Clinical Trials

Multiple completed Phase II clinical trials have evaluated compounds with this profile for oncological and renal indications, specifically targeting diabetic nephropathy and high-grade gliomas, including glioblastoma multiforme, astrocytomas, giant cell glioblastoma, and oligodendrogliomas. Sponsorship for these studies encompasses commercial entities such as Novartis Pharmaceuticals alongside major government research institutions, including the National Cancer Institute and the National Institutes of Health Clinical Center.

NCT Number Recruitment Conditions Sponsor/Collaborators Start Date Phases
NCT03027388 Completed
Astrocytoma Grades II III and IV|Glioblastoma Multiforme|Giant Cell Glioblastoma|Glioma|Oligodendrogliomas
National Cancer Institute (NCI)|National Institutes of Health Clinical Center (CC)
 2019-01-09 Phase 2
NCT03804879 Completed
Diabetic Nephropathy
Novartis Pharmaceuticals|Novartis
 2018-12-17 Phase 2

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

Check the Leptomycin B (LMB) product page for in-depth specifications, including solubility, stock solutions, MOA, and working concentrations.

Compliance for Clinical Use

Mechanism and Biochemical Profile

Leptomycin B covalently binds to and inhibits chromosome region maintenance 1 (CRM1), thereby blocking the nuclear export of essential signaling proteins and regulatory complexes. This transport blockade leads to the nuclear entrapment of nuclear export cargo and rapid induction of cellular cytotoxicity, demonstrating therapeutic relevance in suppressing aggressive neoplasms such as glioblastoma multiforme and astrocytomas.

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.