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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 |
| Contact |
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.
Recruitment
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.