Treatment Trials

4 Clinical Trials for Various Conditions

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      TERMINATED
      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)
      Description

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

      Conditions
      Hereditary Inclusion Body MyopathyDistal Myopathy With Rimmed VacuolesDistal Myopathy, Nonaka TypeGNE MyopathyQuadriceps Sparing Myopathy
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      TERMINATED
      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
      Description

      The primary objective of this Phase 2 study is to evaluate the safety of open-label 6 g/day Ace-ER in GNEM participants with severe ambulatory impairment.

      Conditions
      Hereditary Inclusion Body MyopathyDistal Myopathy With Rimmed VacuolesDistal Myopathy, Nonaka TypeGNE MyopathyQuadriceps Sparing MyopathyInclusion Body Myopathy 2
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      COMPLETED
      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)
      Description

      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.

      Conditions
      Hereditary Inclusion Body MyopathyDistal Myopathy With Rimmed VacuolesDistal Myopathy, Nonaka TypeGNE Myopathy
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      ACTIVE_NOT_RECRUITING
      Multi-Center Study of ManNAc for GNE Myopathy
      Description

      GNE myopathy is a rare genetic muscle disease characterized by progressive muscle atrophy and weakness. The disease is caused by mutations in the gene that encodes the enzyme that initiates and regulates N-acetylneuraminic acid (Neu5Ac) biosynthesis and glycan sialylation. Currently, there is no therapy available for this disease. N-Acetylmannosamine (ManNAc), an orphan drug in development for GNE myopathy, is an uncharged monosaccharide and the first committed precursor in Neu5Ac biosynthesis. In this randomized, double-blind, placebo-controlled trial the efficacy and long-term safety of ManNAc will be evaluated in subjects with GNE myopathy.

      Conditions
      GNE Myopathy
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