Queen Square Centre for Neuromuscular Diseases



A Phase III Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Sialic Acid Extended Release (SA-ER) Tablets in Patients with GNE Myopathy (GNEM) or Hereditary Inclusion Body Myopathy (HIBM)

Sponsor / Funder Ultragenyx
PI Professor Hanns Lochmüller

Newcastle: becky.davis@ncl.ac.uk

London: c.uzowuru@ucl.ac.uk

More information on the trial

This is a randomised, double-blind, placebo-controlled, multi-centre study in subjects with GNEM.

Primary objective

To assess the clinical effect of 6 g/day SA-ER tablets as compared with placebo.


About 80 subjects randomized in a 1:1 ratio to receive 6 g/day of SA-ER tablets or matching placebo for 48 weeks.

Randomisation of subjects will be stratified by gender with a planned enrolment of no more than 60% of subjects of either gender.