Treatment of Mitochondrial Dysfunction in Rett Syndrome With Triheptanoin
Status:
Recruiting
Trial end date:
2022-02-01
Target enrollment:
Participant gender:
Summary
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.
Phase:
Phase 2
Details
Lead Sponsor:
Center for Rare Neurological Diseases, Norcross, GA Emory University
Collaborators:
Rett Syndrome Research Trust Ultragenyx Pharmaceutical Inc