Clinical Trials

Multiple clinical trials have completed recruitment to evaluate topical formulations of ingenol and its derivatives exclusively for the dermatological condition actinic keratosis across Phase 1, Phase 1/2, and Phase 2 stages. Sponsored by academic and industry entities, including LEO Pharma, Peplin, and the Icahn School of Medicine at Mount Sinai, these early-phase studies assess safety, tolerability, pharmacokinetic profiles, and local tissue reactions.

NCT Number Recruitment Conditions Sponsor/Collaborators Start Date Phases
NCT01836367 Completed
Actinic Keratosis
Icahn School of Medicine at Mount Sinai|LEO Pharma
 2013-03 Phase 1
NCT01803477 Completed
Actinic Keratosis
LEO Pharma
 2013-02 Phase 1|Phase 2
NCT01703078 Completed
Actinic Keratosis
LEO Pharma
 2012-11 Phase 1
NCT00852137 Completed
Actinic Keratosis
Peplin
 2009-03 Phase 2

(data from https://clinicaltrials.gov, updated on 2024-05-22)

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

Compliance for Clinical Use

Mechanism and Biochemical Profile

Ingenol functions as a plant-derived diterpenoid that binds to and activates protein kinase C isoforms, which triggers downstream cellular necrosis and stimulates localized immunocompetent responses. This dual action of rapid cell disruption and inflammatory activation targets proliferating dysplastic epidermal cells, resulting in the clinical clearance of actinic keratosis lesions.

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.