Phenylbutyric acid
- Generic Name
- Phenylbutyric acid
- Brand Names
- Ammonaps, Buphenyl, Olpruva 2 Gm Pack, Pheburane, Relyvrio
- Drug Type
- Small Molecule
- Chemical Formula
- C10H12O2
- CAS Number
- 1821-12-1
- Unique Ingredient Identifier
- 7WY7YBI87E
- Background
Phenylbutyric acid is a fatty acid and a derivative of butyric acid naturally produced by colonic bacteria fermentation. It demonstrates a number of cellular and biological effects, such as relieving inflammation and acting as a chemical chaperone. It is used to treat genetic metabolic syndromes, neuropathies, and urea cycle disorders.
- Indication
Phenylbutyric acid is used for the treatment of various conditions, including urea cycle
disorders, neonatal-onset deficiency, late-onset deficiency disease in patients with a history of hyperammonemic encephalopathy. Phenylbutyric acid must be combined with dietary protein restriction and, in some cases, essential amino acid supplementation.
Phenylbutyric acid, as sodium phenylbutyrate, is used in combination with tauroursodeoxycholic acid to treat amyotrophic lateral sclerosis (ALS) in adults.
- Associated Conditions
- Amyotrophic Lateral Sclerosis (ALS), Argininosuccinate Synthetase Deficiency (Citrullinemia), Carbamyl Phosphate Synthetase Deficiency, Ornithine Transcarbamylase Deficiency, Deficiencies in enzymes of the urea cycle, Neonatal-onset deficiency
Study of Sodium Phenylbutyrate (ACER-001) for the Treatment of Pediatric and Adults Patients With Medium Chain Acyl-CoA Dehydrogenase Deficiency (MCADD)
- Conditions
- Medium-chain Acyl-CoA Dehydrogenase Deficiency
- Interventions
- First Posted Date
- 2025-01-14
- Last Posted Date
- 2025-05-04
- Lead Sponsor
- Jerry Vockley, MD, PhD
- Target Recruit Count
- 24
- Registration Number
- NCT06773026
- Locations
- 🇺🇸
UPMC Children's Hospital of Pittsburgh, Pittsburgh, Pennsylvania, United States
Na-Phenylbutyrate VAscular Trial
- Conditions
- Diabetes
- Interventions
- Other: Placebo
- First Posted Date
- 2024-10-17
- Last Posted Date
- 2025-02-20
- Lead Sponsor
- University of Pennsylvania
- Target Recruit Count
- 30
- Registration Number
- NCT06645769
Study of Sodium Phenylbutyrate (ACER-001) for the Treatment of Patients With Medium Chain Acyl-CoA Dehydrogenase Deficiency (MCADD)
- Conditions
- Medium-chain Acyl-CoA Dehydrogenase Deficiency
- Interventions
- First Posted Date
- 2023-10-05
- Last Posted Date
- 2025-04-04
- Lead Sponsor
- Jerry Vockley, MD, PhD
- Target Recruit Count
- 24
- Registration Number
- NCT06069375
- Locations
- 🇺🇸
UPMC Children's Hospital of Pittsburgh, Pittsburgh, Pennsylvania, United States
A Healthy Volunteer Study to Compare Fezagepras (PBI-4050) With Sodium Phenylbutyrate
- First Posted Date
- 2022-04-27
- Last Posted Date
- 2022-08-01
- Lead Sponsor
- Liminal BioSciences Ltd.
- Target Recruit Count
- 8
- Registration Number
- NCT05349435
- Locations
- 🇬🇧
Celerion, Belfast, Northern Ireland, United Kingdom
The Effects of Sodium Phenylbutyrate on Circulating Branched Chain Amino Acids
- First Posted Date
- 2021-08-31
- Last Posted Date
- 2021-08-31
- Lead Sponsor
- University of Nottingham
- Target Recruit Count
- 6
- Registration Number
- NCT05028803
- Locations
- 🇬🇧
University of Nottingham, Derby, Derbyshire, United Kingdom
BSEP Function Rescue During Childhood Inhereditary Cholestatic Diseases
- Conditions
- Hereditary DiseasesCholestasis, Intrahepatic
- Interventions
- First Posted Date
- 2020-08-31
- Last Posted Date
- 2023-02-09
- Lead Sponsor
- Children's Hospital of Fudan University
- Registration Number
- NCT04531878
Safety and Tolerability of Phenylbutyrate in Inclusion Body Myositis
- Conditions
- Sporadic Inclusion Body MyositisInclusion Body Myositis
- Interventions
- First Posted Date
- 2020-06-09
- Last Posted Date
- 2022-05-23
- Lead Sponsor
- University of Kansas Medical Center
- Target Recruit Count
- 10
- Registration Number
- NCT04421677
- Locations
- 🇺🇸
University of Kansas Medical Center, Kansas City, Kansas, United States
PBA Use for Treatment of ATF6-/- Patients
- First Posted Date
- 2019-08-01
- Last Posted Date
- 2025-01-08
- Lead Sponsor
- Columbia University
- Target Recruit Count
- 2
- Registration Number
- NCT04041232
- Locations
- 🇺🇸
Edward S. Harkness Eye Institute, New York, New York, United States
Use of Phenylbutyrate Therapy for Patients With Pyruvate Dehydrogenase Complex Deficiency.
- Conditions
- Pyruvate Dehydrogenase Complex Deficiency
- Interventions
- First Posted Date
- 2018-11-07
- Last Posted Date
- 2021-10-13
- Lead Sponsor
- Fondazione Telethon
- Target Recruit Count
- 1
- Registration Number
- NCT03734263
- Locations
- 🇮🇹
Federico II University, Napoli, Italy
Phenylbutyrate in Proteinuric Nephropathies
- First Posted Date
- 2015-01-21
- Last Posted Date
- 2016-08-26
- Lead Sponsor
- Assistance Publique - Hôpitaux de Paris
- Target Recruit Count
- 26
- Registration Number
- NCT02343094
- Locations
- 🇫🇷
Hôpital Necker Enfants Malades, Paris, France
Drug Development Updates
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