A Phase 3 Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Sialic Acid Extended-Release Tablets in Patients With GNE Myopathy (GNEM) or Hereditary Inclusion Body Myopathy (HIBM)
Phase of Trial: Phase III
Latest Information Update: 21 Sep 2017
At a glance
- Drugs Aceneuramic acid (Primary)
- Indications Nonaka distal myopathy
- Focus Registrational; Therapeutic Use
- Acronyms GNEM
- Sponsors Ultragenyx Pharmaceutical
- 22 Aug 2017 According to an Ultragenyx Pharmaceutical media release, based on results of this trial Ultragenyx plans to discontinue further clinical development of Ace-ER.
- 22 Aug 2017 Results published in an Ultragenyx Pharmaceutical media release.
- 22 Aug 2017 Primary endpoint (Upper Extremity Muscle Strength) has not been met.
Most Recent Events
Table of Contents
- At a glance
- Trial Overview
- Trial Details
- Trial Centres
- Trial History