Glycerol Phenylbutyrate 1100 MG/ML [Ravicti]

DRUG2 trials

Sponsors

Weill Medical College of Cornell University, Assistance Publique - Hôpitaux de Paris

Conditions

Developmental and Epileptic EncephalopathyPyruvate Dehydrogenase Complex Deficiency DiseaseSTXBP1 Encephalopathy With Epilepsy, SLC6A1 Neurodevelopmental Disorder

Early Phase 1

Phenylbutyrate for Monogenetic Developmental and Epileptic Encephalopathy

Active, not recruitingNCT04937062

Weill Medical College of Cornell UniversityDevelopmental and Epileptic Encephalopathy, STXBP1 Encephalopathy With Epilepsy, SLC6A1 Neurodevelopmental Disorder

Start: 2021-03-01End: 2026-12-31Target: 50Updated: 2026-01-22

Phase 2

Efficacy and Safety of the Treatment of Pyruvate Dehydrogenase Deficiency Patients With Glycerol Phenylbutyrate (RAVICTI)

RecruitingNCT06887777

Assistance Publique - Hôpitaux de ParisPyruvate Dehydrogenase Complex Deficiency Disease

Start: 2025-10-01End: 2027-04-01Target: 15Updated: 2026-03-31

Related Papers

Unraveling 4-Phenylbutyrate's Therapeutic Role in SLC6A1 Disorders: Pharmacochaperoning Over HDAC Inhibition.

2026-05-19

Artificial intelligence in the assessment of epilepsy‐related genetic mutations: Learned from <scp> GABA <sub>A</sub> </scp> receptors and <scp>GABA</scp> transporter 1

Epilepsia Open2026-05-08

4-Phenylbutyrate restored GABA uptake, mitigated seizures in SLC6A1 and SLC6A11 microdeletions/3p- syndrome: From cellular models to human patients.

2025-01-314 citations