Trials / Completed
CompletedNCT02377921
Phase 3 Randomized, Double-Blind, Placebo-Controlled Study to Evaluate Sialic Acid in Patients With Glucosamine (UDP-N-acetyl)-2-epimerase Myopathy (GNEM) or Hereditary Inclusion Body Myopathy (HIBM)
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)
- Status
- Completed
- Phase
- Phase 3
- Study type
- Interventional
- Enrollment
- 89 (actual)
- Sponsor
- Ultragenyx Pharmaceutical Inc · Industry
- Sex
- All
- Age
- 18 Years – 55 Years
- Healthy volunteers
- Not accepted
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.
Conditions
- Hereditary Inclusion Body Myopathy
- Distal Myopathy With Rimmed Vacuoles
- Distal Myopathy, Nonaka Type
- GNE Myopathy
Interventions
| Type | Name | Description |
|---|---|---|
| DRUG | aceneuramic acid extended-release (Ace-ER) | tablets for oral use |
| DRUG | Placebo | tablets for oral use |
Timeline
- Start date
- 2015-05-20
- Primary completion
- 2017-06-09
- Completion
- 2017-06-09
- First posted
- 2015-03-04
- Last updated
- 2019-06-27
- Results posted
- 2018-07-09
Locations
13 sites across 7 countries: United States, Bulgaria, Canada, France, Israel, Italy, United Kingdom
Source: ClinicalTrials.gov record NCT02377921. Inclusion in this directory is not an endorsement.