Medical Information Only. Always consult your healthcare provider before enrolling in any clinical trial.
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: Completed Phase 2 study of GNE Myopathy and Hereditary Inclusion Body Myopathy, sponsored by Ultragenyx Pharmaceutical.
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, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (65% lower). The trial reports 4 study locations across 4 states. According to ClinicalTrials.gov, the official US trial registry.
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).
Primary Outcome
To evaluate the effect of SA-ER treatment on improvement of biochemical measures of sialylation and pathology in muscle. On mobility, strength, and function using a series of quantitative and physical performance measures and quality of life using patient-reported outcome measures.
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 |
What the finished NCT01517880 record still lists
NCT01517880 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 46 participants, a relatively small participant target, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (65% lower).
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 a single indexed study location in 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.
Learn More About Clinical Trials
Similar trials for GNE Myopathy
Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.
Where NCT01517880's enrollment target sits among peer trials
46 1286th of 2000 higher than 705 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
Nationwide trials with similar profiles
Cross-condition peers matched on enrollment target and registry start date, not the same-condition list above.
Similar enrollment target
- NCT01890590 · 46 participants · NA
A Phase II Study of Cyberknife Radiosurgery for Renal Cell Carcinoma
- NCT03191149 · 46 participants · Phase 2
Testing Osimertinib as a Treatment for Lung Cancers With an EGFR Exon 20 Change
- NCT03510208 · 46 participants · Phase 1
Panitumumab-IRDye800 in Diagnosing Participants With Malignant Glioma Undergoing Surgery
- NCT04048278 · 46 participants · Early Phase 1
Lidocaine Infusion in Pancreatic Cancer
Similar registry start date
- NCT05988463 · started 2027-01-01 · Phase 1
Dose-Escalation Study of Artesunate Patients With IPF
- NCT07219706 · started 2026-12-10 · NA
Dietary Fiber and Time Restricted Eating
- NCT02831335 · started 2026-12-01
Technical Development of Multi-Parametric Renocerebral MRI
- NCT05962346 · started 2026-12 · NA
Fetal Endoscopic Tracheal Occlusion for Congenital Diaphragmatic Hernia