Clinical Trials

Patupilone (Epothilone B) has been evaluated in numerous Phase I through Phase III clinical trials as both monotherapy and combination regimens across various oncological indications, including advanced malignancies, refractory solid tumors, breast cancer, non-small cell lung cancer, brain metastasis, and hormone-refractory prostate cancer. Sponsored by pharmaceutical leaders such as Novartis Pharmaceuticals alongside academic and federal entities like the National Cancer Institute, these trials encompass completed, terminated, and withdrawn statuses.

NCT Number Recruitment Conditions Sponsor/Collaborators Start Date Phases
NCT00924066 TERMINATED
Cervical Carcinoma; Cervical Adenocarcinoma; Cervical Adenosquamous Carcinoma; Cervical Carcinoma, Non-SquamousType
National Cancer Institute (NCI)
 2008-11 PHASE2
NCT00411528 COMPLETED
Metastatic Hormone Refractory Prostate Cancer
Novartis Pharmaceuticals
 2006-09 PHASE2
NCT00450866 COMPLETED
Breast Cancer; Metastatic Cancer
David Peereboom, MD
 2007-01 PHASE2
NCT00496600 COMPLETED
Refractory Malignancy
University of Medicine and Dentistry of New Jersey
 2007-07 PHASE1
NCT00715013 COMPLETED
Recurrent Glioblastoma Planned for Reoperation
University of Zurich
 2008-07 PHASE1; PHASE2
NCT00426582 COMPLETED
Advanced Solid Tumors
Novartis Pharmaceuticals
 2006-08 PHASE1
NCT00058019 COMPLETED
Anaplastic Large Cell Lymphoma; Recurrent Adult Burkitt Lymphoma; Recurrent Adult Diffuse Large Cell Lymphoma; Recurrent Adult Diffuse Mixed Cell Lymphoma; Recurrent Grade 3 Follicular Lymphoma; Recurrent Mantle Cell Lymphoma
National Cancer Institute (NCI)
 2003-02 PHASE2
NCT00420524 COMPLETED
Advanced Malignancies; Tumors
Novartis Pharmaceuticals
 2006-01 PHASE1
NCT00219297 COMPLETED
Brain Metastasis; Non-small Cell Lung Cancer
Novartis Pharmaceuticals
 2005-11-16 PHASE2
NCT00407251 COMPLETED
Hormone Refractory Prostate Cancer
British Columbia Cancer Agency
 2007-02 PHASE2
NCT00025155 COMPLETED
Primary Peritoneal Cavity Cancer; Recurrent Ovarian Epithelial Cancer
National Cancer Institute (NCI)
 2002-07 PHASE2
NCT00030108 COMPLETED
Brain and Central Nervous System Tumors; Childhood Germ Cell Tumor; Extragonadal Germ Cell Tumor; Kidney Cancer; Leukemia; Liver Cancer; Neuroblastoma; Ovarian Cancer; Sarcoma; Unspecified Childhood Solid Tumor, Protocol Specific
National Institutes of Health Clinical Center (CC)
 2001-11 PHASE1
NCT00262990 COMPLETED
Ovarian Cancer; Fallopian Tube Cancer; Peritoneal Neoplasms
Novartis Pharmaceuticals
 2005-11 PHASE3
NCT00030992 COMPLETED
Renal Cell Carcinoma
National Cancer Institute (NCI)
 2002-02 PHASE2
NCT00171834 COMPLETED
Carcinoma, Non-Small-Cell Lung
Novartis Pharmaceuticals
 2003-08 PHASE1; PHASE2
NCT00420615 COMPLETED
Solid Tumors
Novartis Pharmaceuticals
 2006-12 PHASE1
NCT00328458 COMPLETED
Central Nervous System Neoplasms; Head and Neck Neoplasms
Sidney Kimmel Comprehensive Cancer Center at Thomas Jefferson University
 2004-02 PHASE1
NCT00448396 COMPLETED
Advanced Malignancies
Novartis Pharmaceuticals
 2007-03 PHASE1
NCT00421044 COMPLETED
Advanced Malignancies; Tumors
Novartis Pharmaceuticals
 2006-05 PHASE1
NCT00412789 COMPLETED
Tumors
Novartis Pharmaceuticals
 2006-08 PHASE1
NCT00331643 COMPLETED
Adult Rhabdomyosarcoma; Adult Synovial Sarcoma; Alveolar Childhood Rhabdomyosarcoma; Childhood Synovial Sarcoma; Embryonal Childhood Rhabdomyosarcoma; Previously Treated Childhood Rhabdomyosarcoma; Recurrent Adult Soft Tissue Sarcoma; Recurrent Childhood Rhabdomyosarcoma; Recurrent Childhood Soft Tissue Sarcoma; Recurrent Ewing Sarcoma/Peripheral Primitive Neuroectodermal Tumor; Recurrent Neuroblastoma; Recurrent Osteosarcoma; Recurrent Wilms Tumor and Other Childhood Kidney Tumors
National Cancer Institute (NCI)
 2006-04 PHASE2
NCT00021099 COMPLETED
Distal Urethral Cancer; Metastatic Transitional Cell Cancer of the Renal Pelvis and Ureter; Proximal Urethral Cancer; Recurrent Bladder Cancer; Recurrent Transitional Cell Cancer of the Renal Pelvis and Ureter; Recurrent Urethral Cancer; Regional Transitional Cell Cancer of the Renal Pelvis and Ureter; Stage III Bladder Cancer; Stage IV Bladder Cancer; Transitional Cell Carcinoma of the Bladder; Urethral Cancer Associated With Invasive Bladder Cancer
National Cancer Institute (NCI)
 2001-06 PHASE2
NCT00442741 WITHDRAWN
Solid Tumors
Novartis Pharmaceuticals
 2007-07 PHASE1
NCT00496600 Completed
Refractory Malignancy
University of Medicine and Dentistry of New Jersey|Novartis Pharmaceuticals|National Cancer Institute (NCI)|Rutgers The State University of New Jersey
 2007-07 Phase 1
NCT00442741 Withdrawn
Solid Tumors
Novartis Pharmaceuticals|Novartis
 2007-07 Phase 1
NCT00468260 Terminated
Advanced Malignancies
Novartis Pharmaceuticals|Novartis
 2007-05 Phase 1
NCT00050349 COMPLETED
Carcinoid; Neuroendocrine Tumors
Novartis Pharmaceuticals
 2002-07 PHASE2
NCT00448396 Completed
Advanced Malignancies
Novartis Pharmaceuticals|Novartis
 2007-03 Phase 1
NCT00030706 COMPLETED
Ovarian Cancer; Peritoneal Cavity Cancer
Montefiore Medical Center
 2004-11 PHASE2
NCT00028561 TERMINATED
Unspecified Adult Solid Tumor, Protocol Specific
National Cancer Institute (NCI)
 2001-10 PHASE1
NCT00426140 COMPLETED
Advanced Malignancies; Solid Tumors
Novartis Pharmaceuticals
 2006-08 PHASE1
NCT00969046 COMPLETED
Colon Cancer
Novartis Pharmaceuticals
 2003-11 PHASE1
NCT00080301 COMPLETED
Breast Cancer; Metastases
R-Pharm
 2003-09 PHASE3
NCT00273312 COMPLETED
Hepatocellular Carcinoma
Novartis Pharmaceuticals
 2006-01 PHASE2
NCT00049400 COMPLETED
Lymphoma; Small Intestine Cancer; Unspecified Adult Solid Tumor, Protocol Specific
SWOG Cancer Research Network
 2003-10 PHASE1
NCT00426140 Completed
Advanced Malignancies|Solid Tumors
Novartis Pharmaceuticals|Novartis
 2006-08 Phase 1
NCT00022542 TERMINATED
Recurrent Adult Soft Tissue Sarcoma; Stage III Adult Soft Tissue Sarcoma; Stage IV Adult Soft Tissue Sarcoma
National Cancer Institute (NCI)
 2001-06 PHASE2
NCT00023946 TERMINATED
Adult Primary Cholangiocellular Carcinoma; Adult Primary Hepatocellular Carcinoma; Advanced Adult Primary Liver Cancer; Cholangiocarcinoma of the Extrahepatic Bile Duct; Cholangiocarcinoma of the Gallbladder; Localized Extrahepatic Bile Duct Cancer; Localized Gallbladder Cancer; Localized Resectable Adult Primary Liver Cancer; Localized Unresectable Adult Primary Liver Cancer; Recurrent Adult Primary Liver Cancer; Recurrent Extrahepatic Bile Duct Cancer; Recurrent Gallbladder Cancer; Unresectable Extrahepatic Bile Duct Cancer; Unresectable Gallbladder Cancer
National Cancer Institute (NCI)
 2001-08 PHASE2
NCT00036764 COMPLETED
Recurrent Melanoma; Stage IV Melanoma
National Cancer Institute (NCI)
 2002-02 PHASE2
NCT00005807 COMPLETED
Breast Cancer; Ovarian Cancer; Unspecified Adult Solid Tumor, Protocol Specific
Albert Einstein College of Medicine
 2000-07 PHASE1
NCT00035113 COMPLETED
Prostatic Neoplasms
Novartis Pharmaceuticals
 2002-01 PHASE2
NCT00035126 COMPLETED
Breast Neoplasms
Novartis Pharmaceuticals
 2002-01 PHASE2
NCT00035243 COMPLETED
Kidney Neoplasms
Novartis Pharmaceuticals
 2002-04 PHASE2
NCT00057850 COMPLETED
Head and Neck Cancer
National Cancer Institute (NCI)
 2002-01 PHASE1; PHASE2
NCT00035100 COMPLETED
Ovarian Neoplasms; Peritoneal Neoplasms; Fallopian Tube Neoplasms
Novartis Pharmaceuticals
 2001-09 PHASE2
NCT00035165 COMPLETED
Melanoma
Novartis Pharmaceuticals
 2002-03 PHASE2
NCT00035087 COMPLETED
Colorectal Neoplasms; Colonic Neoplasms
Novartis Pharmaceuticals
 2002-05 PHASE2
NCT00020904 COMPLETED
Breast Cancer
Memorial Sloan Kettering Cancer Center
 2001-02 PHASE2
NCT00022477 COMPLETED
Colorectal Cancer
University of Chicago
 2001-09 PHASE2
NCT00468260 TERMINATED
Advanced Malignancies
Novartis Pharmaceuticals
 2007-05 PHASE1
NCT00016393 COMPLETED
Prostate Cancer
SWOG Cancer Research Network
 2001-06 PHASE2
NCT00025194 COMPLETED
Prostate Cancer
Memorial Sloan Kettering Cancer Center
 2001-07 PHASE1; PHASE2
NCT00016965 COMPLETED
Pancreatic Cancer
SWOG Cancer Research Network
 2001-07 PHASE2
NCT00045097 COMPLETED
Breast Cancer
National Cancer Institute (NCI)
 2002-05 PHASE2
NCT00052572 COMPLETED
Leukemia; Lymphoma
Memorial Sloan Kettering Cancer Center
 2002-10 PHASE2

(data from https://clinicaltrials.gov, updated on 2019-11-21)

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

Compliance for Clinical Use

Mechanism and Biochemical Profile

Patupilone binds to tubulin with high affinity and stabilizes microtubule dynamics at an IC50 of 3.5 nM, effectively suppressing microtubule growth and shortening rates to disrupt mitotic spindle assembly. This sustained suppression prevents proper cell division and induces cell cycle arrest in mitosis, providing a clear mechanistically driven strategy to inhibit tumor proliferation in advanced solid tumors and refractory 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.