HDAC inhibitors 2017-05-19T11:28:32+00:00

To cite this dataset: M. Licheva, A. Neudolt, and I.M. Bennani-Baiti. Clinical trials of histone deacetylase inhibitors in cancer. Cancer Epigenetics Drug Database (CEDD) – Clinical data, v1.1. Cancer Epigenetics Society (https://ces.b2sg.org/cedd/clinical_hdaci/), 2016.

Here you will find drugs that have been approved by the US Food and Drug Administration (FDA) for the treatment of cancers, and/or the clinical trials in which they have or are being tested. Clinical trials are either Phase 0 (enroll a maximum of 15 patients or healthy volunteers who are administered a single sub-therapeutic dose to study the pharmacokinetics and tumor targeting of the drug), Phase I (enroll about 15-30 cancer patients to test if the new treatment is safe, to investigate the best way to administer the experimental drug, and whether cancer is responding to the treatment), Phase II (enroll 100 or less patients to test if the new drug is effective against a cancer, and to monitor side effects in a group of patients larger than in Phase I), Phase III (enroll hundreds to thousands of patients that are double-blindly assigned either to the experimental or standard treatment to test if the new treatment is better than a standard treatment protocol), or Phase IV (to gather more information about the long-term benefits and side effects of the new drug after it has been FDA-approved). Details on the clinical trials and whether they are ongoing, closed or terminated, and whether results have been posted can be found in the provided links below.

Drug* Targets FDA-approved & Clinical trials**
Belinostat (PXD101) HDAC1, HDAC2, HDAC3, HDAC6 FDA-approved: RR-PTCL

Trials: Adult T-cell Leukemia (II: NCT02737046), ALL (I: NCT01075425, NCT00351975), AML (I: NCT01075425, NCT02381548; I/II: NCT00878722; II: NCT00357032), bladder cancer (I/II: NCT00421889), Brenner tumor (II: NCT00993616), CML (I: NCT01075425, NCT02381548, NCT00351975), fallopian tube cancer (I/II: NCT00421889; II: NCT00993616), hepatocellular carcinoma (I/II: NCT00321594), liver cancer (I/II: NCT00321594), lymphoma (I: NCT00413075, NCT02142530, NCT00348985), MDS (I: NCT01075425; II: NCT00357162), mesothelioma (II: NCT00365053, NCT00365053), MM (II: NCT00431340, NCT00131261), NSCLC (I/II: NCT01310244, NCT01188707), ovarian cancer (I/II: NCT00421889; II: NCT00993616), PTCL (I: NCT01839097; II: NCT00865969), relapsed/refractory solid tumor (I: NCT02679131NCT02680795), solid tumor (I: NCT01317927), thymoma (I/II: NCT01100944, II; NCT00589290, NCT00589290).

Panobinostat (LBH589; Farydak) HDAC1, HDAC2,
FDA-approved: MM

Trials: ALL (I/II: NCT00918333), AML (I: NCT01242774; I/II: NCT01451268, NCT00840346NCT02676323; II: NCT00946647), breast cancer (I: NCT00632489, NCT00788931, NCT00567879; I/II: NCT01105312), brain metastasis (I: NCT01324635), CML (I: NCT00686218; II: NCT00946647), CMML (I: NCT01613976), CTCL (I: NCT00412997; II/III: NCT00490776), DIPG (I: NCT02717455), HCC (I: NCT00823290), head and neck cancer (I: NCT00738751), Hodgkin’s lymphoma (I/II: NCT01169636; II: NCT01460940; III: NCT01034163), lung cancer (I: NCT00738751), lymphoma (I/II: NCT00918333; II: NCT01261247), MDS (I/II: NCT01451268, NCT00691938NCT02676323; II: NCT00594230), meningioma (I: NCT01324635), metastatic melanoma (I: NCT02032810; I/II: NCT00925132, NCT02802163; II: NCT02722941, NCT02756663), MM (I: NCT01549431NCT01965353; II: NCT01651039NCT02654990; III: NCT01023308), neuroendocrine tumor (II: NCT00985946), NSCLC (I: NCT01336842, NCT01005797), pancreatic cancer (II: NCT01056601), prostate cancer (I: NCT00663832; I/II: NCT00878436), renal cell carcinoma (I: NCT01005797; I/II: NCT01582009; II: NCT00550277), soft tissue sarcoma (I: NCT01005797), solid tumors (I: NCT00878904), thyroid cancer (II: NCT01013597).

Romidepsin (Depsipeptide; FK-228; Istodax®) HDAC1, HDAC2, HDAC3, HDAC8 FDA-approved: CTCL, PTCL

Trials: Bladder cancer (II: NCT02788201), CTCL (NCT02296398; I: NCT02181218NCT02757248NCT02616965; I/II: NCT02512497), lymphoid malignancies (I/II: NCT01998035, NCT01998035, NCT01947140), Hodgkin’s lymphoma (I/II: NCT01998035, NCT01998035, NCT01947140), lung cancer (I/II: NCT01302808), lymphoma (I: NCT02783625), metastatic breast cancer (I/II: NCT01938833, NCT02393794), MM (I/II: NCT01979276, NCT01755975), non-Hodgkin’s lymphoma (I: NCT01738594; I/II: NCT01998035, NCT01998035, NCT01947140), NSCLC (I NCT00037817; II: NCT00020202), pancreatic cancer (I: NCT00379639), prostate cancer (II: NCT00106418, NCT00106301), PTCL (I: NCT01590732, NCT01846390, NCT02757248, NCT02181218, NCT02783625; I/II: NCT00426764; I/II: NCT02512497; II: NCT01822886; III: NCT01796002), recurrent ovarian epithelial cancer (II: NCT00085527), T-large granulocytic leukemia (I/II: NCT02512497), T-lymphoblastic leukemia/lymphoma (I/II: NCT02512497), T-prolymphocytic leukemia (I/II: NCT02512497), thyroid cancer (II: NCT00098813), urinary bladder neoplasm (II: NCT02788201), urothelial carcinoma (II: NCT02788201), virally mediated cancers and liposarcoma (I: NCT01537744).

Vorinostat (SAHA; MK0683; Zolinza®) HDAC1, HDAC2, HDAC3, HDAC6 FDA-approved: CTCL

Trials: AML (I: NCT00875745, NCT02412475, NCT00275080; I/II: NCT00392353; II: NCT00948064), bladder cancer (II: NCT02788201), BRAF V600 mutated melanoma (II: 2015-005840-33) brain metastases (I: NCT00838929; I/II: NCT01266031), breast cancer (NCT01720602), nasal natural killer T-cell lymphoma (I: NCT00336063), nasopharyngeal carcinoma (I: NCT00336063), cell carcinoma of the oropharynx (I: NCT01064921), gastrointestinal cancer (I/II: NCT01045538), diffuse intrinsic pontine glioma (I: NCT02420613), glioblastoma (I: NCT00268385; I/II: NCT00731731, NCT01189266), liver cancer (I: NCT01075113), lung cancer (I/II: NCT02638090), lymphoma (I: NCT00275080; I/II: NCT02589145; II: NCT00992446), malignant solid tumor (I: NCT01023737), mantle cell lymphoma (II: NCT01578343), MDS (I: NCT00875745; II: NCT00948064), melanoma (I/II: NCT02836548; II: NCT00121225; IV: NCT02645149), MM (I: NCT00111813; II: NCT00773838; III: NCT00773747), neuroblastoma (I: NCT01208454), NSCLC (I: NCT02151721; I/II: NCT00251589, NCT02638090), pancreatic cancer (I: NCT00983268, NCT02349867), renal cell carcinoma (I/Ib: NCT02619253), soft tissue sarcoma (II: NCT00918489), urinary bladder neoplasm (I/Ib: NCT02619253; II: NCT02788201), urothelial carcinoma (II: NCT02788201).

4SC-202 HDAC1, HDAC2, HDAC3 Hematological malignancies (I: NCT01344707).
Abexinostat (S78454; PCI-24781) HDAC1, HDAC2, HDAC3, HDAC6, HDAC10 CLL (I: NCT00562224, I/II: 2014-002251-26, 2009-013691-47), Hodgkin’s lymphoma (I: NCT00562224, NCT01149668; I/II: NCT2014-002251-26, NCT2009-013691-47, NCT00724984), lymphoma (I/II: NCT00724984), metastatic solid tumors (I: NCT01543763), MM (I: NCT00562224NCT01149668), neoplasms (I: NCT00473577), non-Hodgkin’s lymphoma (I: NCT00562224NCT01149668; I/II: 2014-002251-26, 2009-013691-47, NCT00724984), soft tissue sarcoma (I/II: NCT01027910).
AR-42 HDAC Acoustic neuroma (0: NCT02282917), AML (I: NCT01798901NCT01798901), CLL (I: NCT01129193),  lymphoma (I: NCT01129193NCT01129193), meningioma (0:  NCT02282917), MM (1: NCT01129193), NF2 (0: NCT02282917), plasma cell myeloma (I: NCT02569320), renal cell carcinoma (I: NCT02795819), soft tissue carcinoma (I: NCT02795819), vestibular schwannoma (0: NCT02282917).
CG200745 HDAC Advanced solid tumors (I: NCT01226407), pancreatic neoplasm (I/II: NCT02737228).
Chidamide (Epidaza; CS055; HBI-8000) HDAC Breast cancer (III: NCT02482753), DLBCL (II: NCT02733380NCT02753647), Melanoma (I/II: NCT02718066), non-Hodgkin’s lymphoma (I: NCT02697552), NSCLC (II: NCT01836679, NCT02815007NCT02718066), PTCL (I: NCT02809573;  II: NCT02753543), renal cell carcinoma (I/II: NCT02718066).
CHR-3996 HDAC Class I MM (I/IIa: 2011-001914-33), solid tumors (I: NCT00697879).
CI-994 (Acetyldinaline; p-N-acetyldinaline; Tacedinaline) HDAC Class I Lung cancer (III: NCT00005093), MM (II: NCT00005624), pancreatic cancer (II: NCT00004861).
CUDC‑101 HDAC Breast cancer (Ib: NCT01171924), gastric cancer (Ib: NCT01171924), head and neck cancer (I: NCT01384799; Ib: NCT01171924), liver cancer (Ib: NCT01171924), MM (I: NCT01742988), NSCLC (Ib: NCT01171924), solid tumors (I: NCT01702285, NCT00728793).
Entinostat (MS-275) HDAC1, HDAC2 ALL (I: NCT01132573), AML (I: NCT00101179; II: NCT00313586, NCT01305499), brain stem neoplasm (I: NCT02780804), breast cancer (I: NCT01434303, NCT02453620, NCT02623751NCT02820961NCT02833155; I/II: NCT02708680;  II: NCT01349959; III: NCT02115282), CML (II: NCT00313586), colon and rectal cancer (II: NCT01105377), Hodgkin’s lymphoma (II: NCT00866333),  lymphoma (I: NCT02780804), MDS (I: NCT00101179; II: NCT00313586), melanoma (I/II: NCT02437136; II: NCT00185302), metastatic uveal melanoma (II: NCT02697630), NSCLC (I/II: NCT00387465, NCT02437136; II: NCT01935947, NCT01928576), pineal region neoplasm (I: NCT02780804), recurrent central nervous system neoplasm (I: NCT02780804), refractory central nervous system neoplasm (I: NCT02780804), renal cell carcinoma (I/II: NCT01038778), solid neoplasm (I: NCT02780804), visual pathway glioma (I: NCT02780804).
Givinostat (ITF2357 , INN) HDAC Class I/II Hodgkin’s lymphoma (I/II: NCT00792467), myelofibrosis (II: NCT00606307, NCT01761968, NCT01761968).
Mocetinostat (MGCD0103) HDAC1, HDAC2, HDAC3 Advanced solid tumor (I/II: NCT02805660), AML (I/II: NCT00324220), CLL (II: NCT00431873), DLBCL (I/II: NCT02282358), FL (I/II: NCT02282358), Hodgkin’s lymphoma (I/II: NCT02429375), MDS (I: NCT00324194, I/II: NCT02018926, NCT00324220), metastatic leiomyosarcoma (II: NCT02303262), non-Hodgkin’s lymphoma (I: NCT00323934),  NSCLC (I/II: NCT02805660), urothelial carcinoma (II: NCT02236195).
Pivanex (AN-9, pivaloyloxymethyl butyrate) HDAC CLL (II: NCT00083473), malignant melanoma (I/II: NCT00087477), NSCLC (II: NCT00073385), SLL (II: NCT00083473).
Pracinostat (SB939) Pan HDAC AML (II: NCT01912274), hematological malignancies (I: NCT00741234), MDS (I: NCT00741234; II: NCT01993641, NCT01873703), myelofibrosis (II: NCT02267278) prostate cancer (II: NCT01075308).
Quisinostat (JNJ-26481585) HDAC Advanced solid tumors and lymphomas (I: NCT00677105), CTCL (II: NCT01486277), epithelial ovarian cancer (I: NCT02728492), MM (I: NCT01464112), NSCLC (I: NCT02728492) .
Resminostat (4SC-201) HDAC1, HDAC3, HDAC6 Colorectal cancer (I/II: NCT01277406), hepatocellular carcinoma (I/II: NCT02400788; II: NCT00943449), Hodgkin’s lymphoma (II: NCT01037478).
Ricolinostat (ACY-1215) HDAC6 Breast cancer (I: NCT02632071), fallopian tube cancer (I: NCT02661815), lymphoma (I/II: NCT02091063), MM (Ib: NCT01583283, NCT02189343; I/II: NCT01323751, NCT01997840), ovarian cancer (I: NCT02661815), primary peritoneal cancer (I: NCT02661815), recurrent CLL (I: NCT02787369).
Sodium phenyl butyrate HDAC ALL (III: NCT02112916), brain tumors (I: NCT00001565; II: NCT00006450), colorectal cancer (I/II: NCT00002796), leukemia (I: NCT00006239; II: NCT00006019, NCT00006240), lung cancer (II: NCT00006019), lymphoma (I: NCT00002909, NCT00005639; II: NCT00006019), MDS (I: NCT00004871, NCT00006239; II: NCT00006019), MM and plasma cell neoplasm (II: NCT00006019), prostate cancer (II: NCT00006019).
Trichostatin A (TSA) HDAC1, HDAC2 MDS (II: NCT00520468).
Valproic acid HDAC1, HDAC2 AML (I: NCT00079378; II: NCT02124174, NCT00414310), brain tumor (II: NCT00302159; IV: NCT02670161), breast cancer (II: NCT01900730), cervical cancer (III: NCT02446652), chronic myelogenous leukemia (II: NCT01011998), CLL (I: NCT00079378), colorectal cancer (I/II: NCT01898104), DIPG (II: NCT02758366), GBM (I: NCT02648633; II: NCT02758366),  high grade glioma (II: NCT00302159), MDS (II: NCT02124174, NCT00414310), melanoma (II: NCT02068586), nasopharyngeal carcinoma (I: NCT02761291) NSCLC (I: NCT00084981), ovarian cancer (I: NCT02520115), SCLC (II: NCT00759824), SLL (I: NCT00079378), squamous cell carcinoma of head and neck (II: NCT02624128).

© CEDD v1.1, Cancer Epigenetics Society & B² Scientific Group, Ltd. All rights reserved, 2016.

*This database is continuously expanding and updated; please check periodically. Please feel free to write to us at info@ces.b2sg.org under the Subject line: CEDD suggestion, with any suggestion of additional drugs, or to report new epigenetic targets, clinical trials, or FDA-approved cancer applications.

**Abbreviations: ALL: Acute Lymphoblastic Leukemia; AML: Acute Myeloid Leukemia; CLL: Chronic Lymphocytic Leukemia; CML: Chronic Myeloid Leukemia; CMML: Chronic Myelomonocytic Leukemia; CTCL: Cutaneous T-Cell Lymphoma; DIPG: Diffuse Intrinsic Pontine Glioma; DLBCL: Diffuse large B-cell lymphoma; FL: Follicular lymphoma; GBM: Glioblastoma Multiforme; HCC: Hepatocellular carcinoma; MDS: Myelodysplastic Syndrome; MM: Multiple Myeloma; NF2: Neurofibromatosis Type 2; NSCLC: Non-Small Cell Lung Carcinoma; PTCL: Peripheral T-cell Lymphoma; RR-PTCL: PTCL: Relapsed or refractory Peripheral T-cell Lymphoma; SCLC: Small Cell Lung Carcinoma; SLL: Small Lymphocytic Lymphoma. Roman numbers between brackets denote Phase I, II, or III clinical trials.

  • Cancer Epigenetics Society: Belinostat
  • Cancer Epigenetics Society: Panobinostat
  • Cancer Epigenetics Society: Romidepsin
  • Cancer Epigenetics Society: Vorinostat
  • Cancer Epigenetics Society: AR-42
  • Cancer Epigenetics Society: CUDC-101