Status | Study |
Enrolling by invitation |
Study Name: Study to Evaluate the Safety and Efficacy of Ace-ER Tablets in Patients With GNE Myopathy or Hereditary Inclusion Body Myopathy Condition: Hereditary Inclusion Body Myopathy Distal Myopathy With Rimmed Date: 2016-04-04 Interventions: Drug: Aceneuramic Acid Extended-Release Tablets Other Names: UX001 |
Recruiting |
Study Name: A 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 Condition: Hereditary Inclusion Body Myopathy Distal Myopathy With Rimmed Date: 2016-03-24 Interventions: Drug: Aceneuramic Acid Extended-Release Tablets Other Names: Ace-ER |
Active, not recruiting |
Study Name: Phase 3 Randomized, Double-Blind, Placebo-Controlled Study to Evaluate Sialic Acid in Patients With GNE Myopathy (GNEM) or Hereditary Inclusion Body Myopathy (HIBM) Condition: Hereditary Inclusion Body Myopathy Distal Myopathy With Rimmed Date: 2015-02-27 Interventions: Drug: Sialic Acid Tablets Othe |
Recruiting |
Study Name: 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) Condition: Hereditary Inclusion Body Myopathy GNE Myopathy Nonaka Dis Date: 2013-02-04 |
Completed |
Study Name: Phase I Clinical Trial of ManNAc in Patients With GNE Myopathy or Hereditary Inclusion Body Myopathy (HIBM) Condition: Hereditary Inclusion Body Myopathy (HIBM) GNE Myopathy Date: 2012-07-03 Interventions: Drug: ManNAc Single dose |
Completed |
Study Name: 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 Condition: GNE Myopathy Hereditary Inclusion Body Myopathy Date: 2012-01-17 Interventions: Drug: Sialic Acid Extended Release (SA-ER) |
Recruiting |
Study Name: Genetic Study of Familial and Sporadic ALS/Motor Neuron Disease, Miyoshi Myopathy and Other Neuromuscular Disorders Condition: Amyotrophic Lateral Sclerosis Frontotemporal Dementia PLS Date: 2011-10-21 |
Recruiting |
Study Name: A Natural History Study of Patients With GNE Myopathy Condition: Hereditary Inclusion Body Myopathy Date: 2011-08-13 |
Completed |
Study Name: Safety and Pharmacokinetics of Sialic Acid Tables in Patients With Hereditary Inclusion Body Myopathy (HIBM) Condition: Hereditary Inclusion Body Myopathy (HIBM) Date: 2011-05-20 Interventions: Drug: Sialic Acid Extended Release (SA-ER) Tablets |
Completed |
Study Name: Pharmacokinetic Study on N-acetylneuraminic Acid Condition: Nonaka Myopathy Hereditary Inclusion Body Myopathy Date: 2010-11-08 Interventions: Drug: NPC-09 NPC-09 tablet NPC-09 contains 50mg or 100mg N-acetylneuraminic acid (anhydride) |