Safety and Pharmacokinetics of Sialic Acid Tables in Patients With Hereditary Inclusion Body Myopathy (HIBM)
Condition: Hereditary Inclusion Body Myopathy (HIBM)
MeSH terms: Muscular Diseases; Distal Myopathies
Study type: Interventional
Phase: Phase 1

Summary: 
      Hereditary Inclusion Body Myopathy (HIBM) is a severe progressive metabolic myopathy caused
      by a defect in the biosynthetic pathway for sialic acid (SA), a critical component of many
      muscle proteins, resulting in a deficiency in SA in the muscles of HIBM patients.

      The effective replacement of the missing SA substrate is theoretically simple, and, in animal
      models, replacement with SA showed significant restoration of sialylation biochemistry and
      excellent reduction in muscle disease. These data show that replacement can achieve
      significant clinical benefit in muscle pathology, function, and survival.
    


Intervention name(s): Sialic Acid Extended Release (SA-ER) Tablets; Sialic Acid Extended Release (SA-ER) Tables; Sialic Acid Extended Release (SA-ER) Tablets; Sialic Acid Extended Release (SA-ER) Tablets; Sialic Acid Extended Release (SA-ER) Tablets
Intervention type(s): Drug; Drug; Drug; Drug; Drug
Intervention description(s): Patients will receive SA-ER tablets orally at one of five (5) dose levels in the single-dose phase,phase and one of four (4) dose levels in the repeat-dose phase. During repeat dosing, the total daily dose will be divided evenly into three doses given in the morning, in the evening, and at bedtime (qHS). No placebo or active comparator will be administered and the study drug will be administered on an open-label basis.; PPatients will receive SA-ER tablets orally at one of five (5) dose levels in the single-dose phase,phase and one of four (4) dose levels in the repeat-dose phase. During repeat dosing, the total daily dose will be divided evenly into three doses given in the morning, in the evening, and at bedtime (qHS). No placebo or active comparator will be administered and the study drug will be administered on an open-label basis.; Patients will receive SA-ER tablets orally at one of five (5) dose levels in the single-dose phase,phase and one of four (4) dose levels in the repeat-dose phase. During repeat dosing, the total daily dose will be divided evenly into three doses given in the morning, in the evening, and at bedtime (qHS). No placebo or active comparator will be administered and the study drug will be administered on an open-label basis.; Patients will receive SA-ER tablets orally at one of five (5) dose levels in the single-dose phase,phase and one of four (4) dose levels in the repeat-dose phase. During repeat dosing, the total daily dose will be divided evenly into three doses given in the morning, in the evening, and at bedtime (qHS). No placebo or active comparator will be administered and the study drug will be administered on an open-label basis.; Patients will receive SA-ER tablets orally at one of five (5) dose levels in the single-dose phase,phase and one of four (4) dose levels in the repeat-dose phase. During repeat dosing, the total daily dose will be divided evenly into three doses given in the morning, in the evening, and at bedtime (qHS). No placebo or active comparator will be administered and the study drug will be administered on an open-label basis.
Allocation: Non-Randomized
Intervention model: Single Group Assignment
Masking: None (Open Label)
Primary purpose: Treatment
Keywords: Phase 1 Safety and Pharmacokinetic Study