Clinical Trial

Disease: Relapsed or Refractory 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.

Relapsed refers to when a patient has received active treatment, went off treatment and then the disease came back, whereas refractory refers to disease that is progressing despite active treatment.

The American Cancer Society estimates about 81,800 new cases of kidney cancer will be diagnosed in the United States in 2023, and approximately 14,890 will die from the disease.
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

Study Director: Alissa Keegan, MDCRISPR Therapeutics


United States, California

Research Site 2, Duarte, California, United States, 91010


United States, Connecticut

Research Site 5, Hartford, Connecticut, United States, 06520


United States, Texas

Research Site 4, Houston, Texas, United States, 77030


United States, Utah

Research Site 3, Salt Lake City, Utah, United States, 84112


Australia, Victoria

Research Site 1, Melbourne, Victoria, Australia, 3000


Canada, Ontario

Research Site 6, Toronto, Ontario, Canada, M5G 2M9



Research Site 7, Amsterdam, North Holland, Netherlands, 1066

Study start:
Jun. 16, 2020
107 participants
Gene editing method:
Type of edit:
Gene disruption
T-cell receptor α constant (TRAC), β2 microglobulin, and CD70 loci
Delivery method:
Adeno-associated virus vector - Ex-vivo
Safety updates:
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 not 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. 11, 2024
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