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 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 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 Date: 2015-02-27 Interventions: Drug: Sialic Acid Tablets Othe |
Active, not recruiting |
Study Name: An Open Label Phase 2 Study of ManNAc in Subjects With GNE Myopathy Condition: GNE Myopathy Date: 2015-01-24 Interventions: Drug: ManNac. At doses of 3,00 |
Active, not recruiting |
Study Name: Clinical, Biological and NMR Outcome Measures Study for Hereditary Inclusion Body Myopathy Due to Mutation of UDP-N-acetylglucosamine 2-epimerase/N-acetylmannosamine Kinase Gene (GNE) Condition: HIBM Date: 2014-04-08 Interventions: Other: motor function and strength assessment |
Completed |
Study Name: Natural History in CCFDN and IBM Syndromes Condition: Inclusion Body Myositis, Sporadic Inclusion Body Myopathy, Autosomal-r Date: 2013-07-03 Interventions: Other: Natural History Assessment of natural history in IBM and CCFDN |
Active, not recruiting |
Study Name: An Open Label Phase 2 Extension Study of Higher Dose Sialic Acid (ER Tablets + IR Capsules) in Patients With GNE Myopathy Condition: GNE Myopathy HIBM Date: 2013-04-10 Interventions: Drug: SA-ER tablets and SA-IR capsules |
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 My 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) 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 My Date: 2012-01-17 Interventions: Drug: Sialic Acid Extended Release (SA-ER) |