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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 is a Phase 1 study that has completed, run by Ultragenyx Pharmaceutical. The registered enrollment target is 26 participants.
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.
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 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT01359319
The ClinicalTrials.gov registry entry for NCT01359319 describes a study currently listed as completed, categorized as Phase 1. The registered enrollment target is 26 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 0 conditions, and to 2 interventions - of which Sialic Acid Extended Release (SA-ER) Tablets is the first listed.
NCT01359319 reports 0 study locations.
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.
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