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Phenylbutyrate for Monogenetic Developmental and Epileptic Encephalopathy

Trial Profile

Phenylbutyrate for Monogenetic Developmental and Epileptic Encephalopathy

Status: Recruiting
Phase of Trial: Phase 0

Latest Information Update: 07 Oct 2023

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At a glance

  • Drugs Glycerol phenylbutyrate (Primary)
  • Indications Developmental disabilities; Encephalopathy
  • Focus Adverse reactions
  • Most Recent Events

    • 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.
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