Overview

Phase 2 Study of Triheptanoin (UX007) for the Treatment of Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS)

Status:
Completed
Trial end date:
2017-09-20
Target enrollment:
Participant gender:
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).
Phase:
Phase 2
Details
Lead Sponsor:
Ultragenyx Pharmaceutical Inc
Treatments:
Glycerol