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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 is a Phase 2 study that has completed, run by National Human Genome Research Institute (NHGRI). The registered enrollment target is 12 participants.
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.
Interventions
- DRUG ManNAc
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 12 participants |
| Start Date | 2015-02-05 |
| Est. Completion | 2018-11-15 |
| Phase | Phase 2 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT02346461
The ClinicalTrials.gov registry entry for NCT02346461 describes a study currently listed as completed, categorized as Phase 2. The registered enrollment target is 12 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is National Human Genome Research Institute (NHGRI), which has 185 total studies on file at ClinicalTrials.gov.
The record links to 0 conditions, and to 1 intervention - of which ManNAc is the first listed.
NCT02346461 reports 0 study locations.
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.
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