Multi-Center Study of ManNAc for GNE Myopathy
Research Question:
Does ManNAc slow the progression of muscle strength decline in GNE myopathy?
Basic Study Information
Purpose:
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.
Location: University of Rochester
Study Web URL: https://neuronext.org/projects/nn109-magine
Study Reference #: 2017P002505
Lead Researcher (Principal Investigator)
Lead Researcher:
Johanna Hamel
Study Contact Information
Study Coordinator: Erin Richardson
Phone: (585) 275-0420
Email: Erin_Richardson@URMC.Rochester.edu
Additional Study Details
Number of Visits:
6 to 10
Parking:
Reimbursed
Transportation Coverage:
Reimbursement:
Yes
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