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NCT02346461 · ClinicalTrials.gov registry record · Phase 2
An Open Label Phase 2 Study of ManNAc in Subjects With GNE Myopathy
A Phase 2 study, sponsored by National Human Genome Research Institute (NHGRI).
- Completed
- Registry status
- Phase 2
- Development phase
- 12
- Enrollment target
NCT02346461: Completed Phase 2 study, sponsored by National Human Genome Research Institute (NHGRI).
NCT02346461 is a Phase 2 study that has completed, run by National Human Genome Research Institute (NHGRI). The registered enrollment target is 12 participants, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (91% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT02346461, a Phase 2 study, has completed, sponsored by National Human Genome Research Institute (NHGRI).
- COMPLETED
- Registry status
- Phase 2
- Development phase
- 12 participants
- Enrollment target
Study 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.
Primary Outcome
The mean area under the plasma ManNAc concentration-versus time curve from time 0 (dosing) to the time of last quantifiable concentration.
Interventions
- DRUG ManNAc
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 12 participants |
| Start Date | 2015-02-05 |
| Est. Completion | 2018-11-15 |
| Phase | Phase 2 |
What the finished NCT02346461 record still lists
NCT02346461 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 12 participants, a relatively small participant target, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (91% lower).
The record links to 0 conditions, and to 1 intervention - of which ManNAc is the first listed.
NCT02346461 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT02346461 about?
NCT02346461 is a clinical study titled "An Open Label Phase 2 Study of ManNAc in Subjects With GNE Myopathy". 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-acetylneurami...
What is the current status of trial NCT02346461?
This trial is currently completed. It is a Phase 2 study. The enrollment target is 12 participants. The study started on 2015-02-05. Estimated completion is 2018-11-15.
What interventions are being tested in trial NCT02346461?
The interventions under investigation include: ManNAc (DRUG).
Who is sponsoring clinical trial NCT02346461?
This trial is sponsored by National Human Genome Research Institute (NHGRI), which has 185 total clinical trials registered on ClinicalTrials.gov.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT02346461's enrollment target sits among peer trials
12 1948th of 2000 higher than 34 of 2,000 other Phase 2 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 2 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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