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.
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- 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
- Associated Therapies
- -
BSEP Function Rescue During Childhood Inhereditary Cholestatic Diseases
- Conditions
- Interventions
- First Posted Date
- 2020-08-31
- Last Posted Date
- 2023-02-09
- Lead Sponsor
- Children's Hospital of Fudan University
- Registration Number
- NCT04531878
PBA Use for Treatment of ATF6-/- Patients
- First Posted Date
- 2019-08-01
- Last Posted Date
- 2024-10-18
- Lead Sponsor
- Columbia University
- Target Recruit Count
- 2
- Registration Number
- NCT04041232
- Locations
- 🇺🇸
Edward S. Harkness Eye Institute, New York, New York, United States
Buphenyl Therapy for Byler's Disease
- Conditions
- First Posted Date
- 2013-02-06
- Last Posted Date
- 2013-02-06
- Lead Sponsor
- University of Pittsburgh
- Registration Number
- NCT01784718
Phenylbutyrate Therapy for Maple Syrup Urine Disease
- Conditions
- Interventions
- First Posted Date
- 2012-02-08
- Last Posted Date
- 2019-03-19
- Lead Sponsor
- Brendan Lee
- Target Recruit Count
- 20
- Registration Number
- NCT01529060
- Locations
- 🇺🇸
Baylor College of Medicine, Houston, Texas, United States
A Study of Glyceryl Tri-(4-phenylbutyrate) (GT4P)
- First Posted Date
- 2009-09-16
- Last Posted Date
- 2024-06-20
- Lead Sponsor
- Amgen
- Target Recruit Count
- 24
- Registration Number
- NCT00977600
- Locations
- 🇺🇦
Medical Sanitary Division #2, Kharkiv, Ukraine
Phase I and Pharmacokinetic Trial of Phenylbutyrate Given as a Continuous Infusion in Pediatric Patients With Refractory Malignancy
- Conditions
- First Posted Date
- 2002-12-10
- Last Posted Date
- 2008-03-04
- Lead Sponsor
- National Cancer Institute (NCI)
- Target Recruit Count
- 35
- Registration Number
- NCT00001565
- Locations
- 🇺🇸
National Cancer Institute (NCI), Bethesda, Maryland, United States
Phenylbutyrate to Treat Children With Progressive or Recurrent Brain Tumors
- Conditions
- First Posted Date
- 2000-11-08
- Last Posted Date
- 2008-03-04
- Lead Sponsor
- National Cancer Institute (NCI)
- Target Recruit Count
- 120
- Registration Number
- NCT00006450
- Locations
- 🇺🇸
National Cancer Institute (NCI), Bethesda, Maryland, United States
Drug Development Updates
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