Trials / Completed
CompletedNCT01993186
Phase 2 Study of Triheptanoin (UX007) for the Treatment of Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS)
A Randomized, Double-blind, Placebo-controlled, Parallel-group, Study to Assess the Safety and Efficacy of UX007 in Subjects With Glucose Transporter Type 1 Deficiency Syndrome
- Status
- Completed
- Phase
- Phase 2
- Study type
- Interventional
- Enrollment
- 36 (actual)
- Sponsor
- Ultragenyx Pharmaceutical Inc · Industry
- Sex
- All
- Age
- 1 Year – 100 Years
- Healthy volunteers
- Not accepted
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).
Conditions
Interventions
| Type | Name | Description |
|---|---|---|
| DRUG | UX007 | oral liquid |
| DRUG | Placebo | oral liquid |
Timeline
- Start date
- 2014-02-28
- Primary completion
- 2017-09-20
- Completion
- 2017-09-20
- First posted
- 2013-11-25
- Last updated
- 2020-06-19
- Results posted
- 2020-05-29
Locations
14 sites across 7 countries: United States, Australia, France, Israel, Italy, Spain, United Kingdom
Source: ClinicalTrials.gov record NCT01993186. Inclusion in this directory is not an endorsement.