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NCT04937062 · ClinicalTrials.gov registry record · Early Phase 1

Phenylbutyrate for Monogenetic Developmental and Epileptic Encephalopathy

A Early Phase 1 study of Developmental and Epileptic Encephalopathy and STXBP1 Encephalopathy With Epilepsy, SLC6A1 Neurodevelopmental Disorder, sponsored by Weill Medical College of Cornell University.

Active
Registry status
Early Phase 1
Development phase
50
Enrollment target
2
Study locations

NCT04937062 is a Early Phase 1 study of Developmental and Epileptic Encephalopathy and STXBP1 Encephalopathy With Epilepsy, SLC6A1 Neurodevelopmental Disorder that is active but no longer recruiting, run by Weill Medical College of Cornell University. The registered enrollment target is 50 participants, below the 138-participant average among 3 other Developmental and Epileptic Encephalopathy trials with a reported enrollment target (64% lower). The trial reports 2 study locations across 2 states.

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The verdict

NCT04937062, a Early Phase 1 study of Developmental and Epileptic Encephalopathy and STXBP1 Encephalopathy With Epilepsy, SLC6A1 Neurodevelopmental Disorder, is active but no longer recruiting, sponsored by Weill Medical College of Cornell University.

ACTIVE NOT RECRUITING
Registry status
Early Phase 1
Development phase
50 participants
Enrollment target
2
Study locations

Study Summary

This study is to evaluate the use of glycerol phenylbutyrate for monogenetic developmental epileptic encephalopathies (DEEs). DEEs are characterized by epilepsy and developmental delay in early life. Two examples of DEEs are STXBP1 and SLC6A1, though there are dozens of others. STXBP1 Encephalopathy is a severe disease that can cause seizures and developmental delays in infants and children. SLC6A1 neurodevelopmental disorder is characterized by developmental delay and often epilepsy. Both STXBP1 encephalopathy and SLC6A1 neurodevelopmental disorder cause symptoms because there are not enough working proteins made by these genes. It is possible that a medication called phenylbutyrate may help the the remaining proteins work better for STXBP1, SLC6A1, and/or other similar DEEs caused by single genes (i.e. "monogenetic"). This study is to test if glycerol phenylbutyrate is safe and well tolerated in children with monogenetic DEE.

Conditions Studied

Developmental and Epileptic Encephalopathy STXBP1 Encephalopathy With Epilepsy, SLC6A1 Neurodevelopmental Disorder

Interventions

  • DRUG Glycerol Phenylbutyrate 1100 MG/ML [Ravicti]

Study Locations (2)

Colorado

  • Children's Hospital Colorado - Aurora

New York

  • Weill Cornell Medicine - New York

Trial Details

FieldValue
Enrollment Target 50 participants
Start Date 2021-03-01
Est. Completion 2026-12-31
Phase Early Phase 1

Sponsor

Weill Medical College of Cornell University

701 total trials

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Full Details on ClinicalTrials.gov ↗

What the Registry Record Tells You About NCT04937062

The ClinicalTrials.gov registry entry for NCT04937062 describes a study currently listed as active not recruiting, categorized as Early Phase 1. The registered enrollment target is 50 participants, a figure that helps gauge the scale of data the investigators plan to collect, below the 138-participant average among 3 other Developmental and Epileptic Encephalopathy trials with a reported enrollment target (64% lower). The listed sponsor is Weill Medical College of Cornell University, which has 701 total studies on file at ClinicalTrials.gov.

The record links to 2 conditions, with Developmental and Epileptic Encephalopathy appearing as the primary indexed condition, and to 1 intervention - of which Glycerol Phenylbutyrate 1100 MG/ML [Ravicti] is the first listed.

NCT04937062 reports 2 study locations spanning 2 distinct geographic areas - top geographies include Colorado, New York.

Frequently Asked Questions

What is clinical trial NCT04937062 about?

NCT04937062 is a clinical study titled "Phenylbutyrate for Monogenetic Developmental and Epileptic Encephalopathy". This study is to evaluate the use of glycerol phenylbutyrate for monogenetic developmental epileptic encephalopathies (DEEs). DEEs are characterized by epilepsy and developmental delay in early life. Two examples of DEEs are STXBP1 and SLC6A1, though there are dozens of others. STXBP1 Encephalopat...

What is the current status of trial NCT04937062?

This trial is currently active not recruiting. It is a Early Phase 1 study. The enrollment target is 50 participants. The study started on 2021-03-01. Estimated completion is 2026-12-31.

What conditions does trial NCT04937062 study?

This clinical trial studies the following conditions: Developmental and Epileptic Encephalopathy, STXBP1 Encephalopathy With Epilepsy, SLC6A1 Neurodevelopmental Disorder.

What interventions are being tested in trial NCT04937062?

The interventions under investigation include: Glycerol Phenylbutyrate 1100 MG/ML [Ravicti] (DRUG).

Who is sponsoring clinical trial NCT04937062?

This trial is sponsored by Weill Medical College of Cornell University, which has 701 total clinical trials registered on ClinicalTrials.gov.

Where is trial NCT04937062 being conducted?

This trial has 2 study locations across Colorado, New York. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.

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