Industry)

Overall Status

Recruiting

CT.gov ID

NCT04438083

Collaborator

(none)

107

Enrollment

Locations

Arm

81.5

Anticipated Duration (Months)

15.3

Patients Per Site

0.2

Patients Per Site Per Month

Study Details

Study Description

Brief Summary

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.

Condition or Disease	Intervention/Treatment	Phase
Renal Cell Carcinoma	Biological: CTX130	Phase 1

Detailed Description

The study may enroll approximately 107subjects in total.

Study Design

Study Type:

Interventional

Anticipated Enrollment :

107 participants

Allocation:

N/A

Intervention Model:

Sequential Assignment

Masking:

None (Open Label)

Primary Purpose:

Treatment

Official Title:

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

Actual Study Start Date :

Jun 16, 2020

Anticipated Primary Completion Date :

Feb 1, 2027

Anticipated Study Completion Date :

Apr 1, 2027

Arms and Interventions

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.

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.

Outcome Measures

Primary Outcome Measures

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

Secondary Outcome Measures

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]

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

Abbreviated Inclusion Criteria:

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

Abbreviated Exclusion Criteria:

Prior treatment with any anti-CD70 targeting agents.
Prior treatment with any CAR T cells or any other modified T or natural killer (NK) cells.
History of certain central nervous system (CNS), cardiac or pulmonary conditions.
Active HIV, hepatitis B virus or hepatitis C virus infection.
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.
Primary immunodeficiency disorder or active autoimmune disease requiring steroids and/or other immunosuppressive therapy.
Prior solid organ transplantation or bone marrow transplant.
Pregnant or breastfeeding females.

Contacts and Locations

Locations

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