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Phenylbutyrate for Monogenetic Developmental and Epileptic Encephalopathy
NCT04937062Phase 1Active, not recruiting
- Sponsor
- Weill Cornell Medicine
- Start
- Mar 2021
- Primary completion
- Dec 2026
- Modality
- Small Molecules
- Countries
- 1
01
Overview
Phase 1, open-label trial of glycerol phenylbutyrate in developmental and epileptic encephalopathy (DEE), STXBP1 encephalopathy (STXBP1E) and SLC6A1-related neurodevelopmental disorder (SLC6A1-NDD) (2 arms), sponsored by Weill Cornell Medicine. Primary endpoints: Short Term Adverse events at 20 weeks, Long Term Adverse events and 1 more. Started Mar 2021; status: active, not recruiting.
02
Drugs
glycerol phenylbutyrate
03
Conditions
developmental and epileptic encephalopathy (DEE)STXBP1 encephalopathy (STXBP1E)SLC6A1-related neurodevelopmental disorder (SLC6A1-NDD)
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