Phase 3 Randomized, Double-Blind, Placebo-Controlled Study to Evaluate

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) — GNEM

GNE Myopathy Clinical Trial

Official title:
A Phase 3 Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Sialic Acid Extended-Release Tablets in Patients With GNE Myopathy (GNEM) or Hereditary Inclusion Body Myopathy (HIBM)

Status Completed

Clinical Trial 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.

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
Muscular Diseases

Clinical Trial Details

NCT number	NCT02377921
Study type	Interventional
Source	Ultragenyx Pharmaceutical Inc
Contact
Status	Completed
Phase	Phase 3
Start date	May 20, 2015
Completion date	June 9, 2017

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
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)
Terminated	`NCT02731690` - 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	Phase 2