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ManNAc in GNE Myopathy
NCT02346461Phase 2Completed
- Sponsor
- National Human Genome Research Institute
- Start
- Feb 2015
- Primary completion
- Dec 2017
- Modality
- Small Molecules
- Countries
- 1
01
Overview
Phase 2, open-label trial of n-acetylmannosamine in GNE myopathy (HIBM) (2 cohorts), sponsored by National Human Genome Research Institute. Primary endpoints: Mean Area Under the Curve (AUClast) of Plasma ManNAc at Day 7, Maximum Observed Plasma Concentration (Cmax) of ManNAc at Day 7 and 5 more. Started Feb 2015; status: completed.
02
Drugs
n-acetylmannosamine
03
Conditions
GNE myopathy (HIBM)
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