Clinical Trials

Multiple completed clinical trials have evaluated Poly(I:C) derivatives and related immunomodulatory regimens across diverse conditions, ranging from a Phase II trial of in situ autologous therapeutic vaccination against solid malignancies—including melanoma, sarcoma, head and neck, and non-melanoma skin cancers—to an unphased study examining leukocyte responses in immunologic deficiency syndromes. These investigations were supported by major academic, governmental, and industry sponsors, including the National Cancer Institute, the National Institutes of Health, the Icahn School of Medicine at Mount Sinai, Radboud University Medical Center, and Oncovir Inc.

NCT Number Recruitment Conditions Sponsor/Collaborators Start Date Phases
NCT02423863 Completed
Melanoma|Head and Neck Cancer|Sarcoma|Non-Melanoma Skin Cancers
Oncovir Inc.|National Institutes of Health (NIH)|Icahn School of Medicine at Mount Sinai|Bay Hematology Oncology|National Cancer Institute (NCI)|University of Missouri-Columbia|Chevy Chase Regional Cancer Care Associates LLC|Dermatologic Surgery Center of Washington LLC and Skin Cancer Treatment Center
 2015-03 Phase 2
NCT01727895 Completed
Immunologic Deficiency Syndromes
Radboud University Medical Center
 2013-05 Not Applicable

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

Check the Poly(I:C) (Polyinosinic-Polycytidylic Acid) product page for in-depth specifications, including solubility, stock solutions, MOA, and working concentrations.

Compliance for Clinical Use

Mechanism and Biochemical Profile

Poly(I:C) acts as a synthetic double-stranded RNA analog that binds to pattern recognition receptors such as Toll-like receptor 3, triggering downstream signaling through NF-κB and interferon regulatory factors to induce robust type I interferon and pro-inflammatory cytokine production. This molecular cascade stimulates immune cell maturation and enhances cytotoxic T-cell responses, providing the mechanistic basis for its clinical application in boosting anti-tumor immunity in solid cancers—such as melanoma, sarcoma, and head and neck carcinoma—and restoring immune responsiveness in immunologic deficiency syndromes.

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.