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Table 1 Active trials involving immune checkpoint inhibitors

From: Translational landscape of glioblastoma immunotherapy for physicians: guiding clinical practice with basic scientific evidence

NCT

Phase

Intervention

Target population

Location

Sample size

Key outcomes

NCT02337686

Phase II

Pembrolizumab both pre- and postoperatively

Recurrent glioblastoma

USA

20

PFS, OS, ORR, toxicity, cytokine evaluation

NCT02617589

Phase III

Nivolumab/RT vs TMZ/RT

Newly diagnosed MGMT-unmethylated glioblastoma

USA

560

OS, PFS, tumor mutational burden comparison

NCT02667587

Phase III

Nivolumab/TMZ/RT vs placebo/TMZ/RT

Newly diagnosed MGMT-methylated glioblastoma

USA

716

PFS, OS

NCT02852655

Phase I

Neoadjuvant pembrolizumab vs placebo

Surgically accessible recurrent/progressive glioblastoma

USA

25

TIL density, adverse events, PFS

NCT02974621

Phase II

Cediranib/olaparib vs bevacizumab

Recurrent glioblastoma

USA

70

PFS, OS, adverse events, cytokine evaluation, tumor genetics

NCT03047473

Phase II

Adjuvant avelumab with TMZ/RT

Newly diagnosed glioblastoma

Canada

30

Adverse events, MRI evaluation, biomarker evaluation

NCT03158389

Phase I/II

Matches one of 7 drugs to patients (APG101, alectinib, idasanutlin, atezolizumab, vismodegib, Palbociclib, and temsirolimus) based on molecular markers after surgery, in addition to RT

MGMT-unmethylated glioblastoma

Germany

350

PFS, OS, adverse events

NCT03174197

Phase I/II

Atezolizumab/TMZ/RT

Newly diagnosed glioblastoma

USA

80

DLT, OS, adverse events, ORR, PFS

NCT03197506

Phase II

Neoadjuvant pembrolizumab and then adjuvant RT/TMZ/pembrolizumab

Newly diagnosed glioblastoma

USA

50

DLT, OS, adverse events, PFS, biomarkers

NCT03341806

Phase I

Avelumab (PD-L1 inhibitor) with or without MRI-guided laser interstitial thermal therapy

Recurrent glioblastoma

USA

13

DLT, ORR, PFS

NCT03426891

Phase I

Pembrolizumab/vorinostat (histone deacetylase inhibitor)/TMZ/RT in dose escalation

Newly diagnosed glioblastoma

USA

21

MTD, OS

NCT03493932

Phase I

Nivolumab with BMS-986016 (anti-Lag-3 antibody), with active monitoring by cerebral microdialysis

Recurrent glioblastoma

USA

20

IFN–γ levels, microdialysis safety, drug safety

NCT03532295

Phase II

Epacadostat (IDO-1 inhibitor) + INCMGA00012 (anti-PD1 antibody) given alone or in combination prior to RT/bevacizumab

Recurrent glioblastoma

USA

55

OS, PFS, neurological function, safety

NCT03718767

Phase II

Adjuvant nivolumab

IDH mutant glioma with or without hypermutator phenotype

USA

95

PFS, HRQOL, neoantigen burden

NCT03899857

Phase II

Study of standard of care with the addition of pembrolizumab for newly diagnosed GBM

Newly diagnosed glioblastoma

Switzerland

56

OS, PFS, treatment failure, PD-1 expression levels

NCT03925246

Phase II

Nivolumab in varying doses

Recurrent IDH mutant glioblastoma, prior TMZ/RT

France

43

PFS, OS, ORR, safety

NCT03961971

Phase I

MBG453 (anti-Tim3 antibody) and Spartalizumab (anti-PD1 antibody) given 1 week prior to SRS, then in an adjuvant fashion ongoing

Recurrent glioblastoma

USA

15

Adverse events, toxicity, PFS, OS, ORR

NCT04047706

Phase I

BMS 986,205 (IDO1 inhibitor) with nivolumab and TMZ/radiation

Newly diagnosed glioblastoma

USA

30

Adverse events, OS, PFS, MRI evaluation

NCT04145115

Phase II

Combination therapy with ipilimumab and nivolumab

Somatically hypermutated glioblastoma

USA

37

ORR, OS, PFS, adverse events

NCT04225039

Phase II

A Cohort = single priming dose of both drugs prior to RT, then adjuvant schedule for both drugs; B Cohort = neoadjuvant schedule of both drugs, then RT/resection, then adjuvant treatment

Recurrent glioblastoma

USA

32

ORR, adverse events, OS, PFS

NCT04323046

Phase I

Neoadjuvant Ipilimumab/nivolumab plus adjuvant nivolumab after surgery, vs placebo

Recurrent/progressive high-grade glioma in children/young adults

USA

45

Cell cycle gene changes, adverse events, OS, PFS

NCT04396860

Phase II/III

Phase II = ipilimumab alone; Phase III = ipilimumab and nivolumab; all patients received RT as well; active comparator is TMZ/TTF

Newly diagnosed MGMT-unmethylated glioblastoma, IDH wild type

USA

485

PFS, OS, adverse events, HRQOL, MGMT expression

NCT04608812

Phase I

OS2966 (mAb against CD28/b1 integrin) directly infused into the brain tumor and surrounding infiltrated brain

Newly diagnosed glioblastoma that has received resection and standard of care

USA

24

DLT, optimal dose, spatial distribution of drug, MRI evaluation

NCT04656535

Phase 0/I

AB154 (anti-TIGIT) and AB122 (anti-PD-1) in various combinations vs placebo control

Recurrent glioblastoma

USA

46

Adverse events, RNA sequencing, T cell changes

NCT04729959

Phase II

Tocilizumab ± atezolizumab, combination w/ surgery ± RT

Recurrent glioblastoma

USA

12

DLT, MTD, ORR, PFS, OS, adverse events

NCT04817254

Phase II

TMZ/RT and ipilimumab in two different dose regimens

Newly diagnosed glioblastoma or gliosarcoma

USA

48

OS, T cell responses, HRQOL

NCT04826393

Phase Ib

Dose escalation trial of ASP8374 (anti-TIGIT antibody) and cemiplimab (anti-PD1 antibody)

Recurrent high-grade glioma

USA

24

MTD, TIL, adverse events, PFS, OS

NCT04922723

Phase I/II

Adjuvant TMZ/RT plus daratumumab (anti-CD38)

Newly diagnosed glioblastoma

USA

16

DLT, OS, PFS, adverse events

NCT04952571

Phase II

Camrelizumab and bevacizumab

Recurrent glioblastoma

China

94

PFS, OS, ORR, control rate, functional outcomes, adverse events