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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)

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)

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

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