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A Study to Evaluate the Safety of Aceneuramic Acid Extended Release (Ace-ER; UX001) Tablets in Glucosamine (UDP-N-acetyl)-2-Epimerase (GNE) Myopathy (GNEM) (Also Known as Hereditary Inclusion Body Myopathy [HIBM]) Patients With Severe Ambulatory Impairment

NCT02731690 · Status: TERMINATED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 42

Last updated 2019-02-19

Study results available
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Summary

The primary objective of this Phase 2 study is to evaluate the safety of open-label 6 g/day Ace-ER in GNEM participants with severe ambulatory impairment.

Conditions

  • Hereditary Inclusion Body Myopathy
  • Distal Myopathy With Rimmed Vacuoles
  • Distal Myopathy, Nonaka Type
  • GNE Myopathy
  • Quadriceps Sparing Myopathy
  • Inclusion Body Myopathy 2

Interventions

DRUG

Aceneuramic Acid Extended-Release

oral 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
2016-04-29
Primary Completion
2018-01-10
Completion
2018-01-10

Countries

  • United States
  • Bulgaria
  • Canada

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 NCT02731690 on ClinicalTrials.gov