Study to Evaluate the Safety & Efficacy of Aceneuramic Acid

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)

GNE Myopathy Clinical Trial

Official title:
Phase 3B Open-Label Extension Study to Evaluate the Safety and Efficacy of Aceneuramic Acid Extended-Release (Ace-ER) Tablets in Patients With GNE Myopathy (GNEM) or Hereditary Inclusion Body Myopathy (HIBM)

Status Terminated

Clinical Trial Summary

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

Clinical Trial Description

This Phase 3b extension study will assess the long-term safety of Ace-ER in patients who participated in and completed study UX001-CL301 (NCT02377921), study UX001-CL202 (NCT01830972), and study UX001-CL203 (NCT02731690). ;

Study Design

Related Conditions & MeSH terms

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

Clinical Trial Details

NCT number	NCT02736188
Study type	Interventional
Source	Ultragenyx Pharmaceutical Inc
Contact
Status	Terminated
Phase	Phase 3
Start date	May 2, 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
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