Clinical Trials

Numerous clinical trials spanning Early Phase 1 through Phase 3 evaluate the safety and efficacy of neratinib, primarily targeting HER2-positive breast cancer—including early-stage disease, hormone receptor-positive subtypes, and brain metastases—alongside metastatic castration-resistant prostate cancer and hepatic impairment. Conducted by pharmaceutical sponsors like Puma Biotechnology Inc. and academic institutions such as University Health Network Toronto, these studies range from pharmacokinetic evaluations to Phase 2 combination regimens. Recruitment statuses across the portfolio vary, encompassing completed, actively recruiting, and withdrawn protocols.

NCT Number Recruitment Conditions Sponsor/Collaborators Start Date Phases
NCT07729046 NOT_YET_RECRUITING
Metastatic Invasive Breast Cancer; Resistant Breast Cancer; ER+, HER2-, Metastatic Breast Cancer
University of California, San Diego
 2027-07 PHASE2
NCT01042379 RECRUITING
Breast Neoplasms; Breast Cancer; Breast Tumors; Angiosarcoma; TNBC - Triple-Negative Breast Cancer; HER2-positive Breast Cancer; HER2-negative Breast Cancer; Hormone Receptor Positive Tumor; Hormone Receptor Negative Tumor; Early-stage Breast Cancer; Locally Advanced Breast Cancer
QuantumLeap Healthcare Collaborative
 2010-03-01 PHASE2
NCT05933395 RECRUITING
Advanced Breast Cancer
Dartmouth-Hitchcock Medical Center
 2024-09-23 PHASE2
NCT05564377 RECRUITING
Advanced Malignant Solid Neoplasm; Anatomic Stage III Breast Cancer AJCC v8; Anatomic Stage IV Breast Cancer AJCC v8; Locally Advanced Malignant Solid Neoplasm; Malignant Female Reproductive System Neoplasm; Metastatic HER2-Negative Breast Carcinoma; Metastatic Malignant Solid Neoplasm; Recurrent Endometrial Carcinoma; Recurrent Fallopian Tube Carcinoma; Recurrent Malignant Female Reproductive System Neoplasm; Recurrent Malignant Solid Neoplasm; Recurrent Ovarian Carcinoma; Recurrent Primary Peritoneal Carcinoma; Unresectable HER2-Negative Breast Carcinoma; Unresectable Malignant Solid Neoplasm
National Cancer Institute (NCI)
 2023-04-07 PHASE2
NCT05919108 RECRUITING
Anatomic Stage I Breast Cancer; Anatomic Stage II Breast Cancer; Anatomic Stage III Breast Cancer; Invasive Breast Lobular Carcinoma
Vanderbilt-Ingram Cancer Center
 2024-06-11 PHASE2
NCT03919292 SUSPENDED
Solid Tumor, Adult
Virginia Commonwealth University
 2019-05-01 PHASE1; PHASE2
NCT04374305 RECRUITING
Neurofibromatosis Type 2; Vestibular Schwannoma; Non-vestibular Schwannoma; Meningioma; Ependymoma
Scott R. Plotkin, MD, PhD
 2020-06-20 PHASE2
NCT05372614 RECRUITING
Metastatic Malignant Solid Neoplasm; Metastatic Pancreatic Adenocarcinoma; Stage III Pancreatic Cancer AJCC v8; Stage IV Pancreatic Cancer AJCC v8; Unresectable Malignant Solid Neoplasm; Unresectable Pancreatic Adenocarcinoma
National Cancer Institute (NCI)
 2022-10-05 PHASE1
NCT06109467 ACTIVE_NOT_RECRUITING
GastroEsophageal Cancer; Gastric Cancer
H. Lee Moffitt Cancer Center and Research Institute
 2024-01-12 PHASE2
NCT06813079 RECRUITING
Pancreatic Ductal Carcinoma; Advanced Cancer; Epithelial Tumor
University Health Network, Toronto
 2025-04-07 PHASE2
NCT05760612 RECRUITING
Early-stage Breast Cancer; HER2-positive Breast Cancer; Adjuvant Treatment After Trastuzumab; RCB Classification 1-2; Neratini
Sun Yat-Sen Memorial Hospital of Sun Yat-Sen University
 2023-03-14 PHASE3
NCT02977780 RECRUITING
Glioblastoma
David Reardon, MD
 2017-02-09 PHASE2
NCT07333937 NOT_YET_RECRUITING
Brest Cancer
Xijing Hospital
 2025-12-20 PHASE2
NCT07152782 NOT_YET_RECRUITING
Breast Cancer With Brain Metastasis; HER2-positive Breast Cancer
Fudan University
 2025-11 PHASE2
NCT05615818 RECRUITING
Biliary Tract Neoplasms
UNICANCER
 2024-07-18 PHASE3
NCT06892093 RECRUITING
HER2-positive Breast Cancer; Diarrhea Caused by Drug; Neratinib
Sun Yat-Sen Memorial Hospital of Sun Yat-Sen University
 2025-08-07
NCT06126276 RECRUITING
Malignant Female Reproductive System Neoplasm; Malignant Solid Neoplasm; Recurrent Malignant Female Reproductive System Neoplasm; Recurrent Malignant Solid Neoplasm
National Cancer Institute (NCI)
 2024-05-07 PHASE2
NCT04886531 RECRUITING
Breast Cancer; HER2-positive Breast Cancer; ER Positive Breast Cancer; PR-Positive Breast Cancer
Ruth O'Regan
 2022-07-21 PHASE2
NCT03377387 ACTIVE_NOT_RECRUITING
Breast Cancer
Memorial Sloan Kettering Cancer Center
 2017-12-13 PHASE1; PHASE2
NCT02932280 ACTIVE_NOT_RECRUITING
Solid Tumor; Central Nervous System Tumor; Lymphoma; Leukemia
Memorial Sloan Kettering Cancer Center
 2016-10 PHASE1; PHASE2
NCT06374888 NOT_YET_RECRUITING
Her2-positive Advanced Esophageal/Esophagogastric Junction/Gastric Adenocarcinoma With Brain Metastasis
Convalife (Shanghai) Co., Ltd.
 2024-04-30 PHASE2
NCT04965064 RECRUITING
Breast Cancer
University of Rochester
 2022-09-23 PHASE2
NCT05388149 RECRUITING
Breast Cancer; HER2-positive Breast Cancer
University Health Network, Toronto
 2022-12-06 PHASE2
NCT05274048 RECRUITING
Gastric Cancer; Gastrointestinal Cancer; HER2 Gene Mutation
Fox Chase Cancer Center
 2022-06-24 PHASE1
NCT06008275 RECRUITING
Metastatic Triple-Negative Breast Carcinoma; Breast Cancer
Baylor Research Institute
 2024-09-16 EARLY_PHASE1
NCT03065387 TERMINATED
Advanced Malignant Solid Neoplasm; EGFR Gene Amplification; EGFR Gene Mutation; ERBB2 Gene Amplification; ERBB2 Gene Mutation; ERBB3 Gene Mutation; ERBB4 Gene Mutation; KRAS Gene Mutation; Metastatic Malignant Solid Neoplasm; Refractory Malignant Solid Neoplasm
M.D. Anderson Cancer Center
 2017-10-31 PHASE1
NCT06519110 UNKNOWN
Advanced Solid Tumor
Convalife (Shanghai) Co., Ltd.
 2024-08-10 PHASE2
NCT03101748 TERMINATED
Breast Inflammatory Carcinoma; Locally Advanced Breast Carcinoma; Metastatic Breast Carcinoma; Stage III Breast Cancer AJCC v7; Stage IIIA Breast Cancer AJCC v7; Stage IIIB Breast Cancer AJCC v7; Stage IIIC Breast Cancer AJCC v7; Stage IV Breast Cancer AJCC v6 and v7
M.D. Anderson Cancer Center
 2018-02-19 PHASE1; PHASE2
NCT06029816 UNKNOWN
NSCLC
Convalife (Shanghai) Co., Ltd.
 2023-11-20 PHASE2
NCT03812393 UNKNOWN
Triple Negative Breast Cancer; Early-stage Breast Cancer; HER2-positive Breast Cancer
West Cancer Center
 2019-06-21 PHASE2
NCT05243641 TERMINATED
Metastatic Breast Cancer; Breast Cancer
M.D. Anderson Cancer Center
 2022-08-18 PHASE1; PHASE2
NCT04502602 ACTIVE_NOT_RECRUITING
Advanced Solid Tumor; Ovarian Cancer
Virginia Commonwealth University
 2020-08-24 PHASE1
NCT05252988 ACTIVE_NOT_RECRUITING
Early-stage Breast Cancer; HER2 Positive Breast Cancer; Hormone Receptor Positive
Spanish Breast Cancer Research Group
 2022-08-31 PHASE2
NCT04901299 WITHDRAWN
Stage IV (Metastatic) Breast Cancer; Metastatic Breast Cancer; ER Positive Breast Cancer; PR-Positive Breast Cancer; HER2-negative Breast Cancer; Invasive Breast Cancer
Massachusetts General Hospital
 2023-07 PHASE2
NCT05154396 UNKNOWN
HER2-positive Breast Cancer
Chinese Academy of Medical Sciences
 2022-01-01 PHASE2
NCT06083662 COMPLETED
Metastatic Cancer; HER2 Gene Mutation
Korea University Guro Hospital
 2021-06-15 PHASE2
NCT05512182 WITHDRAWN
Recurrent/Advanced Gastric Cancer
Yonsei University
 2022-09-15 PHASE2
NCT01494662 COMPLETED
Breast Cancer
Dana-Farber Cancer Institute
 2012-02 PHASE2
NCT01953926 TERMINATED
Solid Tumors Harboring Somatic HER2 or EGFR Exon 18 Mutations
Puma Biotechnology, Inc.
 2013-09-30 PHASE2
NCT05388149 Recruiting
Breast Cancer|HER2-positive Breast Cancer
University Health Network Toronto
 2022-12-06 Phase 2
NCT03182634 UNKNOWN
Advanced Breast Cancer
Institute of Cancer Research, United Kingdom
 2016-12-15 PHASE2
NCT03094052 COMPLETED
HER2-positive Breast Cancer; Breast Adenocarcinoma; Stage II Breast Cancer AJCC v6 and v7; Stage IIA Breast Cancer AJCC v6 and v7; Stage IIB Breast Cancer AJCC v6 and v7; Stage III Breast Cancer AJCC v7; Stage IIIA Breast Cancer AJCC v7; Stage IIIB Breast Cancer AJCC v7; Stage IIIC Breast Cancer AJCC v7
University of California, San Francisco
 2018-10-09 PHASE2
NCT05252988 Recruiting
Early-stage Breast Cancer|HER2 Positive Breast Cancer|Hormone Receptor Positive
Spanish Breast Cancer Research Group|Puma Biotechnology Inc.|Pierre Fabre Laboratories
 2022-08-31 Phase 2
NCT04366713 COMPLETED
HER2 Amplified Breast Cancer
Puma Biotechnology, Inc.
 2020-06-30 PHASE2
NCT04460430 TERMINATED
Breast Cancer
SOLTI Breast Cancer Research Group
 2020-07-23 PHASE2
NCT04856475 WITHDRAWN
Breast Cancer; Brain Metastases
Jules Bordet Institute
 2021-11-24 PHASE2
NCT04856475 Withdrawn
Breast Cancer|Brain Metastases
Jules Bordet Institute
 2021-11-24 Phase 2
NCT03457896 UNKNOWN
Metastatic Colorectal Cancer
NSABP Foundation Inc
 2018-05-18 PHASE2
NCT04781374 WITHDRAWN
Metastatic Prostate Adenocarcinoma; Castration-resistant Prostate Cancer; Prostate Cancer; Prostate Cancer Metastatic
Beth Israel Deaconess Medical Center
 2021-05-21 PHASE2
NCT03289039 TERMINATED
Breast Cancer
Dana-Farber Cancer Institute
 2017-10-25 PHASE2
NCT04781374 Withdrawn
Metastatic Prostate Adenocarcinoma|Castration-resistant Prostate Cancer|Prostate Cancer|Prostate Cancer Metastatic
Beth Israel Deaconess Medical Center|Puma Biotechnology Inc.|Dana-Farber Cancer Institute
 2021-05-21 Phase 2
NCT02400476 COMPLETED
Early Stage HER2+ Breast Cancer
Puma Biotechnology, Inc.
 2015-02 PHASE2
NCT01670877 COMPLETED
Breast Neoplasms
Washington University School of Medicine
 2012-12-11 PHASE2
NCT03878524 TERMINATED
Accelerated Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive; Anatomic Stage IV Breast Cancer AJCC v8; Anemia; Ann Arbor Stage III Hodgkin Lymphoma; Ann Arbor Stage III Non-Hodgkin Lymphoma; Ann Arbor Stage IV Hodgkin Lymphoma; Ann Arbor Stage IV Non-Hodgkin Lymphoma; Atypical Chronic Myeloid Leukemia, BCR-ABL1 Negative; Blast Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive; Castration-Resistant Prostate Carcinoma; Chronic Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive; Hematopoietic and Lymphoid System Neoplasm; Locally Advanced Pancreatic Adenocarcinoma; Metastatic Breast Carcinoma; Metastatic Malignant Solid Neoplasm; Metastatic Pancreatic Adenocarcinoma; Myelodysplastic/Myeloproliferative Neoplasm With Ring Sideroblasts and Thrombocytosis; Myelodysplastic/Myeloproliferative Neoplasm, Unclassifiable; Primary Myelofibrosis; Recurrent Acute Lymphoblastic Leukemia; Recurrent Acute Myeloid Leukemia; Recurrent Chronic Lymphocytic Leukemia; Recurrent Chronic Myelogenous Leukemia, BCR-ABL1 Positive; Recurrent Hematologic Malignancy; Recurrent Hodgkin Lymphoma; Recurrent Myelodysplastic Syndrome; Recurrent Myelodysplastic/Myeloproliferative Neoplasm; Recurrent Myeloproliferative Neoplasm; Recurrent Non-Hodgkin Lymphoma; Recurrent Plasma Cell Myeloma; Recurrent Small Lymphocytic Lymphoma; Refractory Acute Lymphoblastic Leukemia; Refractory Acute Myeloid Leukemia; Refractory Chronic Lymphocytic Leukemia; Refractory Chronic Myelogenous Leukemia, BCR-ABL1 Positive; Refractory Chronic Myelomonocytic Leukemia; Refractory Hematologic Malignancy; Refractory Hodgkin Lymphoma; Refractory Malignant Solid Neoplasm; Refractory Myelodysplastic Syndrome; Refractory Myelodysplastic/Myeloproliferative Neoplasm; Refractory Non-Hodgkin Lymphoma; Refractory Plasma Cell Myeloma; Refractory Primary Myelofibrosis; Refractory Small Lymphocytic Lymphoma; Stage II Pancreatic Cancer AJCC v8; Stage III Pancreatic Cancer AJCC v8; Stage IV Pancreatic Cancer AJCC v8; Stage IV Prostate Cancer AJCC v8; Unresectable Pancreatic Adenocarcinoma
OHSU Knight Cancer Institute
 2020-04-01 PHASE1
NCT02236000 COMPLETED
Breast Cancer
NSABP Foundation Inc
 2014-08 PHASE1; PHASE2
NCT02673398 COMPLETED
HER2 Positive Breast Carcinoma; Stage IV Breast Cancer AJCC v6 and v7
City of Hope Medical Center
 2016-12-02 PHASE2
NCT01808573 COMPLETED
HER2+ Metastatic Breast Cancer (MBC)
Puma Biotechnology, Inc.
 2013-03-29 PHASE3
NCT02593708 TERMINATED
Solid Tumor
Michelle Melisko
 2015-11-03 PHASE1
NCT01827267 COMPLETED
HER2-mutant Non-Small Cell Lung Cancer
Puma Biotechnology, Inc.
 2013-07-01 PHASE2
NCT01960023 WITHDRAWN
Colorectal Cancer
NSABP Foundation Inc
 2013-10 PHASE1; PHASE2
NCT01111825 COMPLETED
Breast Cancer
Puma Biotechnology, Inc.
 2010-04 PHASE1; PHASE2
NCT01008150 COMPLETED
Breast Cancer
NSABP Foundation Inc
 2010-10 PHASE2
NCT02334501 COMPLETED
Healthy
Puma Biotechnology, Inc.
 2014-08 PHASE1
NCT00878709 COMPLETED
Breast Cancer
Puma Biotechnology, Inc.
 2009-07-09 PHASE3
NCT00915018 COMPLETED
Breast Cancer
Puma Biotechnology, Inc.
 2009-08-21 PHASE2
NCT00932464 WITHDRAWN
Healthy
Puma Biotechnology, Inc.
 2012-08 PHASE1
NCT01423123 COMPLETED
Breast Cancer
NSABP Foundation Inc
 2011-05 PHASE1
NCT00838539 COMPLETED
Neoplasms; Malignant Carcinoma
Puma Biotechnology, Inc.
 2009-04 PHASE1
NCT01128842 COMPLETED
Advanced Malignant Solid Tumors
Puma Biotechnology, Inc.
 2010-04 PHASE1
NCT00777101 COMPLETED
Advanced Breast Cancer; Breast Cancer
Puma Biotechnology, Inc.
 2009-02-04 PHASE2
NCT00768469 COMPLETED
Advanced Malignant Solid Tumors
Puma Biotechnology, Inc.
 2008-10 PHASE1
NCT00741260 COMPLETED
Breast Cancer
Puma Biotechnology, Inc.
 2008-12-09 PHASE1; PHASE2
NCT00958724 COMPLETED
Advanced Malignant Solid Tumors
Puma Biotechnology, Inc.
 2009-07 PHASE1
NCT00781430 COMPLETED
Hepatic Insufficiency
Puma Biotechnology, Inc.
 2009-04 PHASE1
NCT00706030 COMPLETED
Breast Cancer; Advanced Malignant Solid Tumors
Puma Biotechnology, Inc.
 2008-04-29 PHASE1; PHASE2
NCT00864487 COMPLETED
Healthy Subjects
Puma Biotechnology, Inc.
 2009-04 PHASE1
NCT00860223 COMPLETED
Healthy
Puma Biotechnology, Inc.
 2009-04 PHASE1
NCT00814060 COMPLETED
Healthy Subjects
Puma Biotechnology, Inc.
 2009-01 PHASE1
NCT00397046 COMPLETED
Tumors
Puma Biotechnology, Inc.
 2006-11 PHASE1
NCT00757809 COMPLETED
Healthy Subjects
Puma Biotechnology, Inc.
 2008-10 PHASE1
NCT00708903 COMPLETED
Breast Cancer
Puma Biotechnology, Inc.
 2008-05 PHASE1
NCT00300781 COMPLETED
Breast Neoplasms; Neoplasms
Puma Biotechnology, Inc.
 2006-08-04 PHASE2
NCT00550212 COMPLETED
Healthy
Puma Biotechnology, Inc.
 2008-03 PHASE1
NCT00146172 COMPLETED
Breast Neoplasms
Puma Biotechnology, Inc.
 2003-11 PHASE1
NCT00366600 COMPLETED
Healthy
Puma Biotechnology, Inc.
 2006-07 PHASE1
NCT00380328 COMPLETED
Healthy
Puma Biotechnology, Inc.
 2006-10 PHASE1
NCT00498745 COMPLETED
Healthy
Puma Biotechnology, Inc.
 2007-07 PHASE1

(data from https://clinicaltrials.gov, updated on 2026-07-27)

Check the Neratinib (HKI-272) product page for in-depth specifications, including solubility, stock solutions, MOA, and working concentrations.

Compliance for Clinical Use

Mechanism and Biochemical Profile

Neratinib selectively and irreversibly binds to the ATP-binding pocket of HER2 and EGFR, targeting a specific cysteine residue to block receptor autophosphorylation and suppress downstream cell signaling pathways. By inhibiting the proliferation of EGFR- and HER2-dependent cancer cells, this target blockade limits tumor growth, providing a strong rationale for its evaluation in HER2-positive breast cancer and other receptor-driven 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.