A Phase 2 Open-label Study to Evaluate the Safety of Aceneuramic Acid Extended Release (Ace-ER) Tablets in GNE Myopathy (GNEM) (Also Known as Hereditary Inclusion Body Myopathy (HIBM)) Patients With Severe Ambulatory Impairment
Phase of Trial: Phase II
Latest Information Update: 13 Aug 2017
At a glance
- Drugs Aceneuramic acid (Primary)
- Indications Nonaka distal myopathy
- Focus Adverse reactions
- Sponsors Ultragenyx Pharmaceutical
- 10 Jun 2017 Biomarkers information updated
- 14 May 2017 Planned number of patients changed from 30 to 45.
- 12 Apr 2017 Planned primary completion date changed from 1 Aug 2017 to 1 Aug 2018.