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

GNE Myopathy Clinical Trial

Official title:
A Phase 2 Open-label Study to Evaluate the Safety of Aceneuramic Acid Extended Release (Ace-ER) Tablets in GNE Myopathy (GNEM) (Also Known as Hereditary Inclusion Body Myopathy (HIBM)) Patients With Severe Ambulatory Impairment

Status Terminated

Clinical Trial 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.

Clinical Trial Description

n/a

Study Design

Related Conditions & MeSH terms

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

Clinical Trial Details

NCT number	NCT02731690
Study type	Interventional
Source	Ultragenyx Pharmaceutical Inc
Contact
Status	Terminated
Phase	Phase 2
Start date	April 29, 2016
Completion date	January 10, 2018

View Details

See also
Status	Clinical Trial	Phase
Recruiting	`NCT01417533` - A Natural History Study of Patients With GNE Myopathy and GNE-Related Diseases
Completed	`NCT01634750` - Phase I Clinical Trial of ManNAc in Patients With GNE Myopathy or Hereditary Inclusion Body Myopathy (HIBM)	Phase 1
Completed	`NCT01517880` - A Phase 2 Study to Evaluate the Dose and Pharmacodynamic Efficacy of Sialic Acid-Extended Release (SA-ER) Tablets in Patients With GNE Myopathy or Hereditary Inclusion Body Myopathy	Phase 2
Completed	`NCT02377921` - 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)	Phase 3
Terminated	`NCT02736188` - 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)	Phase 3
Recruiting	`NCT04009226` - International GNE Myopathy Patient Registry
Completed	`NCT01830972` - An Open Label Phase 2 Extension Study of Higher Dose Sialic Acid-Extended Release (SA-ER) Tablets and Sialic Acid-Immediate Release (SA-IR) Capsules in Patients With Glucosamine (UDP-N-acetyl)-2-Epimerase (GNE) Myopathy	Phase 2
Completed	`NCT04671472` - Efficacy Confirmation Study of NPC-09	Phase 3
Completed	`NCT02346461` - An Open Label Phase 2 Study of ManNAc in Subjects With GNE Myopathy	Phase 2
Active, not recruiting	`NCT04231266` - Multi-Center Study of ManNAc for GNE Myopathy	Phase 2
Completed	`NCT01784679` - GNE-Myopathy Disease Monitoring Program (GNEM-DMP): A Registry and Prospective Observational Natural History Study to Assess GNE Myopathy or Hereditary Inclusion Body Myopathy (HIBM)