Open Label Trial of Triheptanoin (UX007) in Treatment of Rett Syndrome.

Summary

This is a single-center, exploratory, open-label study in 10 girls diagnosed with Rett Syndrome. The study will consist of the following 4 parts: Screening/Baseline run-in, Titration/Dose-Setting, Treatment, and Washout/Follow-up.

Timeline

Start

2017-04-01

Primary completion

2018-04-01

Completion

2018-08-01

Drugs

Indications