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Table 1 IDO1 inhibitors in clinical trials

From: Indoleamine 2,3-dioxygenase 1 (IDO1) inhibitors in clinical trials for cancer immunotherapy

Drug Strategy Tumor type Phase Clinical efficacy Safety Trial number Status
Indoximod Single agent Healthy Phase I Unknown Unknown NCT03372239 Completed
Healthy Early phase I Unknown Unknown NCT03852446 Completed
Progressive brain tumors or newly diagnosed DIPG Phase II Unknown Unknown NCT04049669 Recruiting
Pembrolizumab/nivolumab Advanced or metastatic melanoma Phase I/II Unknown Unknown NCT02073123 Completed
Temozolomide Refractory metastatic prostate cancer Phase II Stable disease (SD) 50% Constipation, diarrhea, fatigue, pain, NCT01560923 Completed
Adenovirus-p53 transduced dendritic cell (DC) vaccine Breast cancer Not applicable Unknown Unknown NCT01302821 Withdrawn
Epacadostat Single agent Epithelial ovarian, fallopian tube or primary peritoneal carcinoma Early phase I Unknown Unknown NCT02042430 Active, not recruiting
Locally advanced rectal cancer Phase I Unknown Unknown NCT03516708 Recruiting
Pembrolizumab Gastrointestinal stromal tumors Phase II Unknown Unknown NCT03291054 Active, not recruiting
Head and neck cancer Phase II Unknown Unknown NCT03238638 Withdrawn
Squamous cell carcinoma of the head and neck Phase II Unknown Unknown NCT03325465 Withdrawn
Head and neck cancer patients, who failed prior PD-1/PD-L1 therapy Phase II Unknown Unknown NCT03463161 Terminated
Advanced pancreatic cancer Phase II Unknown Unknown NCT03432676 Withdrawn
Recurrent Clear cell carcinoma of the ovary Phase II Unknown Unknown NCT03602586 Suspended
Non-metastatic esophageal/gastroesophageal squamous cell and adenocarcinomas treated with neoadjuvant chemoradiation Phase II Unknown Unknown NCT03592407 Withdrawn
Recurrent/metastatic endometrial carcinoma Phase II Unknown Unknown NCT03310567 Pembrolizumab
Lung cancer Phase II Unknown Unknown NCT03322540 Active, not recruiting
Urothelial cancer (UC) Phase III Unknown Unknown NCT03374488 Active, not recruiting
Renal cell carcinoma (RCC) Phase III PR: 29.7%, CR + PR: 31.3%, [63] Anaemia, diarrhoea, hypothyroidism, nausea, fatigue, pruritus NCT03260894 Recruiting
Muscle-invasive bladder cancer Phase II Unknown Unknown NCT03832673 Not yet recruiting
Head and neck cancer Phase III Unknown Hypothyroidism, vomiting, asthenia NCT03358472 Active, not recruiting
Extensive stage small cell lung carcinoma Phase II Unknown Unknown NCT03402880 Withdrawn
Pembrolizumab/azacitidine Metastatic colorectal cancer Phase I/II Unknown Unknown NCT03182894 Withdrawn
Pembrolizumab/chemotherapy Advanced solid tumors Phase I Unknown Unknown NCT02862457 Active, not recruiting
Lung cancer Phase II Unknown Nausea NCT03322566 Active, not recruiting
INCB001158/pembrolizumab Solid tumors Phase I/II Unknown Unknown NCT03361228 Terminated
Electroporation/pembrolizumab Squamous cell carcinoma of the head and neck Phase II Unknown Unknown NCT03823131 Recruiting
Nivolumab/Ipilimumab Advanced or metastatic malignancies Phase I/II Unknown Unknown NCT03347123 Active, not recruiting
Nivolumab/chemotherapy Lung cancer Phase III Unknown Unknown NCT03348904 Terminated
Nivolumab/chemotherapy Phase III Unknown Unknown NCT03342352 Withdrawn
Durvalumab Unresectable, recurrent, and metastatic EBV + NPC Phase II Unknown Unknown NCT04231864 Not yet recruiting
Cladribine/cytarabine Relapsed/refractory AML patients Phase I Unknown Unknown NCT03491579 Withdrawn
Idarubicin/cytarabine/Daunorubicin Phase 1 Phase I Unknown Unknown NCT03444649 Withdrawn
Rapamycin Advanced malignancy Phase I Unknown Unknown NCT03217669 Recruiting
INCMGA00012 + RT + bevacizumab Recurrent gliomas Phase II Unknown Unknown NCT03532295 Recruiting
M7824 + BN-Brachyury + ALT-803 + Epacadostat (Immunotherapy) Solid tumor Phase I/II Unknown Unknown NCT03493945 Recruiting
INCMGA00012, Epacadostat 600 mg BID, SV-BR-1-GM combination Metastatic or locally recurrent breast cancer patients Phase I/II Unknown Unknown NCT03328026 Recruiting
Intralesional SD101, Radiotherapy Advanced solid tumors lymphoma Phase I/II Unknown Unknown NCT03322384 Recruiting
Itacitinib/INCB050465 Solid tumors Phase I Unknown Unknown NCT02559492 Terminated
BMS-986205 Single agent Healthy volunteers Phase I Unknown Unknown NCT03378310 Completed
NCT03374228
NCT03312426
NCT03362411
NCT03247283
Nivolumab Advanced malignant solid tumors Phase I/ II Unknown Unknown NCT03792750 Active, not recruiting
Recurrent or persistent endometrial carcinoma or endometrial carcinosarcoma Phase II Unknown Unknown NCT04106414 Recruiting
Squamous cell carcinoma of the head and neck Phase II Unknown Unknown NCT03854032 Recruiting
Resectable stage III or IV melanoma Phase II Unknown Unknown NCT04007588 Withdrawn
Relatlimab/nivolumab Advanced malignant tumors Phase I/ II Unknown Unknown NCT03459222 Recruiting
Nivolumab/BCG BCG-unresponsive, high-risk, non-muscle invasive bladder cancer Phase II Unknown Unknown NCT03519256 Recruiting
Itraconazole/rifampin Malignancies multiple Phase I Unknown Unknown NCT03346837 Completed
Nivolumab/chemotherapy Muscle-invasive bladder cancer Phase III Unknown [64]   NCT03661320 Recruiting
Nivolumab/radiotherapy or chemoradiotherapy Glioblastoma Phase I Unknown Unknown NCT04047706 Recruiting
Omeprazole Healthy Phase I Unknown Unknown NCT03936374 Completed
Navoximod (GDC-0919, NLG-919) Single agent Recurrent advanced solid tumors Phase I 8 (36%) had stable disease and 10 (46%) had progressive disease [65, 66] Fatigue (59%), cough, decreased appetite, and pruritus (41% each), nausea (36%), and vomiting (27%). Grade ≥ 3 AEs occurred in 14/22 patients (64%) NCT02048709 Completed
PF-06840003 Single agent Malignant glioma Phase I Disease control occurred in eight patients (47%). Mean duration of stable disease (SD) was 32.1 (12.1–72.3) weeks [67, 68] Grade 4 alanine and aspartate aminotransferase elevations NCT02764151 Terminated
KHK2455 Avelumab Urothelial carcinoma Phase I Unknown Unknown NCT03915405 Recruiting
Mogamulizumab Locally advanced or metastatic solid tumors Phase I Unknown Unknown NCT02867007 Active, not recruiting
LY3381916 Single agent or in combination with anti-programmed cell death ligand 1 (PD-L1) checkpoint antibody (LY3300054) Solid tumor, non-small cell lung cancer, renal cell carcinoma, triple negative breast cancer Phase I Unknown Unknown NCT03343613 Terminated