Clinical trials investigating neoantigen-based immunotherapies

Therapy typeCancer type(s)Trial identifier(s)Therapy combinationsKey outcomes/endpoints
Neoantigen-directed cancer vaccines
Dendritic cell (DC) vaccineMelanomaNCT00683670Induction of neoantigen-specific T cells
Synthetic long peptide (SLP) vaccineMelanomaNCT01970358, NCT02035956No evidence of disease (NED) in some patients, recurrence in others; response to PD-1 therapy
SLP vaccine (GAPVAC)GlioblastomaNCT02149225, NCT02287428Increased neoantigen-specific T cell response
Ribonucleic acid (RNA) vaccine (IVAC MUTANOME)MelanomaNCT02035956Induction of neoantigen-specific T cells
SLP vaccine (NEO-PV-01)NSCLCNCT03380871Anti-PD-1 & ChemotherapyInduction of neoantigen-specific T cells
Adenoviral & self-amplifying messenger RNA (samRNA) vectorsMicrosatellite-Stable colorectal cancer (MSS-CRC), gastroesophageal adenocarcinoma (GEAC), NSCLCNCT03639714Anti-PD-1One complete response (CR) (GEAC); neoantigen-specific T cell induction
Neoantigen-directed adoptive cell transfer (ACT)
Tumor-infiltrating lymphocyte adoptive cell transfer (TIL-ACT)CholangiocarcinomaNCT01174121Anti-PD-1Durable response (~9 years) after retreatment with TIL-ACT
MSS-CRCNCT01174121Regression of all metastases
Breast cancerNCT01174121One CR (>5 years), partial response (PR) in others
T cell receptor-engineered adoptive cell transfer (TCR-ACT)Pancreatic cancerNCT04146298PR, metastasis regression at 6 months
T cell receptor-engineered T cell therapy (TCR-T)
TCR-TMultiple solid tumorsNCT05194735, NCT05105815, NCT04625205, NCT03171220, NCT05020119Safety, overall response rate (ORR), disease-free survival (DFS) endpoints
Various cancersNCT04102436, NCT04596033, NCT02280811, NCT02858310ChemotherapyResponse rate, adverse events (AEs), dose-limiting toxicities (DLTs)
Solid tumorsNCT05349890, NCT04520711, NCT03970382Immune checkpoint blockade (ICB)Safety, tolerability, DLTs
Multiple tumorsNCT03412877, NCT04536922ICB & ChemotherapyResponse rate, treatment effect
Hepatocellular carcinomaNCT03199807RadiotherapyAEs
Tumor-infiltrating lymphocytes (TILs)
TIL therapySolid tumorsNCT05141474AEs, serious adverse events (SAEs), treatment-limiting toxicity (TLT)
TILsGastrointestinal (GI) & pancreatic cancersNCT04426669, NCT03658785, NCT02959905ChemotherapyMaximum tolerated dose (MTD), ORR, AEs
TILsMelanoma, NSCLCNCT03997474, NCT04032847ICBAEs
TILsNSCLC, squamous cell carcinoma (SCC), adenosquamous carcinomaNCT03215810ICB & chemotherapyDLTs
Chimeric antigen receptor t cell therapy (CAR-T)
CAR-TGlioblastoma multiformeNCT02844062ChemotherapySafety
Immune checkpoint blockade (ICB) therapy
ICB monotherapyVarious cancersNCT03600155, NCT02553642, NCT03827044, NCT03718767, NCT03925246, NCT03082534, NCT03357757, NCT03813394, NCT02437279, NCT04825990, NCT03130764, NCT03653052, NCT02113657, NCT03040791, NCT04019964, NCT04293419, NCT04262089Clinical response, safety, DFS, ORR, progression-free survival (PFS)
ICBVarious cancersNCT04214249, NCT03978624, NCT02990845, NCT02453620, NCT03409198, NCT05456165, NCT05201612, NCT03832621, NCT03186326, NCT04659382, NCT04262687, NCT05141721, NCT04014530, NCT03918499, NCT03655002, NCT03126812, NCT03554317, NCT04336943, NCT04068194, NCT05317000, NCT02883062ChemotherapyClinical response, MTD, AEs, PFS, ORR
ICBCutaneous T-cell lymphomaNCT03385226RadiotherapyORR
ICBColorectal cancer, meningioma, rectal cancerNCT03854799, NCT03604978, NCT04340401Chemotherapy & radiotherapyPathologic response, MTD, ORR, AEs