Last updated on February 2018

Treatment of Mitochondrial Dysfunction in Rett Syndrome With Triheptanoin


Brief description of study

The aim of this study is to evaluate the safety and tolerability of triheptanoin in participants with Rett syndrome using laboratory values, electrocardiogram, rate of adverse events (AE), and physical exam.This study also seeks to evaluate the efficacy of UX007 (triheptanoin) in improving overall seizure frequency and dystonia.

Detailed Study Description

The aim of this study is to evaluate the safety and tolerability of triheptanoin in participants with Rett syndrome using laboratory values, electrocardiogram, rate of adverse events (AE), and physical exam. This study also seeks to evaluate the efficacy of UX007 (triheptanoin) in improving overall seizure frequency, dystonia severity, and quality of life. Participants who are eligible will take triheptanoin daily. Participation in this study will last up to 8.5 months.

Clinical Study Identifier: NCT02696044

Contact Investigators or Research Sites near you

Start Over

Daniel Tarquinio, DO

Center for Rare Neurological Diseases
Norcross, GA United States
  Connect »