Page last updated: 2024-10-19

phenylacetic acid and Argininosuccinate Synthase Deficiency Disease

phenylacetic acid has been researched along with Argininosuccinate Synthase Deficiency Disease in 3 studies

phenylacetic acid : A monocarboxylic acid that is toluene in which one of the hydrogens of the methyl group has been replaced by a carboxy group.

Research Excerpts

Excerpt	Relevance	Reference
"We report the results of a 25-year, open-label, uncontrolled study of sodium phenylacetate and sodium benzoate therapy (Ammonul, Ucyclyd Pharma) in 299 patients with urea-cycle disorders in whom there were 1181 episodes of acute hyperammonemia."	5.12	Survival after treatment with phenylacetate and benzoate for urea-cycle disorders. ( Berry, GT; Berry, SA; Brusilow, SW; Enns, GM; Hamosh, A; Rhead, WJ, 2007)
"Citrullinemia is a urea cycle defect requiring long-term care with nutritional and pharmacological management."	1.38	30-year follow-up of a patient with classic citrullinemia. ( Brunetti-Pierri, N; Craigen, WJ; Lamance, KM; Lewis, RA, 2012)

Research

Studies (3)

Timeframe	Studies, this research(%)	All Research%
pre-1990	0 (0.00)	18.7374
1990's	0 (0.00)	18.2507
2000's	2 (66.67)	29.6817
2010's	1 (33.33)	24.3611
2020's	0 (0.00)	2.80

Authors

Authors	Studies
Brunetti-Pierri, N	1
Lamance, KM	1
Lewis, RA	1
Craigen, WJ	1
Scaglia, F	1
Carter, S	1
O'Brien, WE	1
Lee, B	1
Enns, GM	1
Berry, SA	1
Berry, GT	1
Rhead, WJ	1
Brusilow, SW	1
Hamosh, A	1

Clinical Trials (1)

Trial Overview

Trial			Phase	Enrollment	Study Type	Start Date	Status
A Phase 2, Open-Label, Switch-Over, Dose-Escalation Study of the Safety and Tolerability of HPN-100 Compared to Buphenyl® (Sodium Phenylbutyrate) in Patients With Urea Cycle Disorders[NCT00551200]			Phase 2	14 participants (Actual)	Interventional	2007-10-31	Completed
[information is prepared from clinicaltrials.gov, extracted Sep-2024]

Trial Outcomes

Number of Subjects Experienced Adverse Events

(NCT00551200)
Timeframe: during the period on 100% Buphenyl (up to 4 weeks) or HPN-100 (up to 10 weeks)

Intervention	participants (Number)
Buphenyl	7
HPN-100	5

Number of Subjects Experienced Serious Adverse Events

(NCT00551200)
Timeframe: during the period subjects on 100% Buphenyl (up to 4 weeks) or HPN-100 (up to 10 weeks)

Intervention	participants (Number)
Buphenyl	1
HPN-100	0

Drug Preference for HPN-100 or Buphenyl® (as Assessed by Global Preference Question)

(NCT00551200)
Timeframe: End of Study

Intervention	participants (Number)
	prefer Buphenyl	prefer HPN-100
Buphenyl to HPN-100	1	9

Pharmacokinetics (Plasma and Urine PK Parameters of Study Drugs and Their Metabolites)

measured AUC0-24 (Area under the curve from time 0 (pre-dose) to 24 hours) for each metabolite in plasma. Data were collected at 30 minutes and 1, 2, 4, 5, 6, 8, 10, 12, and 24 hours post-first dose. (NCT00551200)
Timeframe: At steady state (1 week) on each medication (Buphenyl® alone, HPN-100 alone)

Intervention	μg*h/mL (Mean)
	AUC0-24 PBA (phenylbutyrate) in plasma	AUC0-24 PAA (phenylacetate) in plasma	AUC0-24 PAGN (phenylacetylglutamine) in plasma
HPN-100 Steady State	540	575	1098
NaPBA Steady State	740	596	1133

Venous Ammonia Levels at the Peak and Mean TNUAC Time-normalized Area Under the Curve)

Data were collected at pre-first dose and at 30 minutes and 1, 2, 4, 5, 6, 8, 10, 12, and 24 hours post first dose. (NCT00551200)
Timeframe: At steady state (1 week) on each medication (Buphenyl® alone, HPN-100 alone), and at steady state (1 week) after each dose escalation

Intervention	μmol/L (Mean)
	in peak	in TNAUC (time-normalized area under the curve)
HPN-100 Steady State	56.3	26.5
NaPBA Steady State	79.1	38.4

Trials

1 trial available for phenylacetic acid and Argininosuccinate Synthase Deficiency Disease

Article	Year
Survival after treatment with phenylacetate and benzoate for urea-cycle disorders. The New England journal of medicine, 2007, May-31, Volume: 356, Issue:22 Topics: Adolescent; Adult; Age Factors; Age of Onset; Amino Acid Metabolism, Inborn Errors; Ammonia; Carbamo	2007

Other Studies

2 other studies available for phenylacetic acid and Argininosuccinate Synthase Deficiency Disease

Article	Year
30-year follow-up of a patient with classic citrullinemia. Molecular genetics and metabolism, 2012, Volume: 106, Issue:2 Topics: Adult; Cardiomyopathy, Hypertrophic; Cataract; Citrullinemia; Female; Follow-Up Studies; Histone Dea	2012
Effect of alternative pathway therapy on branched chain amino acid metabolism in urea cycle disorder patients. Molecular genetics and metabolism, 2004, Volume: 81 Suppl 1 Topics: Adolescent; Adult; Amino Acid Metabolism, Inborn Errors; Amino Acids, Branched-Chain; Citrullinemia;	2004