Clinical Trials

Exatecan has been evaluated across numerous clinical trials spanning early to late phases to assess its therapeutic efficacy in advanced malignancies, including pancreatic, gastric, and ovarian cancers, leukemia, myelodysplastic syndromes, and metastatic solid tumors. Sponsored by pharmaceutical organizations and cooperative research groups such as the National Cancer Institute, these protocols range from completed evaluations to actively recruiting and withdrawn studies.

NCT Number Recruitment Conditions Sponsor/Collaborators Start Date Phases
NCT07454642 RECRUITING
Vulvar Adenocarcinoma; PDAC - Pancreatic Ductal Adenocarcinoma; Gastric Adenocarcinoma; GEJ Adenocarcinoma; Cervical Adenocarcinoma; Cervical Adenosquamous Carcinoma; Small Cell Carcinoma of Lung; Colorectal Cancer; Hormone Receptor Positive Breast Carcinoma
Avacta Life Sciences Ltd
 2026-03-31 PHASE1
NCT07360314 RECRUITING
Advanced Solid Tumors
EMD Serono Research & Development Institute, Inc.
 2026-02-13 PHASE1
NCT06730100 WITHDRAWN
Metastatic Mismatch Repair Proficient Colorectal Carcinoma; Refractory Mismatch Repair Proficient Colorectal Carcinoma; Stage IV Colorectal Cancer AJCC v8
National Cancer Institute (NCI)
 2026-02-17 PHASE2
NCT05691517 RECRUITING
Advanced Malignant Solid Neoplasm; Metastatic Malignant Solid Neoplasm
National Cancer Institute (NCI)
 2024-06-12 PHASE1
NCT00055939 COMPLETED
Sarcoma
Daiichi Sankyo
 2003-01 PHASE2
NCT00055952 COMPLETED
Sarcoma
Daiichi Sankyo
 2003-01 PHASE2
NCT00004047 COMPLETED
Leukemia; Myelodysplastic Syndromes
Daiichi Sankyo
 1999-06 PHASE1
NCT00023972 COMPLETED
Pancreatic Cancer
Daiichi Sankyo
 2001-07 PHASE3
NCT00003951 COMPLETED
Pancreatic Cancer
Daiichi Sankyo
 1999-06 PHASE2
NCT00005938 COMPLETED
Extrahepatic Bile Duct Cancer; Gallbladder Cancer; Liver Cancer
Daiichi Sankyo
 2000-03 PHASE2
NCT00004108 COMPLETED
Liver Cancer
Daiichi Sankyo
 1999-09 PHASE2
NCT00041236 COMPLETED
Sarcoma
European Organisation for Research and Treatment of Cancer - EORTC
 2002-05 PHASE2
NCT00004212 COMPLETED
Brain and Central Nervous System Tumors; Lymphoma; Unspecified Childhood Solid Tumor, Protocol Specific
Daiichi Sankyo
 1999-09 PHASE1
NCT00017212 COMPLETED
Esophageal Cancer; Gastric Cancer
Daiichi Sankyo
 2001-04 PHASE2
NCT00004866 COMPLETED
Cervical Cancer
Daiichi Sankyo
 2000-01 PHASE2
NCT00004046 COMPLETED
Breast Cancer
Daiichi Sankyo
 1999-06 PHASE2
NCT00004045 COMPLETED
Prostate Cancer
Daiichi Sankyo
 1999-06 PHASE2
NCT00004060 COMPLETED
Fallopian Tube Cancer; Ovarian Cancer; Primary Peritoneal Cavity Cancer
Daiichi Sankyo
 1999-07 PHASE2
NCT00045318 COMPLETED
Unspecified Adult Solid Tumor, Protocol Specific
Jonsson Comprehensive Cancer Center
 2002-05 PHASE1
NCT00005091 COMPLETED
Lung Cancer
Daiichi Pharmaceuticals
 1999-02 PHASE2

(data from https://clinicaltrials.gov, updated on 2026-06-02)

Check the Exatecan product page for in-depth specifications, including solubility, stock solutions, MOA, and working concentrations.

Compliance for Clinical Use

Mechanism and Biochemical Profile

Exatecan selectively binds to and inhibits DNA topoisomerase I with an IC50 value of 1.906 μM, stabilizing DNA-protein cleavage complexes and preventing DNA strand religation. The resulting accumulation of double-strand DNA damage triggers apoptotic cell death, which underlies its antitumor activity evaluated in clinical trials for advanced solid tumors, pancreatic cancer, and hematologic 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.