drugset / Trial / NCT02696044
Treatment of Mitochondrial Dysfunction in Rett Syndrome With Triheptanoin
Phase 2
Unknown
12 enrolled
Center for Rare Neurological Diseases, Norcross, GA
Rett Syndrome Research Trust · collabUltragenyx Pharmaceutical Inc · collab
Non-randomizedSingle-groupOpen-labelTreatment
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.
Timeline
- Start
- 2016-06
- Primary completion
- 2021-02
- Completion
- 2022-02
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | Triheptanoin | Other / unclassified | 1.2 g/kg | Oral |
| Subject | Triheptanoin | Other / unclassified | 1.5 g/kg | Oral |
| Subject | Triheptanoin | Other / unclassified | 2 g/kg | Oral |
| Subject | Triheptanoin | Other / unclassified | 2.5 g/kg | Oral |
| Subject | Triheptanoin | Other / unclassified | 3 g/kg | Oral |
| Subject | Triheptanoin | Other / unclassified | 4 g/kg | Oral |