CI

At a glance

ClinicalIndex Comparison Record
Phase 2Completed· 12 enrolled
Drug / intervention
ManNAc +1 moredrug
Likely dose
ManNAc 3 gfrom 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.

Search/NCT02346461
NCT02346461Phase 2Completed
CompareCT.gov

An Open-Label Phase 2 Study of ManNAc in Subjects With GNE Myopathy

National Human Genome Research Institute (NHGRI)·interventional·Posted Jan 27, 2015·Updated Apr 16, 2019

In Brief

A Phase 2 clinical trial evaluating ManNAc for GNE Myopathy. Completed, enrolled 12 participants across 1 site.

Detailed Summary

Background: Patients with GNE myopathy have progressive muscle weakness and can have difficulty walking and decreased mobility. The disease is a rare genetic disorder that results from a gene mutation in a key step in the body's production of a sugar called sialic acid, (also called N-acetylneuraminic acid, Neu5Ac). Researchers think decreased sialic acid bound to muscle proteins may be the cause of muscle wasting in GNE myopathy. Researchers are testing the drug ManNAc which is a precursor in the production of sialic acid within cells. ManNAc is provided as a powder dissolved in water to be administered orally.

Study Details

Study Typeinterventional
Allocation--
Masking--
Primary Purpose--
ConditionsGNE Myopathy
CountriesUnited States
CollaboratorsNational Center for Advancing Translational Sciences (NCATS)

Timeline

Phase 2CompletedFinished
201520162017201820192020202120222023202420252026
First PostedJan 27, 2015
Enrollment StartFeb 5, 2015
Primary CompletionDec 30, 2017
Study CompletionNov 15, 2018
TodayJul 2, 2026
Enrollment to primary: 2.9 yearsPosted 11.4 years ago

Interventions

ManNAcdrug

At doses of 3 g and 6 g twice daily for a total dose of 6 and 12 g per day.

ManNAcdrug

At doses of 6 g twice daily (12 g per day).