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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Withdrawn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Withdrawn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Withdrawn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Withdrawn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Withdrawn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