Clinical Trials

Multiple clinical trials have evaluated N-acetylneuraminic acid for sialic acid deficiency states, metabolic conditions, and neuromuscular disorders—such as N-acetylneuraminic acid storage disease, GNE myopathy, and Nonaka myopathy—alongside health behavior studies. Featuring completed recruitment, these Phase 1, Phase 2, and unassigned-phase designs focused on compound pharmacokinetics and sialic acid supplementation. Prominent sponsors include academic and government institutions, such as the National Institutes of Health, Tohoku University, and the University of Lausanne.

NCT Number Recruitment Conditions Sponsor/Collaborators Start Date Phases
NCT03545568 Completed
N-Acetylneuraminic Acid Storage Disease
University of Lausanne|University of Modena and Reggio Emilia
 2018-05-01 Not Applicable
NCT02346461 Completed
GNE Myopathy
National Human Genome Research Institute (NHGRI)|National Center for Advancing Translational Sciences (NCATS)|National Institutes of Health Clinical Center (CC)
 2015-02-05 Phase 2
NCT01236898 COMPLETED
Nonaka Myopathy; Hereditary Inclusion Body Myopathy
Tohoku University
 2010-11 PHASE1
NCT01236898 Completed
Nonaka Myopathy|Hereditary Inclusion Body Myopathy
Tohoku University
 2010-11 Phase 1

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

Check the N-Acetylneuraminic acid product page for in-depth specifications, including solubility, stock solutions, MOA, and working concentrations.

Compliance for Clinical Use

Mechanism and Biochemical Profile

As a predominant mammalian sialic acid, N-acetylneuraminic acid undergoes enzymatic incorporation into cell-surface glycoconjugates by sialyltransferases, restoring terminal sialylation on membrane glycoproteins and glycolipids to maintain structural integrity and signaling. This restoration of cellular sialylation prevents cellular degeneration and improves muscle cell function, which directly addresses the underlying pathophysiological deficit in conditions such as GNE myopathy and N-acetylneuraminic acid storage 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>).

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Note: Technical data last updated: Sep 1, 2026.