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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


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

See also
  Status Clinical Trial Phase
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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