NCT01830972

An Open Label Phase 2 Extension Study of Higher Dose Sialic Acid-Extended Release (SA-ER) Tablets and Sialic Acid-Immediate Release (SA-IR) Capsules in Patients With Glucosamine (UDP-N-acetyl)-2-Epimerase (GNE) Myopathy

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 GNE Myopathy or Hereditary Inclusion Body Myopathy

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 59 (actual)
• Sponsor: Ultragenyx Pharmaceutical Inc · Industry
• Sex: All
• Age: 18 Years – 65 Years
• Healthy volunteers: Not accepted

Summary

The safety objectives of the study are to: evaluate additional long-term safety of SA-ER treatment of participants with GNE myopathy previously treated with SA-ER at dose of 6g/day (Part I); evaluate the safety of 12g /day SA (delivered by 1.5g of SA-ER tablets and 1.5g of SA-IR capsules 4 times per day) in the treatment of participants with GNE myopathy (Part II) over a 6 month treatment period; evaluate the safety of SA treatment at both 6g/day and 12 g/day (Part III \[SA-ER/SA-IR\] and Part IV \[SA-ER\]).

Conditions

• GNE Myopathy
• Hereditary Inclusion Body Myopathy (HIBM)

Interventions

Type	Name	Description
DRUG	SA-ER 500 mg	oral tablets
DRUG	SA-IR 500 mg	oral capsules

Timeline

• Start date: 2013-06-04
• Primary completion: 2017-02-14
• Completion: 2017-02-14
• First posted: 2013-04-12
• Last updated: 2018-04-11
• Results posted: 2018-03-13

Locations

4 sites across 2 countries: United States, Israel

Regulatory

• FDA-regulated drug study