Clinical Trials

Several completed clinical trials, spanning Phase 1, Phase 2, and non-phase proof-of-concept evaluations, have investigated N-Acetyl-D-mannosamine for rare metabolic and neuromuscular disorders, specifically N-Acetylneuraminic Acid Storage Disease, GNE Myopathy, and Hereditary Inclusion Body Myopathy. This early-stage research landscape is predominantly supported by governmental and academic institutions, including institutes within the National Institutes of Health, the University of Lausanne, and the University of Modena and Reggio Emilia.

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
NCT01634750 Completed
Hereditary Inclusion Body Myopathy (HIBM)|GNE Myopathy
National Human Genome Research Institute (NHGRI)|National Center for Advancing Translational Sciences (NCATS)|Therapeutics for Rare and Neglected Diseases (TRND)|National Institutes of Health Clinical Center (CC)
 2012-09-11 Phase 1

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

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

Compliance for Clinical Use

Mechanism and Biochemical Profile

N-Acetyl-D-mannosamine functions as an essential biochemical precursor in the sialic acid biosynthetic pathway, bypassing rate-limiting enzymatic steps to restore downstream N-acetylneuraminic acid synthesis and promote cellular protein sialylation. This restoration of normal glycosylation patterns improves synaptic transmission and mitigates disease pathology in clinical 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.