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Study to Evaluate the Safety and Efficacy of Aceneuramic Acid Extended-Release (Ace-ER) Tablets in Patients With Glucosamine (UDP-N-acetyl)-2-epimerase Myopathy (GNEM) or Hereditary Inclusion Body Myopathy (HIBM)

NCT02736188 · Status: TERMINATED · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 143

Last updated 2023-03-24

Study results available
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Summary

The objective of this study is to evaluate the long-term safety and efficacy of Ace-ER treatment in subjects with GNEM.

Conditions

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

Interventions

DRUG

Aceneuramic Acid Extended-Release Tablets

Sponsors & Collaborators

Principal Investigators

  • Medical Director · Ultragenyx Pharmaceutical Inc

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2016-05-02
Primary Completion
2018-01-10
Completion
2018-01-10

Countries

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

Study Locations

More Related Trials

Entities

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 NCT02736188 on ClinicalTrials.gov