Clinical Trial

Disease: Renal Cell Carcinoma, RCC, (NCT04438083)

Disease info:

Renal cell cancer (also called kidney cancer or renal cell adenocarcinoma) is a disease in which malignant (cancer) cells are found in the lining of tubules (very small tubes) in the kidney. There are 2 kidneys, one on each side of the backbone, above the waist. Tiny tubules in the kidneys filter and clean the blood. 

Cancer that starts in the ureters or the renal pelvis (the part of the kidney that collects urine and drains it to the ureters) is different from renal cell cancer. 

The prognosis and treatment options depend on the stage of the disease, and the patient's age and general health. After renal cell cancer has been diagnosed, tests are done to find out if cancer cells have spread within the kidney or to other parts of the body. The cancer may come back in the kidney or in other parts of the body after initial treatment.

There are different types of treatment for patients with renal cell cancer. Five types of standard treatment are used:

  • Surgery
  • Radiation therapy
  • Chemotherapy
  • Immunotherapy
  • Targeted therapy

New types of treatment are being tested in clinical trials. Patients can enter clinical trials before, during, or after starting their cancer treatment. Follow-up tests may be needed.

Renal cell carcinoma (RCC), affects more than 30,000 people annually and 40% of them die due to complications from this cancer. Kidney cancer is the ninth and fourteenth common cancer cases in men and women, respectively worldwide according to 2018 data.
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

Matthias Will, MD CRISPR Therapeutics


United States, California

United States, Connecticut

United States, Texas

United States, Utah

Australia, Victoria

Canada, Ontario

Netherlands, North Holland

Study start:
Jun. 16, 2020
107 participants
Gene editing method:
Type of edit:
Gene insertion, gene knock-out
anti-CD70 CAR, Major Histocompatibility Complex Class I (MHC-I) , T Cell Receptor (TCR)
Delivery method:
- Ex-vivo
Safety updates:

10-04-2021 CRISPR Therapeutics Presents Preclinical Data at AACR 2021 Supporting CD70 Knockout as a Novel Approach to Increasing CAR-T Cell Function

CTX130 allogeneic CAR-T cells targets CD70, an antigen expressed on both hematologic cancers, including certain lymphomas, and solid tumors, including renal cell carcinoma.
IND Enabling Pre-clinical
Phase I Safety
Phase II Safety and Dosing
Phase III Safety and Efficacy

Status: Active recruiting


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.

CTX130 CD70-directed T-cell immunotherapy comprised of off-the-shelf (allogeneic) T cells genetically modified ex vivo using CRISPR-Cas9 gene editing components.

CRISPR-Cas9 is used to make three modifications to healthy donor T cells.

  • Insert the CAR construct precisely into the T-cell Receptor (TCR) alpha constant (TRAC) locus.
  • TCR knock-out - eliminate the endogenous TCR with high efficiency, which reduces the risk of graft versus host disease occurring during off-the-shelf use.

  • MCH-I knock-out - eliminate the class I major histocompatibility complex (MHC I) expressed on the surface of our CAR-T product candidates. If present, MHC I could lead to rejection of the CAR-T product by the patient’s own T cells.

Last updated: Apr. 8, 2022
Source: US National Institutes of Health (NIH)
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