clinical trial · NCT07511556
First-in-human Study of UX016 in GNEM
GNE MyopathyUX016Placebo
brief summary
The main goal of this study is to evaluate the safety of UX016 and to evaluate the impact of UX016 on muscle strength in adults with GNE Myopathy (GNEM).
started
Oct 1, 2026
primary completion
Dec 1, 2028
completion
Dec 1, 2028
last updated
Apr 6, 2026
official title
A Phase 1/2, First-in-human, Double-blind, Placebo-controlled Study to Assess Dose, Safety, and Efficacy of UX016 (Sialic Acid-C16 Prodrug) in Adults With GNE Myopathy
sourced from ClinicalTrials.gov · pharmadog mirrors structured fields, not the full protocol