Clinical Trials

Multiple clinical trials have evaluated Combretastatin A4 across Phase I, Phase II, and Phase III for oncology and ophthalmology indications, sponsored by academic institutions such as Johns Hopkins University alongside industry entities like Mateon Therapeutics. Investigated conditions include solid tumors, head and neck cancer, anaplastic thyroid cancer, age-related macular degeneration, choroidal neovascularization, and degenerative myopia. While several trials assessing safety, tolerability, and combination regimens were successfully completed, certain Phase II and Phase III trials in thyroid cancer were ultimately terminated.

NCT Number Recruitment Conditions Sponsor/Collaborators Start Date Phases
NCT00507429 TERMINATED
Anaplastic Thyroid Cancer
Mateon Therapeutics
2007-08 PHASE2; PHASE3
NCT00653939 COMPLETED
Tumors
Mateon Therapeutics
2008-03 PHASE2
NCT00113438 COMPLETED
Cancer; Tumor
Mateon Therapeutics
2005-03 PHASE2
NCT00060242 COMPLETED
Head and Neck Cancer
Case Comprehensive Cancer Center
2003-02 PHASE2
NCT00077103 TERMINATED
Head and Neck Cancer
Case Comprehensive Cancer Center
2003-11 PHASE1; PHASE2
NCT00395434 COMPLETED
Tumors
Mateon Therapeutics
2006-09 PHASE1
NCT01423149 COMPLETED
Choroidal Neovascularization; Myopia, Degenerative
Mateon Therapeutics
2005-03 PHASE2
NCT01570790 COMPLETED
Combretastatin A4 Phosphate; Age-related Macular Degeneration; AMD; CNV; Choroidal Neovascularization
Johns Hopkins University
2003-05 PHASE1; PHASE2
NCT00003698 COMPLETED
Unspecified Adult Solid Tumor, Protocol Specific
University of Glasgow
1998-07 PHASE1

(data from https://clinicaltrials.gov, updated on 2014-06-09)

Check the Combretastatin A4 product page for in-depth specifications, including solubility, stock solutions, MOA, and working concentrations.

Compliance for Clinical Use

Mechanism and Biochemical Profile

Combretastatin A4 selectively binds to β-tubulin with a Kd of 0.4 μM, inhibiting microtubule polymerization and destabilizing the dynamic endothelial cytoskeleton. This acute cytoskeletal disruption triggers vascular shutdown and tumor necrosis, underlying its therapeutic investigation in solid tumors, anaplastic thyroid cancer, and choroidal neovascularization.

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>).

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