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Multi-Center Study of ManNAc for GNE Myopathy (MAGiNE)
GNE myopathy is a rare genetic muscle disease characterized by progressive muscle atrophy and weakness. The disease is caused by mutations in the gene that encodes the enzyme that initiates and regulates N-acetylneuraminic acid (Neu5Ac) biosynthesis and glycan sialylation. Currently, there is no therapy available for this disease. N-Acetylmannosamine (ManNAc), an orphan drug in development for GNE myopathy, is an uncharged monosaccharide and the first committed precursor in Neu5Ac biosynthesis. In this randomized, double-blind, placebo-controlled trial the efficacy and long-term safety of ManNAc will be evaluated in subjects with GNE myopathy.
Details
| Lead sponsor | Leadiant Biosciences, Inc. |
|---|---|
| Phase | PHASE2 |
| Status | ACTIVE_NOT_RECRUITING |
| Enrolment | 54 |
| Start date | Tue Apr 05 2022 00:00:00 GMT+0000 (Coordinated Universal Time) |
| Completion | Thu Oct 30 2025 00:00:00 GMT+0000 (Coordinated Universal Time) |
Conditions
- GNE Myopathy
Interventions
- ManNAc
- Placebo
Countries
United States