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NCT01359319 · ClinicalTrials.gov registry record · Phase 1
Safety and Pharmacokinetics of Sialic Acid Tables in Patients With Hereditary Inclusion Body Myopathy (HIBM)
A Phase 1 study, sponsored by Ultragenyx Pharmaceutical.
- Completed
- Registry status
- Phase 1
- Development phase
- 26
- Enrollment target
NCT01359319: Completed Phase 1 study, sponsored by Ultragenyx Pharmaceutical.
NCT01359319 is a Phase 1 study that has completed, run by Ultragenyx Pharmaceutical. The registered enrollment target is 26 participants, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (57% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT01359319, a Phase 1 study, has completed, sponsored by Ultragenyx Pharmaceutical.
- COMPLETED
- Registry status
- Phase 1
- Development phase
- 26 participants
- Enrollment target
Study Summary
Hereditary Inclusion Body Myopathy (HIBM) is a severe progressive metabolic myopathy caused by a defect in the biosynthetic pathway for sialic acid (SA), a critical component of many muscle proteins, resulting in a deficiency in SA in the muscles of HIBM patients. The effective replacement of the missing SA substrate is theoretically simple, and, in animal models, replacement with SA showed significant restoration of sialylation biochemistry and excellent reduction in muscle disease. These data show that replacement can achieve significant clinical benefit in muscle pathology, function, and survival.
Primary Outcome
Safety will be evaluated in terms of the incidence and frequency of treatment-emergent adverse events (TEAEs), including clinically significant changes from baseline to scheduled timepoints in clinical laboratory values, vital signs, or physical and neurologic examination findings. Medical history and reported clinical symptoms, including increasing muscle weakness, fatigue, or pain.
Interventions
- DRUG Sialic Acid Extended Release (SA-ER) Tablets
- DRUG Sialic Acid Extended Release (SA-ER) Tables
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 26 participants |
| Start Date | 2011-07 |
| Est. Completion | 2012-05 |
| Phase | Phase 1 |
What the finished NCT01359319 record still lists
NCT01359319 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 26 participants, a relatively small participant target, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (57% lower).
The record links to 0 conditions, and to 2 interventions - of which Sialic Acid Extended Release (SA-ER) Tablets is the first listed.
NCT01359319 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT01359319 about?
NCT01359319 is a clinical study titled "Safety and Pharmacokinetics of Sialic Acid Tables in Patients With Hereditary Inclusion Body Myopathy (HIBM)". Hereditary Inclusion Body Myopathy (HIBM) is a severe progressive metabolic myopathy caused by a defect in the biosynthetic pathway for sialic acid (SA), a critical component of many muscle proteins, resulting in a deficiency in SA in the muscles of HIBM patients. The effective replacement of the m...
What is the current status of trial NCT01359319?
This trial is currently completed. It is a Phase 1 study. The enrollment target is 26 participants. The study started on 2011-07. Estimated completion is 2012-05.
What interventions are being tested in trial NCT01359319?
The interventions under investigation include: Sialic Acid Extended Release (SA-ER) Tablets (DRUG), Sialic Acid Extended Release (SA-ER) Tables (DRUG).
Who is sponsoring clinical trial NCT01359319?
This trial is sponsored by Ultragenyx Pharmaceutical, which has 52 total clinical trials registered on ClinicalTrials.gov.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT01359319's enrollment target sits among peer trials
26 1482nd of 2000 higher than 506 of 2,000 other Phase 1 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 1 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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