NCT02960217

Crossover Study to Assess the Efficacy and Safety of UX007 in the Treatment of Movement Disorders Associated With Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS)

A Phase 3, Randomized, Double-blind, Placebo-controlled, Crossover Study to Assess the Efficacy and Safety of UX007 in the Treatment of Movement Disorders Associated With Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS)

• Status: Terminated
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 44 (actual)
• Sponsor: Ultragenyx Pharmaceutical Inc · Industry
• Sex: All
• Age: 6 Years
• Healthy volunteers: Not accepted

Summary

The primary objective of the study was to evaluate the efficacy and safety of UX007 in the treatment of disabling paroxysmal movement disorders associated with Glut1 DS.

Conditions

• Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS)

Interventions

Type	Name	Description
DRUG	UX007	liquid for oral (PO) administration
DRUG	Placebo	liquid safflower oil for PO administration

Timeline

• Start date: 2017-04-19
• Primary completion: 2019-10-09
• Completion: 2019-10-09
• First posted: 2016-11-09
• Last updated: 2020-06-16
• Results posted: 2020-04-24

Locations

12 sites across 6 countries: United States, France, Germany, Italy, Spain, United Kingdom

Regulatory

• FDA-regulated drug study