Overview

Efficacy Confirmation Study of NPC-09

Status:
Active, not recruiting
Trial end date:
2022-04-30
Target enrollment:
Participant gender:
Summary
GNE myopathy is a distal myopathy that is thought to be caused by a mutation in the GNE gene that encodes an enzyme in the biosynthetic process of aceneuramic acid (typical sialic acid). The investigators will examine the efficacy and safety of aceneuramic acid (SA-ER tablets) 6g daily for 48 weeks in patients with GNE myopathy in a placebo-controlled, double-blind, controlled trial.
Phase:
Phase 3
Details
Lead Sponsor:
Nobelpharma