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Phase 3 Randomized, Double-Blind, Placebo-Controlled Study to Evaluate Sialic Acid in Patients With Glucosamine (UDP-N-acetyl)-2-epimerase Myopathy (GNEM) or Hereditary Inclusion Body Myopathy (HIBM)

NCT02377921 · Status: COMPLETED · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 89

Last updated 2019-06-27

Study results available
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Summary

The primary objective of this study is to evaluate the effect of 6 g/day aceneuramic acid extended-release (Ace-ER) treatment of participants with GNEM on upper extremity muscle strength (upper extremity composite \[UEC\] score) as measured by dynamometry.

Conditions

  • Hereditary Inclusion Body Myopathy
  • Distal Myopathy With Rimmed Vacuoles
  • Distal Myopathy, Nonaka Type
  • GNE Myopathy

Interventions

DRUG

aceneuramic acid extended-release (Ace-ER)

tablets for oral use

DRUG

Placebo

tablets for oral use

Sponsors & Collaborators

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
PARALLEL

Eligibility

Min Age
18 Years
Max Age
55 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2015-05-20
Primary Completion
2017-06-09
Completion
2017-06-09

Countries

  • United States
  • Bulgaria
  • Canada
  • France
  • Israel
  • Italy
  • United Kingdom

Study Locations

More Related Trials

Entities

Drugs
Companies

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 NCT02377921 on ClinicalTrials.gov