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Table 1 IDO1 inhibitors in clinical trials

From: Targeting the IDO1 pathway in cancer: from bench to bedside

Drug

Strategies

Tumor type

Phase

Clinical efficacy

Safety (% patients)

Trial number

Status

Indoximod

Single agent

Metastatic or refractory solid tumors

I

NR

NR

NCT00739609

Terminated

Metastatic or refractory solid tumors

I

ORR 10% (5/48)

Fatigue (56.3%), anemia (37.5%), anorexia (37.5%) dyspnea (35.4%) cough (33.3%) nausea (29.2%)

NCT00567931

Completed

Docetaxel

Metastatic solid tumors

I

4/22PRs, 9/22 SD, and 9/22 PD

Fatigue (58.6%) anemia (51.7%) hyperglycemia (48.3%), infection (44.8%), nausea (41.4%)

NCT01191216

Completed

Temozolomide/bevacizumab

Primary malignant brain tumors

I/II

NR

NR

NCT02052648

Recruiting

Temozolomide

Progressive primary malignant brain tumors

I

  

NCT02502708

Recruiting

Docetaxel/paclitaxel

Metastatic breast cancer

II

  

NCT01792050

Unknown

Nab-Paclitaxel/gemcitabine

Metastatic pancreatic cancer

I/II

ORR 11/30 (37%)

1/30 (colitis)

NCT02077881

Recruiting

Idarubicin/cytarabine

Acute myeloid leukemia

I/II

  

NCT02835729

Recruiting

Adenovirus-p53 transduced dendritic cell (DC) vaccine

Metastatic breast cancer

I/II

Chemosensitization effect, median PFS 13.3 weeks and median OS 20.71 weeks. 9/22 patients benefitted from chemotherapy after vaccination.

Most common grade 1–2 (fatigue, anemia, transient lymphopenia, nausea, anorexia)

NCT01042535

Completed

Sipuleucel-T

Refractory metastatic prostate cancer

II

NR

NR

NCT01560923

Active, not recruiting

Tergenpumatucel-L/docetaxel

Advanced previously treated non-small cell lung cancer

I/II

  

NCT02460367

Unknown

Ipilimumab/nivolumab/pembrolizumab

Metastatic melanoma

II/ III

  

NCT03301636

Recruiting

Epacadostat

Single agent

Advanced malignancy

I/II

NR

Fatigue, nausea, decreased appetite, vomiting, constipation, abdominal pain, diarrhea, dyspnea, back pain, cough

NCT01195311

Completed

Solid tumor

I

  

NCT03471286

Not yet recruiting

Myelodysplastic syndromes

II

  

NCT01822691

Completed

Fludarabine/cyclophosphamide/NK cells/IL-2

Recurrent ovarian, fallopian tube, and primary peritoneal cancer

I

  

NCT02118285

Completed

Tamoxifen

Ovarian cancer genitourinary tumors

II

Median PFS, 3.75 months

Fatigue (36.4%)

rash (18.2%)

pruritus (9.1%)

NCT01685255

Terminated

Azacitidine + pembrolizumab

Advanced solid tumors

I/II

NR

NR

NCT02959437

Recruiting

MELITAC 12.1 peptide vaccine

Stage III-IV melanoma

II

NR

NR

NCT01961115

Active, not recruiting

DPX-survivac/cyclophosphamide

Ovarian cancer

I

NR

NR

NCT02785250

Recruiting

ALVAC(2)-NY-ESO-1 (M)/TRICOM vaccine

Ovarian, fallopian tube, or primary peritoneal cancer

I/II

NR

NR

NCT01982487

Withdrawn

DEC-205/NY-ESO-1 fusion protein CDX-1401/poly ICLC

Ovarian, fallopian tube, or primary peritoneal cancer

I/II

NR

NR

NCT02166905

Recruiting

CRS-207/pembrolizumab

Metastatic pancreas cancer, platinum-resistant ovarian, fallopian, or peritoneal cancer

II,I/II

NR

NR

NCT03006302

Recruiting active, not recruiting

NCT02575807

Atezolizumab

Non-small cell lung cancer and urothelial carcinoma

I

NR

NR

NCT02298153

Terminated

Durvalumab

Advanced solid tumor

I/II

NR

NR

NCT02318277

Recruiting

Ipilimumab

Melanoma

I/II

  

NCT01604889

Terminated

Nivolumab/ipilimumab/lirilumab

Solid tumors

I/II

  

NCT03347123

Recruiting

Nivolumab/chemotherapy

Advanced cancers

I/II

ORR 75%(melanoma) 11% (ovarian) 4% (colorectal)

Rash (10% and 12% in epacadostat 100 and 300 mg subgroups)

NCT02327078

Recruiting

Pembrolizumab/chemotherapy

Advanced solid tumors

I, I/II

NR

NR

NCT02862457

Recruiting

NCT03085914

Recruiting

pembrolizumab

Solid tumors, thymic carcinoma, sarcoma, junction or gastric cancer, lung cancer, urothelial cancer, metastatic melanoma, and others.

I, I/II, III

Melanoma (ORR 57% and DCR 86%), Renal cell carcinoma (ORR 40% and DCR 80%)

Fatigue, diarrhea, rash, arthralgia, and nausea

NCT02178722

Recruiting

NCT02364076

Recruiting

NCT03414229

Recruiting

NCT03196232

Recruiting

NCT03322540

Recruiting

NCT03361865

Recruiting

 

Pembrolizumab

Melanoma

III

  

NCT02752074

Halted

Navoximod

Single agent

Advanced solid tumors

I

NR

NR

NCT02048709

Completed

Atezolizumab

Solid tumors

I

  

NCT02471846

Active, not recruiting

PF-06840003

Single agent

Malignant gliomas

I

  

NCT02764151

Active, not recruiting

BMS-986205

Nivolumab

Melanoma advanced cancers

III, I/II

NR

3/42 patients with grade 3 autoimmune hepatitis

NCT03329846

Recruiting

NCT03192943

Recruiting

Nivolumab/ipilimumab

Advanced cancer melanoma non-small cell lung cancer

I/II

ORR 32%(bladder cancer)

14%(cervical cancer)

PD-L1 > 1%: 46% (bladder cancer)and 25% (cervical cancer)

Fatigue (18.2% nausea (18.2%) decreased appetite (13.6%) vomiting (6.8%)

NCT02658890

Recruiting

Nivolumab/ipilimumab/relatlimab

Advanced gastric cancer, advanced renal cell carcinoma, advanced cancer

II, I/II

NR

NR

NCT02935634

Recruiting

NCT02996110

Recruiting

NCT03459222

Not yet recruiting

NCT03335540

Recruiting

Nivolumab/cetuximab/chemotherapy

Head and neck cancer

III

NR

NR

NCT03386838

Halted

Nivolumab/chemotherapy

Lung cancer

III

  

NCT03417037

Halted