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

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).

Timeline

Start

2014-02-28

Primary completion

2017-09-20

Completion

2017-09-20

Outcome

Missed primary endpoint

registry analysis (superiority test); UX007 vs Placebo; p = 0.5812; Hodges-Lehmann estimate 13.45 (90% CI -38.63 to 80.95); Wilcoxon rank-sum test NCT01993186 ↗

paper SIGNIFICANCE: Triheptanoin did not significantly reduce seizure frequency in patients with Glut1DS not on the ketogenic diet. PMID 35441706 ↗

Publications

• Striano P, Auvin S, Collins A, Horvath R, Scheffer IE, Tzadok M, Miller I, Kay Koenig M, Lacy A, Davis R, Garcia-Cazorla A, Saneto RP, Brandabur M, Blair S, Koutsoukos T, De Vivo D. A randomized, double-blind trial of triheptanoin for drug-resistant epilepsy in glucose transporter 1 deficiency syndrome. Epilepsia. 2022 Jul;63(7):1748-1760. doi: 10.1111/epi.17263. Epub 2022 May 21.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Triheptanoin	Other / unclassified	35 %	Oral

Indications