Table 4 Summary of histone acetylation-targeted drugs for different diseases in clinical trials

KAT inhibitor

CCS1477

P300/CBP (KAT3A/KAT3B)

NHL, MM, AML, MDS, PTCL

Recruiting

Phase I/II

With or without Pomalidomide plus Dexamethasone, or Azacitidine plus Venetoclax

NCT04068597

CCS1477

P300/CBP (KAT3A/KAT3B)

CRPC, BC, NSCLC

Recruiting

Phase I/II

With or without Abiraterone acetate or Enzalutamide or Darolutamide or Olaparib or Atezolizumab

NCT03568656

FT-7051

P300/CBP (KAT3A/KAT3B)

CRPC

Terminated (due to sponsors’ decision)

Phase I

—

NCT04575766

NEO2734

P300/CBP (KAT3A/KAT3B), BET

CRPC, NUT carcinoma

Recruiting

Phase I

—

NCT05488548

PRI-724

CBP/β-catenin

HCV-induced cirrhosis

Completed (causes liver injury in the high-dose cohort)

Phase I

—

NCT02195440³⁹⁷

PRI-724

CBP/β-catenin

PDAC

Completed (unpublished)

Phase I

—

NCT01764477

PRI-724

CBP/β-catenin

Solid tumors

Terminated (due to low enrollment)

Phase I

—

NCT01302405

PRI-724

CBP/β-catenin

HIV/HCV co-induced cirrhosis

Completed (unpublished)

Phase I

—

NCT04688034

PRI-724

CBP/β-catenin

PBC

Completed (unpublished)

Phase I

—

NCT04047160

PRI-724

CBP/β-catenin

HIV/HCV co-induced cirrhosis

Recruiting

Phase II

—

NCT06144086

PRI-724

CBP/β-catenin

AML, CML

Completed (unpublished)

Phase I/II

—

NCT01606579

PRI-724

CBP/β-catenin

HCV-induced cirrhosis, HBV-induced cirrhosis

Completed (exhibits insufficient evidence of improvement in hepatic function)

Phase I/II

—

NCT03620474³⁹⁶

PF-07248144

KAT6

HR-positive, HER2-negative BC, CRPC, NSCLC

Recruiting

Phase I

With or without Fulvestrant, or Letrozole plus Palbociclib, or Fulvestrant plus PF-07220060

NCT04606446

HDAC inhibitor

Ivaltinostat

Pan-HDAC

Malignant tumors

Active, not recruiting

Phase I

Placebo-controlled

NCT05716919

Ivaltinostat

Pan-HDAC

Healthy volunteers

Completed (the oral formulation of Ivaltinostat is well tolerated)

Phase I

Placebo-controlled

NCT05345912¹⁰²⁹

Ivaltinostat

Pan-HDAC

PDAC

Unknown (exhibits good efficacy and an acceptable safe profile according to disclosed data)

Phase I/II

In combination with Gemcitabine and Erlotinib

NCT02737228⁴⁰¹

Ivaltinostat

Pan-HDAC

PDAC

Recruiting

Phase I/II

Capecitabine (active comparator/in combination with Ivaltinostat)

NCT05249101

Abexinostat

Pan-HDAC

High-grade glioma

Recruiting

Phase I

In combination with Temozolomide

NCT05698524

Abexinostat

Pan-HDAC

DLBCL, MCL

Active, not recruiting

Phase I

In combination with Ibrutinib

NCT03939182

Abexinostat

Pan-HDAC

Melanoma, squamous cell carcinoma of head and neck, urothelial carcinoma, NSCLC

Completed (unpublished)

Phase I

In combination with Pembrolizumab

NCT03590054

Abexinostat

Pan-HDAC

NHL, HL, MM

Completed (unpublished)

Phase I

—

NCT01149668

Abexinostat

Pan-HDAC

Solid tumors

Active, not recruiting (exhibits good tolerability and antitumor effects according to disclosed data)

Phase I

In combination with Pazopanib

NCT01543763⁴⁰⁴

Abexinostat

Pan-HDAC

NHL, HL, MM

Completed (unpublished)

Phase I

—

NCT00562224

Abexinostat

Pan-HDAC

Malignant tumors

Completed (unpublished)

Phase I

—

NCT00473577

Abexinostat

Pan-HDAC

MDS, AML, ALL

Terminated (due to limited clinical benefit)

Phase I

—

ISRCTN 99680465¹⁰³⁰

Abexinostat

Pan-HDAC

FL

Active, not recruiting

Phase II

—

NCT03600441

Abexinostat

Pan-HDAC

DLBCL

Recruiting

Phase II

—

NCT03936153

Abexinostat

Pan-HDAC

FL

Recruiting

Phase II

—

NCT03934567

Abexinostat

Pan-HDAC

NHL

Active, not recruiting

Phase I/II

—

NCT04024696

Abexinostat

Pan-HDAC

NHL, HL

Completed (exhibits tolerable safety and significant clinical activity in FL)

Phase I/II

—

NCT00724984⁴⁰⁶

Abexinostat

Pan-HDAC

Sarcoma

Completed (exhibits manageable toxicities and tumor responses)

Phase I/II

In combination with Doxorubicin and GCSF

NCT01027910¹⁰³¹

Abexinostat

Pan-HDAC

B cell lymphoma, CML

Completed (exhibits manageable toxicity and partial responses)

Phase I/II

—

EudraCT 2009-013691-47^405,1032

Abexinostat

Pan-HDAC

RCC

Recruiting

Phase III

Pazopanib (active comparator/in combination with Abexinostat)

NCT03592472

AR-42

Pan-HDAC

Vestibular schwannoma, meningioma, acoustic neuroma, neurofibromatosis type 2

Terminated (due to a lack in drug supply)

Phase I

—

NCT02282917

AR-42

Pan-HDAC

RCC, soft tissue sarcoma

Terminated (due to a lack in drug supply)

Phase I

In combination with Pazopanib

NCT02795819

AR-42

Pan-HDAC

AML

Completed (exits possibilities of serious treatment-associated adverse events)

Phase I

In combination with Decitabine

NCT01798901¹⁰³³

AR-42

Pan-HDAC

Hematologic malignancies

Completed (exhibits tolerable safety)

Phase I

—

NCT01129193^402,1034

AR-42

Pan-HDAC

Plasma cell myeloma

Completed (unpublished)

Phase I

In combination with Dexamethasone and Pomalidomide

NCT02569320

AR-42

Pan-HDAC

Neurofibromatosis type 2

Recruiting

Phase II/III

Placebo-controlled

NCT05130866

Pracinostat

HDAC class I/II/IV

Healthy volunteers

Completed (unpublished)

Phase I

—

NCT03495934

Pracinostat

HDAC class I/II/IV

Healthy volunteers

Completed (unpublished)

Phase I

Fasted or fed conditions

NCT02058784

Pracinostat

HDAC class I/II/IV

Healthy volunteers

Completed (unpublished)

Phase I

In combination with Ciprofloxacin or Itraconazole

NCT02118909

Pracinostat

HDAC class I/II/IV

Solid tumors, leukemia

Completed (unpublished)

Phase I

—

NCT01184274

Pracinostat

HDAC class I/II/IV

Solid tumors, hematologic malignancies

Completed (exhibits safety and modest single-agent activity in hematologic malignancies)

Phase I

With or without Azacitidine

NCT00741234¹⁰³⁵

Pracinostat

HDAC class I/II/IV

AML

Completed (unpublished)

Phase I

Gemtuzumab Ozogamicin (active comparator/in combination with Pracinostat)

NCT03848754

Pracinostat

HDAC class I/II/IV

Solid tumors

Completed (unpublished)

Phase I

—

NCT00504296

Pracinostat

HDAC class I/II/IV

Solid tumors

Completed (exhibits good tolerability and inhibitory effects)

Phase I

—

SCS-PN0022¹⁰³⁶

Pracinostat

HDAC class I/II/IV

Solid tumors

Completed (exhibits good tolerability)

Phase I

—

¹⁰³⁷

Pracinostat

HDAC class I/II/IV

Solid tumors

Completed (exhibits good tolerability and inhibitory effects)

Phase II

—

NCT01912274¹⁰³⁸

Pracinostat

HDAC class I/II/IV

MDS

Terminated (due to sponsors’ decision)

Phase II

In combination with Azacitidine

NCT03151304

Pracinostat

HDAC class I/II/IV

Myelofibrosis

Completed (worsening anemia and other adverse events do not support the continued development)

Phase II

In combination with Ruxolitinib and Questionnaire

NCT02267278¹⁰³⁹

Pracinostat

HDAC class I/II/IV

MDS

Completed (reduced doses exhibit improved tolerability and efficacy)

Phase II

In combination with Azacitidine and Decitabine

NCT01993641¹⁰⁴⁰

Pracinostat

HDAC class I/II/IV

CRPC

Completed (exhibits insufficient activity as a single agent)

Phase II

—

NCT01075308¹⁰⁴¹

Pracinostat

HDAC class I/II/IV

MDS

Completed (fails to improve outcomes at the available dosing regimen)

Phase II

Azacitidine (active comparator/in combination with Pracinostat)

NCT01873703¹⁰⁴²

Pracinostat

HDAC class I/II/IV

Myeloproliferative disorders

Completed (exhibits reasonable tolerability and modest activity in myelofibrosis)

Phase II

—

NCT01200498¹⁰⁴³

Pracinostat

HDAC class I/II/IV

Sarcoma

Completed (premature stop due to the prolonged unavailability)

Phase II

—

NCT01112384¹⁰⁴⁴

Pracinostat

HDAC class I/II/IV

AML

Terminated (due to a lack of efficacy)

Phase III

Azacitidine (active comparator/in combination with Pracinostat)

NCT03151408¹⁰⁴⁵

Resminostat

HDAC class I/IIb/IV

CTCL, MF

Completed (unpublished)

Phase I

—

NCT04955340

Resminostat

HDAC class I/IIb/IV

Biliary tract cancer, pancreatic cancer

Completed (exhibits acceptable tolerability)

Phase I

In combination with chemotherapy

JapicCTI-152864¹⁰⁴⁶

Resminostat

HDAC class I/IIb/IV

Solid tumors

Completed (exhibits on-target pharmacodynamic activity at dose levels ≥400 mg and signs of antitumor efficacy)

Phase I

—

¹⁰⁴⁷

Resminostat

HDAC class I/IIb/IV

CTCL, MF

Active, not recruiting

Phase II

Placebo-controlled

NCT02953301

Resminostat

HDAC class I/IIb/IV

HL

Completed (exhibits acceptable safety and efficacy)

Phase II

—

NCT01037478¹⁰⁴⁸

Resminostat

HDAC class I/IIb/IV

HCC

Completed (exhibits early signs of efficacy and good tolerability)

Phase II

With or without Sorafenib

NCT00943449¹⁰⁴⁹

Resminostat

HDAC class I/IIb/IV

Biliary tract cancer

Completed (exhibits no significant improve in clinical activity)

Phase II

In combination with chemotherapy

JapicCTI-183883¹⁰⁵⁰

Resminostat

HDAC class I/IIb/IV

HCC

Completed (no significant efficacy advantage over sorafenib monotherapy)

Phase I/II

Sorafenib (active comparator/in combination with Resminostat)

NCT02400788¹⁰⁵¹

Resminostat

HDAC class I/IIb/IV

NSCLC

Completed (fails to improve progression-free survival and increases toxicity)

Phase I/II

In combination with Docetaxel

JapicCTI-132123¹⁰⁵²

Resminostat

HDAC class I/IIb/IV

Colorectal carcinoma

Completed (unpublished)

Phase I/II

Chemotherapy (active comparator/in combination with Resminostat)

NCT01277406

Tacedinaline

HDAC class I/II/III

Solid tumors

Completed (exhibits antitumor activity)

Phase I

In combination with Carboplatin and Paclitaxel

¹⁰⁵³

Tacedinaline

HDAC class I/II/III

Solid tumors

Completed (thrombocytopenia is the main principal dose-limiting toxicity)

Phase I

In combination with Capecitabine

¹⁰⁵⁴

Tacedinaline

HDAC class I/II/III

Solid tumors

Completed (exhibits preliminary efficacy and potential adverse events)

Phase I

In combination with Gemcitabine hydrochloride

¹⁰⁵⁵

Tacedinaline

HDAC class I/II/III

Solid tumors

Completed (exhibits preliminary efficacy and potential adverse events)

Phase I

—

¹⁰⁵⁶

Tacedinaline

HDAC class I/II/III

MM

Completed (unpublished)

Phase II

—

NCT00005624

Tacedinaline

HDAC class I/II/III

Pancreatic cancer

Completed (exhibits no evidence for improving efficacy)

Phase II

In combination with Gemcitabine hydrochloride; placebo-controlled

NCT00004861¹⁰⁵⁷

Tacedinaline

HDAC class I/II/III

NSCLC

Completed (unpublished)

Phase III

In combination with Gemcitabine hydrochloride; placebo-controlled

NCT00005093

FRM-0334

HDAC class I/II

Frontotemporal dementia with granulin mutation

Unknown (exhibits tolerable safety while insufficient efficacy according to disclosed data)

Phase II

Placebo-controlled

NCT02149160¹⁰⁵⁸

Trichostatin A

HDAC class I/II

Hematologic malignancies

Unknown

Phase I

—

NCT03838926

Quisinostat

HDAC class I/II

Leukemia, MDS

Terminated (due to sponsors’ decision)

Phase I

—

NCT00676728

Quisinostat

HDAC class I/II

Solid tumors, lymphomas

Completed (intermittent schedules exhibit better tolerated than continuous schedules)

Phase I

—

NCT00677105¹⁰⁵⁹

Quisinostat

HDAC class I/II

NSCLC, ovarian cancer

Completed (unpublished)

Phase I

In combination with Cisplatin plus Gemcitabine, or Paclitaxel plus Carboplatin

NCT02728492

Quisinostat

HDAC class I/II

MM

Completed (exhibits efficacy and tolerable safety)

Phase I

In combination with Dexamethasone and Bortezonib

NCT01464112¹⁰⁶⁰

Quisinostat

HDAC class I/II

CTCL

Completed (exhibits an acceptable safety profile)

Phase II

—

NCT01486277¹⁰⁶¹

Quisinostat

HDAC class I/II

Ovarian cancer

Completed (unpublished)

Phase II

In combination with Paclitaxel and Carboplatin

NCT02948075

CXD101

HDAC class I

Malignant tumors

Completed (exhibits acceptable tolerability with efficacy in HL, T cell lymphoma, and FL)

Phase I

—

NCT01977638¹⁰⁶²

CXD101

HDAC class I

HCC

Recruiting

Phase II

In combination with Geptanolimab, Lenvatinib and Sorafenib (active comparator)

NCT05873244

CXD101

HDAC class I

Colorectal carcinoma

Unknown (exhibits good tolerability and efficacy according to disclosed data)

Phase I/II

In combination with Nivolumab

NCT03993626¹⁰⁶³

CXD101

HDAC class I

DLBCL

Withdrawn (due to insufficent funds)

Phase I/II

In combination with Pembrolizumab

NCT03873025

Magnesium valproate

HDAC class I

Solid tumors

Completed (exhibits the potential to overcome chemotherapy resistance)

Phase II

In combination with Hydralazine

NCT00404508³⁰⁴

Magnesium valproate

HDAC class I

Cervical cancer

Completed (unpublished)

Phase II

In combination with Hydralazine

NCT00404326

Magnesium valproate

HDAC class I

Colorectal carcinoma

Recruiting

Phase II

With or without Panitumumab and Cetuximab

NCT05694936

Magnesium valproate

HDAC class I

BC

Terminated (treatment is well-tolerated)

Phase II

In combination with Hydralazine

NCT00395655³⁰¹

Magnesium valproate

HDAC class I

Ovarian cancer

Unknown

Phase III

In combination with Hydralazine, placebo-controlled

NCT00533299

Magnesium valproate

HDAC class I

Cervical cancer

Completed (exhibits advantages in progression-free survival)

Phase III

In combination with Hydralazine; placebo-controlled

NCT00532818

Magnesium valproate

HDAC class I

Cervical cancer

Unknown

Phase III

In combination with Hydralazine, Carboplatin, and Paclitaxel; placebo-controlled

NCT02446652

OBP-801

HDAC class I

Solid tumors

Unknown (large-scale trials should be hold according to disclosed data)

Phase I

—

NCT02414516¹⁰⁶⁴

Nanatinostat

HDAC class I

Malignant tumors (excluding gastrointestinal tumors)

Recruiting

Phase I

With or without Valganciclovir

NCT06302140

Nanatinostat

HDAC class I

EBV-associated lymphoma, PTCL, PTLD

Recruiting

Phase II

In combination with Valganciclovir

NCT05011058

Nanatinostat

HDAC class I

EBV-associated lymphoma

Completed (exhibits encouraging efficacy)

Phase I/II

In combination with Valganciclovir

NCT03397706¹⁰⁶⁵

Nanatinostat

HDAC class I

EBV-associated solid tumors

Recruiting

Phase I/II

In combination with Valganciclovir, with or without Pembrolizumab

NCT05166577

Entinostat

HDAC class 1

TNBC

Terminated (due to funding withdrawn)

Early phase I

—

NCT03361800

Entinostat

HDAC class 1

Healthy volunteers

Completed (unpublished)

Phase I

—

NCT02922946

Entinostat

HDAC class 1

Healthy volunteers; renal impairment

Completed (unpublished)

Phase I

—

NCT03192111

Entinostat

HDAC class 1

Solid tumors

Completed (unpublished)

Phase I

Placebo-controlled

NCT02897778

Entinostat

HDAC class 1

CRPC

Completed (exhibits an acceptable safety profile)

Phase I

In combination with Enzalutamide

NCT03829930¹⁰⁶⁶

Entinostat

HDAC class 1

HR-positive HER2-negative BC

Completed (exhibits reasonable safety, tolerability, and encouraging efficacy)

Phase I

In combination with Exemestane

NCT02833155¹⁰⁶⁷

Entinostat

HDAC class 1

MM, MDS, myeloproliferative diseases

Completed (exhibits effective inhibition on HDAC in vivo)

Phase I

—

NCT00015925¹⁰⁶⁸

Entinostat

HDAC class 1

Solid tumors, lymphomas

Completed (exhibits good tolerability at the studied doses)

Phase I

—

NCT00020579¹⁰⁶⁹

Entinostat

HDAC class 1

Healthy volunteers; renal impairment

Completed (unpublished)

Phase I

In combination with Midazolam

NCT03187015

Entinostat

HDAC class 1

Solid tumors

Completed (unpublished)

Phase I

In combination with Pembrolizumab

NCT02909452

Entinostat

HDAC class 1

MDS

Active, not recruiting (exhibits limited clinical efficacy and substantial toxicity according to disclosed data)

Phase I

In combination with Pembrolizumab

NCT02936752¹⁰⁷⁰

Entinostat

HDAC class 1

BC

Completed (unpublished)

Phase I

In combination with Capecitabine

NCT03473639

Entinostat

HDAC class 1

Ovarian cancer, peritoneal cancer, fallopian tube cancer

Terminated (due to changes in participant landscape and other treatment availability)

Phase I

In combination with Olaparib

NCT03924245

Entinostat

HDAC class 1

HR-positive BC

Completed (unpublished)

Phase I

In combination with Exemestane

NCT02820961

Entinostat

HDAC class 1

HR-positive BC, NSCLC

Completed (results published along with phase II studies)

Phase I

In combination with Erlotinib and Exemestane

NCT01594398

Entinostat

HDAC class 1

Lymphoma

Completed (exhibits tolerable safety)

Phase I

In combination with Isotretinoin

NCT00098891¹⁰⁷¹

Entinostat

HDAC class 1

AML, MDS, CMML

Completed (increases toxicity in treating myeloid neoplasms)

Phase I

In combination with Azacitidine

NCT00101179^{1072,1073,1074}

Entinostat

HDAC class 1

Healthy volunteers

Completed (unpublished)

Phase I

Dietary supplements (Omeprazole and Famotidine)

NCT02922933

Entinostat

HDAC class 1

HR-positive BC

Completed (exhibits no additional safety concerns)

Phase I

In combination with KHK2375

NCT02623751¹⁰⁷⁵

Entinostat

HDAC class 1

Colorectal carcinoma

Completed (the combination is poorly tolerated without evident activity)

Phase I

In combination with Hydroxychloroquine and Regorafenib

NCT03215264⁴¹³

Entinostat

HDAC class 1

SCLC

Completed (further exploration should not be applied)

Phase I

In combination with Atezolizumab, Carboplatin, and Etoposide

NCT04631029⁴¹⁴

Entinostat

HDAC class 1

CNS tumors, lymphoma

Completed (exhibits good tolerability)

Phase I

—

NCT02780804¹⁰⁷⁶

Entinostat

HDAC class 1

Endometrial endometrioid adenocarcinoma

Completed (no immediate effect on the regulation of progesterone receptor)

Phase I

With or without Medroxyprogesterone acetate

NCT03018249⁴¹⁵

Entinostat

HDAC class 1

HER2-positive BC, TNBC

Terminated (due to slow accrual and company reasons)

Phase I

M7824 and Ado-trastuzumab emtansine (active comparator/in combination with Entinostat)

NCT04296942

Entinostat

HDAC class 1

HER2-positive BC

Completed (exhibits acceptable tolerability and antitumor activity)

Phase I

In combination with Lapatinib ditosylate

NCT01434303¹⁰⁷⁷

Entinostat

HDAC class 1

ALL, ABL

Completed (exhibits less activities in relapsed/refractory patients)

Phase I

In combination with Clofarabine

NCT01132573⁴¹⁶

Entinostat

HDAC class 1

Solid tumors

Terminated (exhibits good tolerability according to disclosed data)

Phase I

In combination with Sorafenib

NCT01159301¹⁰⁷⁸

Entinostat

HDAC class 1

NSCLC

Terminated

Phase I

In combination with Azacitidine

NCT01886573

Entinostat

HDAC class 1

RCC

Active, not recruiting (exhibits acceptable safety and efficacy according to disclosed data)

Phase I

In combination with Aldesleukin

NCT01038778

Entinostat

HDAC class 1

BC

Terminated

Phase I

—

NCT00754312

Entinostat

HDAC class 1

HR-positive BC, TNBC

Active, not recruiting (exhibits good efficacy according to disclosed data)

Phase I

In combination with Ipilimumab and Nivolumab

NCT02453620¹⁰⁷⁹

Entinostat

HDAC class 1

BC

Completed (exhibits acceptable safety)

Phase II

Exemestane (active comparator/in combination with Entinostat); placebo-controlled

NCT03291886¹⁰⁸⁰

Entinostat

HDAC class 1

Uveal melanoma

Completed (exhibits durable responses in a subset of patients)

Phase II

In combination with Pembrolizumab

NCT02697630^1081,1082

Entinostat

HDAC class 1

TNBC

Active, not recruiting (exhibits good tolerability but fails to meet primary endpoint according to disclosed data)

Phase II

In combination with Azacitidine

NCT01349959

Entinostat

HDAC class 1

HL

Terminated (due to corporate decision)

Phase II

—

NCT00866333¹⁰⁸³

Entinostat

HDAC class 1

MDS, AML

Completed (increases toxicity in treating myeloid neoplasms)

Phase II

Azacitidine (active comparator/in combination with Entinostat)

NCT00313586^1072,1084

Entinostat

HDAC class 1

HR-positive BC

Completed (exhibits good tolerability and clinical activity)

Phase II

Exemestane (active comparator); placebo-controlled

NCT00676663¹⁰⁸⁵

Entinostat

HDAC class 1

Neuroendocrine tumors

Terminated (due to a lack of funding and drug supply)

Phase II

—

NCT03211988

Entinostat

HDAC class 1

Cholangiocarcinoma, PDAC

Completed (exhibits promising efficacy)

Phase II

In combination with Nivolumab

NCT03250273

Entinostat

HDAC class 1

Melanoma

Completed (unpublished)

Phase II

—

NCT00185302

Entinostat

HDAC class 1

Lymphomas

Active, not recruiting

Phase II

In combination with Pembrolizumab

NCT03179930

Entinostat

HDAC class 1

RCC

Active, not recruiting

Phase II

Interleukin-2 (active comparator/in combination with Entinostat)

NCT03501381

Entinostat

HDAC class 1

Melanoma

Completed (exhibits preliminary antitumor effects)

Phase II

In combination with Pembrolizumab

NCT03765229

Entinostat

HDAC class 1

Bladder cancer

Active, not recruiting

Phase II

Pembrolizumab (active comparator/in combination with Entinostat)

NCT03978624

Entinostat

HDAC class 1

RCC

Active, not recruiting

Phase II

In combination with Nivolumab and Ipilimumab

NCT03552380

Entinostat

HDAC class 1

AML, ALL

Completed (exhibits preliminary antitumor effects)

Phase II

In combination with Sargramostim

NCT00462605

Entinostat

HDAC class 1

NSCLC

Terminated (due to business reasons)

Phase II

In combination with Erlotinib

NCT00750698

Entinostat

HDAC class 1

AML

Active, not recruiting

Phase II

In combination with Azacitidine

NCT01305499

Entinostat

HDAC class 1

NSCLC

Terminated (due to slow accrual)

Phase II

In combination with Azacitidine

NCT01207726

Entinostat

HDAC class 1

Colon cancer, rectal cancer

Completed (exhibits preliminary antitumor effects)

Phase II

In combination with Azacitidine

NCT01105377

Entinostat

HDAC class 1

HR-positive BC

Completed (risks of treatment-associated adverse events are high)

Phase II

In combination with Aromatase inhibitor

NCT00828854

Entinostat

HDAC class 1

NSCLC

Completed (combination is a promising tool in future exploration)

Phase II

In combination with Azacitidine and Nivolumab; Nivolumab with or without CC-486 300 (active comparator)

NCT01928576

Entinostat

HDAC class 1

TNBC

Terminated (due to slow accrual)

Phase II

In combination with Anastrozole

NCT01234532

Entinostat

HDAC class 1

NSCLC

Terminated

Phase II

In combination with Azacitidine and chemotherapy; chemotherapy (active comparator)

NCT01935947

Entinostat

HDAC class 1

NSCLC

Completed (exhibits clinically meaningful benefit)

Phase I/II

In combination with Pembrolizumab

NCT02437136¹⁰⁸⁶

Entinostat

HDAC class 1

CNS tumors

Recruiting

Phase I/II

In combination with Nivolumab; placebo-controlled

NCT03838042¹⁰⁸⁷

Entinostat

HDAC class 1

RCC

Active, not recruiting (exhibits promising clinical activities according to disclosed data)

Phase I/II

In combination with Aldesleukin

NCT01038778¹⁰⁸⁸

Entinostat

HDAC class 1

NSCLC

Completed (exhibits improvement in progression-free rates and overall survival)

Phase I/II

With or without Azacitidine

NCT00387465¹⁰⁸⁹

Entinostat

HDAC class 1

NSCLC

Completed (the combination fails to improve the outcomes)

Phase I/II

Erlotinib (active comparator/in combination with Entinostat); placebo-controlled

NCT00602030¹⁰⁹⁰

Entinostat

HDAC class 1

Ovarian cancer, peritoneal cancer, fallopian tube cancer

Completed (exhibits comparable efficacy and tolerability)

Phase I/II

Avelumab (active comparator/in combination with Entinostat); placebo-controlled

NCT02915523

Entinostat

HDAC class 1

BC

Completed (exhibits clinical activity)

Phase I/II

Atezolizumab (active comparator/in combination with Entinostat); placebo-controlled

NCT02708680

Entinostat

HDAC class 1

RCC

Suspended (due to major review underway)

Phase I/II

In combination with Atezolizumab and Bevacizumab

NCT03024437

Entinostat

HDAC class 1

HPV-associated malignancies, small bowel cancer,colon cancer

Recruiting

Phase I/II

In combination with Bintrafusp Alfa/NHS-IL12, or NHS-IL12

NCT04708470

Entinostat

HDAC class 1

Solid tumors

Recruiting

Phase I/II

In combination with ZEN-3694

NCT05053971

Entinostat

HDAC class 1

Esophageal cancer

Suspended (due to revisions to design)

Phase I/II

In combination with Nivolumab, Montanide(R) ISA-51 VG Adjuvant, and H1299 Cell Lysates

NCT05898828

Entinostat

HDAC class 1

ALL

Terminated (due to low accrual)

Phase I/II

In combination with Imatinib mesylate

NCT01383447

Entinostat

HDAC class 1

BC

Active, not recruiting

Phase I/II

Umbrella study

NCT03280563

Entinostat

HDAC class 1

HR-positive HER2-negative BC

Active, not recruiting (the combination fails to improve survival according to disclosed data)

Phase III

Exemestane/Goserelin/Goserelin acetate (active comparator/in combination with Entinostat); placebo-controlled

NCT02115282^1091,1092

Entinostat

HDAC class 1

HR-positive BC

Unknown (exhibits encouraging outcomes according to disclosed data)

Phase III

Exemestane (active comparator/in combination with Entinostat); placebo-controlled

NCT03538171⁴¹²

Mocetinostat

HDAC class 1

CRPC, BC, NSCLC

Terminated (due to terminated collaboration)

Phase I

In combination with Docetaxel

NCT00511576

Mocetinostat

HDAC class 1

MDS, lymphomas

Completed (unpublished)

Phase I

Given twice weekly

NCT00324194

Mocetinostat

HDAC class 1

MDS, lymphomas

Completed (dose-limiting toxicities of fatigue, nausea, vomiting, and diarrhea observed at higher doses)

Phase I

Given three-times weekly

NCT00324129¹⁰⁹³

Mocetinostat

HDAC class 1

NHL

Completed (unpublished)

Phase I

Given twice weekly

NCT00323934

Mocetinostat

HDAC class 1

Squamous cell carcinoma of head and neck, squamous cell carcinoma of oral cavity

Withdrawn (due to a change in internal prioritization)

Phase I

In combination with Durvalumab

NCT02993991

Mocetinostat

HDAC class 1

Rhabdomyosarcoma

Recruiting

Phase I

In combination with Vinorelbine

NCT04299113

Mocetinostat

HDAC class 1

Lung cancer

Active, not recruiting

Phase I

In combination with Pembrolizumab and Guadecitabine

NCT03220477

Mocetinostat

HDAC class 1

Melanoma

Terminated (exhibits favorable response rates but with high levels of toxicity according to disclosed data)

Phase I

In combination with Ipilimumab and Nivolumab

NCT03565406¹⁰⁹⁴

Mocetinostat

HDAC class 1

Urothelial carcinoma

Completed (exhibits modest clinical activity)

Phase II

—

NCT02236195¹⁰⁹⁵

Mocetinostat

HDAC class 1

HL

Terminated (exhibits single-agent clinical activity with manageable toxicity according to disclosed data)

Phase II

—

NCT00358982¹⁰⁹⁶

Mocetinostat

HDAC class 1

Lymphoma

Completed (exhibits limited single-agent activity in DLBCL and FL but long-term clinical benefit)

Phase II

—

NCT00359086¹⁰⁹⁷

Mocetinostat

HDAC class 1

AML, MDS

Terminated (due to terminated collaboration)

Phase II

Azacitidine (active comparator/in combination with Mocetinostat)

NCT00666497

Mocetinostat

HDAC class 1

NHL, HL

Terminated (due to terminated collaboration)

Phase II

In combination with Azacitidine

NCT00543582

Mocetinostat

HDAC class 1

CLL

Completed (exhibits limited activity)

Phase II

—

NCT00431873¹⁰⁹⁸

Mocetinostat

HDAC class 1

AML, MDS

Terminated (due to the re-evaluation of clinical development program)

Phase II

—

NCT00374296

Mocetinostat

HDAC class 1

Leiomyosarcoma

Completed (exhibits insufficient activity)

Phase II

In combination with Gemcitabine

NCT02303262

Mocetinostat

HDAC class 1

NSCLC

Terminated (due to sponsors’ decision)

Phase II

In combination with Nivolumab; Nivolumab with Sitravatinib or Glesatinib (active comparator)

NCT02954991

Mocetinostat

HDAC class 1

Malignant tumors

Completed (exhibits significant toxicities in advanced pancreatic cancer)

Phase I/II

In combination with Gemcitabine

NCT00372437¹⁰⁹⁹

Mocetinostat

HDAC class 1

DLBCL,lymphomas

Terminated (due to slow accrual)

Phase I/II

—

NCT02282358¹¹⁰⁰

Mocetinostat

HDAC class 1

NSCLC, solid tumors

Terminated (due to sponsors’ decision)

Phase I/II

In combination with Durvalumab

NCT02805660¹¹⁰¹

Mocetinostat

HDAC class 1

MDS, AML

Completed (unpublished)

Phase I/II

—

NCT00324220

Mocetinostat

HDAC class 1

MDS

Completed (unpublished)

Phase I/II

In combination with Azacitidine

NCT02018926

Mocetinostat

HDAC class 1

HL

Completed (exhibits preliminary clinical activity)

Phase I/II

In combination with Brentuximab vedotin

NCT02429375

Domatinostat

LSD1/HDAC

Hematologic malignancies

Completed (exhibits safety and early signs of antitumor activity)

Phase I

—

NCT01344707¹¹⁰²

Domatinostat

LSD1/HDAC

GastrointEstinal cancer

Unknown (exhibits an acceptable safety profile according to disclosed data)

Phase II

In combination with Avelumab

NCT03812796¹¹⁰³

Domatinostat

LSD1/HDAC

Merkel cell carcinoma

Withdrawn (due to sponsors’ decision)

Phase II

In combination with Avelumab

NCT04874831

Domatinostat

LSD1/HDAC

Merkel cell carcinoma

Completed (unpublished)

Phase II

In combination with Avelumab

NCT04393753

Domatinostat

LSD1/HDAC

Melanoma

Active, not recruiting (Domatinostat addition fails to increase treatment efficacy according to disclosed data)

Phase I/II

Nivolumab (active comparator/in combination with Domatinostat); in combination with Nivolumab and Ipilimumab

NCT04133948⁴²⁴

CUDC101

EGFR/HER2/HDAC

Solid tumors

Terminated

Phase I

—

NCT01702285

CUDC101

EGFR/HER2/HDAC

Squamous cell carcinoma of head and neck, gastric cancer, BC, HCC, NSCLC

Completed (exhibits acceptable safety)

Phase I

—

NCT01171924

CUDC101

EGFR/HER2/HDAC

Squamous cell carcinoma of head and neck

Completed (the combination exhibits promising feasibility)

Phase I

In combination with Cisplatin and radiation therapy

NCT01384799⁴²⁰

CUDC101

EGFR/HER2/HDAC

Solid tumors

Completed (exhibits good tolerability and antitumor activity)

Phase I

—

NCT00728793⁴²¹

CUDC-907

PI3K/HDAC

Lymphoma

Completed (exhibits tolerable safety profile and durable antitumor activity)

Phase I

With or without Rituximab or Venetoclax

NCT01742988^422,1104

CUDC-907

PI3K/HDAC

Diffuse intrinsic pontine glioma, medulloblastoma, high-grade glioma

Active, not recruiting

Phase I

—

NCT03893487

CUDC-907

PI3K/HDAC

TNBC, ovarian cancer, NUT carcinoma

Completed (unpublished)

Phase I

—

NCT02307240

CUDC-907

PI3K/HDAC

CNS tumors, lymphoma

Active, not recruiting

Phase I

—

NCT02909777

CUDC-907

PI3K/HDAC

DLBCL

Completed (exhibits preliminary antitumor effects)

Phase II

—

NCT02674750⁴²³

CUDC-907

PI3K/HDAC

Cushing disease

Not yet recruiting

Phase II

—

NCT05971758

CUDC-907

PI3K/HDAC

Thyroid cancer

Terminated (due to investigator’s reasons)

Phase II

—

NCT03002623

Sodium phenylbutyrate

PRKCA/HDAC

MCAD deficiency

Recruiting

Phase II

—

NCT06069375

Tinostamustine

DNA/HDAC

Melanoma

Unknown

Phase I

—

NCT03903458

Tinostamustine

DNA/HDAC

Glioblastoma multiforme

Active, not recruiting

Phase I

—

NCT05432375

Tinostamustine

DNA/HDAC

MM, HL, CTCL

Active, not recruiting

Phase I

—

NCT02576496

Tinostamustine

DNA/HDAC

MGMT-promoter unmethylated glioblastoma

Completed (unpublished)

Phase I

With or without radiation therapy

NCT03452930

Tinostamustine

DNA/HDAC

DLBCL

Withdrawn (given the safety data on drug)

Phase I

In combination with Pembrolizumab and Rituximab

NCT04279938

Tinostamustine

DNA/HDAC

MM

Terminated (due to sponsors’ decision based on adverse events)

Phase I/II

ASCT

NCT03687125

Tinostamustine

DNA/HDAC

SCLC, soft tissue sarcoma, TNBC, ovarian cancer, endometrial cancer

Completed (unpublished)

Phase I/II

—

NCT03345485

GSK3117391

Esterase-sensitive motif

RA

Terminated (due to development portfolio)

Phase II

Placebo-controlled

NCT02965599

Tefinostat

Esterase-sensitive motif

Hematologic malignancies

Completed (exhibits early signs of efficacy and absence of significant toxicity)

Phase I

—

NCT00820508¹¹⁰⁵

Tefinostat

Esterase-sensitive motif

HCC

Unknown

Phase I/II

—

NCT02759601

HDAC agonist

Theophylline

Pan-HDAC

COPD

Completed (exhibits an increase in total HDAC activity and potential clinical benefit)

Phase II

With or without Fluticasone propionate

NCT00241631¹¹⁰⁶

Theophylline

Pan-HDAC

COPD

Completed (fails to enhance the anti-inflammatory properties of ICS)

Phase III

In combination with ICS; placebo-controlled

NCT01599871⁴²⁶

Theophylline

Pan-HDAC

Bronchiectasis

Completed (unpublished)

Phase IV

With or without Formoterol-budesonide

NCT01769898

Theophylline

Pan-HDAC

COPD

Completed (exhibits an increase in total HDAC activity and potential clinical benefit)

Not applicable

With or without standard therapy

NCT00671151¹¹⁰⁷

Sirtuin agonist

Resveratrol

SIRT1

T2DM

Completed (H3K56ac is located in the key position between SIRT1 and T2DM)

Phase III

Placebo-controlled

NCT02244879¹¹⁰⁸

BET inhibitor

ZEN-3694

Pan-BET

Colorectal carcinoma

Recruiting

Phase I

In combination with Capecitabine

NCT05803382

ZEN-3694

Pan-BET

Endometrial carcinoma

Recruiting

Phase I

In combination with Tuvusertib

NCT05950464

ZEN-3694

Pan-BET

Platinum-resistant ovarian carcinoma

Recruiting

Phase I

In combination with Nivolumab or Nivolumab plus Ipilimumab

NCT04840589

ZEN-3694

Pan-BET

Colorectal carcinoma

Recruiting

Phase I

In combination with Cetuximab and Encorafenib

NCT06102902

ZEN-3694

Pan-BET

BC, NUT carcinoma

Recruiting

Phase I

In combination with Abemaciclib

NCT05372640

ZEN-3694

Pan-BET

Ovarian cancer, solid tumors

Recruiting

Phase I

In combination with Niraparib

NCT06161493

ZEN-3694

Pan-BET

Ovarian cancer, solid tumors

Recruiting

Phase I

In combination with Binimetinib

NCT05111561

ZEN-3694

Pan-BET

CRPC

Completed (unpublished)

Phase I

—

NCT02705469

ZEN-3694

Pan-BET

CRPC

Recruiting

Phase II

In combination with Enzalutamide and Pembrolizumab

NCT04471974

ZEN-3694

Pan-BET

Solid tumors

Recruiting

Phase II

In combination with Talazoparib

NCT05327010

ZEN-3694

Pan-BET

CRPC

Recruiting

Phase II

Enzalutamide (active comparator/in combination with ZEN-3694)

NCT04986423

ZEN-3694

Pan-BET

Squamous cell lung cancer

Recruiting

Phase II

—

NCT05607108

ZEN-3694

Pan-BET

Ovarian cancer, peritoneal cancer, fallopian tube cancer

Recruiting

Phase II

In combination with Talazoparib

NCT05071937

ZEN-3694

Pan-BET

TNBC

Terminated (based on results from an interim futility analysis and not due to safety concerns)

Phase II

In combination with Talazoparib

NCT03901469⁴⁸⁵

ZEN-3694

Pan-BET

NUT carcinoma

Recruiting

Phase I/II

In combination with Cisplatin and Etoposide

NCT05019716

ZEN-3694

Pan-BET

Solid tumors, lymphomas

Recruiting

Phase I/II

In combination with Entinostat

NCT05053971

ZEN-3694

Pan-BET

CRPC

Completed (exhibits acceptable safety and efficacy)

Phase I/II

In combination with Enzalutamide

NCT02711956⁴⁸⁶

ZEN-3694

Pan-BET

CRPC

Enrolling by invitation

Phase I/II

In combination with Enzalutamide

NCT04145375

Trotabresib

Pan-BET

HER2-positive BC with CNS metastasis and leptomeningeal disease

Withdrawn (due to sponsors’ decision)

Phase I

In combination with Vinorelbine and radiation therapy

NCT06137651

Trotabresib

Pan-BET

Astrocytoma, glioblastoma

Terminated (due to a change in business objectives)

Phase I

—

NCT04047303¹¹⁰⁹

Trotabresib

Pan-BET

Solid tumors, NHL

Active, not recruiting (exhibits good tolerability and single-agent activity in advanced solid tumors according to disclosed data)

Phase I

—

NCT03220347^1110,1111

Trotabresib

Pan-BET

Pediatric cancer

Active, not recruiting

Phase I

BMS-986158 (active comparator)

NCT03936465

Trotabresib

Pan-BET

Solid tumors

Withdrawn (due to a change in business objectives)

Phase I

—

NCT05678283

Trotabresib

Pan-BET

Glioblastoma

Active, not recruiting

Phase I

In combination with Temozolomide and radiation therapy; radiation therapy (active comparator)

NCT04324840

Alobresib

Pan-BET

Solid tumors, lymphomas, HR-positive BC

Completed (reports pharmacokinetics and pharmacodynamics)

Phase I

With or without Exemestane or Fulvestrant

NCT02392611

Alobresib

Pan-BET

CRPC

Terminated (exhibits acceptable tolerability)

Phase I/II

With or without Enzalutamide

NCT02607228

GSK3358699

Pan-BET

Healthy volunteers

Terminated (due to strategic reasons)

Phase I

Placebo-controlled

NCT03426995¹¹¹²

TEN-010

Pan-BET

AML, MDS

Completed (exhibits insufficient activity as a single agent)

Phase I

—

NCT02308761¹¹¹³

TEN-010

Pan-BET

Solid tumors

Completed (exhibits evidence of target engagement and preliminary single-agent activity)

Phase I

—

NCT01987362¹¹¹⁴

TEN-010

Pan-BET

Ovarian cancer, TNBC

Terminated (due to development portfolio)

Phase I

In combination with Atezolizumab

NCT03292172

TEN-010

Pan-BET

MM

Completed (exhibits infrequent and short duration of clinical response rates)

Phase I

With or without Daratumumab

NCT03068351¹¹¹⁵

TEN-010

Pan-BET

DLBCL

Completed (unpublished)

Phase I

In combination with Venetoclax and Rituximab

NCT03255096¹¹¹⁶

ODM-207

Pan-BET

Solid tumors

Completed (exhibits safety at doses up to 2 mg/kg but has a narrow therapeutic window)

Phase I/II

—

NCT03035591¹¹¹⁷

ABBV-744

Pan-BET

Myelofibrosis

Terminated (due to strategic reasons)

Phase I

—

NCT03360006

ABBV-744

Pan-BET

AML

Active, not recruiting

Phase I

With or without Ruxolitinib or Navitoclax

NCT04454658

Birabresib

BRD2/3/4

Solid tumors

Completed (exhibits a favorable safety profile with clinical activity in NUT carcinoma)

Phase I

—

NCT02259114¹¹¹⁸

Birabresib

BRD2/3/4

AML, ALL, DLBCL, MM

Completed (exhibits evidence of clinical activity though does not meet objective response criteria in non-leukemia cohort)

Phase I

—

NCT01713582^1119,1120

Birabresib

BRD2/3/4

AML, DLBCL

Terminated (due to limited efficacy)

Phase I

—

NCT02698189

Birabresib

BRD2/3/4

NUT carcinoma, TNBC, NSCLC, CRPC

Terminated (due to limited efficacy)

Phase I

—

NCT02698176

Birabresib

BRD2/3/4

GBM

Terminated (due to limited efficacy)

Phase II

—

NCT02296476

Birabresib

BRD2/3/4

AML

Withdrawn

Phase I/II

Azacitidine (active comparator/in combination with Birabresib)

NCT02303782

Molibresib

BRD2/3/4

Solid tumors, lymphomas

Withdrawn (due to disapproved protocal)

Phase I

In combination with Entinostat

NCT03925428

Molibresib

BRD2/3/4

NUT carcinoma, solid tumors

Completed (exhibits acceptable safety)

Phase I

—

NCT01587703^1121,1122

Molibresib

BRD2/3/4

HR-positive HER2-negative BC

Terminated (due to meeting protocol-defined futility)

Phase I

In combination with Fulvestrant; placebo-controlled

NCT02964507¹¹²³

Molibresib

BRD2/3/4

CRPC

Terminated (due to meeting protocol-defined futility)

Phase I

In combination with Abiraterone plus Prednisone or Enzalutamide

NCT03150056

Molibresib

BRD2/3/4

Healthy volunteers

Completed (CYP3A enzymes play a major role in the elimination of Molibresib)

Phase I

In combination with Itraconazole or Rifampicin

NCT02706535¹¹²⁴

Molibresib

BRD2/3/4

Hematologic malignancies

Completed (exhibits antitumor activity but is limited by gastrointestinal and thrombocytopenia toxicities)

Phase II

—

NCT01943851¹¹²⁵

Molibresib

BRD2/3/4

SCLC, solid tumors

Withdrawn (due to reevaluation)

Phase II

In combination with Trametinib

NCT03266159

Molibresib

BRD2/3/4

NUT carcinoma

Withdrawn (due to disapproved protocal)

Phase I/II

In combination with Cisplatin, Etoposide, and Etoposide phosphate

NCT04116359

Mivebresib

BRD2/4/T

Myelofibrosis

Terminated (due to strategic reasons)

Phase I

WIth or without Ruxolitinib or Navitoclax

NCT04480086

Mivebresib

BRD2/4/T

BC, NSCLC, AML, MM PC, SCLC, NHL

Completed (exhibits good tolerability and potential efficacy in advanced solid tumors)

Phase I

With or without Venetoclax

NCT02391480^1126,1127

BAY1238097

BRD4-BD1; BRD2/3

Malignant tumors

Terminated

Phase I

—

NCT02369029

PLX-2853

BRD4

AML, MDS

Completed (unpublished)

Phase I

—

NCT03787498

PLX-2853

BRD4

Malignant tumors

Completed (unpublished)

Phase I

—

NCT03297424

PLX-2853

BRD4

CRPC

Terminated (due to business realignment)

Phase I/II

In combination with Abiraterone acetate plus Prednisone, or Olaparib

NCT04556617

PLX-2853

BRD4

Uveal melanoma

Withdrawn (drug company has withdrawn support)

Phase I/II

In combination with Trametinib

NCT05677373

PLX-2853

BRD4

Platinum-resistant ovarian carcinoma

Terminated (due to business realignment)

Phase I/II

With or without Carboplatin

NCT04493619

INCB054329

BRD4

Solid tumors, hematologic malignancies

Terminated (due to interindividual pharmacokinetic variability)

Phase I/II

—

NCT02431260¹¹²⁸

SYHA1801

BRD4

Solid tumors

Unknown

Phase I

—

NCT04309968

CC-95775

BRD4

AML, MDS, NHL

Completed (unpublished)

Phase I

With or without Azacitidine

NCT02543879

CC-95775

BRD4

Solid tumors, NHL

Completed (unpublished)

Phase I

—

NCT04089527

PLX51107

BRD4

Solid tumors, hematologic malignancies

Terminated (due to business reasons)

Phase I

—

NCT02683395

PLX51107

BRD4

AML, MDS

Completed (unpublished)

Phase I

In combination with Azacitidine

NCT04022785

PLX51107

BRD4

Acute GVHD

Terminated (due to sponsors’ decision)

Phase I/II

—

NCT04910152

BMS-986158

BRD4

Pediatric Cancer

Active, not recruiting

Phase I

BMS-986378 (active comparator)

NCT03936465

BMS-986158

BRD4

Myelofibrosis

Active, not recruiting

Phase I/II

In combination with Ruxolitinib or Fedratinib

NCT04817007

BMS-986158

BRD4

Solid tumors, hematologic malignancies

Completed (exhibits insufficient evidence of improvement)

Phase I/II

With or without Nivolumab

NCT02419417

BMS-986158

BRD4

MM

Recruiting

Phase I/II

In combination with Tazemetostat plus Dexamethasone or BMS-986158 plus Dexamethasone or Trametinib plus Dexamethasone, or Dexamethasone

NCT05372354

AZD5153

BRD4

Solid tumors, lymphomas

Completed (exhibits tolerable safety as monotherapy and in combination)

Phase I

With or without Olaparib

NCT03205176¹¹²⁹

AZD5153

BRD4

NHL, DLBCL

Completed (unpublished)

Phase I

In combination with Acalabrutinib; Acalabrutinib, Rituximab, plus Hu5F9-G4 (active comparator)

NCT03527147

AZD5153

BRD4

AML

Recruiting

Phase I/II

Umbrella study

NCT03013998

BI894999

BRD4-BD1/BD2

Malignant tumors, NUT carcinoma

Completed (exhibits preliminary antitumor effects and reports the maximum tolerated dose at different cohorts)

Phase I

—

NCT02516553¹¹³⁰

Apabetalone

BRD4-BD2

PAH

Completed (exhibits good tolerability and clinical benefits)

Early phase I

—

NCT03655704¹¹³¹

Apabetalone

BRD4-BD2

PAH

Not yet recruiting

Phase II

Placebo-controlled

NCT04915300

Apabetalone

BRD4-BD2

Atherosclerosis, CAD

Completed (exhibits good tolerability)

Phase II

Placebo-controlled

NCT01058018¹¹³²

Apabetalone

BRD4-BD2

T2DM

Completed (exhibits potential to against the disease development)

Phase II

Placebo-controlled

NCT01728467¹¹³³

Apabetalone

BRD4-BD2

Dyslipidemia, CAD

Completed (exhibits good tolerability)

Phase II

Placebo-controlled

NCT01423188¹¹³⁴

Apabetalone

BRD4-BD2

CAD

Completed (exhibits no significant improvement)

Phase II

Placebo-controlled

NCT01067820^{1134,1135,1136,1137}

Apabetalone

BRD4-BD2

Dyslipidemia, CAD

Terminated

Phase II

In combination with Rosuvastatin or Atorvastatin

NCT01863225

Apabetalone

BRD4-BD2

Chronic kidney failure

Not yet recruiting

Phase I/II

Placebo-controlled

NCT03160430

Apabetalone

BRD4-BD2

Fabry disease

Withdrawn (due to changed development priorities)

Phase I/II

—

NCT03228940

Apabetalone

BRD4-BD2

Healthy volunteers, dyslipidemia, atherosclerosis, CAD

Completed (unpublished)

Phase I/II

Placebo-controlled

NCT00768274

Apabetalone

BRD4-BD2

T2DM, CAD

Completed (fails to reduce the risk of major adverse cardiovascular events)

Phase III

In combination with Rosuvastatin or Atorvastatin; placebo-controlled

NCT02586155^{477,1137,1138,1139,1140,1141,1142}

Apabetalone

BRD4-BD2

COVID-19 infection

Terminated (fails to recruit subjects)

Phase II/III

Standard of care (active comparator/in combination with Apabetalone)

NCT04894266

NUV-868

BRD4-BD2

Solid tumors

Recruiting

Phase I/II

With or without Olaparib or Enzalutamide

NCT05252390

Pelabresib

BRD4-BD1

Malignant tumors

Completed (unpublished)

Phase I

—

NCT05391022

Pelabresib

BRD4-BD1

Lymphoma

Completed (exhibits good tolerability and inhibitory effects)

Phase I

—

NCT01949883¹¹⁴³

Pelabresib

BRD4-BD1

MM

Completed (unpublished)

Phase I

—

NCT02157636

Pelabresib

BRD4-BD1

Peripheral nerve tumor

Withdrawn (due to a lack of enrollment)

Phase II

—

NCT02986919

Pelabresib

BRD4-BD1

Myelofibrosis, AML, MDS, myeloproliferative disorders

Active, not recruiting (exhibits potential disease-modifying activity in myelofibrosis according to disclosed data)

Phase I/II

With or without Ruxolitinib

NCT02158858^484,1144

Pelabresib

BRD4-BD1

Malignant tumors

Not yet recruiting

Phase III

Placebo-controlled

NCT06401356

Pelabresib

BRD4-BD1

Myelofibrosis

Active, not recruiting

Phase III

In combination with Ruxolitinib, placebo-controlled

NCT04603495

TQB3617

BET

Malignant tumors

Unknown

Phase I

—

NCT05110807

TQB3617

BET

Myelofibrosis

Recruiting

Phase I/II

In combination with TQ05105

NCT06122831

TQB3617

BET

Esophageal squamous cell carcinoma

Not yet recruiting

Phase I/II

In combination with TQB2618, Paclitaxel, and Cisplatin, or Paclitaxel plus Cisplatin, or TQB2618 plus Penpulimab

NCT05834543

EP31670

P300(CBP)/BET

CRPC, NUT carcinoma

Recruiting

Phase I

—

NCT05488548¹¹⁴⁵