Phase 3B Open-Label Extension Study to Evaluate the Safety and Efficacy of Aceneuramic Acid Extended-Release (Ace-ER) Tablets in Patients With GNE Myopathy (GNEM) or Hereditary Inclusion Body Myopathy (HIBM)
Phase of Trial: Phase III
Latest Information Update: 04 Aug 2017
At a glance
- Drugs Aceneuramic acid (Primary)
- Indications Nonaka distal myopathy
- Focus Adverse reactions
- Sponsors Ultragenyx Pharmaceutical
- 12 Apr 2017 Planned primary completion date changed from 1 Jan 2020 to 1 Apr 2020.
- 01 Nov 2016 Planned number of patients changed from 110 to 165.
- 15 Apr 2016 New trial record