Clinical Trials

Alisertib (MLN8237) has been evaluated across numerous clinical trials spanning Phase 1, Phase 2, and Phase 3 for malignancies including non-small cell and small cell lung carcinomas, metastatic breast, ovarian, and gastrointestinal cancers, acute myeloid leukemia, and aggressive lymphomas. Spearheaded by academic institutions and industry sponsors—including M.D. Anderson Cancer Center, Millennium Pharmaceuticals, Takeda, and Puma Biotechnology—these studies reflect diverse recruitment statuses, ranging from completed, terminated, and withdrawn to protocols not yet recruiting combination therapies.

NCT Number Recruitment Conditions Sponsor/Collaborators Start Date Phases
NCT07706179 NOT_YET_RECRUITING
HR+/HER2+ Breast Cancer; Metastatic Breast Cancer
University of Wisconsin, Madison
 2026-09 PHASE2
NCT07465757 NOT_YET_RECRUITING
Small Cell Lung Cancer ( SCLC )
Puma Biotechnology, Inc.
 2026-09-30 PHASE2
NCT06369285 RECRUITING
Hormone Receptor Positive HER-2 Negative Breast Cancer; Metastatic Breast Cancer; Recurrent Breast Cancer
Puma Biotechnology, Inc.
 2024-11-19 PHASE2
NCT06095505 RECRUITING
Small Cell Lung Cancer
Puma Biotechnology, Inc.
 2024-02-08 PHASE2
NCT04085315 COMPLETED
Lung Cancer Metastatic; EGFR Gene Mutation
Collin Blakely
 2019-11-12 PHASE1
NCT02114229 ACTIVE_NOT_RECRUITING
Malignant Rhabdoid Tumor; Atypical Teratoid Rhabdoid Tumor
St. Jude Children's Research Hospital
 2014-05-14 PHASE2
NCT04555837 COMPLETED
Head and Neck Squamous Cell Carcinoma; Malignant Solid Neoplasm
M.D. Anderson Cancer Center
 2020-09-15 PHASE1; PHASE2
NCT02187991 COMPLETED
Breast Cancer; Breast Carcinoma; Breast Tumors; Malignant Neoplasm of Breast
US Oncology Research
 2015-02-12 PHASE2
NCT04479306 COMPLETED
Recurrent Lung 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
M.D. Anderson Cancer Center
 2020-06-18 PHASE1
NCT02860000 COMPLETED
Estrogen Receptor Status; HER2/Neu Negative; Invasive Breast Carcinoma; Postmenopausal; Stage III Breast Cancer; Stage IIIA Breast Cancer; Stage IIIB Breast Cancer; Stage IIIC Breast Cancer; Stage IV Breast Cancer
Mayo Clinic
 2017-07-06 PHASE2
NCT02812056 WITHDRAWN
Malignant Neoplasms of Digestive Organs; Malignant Neoplasms of Female Genital Organs; Malignant Neoplasms of Lip Oral Cavity and Pharynx; Malignant Neoplasms of Male Genital Organs
M.D. Anderson Cancer Center
 2016-09 PHASE1
NCT02293005 COMPLETED
Lung Cancer; Mesothelioma
M.D. Anderson Cancer Center
 2015-05-20 PHASE2
NCT04479306 Completed
Recurrent Lung 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
M.D. Anderson Cancer Center
 2020-06-18 Phase 1
NCT02719691 COMPLETED
Metastatic Breast Cancer; Solid Tumors
University of Colorado, Denver
 2016-05-13 PHASE1
NCT01897012 COMPLETED
MYC Positive; Recurrent B-Cell Non-Hodgkin Lymphoma; Recurrent Burkitt Lymphoma; Recurrent Diffuse Large B-Cell Lymphoma; Recurrent Follicular Lymphoma; Recurrent High Grade B-Cell Lymphoma With MYC and BCL2 or BCL6 Rearrangements; Recurrent Hodgkin Lymphoma; Recurrent Mantle Cell Lymphoma; Recurrent Mature T- and NK-Cell Non-Hodgkin Lymphoma; Refractory B-Cell Non-Hodgkin Lymphoma; Refractory Burkitt Lymphoma; Refractory Diffuse Large B-Cell Lymphoma; Refractory Follicular Lymphoma; Refractory High Grade B-Cell Lymphoma With MYC and BCL2 or BCL6 Rearrangements; Refractory Hodgkin Lymphoma; Refractory Mantle Cell Lymphoma; Refractory Mature T-Cell and NK-Cell Non-Hodgkin Lymphoma
National Cancer Institute (NCI)
 2013-07-17 PHASE1
NCT02319018 COMPLETED
Malignant Digestive System Neoplasm
National Cancer Institute (NCI)
 2015-08-27 PHASE1
NCT02780011 WITHDRAWN
CD30-positive Lymphoma; CD30-positive Solid Tumor
Eric Bernicker, MD
 2015-12 PHASE1
NCT01601535 COMPLETED
Neuroblastoma
New Approaches to Neuroblastoma Therapy Consortium
 2012-05 PHASE1; PHASE2
NCT02327169 COMPLETED
Advanced Nonhematologic Malignancies
Millennium Pharmaceuticals, Inc.
 2015-01-14 PHASE1
NCT01695941 ACTIVE_NOT_RECRUITING
Recurrent B-Cell Non-Hodgkin Lymphoma; Recurrent Mantle Cell Lymphoma; Refractory B-Cell Non-Hodgkin Lymphoma; Refractory Mantle Cell Lymphoma
National Cancer Institute (NCI)
 2012-08-31 PHASE1
NCT02530619 UNKNOWN
Acute Megakaryoblastic Leukemia; Myelofibrosis; Primary Myelofibrosis
Northwestern University
 2015-10-09
NCT01567709 COMPLETED
Adult B Acute Lymphoblastic Leukemia; Adult T Acute Lymphoblastic Leukemia; Anaplastic Large Cell Lymphoma; Angioimmunoblastic T-Cell Lymphoma; Chronic Lymphocytic Leukemia; Extranodal Marginal Zone Lymphoma of Mucosa-Associated Lymphoid Tissue; Hepatosplenic T-Cell Lymphoma; Intraocular Lymphoma; Lymphomatous Involvement of Non-Cutaneous Extranodal Site; Mature T-Cell and NK-Cell Non-Hodgkin Lymphoma; Nodal Marginal Zone Lymphoma; Primary Cutaneous B-Cell Non-Hodgkin Lymphoma; Recurrent Adult Acute Lymphoblastic Leukemia; Recurrent Adult Burkitt Lymphoma; Recurrent Adult Grade III Lymphomatoid Granulomatosis; Recurrent Adult Hodgkin Lymphoma; Recurrent Adult Immunoblastic Lymphoma; Recurrent Adult Lymphoblastic Lymphoma; Recurrent Adult T-Cell Leukemia/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 and Sezary Syndrome; Recurrent Non-Hodgkin Lymphoma; Recurrent Primary Cutaneous T-Cell Non-Hodgkin Lymphoma; Recurrent Small Lymphocytic Lymphoma; Refractory Chronic Lymphocytic Leukemia; Refractory Hairy Cell Leukemia; Small Intestinal Lymphoma; Splenic Marginal Zone Lymphoma; T-Cell Large Granular Lymphocyte Leukemia; Testicular Lymphoma; Waldenstrom Macroglobulinemia
National Cancer Institute (NCI)
 2012-04-16 PHASE1
NCT02560025 COMPLETED
Acute Myeloid Leukemia
Massachusetts General Hospital
 2015-12 PHASE2
NCT02367352 TERMINATED
Advanced Solid Tumors; Ovarian Cancer; Small Cell Lung Cancer
Millennium Pharmaceuticals, Inc.
 2015-03-19 PHASE1
NCT02186509 COMPLETED
Adult Anaplastic Astrocytoma; Adult Anaplastic Ependymoma; Adult Anaplastic Oligodendroglioma; Adult Brain Stem Glioma; Adult Diffuse Astrocytoma; Adult Giant Cell Glioblastoma; Adult Glioblastoma; Adult Gliosarcoma; Adult Mixed Glioma; Adult Oligodendroglioma; Adult Pilocytic Astrocytoma; Adult Pineal Gland Astrocytoma; Adult Subependymal Giant Cell Astrocytoma; Recurrent Adult Brain Tumor
Sidney Kimmel Comprehensive Cancer Center at Thomas Jefferson University
 2015-01-30 PHASE1
NCT02551055 TERMINATED
Advanced and Metastatic Gastric or Gastroesophageal Adenocarcinoma
Millennium Pharmaceuticals, Inc.
 2015-10-15 PHASE1
NCT01924260 COMPLETED
Acinar Cell Adenocarcinoma of the Pancreas; Duct Cell Adenocarcinoma of the Pancreas; Recurrent Pancreatic Cancer; Stage III Pancreatic Cancer; Stage IV Pancreatic Cancer; Unspecified Adult Solid Tumor, Protocol Specific
Edward Kim
 2013-08-09 PHASE1
NCT01471964 TERMINATED
Non-small Cell Lung Cancer Metastatic; Non-small Cell Lung Cancer Recurrent
Fox Chase Cancer Center
 2011-10-20 PHASE1; PHASE2
NCT02700022 TERMINATED
Diffuse Large B-cell Lymphoma; Follicular Lymphoma; Burkitt Lymphoma
UNC Lineberger Comprehensive Cancer Center
 2016-10 PHASE1
NCT01923337 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; Unspecified Adult Solid Tumor, Protocol Specific
University of California, Davis
 2013-08 PHASE1
NCT00697346 COMPLETED
B-cell Follicular Lymphoma; B-cell Marginal Zone Lymphoma; Diffuse Large B-cell Lymphoma; B-cell Mantle Cell Lymphoma; B-cell Small Lymphocytic Lymphoma; B-Cell Chronic Lymphocytic Leukemia; Multiple Myeloma; Waldenstrom's Macroglobulinemia; Noncutaneous Peripheral T-cell Lymphoma Not Otherwise Specified; Angioimmunoblastic T-cell Lymphoma; Anaplastic Large Cell Lymphoma; Enteropathy Associated T-cell Lymphoma; NK Lymphoma
Millennium Pharmaceuticals, Inc.
 2008-07-11 PHASE1
NCT02812056 Withdrawn
Malignant Neoplasms of Digestive Organs|Malignant Neoplasms of Female Genital Organs|Malignant Neoplasms of Lip Oral Cavity and Pharynx|Malignant Neoplasms of Male Genital Organs
M.D. Anderson Cancer Center|Millennium Pharmaceuticals Inc.
 2016-09 Phase 1
NCT01639911 COMPLETED
Malignant Neoplasm of Breast; CNS Malignancy; Malignant Neoplasm of Gastrointestinal Tract; Genitourinary Neoplasms Malignancy and Gender Unspecified; Head and Neck Neoplasms; Melanoma; Malignant Neoplasm of Thorax
University of Illinois at Chicago
 2013-08-14 PHASE1
NCT01799278 COMPLETED
Small Cell Prostate Cancer; Neuroendocrine Prostate Cancer; Prostate Adenocarcinoma Plus > 50% Immunohistochemical Staining for Neuroendocrine Markers
Weill Medical College of Cornell University
 2013-02 PHASE2
NCT02719691 Completed
Metastatic Breast Cancer|Solid Tumors
University of Colorado Denver
 2016-05-13 Phase 1
NCT02214147 COMPLETED
Advanced Solid Tumors; Relapsed/Refractory Lymphoma
Millennium Pharmaceuticals, Inc.
 2014-08-21 PHASE1
NCT02219789 COMPLETED
Estrogen Receptor Positive; Progesterone Receptor Positive; Recurrent Breast Carcinoma; Stage IIIB Breast Cancer; Stage IIIC Breast Cancer; Stage IV Breast Cancer
Mayo Clinic
 2014-12-05 PHASE1
NCT02038647 COMPLETED
Small Cell Lung Cancer
Millennium Pharmaceuticals, Inc.
 2014-05-12 PHASE2
NCT01154816 COMPLETED
Hepatoblastoma; Previously Treated Childhood Rhabdomyosarcoma; Recurrent Childhood Acute Lymphoblastic Leukemia; Recurrent Childhood Acute Myeloid Leukemia; Recurrent Childhood Kidney Neoplasm; Recurrent Childhood Malignant Germ Cell Tumor; Recurrent Childhood Rhabdomyosarcoma; Recurrent Childhood Soft Tissue Sarcoma; Recurrent Ewing Sarcoma/Peripheral Primitive Neuroectodermal Tumor; Recurrent Neuroblastoma; Recurrent Osteosarcoma
Children's Oncology Group
 2011-02 PHASE2
NCT01779843 COMPLETED
Acute Myelogenous Leukemia
Massachusetts General Hospital
 2013-04 PHASE1
NCT01540682 COMPLETED
Head and Neck Cancer
Abramson Cancer Center at Penn Medicine
 2012-02 PHASE1
NCT01812005 TERMINATED
Extranodal Marginal Zone B-cell Lymphoma of Mucosa-associated Lymphoid Tissue; Nodal Marginal Zone B-cell Lymphoma; Recurrent Adult Burkitt Lymphoma; Recurrent Adult Diffuse Large Cell Lymphoma; Recurrent Grade 1 Follicular Lymphoma; Recurrent Grade 2 Follicular Lymphoma; Recurrent Grade 3 Follicular Lymphoma; Recurrent Mantle Cell Lymphoma; Recurrent Marginal Zone Lymphoma; Splenic Marginal Zone Lymphoma; Waldenström Macroglobulinemia
Ohio State University Comprehensive Cancer Center
 2013-05-21 PHASE2
NCT01613261 WITHDRAWN
Advanced Nonhematologic Malignancies
Millennium Pharmaceuticals, Inc.
 2013-08 PHASE1
NCT01653028 COMPLETED
Myxofibrosarcoma; Recurrent Adult Soft Tissue Sarcoma; Recurrent Leiomyosarcoma; Recurrent Liposarcoma; Recurrent Malignant Peripheral Nerve Sheath Tumor; Recurrent Undifferentiated Pleomorphic Sarcoma; Stage III Soft Tissue Sarcoma AJCC v7; Stage IV Soft Tissue Sarcoma AJCC v7
National Cancer Institute (NCI)
 2012-08-22 PHASE2
NCT02109328 COMPLETED
Bladder Cancer; Transitional Cell Carcinoma
Fondazione IRCCS Istituto Nazionale dei Tumori, Milano
 2014-08-28 PHASE2
NCT01482962 COMPLETED
Relapsed Peripheral T-Cell Lymphoma; Refractory Peripheral T-Cell Lymphoma
Millennium Pharmaceuticals, Inc.
 2012-06-11 PHASE3
NCT02259010 COMPLETED
Advanced Solid Tumors; Relapsed/Refractory Lymphoma
Millennium Pharmaceuticals, Inc.
 2014-10-22 PHASE1
NCT01848067 COMPLETED
Adenocarcinoma of the Prostate; Hormone-resistant Prostate Cancer; Recurrent Prostate Cancer; Stage IV Prostate Cancer
Sidney Kimmel Comprehensive Cancer Center at Thomas Jefferson University
 2013-08-14 PHASE1; PHASE2
NCT02367352 Terminated
Advanced Solid Tumors|Ovarian Cancer|Small Cell Lung Cancer
Millennium Pharmaceuticals Inc.|Takeda
 2015-03-19 Phase 1
NCT01397825 COMPLETED
Diffuse Large B-Cell Lymphoma; Transformed Follicular Lymphoma; Mantle Cell Lymphoma; Burkitt's Lymphoma
Millennium Pharmaceuticals, Inc.
 2011-08-09 PHASE1; PHASE2
NCT01466881 COMPLETED
Adult Nasal Type Extranodal NK/T-Cell Lymphoma; Anaplastic Large Cell Lymphoma; Angioimmunoblastic T-Cell Lymphoma; Hepatosplenic T-Cell Lymphoma; Mature T-Cell and NK-Cell Non-Hodgkin Lymphoma; Recurrent Adult Non-Hodgkin Lymphoma; Recurrent Adult T-Cell Leukemia/Lymphoma
National Cancer Institute (NCI)
 2011-10 PHASE2
NCT01316692 TERMINATED
Recurrent Melanoma; Stage IIIc Melanoma; Stage IV Melanoma
Vanderbilt-Ingram Cancer Center
 2011-10 PHASE2
NCT01677559 COMPLETED
Adenocarcinoma; Pancreatic Neoplasms
Washington University School of Medicine
 2013-05-07 PHASE1
NCT01034553 COMPLETED
Refractory Multiple Myeloma
Mayo Clinic
 2010-02 PHASE1; PHASE2
NCT01091428 COMPLETED
Ovarian Carcinoma; Fallopian Tube Cancer; Peritoneal Cancer; Breast Carcinoma
Millennium Pharmaceuticals, Inc.
 2010-04-16 PHASE2
NCT01844583 COMPLETED
Solid Tumors; Lymphoma
Millennium Pharmaceuticals, Inc.
 2013-06-25 PHASE1
NCT01637961 COMPLETED
Recurrent Uterine Corpus Sarcoma; Uterine Corpus Leiomyosarcoma
National Cancer Institute (NCI)
 2012-08 PHASE2
NCT01898078 COMPLETED
Advanced Solid Tumors; Lymphoma
Millennium Pharmaceuticals, Inc.
 2013-07-16 PHASE1
NCT01094288 COMPLETED
Advanced Solid Tumors; Adenocarcinoma of the Prostate
Millennium Pharmaceuticals, Inc.
 2010-08-17 PHASE1
NCT01512758 COMPLETED
Advanced Solid Tumors; Lymphomas
Millennium Pharmaceuticals, Inc.
 2012-02-06 PHASE1
NCT01045421 COMPLETED
Advanced Nonhematological Malignancies; Non-Small Cell Lung Cancer; Small Cell Lung Cancer; Metastatic Breast Cancer; Head and Neck Squamous Cell Carcinoma; Gastroesophageal Adenocarcinoma
Millennium Pharmaceuticals, Inc.
 2010-02 PHASE1; PHASE2
NCT01714947 COMPLETED
Advanced Solid Tumors; Lymphoma
Millennium Pharmaceuticals, Inc.
 2013-01-24 PHASE1
NCT00962091 COMPLETED
Advanced Solid Tumors
Millennium Pharmaceuticals, Inc.
 2009-09-25 PHASE1
NCT02444884 COMPLETED
Unspecified Childhood Solid Tumor, Excluding CNS; Neuroblastoma
Children's Oncology Group
 2008-09 PHASE1
NCT00807495 COMPLETED
Diffuse Large B-cell Lymphoma; Mantle Cell Lymphoma; Burkitt's Lymphoma; T-cell Lymphoma, Excluding Primary Cutaneous T-cell Lymphoma; Transformed Follicular Lymphoma With ≥ 50% Diffuse Large Cell Component
Millennium Pharmaceuticals, Inc.
 2009-02-10 PHASE2
NCT00651664 COMPLETED
Advanced Malignancies
Millennium Pharmaceuticals, Inc.
 2007-10-22 PHASE1
NCT00500903 COMPLETED
Advanced Malignancies
Millennium Pharmaceuticals, Inc.
 2007-05-15 PHASE1
NCT00830518 COMPLETED
Acute Myelogenous Leukemia; High-Grade Myelodysplastic Syndrome
Millennium Pharmaceuticals, Inc.
 2009-02-10 PHASE2
NCT00853307 COMPLETED
Ovarian Carcinoma
Millennium Pharmaceuticals, Inc.
 2009-03-23 PHASE2

(data from https://clinicaltrials.gov, updated on 2026-08-26)

Check the Alisertib (MLN8237) product page for in-depth specifications, including solubility, stock solutions, MOA, and working concentrations.

Compliance for Clinical Use

Mechanism and Biochemical Profile

Alisertib selectively binds to and inhibits Aurora A kinase, disrupting mitotic spindle apparatus assembly and chromosome segregation to induce cell cycle arrest, apoptosis, and inhibition of cell proliferation. This targeted inhibition suppresses tumor cell viability, demonstrating clinical relevance for treating advanced solid tumors, non-small cell lung cancer, and aggressive lymphomas.

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>).

Jump to

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