Clinical Trial: Phase 3 Randomized, Double-Blind, Placebo-Controlled Study to Evaluate Sialic Acid in Patients With GNE Myopathy (GNEM) or Hereditary Inclusion Body Myopathy (HIBM)

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

Official Title: A Phase 3 Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Sialic Acid Extended-Release Tablets in Patients With GNE Myopathy (GNEM) or Hereditary Inclusion Bo

Brief Summary: UX001-CL301 is a randomized, double-blind, placebo-controlled study to assess the safety and efficacy of 6 g/day sialic acid - extended release (SA-ER) treatment as compared with placebo in subjects with GNE Myopathy.

Detailed Summary:
Sponsor: Ultragenyx Pharmaceutical Inc

Current Primary Outcome: Upper Extremity Muscle Strength [ Time Frame: Change from Baseline at Weeks 8, 16, 24, 32, 40, and 48 ]

Upper extremity muscle strength will be measured by dynamometry


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • GNEM-FAS Mobility Domain Score [ Time Frame: Change from Baseline at Weeks 8, 16, 24, 32, 40, and 48 ]
  • GNEM-FAS Upper Extremity Domain Score [ Time Frame: Change from Baseline at Weeks 8, 16, 24, 32, 40, and 48 ]
  • Lower Extremity Muscle Strength [ Time Frame: Change from Baseline at Weeks 8, 16, 24, 32, 40, and 48 ]
    Lower extremity muscle strength will be measured by dynamometry
  • Timed Sit-to-Stand Test [ Time Frame: Change from Baseline at Weeks 8, 16, 24, 32, 40, and 48 ]
  • Timed Weighted Arm Lift Test [ Time Frame: Change from Baseline at Weeks 8, 16, 24, 32, 40, and 48 ]
  • Lower Extremity Muscle Strength in the Knee Extensors [ Time Frame: Change from Baseline at Weeks 8, 16, 24, 32, 40, and 48 ]
    Lower extremity muscle strength in the knee extensors will be measured by dynamometry
  • Distance Walked in the Six-minute Walk Test [ Time Frame: Change from Baseline at Weeks 8, 16, 24, 32, 40, and 48 ]


Original Secondary Outcome: Same as current

Information By: Ultragenyx Pharmaceutical Inc

Dates:
Date Received: February 27, 2015
Date Started: May 2015
Date Completion: July 2017
Last Updated: June 23, 2016
Last Verified: June 2016