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NCT01993186 · ClinicalTrials.gov registry record · Phase 2
Phase 2 Study of Triheptanoin (UX007) for the Treatment of Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS)
A Phase 2 study, sponsored by Ultragenyx Pharmaceutical.
- Completed
- Registry status
- Phase 2
- Development phase
- 36
- Enrollment target
NCT01993186: Completed Phase 2 study, sponsored by Ultragenyx Pharmaceutical.
NCT01993186 is a Phase 2 study that has completed, run by Ultragenyx Pharmaceutical. The registered enrollment target is 36 participants, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (73% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT01993186, a Phase 2 study, has completed, sponsored by Ultragenyx Pharmaceutical.
- COMPLETED
- Registry status
- Phase 2
- Development phase
- 36 participants
- Enrollment target
Study Summary
The primary objectives of the study are to evaluate the efficacy of UX007 compared to placebo as measured by the reduction from randomization to Week 8 in frequency of seizures and to evaluate the safety of UX007 via adverse event (AE) rates, laboratory values, and electrocardiogram (ECG).
Primary Outcome
Reduction from baseline to Week 8 in frequency of seizures (normalized to a 4-week rate): observable seizures measured for 6 weeks after 2-week titration by diary and absence seizures measured overnight by EEG. Observable seizures from the diary include generalized tonic-clonic, generalized tonic, generalized clonic, generalized atonic, partial/focal with secondary generalization, myoclonic, myoclonic (astatic) atonic, myoclonic tonic, complex partial/focal, simple partial/focal motor, simple pa
Interventions
- DRUG Placebo
- DRUG UX007
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 36 participants |
| Start Date | 2014-02-28 |
| Est. Completion | 2017-09-20 |
| Phase | Phase 2 |
What the finished NCT01993186 record still lists
NCT01993186 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 36 participants, a relatively small participant target, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (73% lower).
The record links to 0 conditions, and to 2 interventions - of which Placebo is the first listed.
NCT01993186 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT01993186 about?
NCT01993186 is a clinical study titled "Phase 2 Study of Triheptanoin (UX007) for the Treatment of Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS)". The primary objectives of the study are to evaluate the efficacy of UX007 compared to placebo as measured by the reduction from randomization to Week 8 in frequency of seizures and to evaluate the safety of UX007 via adverse event (AE) rates, laboratory values, and electrocardiogram (ECG).
What is the current status of trial NCT01993186?
This trial is currently completed. It is a Phase 2 study. The enrollment target is 36 participants. The study started on 2014-02-28. Estimated completion is 2017-09-20.
What interventions are being tested in trial NCT01993186?
The interventions under investigation include: Placebo (DRUG), UX007 (DRUG).
Who is sponsoring clinical trial NCT01993186?
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 NCT01993186's enrollment target sits among peer trials
36 1496th of 2000 higher than 477 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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