Clinical Trials

A clinical trial sponsored by EMD Serono Research & Development Institute, Inc., has completed recruitment for the evaluation of VX-984 in patients with advanced solid tumors. This open-label, first-in-human Phase 1 study assessed the safety, tolerability, pharmacokinetics, and pharmacodynamics of the compound administered in therapeutic combinations. Consequently, clinical evidence reflects an early-phase effort dedicated to establishing safety profiles and therapeutic regimens for VX-984 in solid tumor oncology.

NCT Number Recruitment Conditions Sponsor/Collaborators Start Date Phases
NCT02644278 COMPLETED
Advanced Solid Tumor
EMD Serono Research & Development Institute, Inc.
 2016-02-29 PHASE1

(data from https://clinicaltrials.gov, updated on 2019-09-09)

Check the VX-984 product page for in-depth specifications, including solubility, stock solutions, MOA, and working concentrations.

Compliance for Clinical Use

Mechanism and Biochemical Profile

VX-984 functions as a potent, ATP-competitive inhibitor of DNA-dependent protein kinase (DNA-PK) that directly reduces DNA-PKcs autophosphorylation and suppresses non-homologous end joining (NHEJ) repair pathways. By inhibiting double-strand break repair and causing an accumulation of lethal DNA damage, the compound potentiates cellular cytotoxicity, providing a mechanistic basis for targeting advanced solid tumors.

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.