GNE SA-ER Phase III
Research type
Research Study
Full 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 Body Myopathy (HIBM)
IRAS ID
180194
Contact name
Hanns Lochmuller
Contact email
Sponsor organisation
Ultragenyx Pharmaceutical Inc.
Eudract number
2014-005432-33
Clinicaltrials.gov Identifier
Duration of Study in the UK
1 years, 7 months, 1 days
Research summary
Study 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) 0r Hereditarty Inclusion Body Myopathy (HIBM).
Study Sites: This study will be conducted across approximately 15 sites globally, with 1 NHS site in the UK.
Study Phase: Phase 3
This study aims to further confirm the long term efficacy and safety of sialic acid extended- release (SAER) tablets, at a dose of 6g/day, in the treatment of patients with GNEM and HIBM, with regards to the effect on upper extremity muscle strength. This study will also aim to evaluate the effect of 6g/day SAER on physical function and muscle strength in the upper and lower extremities, and patient-reported ability and quality of life.
REC name
North East - Newcastle & North Tyneside 1 Research Ethics Committee
REC reference
15/NE/0183
Date of REC Opinion
3 Jul 2015
REC opinion
Further Information Favourable Opinion