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Multi-Center Study of ManNAc for GNE Myopathy
NCT04231266 · View on ClinicalTrials.gov ↗
Study Summary
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.
Conditions Studied
Interventions
- OTHER Placebo
- DRUG ManNAc
Study Locations (10)
New York
- Columbia University Medical Center — New York
- University of Rochester — Rochester
California
- UCLA — Los Angeles
Iowa
- University of Iowa — Iowa City
Kansas
- University of Kansas, Medical Center — Kansas City
Maryland
- NIH Clinical Center — Bethesda
Massachusetts
- Brigham and Women's Hospital — Boston
Missouri
- Washington University — St Louis
Ohio
- Ohio State University, Wexner Medical Center — Columbus
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 54 participants |
| Start Date | 2022-04-05 |
| Est. Completion | 2025-10-30 |
| Phase | Phase 2 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT04231266
The ClinicalTrials.gov registry entry for NCT04231266 describes a study currently listed as active not recruiting. It is categorized as Phase 2, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 54 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Leadiant Biosciences, which has 2 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.
The record links to 1 condition, with GNE Myopathy appearing as the primary indexed condition, and to 2 interventions — of which Placebo is the first listed. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.
Geographic footprint matters for practical reasons: NCT04231266 reports 10 study locations spanning 9 distinct geographic areas — top geographies include New York, California, Iowa. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.
Frequently Asked Questions
What is clinical trial NCT04231266 about?
NCT04231266 is a clinical study titled "Multi-Center Study of ManNAc for GNE Myopathy". 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 the...
What is the current status of trial NCT04231266?
This trial is currently active not recruiting. It is a Phase 2 study. The enrollment target is 54 participants. The study started on 2022-04-05. Estimated completion is 2025-10-30.
What conditions does trial NCT04231266 study?
This clinical trial studies the following conditions: GNE Myopathy. These conditions were identified from the trial registry and reflect the primary focus areas of the research.
What interventions are being tested in trial NCT04231266?
The interventions under investigation include: Placebo (OTHER), ManNAc (DRUG). Each intervention is being evaluated for safety and efficacy as part of this clinical study.
Who is sponsoring clinical trial NCT04231266?
This trial is sponsored by Leadiant Biosciences, which has 2 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.
Where is trial NCT04231266 being conducted?
This trial has 10 study locations across California, Iowa, Kansas, Maryland, Massachusetts. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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