Clinical Trials

Numerous clinical trials evaluate MLN4924 (pevonedistat) across Phase 1 through Phase 3 development for hematological malignancies and solid neoplasms, including myelodysplastic syndromes, acute myeloid leukemia, multiple myeloma, and non-small cell lung cancer. Sponsored by both pharmaceutical companies—such as Takeda and Millennium Pharmaceuticals—and academic or federal entities, including the National Cancer Institute and M.D. Anderson Cancer Center, these studies exhibit diverse recruitment statuses ranging from active and completed to terminated or withdrawn.

NCT Number Recruitment Conditions Sponsor/Collaborators Start Date Phases
NCT03013998 RECRUITING
Previously Untreated Relapsed Refractory Acute Myeloid Leukemia
Beat AML, LLC
 2016-11 PHASE2; PHASE3
NCT03862157 ACTIVE_NOT_RECRUITING
Acute Myeloid Leukemia; Atypical Chronic Myeloid Leukemia, BCR-ABL1 Negative; Chronic Eosinophilic Leukemia, Not Otherwise Specified; Chronic Myelomonocytic Leukemia; Chronic Neutrophilic Leukemia; Essential Thrombocythemia; Myelodysplastic Syndrome; Myelodysplastic/Myeloproliferative Neoplasm With Ring Sideroblasts and Thrombocytosis; Myelodysplastic/Myeloproliferative Neoplasm, Unclassifiable; Myeloid Neoplasm; Myeloproliferative Neoplasm; Myeloproliferative Neoplasm, Unclassifiable; Overt Primary Myelofibrosis; Polycythemia Vera; Polycythemia Vera, Post-Polycythemic Myelofibrosis Phase; Prefibrotic/Early Primary Myelofibrosis
M.D. Anderson Cancer Center
 2019-02-27 PHASE1; PHASE2
NCT03330821 ACTIVE_NOT_RECRUITING
Acute Myeloid Leukemia Arising From Previous Myelodysplastic Syndrome; Acute Myeloid Leukemia With Myelodysplasia-Related Changes; Therapy-Related Acute Myeloid Leukemia
University of Southern California
 2018-04-18 PHASE1; PHASE2
NCT03479268 ACTIVE_NOT_RECRUITING
B-Cell Prolymphocytic Leukemia; Recurrent Chronic Lymphocytic Leukemia; Recurrent Diffuse Large B-Cell Lymphoma; Recurrent Follicular Lymphoma; Recurrent Lymphoplasmacytic Lymphoma; Recurrent Mantle Cell Lymphoma; Recurrent Marginal Zone Lymphoma; Recurrent Non-Hodgkin Lymphoma; Recurrent Small Lymphocytic Lymphoma; Refractory Chronic Lymphocytic Leukemia; Refractory Diffuse Large B-Cell Lymphoma; Refractory Follicular Lymphoma; Refractory Lymphoplasmacytic Lymphoma; Refractory Mantle Cell Lymphoma; Refractory Marginal Zone Lymphoma; Refractory Non-Hodgkin Lymphoma; Refractory Small Lymphocytic Lymphoma; Richter Syndrome
City of Hope Medical Center
 2018-04-27 PHASE1
NCT03009240 COMPLETED
Acute Myeloid Leukemia; Recurrent Acute Myeloid Leukemia; Refractory Acute Myeloid Leukemia; Secondary Acute Myeloid Leukemia; Therapy-Related Acute Myeloid Leukemia; Untreated Adult Acute Myeloid Leukemia
City of Hope Medical Center
 2017-08-21 PHASE1
NCT04985656 WITHDRAWN
Myelodysplastic Syndromes (MDS)
Takeda
 2021-10-01 PHASE2
NCT04175912 ACTIVE_NOT_RECRUITING
Metastatic Cholangiocarcinoma; Metastatic Hepatocellular Carcinoma; Stage III Hepatocellular Carcinoma AJCC v8; Stage III Intrahepatic Cholangiocarcinoma AJCC v8; Stage IIIA Hepatocellular Carcinoma AJCC v8; Stage IIIA Intrahepatic Cholangiocarcinoma AJCC v8; Stage IIIB Hepatocellular Carcinoma AJCC v8; Stage IIIB Intrahepatic Cholangiocarcinoma AJCC v8; Stage IV Hepatocellular Carcinoma AJCC v8; Stage IV Intrahepatic Cholangiocarcinoma AJCC v8; Stage IVA Hepatocellular Carcinoma AJCC v8; Stage IVB Hepatocellular Carcinoma AJCC v8; Unresectable Cholangiocarcinoma; Unresectable Hepatocellular Carcinoma; Unresectable Intrahepatic Cholangiocarcinoma
National Cancer Institute (NCI)
 2020-03-16 PHASE2
NCT04090736 ACTIVE_NOT_RECRUITING
Leukemia, Myeloid, Acute
PETHEMA Foundation
 2019-09-24 PHASE3
NCT03319537 TERMINATED
Mesothelioma
Memorial Sloan Kettering Cancer Center
 2017-10-05 PHASE1; PHASE2
NCT04712942 COMPLETED
Acute Myeloid Leukemia in Remission; Myelodysplastic Syndromes; Minimal Residual Disease
University of Leipzig
 2021-01-01 PHASE2
NCT04800627 TERMINATED
Locally Advanced Malignant Solid Neoplasm; Metastatic Malignant Solid Neoplasm; Unresectable Malignant Solid Neoplasm
M.D. Anderson Cancer Center
 2021-03-29 PHASE1; PHASE2
NCT03238248 COMPLETED
Myelodysplastic Syndromes; Myeloproliferative Neoplasm
Vanderbilt-Ingram Cancer Center
 2017-08-07 PHASE2
NCT03770260 COMPLETED
Recurrent Multiple Myeloma; Refractory Multiple Myeloma
National Cancer Institute (NCI)
 2020-02-10 PHASE1
NCT03965689 ACTIVE_NOT_RECRUITING
Metastatic Lung Non-Small Cell Squamous Carcinoma; Metastatic Lung Non-Squamous Non-Small Cell Carcinoma; Stage IIIB Lung Cancer AJCC v8; Stage IV Lung Cancer AJCC v8; Stage IVA Lung Cancer AJCC v8; Stage IVB Lung Cancer AJCC v8; Unresectable Lung Non-Small Cell Carcinoma; Unresectable Lung Non-Squamous Non-Small Cell Carcinoma
National Cancer Institute (NCI)
 2020-04-16 PHASE2
NCT03772925 TERMINATED
Recurrent Acute Myeloid Leukemia; Recurrent Myelodysplastic Syndrome; Refractory Acute Myeloid Leukemia; Refractory Myelodysplastic Syndrome
National Cancer Institute (NCI)
 2019-06-20 PHASE1
NCT04266795 COMPLETED
Acute Myeloid Leukemia (AML)
Takeda
 2020-10-13 PHASE2
NCT02782468 COMPLETED
Leukemia, Myeloid, Acute; Myelodysplastic Syndromes
Millennium Pharmaceuticals, Inc.
 2016-05-16 PHASE1
NCT04172844 TERMINATED
Acute Myelogenous Leukemia
Medical College of Wisconsin
 2020-01-13 PHASE1
NCT03228186 TERMINATED
Non-small Cell Lung Cancer
University of Michigan Rogel Cancer Center
 2018-03-05 PHASE2
NCT04985656 Withdrawn
Myelodysplastic Syndromes (MDS)
Takeda
 2021-10-01 Phase 2
NCT03813147 COMPLETED
Acute Myeloid Leukemia Arising From Previous Myelodysplastic Syndrome; Recurrent Acute Myeloid Leukemia; Recurrent Myelodysplastic Syndrome; Refractory Acute Myeloid Leukemia; Refractory Myelodysplastic Syndrome
National Cancer Institute (NCI)
 2019-05-17 PHASE1
NCT03323034 COMPLETED
Recurrent Lymphoma; Recurrent Malignant Solid Neoplasm; Recurrent Primary Central Nervous System Neoplasm; Refractory Lymphoma; Refractory Malignant Solid Neoplasm; Refractory Primary Central Nervous System Neoplasm
Children's Oncology Group
 2018-01-11 PHASE1
NCT03386214 TERMINATED
Myelofibroses
Washington University School of Medicine
 2018-04-23 PHASE1
NCT03745352 WITHDRAWN
Recurrent Acute Myeloid Leukemia; Refractory Acute Myeloid Leukemia
National Cancer Institute (NCI)
 2019-05-20 PHASE2
NCT03349281 COMPLETED
Refractory Acute Lymphoblastic Leukemia; Relapsed Acute Lymphoblastic Leukemia
Julio Barredo, MD
 2019-03-25 PHASE1
NCT03459859 COMPLETED
Acute Myelogenous Leukemia; AML; Advanced Myelodysplastic Syndromes; MDS
Justin Watts, MD
 2018-05-21 PHASE1
NCT03268954 COMPLETED
Myelodysplastic Syndrome; Leukemia, Myelomonocytic, Chronic; Leukemia, Myeloid, Acute
Takeda
 2017-11-28 PHASE3
NCT03814005 COMPLETED
Myelodysplastic Syndromes; Leukemia, Myelomonocytic, Chronic; Leukemia, Myeloid, Acute; Renal Insufficiency; Liver Disease; Neoplasms
Takeda
 2019-07-10 PHASE1
NCT04800627 Terminated
Locally Advanced Malignant Solid Neoplasm|Metastatic Malignant Solid Neoplasm|Unresectable Malignant Solid Neoplasm
M.D. Anderson Cancer Center
 2021-03-29 Phase 1|Phase 2
NCT04266795 Active not recruiting
Acute Myeloid Leukemia (AML)
Takeda
 2020-10-13 Phase 2
NCT03770260 Completed
Recurrent Multiple Myeloma|Refractory Multiple Myeloma
National Cancer Institute (NCI)
 2020-02-10 Phase 1
NCT04172844 Active not recruiting
Acute Myelogenous Leukemia
Medical College of Wisconsin
 2020-01-13 Phase 1
NCT02610777 COMPLETED
Myelodysplastic Syndromes; Leukemia, Myelomonocytic, Chronic; Leukemia, Myeloid, Acute
Millennium Pharmaceuticals, Inc.
 2016-04-14 PHASE2
NCT03709576 TERMINATED
Acute Myeloid Leukemia (AML)
Milton S. Hershey Medical Center
 2018-07-18 PHASE2
NCT03486314 COMPLETED
Advanced Solid Neoplasm
Millennium Pharmaceuticals, Inc.
 2018-08-13 PHASE1
NCT03330106 COMPLETED
Advanced Solid Neoplasm
Millennium Pharmaceuticals, Inc.
 2017-11-15 PHASE1
NCT01862328 COMPLETED
Solid Tumors
Millennium Pharmaceuticals, Inc.
 2013-06-10 PHASE1
NCT03057366 COMPLETED
Advanced Solid Tumors, Neoplasms, Advanced Solid
Millennium Pharmaceuticals, Inc.
 2017-05-11 PHASE1
NCT02122770 COMPLETED
Advanced Solid Tumors
Millennium Pharmaceuticals, Inc.
 2014-04-01 PHASE1
NCT01814826 COMPLETED
Acute Myelogenous Leukemia
Millennium Pharmaceuticals, Inc.
 2013-04-10 PHASE1
NCT01415765 WITHDRAWN
Lymphoma, Diffuse Large-Cell B-cell; Diffuse, Large B-cell Lymphoma; Lymphoma, Diffuse Large-Cell; Large-Cell Lymphoma, Diffuse
National Cancer Institute (NCI)
 2011-07-15 PHASE1; PHASE2
NCT00911066 COMPLETED
Acute Myelogenous Leukemia; Acute Lymphoblastic Leukemia; Myelodysplastic Syndrome
Millennium Pharmaceuticals, Inc.
 2009-06 PHASE1
NCT00722488 COMPLETED
Hematologic Malignancies; Multiple Myeloma; Lymphoma; Hodgkin Lymphoma
Millennium Pharmaceuticals, Inc.
 2008-06 PHASE1
NCT01011530 COMPLETED
Metastatic Melanoma
Millennium Pharmaceuticals, Inc.
 2009-12 PHASE1
NCT00677170 COMPLETED
Advanced Nonhematologic Malignancies
Millennium Pharmaceuticals, Inc.
 2008-04 PHASE1

(data from https://clinicaltrials.gov, updated on 2026-09-03)

Check the MLN4924 (Pevonedistat) product page for in-depth specifications, including solubility, stock solutions, MOA, and working concentrations.

Compliance for Clinical Use

Mechanism and Biochemical Profile

MLN4924 selectively binds to and inhibits Nedd8 activating enzyme, which blocks cullin neddylation and prevents the proteasomal degradation of critical regulatory proteins. The resulting disruption of protein homeostasis triggers cell-cycle arrest and apoptosis, directly driving tumor growth inhibition in hematological malignancies like acute myeloid leukemia and myelodysplastic syndromes evaluated in clinical trials.

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.