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: 14 Aug 2017
At a glance
- Drugs Aceneuramic acid (Primary)
- Indications Nonaka distal myopathy
- Focus Registrational; Therapeutic Use
- Acronyms GNEM
- Sponsors Ultragenyx Pharmaceutical
- 09 Aug 2017 Status changed from active, no longer recruiting to completed.
- 16 Feb 2017 According to an Ultragenyx Pharmaceutical media release, the company plans to submit an NDA based on the results of this trial, if positive.
- 11 Nov 2016 According to an Ultragenyx Pharmaceutical media release, the company plans to submit an MAA for full approval after data from this study are available in the second half of 2017.