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)

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.

Timeline

Start

2017-04-19

Primary completion

2019-10-09

Completion

2019-10-09

Outcome

Missed primary endpoint

Stopped: “Study was halted prematurely due to lack of efficacy.”

registry analysis (superiority test); Double-Blind UX007 vs Double-Blind Placebo; p = 0.2684; Hodges-Lehmann estimate (Median Diff.) 1.46 (95% CI -1.12 to 4.36); Wilcoxon Rank Sum test NCT02960217 ↗

release “Phase 3 study evaluating UX007 in patients with Glut1 DS did not achieve its primary endpoint compared to placebo.” ultragenyx.com ↗

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Triheptanoin	Other / unclassified	130 g	Oral

Indications