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. 2023 Feb 28:1–19. Online ahead of print. doi: 10.1038/s41417-023-00597-z

Table 2.

Clinical trials on CRISPR/Cas-mediated cancer therapy (https://www.clinicaltrials.gov).

Study title Status Conditions Interventions Gender Age Phases Clinical trial identifier
Study of CRISPR-Cas9 Mediated PD-1 and TCR Gene-knocked Out Mesothelin-directed CAR-T Cells in Patients with Mesothelin Positive Multiple Solid Tumors. Recruiting Solid Tumor Biological: anti-mesothelin CAR-T cells All 18–70 Phase 1 NCT03545815
A Study of Metastatic Gastrointestinal Cancers Treated with Tumor Infiltrating Lymphocytes in Which the Gene Encoding the Intracellular Immune Checkpoint CISH Is Inhibited Using CRISPR Genetic Engineering Recruiting Gastrointestinal Cancer Drug: Cyclophosphamide|Drug: Fludarabine| Biological: Tumor-Infiltrating Lymphocytes (TIL)|Drug: Aldesleukin All 18–70 Phase1 and Phase 2 NCT04426669
A Safety and Efficacy Study of TALEN and CRISPR/Cas9 in the Treatment of HPV-related Cervical Intraepithelial Neoplasia Unknown status Human Papillomavirus-Related Malignant Neoplasm Biological: TALEN|Biological: CRISPR/Cas9 Female 18–50 Phase 1 NCT03057912
Study of PD-1 Gene-knocked Out Mesothelin-directed CAR-T Cells with the Conditioning of PC in Mesothelin Positive Multiple Solid Tumors Unknown status Solid Tumor Biological: Mesothelin-directed CAR-T cells All 18–70 Phase 1 NCT03747965
A Safety and Efficacy Study Evaluating CTX110 in Subjects With Relapsed or Refractory B-Cell Malignancies (CARBON) Recruiting B-cell Malignancy|Non-Hodgkin Lymphoma|B-cell Lymphoma|Adult B Cell ALL Biological: CTX110 All 18< Phase 1 NCT04035434
PD-1 Knockout Engineered T Cells for Advanced Esophageal Cancer Completed Esophageal Cancer Other: PD-1 Knockout T Cells All 18–80 Not Applicable NCT03081715
PD-1 Knockout Engineered T Cells for Metastatic Non-small Cell Lung Cancer Completed Metastatic Non-small Cell Lung Cancer Drug: Cyclophosphamide|Other: PD-1 Knockout T Cells All 18–70 Phase 1 NCT02793856
Stem Cells in NF1 Patients With Tumors of the Central Nervous System Suspended Neurofibromatosis Type 1|Tumors of the Central Nervous System Diagnostic Test: Collection of Stem Cells All Child, Adult, Older Adult NCT03332030
A Safety and Efficacy Study Evaluating CTX130 in Subjects With Relapsed or Refractory T or B Cell Malignancies (COBALT-LYM) Recruiting T Cell Lymphoma Biological: CTX130 All 18< Phase 1 NCT04502446
First-time-in-human (FTIH) Study of GSK3145095 Alone and in Combination With Other Anticancer Agents in Adults With Advanced Solid Tumors Terminated Neoplasms, Pancreatic Drug: GSK3145095|Drug: Pembrolizumab All 18< Phase 2 NCT03681951
Lavage of the Uterine Cavity for Diagnosis of Ovarian Cancer Recruiting Ovarian Cancer Other: Biospecimen Collection|Other: Laboratory Biomarker Analysis|Device: Lavage|Other: Pap Smear Female 18< Not Applicable NCT03606486
TGF²R-KO CAR-EGFR T Cells in Previously Treated Advanced EGFR-positive Solid Tumors Recruiting Solid Tumor| EGFR Overexpression Biological: TGF²R-KO CAR-EGFR T Cells All 18–75 Phase 1 NCT04976218
PD-1 Knockout Engineered T Cells for Castration Resistant Prostate Cancer Withdrawn Hormone Refractory Prostate Cancer Biological: PD-1 Knockout T Cells|Drug: Cyclophosphamide|Drug: IL-2 Male 45–85 NCT02867345
PD-1 Knockout Engineered T Cells for Muscle-invasive Bladder Cancer Withdrawn Invasive Bladder Cancer Stage IV Biological: PD-1 Knockout T Cells|Drug: Cyclophosphamide|Drug: IL-2 All 18–75 Phase 1 NCT02863913
CRISPR (HPK1) Edited CD19-specific CAR-T Cells (XYF19 CAR-T Cells) for CD19 + Leukemia or Lymphoma. Recruiting Leukemia Lymphocytic Acute (ALL) in Relapse|Leukemia Lymphocytic Acute (All) Refractory|Lymphoma, B-Cell|CD19 Positive Genetic: XYF19 CAR-T cell|Drug: Cyclophosphamide|Drug: Fludarabine All 18–55 Phase 1 NCT04037566
Safety and Efficacy of CT125A Cells for Treatment of Relapsed/Refractory CD5 + Hematopoietic Malignancies Not yet recruiting CD5+ Relapsed/Refractory Hematopoietic Malignancies|Chronic Lymphocytic Leukemia (CLL)|Mantle Cell Lymphoma (MCL)|Diffuse Large B-cell Lymphoma (DLBCL)|Follicular Lymphoma (FL)|Peripheral T-cell Lymphomas (PTCL) Biological: CT125A cells|Drug: Cyclophosphamide, fludarabine All 18–70 Early Phase 1 NCT04767308
A Safety and Efficacy Study Evaluating CTX120 in Subjects With Relapsed or Refractory Multiple Myeloma Recruiting Multiple Myeloma Biological: CTX120 All 18< Phase 1 NCT04244656
Study of Base Edited CAR7 T Cells to Treat T Cell Malignancies (TvT CAR7) Recruiting Relapsed/Refractory T-cell Acute Lymphoid Leukemia Biological: Cryopreserved BE CAR7 T cells (BE752TBCCLCAR7PBL) All 6–16 Phase 1 NCT05397184
PD-1 Knockout EBV-CTLs for Advanced Stage Epstein-Barr Virus (EBV) Associated Malignancies Unknown status Stage IV Gastric Carcinoma|Stage IV Nasopharyngeal Carcinoma|T-Cell Lymphoma Stage IV|Stage IV Adult Hodgkin Lymphoma|Stage IV Diffuse Large B-Cell Lymphoma Drug: Fludarabine|Drug: Cyclophosphamide|Drug: Interleukin-2 All 18–75 Phase 1|Phase 2 NCT03044743
Study Investigating NTLA-5001 in Subjects With Acute Myeloid Leukemia Recruiting Acute Myeloid Leukemia Genetic: Arm 1: NTLA-5001|Genetic: Arm 2: NTLA-5001 All 18< Phase 1|Phase 2 NCT05066165
A Study Evaluating UCART019 in Patients With Relapsed or Refractory CD19+ Leukemia and Lymphoma Unknown status B Cell Leukemia|B Cell Lymphoma Biological: UCART019 All 12–75 Phase 1|Phase 2 NCT03166878
A Long-term Follow-up Study of Patients Who Received VOR33 Recruiting Leukemia, Myeloid, Acute Genetic: VOR33 All Child, Adult, Older Adult NCT05309733
NY-ESO-1-redirected CRISPR (TCRendo and PD1) Edited T Cells (NYCE T Cells) Terminated Multiple Myeloma |Melanoma| Synovial Sarcoma |Myxoid/Round Cell Liposarcoma Biological: NY-ESO-1 redirected autologous T cells with CRISPR edited endogenous TCR and PD-1| Drug: Cyclophosphamide|Drug: Fludarabine|Device: NY-ESO-1 expression testing All 18< Phase 1 NCT03399448
A Safety and Efficacy Study Evaluating CTX130 in Subjects With Relapsed or Refractory Renal Cell Carcinoma (COBALT-RCC) Recruiting Renal Cell Carcinoma Biological: CTX130 All 18< Phase 1 NCT04438083
CRISPR-Edited Allogeneic Anti-CD19 CAR-T Cell Therapy for Relapsed/Refractory B Cell Non-Hodgkin Lymphoma Recruiting Lymphoma Genetic: CB-010|Drug: Cyclophosphamide|Drug: Fludarabine All 18< Phase 1 NCT04637763
Programmed Allogeneic CRISPR-edited T Cells Engineered to Express Anti-CD19 Chimeric Antigen Receptor (PACE CART19) in Patients With Relapsed Or Refractory CD19 + Leukemia and Lymphoma Withdrawn Acute Lymphoblastic Leukemia |Chronic Lymphocytic Leukemia| Non-Hodgkin Lymphoma Biological: PACE CART19 All 18< Phase 1 NCT05037669
TT52CAR19 Therapy for B-cell Acute Lymphoblastic Leukemia (B-ALL) Recruiting B Acute Lymphoblastic Leukemia Drug: PBLTT52CAR19 All 6 Months to 18 Years Phase 1 NCT04557436
A Feasibility and Safety Study of Universal Dual Specificity CD19 and CD20 or CD22 CAR-T Cell Immunotherapy for Relapsed or Refractory Leukemia and Lymphoma Unknown status B Cell Leukemia Cell Lymphoma Biological: Universal Dual Specificity CD19 and CD20 or CD22 CAR-T Cells All 12–70 Phase 1|Phase 2 NCT03398967
TACE Combined With PD-1 Knockout Engineered T Cell in Advanced Hepatocellular Carcinoma. Unknown status Advanced Hepatocellular Carcinoma Procedure: Transcatheter arterial chemoembolization Biological: PD-1 knockout engineered T cells All 18–70 Phase 1 NCT04417764
PD-1 Knockout Engineered T Cells for Metastatic Renal Cell Carcinoma. Withdrawn Metastatic Renal Cell Carcinoma Biological: PD-1 Knockout T Cells Drug: Cyclophosphamide Drug: IL-2 All 18–75 Phase 1 NCT02867332