1-Deoxynojirimycin Clinical Trials

Size	Price	Qty
5mg	USD 147
25mg	USD 370

Clinical Trials

A clinical trial evaluated 1-deoxynojirimycin (duvoglustat or AT2220) as an investigational chaperone therapy for Pompe disease. Sponsored by Amicus Therapeutics, this completed phase 2, open-label, multi-center study assessed potential drug-drug interactions between duvoglustat and recombinant human acid alpha-glucosidase (alglucosidase alfa) in affected individuals.

NCT Number	Recruitment	Conditions	Sponsor/Collaborators	Start Date	Phases
NCT01380743	Completed	Pompe Disease	Amicus Therapeutics	2011-10-31	Phase 2

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

Check the 1-Deoxynojirimycin product page for in-depth specifications, including solubility, stock solutions, MOA, and working concentrations.

Compliance for Clinical Use

Mechanism and Biochemical Profile

1-Deoxynojirimycin functions as a potent iminosugar inhibitor of alpha-glucosidase enzymes, binding competitively to the active catalytic site to suppress the enzymatic hydrolysis of complex carbohydrates into free glucose. By modulating carbohydrate processing and stabilizing lysosomal alpha-glucosidase, this target-specific enzymatic inhibition provides therapeutic potential for managing metabolic disorders such as diabetes mellitus and lysosomal storage conditions like Pompe disease.

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>).