MedTrace Logo

Efficacy Confirmation Study of NPC-09

NCT04671472 · Status: COMPLETED · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 14

Last updated 2022-05-16

No results posted yet for this study

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.

Conditions

  • GNE Myopathy
  • Distal Myopathy With Rimmed Vacuoles (DMRV)
  • Hereditary Inclusion Body Myopathy (hIBM)
  • Nonaka Disease

Interventions

DRUG

NPC-09

The drug will be administered by the oral route with the same manner

DRUG

NPC-09 placebo

The drug will be administered by the oral route with the same manner

Sponsors & Collaborators

  • Nobelpharma

    lead INDUSTRY

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
PARALLEL

Eligibility

Min Age
20 Years
Max Age
50 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2021-02-08
Primary Completion
2022-03-29
Completion
2022-03-29

Countries

  • Japan

Study Locations

More Related Trials

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04671472 on ClinicalTrials.gov