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NCT01830972 · ClinicalTrials.gov registry record · Phase 2
An Open Label Phase 2 Extension Study of Higher Dose Sialic Acid-Extended Release (SA-ER) Tablets and Sialic Acid-Immediate Release (SA-IR) Capsules in Patients With Glucosamine (UDP-N-acetyl)-2-Epimerase (GNE) Myopathy
A Phase 2 study, sponsored by Ultragenyx Pharmaceutical.
- Completed
- Registry status
- Phase 2
- Development phase
- 59
- Enrollment target
NCT01830972: Completed Phase 2 study, sponsored by Ultragenyx Pharmaceutical.
NCT01830972 is a Phase 2 study that has completed, run by Ultragenyx Pharmaceutical. The registered enrollment target is 59 participants, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (56% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT01830972, a Phase 2 study, has completed, sponsored by Ultragenyx Pharmaceutical.
- COMPLETED
- Registry status
- Phase 2
- Development phase
- 59 participants
- Enrollment target
Study Summary
The safety objectives of the study are to: evaluate additional long-term safety of SA-ER treatment of participants with GNE myopathy previously treated with SA-ER at dose of 6g/day (Part I); evaluate the safety of 12g /day SA (delivered by 1.5g of SA-ER tablets and 1.5g of SA-IR capsules 4 times per day) in the treatment of participants with GNE myopathy (Part II) over a 6 month treatment period; evaluate the safety of SA treatment at both 6g/day and 12 g/day (Part III \[SA-ER/SA-IR\] and Part IV \[SA-ER\]).
Primary Outcome
An adverse event (AE) is defined as any untoward medical occurrence, whether or not considered drug related. A serious AE (SAE) is an AE that at any dose results in any of the following: death, a life-threatening AE; inpatient hospitalization or prolongation of existing hospitalization; persistent or significant incapacity or substantial disruption of the ability to conduct normal life functions; a congenital anomaly/birth defect. TEAEs include all adverse events that start on or after the first
Interventions
- DRUG SA-ER 500 mg
- DRUG SA-IR 500 mg
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 59 participants |
| Start Date | 2013-06-04 |
| Est. Completion | 2017-02-14 |
| Phase | Phase 2 |
What the finished NCT01830972 record still lists
NCT01830972 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 59 participants, a relatively small participant target, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (56% lower).
The record links to 0 conditions, and to 2 interventions - of which SA-ER 500 mg is the first listed.
NCT01830972 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT01830972 about?
NCT01830972 is a clinical study titled "An Open Label Phase 2 Extension Study of Higher Dose Sialic Acid-Extended Release (SA-ER) Tablets and Sialic Acid-Immediate Release (SA-IR) Capsules in Patients With Glucosamine (UDP-N-acetyl)-2-Epimerase (GNE) Myopathy". The safety objectives of the study are to: evaluate additional long-term safety of SA-ER treatment of participants with GNE myopathy previously treated with SA-ER at dose of 6g/day (Part I); evaluate the safety of 12g /day SA (delivered by 1.5g of SA-ER tablets and 1.5g of SA-IR capsules 4 times per...
What is the current status of trial NCT01830972?
This trial is currently completed. It is a Phase 2 study. The enrollment target is 59 participants. The study started on 2013-06-04. Estimated completion is 2017-02-14.
What interventions are being tested in trial NCT01830972?
The interventions under investigation include: SA-ER 500 mg (DRUG), SA-IR 500 mg (DRUG).
Who is sponsoring clinical trial NCT01830972?
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 NCT01830972's enrollment target sits among peer trials
59 1133rd of 2000 higher than 866 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
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