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Phenylbutyrate for Neurodevelopmental Disorders
Study Summary
This trial will test if phenylbutyrate is safe and well tolerated in children with STXBP1 encephalopathy or SLC6A1 neurodevelopmental disorder.
Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowTimeline
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Trial Design
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Who is running the clinical trial?
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- I have SLC6A1-NDD, but seizures are not a main concern for my condition.Your heart's electrical activity measured on a test is less than 450 milliseconds.I am between 2 months and 17 years old.I have a confirmed genetic condition related to STXBP1 or SLC6A1.My kidney function is normal, with a filtration rate above 90 mL/min.
- Group 1: Monogenetic Epileptic Encephalopathy
- Group 2: SLC6A1 and STXBP1
- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
Frequently Asked Questions
Does one have to exceed a certain age limit in order to participate in the experiment?
"In order to join this medical trial, participants must be between two months and seventeen years old. Of the 100 trials administered, 95 are designed for patients aged 65 or older while only 5 address minors under 18."
Are there any records of prior research concerning Glycerol Phenylbutyrate 1100 MG/ML [Ravicti]?
"Currently, there are 5 clinical trials for Glycerol Phenylbutyrate 1100 MG/ML [Ravicti], with one of them being in the third phase. With a focus on Bern, Utah as its primary venue, these studies span 18 different locations."
What is the population size of this clinical experiment?
"At this time, enrolment for the trial is closed. Initially posted on March 1st 2021, and last updated September 27th 2022; if you are exploring other studies there are a total of 175 trials recruiting patients with epilepsy and 5 clinical investigations still open to participants taking Glycerol Phenylbutyrate 1100 MG/ML [Ravicti]."
Who is eligible for inclusion in this experimental program?
"This clinical trial is currently taking on 21 individuals, ranging in age from 2 Months to 17 years old, who are diagnosed with epilepsy. Furthermore, entrants must meet the following criteria: no concurrent medical illness that could increase risk of adverse drug reactions or interfere with study follow-up; estimated glomerular filtration rate > 90 mL/minute/1.73 m2 at Screening; QT interval corrected < 450 msec on the Screening EKG.; diagnosis confirmed by laboratory report (i.e., a genetic test and corresponding clinical picture); normal laboratory test results for serum aminotransferase concentrations and ammonia;"
Is this a pioneering study of its kind?
"Currently, 5 clinical trials for Glycerol Phenylbutyrate 1100 MG/ML [Ravicti] are underway in 12 cities and 6 countries. The first of these studies was conducted by Horizon Therapeutics, LLC in 2018 on 18 participants as part of the Phase 4 drug approval process. Since then, 30 further research initiatives have been undertaken."
What diseases or afflictions does Glycerol Phenylbutyrate 1100 MG/ML [Ravicti] primarily target?
"Ravicti (Glycerol Phenylbutyrate 1100 MG/ML) is a viable therapeutic option for those dealing with enzyme deficiencies, carbamyl phosphate accumulation, and citrullinemia."
Is this research program still accepting volunteers?
"At present, this trial has ceased its recruitment process. The original posting date for the study was March 1st 2021 and it's most recent update happened on September 27th 2022. Alternatively, there are 175 clinical trials actively recruiting participants who have epilepsy and 5 studies that involve Glycerol Phenylbutyrate 1100 MG/ML [Ravicti]."
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