Table 2 Epitherapy in clinical trials for cancer and CVDs
| Drug | Conditions | Study type | Study title | Aim | Status/phase | ID |
|---|---|---|---|---|---|---|
| Drugs in clinical trial | ||||||
| SGI-110 (guadecitabine) | AML |
302 participants, Interventional, Randomized, Parallel Assignment, Treatment | Phase 3 Randomized, Open-Label Study of Guadecitabine vs Treatment Choice in Previously Treated Acute Myeloid Leukemia | Multicenter, randomized, open-label, parallel-group study of guadecitabine vs treatment choice | Active/Phase 3 | NCT02920008 |
|
MDS CMML |
408 participants, Interventional, Randomized, Parallel Assignment, Treatment | Guadecitabine (SGI-110) vs Treatment Choice in Adults With MDS or CMML Previously Treated With HMAs | To evaluate the efficacy and safety of guadecitabine in MDS or CMML who failed or relapsed after treatment with azacitidine, decitabine, or both | Active/Phase 3 | NCT02907359 | |
| AML |
815 participants, Interventional, Randomized, Parallel Assignment, Treatment | SGI-110 in Adults With Untreated Acute Myeloid Leukemia (AML), Not Considered Candidates for Intensive Remission Induction | To compare efficacy and safety between SGI-110 and treatment in adults with previously untreated AML |
Completed/ Phase 3 | NCT02348489 | |
| RX-3117 | MPC |
46 participants, Interventional, N/A, Single group Assignment, Treatment | RX-3117 in Combination With Abraxane® in Subjects With Metastatic Pancreatic Cancer | To determine the safety profile, dose modification, and pharmacokinetics of oral RX-3117 administered in combination with Abraxane® to subjects with metastatic pancreatic cancer |
Completed/ Phase 1/2 | NCT03189914 |
|
Solid tumor Metastatic Bladder Cancer |
124 participants, Interventional, N/A, Single group Assignment, Treatment | Dose-Finding and Safety Study for Oral Single-Agent to Treat Advanced Malignancies | To determine the maximum tolerated dose in metastatic solid tumors and to estimate anti-tumor activity in subjects with relapsed or refractory pancreatic or advanced bladder cancer |
Completed/ Phase 1/2 | NCT02030067 | |
| INCB059872 |
Solid tumors Advanced Malignancies Metastatic Cancer |
70 participants, Interventional, N/A, Single group Assignment, Treatment | Azacitidine Combined With Pembrolizumab and Epacadostat in Subjects With Advanced Solid Tumors (ECHO-206) | To evaluate the safety and tolerability of INCB059872 with pembrolizumab and epacadostat |
Completed Phase 1/2 | NCT02959437 |
| Solid tumors and hematologic malignancy |
215 participants, Interventional, N/A, Single group Assignment, Treatment | An Open-Label, Dose-Escalation/Dose-Expansion Safety Study of INCB059872 in Subjects With Advanced Malignancies | To determine the safety, tolerability, efficacy, PK, PD, and the recommended dose(s) of azacitadine and all-trans retinoic acid in AML and in combination with nivolumab in SCLC |
Recruiting Phase 1/2 | NCT02712905 | |
| CI-994 | MM |
6 participants, Interventional, N/A, Single group Assignment, Treatment | CI-994 in Treating Patients With Advanced Myeloma | To study the effectiveness of CI-994 in treating patients who have advanced myeloma |
Completed Phase 2 | NCT00005624 |
| Lung cancer |
Interventional, Randomized, Treatment | Gemcitabine With or Without CI-994 in Treating Patients With Advanced Non-small Cell Lung Cancer | To compare the effectiveness of gemcitabine with or without CI-994 in treating patients who have advanced non-small cell lung cancer |
Completed Phase 3 | NCT00005093 | |
| Pancreatic cancer |
Interventional, Randomized, Treatment | Gemcitabine With or Without CI-994 in Treating Patients With Advanced Pancreatic Cancer | To compare the effectiveness of gemcitabine with or without CI-994 in treating patients who have advanced pancreatic cancer |
Completed Phase 2 | NCT00004861 | |
|
EPZ-5676 (Pinometostat) | AML with 11q23 rearrangement |
36 participants, Interventional, N/A, Single group Assignment, Treatment | Pinometostat and Azacitidine in Treating Patients With Relapsed, Refractory, or Newly Diagnosed Acute Myeloid Leukemia With 11q23 Rearrangement | To study the side effects and the dose of pinometostat with azacitidine in AML with 11q23 rearrangement |
Recruiting Phase 1/2 | NCT03701295 |
| AML with 11q23 rearrangement |
37 participants, Interventional, N/A, Single group Assignment, Treatment | Pinometostat With Standard Chemotherapy in Treating Patients With Newly Diagnosed Acute Myeloid Leukemia and MLL Gene Rearrangement | To study the side effects and the dose of pinometostat in AML with 11q23 rearrangement |
Recruiting Phase 1/2 | NCT03724084 | |
| TCP | AML |
16 participants, Interventional, N/A, Single group Assignment, Treatment | Phase I/II Trial of ATRA and TCP in Patients With Relapsed or Refractory AML and no Intensive Treatment is Possible | To study the feasibility, pharmacodynamics, and effectivity of ATRA and TCP in AML | Phase 1/2 | NCT02261779 |
|
AML, MDS |
60 participants, Interventional, N/A, Single group Assignment, Treatment | Study of Sensitization of Non-M3 AML Blasts to ATRA by Epigenetic Treatment With Tranylcypromine (TCP) | To study the maximum tolerated and the efficacy dose of TCP with ATRA and Cytarabine |
Recruiting Phase 1/2 | NCT02717884 | |
| CPI-0610 |
Myelofibrosis AML, MDS/MPD, MDS |
271 participants, Interventional, Non-Randomized, Parallel Assignment, Treatment | A Phase 2 Study of CPI-0610 With and Without Ruxolitinib in Patients With Myelofibrosis | Open-label, sequential dose escalation study of CPI-0610 and open-label study of CPI-0610 with and without Ruxolitinib in patients with Myelofibrosis |
Recruiting Phase 1/2 | NCT02158858 |
| Rocilinostat (ACY-1215) | MM |
101 participants, Interventional, Single Group Assignment, Treatment | ACY-1215 (Ricolinostat) in Combination With Pomalidomide and Low-dose Dex in Relapsed-and-Refractory Multiple Myeloma | To evaluate the side effects, determine the best dose and the response rate of ACY-1215 in combination with Pomalidomide and low-dose dexamethasone in patients with relapsed-and-refractory multiple myeloma |
Active, not recruiting Phase 1b/2 | NCT01997840 |
| Mocetinostat | Urothelial carcinoma |
17 participants, Interventional, N/A, Single group Assignment, Treatment | Study of Mocetinostat in Patients With Urothelial Carcinoma Having Inactivating Alterations of Specific Genes | To evaluate whether the number of patients responding to treatment is substantially higher than other available treatments | Completed/ Phase II | NCT02236195 |
| Hodgkin’s lymphoma |
51 participants, Interventional, Non-Randomized, Single group Assignment, Treatment | Study of MGCD0103 (MG-0103) in Patients With Relapsed or Refractory Hodgkin’s Lymphoma | To study the effect of Mocetinostat on patients with relapsed and refractory Hodgkin’s lymphoma | Terminated/ Phase II | NCT00358982 | |
| CPI-1205 | mCRPC |
242 participants, Interventional, Randomized, Parallel Assignment, Treatment | ProSTAR: A Study Evaluating CPI-1205 in Patients With Metastatic Castration Resistant Prostate Cancer | To study the oral administration of CPI-1205 in combination with enzalutamide or abiraterone/prednisone in male patients with mCRPC | Active, not recruiting/ Phase I/II | NCT03480646 |
| Advanced Solid Tumors |
24 participants, Interventional, N/A, Single group Assignment, Treatment | ORIOn-E: A Study Evaluating CPI-1205 in Patients With Advanced Solid Tumors | To determine the maximum tolerated dose and recommended phase 2 dose of CPI-1205 + ipilimumab in patients with advanced solid tumors | Active, not recruiting/ Phase I/II | NCT03525795 | |
| Approved epi-drug ongoing in clinical trial for other treatments | ||||||
| Vorinostat | ALL, LLy |
1000 participants, Interventional, Randomized, Parallel Assignment, Treatment | Total Therapy XVII for Newly Diagnosed Patients With Acute Lymphoblastic Leukemia and Lymphoma | To study a novel strategies based on inherited and acquired leukemia-specific genomic features and treatment to improve the cure rate and quality of life of ALL and LLy children |
Recruiting/ Phase2/3 | NCT03117751 |
| MM |
637 participants, Interventional, Randomized, Single group Assignment, Treatment | Study of Vorinostat (MK-0683) an HDAC Inhibitor, or Placebo in Combination with Bortezomib in Patients With Multiple Myeloma (MK-0683-088 AMN) | To study of the efficacy and safety of bortezomib administered in combination with vorinostat in patients with relapsed or refractory MM |
Complete/ Phase 3 | NCT00773747 | |
| MLC |
662 participants, Interventional, Randomized, Single group Assignment, Treatment | Suberoylanilide Hydroxamic Acid (Vorinostat, MK-0683) Versus Placebo in Advanced Malignant Pleural Mesothelioma (0683-014 AM5, EXT1) | To study the safety, tolerability, and anti-tumor effectiveness of vorinostat in oral administration, for MLC |
Complete/ Phase 3 | NCT00128102 | |
| Panobinostat | AML, MDS |
350 participants, Interventional, Randomized, Parallel Assignment Treatment | Panobinostat Maintenance After HSCT for High-risk AML and MDS | To compare maintenance treatment with panobinostat versus the standard approach of preemptive DLI alone in patients with poor-risk AML/MDS |
Recruiting/ Phase 3 | NCT04326764 |
| PMF, CIM, post-PVMF, PET-MF, SR-GVHD |
356 participants, Interventional, Non-Randomized, Parallel Assignment Treatment | CINC424A2X01B Rollover Protocol | To study the efficacy of ruxolitinib and panobinostat in combination for patients that use the study treatment based on the parent protocol | Recruiting/ Phase 4 | NCT02386800 | |
| Vidaza | AML |
488 participants, Interventional, Randomized, Parallel Assignment, Treatment | Study of Vidaza Versus Conventional Care Regimens for the Treatment of Acute Myeloid Leukemia (AML) | To compare the effect of azacitidine to conventional care regimens in AML patients |
Complete/ Phase 3 | NCT01074047 |
| AML |
47 participants, Interventional, Non-Randomized, Parallel Assignment, Treatment | zacitidine Combined With Homoharringtonie in AML | To validate the efficacy and safety advantages of the regimens that contain homoharringtonie and azacitidine, and to determine which regimen would receive more living benefits in AML | Recruiting/ Phase 3 | NCT04248595 | |
|
AML, MDS, CMML |
187 participants, Interventional, Randomized, Single group Assignment, Treatment | Controlled Study of Post-transplant Azacitidine for Prevention of Acute Myelogenous Leukemia and Myelodysplastic Syndrome Relapse (VZ-AML-PI-0129) | To study if Vidaza will help to control the disease in patients with AML, CMML, or MDS after a stem cell transplant |
Complete/ Phase 3 | NCT00887068 | |
|
AML, MDS, CML |
114 participants, Interventional, Randomized, Parallel Assignment, Treatment | Azacytidine + HAG Regimen vs. Azacytidine for Elderly Patients With Newly Diagnosed Myeloid Malignancy | To explore the efficacy and safety of azacytidine and HAG regimen versus azacytidine for elderly patients with Newly Diagnosed MDS/AML/CMML in China | Recruiting/ Phase 4 | NCT03873311 | |
| Decitabine | T-ALL, T-LBL, T/M-MPAL |
100 participants, Interventional, N/A, Single group Assignment Treatment | Study of Decitabine Combined With HAAG Regimen in Newly Diagnosed ETP-ALL/LBL, T/M-MPAL and ALL/LBL With Myeloid or Stem Cell Markers Patients | To evaluate the efficacy and safety of decitabine combined with HAAG | Recruiting/ Phase 3 | NCT04446130 |
| DLBCL |
60 participants, Interventional, Randomized, Parallel Assignment Treatment | A Clinical Trial of Decitabine in Relapse and Refractory Diffuse Large B Cell Lymphoma | To evaluate the safety, tolerability, and clinical effects of decitabine with R ± DHAP | Recruiting/ Phase 4 | NCT03579082 | |
| Tazametostat | Solid tumor |
49 participants, Interventional, N/A, Single group Assignment Treatment | Tazemetostat in Treating Patients With Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphoma, or Histiocytic Disorders With EZH2, SMARCB1, or SMARCA4 Gene Mutations (A Pediatric MATCH Treatment Trial) | To study the action of tazemetostat in patients with brain tumors, solid tumors, non-Hodgkin lymphoma, or histiocytic disorders that have come back (relapsed) or do not respond to treatment (refractory) and have EZH2, SMARCB1, or SMARCA4 gene mutations | Phase 2 | NCT03213665 |
| Drug repositioning in CVDs | ||||||
| Trimetazidine | HFpEF |
25 participants, Interventional, Randomized, Placebo-controlled | TrimetaziDine as a Performance-enhancING drug in Heart Failure with Preserved Ejection Fraction | To assess whether trimetazidine improves LV diastolic function by improving myocardial energy metabolism in HFpEF |
Ongoing/ Phase 2 |
EU Clinical Trial Register: 2018-002170-52; NTR registration: NL7830 [van de Bovenkamp AA, |
| PAH |
26 participants, Interventional, Randomized, Placebo-controlled | The Role of Trimetazidine on Right Ventricle Function in Pulmonary Arterial Hypertension (TRIMETA-PH) | To evaluate the effect of trimetazidine versus placebo in addition to standard PAH regime on right ventricular function | Completed/Phase 2/ 3 | NCT03273387 | |
| Tocilizumab | PAH |
29 participants, Interventional, Single‐arm | A Therapeutic Open Label Study of Tocilizumab in the Treatment of Pulmonary Arterial Hypertension (TRANSFORM-UK) | To assess the safety and efficacy of tocilizumab in PAH | Completed/Phase 3 | NCT02676947 |
| Metformin | MetS |
40 participants, Interventional, Randomized, Placebo-controlled | Combination of Metformin/Inulin vs Inulin on Adiponectin in Metabolic Syndrome | To compare the effect of the administration of Metformin/agave inulin vs. Agave inulin on adiponectin in patients with MetS |
Completed/ Phase 3 | NCT02773927 |
|
T2D, HF |
30 participants, Observational, Cohort, Prospective | Lipid Accumulation in Heart Transplant From Non-diabetic Donors to Diabetic Recipients (DCM-AHEAD) | To evaluate in the explanted diabetic heart the presence of possible cellular alterations attributable to the diabetic disease discerning from treated and non-treated with metformin | Completed/NA | NCT03546062 | |
| PAH |
1899 participants, Observational, Cohort, Prospective | Hormonal, Metabolic, and Signaling Interactions in PAH | To evaluate if an optimal treatment of the dysfunctional metabolic pathways underlying PAH may improve pulmonary vascular function and consequences of the disease | Recruiting/NA | NCT01884051 | |
|
Apabetalone (RVX 208) | PAH |
10 participants, Interventional, Single‐arm, 2‐center study | Apabetalone for Pulmonary Arterial Hypertension: a Pilot Study | To evaluate the possible efficacy of apabetalone in treating PAH | Recruiting/Early Phase 1 | NCT03655704 |
| T2D-CHD |
2425 participants, Interventional, Randomized, Placebo-controlled | Effect of RVX000222 on Time to Major Adverse Cardiovascular Events in High-Risk T2DM Subjects With CAD (BETonMACE) | To determine whether RVX000222 in high-risk type 2 diabetes mellitus patients with coronary artery disease increases the time to major adverse cardiovascular events | Phase 3/Active | NCT02586155 | |
|
n-3 PUFA or Rosuvastatin | HF |
6975 participants, Interventional, Randomized, Placebo-controlled | GISSI-HF- Effects of n-3 PUFA and Rosuvastatin on Mortality-Morbidity of Patients With Symptomatic CHF | To demonstrate that, in HF patients treated at the best of recommended therapies, long-term administration of n-3 PUFA or rosuvastatin is more effective than the corresponding placebo |
Completed/ Phase 3 |
NCT00336336 Tavazzi et al. [128] |