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NPC-09 efficacy confirmation study in GNE myopathy
jRCT2021200030Active, not recruiting
- Sponsor
- Nobelpharma Co., Ltd.
- Start
- Feb 2021
- Modality
- Small Molecules
- Countries
- 1
01
Overview
Randomised, double-blind trial in GNE myopathy (HIBM) comparing NPC-09 arm with Placebo arm (2 arms). Primary endpoint: Change in upper extremity composite score from baseline to Week 48. Started Feb 2021; status: active, not recruiting.
02
Drugs
aceneuramic acid
03
Conditions
GNE myopathy (HIBM)
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