Table 3 Ongoing and completed Phase III/IV clinical trials (≥ 100 subjects) on DNA methylation-targeted therapy (listed on https://clinicaltrials.gov/—last accessed October 16, 2024)

NCT01074047

AML

5-Aza

488

III

C

[338, 339]

NCT00887068

AML, MDS

5-Aza

187

III

C

[340]

NCT04173533

AML, MDS

5-Aza

326

III

A, NR

N/A

NCT02319135

AML

5-Aza

289

III

C

N/A

NCT05175508

AML, MDS

5-Aza + ATRA

180

II/III

US

N/A

NCT05469737

MDS

5-Aza + BSC

230

II/III

A, NR

N/A

NCT01566695

MDS

5-Aza + BSC

216

III

C

[341]

NCT01757535

AML

5-Aza + BSC

472

III

C

[342,343,344,345]

NCT00071799

MDS

5-Aza + BSC

358

III

C

[346,347,348,349,350]

NCT03173248

AML, MDS

5-Aza + ivosidenib

146

III

A, NR

[351]

NCT05907057

AML

5-Aza + ivosidenib

245

III

R

N/A

NCT04256317

AML, MDS, CMML

5-Aza + cedazuridine

317

II/III

R

N/A

NCT04102020

AML

5-Aza + venetoclax

112

III

A, NR

N/A

NCT05404906

AML

5-Aza + venetoclax

124

II/III

R

N/A

NCT02993523

AML

5-Aza + venetoclax

443

III

A, NR

[352,353,354,355]

NCT04161885

AML

5-Aza + venetoclax

465

III

A, NR

N/A

NCT04401748

MDS

5-Aza + venetoclax

531

III

A, NR

N/A

NCT05939180

AML

5-Aza + venetoclax

116

II/III

R

N/A

NCT05079230

AML

5-Aza + venetoclax + magrolimab

378

III

T

N/A

NCT04778397

AML

5-Aza + venetoclax/magrolimab

258

III

T

N/A

NCT04799275

DLBC

5-Aza + chemotherapy

422

II/III

R

N/A

NCT05678933

PTCL

5-Aza + CHOP regimen

200

III

EBI

N/A

NCT03765541

AML

5-Aza + dexamethasone

142

III

R

N/A

NCT02158936

MDS

5-Aza + eltrombopag

356

III

T

[356]

NCT03745716

MDS

5-Aza + eprenetapopt

154

III

C

N/A

NCT02752035

AML

5-Aza + gilteritinib

183

III

A, NR

N/A

NCT03873311

AML, MDS, CMML

5-Aza + HAG regimen

114

IV

US

N/A

NCT05709093

MDS

5-Aza + lemzoparlimab

552

III

R

N/A

NCT04313881

MDS

5-Aza + magrolimab

539

III

T

N/A

NCT04090736

AML

5-Aza + pevonedistat

302

III

US

N/A

NCT03268954

AML, MDS, CMML

5-Aza + pevonedistat

454

III

A, NR

[357]

NCT03151408

AML

5-Aza + pracinostat

406

III

T

N/A

NCT04266301

MDS, CMML

5-Aza + sabatolimab

530

III

A, NR

N/A

NCT04797780

MDS

5-Aza + tamibarotene

550

III

R

N/A

NCT05075460

PTCL

5-Aza + tucidinostat + CHOP regimen

107

III

R

N/A

NCT02785900

AML

5-Aza/DAC + vadastuximab talirine

240

III

T

N/A

NCT03926624

AML

5-Aza/DAC + venetoclax

450

III

US

N/A

NCT06073730

AML

5-Aza/DAC + venetoclax

154

III

NYR

N/A

NCT05264883

AML

5-Aza/DAC + venetoclax + aclarubicin

170

III

R

N/A

NCT03257241

AML

DAC

582

III

US

N/A

NCT02072811

AML

DAC

400

III

US

N/A

NCT04292769

Solid tumors

DAC

200

III

US

N/A

NCT00043381

MDS

DAC

160

III

C

N/A

NCT00043134

MDS

DAC

220

III

US

N/A

NCT01751867

MDS

DAC

135

III

C

[358]

NCT00260832

AML

DAC

485

III

C

[359]

NCT02013102

MDS

DAC

240

IV

US

N/A

NCT02172872

AML

DAC

606

III

A, NR

N/A

NCT03026842

AML

DAC

180

IV

US

N/A

NCT04098653

Myeloid tumors

DAC + BUCY

196

II/III

US

N/A

NCT04123392

Myeloid tumors

DAC + BUCY

196

II/III

US

N/A

NCT03596892

AML, MLL

DAC + BUCY

122

II/III

US

N/A

NCT04510610

HL

DAC + camrelizumab

100

II/III

R

N/A

NCT02159820

OV

DAC + carboplatin + paclitaxel

500

II/III

US

N/A

NCT03306264

AML, MDS, CMML

DAC + cedazuridine

227

III

C

N/A

NCT03553537

PTCL

DAC + CHOP

100

III

US

N/A

NCT02085408

AML

DAC + cytarabine + clofarabine/daunorubicin

727

III

A, NR

N/A

NCT05449899

AML

DAC + G-CSF + BUCY/BF

232

II/III

R

N/A

NCT04087967

AML

DAC + HAAG regimen

162

III

US

N/A

NCT04446130

AML, T-ALL

DAC + HAAG regimen

100

III

US

N/A

NCT01303796

AML

DAC + sapacitabine

482

III

C

[360]

NCT02472145

AML

DAC + talacotuzumab

326

II/III

C

[361]

NCT02272478

AML, MDS

DAC + vosaroxin

1,600

II/III

US

N/A

NCT02348489

AML

SGI-110

815

III

C

N/A

NCT02920008

AML

SGI-110

302

III

C

N/A

NCT02907359

MDS, CMML

SGI-110

417

III

C

N/A