Multi-Center Study of ManNAc for GNE Myopathy (NCT04231266)
A Randomized, Double-Blind, Placebo-Controlled, Multi-Center Study to Evaluate the Efficacy of ManNAc in Subjects With GNE Myopathy
🩺 Plain-English Summary (8th-Grade Level)
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.
Who can join: Review the eligibility criteria below.
Location: UCLA — Los Angeles, California
Age range: 18 Years to 70 Years
🧪 Interventions in This Study
📋 "Do I Qualify?" — 1-Minute Self Screener
Instant ChecklistFull eligibility criteria are available on the official NIH protocol. View the criteria ↗
📍 All Participating Trial Locations (10 Sites)
- UCLA — Los Angeles, California
- University of Iowa — Iowa City, Iowa
- University of Kansas, Medical Center — Kansas City, Kansas
- NIH Clinical Center — Bethesda, Maryland
- Brigham and Women's Hospital — Boston, Massachusetts
- Washington University — St Louis, Missouri
- Columbia University Medical Center — New York, New York
- University of Rochester — Rochester, New York
- Ohio State University, Wexner Medical Center — Columbus, Ohio
- University of Utah — Salt Lake City, Utah
💰 Cost, Insurance & Patient Rights
- 100% Free Items: The investigational treatment and protocol-required procedures are supplied at zero cost by the sponsor.
- Routine Patient Care: Under federal law (ACA § 2709) and many state statutes, routine care costs are covered during participation.
- Voluntary Participation: You may withdraw at any time without affecting your standard medical care.
More studies in GNE Myopathy
- First-in-human Study of UX016 in GNEM — Orange, California
Explore by condition: All GNE Myopathy clinical trials
💡 How to Participate
- Check the checklist above to see if you may qualify.
- Review the official NIH record below to find the coordinating site.
- Ask about the visit schedule, what is covered at no cost, and any travel support.
- Confirm with your own doctor before making a decision.