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|
|More information on the trial|
This is a randomised, double-blind, placebo-controlled, multi-centre study in subjects with GNEM.
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.