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A Site-Less Feasibility Trial of Phenylbutyrate for SLC6A1-Related Disorders
NCT07847918Phase 2Recruiting
- Sponsor
- Scripps Research
- Start
- Oct 2026
- Primary completion
- Mar 2029
- Countries
- 1
01
Overview
Phase 2, randomised, open-label trial of glycerol phenylbutyrate in SLC6A1-related disorder (2 arms), sponsored by Scripps Research. Primary endpoints: Recruitment efficiency at 2 years, Protocol adherence: seizure diary completion at 18 weeks and 16 more. Started Oct 2026; status: recruiting.
02
Drugs
glycerol phenylbutyrate
03
Conditions
SLC6A1-related disorder
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