Phenylbutyrate for Monogenetic Developmental and Epileptic Encephalopathy
Latest Information Update: 07 Oct 2023
At a glance
- Drugs Glycerol phenylbutyrate (Primary)
- Indications Developmental disabilities; Encephalopathy
- Focus Adverse reactions
- 29 Sep 2023 Number of treatment arms have been increased from 1 to 2 by the addition of Experimental: Monogenetic Epileptic Encephalopathy arm comprising 30 more children. Inclusion criteria is updated to decrease the minimum age limit from 2 months to 0 months. Timelines for all the primary end-points have been modified.
- 29 Sep 2023 Planned number of patients changed from 20 to 50.
- 29 Sep 2023 Planned End Date changed from 1 Jan 2025 to 31 Dec 2025.