Study design & results
Available in the Gosset platform where reported.
Trial intelligence / Profile preview
Phase 2 Open-label Study of Aceneuramic Acid Extended-Release in GNE Myopathy
2015-004553-41Phase 2Terminated
- Sponsor
- Ultragenyx Pharmaceutical Inc.
- Start
- Sep 2016
- Modality
- Small Molecules
- Countries
- 1
01
Overview
Phase 2, open-label, single-arm trial of aceneuramic acid in GNE myopathy (HIBM), sponsored by Ultragenyx Pharmaceutical Inc. Primary endpoint: Incidence and frequency of adverse events related to Ace-ER. Started Sep 2016; status: terminated.
02
Drugs
aceneuramic acid
03
Conditions
GNE myopathy (HIBM)
Beyond the preview
Go deeper on 2015-004553-41.
Study design, results and competitive context are available in the Gosset platform where reported.
Competitive context
Available in the Gosset platform where reported.
Bring the full picture into focus.
See how Gosset can support your research on 2015-004553-41.