Clinical Trials

Sponsored by Karyopharm Therapeutics Inc, multiple clinical trials evaluated oral Verdinexor in healthy volunteers within Phase 1 investigations. These randomized, double-blind, placebo-controlled, dose-escalation studies analyzed the safety profile, tolerability, and pharmacokinetic properties of the agent. According to public records, recruitment for these trials has been terminated.

NCT Number Recruitment Conditions Sponsor/Collaborators Start Date Phases
NCT02431364 TERMINATED
Healthy
Karyopharm Therapeutics Inc
 2015-05-26 PHASE1
NCT02431364 Terminated
Healthy
Karyopharm Therapeutics Inc
 2015-05-26 Phase 1

(data from https://clinicaltrials.gov, updated on 2023-01-20)

Check the Verdinexor (KPT-335) product page for in-depth specifications, including solubility, stock solutions, MOA, and working concentrations.

Compliance for Clinical Use

Mechanism and Biochemical Profile

Verdinexor functions as a selective inhibitor of exportin-1 (XPO1/CRM1), binding to XPO1 to block the nuclear export of tumor suppressor proteins and regulatory factors, thereby forcing their retention in the nucleus and promoting cellular apoptotic pathways or antiviral cascades. This inhibition of nuclear export mechanisms provides the pharmacological rationale for evaluating the compound's safety, tolerability, and pharmacokinetic profile in healthy volunteers during early-phase clinical development.

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.