Glycerol phenylbutyrate

For research use only.

Catalog No.S6981 Synonyms: GT4P, HPN-100M, Ravicti

Glycerol phenylbutyrate Chemical Structure

CAS No. 611168-24-2

Glycerol phenylbutyrate (GT4P, HPN-100M, Ravicti) is a nitrogen-binding agent and acts as a sigma-2 (σ2) receptor ligand with a pKi of 8.02. Glycerol phenylbutyrate is a prodrug in which phenylbutyrate (PBA) is released from the glycerol backbone by lipases in the gastrointestinal tract.

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Description Glycerol phenylbutyrate (GT4P, HPN-100M, Ravicti) is a nitrogen-binding agent and acts as a sigma-2 (σ2) receptor ligand with a pKi of 8.02. Glycerol phenylbutyrate is a prodrug in which phenylbutyrate (PBA) is released from the glycerol backbone by lipases in the gastrointestinal tract.
Targets
σ2 receptor [2]
()
8.02(pKi)

Protocol

Solubility (25°C)

Chemical Information

Molecular Weight 530.65
Formula

C33H38O6

Density 1.13 g/mL
CAS No. 611168-24-2
Storage powder
in solvent
Synonyms GT4P, HPN-100M, Ravicti

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Clinical Trial Information

NCT Number Recruitment interventions Conditions Sponsor/Collaborators Start Date Phases
NCT05019417 Recruiting Drug: Glycerol Phenylbutyrate 1100 MG/ML Monocarboxylate Transporter 8 Deficiency Kaplan Medical Center|Weizmann Institute of Science June 30 2021 Phase 2|Phase 3
NCT02323100 Recruiting Drug: Ravicti low dose|Drug: Ravicti high dose|Drug: Placebo Cystic Fibrosis National Jewish Health|University of Alabama at Birmingham|Children''s Hospital of Philadelphia|Johns Hopkins University|Horizon Pharma Ireland Ltd. Dublin Ireland December 2 2018 Phase 1|Phase 2
NCT02246218 Completed Drug: RAVICTI Urea Cycle Disorder Horizon Therapeutics LLC|Horizon Pharma Ireland Ltd. Dublin Ireland December 31 2014 Phase 4

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Cell Lines Assay Type Concentration Incubation Time Formulation Activity Description PMID