phenylacetic acid has been researched along with Ornithine Carbamoyltransferase Deficiency Disease in 10 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phenylacetic acid : A monocarboxylic acid that is toluene in which one of the hydrogens of the methyl group has been replaced by a carboxy group.
Ornithine Carbamoyltransferase Deficiency Disease: An inherited urea cycle disorder associated with deficiency of the enzyme ORNITHINE CARBAMOYLTRANSFERASE, transmitted as an X-linked trait and featuring elevations of amino acids and ammonia in the serum. Clinical features, which are more prominent in males, include seizures, behavioral alterations, episodic vomiting, lethargy, and coma. (Menkes, Textbook of Child Neurology, 5th ed, pp49-50)
| 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) |
| "Patients with neonatal urea cycle defects (UCDs) typically experience severe hyperammonemia during the first days of life, which results in serious neurological injury or death." | 3.88 | Prenatal treatment of ornithine transcarbamylase deficiency. ( Akula, VP; Alcorn, D; Benitz, WE; Bernstein, JA; Berquist, WE; Blumenfeld, YJ; Castillo, RO; Concepcion, W; Cowan, TM; Cox, KL; Cusmano, K; Enns, GM; Esquivel, CO; Hintz, SR; Homeyer, M; Hudgins, L; Hurwitz, M; Lyell, DJ; Palma, JP; Schelley, S; Summar, ML; Wilnai, Y, 2018) |
| "Girls with symptomatic ornithine transcarbamylase deficiency who are treated with drugs that activate new pathways of waste-nitrogen excretion have fewer hyperammonemic episodes and a reduced risk of further cognitive decline." | 2.68 | Long-term treatment of girls with ornithine transcarbamylase deficiency. ( Bassett, SS; Brusilow, SW; Clissold, DB; Maestri, NE, 1996) |
| " Ambiguous medical prescriptions and inadequate cross-checking of drug dosage by physicians, nurses and pharmacists were the main causes of these incidents." | 1.31 | Three cases of intravenous sodium benzoate and sodium phenylacetate toxicity occurring in the treatment of acute hyperammonaemia. ( Boyadjiev, SA; Brusilow, SW; Geraghty, MT; Praphanphoj, V; Waber, LJ, 2000) |
| " In the child, when the benzoate/phenylacetate dosage was increased from 200 to 375 mg/kg/day each, feeding decreased." | 1.27 | Effect of sodium benzoate and sodium phenylacetate on brain serotonin turnover in the ornithine transcarbamylase-deficient sparse-fur mouse. ( Batshaw, ML; Coyle, JT; Hyman, SL; Mellits, ED; Quaskey, S; Qureshi, IA; Robinson, MB, 1988) |
| Timeframe | Studies, this research(%) | All Research% |
|---|---|---|
| pre-1990 | 2 (20.00) | 18.7374 |
| 1990's | 2 (20.00) | 18.2507 |
| 2000's | 3 (30.00) | 29.6817 |
| 2010's | 3 (30.00) | 24.3611 |
| 2020's | 0 (0.00) | 2.80 |
| Authors | Studies |
|---|---|
| Wilnai, Y | 1 |
| Blumenfeld, YJ | 1 |
| Cusmano, K | 1 |
| Hintz, SR | 1 |
| Alcorn, D | 1 |
| Benitz, WE | 1 |
| Berquist, WE | 1 |
| Bernstein, JA | 1 |
| Castillo, RO | 1 |
| Concepcion, W | 1 |
| Cowan, TM | 1 |
| Cox, KL | 1 |
| Lyell, DJ | 1 |
| Esquivel, CO | 1 |
| Homeyer, M | 1 |
| Hudgins, L | 1 |
| Hurwitz, M | 1 |
| Palma, JP | 1 |
| Schelley, S | 1 |
| Akula, VP | 1 |
| Summar, ML | 1 |
| Enns, GM | 2 |
| Nagasaka, H | 1 |
| Yorifuji, T | 1 |
| Kobayashi, K | 1 |
| Takikawa, H | 1 |
| Komatsu, H | 1 |
| Inui, A | 1 |
| Fujisawa, T | 1 |
| Miida, T | 1 |
| Tsukahara, H | 1 |
| Takatani, T | 1 |
| Hayashi, H | 1 |
| Houston, B | 1 |
| Reiss, KA | 1 |
| Merlo, C | 1 |
| Scaglia, F | 1 |
| Carter, S | 1 |
| O'Brien, WE | 1 |
| Lee, B | 1 |
| Berry, SA | 1 |
| Berry, GT | 1 |
| Rhead, WJ | 1 |
| Brusilow, SW | 4 |
| Hamosh, A | 1 |
| Danney, M | 1 |
| Waber, LJ | 2 |
| Batshaw, M | 1 |
| Burton, B | 1 |
| Levitsky, L | 1 |
| Roth, K | 1 |
| McKeethren, C | 1 |
| Ward, J | 1 |
| Maestri, NE | 1 |
| Clissold, DB | 1 |
| Bassett, SS | 1 |
| Thoene, JG | 1 |
| Praphanphoj, V | 1 |
| Boyadjiev, SA | 1 |
| Geraghty, MT | 1 |
| Batshaw, ML | 1 |
| Hyman, SL | 1 |
| Coyle, JT | 1 |
| Robinson, MB | 1 |
| Qureshi, IA | 1 |
| Mellits, ED | 1 |
| Quaskey, S | 1 |
| 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] | |||||||
(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 |
(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 |
(NCT00551200)
Timeframe: End of Study
| Intervention | participants (Number) | |
|---|---|---|
| prefer Buphenyl | prefer HPN-100 | |
| Buphenyl to HPN-100 | 1 | 9 |
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 |
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 |
1 review available for phenylacetic acid and Ornithine Carbamoyltransferase Deficiency Disease
| Article | Year |
|---|---|
Treatment of urea cycle disorders.
Topics: Amino Acid Metabolism, Inborn Errors; Ammonia; Antimetabolites; Child, Preschool; Drug Combinations; | 1999 |
2 trials available for phenylacetic acid and Ornithine Carbamoyltransferase Deficiency Disease
| Article | Year |
|---|---|
Survival after treatment with phenylacetate and benzoate for urea-cycle disorders.
Topics: Adolescent; Adult; Age Factors; Age of Onset; Amino Acid Metabolism, Inborn Errors; Ammonia; Carbamo | 2007 |
Long-term treatment of girls with ornithine transcarbamylase deficiency.
Topics: Adolescent; Amino Acid Metabolism, Inborn Errors; Ammonia; Benzoates; Benzoic Acid; Brain Diseases; | 1996 |
7 other studies available for phenylacetic acid and Ornithine Carbamoyltransferase Deficiency Disease
| Article | Year |
|---|---|
Prenatal treatment of ornithine transcarbamylase deficiency.
Topics: Ammonia; Drug Combinations; Female; Glutamine; Humans; Hyperammonemia; Infant, Newborn; Male; Mutati | 2018 |
Favorable effect of 4-phenylacetate on liver functions attributable to enhanced bile salt export pump expression in ornithine transcarbamylase-deficient children.
Topics: Adenosine Triphosphatases; Adolescent; Arginine; ATP Binding Cassette Transporter, Subfamily B, Memb | 2010 |
Healthy, but comatose.
Topics: Adult; Brain Edema; Carbohydrates; Enteral Nutrition; Glucose; Humans; Hyperammonemia; Insulin; Male | 2011 |
Effect of alternative pathway therapy on branched chain amino acid metabolism in urea cycle disorder patients.
Topics: Adolescent; Adult; Amino Acid Metabolism, Inborn Errors; Amino Acids, Branched-Chain; Citrullinemia; | 2004 |
Treatment of episodic hyperammonemia in children with inborn errors of urea synthesis.
Topics: Amino Acid Metabolism, Inborn Errors; Ammonia; Arginine; Argininosuccinate Synthase; Benzoates; Benz | 1984 |
Three cases of intravenous sodium benzoate and sodium phenylacetate toxicity occurring in the treatment of acute hyperammonaemia.
Topics: Child; Child, Preschool; Drug Overdose; Fatal Outcome; Female; Humans; Injections, Intravenous; Male | 2000 |
Effect of sodium benzoate and sodium phenylacetate on brain serotonin turnover in the ornithine transcarbamylase-deficient sparse-fur mouse.
Topics: Administration, Oral; Amino Acid Metabolism, Inborn Errors; Ammonia; Animals; Benzoates; Benzoic Aci | 1988 |