Clinical trials investigating neoantigen-based immunotherapies
| Therapy type | Cancer type(s) | Trial identifier(s) | Therapy combinations | Key outcomes/endpoints |
|---|---|---|---|---|
| Neoantigen-directed cancer vaccines | ||||
| Dendritic cell (DC) vaccine | Melanoma | NCT00683670 | Induction of neoantigen-specific T cells | |
| Synthetic long peptide (SLP) vaccine | Melanoma | NCT01970358, NCT02035956 | No evidence of disease (NED) in some patients, recurrence in others; response to PD-1 therapy | |
| SLP vaccine (GAPVAC) | Glioblastoma | NCT02149225, NCT02287428 | Increased neoantigen-specific T cell response | |
| Ribonucleic acid (RNA) vaccine (IVAC MUTANOME) | Melanoma | NCT02035956 | Induction of neoantigen-specific T cells | |
| SLP vaccine (NEO-PV-01) | NSCLC | NCT03380871 | Anti-PD-1 & Chemotherapy | Induction of neoantigen-specific T cells |
| Adenoviral & self-amplifying messenger RNA (samRNA) vectors | Microsatellite-Stable colorectal cancer (MSS-CRC), gastroesophageal adenocarcinoma (GEAC), NSCLC | NCT03639714 | Anti-PD-1 | One complete response (CR) (GEAC); neoantigen-specific T cell induction |
| Neoantigen-directed adoptive cell transfer (ACT) | ||||
| Tumor-infiltrating lymphocyte adoptive cell transfer (TIL-ACT) | Cholangiocarcinoma | NCT01174121 | Anti-PD-1 | Durable response (~9 years) after retreatment with TIL-ACT |
| MSS-CRC | NCT01174121 | Regression of all metastases | ||
| Breast cancer | NCT01174121 | One CR (>5 years), partial response (PR) in others | ||
| T cell receptor-engineered adoptive cell transfer (TCR-ACT) | Pancreatic cancer | NCT04146298 | PR, metastasis regression at 6 months | |
| T cell receptor-engineered T cell therapy (TCR-T) | ||||
| TCR-T | Multiple solid tumors | NCT05194735, NCT05105815, NCT04625205, NCT03171220, NCT05020119 | Safety, overall response rate (ORR), disease-free survival (DFS) endpoints | |
| Various cancers | NCT04102436, NCT04596033, NCT02280811, NCT02858310 | Chemotherapy | Response rate, adverse events (AEs), dose-limiting toxicities (DLTs) | |
| Solid tumors | NCT05349890, NCT04520711, NCT03970382 | Immune checkpoint blockade (ICB) | Safety, tolerability, DLTs | |
| Multiple tumors | NCT03412877, NCT04536922 | ICB & Chemotherapy | Response rate, treatment effect | |
| Hepatocellular carcinoma | NCT03199807 | Radiotherapy | AEs | |
| Tumor-infiltrating lymphocytes (TILs) | ||||
| TIL therapy | Solid tumors | NCT05141474 | AEs, serious adverse events (SAEs), treatment-limiting toxicity (TLT) | |
| TILs | Gastrointestinal (GI) & pancreatic cancers | NCT04426669, NCT03658785, NCT02959905 | Chemotherapy | Maximum tolerated dose (MTD), ORR, AEs |
| TILs | Melanoma, NSCLC | NCT03997474, NCT04032847 | ICB | AEs |
| TILs | NSCLC, squamous cell carcinoma (SCC), adenosquamous carcinoma | NCT03215810 | ICB & chemotherapy | DLTs |
| Chimeric antigen receptor t cell therapy (CAR-T) | ||||
| CAR-T | Glioblastoma multiforme | NCT02844062 | Chemotherapy | Safety |
| Immune checkpoint blockade (ICB) therapy | ||||
| ICB monotherapy | Various cancers | NCT03600155, NCT02553642, NCT03827044, NCT03718767, NCT03925246, NCT03082534, NCT03357757, NCT03813394, NCT02437279, NCT04825990, NCT03130764, NCT03653052, NCT02113657, NCT03040791, NCT04019964, NCT04293419, NCT04262089 | Clinical response, safety, DFS, ORR, progression-free survival (PFS) | |
| ICB | Various cancers | NCT04214249, NCT03978624, NCT02990845, NCT02453620, NCT03409198, NCT05456165, NCT05201612, NCT03832621, NCT03186326, NCT04659382, NCT04262687, NCT05141721, NCT04014530, NCT03918499, NCT03655002, NCT03126812, NCT03554317, NCT04336943, NCT04068194, NCT05317000, NCT02883062 | Chemotherapy | Clinical response, MTD, AEs, PFS, ORR |
| ICB | Cutaneous T-cell lymphoma | NCT03385226 | Radiotherapy | ORR |
| ICB | Colorectal cancer, meningioma, rectal cancer | NCT03854799, NCT03604978, NCT04340401 | Chemotherapy & radiotherapy | Pathologic response, MTD, ORR, AEs |