At a glance
ClinicalIndex Comparison RecordPhase 3Completed· 89 enrolled
Drug / intervention
aceneuramic acid extended-release (Ace-ER) +1 moredrug
Likely dose
Not stated in record
Structured eligibility isn't available for this trial yet — see the full criteria in the Eligibility tab below.
Standardized by ClinicalIndex from the ClinicalTrials.gov record · verify against the source.
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 Body Myopathy (HIBM)
In Brief
A Phase 3 clinical trial evaluating aceneuramic acid extended-release (Ace-ER) and Placebo for Hereditary Inclusion Body Myopathy and 3 related conditions. Completed, enrolled 89 participants across 13 sites in 7 countries.
Detailed Summary
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.
Study Details
Study Typeinterventional
Allocation--
Masking--
Primary Purpose--
ConditionsHereditary Inclusion Body Myopathy, Distal Myopathy With Rimmed Vacuoles, Distal Myopathy, Nonaka Type, GNE Myopathy
CountriesBulgaria, Canada, France, Israel, Italy, United Kingdom, United States
Collaborators--
Timeline
Phase 3CompletedFinished
201520162017201820192020202120222023202420252026
First PostedMar 2015
Enrollment StartMay 2015
Primary CompletionJun 2017
TodayJul 2026
First PostedMar 4, 2015
Enrollment StartMay 20, 2015
Primary CompletionJun 9, 2017
TodayJul 2, 2026
Enrollment to primary: 2.1 yearsPosted 11.3 years ago
Interventions
aceneuramic acid extended-release (Ace-ER)drug
tablets for oral use
Placebodrug
tablets for oral use