Clinical Trials

CPI-613 (Devimistat) has been evaluated in numerous clinical trials spanning Phase I through Phase III for solid tumors—including pancreatic adenocarcinoma, biliary tract carcinoma, colorectal cancer, and clear cell sarcoma—as well as hematologic malignancies like lymphoma and leukemia. Sponsored by academic institutions such as Memorial Sloan Kettering Cancer Center and Northwestern University alongside industry partners like Cornerstone Pharmaceuticals, these studies encompass recruitment statuses ranging from actively recruiting to completed, terminated, and withdrawn.

NCT Number Recruitment Conditions Sponsor/Collaborators Start Date Phases
NCT05733000 RECRUITING
Advanced Biliary Tract Carcinoma; Advanced Colorectal Carcinoma; Advanced Gastroesophageal Junction Adenocarcinoma; Advanced Lung Adenocarcinoma; Advanced Malignant Solid Neoplasm; Advanced Ovarian Carcinoma; Advanced Pancreatic Carcinoma; Advanced Urothelial Carcinoma; Clinical Stage III Gastroesophageal Junction Adenocarcinoma AJCC v8; Clinical Stage IV Gastroesophageal Junction Adenocarcinoma AJCC v8; Metastatic Biliary Tract Carcinoma; Metastatic Colorectal Carcinoma; Metastatic Gastroesophageal Junction Adenocarcinoma; Metastatic Lung Adenocarcinoma; Metastatic Ovarian Carcinoma; Metastatic Pancreatic Carcinoma; Metastatic Urothelial Carcinoma; Refractory Biliary Tract Carcinoma; Refractory Colorectal Carcinoma; Refractory Gastroesophageal Junction Adenocarcinoma; Refractory Lung Adenocarcinoma; Refractory Ovarian Carcinoma; Refractory Pancreatic Carcinoma; Refractory Urothelial Carcinoma; Stage II Pancreatic Cancer AJCC v8; Stage III Colorectal Cancer AJCC v8; Stage III Lung Cancer AJCC v8; Stage III Ovarian Cancer AJCC v8; Stage III Pancreatic Cancer AJCC v8; Stage IV Colorectal Cancer AJCC v8; Stage IV Lung Cancer AJCC v8; Stage IV Ovarian Cancer AJCC v8; Stage IV Pancreatic Cancer AJCC v8
Northwestern University
 2023-03-08 PHASE2
NCT05926206 WITHDRAWN
Metastatic Pancreatic Adenocarcinoma
University of Michigan Rogel Cancer Center
 2023-07 PHASE1; PHASE2
NCT03793140 COMPLETED
Lymphoma; Leukemia
Memorial Sloan Kettering Cancer Center
 2018-12-31 PHASE2
NCT06518538 WITHDRAWN
Pancreatic Cancer Stage IV
Cornerstone Pharmaceuticals
 2024-08-15 PHASE2
NCT05325281 TERMINATED
Pancreas Adenocarcinoma
Medical College of Wisconsin
 2022-10-31 PHASE1
NCT05854966 WITHDRAWN
Acute Myeloid Leukemia, in Relapse; Acute Myeloid Leukemia Refractory; Granulocytic Sarcoma
Wake Forest University Health Sciences
 2024-12 PHASE2
NCT03699319 COMPLETED
Pancreatic Cancer
David Bajor, MD
 2018-12-07 PHASE1; PHASE2
NCT04203160 TERMINATED
Biliary Tract Cancer
University of Michigan Rogel Cancer Center
 2020-06-23 PHASE1; PHASE2
NCT05070104 WITHDRAWN
C04.588.274.476.411.307
Cornerstone Pharmaceuticals
 2023-03-30 PHASE1
NCT05926206 Withdrawn
Metastatic Pancreatic Adenocarcinoma
University of Michigan Rogel Cancer Center
 2023-07 Phase 1|Phase 2
NCT04217317 TERMINATED
Relapsed T-Cell Lymphoma; Refractory T-Cell Lymphoma; Non Hodgkin Lymphoma
Wake Forest University Health Sciences
 2020-09-16 PHASE2
NCT04593758 COMPLETED
Sarcoma, Clear Cell
Cornerstone Pharmaceuticals
 2021-09-01 PHASE1; PHASE2
NCT03929211 WITHDRAWN
Myelodysplastic Syndromes; Progressive Disease
Wake Forest University Health Sciences
 2021-05 PHASE1; PHASE2
NCT03504410 TERMINATED
Relapsed/Refractory Acute Myeloid Leukemia
Cornerstone Pharmaceuticals
 2018-11-12 PHASE3
NCT03504423 COMPLETED
Pancreatic Cancer Metastatic
Cornerstone Pharmaceuticals
 2018-11-09 PHASE3
NCT03374852 WITHDRAWN
Pancreatic Ductal Adenocarcinoma
Sidney Kimmel Comprehensive Cancer Center at Thomas Jefferson University
 2018-08 PHASE2
NCT02168140 COMPLETED
Adult Lymphocyte Depletion Hodgkin Lymphoma; Adult Lymphocyte Predominant Hodgkin Lymphoma; Adult Mixed Cellularity Hodgkin Lymphoma; Adult Nasal Type Extranodal NK/T-cell Lymphoma; Adult Nodular Sclerosis Hodgkin Lymphoma; Anaplastic Large Cell Lymphoma; Angioimmunoblastic T-cell Lymphoma; Hepatosplenic T-cell Lymphoma; Noncutaneous Extranodal Lymphoma; Peripheral T-cell Lymphoma; Recurrent Adult Acute Lymphoblastic Leukemia; Recurrent Adult Hodgkin Lymphoma; Recurrent Adult T-cell Leukemia/Lymphoma; Recurrent Cutaneous T-cell Non-Hodgkin Lymphoma; Recurrent Mycosis Fungoides/Sezary Syndrome; T-cell Adult Acute Lymphoblastic Leukemia; T-cell Large Granular Lymphocyte Leukemia
Wake Forest University Health Sciences
 2014-09 PHASE1
NCT03370159 WITHDRAWN
Stage IIIB Non-Small Cell Lung Cancer AJCC v7; Stage IV Non-Small Cell Lung Cancer AJCC v7
Wake Forest University Health Sciences
 2018-06 PHASE1; PHASE2
NCT02232152 COMPLETED
Mucinous Adenocarcinoma of the Colon; Mucinous Adenocarcinoma of the Rectum; Recurrent Colon Cancer; Recurrent Rectal Cancer; Signet Ring Adenocarcinoma of the Colon; Signet Ring Adenocarcinoma of the Rectum; Stage IIIA Colon Cancer; Stage IIIA Rectal Cancer; Stage IIIB Colon Cancer; Stage IIIB Rectal Cancer; Stage IIIC Colon Cancer; Stage IIIC Rectal Cancer; Stage IVA Colon Cancer; Stage IVA Rectal Cancer; Stage IVB Colon Cancer; Stage IVB Rectal Cancer
Wake Forest University Health Sciences
 2015-01-06 PHASE1
NCT01830322 WITHDRAWN
Metastatic Pancreatic Adenocarcinoma
Cornerstone Pharmaceuticals
 2014-01 PHASE2
NCT01520805 WITHDRAWN
Acute Myeloid Leukemia (AML); Myelodysplastic Syndrome (MDS)
Cornerstone Pharmaceuticals
 2016-01 PHASE2
NCT03793140 Active not recruiting
Lymphoma|Leukemia
Memorial Sloan Kettering Cancer Center|City of Hope Medical Center|Massachusetts General Hospital|M.D. Anderson Cancer Center|George Washington University
 2018-12-31 Phase 2
NCT01902381 TERMINATED
Previously Treated Myelodysplastic Syndromes
Wake Forest University Health Sciences
 2013-08 PHASE2
NCT03435289 UNKNOWN
Pancreatic Cancer; Metastatic Pancreatic Cancer
Atlantic Health System
 2017-10-19 PHASE1
NCT02484391 COMPLETED
Granulocytic Sarcoma; Recurrent Adult Acute Myeloid Leukemia
Wake Forest University Health Sciences
 2015-09 PHASE1
NCT01766219 COMPLETED
Adult Primary Cholangiocellular Carcinoma; Advanced Adult Primary Liver Cancer; Cholangiocarcinoma of the Extrahepatic Bile Duct; Cholangiocarcinoma of the Gallbladder; Localized Unresectable Adult Primary Liver Cancer; Metastatic Extrahepatic Bile Duct Cancer; Recurrent Adult Primary Liver Cancer; Recurrent Extrahepatic Bile Duct Cancer; Unresectable Extrahepatic Bile Duct Cancer
Wake Forest University Health Sciences
 2013-05 PHASE1; PHASE2
NCT00907166 TERMINATED
Cancer; Pancreatic Cancer; Pancreatic Carcinoma
Cornerstone Pharmaceuticals
 2009-05 PHASE1; PHASE2
NCT01835041 COMPLETED
Acinar Cell Adenocarcinoma of the Pancreas; Duct Cell Adenocarcinoma of the Pancreas; Recurrent Pancreatic Cancer; Stage IV Pancreatic Cancer
Wake Forest University Health Sciences
 2013-04 PHASE1
NCT00741403 COMPLETED
Advanced Cancer; Metastatic Cancer; Lymphoma; Solid Tumors; Advanced Malignancies
Cornerstone Pharmaceuticals
 2008-08 PHASE1
NCT01832857 TERMINATED
Cancer
Cornerstone Pharmaceuticals
 2013-06 PHASE2
NCT02472626 WITHDRAWN
Untreated Adult Acute Myeloid Leukemia
Wake Forest University Health Sciences
 2015-12 PHASE1; PHASE2
NCT01768897 COMPLETED
Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities; Adult Acute Myeloid Leukemia With Del(5q); Adult Acute Myeloid Leukemia With Inv(16)(p13;q22); Adult Acute Myeloid Leukemia With t(15;17)(q22;q12); Adult Acute Myeloid Leukemia With t(16;16)(p13;q22); Adult Acute Myeloid Leukemia With t(8;21)(q22;q22); Recurrent Adult Acute Myeloid Leukemia
Wake Forest University Health Sciences
 2013-01 PHASE1
NCT01931787 COMPLETED
Recurrent Small Cell Lung Cancer
Wake Forest University Health Sciences
 2013-10 PHASE1
NCT01839981 COMPLETED
Acinar Cell Adenocarcinoma of the Pancreas; Duct Cell Adenocarcinoma of the Pancreas; Recurrent Pancreatic Cancer; Stage III Pancreatic Cancer; Stage IV Pancreatic Cancer
Wake Forest University Health Sciences
 2013-07 PHASE1
NCT02168907 TERMINATED
B-cell Adult Acute Lymphoblastic Leukemia; B-cell Chronic Lymphocytic Leukemia; Cutaneous B-cell Non-Hodgkin Lymphoma; Extranodal Marginal Zone B-cell Lymphoma of Mucosa-associated Lymphoid Tissue; Intraocular Lymphoma; Nodal Marginal Zone B-cell Lymphoma; Recurrent Adult Acute Lymphoblastic Leukemia; Recurrent Adult Burkitt Lymphoma; Recurrent Adult Diffuse Large Cell Lymphoma; Recurrent Adult Diffuse Mixed Cell Lymphoma; Recurrent Adult Diffuse Small Cleaved Cell Lymphoma; Recurrent Adult Grade III Lymphomatoid Granulomatosis; Recurrent Adult Immunoblastic Large Cell Lymphoma; Recurrent Adult Lymphoblastic Lymphoma; Recurrent Grade 1 Follicular Lymphoma; Recurrent Grade 2 Follicular Lymphoma; Recurrent Grade 3 Follicular Lymphoma; Recurrent Mantle Cell Lymphoma; Recurrent Marginal Zone Lymphoma; Recurrent Mycosis Fungoides/Sezary Syndrome; Recurrent Small Lymphocytic Lymphoma; Refractory Chronic Lymphocytic Leukemia; Refractory Hairy Cell Leukemia; Small Intestine Lymphoma; Splenic Marginal Zone Lymphoma; Testicular Lymphoma; Waldenström Macroglobulinemia
Wake Forest University Health Sciences
 2014-12 PHASE1
NCT01034475 COMPLETED
Advanced Hematologic Malignancies
Wake Forest University Health Sciences
 2010-03 PHASE1

(data from https://clinicaltrials.gov, updated on 2023-03-10)

Check the CPI-613 (Devimistat) product page for in-depth specifications, including solubility, stock solutions, MOA, and working concentrations.

Compliance for Clinical Use

Mechanism and Biochemical Profile

CPI-613 selectively inhibits the mitochondrial enzymes pyruvate dehydrogenase and α-ketoglutarate dehydrogenase, thereby disrupting tumor cell mitochondrial energy metabolism and reducing mitochondrial membrane potential. This metabolic collapse triggers apoptotic cell death, resulting in tumor growth inhibition in clinical conditions such as pancreatic adenocarcinoma, biliary tract carcinoma, and hematologic malignancies.

Appendix RUO and cGMP Quality Standards

Quality Dimension RUO (Research Use Only) cGMP (Current Good Manufacturing Practice)
Clinical Applicability Prohibited in human clinical trials or medical diagnostics. Mandatory for human clinical trials (Phase I–III) and therapies.
Regulatory Status Non-regulated grade; exempt from drug manufacturing laws. Legally enforced by health authorities (e.g., FDA, EMA, NMPA).
Facility Environment Unclassified analytical or research laboratories. Validated Cleanrooms (ISO Class 5–8) with continuous monitoring.
Quality Control Basic purity and activity testing. Rigorous safety release testing (Sterility, Endotoxin, Mycoplasma).
Process Validation Basic equipment calibration; no process validation required. Full qualification (IQ/OQ/PQ) and complete batch records.
Quality Assurance Vendor self-declared without required formal QMS. Mandatory QA/QC unit, Change Control, CAPA, and vendor audits.
Regulatory Impact High risk of IND rejection if used as a critical raw material. Required for IND/NDA filings, supported by Drug Master Files (DMF).

Footnotes

Regulatory Note: Governed by FDA (21 CFR Parts 210/211/312), EMA (EudraLex Vol 4), ICH Guidelines (E6/Q7/Q9/Q10), and compendial standards (USP <71>/<85>/<1043>).

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Note: Technical data last updated: Sep 1, 2026.