Clinical Trials

Buparlisib (BKM120) has been evaluated in numerous clinical trials from Early Phase I through Phase III across diverse malignancies, including head and neck, breast, cervical, lung, urothelial, leukemia, and melanoma. Sponsored by pharmaceutical companies and academic medical centers, these studies encompass active, completed, terminated, and withdrawn recruitment statuses. Advanced evaluation includes an ongoing Phase III clinical trial investigating buparlisib combined with paclitaxel for recurrent or metastatic head and neck squamous cell carcinoma.

NCT Number Recruitment Conditions Sponsor/Collaborators Start Date Phases
NCT04338399 COMPLETED
Head and Neck Cancer
Adlai Nortye Biopharma Co., Ltd.
 2020-12-12 PHASE3
NCT02159066 COMPLETED
Melanoma
Pfizer
 2014-07-23 PHASE2
NCT02756247 COMPLETED
Lymphoma; Mantle Cell Lymphoma; Follicular Lymphoma; Diffuse Large B Cell Lymphoma
Memorial Sloan Kettering Cancer Center
 2016-05-09 PHASE1
NCT04338399 Active not recruiting
Head and Neck Cancer
Adlai Nortye Biopharma Co. Ltd.
 2020-12-12 Phase 3
NCT01551030 COMPLETED
Metastatic Transitional Cell Carcinoma of the Urothelium
Memorial Sloan Kettering Cancer Center
 2012-03 PHASE2
NCT02113878 COMPLETED
Carcinoma, Squamous Cell of Head and Neck; HPV Positive Oropharyngeal Squamous Cell Carcinoma; Hypopharyngeal Cancer; Early Invasive Cervical Squamous Cell Carcinoma; Carcinoma of Larynx; Cancer of Nasopharynx
Dana-Farber Cancer Institute
 2014-09-29 PHASE1
NCT02614508 TERMINATED
Recurrent Chronic Lymphocytic Leukemia; Recurrent Small Lymphocytic Lymphoma; Refractory Chronic Lymphocytic Leukemia; Refractory Small Lymphocytic Lymphoma
Emory University
 2016-01 PHASE1
NCT01623349 COMPLETED
Ovarian Cancer; Breast Cancer
Dana-Farber Cancer Institute
 2012-09 PHASE1
NCT02000882 COMPLETED
Brain Metastases; Breast Cancer; Metastatic Breast Cancer
US Oncology Research
 2014-05-29 PHASE2
NCT02340780 COMPLETED
Chronic Lymphocytic Leukemia
Canadian Cancer Trials Group
 2015-04-27 PHASE2
NCT02049541 COMPLETED
Extranodal Marginal Zone B-cell Lymphoma of Mucosa-associated Lymphoid Tissue; Nodal Marginal Zone B-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
Kami Maddocks, MD
 2014-07-21 PHASE1
NCT01737450 COMPLETED
Head and Neck Neoplasms; Neoplasm Metastasis; Recurrent Disease; Progressive Disease
Centre Leon Berard
 2013-01 PHASE2
NCT02058381 COMPLETED
Pre-menopausal Breast Cancer; PI3K Pathway Inhibition
Novartis Pharmaceuticals
 2014-05-06 PHASE1
NCT02088684 COMPLETED
Breast Cancer
Novartis Pharmaceuticals
 2014-05-19 PHASE1
NCT01816984 COMPLETED
Metastatic Squamous Neck Cancer With Occult Primary Squamous Cell Carcinoma; Recurrent Metastatic Squamous Neck Cancer With Occult Primary; Recurrent Salivary Gland Cancer; Recurrent Squamous Cell Carcinoma of the Hypopharynx; Recurrent Squamous Cell Carcinoma of the Larynx; Recurrent Squamous Cell Carcinoma of the Lip and Oral Cavity; Recurrent Squamous Cell Carcinoma of the Nasopharynx; Recurrent Squamous Cell Carcinoma of the Oropharynx; Recurrent Squamous Cell Carcinoma of the Paranasal Sinus and Nasal Cavity; Recurrent Verrucous Carcinoma of the Larynx; Recurrent Verrucous Carcinoma of the Oral Cavity; Salivary Gland Squamous Cell Carcinoma; Stage IV Squamous Cell Carcinoma of the Hypopharynx; Stage IV Squamous Cell Carcinoma of the Nasopharynx; Stage IVA Salivary Gland Cancer; Stage IVA Squamous Cell Carcinoma of the Larynx; Stage IVA Squamous Cell Carcinoma of the Lip and Oral Cavity; Stage IVA Squamous Cell Carcinoma of the Oropharynx; Stage IVA Squamous Cell Carcinoma of the Paranasal Sinus and Nasal Cavity; Stage IVA Verrucous Carcinoma of the Larynx; Stage IVA Verrucous Carcinoma of the Oral Cavity; Stage IVB Salivary Gland Cancer; Stage IVB Squamous Cell Carcinoma of the Larynx; Stage IVB Squamous Cell Carcinoma of the Lip and Oral Cavity; Stage IVB Squamous Cell Carcinoma of the Oropharynx; Stage IVB Squamous Cell Carcinoma of the Paranasal Sinus and Nasal Cavity; Stage IVB Verrucous Carcinoma of the Larynx; Stage IVB Verrucous Carcinoma of the Oral Cavity; Stage IVC Salivary Gland Cancer; Stage IVC Squamous Cell Carcinoma of the Larynx; Stage IVC Squamous Cell Carcinoma of the Lip and Oral Cavity; Stage IVC Squamous Cell Carcinoma of the Oropharynx; Stage IVC Squamous Cell Carcinoma of the Paranasal Sinus and Nasal Cavity; Stage IVC Verrucous Carcinoma of the Larynx; Stage IVC Verrucous Carcinoma of the Oral Cavity; Tongue Cancer
University of Chicago
 2013-05 PHASE1; PHASE2
NCT02452294 UNKNOWN
Malignant Melanoma; Metastases
University Hospital Tuebingen
 2015-07 PHASE2
NCT02487823 TERMINATED
Non Castrate Metastatic Prostate Cancer
Institut Paoli-Calmettes
 2014-10 PHASE1
NCT02128724 COMPLETED
Carcinoma, Non-Small-Cell Lung
University of Oxford
 2013-04 PHASE1
NCT01589861 TERMINATED
Breast Cancer
Institut Paoli-Calmettes
 2011-12 PHASE1; PHASE2
NCT01730248 TERMINATED
Myelofibrosis
Novartis Pharmaceuticals
 2012-12-18 PHASE1
NCT02404844 COMPLETED
Breast Cancer
University Hospital, Essen
 2014-12 PHASE2
NCT01613677 WITHDRAWN
Treatment for Metastatic or Locally Advanced Cervical Cancer
Novartis Pharmaceuticals
 2015-11 PHASE2
NCT01693614 COMPLETED
Diffuse Large B-cell Lymphoma, Mantle Cell Lymphoma, Follicular Lymphoma
Novartis Pharmaceuticals
 2013-02-28 PHASE2
NCT01923168 COMPLETED
Breast Cancer
Novartis Pharmaceuticals
 2014-03-11 PHASE2
NCT02439489 COMPLETED
Solid Tumors
Fondazione Michelangelo
 2012-12 PHASE1
NCT01473901 COMPLETED
Glioblastoma
Novartis Pharmaceuticals
 2011-12-30 PHASE1
NCT01470209 COMPLETED
Solid Tumors; Lung Cancer
Emory University
 2012-01 PHASE1
NCT02303041 TERMINATED
Carcinoma, Basal Cell; Recurrent Skin Cancer; Skin Neoplasms; Basal Cell Nevus Syndrome
Anne Chang
 2015-02 PHASE2
NCT01852292 TERMINATED
Head and Neck Squamous Cell Carcinoma
Novartis Pharmaceuticals
 2013-10-01 PHASE2
NCT01297452 COMPLETED
Solid Tumors
Memorial Sloan Kettering Cancer Center
 2011-02-15 PHASE1
NCT02220855 COMPLETED
Thymoma
Patrick Joseph Loehrer Sr.
 2014-10-01 PHASE2
NCT01300962 COMPLETED
Metastatic Breast Cancer
UNC Lineberger Comprehensive Cancer Center
 2011-08 PHASE1
NCT01870726 TERMINATED
c-MET Inhibitor; PI3K Inhibitor, PTEN Mutations, Homozygous Del. of PTEN or PTEN Neg. by IHC, c-Met Ampli. by FISH, INC280, BKM120, Buparlisib; Recurrent GBM
Novartis Pharmaceuticals
 2014-01-09 PHASE1; PHASE2
NCT01339442 COMPLETED
Estrogen Receptor-positive Breast Cancer; Recurrent Breast Cancer; Stage IV Breast Cancer
Washington University School of Medicine
 2011-11-14 PHASE1
NCT01349660 COMPLETED
Glioblastoma Multiforme
SCRI Development Innovations, LLC
 2011-12 PHASE1; PHASE2
NCT02154776 COMPLETED
Advanced or Metastatic Breast Cancer
Novartis Pharmaceuticals
 2014-06-27 PHASE1
NCT02301364 COMPLETED
Lymphoma; Primary Central Nervous System Lymphoma; Recurrent/Refractory Secondary Central Nervous System Lymphoma
Memorial Sloan Kettering Cancer Center
 2014-11-20 PHASE2
NCT01396499 COMPLETED
Leukemia
M.D. Anderson Cancer Center
 2012-07 PHASE1
NCT01339052 COMPLETED
Glioblastoma
L. Nicolas Gonzalez Castro, MD, PhD
 2011-09 PHASE2
NCT01833169 COMPLETED
PI3K Pathway Activated Tumors
Novartis Pharmaceuticals
 2013-03-29 PHASE2
NCT01719250 COMPLETED
Recurrent Adult Diffuse Large Cell Lymphoma; Recurrent Grade 3 Follicular Lymphoma; Recurrent Mantle Cell Lymphoma; Refractory Mantle Cell Lymphoma; Transformed Recurrent Non-Hodgkin Lymphoma
Mayo Clinic
 2012-12 EARLY_PHASE1
NCT01723800 COMPLETED
Recurrent Non-small Cell Lung Cancer; Stage IV Non-small Cell Lung Cancer
City of Hope Medical Center
 2013-07 PHASE1
NCT01283048 COMPLETED
Renal Cell Carcinoma
Toni Choueiri, MD
 2011-09 PHASE1
NCT01971489 WITHDRAWN
Adult Solid Neoplasm; Recurrent Non-Small Cell Lung Carcinoma; Stage IIIA Non-Small Cell Lung Cancer; Stage IIIB Non-Small Cell Lung Cancer; Stage IV Non-Small Cell Lung Cancer
Roswell Park Cancer Institute
 2015-09 PHASE1
NCT01468688 COMPLETED
3rd Line GIST
Novartis Pharmaceuticals
 2012-04-20 PHASE1
NCT01934361 COMPLETED
Recurrent Glioblastoma Multiforme
Novartis Pharmaceuticals
 2014-02-28 PHASE1
NCT01487265 COMPLETED
Non Small Cell Lung Cancer
SCRI Development Innovations, LLC
 2014-03 PHASE2
NCT01633060 TERMINATED
Metastatic Breast Cancer
Novartis Pharmaceuticals
 2012-10-03 PHASE3
NCT01806649 TERMINATED
Esophageal Cancer
Prince of Songkla University
 2013-07-01 PHASE2
NCT02194049 COMPLETED
Extensive Stage Small Cell Lung Cancer; Unspecified Adult Solid Tumor, Protocol Specific
University of California, Davis
 2014-07 PHASE1
NCT01570296 COMPLETED
Non-Small Cell Lung Cancer; Solid Tumors
National Cancer Centre, Singapore
 2011-10-03 PHASE1
NCT01385293 TERMINATED
Prostate Cancer; Metastatic (Spread to Other Areas of the Body)
Andrew J. Armstrong, MD
 2011-08 PHASE2
NCT02614508 Terminated
Recurrent Chronic Lymphocytic Leukemia|Recurrent Small Lymphocytic Lymphoma|Refractory Chronic Lymphocytic Leukemia|Refractory Small Lymphocytic Lymphoma
Emory University|Novartis
 2016-01 Phase 1
NCT01613677 Withdrawn
Treatment for Metastatic or Locally Advanced Cervical Cancer
Novartis Pharmaceuticals|Novartis
 2015-11 Phase 2
NCT01629615 COMPLETED
Breast Cancer
SOLTI Breast Cancer Research Group
 2012-06 PHASE2
NCT01591421 COMPLETED
Metastatic Colorectal Cancer
Canadian Cancer Trials Group
 2012-09-06 PHASE1; PHASE2
NCT01790932 COMPLETED
Breast Cancer
Dana-Farber Cancer Institute
 2012-06 PHASE2
NCT01911325 TERMINATED
Squamous Non-small Cell Lung Cancer
Novartis Pharmaceuticals
 2013-10 PHASE1
NCT01634061 COMPLETED
Castration-resistant Prostate Cancer
Novartis Pharmaceuticals
 2012-09 PHASE1
NCT01540253 COMPLETED
Unspecified Adult Solid Tumor, Protocol Specific
Roswell Park Cancer Institute
 2012-05 PHASE1
NCT01953445 WITHDRAWN
Breast Neoplasms
Washington University School of Medicine
 2014-08 PHASE2
NCT01572727 COMPLETED
Breast Cancer
Novartis Pharmaceuticals
 2012-08 PHASE2; PHASE3
NCT01571024 COMPLETED
Advanced Solid Tumors; Metastatic Colorectal Cancer; Metastatic Pancreatic Cancer
UNC Lineberger Comprehensive Cancer Center
 2012-05 PHASE1
NCT01610284 COMPLETED
Breast Cancer
Novartis Pharmaceuticals
 2012-08-07 PHASE3
NCT01576666 COMPLETED
Dose Escalation; Safety; Preliminary Efficacy; Advanced Solid Tumors; Metastatic Breast Cancer; Advanced Pancreatic Adenocarcinoma; Metastatic Colorectal Cancer; Recurrent Glioblastoma Multiforme; Gastric Cancer; Gastroesophageal Junction Cancer; Triple Negative Metastatic Breast Cancer; Hormone Receptor Positive (ER+/PR+, and Her2-) Metastatic Breast Cancer
Novartis Pharmaceuticals
 2012-07 PHASE1
NCT02048787 COMPLETED
Renal Impairment
Novartis Pharmaceuticals
 2014-03 PHASE1
NCT02117817 WITHDRAWN
Ovarian Cancer; Endometrial Cancer; Recurrent Ovarian Cancer; Recurrent Endometrial Cancer
Rutgers, The State University of New Jersey
 2015-02 PHASE1
NCT01816594 COMPLETED
HER2-positive Newly Diagnosed, Primary Breast Cancer
Novartis Pharmaceuticals
 2013-09-03 PHASE2
NCT01695473 TERMINATED
High Risk Prostate Cancer
Won Kim
 2013-04-23 PHASE2
NCT02035124 WITHDRAWN
Advanced Prostate Cancer
SCRI Development Innovations, LLC
 2014-04 PHASE2
NCT01741753 TERMINATED
Prostate Cancer
Dana-Farber Cancer Institute
 2012-11 PHASE1
NCT01155453 COMPLETED
Advanced and Selected Solid Tumors
Novartis Pharmaceuticals
 2010-04 PHASE1
NCT01397877 COMPLETED
Endometrial Cancer
ARCAGY/ GINECO GROUP
 2011-12 PHASE2
NCT01297491 COMPLETED
Non-small Cell Lung Cancer
Novartis Pharmaceuticals
 2011-05 PHASE2
NCT01285466 COMPLETED
Metastatic or Locally Advanced Solid Tumors
Novartis Pharmaceuticals
 2011-01 PHASE1
NCT01132664 TERMINATED
Metastatic Breast Cancer; HER2+ Breast Cancer
Novartis Pharmaceuticals
 2010-05 PHASE1; PHASE2
NCT01550380 WITHDRAWN
Endometrial Cancer
University of Utah
 2014-05 PHASE2
NCT01820325 TERMINATED
Non-Small Cell Lunch Cancer
Novartis Pharmaceuticals
 2013-09-09 PHASE1
NCT01304602 COMPLETED
Colorectal Cancer
University of Kansas Medical Center
 2011-02 PHASE1
NCT01626209 COMPLETED
Advanced Breast Cancer, Advanced Carcinomas With Squamous Cell Histology
Novartis Pharmaceuticals
 2012-07 PHASE1
NCT01363232 COMPLETED
Advanced Solid Tumors; Selected Solid Tumors
Array Biopharma, now a wholly owned subsidiary of Pfizer
 2011-08 PHASE1
NCT01289041 COMPLETED
Advanced Endometrial Cancer
Novartis Pharmaceuticals
 2011-02 PHASE2
NCT01513356 COMPLETED
Breast Cancer
Sofia Perea, Director Clinical Trials Unit.
 2012-10 EARLY_PHASE1
NCT01512251 COMPLETED
BRAF Mutant Metastatic Melanoma
University of California, San Francisco
 2012-06-09 PHASE1; PHASE2
NCT01527877 UNKNOWN
Head Neck Cancer Squamous Cell Metastatic; Head Neck Cancer Squamous Cell Recurrent
Yonsei University
 2012-09 PHASE2
NCT01727128 COMPLETED
Hepatic Impairment
Novartis Pharmaceuticals
 2011-10 PHASE1
NCT01501604 WITHDRAWN
Lung Cancer; Breast Cancer; Colorectal Cancer; Cholangiocarcinoma; Solid Tumors
Massachusetts General Hospital
 2012-01 PHASE2
NCT01830504 UNKNOWN
Thyroid Cancers
Hospices Civils de Lyon
 2013-04 PHASE2
NCT01248494 COMPLETED
Metastatic Breast Cancer
Vanderbilt-Ingram Cancer Center
 2010-11 PHASE1
NCT01283503 COMPLETED
Advanced Solid Tumor
Novartis Pharmaceuticals
 2009-10 PHASE1
NCT01068483 COMPLETED
Breast Cancer; Colon Cancer; Ovarian Cancer; Endometrium Cancer
Novartis Pharmaceuticals
 2008-11 PHASE1

(data from https://clinicaltrials.gov, updated on 2025-11-17)

Check the Buparlisib (BKM120) product page for in-depth specifications, including solubility, stock solutions, MOA, and working concentrations.

Compliance for Clinical Use

Mechanism and Biochemical Profile

Buparlisib (BKM120) is an oral, ATP-competitive pan-class I phosphatidylinositol 3-kinase (PI3K) inhibitor that binds the p110α, p110β, p110δ, and p110γ catalytic subunits, thereby blocking phosphatidylinositol-3,4,5-trisphosphate production and inhibiting downstream AKT pathway activation. This biochemical blockade suppresses cell survival pathways and induces tumor cell apoptosis, providing the pharmacological rationale for its clinical evaluation in PI3K-driven malignancies such as head and neck cancer and breast cancer.

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.