Clinical Trial: An Open Label Phase 2 Extension Study of Higher Dose Sialic Acid (ER Tablets + IR Capsules) in Patients With GNE Myopathy

Study Status: Active, not recruiting
Recruit Status: Active, not recruiting
Study Type: Interventional




Official Title: An Open-label Phase 2 Extension Study to Evaluate the Long Term Safety and Efficacy of Sialic Acid-Extended Release (SA-ER) Tablets and Sialic Acid-Immediate Release (SA-IR) Capsules in Patients With

Brief Summary: GNE myopathy or hereditary inclusion body myopathy (HIBM) is a severe progressive metabolic myopathy caused by a defect in the biosynthetic pathway for sialic acid (SA). The purpose of the study is to measure long term safety and the effects of Sialic Acid-Extended Release (SA-ER) tablets and Sialic Acid-Immediate Release (SA-IR) capsules.