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NCT01517880 · ClinicalTrials.gov registry record · Phase 2
A Phase 2 Study to Evaluate the Dose and Pharmacodynamic Efficacy of Sialic Acid-Extended Release (SA-ER) Tablets in Patients With GNE Myopathy or Hereditary Inclusion Body Myopathy
A Phase 2 study of GNE Myopathy and Hereditary Inclusion Body Myopathy, sponsored by Ultragenyx Pharmaceutical.
- Completed
- Registry status
- Phase 2
- Development phase
- 46
- Enrollment target
- 4
- Study locations
NCT01517880 is a Phase 2 study of GNE Myopathy and Hereditary Inclusion Body Myopathy that has completed, run by Ultragenyx Pharmaceutical. The registered enrollment target is 46 participants. The trial reports 4 study locations across 4 states.
The verdict
NCT01517880, a Phase 2 study of GNE Myopathy and Hereditary Inclusion Body Myopathy, has completed, sponsored by Ultragenyx Pharmaceutical.
- COMPLETED
- Registry status
- Phase 2
- Development phase
- 46 participants
- Enrollment target
- 4
- Study locations
Study Summary
GNE myopathy or hereditary inclusion body myopathy (HIBM) is a severe progressive metabolic myopathy caused by a defect in the biosynthetic pathway for sialic acid (SA).
Conditions Studied
Interventions
- DRUG Placebo
- DRUG Sialic Acid Extended Release (SA-ER)
Study Locations (4)
California
- UCLA Medical Center - Los Angeles
Missouri
- Washington University School of Medicine - St Louis
New York
- New York University School of Medicine - New York
Other
- Hadassah University Hospital - Jerusalem
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 46 participants |
| Start Date | 2012-05 |
| Est. Completion | 2013-11 |
| Phase | Phase 2 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT01517880
The ClinicalTrials.gov registry entry for NCT01517880 describes a study currently listed as completed, categorized as Phase 2. The registered enrollment target is 46 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Ultragenyx Pharmaceutical, which has 52 total studies on file at ClinicalTrials.gov.
The record links to 2 conditions, with GNE Myopathy appearing as the primary indexed condition, and to 2 interventions - of which Placebo is the first listed.
NCT01517880 reports 4 study locations spanning 4 distinct geographic areas - top geographies include California, Missouri, New York.
Frequently Asked Questions
What is clinical trial NCT01517880 about?
NCT01517880 is a clinical study titled "A Phase 2 Study to Evaluate the Dose and Pharmacodynamic Efficacy of Sialic Acid-Extended Release (SA-ER) Tablets in Patients With GNE Myopathy or Hereditary Inclusion Body Myopathy". GNE myopathy or hereditary inclusion body myopathy (HIBM) is a severe progressive metabolic myopathy caused by a defect in the biosynthetic pathway for sialic acid (SA).
What is the current status of trial NCT01517880?
This trial is currently completed. It is a Phase 2 study. The enrollment target is 46 participants. The study started on 2012-05. Estimated completion is 2013-11.
What conditions does trial NCT01517880 study?
This clinical trial studies the following conditions: GNE Myopathy, Hereditary Inclusion Body Myopathy.
What interventions are being tested in trial NCT01517880?
The interventions under investigation include: Placebo (DRUG), Sialic Acid Extended Release (SA-ER) (DRUG).
Who is sponsoring clinical trial NCT01517880?
This trial is sponsored by Ultragenyx Pharmaceutical, which has 52 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT01517880 being conducted?
This trial has 4 study locations across California, Missouri, New York. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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