ammonium hydroxide has been researched along with Urea Cycle Disorders, Inborn in 53 studies
azane : Saturated acyclic nitrogen hydrides having the general formula NnHn+2.
Urea Cycle Disorders, Inborn: Rare congenital metabolism disorders of the urea cycle. The disorders are due to mutations that result in complete (neonatal onset) or partial (childhood or adult onset) inactivity of an enzyme, involved in the urea cycle. Neonatal onset results in clinical features that include irritability, vomiting, lethargy, seizures, NEONATAL HYPOTONIA; RESPIRATORY ALKALOSIS; HYPERAMMONEMIA; coma, and death. Survivors of the neonatal onset and childhood/adult onset disorders share common risks for ENCEPHALOPATHIES, METABOLIC, INBORN; and RESPIRATORY ALKALOSIS due to HYPERAMMONEMIA.
| Excerpt | Relevance | Reference |
|---|---|---|
| " Urea cycle disorders with hyperammonemia remain difficult to treat and eventually necessitate liver transplantation." | 8.12 | Glutaminase 2 knockdown reduces hyperammonemia and associated lethality of urea cycle disorder mouse model. ( Burczynski, ME; Chen, H; Cheng, X; Halasz, G; Kim, S; Lin, AZ; Mao, X; Murphy, AJ; Na, E; Okamoto, H; Sleeman, MW, 2022) |
| "The urea cycle disorders are a group of inherited biochemical diseases caused by a complete or partial deficiency of any one of the enzymes or transport proteins required to convert toxic ammonia into urea and to produce arginine and citrulline." | 6.58 | Inborn Errors of Metabolism with Hyperammonemia: Urea Cycle Defects and Related Disorders. ( Mew, NA; Summar, ML, 2018) |
| "Leukoencephalopathy was resolved following the administration of L-arginine and lactulose with a decrease in plasma ammonia levels and glutamine-glutamate peak on magnetic resonance spectroscopy." | 5.72 | Reversible Leukoencephalopathy in a Man with Childhood-onset Hyperornithinemia-Hyperammonemia-Homocitrullinuria Syndrome. ( Fukuyama, T; Hoshino, Y; Kaneko, T; Kodaira, M; Matsuno, A; Sekijima, Y; Takano, K; Yazaki, M, 2022) |
| " Urea cycle disorders with hyperammonemia remain difficult to treat and eventually necessitate liver transplantation." | 4.12 | Glutaminase 2 knockdown reduces hyperammonemia and associated lethality of urea cycle disorder mouse model. ( Burczynski, ME; Chen, H; Cheng, X; Halasz, G; Kim, S; Lin, AZ; Mao, X; Murphy, AJ; Na, E; Okamoto, H; Sleeman, MW, 2022) |
| "Transmembrane pH gradient poly(isoprene)-block-poly(ethylene glycol) (PI-b-PEG) polymersomes were investigated for their potential use in the detoxification of ammonia, a metabolite that is excessively present in patients suffering from urea cycle disorders and advanced liver diseases, and which causes neurotoxic effects (e." | 3.96 | Ammonia uptake by transmembrane pH gradient poly(isoprene)-block-poly(ethylene glycol) polymersomes. ( Altmann, KH; Bao, Y; Hebels, ER; Leroux, JC; Schmidt, AC; Stoessel, B; Weitzel, C, 2020) |
| "UCD patients (n=26) ages 2months through 17years were treated with GPB and sodium phenylbutyrate (NaPBA) in two short-term, open-label crossover studies, which compared 24-hour ammonia exposure (AUC0-24) and glutamine levels during equivalent steady-state dosing of GPB and sodium phenylbutyrate (NaPBA)." | 2.79 | Glycerol phenylbutyrate treatment in children with urea cycle disorders: pooled analysis of short and long-term ammonia control and outcomes. ( Berry, SA; Coakley, DF; Diaz, GA; Lee, B; Lemons, C; Lichter-Konecki, U; McCandless, SE; Mokhtarani, M; Nagamani, SC; Rhead, W; Scharschmidt, BF; Smith, W, 2014) |
| "We have analyzed pharmacokinetic data for glycerol phenylbutyrate (also GT4P or HPN-100) and sodium phenylbutyrate with respect to possible dosing biomarkers in patients with urea cycle disorders (UCD)." | 2.77 | Urinary phenylacetylglutamine as dosing biomarker for patients with urea cycle disorders. ( Bart, S; Bartholomew, D; Bartley, J; Berquist, W; Berry, SA; Cederbaum, S; Coakley, DF; Diaz, GA; Dickinson, K; Dorrani, N; Feigenbaum, A; Gallagher, R; Harding, CO; Korson, MS; Kronn, D; Lee, B; Lemons, C; Lichter-Konecki, U; Longo, N; McCandless, SE; Merritt, JL; Mokhtarani, M; Moors, TL; Rhead, W; Scharschmidt, BF; Smith, W; Sreenath-Nagamani, S; Summar, M; Vockley, J; Zori, R, 2012) |
| " No statistically significant differences were observed in plasma phenylacetic acid and PAGN exposure during dosing with GPB vs." | 2.76 | Ammonia control in children with urea cycle disorders (UCDs); phase 2 comparison of sodium phenylbutyrate and glycerol phenylbutyrate. ( Beliveau, M; Diaz, GA; Dickinson, K; Feigenbaum, A; Jomphe, C; Lichter-Konecki, U; Marier, JF; Martinez, A; Mauney, J; Merritt, JL; Mokhtarani, M; Rhead, W; Scharschmidt, B, 2011) |
| " Adverse events were comparable for the two drugs; 2 subjects experienced hyperammonemic events on NaPBA while none occurred on GPB." | 2.75 | Phase 2 comparison of a novel ammonia scavenging agent with sodium phenylbutyrate in patients with urea cycle disorders: safety, pharmacokinetics and ammonia control. ( Beliveau, M; Berry, SA; Diaz, GA; Dickinson, K; Gargosky, S; Lee, B; Marier, JF; Martinez, A; Mauney, J; Mian, A; Mokhtarani, M; Rhead, W; Scharschmidt, BF; Shchelochkov, O, 2010) |
| "The urea cycle disorders are a group of inherited biochemical diseases caused by a complete or partial deficiency of any one of the enzymes or transport proteins required to convert toxic ammonia into urea and to produce arginine and citrulline." | 2.58 | Inborn Errors of Metabolism with Hyperammonemia: Urea Cycle Defects and Related Disorders. ( Mew, NA; Summar, ML, 2018) |
| "Gene therapy for OTC deficiency is effective in animals, and work is ongoing to improve persistence and safety." | 2.45 | Ammonia toxicity and its prevention in inherited defects of the urea cycle. ( Walker, V, 2009) |
| "The most prevalent UCD detected were ornithine transcarbamylase deficiency, followed by citrullinemia type 1, hyperargininemia, carbamoyl phosphate synthase 1 deficiency, and argininosuccinic aciduria." | 1.72 | Clinical findings of patients with hyperammonemia affected by urea cycle disorders with hepatic encephalopathy. ( de Moura Coelho, D; Dos Reis, BG; Faverzani, JL; Lopes, FF; Ribas, GS; Sitta, A; Vargas, CR; Wajner, M, 2022) |
| "Leukoencephalopathy was resolved following the administration of L-arginine and lactulose with a decrease in plasma ammonia levels and glutamine-glutamate peak on magnetic resonance spectroscopy." | 1.72 | Reversible Leukoencephalopathy in a Man with Childhood-onset Hyperornithinemia-Hyperammonemia-Homocitrullinuria Syndrome. ( Fukuyama, T; Hoshino, Y; Kaneko, T; Kodaira, M; Matsuno, A; Sekijima, Y; Takano, K; Yazaki, M, 2022) |
| " Glycerol phenylbutyrate (GPB) is safe and effective in reducing ammonia levels in patients with UCD above 2 months of age." | 1.62 | Glycerol phenylbutyrate efficacy and safety from an open label study in pediatric patients under 2 months of age with urea cycle disorders. ( Ah Mew, N; Bannick, AA; Berry, SA; Canavan, C; Conway, RL; Diaz, GA; Hainline, B; Inbar-Feigenberg, M; Kok, T; Lichter-Konecki, U; Longo, N; McCandless, SE; Porter, MH; Schulze, A; Vescio, T; Zori, R, 2021) |
| "Urea cycle disorders are congenital metabolism errors that affect ammonia elimination." | 1.56 | Perioperative management of children with urea cycle disorders. ( Del Río, C; Martín-Hernández, E; Quijada-Fraile, P; Rubio, P; Ruiz, A, 2020) |
| "Hyperammonemia was diagnosed in all cases during the evaluation of altered mental status, with 22% presenting with seizures." | 1.56 | A retrospective study of adult patients with noncirrhotic hyperammonemia. ( Baker, JJ; Barkoudah, E; Berry, GT; Khoury, CC; Krier, JB; Lin, AP; Mogensen, KM; Peake, RW; Sahai, I; Stergachis, AB; Sweetser, DA, 2020) |
| "In nonalcoholic fatty liver disease (NAFLD), fibrosis is the most important factor contributing to NAFLD-associated morbidity and mortality." | 1.56 | Ammonia Scavenging Prevents Progression of Fibrosis in Experimental Nonalcoholic Fatty Liver Disease. ( Andreola, F; Davies, N; De Chiara, F; Gracia-Sancho, J; Habtesion, A; Hall, A; Jalan, R; Jones, H; Leslie, J; Manicardi, N; Mann, J; Mookerjee, RP; Oakley, F; Paish, HL; Reed, LH; Rombouts, K; Thomsen, KL; Watson, AA, 2020) |
| "Primary hyperammonemic encephalopathy due to urea cycle disorders (UCD) typically manifests with episodic unresponsiveness and this clinical entity is not often included in the differential diagnosis of presumed non-convulsive status epilepticus (NCSE)." | 1.48 | Encephalopathy mimicking non-convulsive status Epilepticus. ( Al-Busaidi, A; Al-Busaidi, M; Al-Murshedi, F; Nandhagopal, R, 2018) |
| " Safety endpoints included adverse events, hyperammonemic crises (HACs), and growth and development." | 1.46 | Safety and efficacy of glycerol phenylbutyrate for management of urea cycle disorders in patients aged 2months to 2years. ( Berry, SA; Diaz, GA; Ficicioglu, C; Harding, CO; Lichter-Konecki, U; Longo, N; McCandless, SE; Robinson, B; Smith, WE; Vockley, J; Zori, R, 2017) |
| "Urea cycle disorders are inborn errors of metabolism that, in rare cases, can present for the first time in adulthood." | 1.42 | Urea cycle disorders: a life-threatening yet treatable cause of metabolic encephalopathy in adults. ( Blair, NF; Cremer, PD; Tchan, MC, 2015) |
| "Hyperammonemia is a metabolic derangement that can be potentially fatal." | 1.39 | A rare case of hyperammonemia complication of high-protein parenteral nutrition. ( Bhat, Z; Cadnapaphorncai, P; Kahlon, R; Pillai, U; Sondheimer, J, 2013) |
| "Specific treatments of hyperammonemia are rarely used in liver failure when compared with urea cycle defect even though use of ammonia scavengers may help to decrease ammonemia." | 1.38 | Threshold for toxicity from hyperammonemia in critically ill children. ( Alvarez, F; Cousineau, J; Ducruet, T; Jouvet, P; Lambert, M; Mitchell, G; Nelson, J; Ozanne, B; Phan, V, 2012) |
| "The most common UCD was ornithine transcarbamylase deficiency (OTCD), which accounted for 116 out of 177 patients." | 1.38 | Long-term outcome and intervention of urea cycle disorders in Japan. ( Endo, F; Horikawa, R; Kasahara, M; Kido, J; Matsuo, M; Mitsubuchi, H; Nakamura, K; Ohura, T; Shigematsu, Y; Takayanagi, M; Yorifuji, T; Yoshino, M, 2012) |
| Timeframe | Studies, this research(%) | All Research% |
|---|---|---|
| pre-1990 | 0 (0.00) | 18.7374 |
| 1990's | 0 (0.00) | 18.2507 |
| 2000's | 1 (1.89) | 29.6817 |
| 2010's | 33 (62.26) | 24.3611 |
| 2020's | 19 (35.85) | 2.80 |
| Authors | Studies |
|---|---|
| Hajaj, E | 1 |
| Sciacovelli, M | 1 |
| Frezza, C | 1 |
| Erez, A | 1 |
| Mao, X | 1 |
| Chen, H | 1 |
| Lin, AZ | 1 |
| Kim, S | 1 |
| Burczynski, ME | 1 |
| Na, E | 1 |
| Halasz, G | 1 |
| Sleeman, MW | 1 |
| Murphy, AJ | 1 |
| Okamoto, H | 1 |
| Cheng, X | 1 |
| Lopes, FF | 1 |
| Sitta, A | 1 |
| de Moura Coelho, D | 1 |
| Ribas, GS | 1 |
| Faverzani, JL | 1 |
| Dos Reis, BG | 1 |
| Wajner, M | 1 |
| Vargas, CR | 1 |
| Lichter-Konecki, U | 10 |
| Sanz, JH | 1 |
| McCarter, R | 1 |
| Imbard, A | 1 |
| Bouchereau, J | 1 |
| Arnoux, JB | 2 |
| Brassier, A | 2 |
| Schiff, M | 1 |
| Bérat, CM | 1 |
| Pontoizeau, C | 2 |
| Benoist, JF | 1 |
| Josse, C | 1 |
| Montestruc, F | 1 |
| de Lonlay, P | 2 |
| Sacchini, M | 1 |
| Procopio, E | 1 |
| Pochiero, F | 1 |
| Scaturro, G | 1 |
| Daniotti, M | 1 |
| Donati, MA | 1 |
| Schmidt, AC | 1 |
| Hebels, ER | 1 |
| Weitzel, C | 1 |
| Stoessel, B | 1 |
| Bao, Y | 1 |
| Altmann, KH | 1 |
| Leroux, JC | 1 |
| Roda, C | 1 |
| Vignolo-Diard, P | 1 |
| Habarou, F | 1 |
| Barbier, V | 1 |
| Grisel, C | 1 |
| Abi-Warde, MT | 1 |
| Boddaert, N | 1 |
| Kuster, A | 1 |
| Servais, A | 1 |
| Kaminska, A | 1 |
| Hennequin, C | 1 |
| Dupic, L | 1 |
| Lesage, F | 1 |
| Touati, G | 1 |
| Valayannopoulos, V | 1 |
| Chadefaux-Vekemans, B | 1 |
| Oualha, M | 1 |
| Eisermann, M | 1 |
| Ottolenghi, C | 1 |
| Ikeri, K | 1 |
| Cardona, VQ | 1 |
| Hagan-Brown, A | 1 |
| Young, M | 1 |
| Schneider, M | 1 |
| Menkiti, O | 1 |
| Del Río, C | 1 |
| Martín-Hernández, E | 1 |
| Ruiz, A | 1 |
| Quijada-Fraile, P | 1 |
| Rubio, P | 1 |
| Stergachis, AB | 1 |
| Mogensen, KM | 1 |
| Khoury, CC | 1 |
| Lin, AP | 1 |
| Peake, RW | 1 |
| Baker, JJ | 1 |
| Barkoudah, E | 1 |
| Sahai, I | 1 |
| Sweetser, DA | 1 |
| Berry, GT | 1 |
| Krier, JB | 1 |
| Kenneson, A | 1 |
| Singh, RH | 1 |
| Häberle, J | 3 |
| Nitzahn, M | 1 |
| Lipshutz, GS | 1 |
| Longo, N | 6 |
| Diaz, GA | 10 |
| Schulze, A | 3 |
| Inbar-Feigenberg, M | 1 |
| Conway, RL | 1 |
| Bannick, AA | 1 |
| McCandless, SE | 8 |
| Zori, R | 5 |
| Hainline, B | 1 |
| Ah Mew, N | 2 |
| Canavan, C | 1 |
| Vescio, T | 1 |
| Kok, T | 1 |
| Porter, MH | 1 |
| Berry, SA | 9 |
| Kido, J | 2 |
| Matsumoto, S | 1 |
| Nakajima, Y | 1 |
| Wada, Y | 1 |
| Mochizuki, N | 1 |
| Murayama, K | 1 |
| Lee, T | 1 |
| Mochizuki, H | 1 |
| Watanabe, Y | 1 |
| Horikawa, R | 2 |
| Kasahara, M | 2 |
| Nakamura, K | 2 |
| Vinegrad, N | 1 |
| Staretz-Chacham, O | 1 |
| Barski, L | 1 |
| Bartal, C | 1 |
| Hoshino, Y | 1 |
| Kodaira, M | 1 |
| Matsuno, A | 1 |
| Kaneko, T | 1 |
| Fukuyama, T | 1 |
| Takano, K | 1 |
| Yazaki, M | 1 |
| Sekijima, Y | 1 |
| Jiang, Y | 1 |
| Almannai, M | 1 |
| Sutton, VR | 1 |
| Sun, Q | 1 |
| Elsea, SH | 1 |
| Smith, WE | 1 |
| Harding, CO | 6 |
| Ficicioglu, C | 1 |
| Robinson, B | 1 |
| Vockley, J | 3 |
| Meyburg, J | 1 |
| Opladen, T | 1 |
| Spiekerkötter, U | 1 |
| Schlune, A | 1 |
| Schenk, JP | 1 |
| Schmidt, J | 1 |
| Weitz, J | 1 |
| Okun, J | 1 |
| Bürger, F | 1 |
| Omran, TB | 1 |
| Abdoh, G | 1 |
| Al Rifai, H | 1 |
| Monavari, A | 1 |
| Konstantopoulou, V | 1 |
| Kölker, S | 1 |
| Yudkoff, M | 1 |
| Hoffmann, GF | 1 |
| Celik, M | 1 |
| Akdeniz, O | 1 |
| Ozgun, N | 1 |
| Nandhagopal, R | 1 |
| Al-Murshedi, F | 1 |
| Al-Busaidi, M | 1 |
| Al-Busaidi, A | 1 |
| Summar, ML | 1 |
| Mew, NA | 1 |
| Hediger, N | 1 |
| Landolt, MA | 1 |
| Diez-Fernandez, C | 1 |
| Huemer, M | 1 |
| Soria, LR | 1 |
| Brunetti-Pierri, N | 1 |
| De Chiara, F | 1 |
| Thomsen, KL | 1 |
| Habtesion, A | 1 |
| Jones, H | 1 |
| Davies, N | 1 |
| Gracia-Sancho, J | 1 |
| Manicardi, N | 1 |
| Hall, A | 1 |
| Andreola, F | 1 |
| Paish, HL | 1 |
| Reed, LH | 1 |
| Watson, AA | 1 |
| Leslie, J | 1 |
| Oakley, F | 1 |
| Rombouts, K | 1 |
| Mookerjee, RP | 1 |
| Mann, J | 1 |
| Jalan, R | 1 |
| Cooper, AJ | 2 |
| Kuhara, T | 2 |
| Rhead, W | 7 |
| Smith, W | 6 |
| Lemons, C | 4 |
| Nagamani, SC | 3 |
| Coakley, DF | 6 |
| Mokhtarani, M | 8 |
| Scharschmidt, BF | 7 |
| Lee, B | 7 |
| Blair, NF | 1 |
| Cremer, PD | 1 |
| Tchan, MC | 1 |
| Feigenbaum, A | 5 |
| Le Mons, C | 2 |
| Bartley, JA | 1 |
| Berquist, W | 4 |
| Gallagher, R | 3 |
| Bartholomew, D | 3 |
| Korson, MS | 3 |
| Cederbaum, S | 3 |
| Wong, D | 1 |
| Merritt, JL | 4 |
| Vockley, G | 2 |
| Kronn, D | 3 |
| Summar, M | 2 |
| Milikien, DA | 1 |
| Marino, M | 2 |
| Bartley, J | 3 |
| Gallagher, RC | 1 |
| Rowell, R | 1 |
| Singh, S | 1 |
| Pal, S | 1 |
| Dubrey, SW | 1 |
| Brannelly, NT | 1 |
| Hamilton-Shield, JP | 1 |
| Killard, AJ | 1 |
| Açıkalın, A | 1 |
| Dişel, NR | 1 |
| De Las Heras, J | 1 |
| Aldámiz-Echevarría, L | 1 |
| Martínez-Chantar, ML | 1 |
| Delgado, TC | 1 |
| Walker, V | 2 |
| Mian, A | 1 |
| Shchelochkov, O | 1 |
| Marier, JF | 2 |
| Beliveau, M | 2 |
| Mauney, J | 4 |
| Dickinson, K | 5 |
| Martinez, A | 2 |
| Gargosky, S | 1 |
| Bezinover, D | 1 |
| Douthitt, L | 1 |
| McQuillan, PM | 1 |
| Khan, A | 1 |
| Dalal, P | 1 |
| Stene, J | 1 |
| Uemura, T | 1 |
| Kadry, Z | 1 |
| Janicki, PK | 1 |
| Inoue, Y | 1 |
| Ohse, M | 1 |
| Krasnikov, BF | 1 |
| Serrano, M | 1 |
| Ormazábal, A | 1 |
| Vilaseca, MA | 1 |
| Lambruschini, N | 1 |
| Garcia-Romero, R | 1 |
| Meavilla, S | 1 |
| Perez-Dueñas, B | 1 |
| Pineda, M | 1 |
| Garcia-Cazorla, A | 1 |
| Campistol, J | 1 |
| Artuch, R | 1 |
| Jomphe, C | 1 |
| Scharschmidt, B | 1 |
| Ozanne, B | 1 |
| Nelson, J | 1 |
| Cousineau, J | 1 |
| Lambert, M | 1 |
| Phan, V | 1 |
| Mitchell, G | 1 |
| Alvarez, F | 1 |
| Ducruet, T | 1 |
| Jouvet, P | 1 |
| Mitsubuchi, H | 1 |
| Ohura, T | 1 |
| Takayanagi, M | 1 |
| Matsuo, M | 1 |
| Yoshino, M | 1 |
| Shigematsu, Y | 1 |
| Yorifuji, T | 1 |
| Endo, F | 1 |
| Pillai, U | 1 |
| Kahlon, R | 1 |
| Sondheimer, J | 1 |
| Cadnapaphorncai, P | 1 |
| Bhat, Z | 1 |
| Bart, S | 1 |
| Dorrani, N | 1 |
| Sreenath-Nagamani, S | 1 |
| Moors, TL | 2 |
| Krivitzky, LS | 1 |
| Bart, SA | 1 |
| C S Nagamani, S | 1 |
| Mistry, PK | 1 |
| Krauss, M | 1 |
| Schaller, S | 1 |
| Borchers, S | 1 |
| Findeisen, R | 1 |
| Lippert, J | 1 |
| Kuepfer, L | 1 |
| Cartagena, A | 1 |
| Prasad, AN | 1 |
| Rupar, CA | 1 |
| Strong, M | 1 |
| Tuchman, M | 1 |
| Prasad, C | 1 |
| Calligaris, L | 1 |
| Vidoni, A | 1 |
| Bruno, I | 1 |
| Vidoni, M | 1 |
| Barbi, E | 1 |
| Moors, T | 1 |
| Trial | Phase | Enrollment | Study Type | Start Date | Status | ||
|---|---|---|---|---|---|---|---|
| An Open Label Study of the Safety, Efficacy and Pharmacokinetics of Glycerol Phenylbutyrate (GPB; RAVICTI®) in Pediatric Subjects Under Two Years of Age With Urea Cycle Disorders (UCDs)[NCT02246218] | Phase 4 | 27 participants (Actual) | Interventional | 2014-12-31 | Completed | ||
| A Phase 2, Fixed-Sequence, Open-Label, Switch-Over Study of the Safety and Tolerability of HPN-100 Compared to Sodium Phenylbutyrate in Children 6-17 Years of Age With Urea Cycle Disorders, With a Long-Term Safety Extension[NCT00947544] | Phase 2 | 17 participants (Actual) | Interventional | 2010-03-31 | Completed | ||
| A Phase 3, Randomized, Double-Blind, Cross-Over, Active-Controlled Study of the Efficacy and Safety of HPN-100, Glyceryl Tri-(4-phenylbutyrate), for the Treatment of Adults With Urea Cycle Disorders (Help UCD)[NCT00992459] | Phase 3 | 46 participants (Actual) | Interventional | 2009-10-31 | Completed | ||
| A Phase 3, Open-Label Study of the Safety of HPN-100 for the Long-Term Treatment of Urea Cycle Disorders (Treat UCD)[NCT00947297] | Phase 3 | 60 participants (Actual) | Interventional | 2009-11-30 | Completed | ||
| 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 | ||
| A Switch-Over, Open-Label Study of the Safety, Pharmacokinetics, and Efficacy of HPN-100, Followed by Long-Term Treatment With HPN-100, in Pediatric Subjects Under 6 Years of Age With Urea Cycle Disorders (UCDs)[NCT01347073] | Phase 3 | 23 participants (Actual) | Interventional | 2011-07-31 | Completed | ||
| [information is prepared from clinicaltrials.gov, extracted Sep-2024] | |||||||
"The percentage of participants with successful transition is based on Investigator response to the question, Has transition to 100% RAVICTI been successful with controlled ammonia? For participants < 2 months of age, after a minimum of 24 hours of ammonia monitoring following the first full dose of RAVICTI alone, the participant was effectively transitioned when following conditions were met: no signs and symptoms of hyperammonemia; ammonia level less than 100 μmol/L (without normalization of ammonia, ie, without conversion of values from local laboratories with varying normal ranges to standardized values); and eligible for discharge per Investigator judgment." (NCT02246218)
Timeframe: Up to Day 4
| Intervention | percentage of participants (Number) |
|---|---|
| RAVICTI: Age 0 to < 2 Months | 100 |
"The percentage of participants with successful transition is based on Investigator response to the question, Has transition to 100% RAVICTI been successful with controlled ammonia? For participants 2 months of age and older, after a minimum of 24 hours of ammonia monitoring following the first full dose of RAVICTI alone, the participant was effectively transitioned when following conditions were met: no signs and symptoms of hyperammonemia; ammonia level less than 100 μmol/L (without normalization of ammonia, ie, without conversion of values from local laboratories with varying normal ranges to standardized values); and eligible for discharge per Investigator judgment." (NCT02246218)
Timeframe: Up to Day 4
| Intervention | percentage of participants (Number) |
|---|---|
| RAVICTI: Age 2 Months to < 2 Years | 100 |
(NCT02246218)
Timeframe: Hour 0 and between 4 and 6 hours, 8 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HAC
| Intervention | μg*hr/mL (Mean) |
|---|---|
| RAVICTI: Age 0 to < 2 Months | 1321.18 |
(NCT02246218)
Timeframe: Hour 0 and between 4 and 6 hours, 8 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HAC
| Intervention | μg*hr/mL (Mean) |
|---|---|
| RAVICTI: Age 2 Months to < 2 Years | 246.126 |
(NCT02246218)
Timeframe: Hour 0 and between 4 and 6 hours, 8 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HAC
| Intervention | μg/mL (Mean) |
|---|---|
| RAVICTI: Age 0 to < 2 Months | 115.3 |
(NCT02246218)
Timeframe: Hour 0 and between 4 and 6 hours, 8 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HAC
| Intervention | μg/mL (Mean) |
|---|---|
| RAVICTI: Age 0 to < 2 Months | 98.98 |
(NCT02246218)
Timeframe: Hour 0 and between 4 and 6 hours, 8 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HAC
| Intervention | μg/mL (Mean) |
|---|---|
| RAVICTI: Age 2 Months to < 2 Years | 4.197 |
(NCT02246218)
Timeframe: Hour 0 and between 4 and 6 hours, 8 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HAC
| Intervention | hours (Mean) |
|---|---|
| RAVICTI: Age 0 to < 2 Months | 9.85 |
(NCT02246218)
Timeframe: Hour 0 and between 4 and 6 hours, 8 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HAC
| Intervention | hours (Mean) |
|---|---|
| RAVICTI: Age 2 Months to < 2 Years | 7.422 |
(NCT02246218)
Timeframe: Hour 0 and between 4 and 6 hours, 8 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HAC
| Intervention | μg*hr/mL (Mean) |
|---|---|
| RAVICTI: Age 0 to < 2 Months | 1384.12 |
(NCT02246218)
Timeframe: Hour 0 and between 4 and 6 hours, 8 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HAC
| Intervention | μg*hr/mL (Mean) |
|---|---|
| RAVICTI: Age 2 Months to < 2 Years | 583.835 |
(NCT02246218)
Timeframe: Hour 0 and between 4 and 6 hours, 8 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HAC
| Intervention | μg/mL (Mean) |
|---|---|
| RAVICTI: Age 0 to < 2 Months | 102.1 |
(NCT02246218)
Timeframe: Hour 0 and between 4 and 6 hours, 8 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HAC
| Intervention | μg/mL (Mean) |
|---|---|
| RAVICTI: Age 0 to < 2 Months | 69.39 |
(NCT02246218)
Timeframe: Hour 0 and between 4 and 6 hours, 8 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HAC
| Intervention | μg/mL (Mean) |
|---|---|
| RAVICTI: Age 2 Months to < 2 Years | 20.62 |
(NCT02246218)
Timeframe: Hour 0 and between 4 and 6 hours, 8 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HAC
| Intervention | hours (Mean) |
|---|---|
| RAVICTI: Age 0 to < 2 Months | 11.72 |
(NCT02246218)
Timeframe: Hour 0 and between 4 and 6 hours, 8 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HAC
| Intervention | hours (Mean) |
|---|---|
| RAVICTI: Age 2 Months to < 2 Years | 6.573 |
(NCT02246218)
Timeframe: Hour 0 and between 4 and 6 hours, 8 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HAC
| Intervention | μg*hr/mL (Mean) |
|---|---|
| RAVICTI: Age 2 Months to < 2 Years | 280.936 |
(NCT02246218)
Timeframe: Hour 0 and between 4 and 6 hours, 8 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HAC
| Intervention | μg*hr/mL (Mean) |
|---|---|
| RAVICTI: Age 0 to < 2 Months | 374.53 |
(NCT02246218)
Timeframe: Hour 0 and between 4 and 6 hours, 8 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HAC
| Intervention | μg/mL (Mean) |
|---|---|
| RAVICTI: Age 0 to < 2 Months | 46.2 |
(NCT02246218)
Timeframe: Hour 0 and between 4 and 6 hours, 8 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HAC
| Intervention | μg/mL (Mean) |
|---|---|
| RAVICTI: Age 0 to < 2 Months | 4.8 |
(NCT02246218)
Timeframe: Hour 0 and between 4 and 6 hours, 8 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HAC
| Intervention | μg/mL (Mean) |
|---|---|
| RAVICTI: Age 2 Months to < 2 Years | 1.697 |
(NCT02246218)
Timeframe: Hour 0 and between 4 and 6 hours, 8 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HAC
| Intervention | hours (Mean) |
|---|---|
| RAVICTI: Age 2 Months to < 2 Years | 8.383 |
(NCT02246218)
Timeframe: Hour 0 and between 4 and 6 hours, 8 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HAC
| Intervention | hours (Mean) |
|---|---|
| RAVICTI: Age 0 to < 2 Months | 9.39 |
(NCT02246218)
Timeframe: Hour 0 and between 4 and 6 hours, 8 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HAC
| Intervention | μg/mL (Mean) |
|---|---|
| RAVICTI: Age 2 Months to < 2 Years | 36.52 |
(NCT02246218)
Timeframe: Hour 0 and between 4 and 6 hours, 8 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HAC
| Intervention | μg/mL (Mean) |
|---|---|
| RAVICTI: Age 2 Months to < 2 Years | 62.45 |
(NCT02246218)
Timeframe: Hour 0 and between 4 and 6 hours, 8 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HAC
| Intervention | μg/mL (Mean) |
|---|---|
| RAVICTI: Age 2 Months to < 2 Years | 42.44 |
HAC is defined as having signs and symptoms consistent with hyperammonemia (such as but not limited to frequent vomiting, nausea, headache, lethargy, irritability, combativeness, and/or somnolence) associated with high blood ammonia and requiring medical intervention. Rate of HACs per 6 months during the safety extension was calculated as sum of (number of HAC) / sum of (days during first 6 months starting on Day 8 or number days on RAVICTI, whichever is less) across all participants in the corresponding group. (NCT02246218)
Timeframe: Day 8 through up to Month 6
| Intervention | HACs per half-year of patient exposure (Number) |
|---|---|
| RAVICTI: Age 0 to < 2 Months | 0.003 |
HAC is defined having signs and symptoms consistent with hyperammonemia (such as but not limited to frequent vomiting, nausea, headache, lethargy, irritability, combativeness, and/or somnolence) associated with high blood ammonia and requiring medical intervention. Rate of HACs per 6 months during the safety extension is calculated as sum of (number of HAC) / sum of (days during first 6 months starting on Day 8 or number days on RAVICTI, whichever is less) across all participants in the corresponding group. (NCT02246218)
Timeframe: Day 8 through up to Month 6
| Intervention | HACs per half-year of patient exposure (Number) |
|---|---|
| RAVICTI: Age 2 Months to < 2 Years | 0.005 |
(NCT02246218)
Timeframe: Baseline, Day 7, Month 2, Month 3, Month 4, Month 5, Month 6, Month 9, Month 12, Month 15, Month 18, Month 24
| Intervention | μmol/L (Mean) | |||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Baseline | Day 7 change from Baseline | Month 2 change from Baseline | Month 3 change from Baseline | Month 4 change from Baseline | Month 5 change from Baseline | Month 6 change from Baseline | Month 9 change from Baseline | Month 12 change from Baseline | Month 15 change from Baseline | Month 18 change from Baseline | Month 24 change from Baseline | |
| RAVICTI: Age 0 to < 2 Months | 84.97 | 26.81 | 25.16 | 50.05 | 18.77 | 57.43 | 43.65 | 33.41 | 8.75 | 25.75 | 2.50 | 16.10 |
(NCT02246218)
Timeframe: Baseline, Day 7, Month 2, Month 3, Month 4, Month 5, Month 6, Month 9, Month 12, Month 15, Month 24
| Intervention | µmol/L (Mean) | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|
| Baseline | Day 7 change from Baseline | Month 2 change from Baseline | Month 3 change from Baseline | Month 4 change from Baseline | Month 5 change from Baseline | Month 6 change from Baseline | Month 9 change from Baseline | Month 12 change from Baseline | Month 15 change from Baseline | Month 24 change from Baseline | |
| RAVICTI: Age 2 Months to < 2 Years | 122.43 | -54.50 | 7.80 | -16.33 | -13.00 | 0.25 | -2.20 | 30.80 | 22.20 | 39.00 | 48.00 |
(NCT02246218)
Timeframe: Baseline, Day 7, Month 2, Month 3, Month 4, Month 5, Month 6, Month 9, Month 12, Month 15, Month 18, Month 24
| Intervention | μmol/L (Mean) | |||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Baseline | Day 7 change from Baseline | Month 2 change from Baseline | Month 3 change from Baseline | Month 4 change from Baseline | Month 5 change from Baseline | Month 6 change from Baseline | Month 9 change from Baseline | Month 12 change from Baseline | Month 15 change from Baseline | Month 18 change from Baseline | Month 24 change from Baseline | |
| RAVICTI: Age 0 to < 2 Months | 508.83 | 21.04 | -27.62 | -15.09 | -113.98 | -99.82 | -138.16 | -56.08 | -181.50 | -103.75 | -184.00 | -219.93 |
(NCT02246218)
Timeframe: Baseline, Day 7, Month 2, Month 3, Month 4, Month 5, Month 6, Month 9, Month 12, Month 15, Month 24
| Intervention | µmol/L (Mean) | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|
| Baseline | Day 7 change from Baseline | Month 2 change from Baseline | Month 3 change from Baseline | Month 4 change from Baseline | Month 5 change from Baseline | Month 6 change from Baseline | Month 9 change from Baseline | Month 12 change from Baseline | Month 15 change from Baseline | Month 24 change from Baseline | |
| RAVICTI: Age 2 Months to < 2 Years | 750.43 | -184.33 | -174.60 | -374.00 | -252.75 | -370.25 | -113.20 | -446.53 | -450.50 | -149.00 | 195.00 |
(NCT02246218)
Timeframe: Baseline, Day 7, Month 2, Month 3, Month 4, Month 5, Month 6, Month 9, Month 12, Month 15, Month 18, Month 24
| Intervention | μmol/L (Mean) | |||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Baseline | Day 7 change from Baseline | Month 2 change from Baseline | Month 3 change from Baseline | Month 4 change from Baseline | Month 5 change from Baseline | Month 6 change from Baseline | Month 9 change from Baseline | Month 12 change from Baseline | Month 15 change from Baseline | Month 18 change from Baseline | Month 24 change from Baseline | |
| RAVICTI: Age 0 to < 2 Months | 142.68 | -49.09 | -1.62 | -20.46 | -67.32 | -75.45 | -35.94 | -73.09 | -178.50 | -139.50 | 1.00 | -55.31 |
(NCT02246218)
Timeframe: Baseline, Day 7, Month 2, Month 3, Month 4, Month 5, Month 6, Month 9, Month 12, Month 15, Month 24
| Intervention | µmol/L (Mean) | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|
| Baseline | Day 7 change from Baseline | Month 2 change from Baseline | Month 3 change from Baseline | Month 4 change from Baseline | Month 5 change from Baseline | Month 6 change from Baseline | Month 9 change from Baseline | Month 12 change from Baseline | Month 15 change from Baseline | Month 24 change from Baseline | |
| RAVICTI: Age 2 Months to < 2 Years | 54.86 | 2.67 | 4.20 | -25.67 | -20.25 | -20.00 | -16.40 | -6.73 | -13.33 | -18.00 | 1.50 |
(NCT02246218)
Timeframe: Baseline, Day 7, Month 2, Month 3, Month 4, Month 5, Month 6, Month 9, Month 12, Month 15, Month 18, Month 24
| Intervention | μmol/L (Mean) | |||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Baseline | Day 7 change from Baseline | Month 2 change from Baseline | Month 3 change from Baseline | Month 4 change from Baseline | Month 5 change from Baseline | Month 6 change from Baseline | Month 9 change from Baseline | Month 12 change from Baseline | Month 15 change from Baseline | Month 18 change from Baseline | Month 24 change from Baseline | |
| RAVICTI: Age 0 to < 2 Months | 133.67 | -81.91 | -60.80 | -51.66 | -82.82 | -118.55 | -11.85 | -115.09 | -249.50 | -195.75 | 6.00 | -82.54 |
(NCT02246218)
Timeframe: Baseline, Day 7, Month 2, Month 3, Month 4, Month 5, Month 6, Month 9, Month 12, Month 15, Month 24
| Intervention | µmol/L (Mean) | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|
| Baseline | Day 7 change from Baseline | Month 2 change from Baseline | Month 3 change from Baseline | Month 4 change from Baseline | Month 5 change from Baseline | Month 6 change from Baseline | Month 9 change from Baseline | Month 12 change from Baseline | Month 15 change from Baseline | Month 24 change from Baseline | |
| RAVICTI: Age 2 Months to < 2 Years | 90.86 | -0.83 | 9.80 | -33.00 | -31.25 | -39.50 | -25.40 | -19.13 | -34.37 | -40.00 | -1.50 |
(NCT02246218)
Timeframe: Baseline, Day 7, Month 2, Month 3, Month 4, Month 5, Month 6, Month 9, Month 12, Month 15, Month 18, Month 24
| Intervention | μmol/L (Mean) | |||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Baseline | Day 7 change from Baseline | Month 2 change from Baseline | Month 3 change from Baseline | Month 4 change from Baseline | Month 5 change from Baseline | Month 6 change from Baseline | Month 9 change from Baseline | Month 12 change from Baseline | Month 15 change from Baseline | Month 18 change from Baseline | Month 24 change from Baseline | |
| RAVICTI: Age 0 to < 2 Months | 593.80 | 47.85 | -2.46 | 34.96 | -95.21 | -42.39 | -94.51 | -22.66 | -172.75 | -78.00 | -181.50 | -203.83 |
(NCT02246218)
Timeframe: Baseline, Day 7, Month 2, Month 3, Month 4, Month 5, Month 6, Month 9, Month 12, Month 15, Month 24
| Intervention | µmol/L (Mean) | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|
| Baseline | Day 7 change from Baseline | Month 2 change from Baseline | Month 3 change from Baseline | Month 4 change from Baseline | Month 5 change from Baseline | Month 6 change from Baseline | Month 9 change from Baseline | Month 12 change from Baseline | Month 15 change from Baseline | Month 24 change from Baseline | |
| RAVICTI: Age 2 Months to < 2 Years | 872.86 | -238.83 | -166.80 | -390.33 | -265.75 | -370.00 | -115.40 | -415.73 | -428.30 | -110.00 | 243.00 |
(NCT02246218)
Timeframe: Baseline, Day 7, Month 2, Month 3, Month 4, Month 5, Month 6, Month 9, Month 12, Month 15, Month 18, Month 24
| Intervention | μmol/L (Mean) | |||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Baseline | Day 7 change from Baseline | Month 2 change from Baseline | Month 3 change from Baseline | Month 4 change from Baseline | Month 5 change from Baseline | Month 6 change from Baseline | Month 9 change from Baseline | Month 12 change from Baseline | Month 15 change from Baseline | Month 18 change from Baseline | Month 24 change from Baseline | |
| RAVICTI: Age 0 to < 2 Months | 181.49 | -63.96 | -39.04 | -23.86 | -74.41 | -98.67 | 2.64 | -90.40 | -238.25 | -137.00 | 38.00 | -72.78 |
(NCT02246218)
Timeframe: Baseline, Day 7, Month 2, Month 3, Month 4, Month 5, Month 6, Month 9, Month 12, Month 15, Month 24
| Intervention | µmol/L (Mean) | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|
| Baseline | Day 7 change from Baseline | Month 2 change from Baseline | Month 3 change from Baseline | Month 4 change from Baseline | Month 5 change from Baseline | Month 6 change from Baseline | Month 9 change from Baseline | Month 12 change from Baseline | Month 15 change from Baseline | Month 24 change from Baseline | |
| RAVICTI: Age 2 Months to < 2 Years | 171.43 | 4.00 | 40.60 | -27.33 | -31.50 | -56.00 | -21.60 | -11.90 | -48.87 | -46.00 | 5.00 |
To assess any effect of study drug treatment on growth, Z-scores were calculated to express the deviation from a reference population for BMI. The Z-scores are based on the World Health Organization's Child Growth Standards charts. Negative Z-scores indicate lower than typical for age and gender while positive scores indicate higher than typical for age and gender. (NCT02246218)
Timeframe: Baseline, Month 1, Month 2, Month 3, Month 4, Month 5, Month 6, Month 9, Month 12, Month 15, Month 18, Month 24
| Intervention | z-score (Mean) | |||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Baseline | Month 1 change from Baseline | Month 2 change from Baseline | Month 3 change from Baseline | Month 4 change from Baseline | Month 5 change from Baseline | Month 6 change from Baseline | Month 9 change from Baseline | Month 12 change from Baseline | Month 15 change from Baseline | Month 18 change from Baseline | Month 24 change from Baseline | |
| RAVICTI: Age 0 to < 2 Months | -0.0544 | -0.2158 | -0.2598 | -0.1617 | -0.0264 | 0.0828 | 0.0136 | 0.4614 | 0.6646 | 0.6830 | 0.3308 | 0.7743 |
To assess any effect of study drug treatment on growth, Z-scores were calculated to express the deviation from a reference population for BMI. The Z-scores are based on the World Health Organization's Child Growth Standards charts. Negative Z-scores indicate lower than typical for age and gender while positive scores indicate higher than typical for age and gender. (NCT02246218)
Timeframe: Baseline, Month 1, Month 2, Month 3, Month 4, Month 5, Month 6, Month 9, Month 12, Month 15, Month 18, Month 24
| Intervention | z-score (Mean) | |||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Baseline | Month 1 change from Baseline | Month 2 change from Baseline | Month 3 change from Baseline | Month 4 change from Baseline | Month 5 change from Baseline | Month 6 change from Baseline | Month 9 change from Baseline | Month 12 change from Baseline | Month 15 change from Baseline | Month 18 change from Baseline | Month 24 change from Baseline | |
| RAVICTI: Age 2 Months to < 2 Years | 0.8107 | -0.2385 | -0.0249 | 0.1815 | 0.4434 | 0.1484 | 0.2497 | 0.6407 | 0.4164 | -0.2997 | -0.2038 | 0.5581 |
To assess any effect of study drug treatment on growth, Z-scores were calculated to express the deviation from a reference population for BSA. The Z-scores are based on weight-for-length charts. Negative Z-scores indicate lower than typical for age and gender while positive scores indicate higher than typical for age and gender. (NCT02246218)
Timeframe: Baseline, Month 1, Month 2, Month 3, Month 4, Month 5, Month 6, Month 9, Month 12, Month 15, Month 18, Month 24
| Intervention | z-score (Mean) | |||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Baseline | Month 1 change from Baseline | Month 2 change from Baseline | Month 3 change from Baseline | Month 4 change from Baseline | Month 5 change from Baseline | Month 6 change from Baseline | Month 9 change from Baseline | Month 12 change from Baseline | Month 15 change from Baseline | Month 18 change from Baseline | Month 24 change from Baseline | |
| RAVICTI: Age 2 Months to < 2 Years | 0.7143 | -0.2105 | -0.0704 | 0.1065 | 0.3365 | 0.1043 | 0.1842 | 0.4875 | 0.2944 | -0.3661 | -0.2214 | 0.4310 |
To assess any effect of study drug treatment on growth, Z-scores were calculated to express the deviation from a reference population for BSA. The Z-scores are based on weight-for-length charts. Negative Z-scores indicate lower than typical for age and gender while positive scores indicate higher than typical for age and gender. (NCT02246218)
Timeframe: Baseline, Month 1, Month 2, Month 3, Month 4, Month 5, Month 6, Month 9, Month 12, Month 15, Month 18, Month 24
| Intervention | z-score (Mean) | |||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Baseline | Month 1 change from Baseline | Month 2 change from Baseline | Month 3 change from Baseline | Month 4 change from Baseline | Month 5 change from Baseline | Month 6 change from Baseline | Month 9 change from Baseline | Month 12 change from Baseline | Month 15 change from Baseline | Month 18 change from Baseline | Month 24 change from Baseline | |
| RAVICTI: Age 0 to < 2 Months | -0.1980 | 0.2336 | 0.2006 | 0.2684 | 0.2372 | 0.1810 | 0.2902 | 0.1679 | 0.1308 | 0.1595 | 0.1050 | 0.7341 |
(NCT02246218)
Timeframe: Hour 0 and between 0.5 and 1.5 hours, 1.5 and 2.5 hours, 4 and 6 hours, 7.5 and 8.5 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HAC
| Intervention | μg/mL (Mean) | |||||
|---|---|---|---|---|---|---|
| Hour 0 | 0.5 to 1.5 hours | 1.5 to 2.5 hours | 4 to 6 hours | 7.5 to 8.5 hours | 12 to 24 hours | |
| RAVICTI: Age 0 to < 2 Months | 11.1 | 46.2 | 62.5 | 34.6 | 22.8 | 35.2 |
(NCT02246218)
Timeframe: Hour 0 and between 0.5 and 1.5 hours, 1.5 and 2.5 hours, 4 and 6 hours, 7.5 and 8.5 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HAC
| Intervention | μg/mL (Mean) | |||||
|---|---|---|---|---|---|---|
| Hour 0 | 0.5 to 1.5 hours | 1.5 to 2.5 hours | 4 to 6 hours | 7.5 to 8.5 hours | 12 to 24 hours | |
| RAVICTI: Age 2 Months to < 2 Years | 18.78 | 6.50 | 7.29 | 2.60 | 4.48 | 4.31 |
(NCT02246218)
Timeframe: Day 7, Month 1, Month 2, Month 3, Month 4, Month 5, Month 6, Month 9, Month 15, End of Trial (up to Month 15)
| Intervention | μg/mL (Mean) | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|
| Day 7 | Month 1 | Month 2 | Month 3 | Month 4 | Month 5 | Month 6 | Month 9 | Month 12 | Month 15 | End of trial | |
| RAVICTI: Age 0 to < 2 Months | 23.7 | 14.6 | 12.3 | 14.4 | 6.4 | 13.2 | 5.5 | 11.8 | 6.0 | 4.9 | 11.6 |
(NCT02246218)
Timeframe: Day 7, Month 1, Month 2, Month 3, Month 4, Month 5, Month 6, Month 9, Month 15, Month 18, End of Trial (up to Month 18)
| Intervention | μg/mL (Mean) | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|
| Day 7 | Month 1 | Month 2 | Month 3 | Month 4 | Month 5 | Month 6 | Month 9 | Month 15 | Month 18 | End of trial | |
| RAVICTI: Age 2 Months to < 2 Years | 5.82 | 4.44 | 3.69 | 4.65 | 7.14 | 3.27 | 1.59 | 4.10 | 2.04 | 1.64 | 7.0 |
(NCT02246218)
Timeframe: Hour 0 and between 0.5 and 1 hour, 1.5 and 2.5 hours, 4 and 6 hours, 7.5 and 8.5 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HAC
| Intervention | μg/mL (Mean) | |||||
|---|---|---|---|---|---|---|
| Hour 0 | 0.5 to 1.5 hours | 1.5 to 2.5 hours | 4 to 6 hours | 7.5 to 8.5 hours | 12 to 24 hours | |
| RAVICTI: Age 0 to < 2 Months | 3530.43 | 1828 | 1746 | 2260 | 3530.43 | 4404 |
(NCT02246218)
Timeframe: Hour 0 and between 0.5 and 1 hour, 1.5 and 2.5 hours, 4 and 6 hours, 7.5 and 8.5 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HAC
| Intervention | μg/mL (Mean) | |||||
|---|---|---|---|---|---|---|
| Hour 0 | 0.5 to 1.5 hours | 1.5 to 2.5 hours | 4 to 6 hours | 7.5 to 8.5 hours | 12 to 24 hours | |
| RAVICTI: Age 2 Months to < 2 Years | 3273 | 4140 | 3145 | 5202 | 3950 | 7561 |
(NCT02246218)
Timeframe: Day 7, Month 1, Month 2, Month 3, Month 4, Month 5, Month 6, Month 9, Month 12, Month 15, End of Trial (up to Month 15)
| Intervention | μg/mL (Mean) | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|
| Day 7 | Month 1 | Month 2 | Month 3 | Month 4 | Month 5 | Month 6 | Month 9 | Month 12 | Month 15 | End of trial | |
| RAVICTI: Age 0 to < 2 Months | 4643 | 4517 | 4116 | 7037 | 2826 | 6973 | 5883 | 7006 | 5847 | 3915 | 6939 |
(NCT02246218)
Timeframe: Day 7, Month 1, Month 2, Month 3, Month 4, Month 5, Month 6, Month 9, Month 12, Month 15, Month 18, End of Trial (up to Month 18)
| Intervention | μg/mL (Mean) | |||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Day 7 | Month 1 | Month 2 | Month 3 | Month 4 | Month 5 | Month 6 | Month 9 | Month 12 | Month 15 | Month 18 | End of trial | |
| RAVICTI: Age 2 Months to < 2 Years | 8859 | 6274 | 7386 | 11456 | 21416 | 6129 | 5347 | 9357 | 2580 | 6400 | 5250 | 25333 |
An AE is any untoward medical occurrence, whether or not the event is considered related to the study drug. A serious AE is any AE that: results in death; is life threatening; requires hospitalization or prolongation of existing hospitalization; results in disability/incapacity; is a congenital anomaly/birth defect; is an important medical event. TEAEs are defined as AEs with an onset date on or after the first dose of study medication until study discontinuation. The Investigator assessed the causal relationship of each TEAE to the study drug as not related, possibly related, or probably related. (NCT02246218)
Timeframe: From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 10.67 [6.142] months).
| Intervention | Participants (Count of Participants) | |||||
|---|---|---|---|---|---|---|
| ≥ 1 TEAE | ≥ 1 Related TEAE | ≥ 1 Serious TEAE | ≥ 1 Serious Related TEAE | Fatal Outcome TEAE | ≥ 1 TEAE Leading to Study Discontinuation | |
| RAVICTI: Age 0 to < 2 Months | 16 | 10 | 11 | 0 | 0 | 1 |
An adverse event (AE) is any untoward medical occurrence, whether or not the event is considered related to the study drug. A serious AE is any AE that: results in death; is life threatening; requires hospitalization or prolongation of existing hospitalization; results in disability/incapacity; is a congenital anomaly/birth defect; is an important medical event. TEAEs are defined as AEs with an onset date on or after the first dose of study medication until study discontinuation. The Investigator assessed the causal relationship of each TEAE to the study drug as not related, possibly related, or probably related. (NCT02246218)
Timeframe: From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months).
| Intervention | Participants (Count of Participants) | |||||
|---|---|---|---|---|---|---|
| ≥ 1 TEAE | ≥ 1 Related TEAE | ≥ 1 Serious TEAE | ≥ 1 Serious Related TEAE | Fatal Outcome TEAE | ≥ 1 TEAE Leading to Study Discontinuation | |
| RAVICTI: Age 2 Months to < 2 Years | 10 | 4 | 6 | 0 | 1 | 1 |
blood samples were collected at pre-dose, 4, 8, 12, 16, 20, and 24 hour post dose on both Day 7 (NaPBA) and Day 14 (HPN-100). (NCT00947544)
Timeframe: Day 7 (NaPBA) and Day 14 (HPN-100)
| Intervention | µmol/L (Mean) |
|---|---|
| HPN-100 | 28.68 |
| NaPBA | 37.75 |
To evaluate control of blood ammonia by HPN-100 compared with NaPBA in pediatric patients with UCDs. (NCT00947544)
Timeframe: Day 7 (NaPBA) and Day 14 (HPN-100)
| Intervention | μmol∙h/L (Mean) |
|---|---|
| HPN-100 | 603.83 |
| NaPBA | 814.62 |
blood samples were collected at pre-dose, 4, 8, 12, 16, 20, and 24 hour post dose on both Day 7 (NaPBA) and Day 14 (HPN-100). (NCT00947544)
Timeframe: Day 7 (NaPBA) and Day 14 (HPN-100)
| Intervention | μmol/L (Mean) |
|---|---|
| HPN-100 | 47.77 |
| NaPBA | 55.66 |
blood samples were collected at pre-dose, 4, 8, 12, 16, 20, and 24 hour post dose on both Day 7 (NaPBA) and Day 14 (HPN-100). (NCT00947544)
Timeframe: Day 7 (NaPBA) and Day 14 (HPN-100)
| Intervention | μg•h/mL AUC 0-24 (Mean) |
|---|---|
| HPN-100 | 964 |
| NaPBA | 773 |
blood samples were collected at pre-dose, 4, 8, 12, 16, 20, and 24 hour post dose on both Day 7 (NaPBA) and Day 14 (HPN-100). (NCT00947544)
Timeframe: Day 7 (NaPBA) and Day 14 (HPN-100)
| Intervention | μg*h/mL AUC 0-24 (Mean) |
|---|---|
| HPN-100 | 1378 |
| NaPBA | 1015 |
blood samples were collected at pre-dose, 4, 8, 12, 16, 20, and 24 hour post dose on both Day 7 (NaPBA) and Day 14 (HPN-100). (NCT00947544)
Timeframe: Day 7 (NaPBA) and Day 14 (HPN-100)
| Intervention | µg*h/ml AUC 0-24 (Mean) |
|---|---|
| HPN-100 | 631 |
| NaPBA | 236 |
"change from baseline to Month 12.~The SF 15 questionnaire consists of 15 questions that assess the following:~Physical functioning (5 questions)~Emotional functioning (4 questions)~Social functioning (3 questions)~School functioning (3 questions) Items were scored on a 5-point Likert scale from 0 (never) to 4 (almost always) or a 3-point scale (0 [not at all], 2 [sometimes], or 4 [a lot] for the young child self-report). Items were reverse-scored and linearly transformed to a 0-100 scale as follows: 0=100, 1=75, 2=50, 3=25, and 4=0. Total score was 0-100 scale (averaged from each functional areas). In the 0-100 scale, 0 is the worst score and 100 is best score.~Improved quality of life was shown by increased total score from baseline to Month 12." (NCT00947544)
Timeframe: 1 year
| Intervention | score on a scale (Mean) |
|---|---|
| HPN-100 | 4.0 |
blood samples were collected at pre-dose, 4, 8, 12, 16, 20, and 24 hour post dose on both Day 7 (NaPBA) and Day 14 (HPN-100). (NCT00947544)
Timeframe: Day 7 (NaPBA) and Day 14 (HPN-100)
| Intervention | percentage of sample (Number) |
|---|---|
| HPN-100 | 18.4 |
| NaPBA | 31.6 |
To evaluate the safety and PK characteristics of HPN-100 compared with sodium phenylbutyrate (NaPBA) in pediatric patients with urea cycle disorders (UCDs) (NCT00947544)
Timeframe: 1 week on each treatment for a total of 2 week.
| Intervention | participants (Number) |
|---|---|
| HPN-100 | 4 |
| NaPBA | 2 |
Urinary PAGN (phenylacetylglutamine) 24-hour excretion. Urine was collect during 0-12 hrs and 12-24 hrs. (NCT00947544)
Timeframe: Day 7 (NaPBA) and Day 14 (HPN-100)
| Intervention | μg (Mean) |
|---|---|
| HPN-100 | 12501037 |
| NaPBA | 12512426 |
"Number of Subjects with at Least One Hyperammonemic Crisis.~Hyperammonemic crisis is defined as follows:~• Clinical symptoms associated with ammonia of ≥ 100 µmol/L" (NCT00947544)
Timeframe: 1 year
| Intervention | participants (Number) | |
|---|---|---|
| Number of subjects with at least 1 HAC | Number of Crises | |
| Pre-Enrollment (NaPBA) | 5 | 8 |
| Safety Extension (HPN-100) | 3 | 3 |
Blood samples were collected at pre-dose, 2, 4, 8, 12, 16, 20 and 24 hours after first dose on days 14 and 28. (NCT00992459)
Timeframe: pre-dose, 2, 4, 8, 12, 16, 20 and 24 hours after first dose on days 14 and 28
| Intervention | μg/mL (Mean) |
|---|---|
| NaPBA | 52.2 |
| HPN-100 | 38.5 |
Blood samples were collected at pre-dose, 2, 4, 8, 12, 16, 20 and 24 hours after first dose on days 14 and 28. (NCT00992459)
Timeframe: pre-dose, 2, 4, 8, 12, 16, 20 and 24 hours after first dose on days 14 and 28
| Intervention | μg/mL (Mean) |
|---|---|
| NaPBA | 80.9 |
| HPN-100 | 51.9 |
Blood samples were collected at pre-dose, 2, 4, 8, 12, 16, 20 and 24 hours after first dose on days 14 and 28. (NCT00992459)
Timeframe: pre-dose, 2, 4, 8, 12, 16, 20 and 24 hours after first dose on days 14 and 28
| Intervention | μg/mL (Mean) |
|---|---|
| NaPBA | 78.6 |
| HPN-100 | 86.8 |
The correlation between 24-hour urinary phenylacetylglutamine (PAGN) excretion (U-PAGN24-hour Excr) and venous ammonia AUC0-24 was summarized and the correlation was tested using the Spearman rank-order correlation. (NCT00992459)
Timeframe: 28 Days
| Intervention | correlation coefficient (Number) |
|---|---|
| NaPBA | 0.437 |
| HPN-100 | 0.219 |
Blood samples were collected at pre-dose, 2, 4, 8, 12, 16, 20 and 24 hours after first dose on days 14 and 28. (NCT00992459)
Timeframe: pre-dose, 2, 4, 8, 12, 16, 20 and 24 hours after first dose on days 14 and 28
| Intervention | µmol/L (Mean) |
|---|---|
| NaPBA | 70.83 |
| HPN-100 | 60.94 |
Severity of symptomatic hyperammonemic crises was measured by peak ammonia level (µmol/L) when it is >= 100 µmol/L. (NCT00992459)
Timeframe: 29 Days
| Intervention | events (Number) |
|---|---|
| NaPBA | 1 |
| HPN-100 | 0 |
NaPBA treated arm: total 345 blood samples were collected. HPN-100 treated arm: 343 blood samples were collected. (NCT00992459)
Timeframe: on Day 14 and Day 28
| Intervention | samples (Number) |
|---|---|
| NaPBA | 125 |
| HPN-100 | 122 |
(NCT00992459)
Timeframe: 29 Days
| Intervention | participants (Number) |
|---|---|
| NaPBA | 23 |
| HPN-100 | 27 |
Blood samples were collected at pre-dose, 2, 4, 8, 12, 16, 20 and 24 hours after first dose on days 14 and 28. Arm A day 14 and Arm B day 28 data were combined as a NaPBA treatment Arm. Arm B day 14 and Arm A day 28 data were combined as a HPN-100 treatment Arm. (NCT00992459)
Timeframe: pre-dose, 2, 4, 8, 12, 16, 20 and 24 hours after first dose on days 14 and 28
| Intervention | μmol∙h/L (Mean) |
|---|---|
| NaPBA | 976.6 |
| HPN-100 | 865.35 |
(NCT00992459)
Timeframe: 24 hours on Day 14 of each treatments
| Intervention | μg (Mean) |
|---|---|
| NaPBA | 13627515 |
| HPN-100 | 13502745 |
Number of hyperammonemic crises per patient (NCT00947297)
Timeframe: 1 year
| Intervention | hyperammonemic events (Mean) |
|---|---|
| HPN-100 | 0.20 |
Drug preference will be noted at week 3 (NCT00947297)
Timeframe: Month 1 post dose
| Intervention | % preferred HPN-100 (Number) |
|---|---|
| HPN-100 | 90 |
(NCT00947297)
Timeframe: 1 year
| Intervention | participants (Number) |
|---|---|
| HPN-100 | 33 |
Venous Ammonia levels over time (NCT00947297)
Timeframe: 1 Year
| Intervention | Umol/L (Mean) | |
|---|---|---|
| Baseline | Month 12 | |
| HPN-100 | 27.623 | 24.202 |
(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 |
Rate of adverse events during the Safety Extension portion of the protocol ( please note: HPN-100 treatment only during Safety Extension ) (NCT01347073)
Timeframe: 12 months
| Intervention | participants (Number) |
|---|---|
| HPN-100 | 23 |
Rate of adverse events during the Switch-Over portion of the Protocol (NCT01347073)
Timeframe: 2 weeks
| Intervention | participants (Number) |
|---|---|
| NaPBA | 0 |
| HPN-100 | 6 |
24-hour ammonia AUC of blood ammonia levels on Days 1 (NaPBA) and 10 (HPN-100) were compared. Ammonia was assessed at Hour 0 (pre-first dose, fasted), Hour 8 (~2-4 hours after lunch or the second main meal and dose of NaPBA), Hour 12 (~4 hours after the last main meal) and 24 hours post-first dose (pre-first dose on following day, fasted). (NCT01347073)
Timeframe: 2 weeks
| Intervention | umol/L*hours (Mean) |
|---|---|
| NaPBA | 914.43 |
| HPN-100 | 647.63 |
Ammonia values were converted to SI units (umol/L) and normalized to a standard ULN of 35 umol/L prior to analysis (NCT01347073)
Timeframe: 2 weeks
| Intervention | Ammonia Values > ULN (Number) |
|---|---|
| NaPBA | 22 |
| HPN-100 | 8 |
Rate of HAC during pre-enrollment on NaPBA compared to HAC during HPN-100 treatment (NCT01347073)
Timeframe: 1 year
| Intervention | number of crises (Number) |
|---|---|
| Pre-enrollment | 29 |
| Long-term Phase | 12 |
14 reviews available for ammonium hydroxide and Urea Cycle Disorders, Inborn
| Article | Year |
|---|---|
The context-specific roles of urea cycle enzymes in tumorigenesis.
Topics: Ammonia; Carcinogenesis; Cell Line, Tumor; Cell Proliferation; Cell Transformation, Neoplastic; Gene | 2021 |
Presentation and management of N-acetylglutamate synthase deficiency: a review of the literature.
Topics: Amino-Acid N-Acetyltransferase; Ammonia; Humans; Hyperammonemia; Infant, Newborn; Urea Cycle Disorde | 2020 |
Primary hyperammonaemia: Current diagnostic and therapeutic strategies.
Topics: Ammonia; Early Diagnosis; Female; Humans; Hyperammonemia; Infant; Infant, Newborn; Male; Renal Dialy | 2020 |
CPS1: Looking at an ancient enzyme in a modern light.
Topics: Ammonia; Carbamoyl-Phosphate Synthase (Ammonia); Carbamoyl-Phosphate Synthase I Deficiency Disease; | 2020 |
Nonhepatic hyperammonemic encephalopathy complications following bariatric surgery: a case report and review of the literature.
Topics: Adult; Ammonia; Bariatric Surgery; Female; Humans; Hyperammonemia; Obesity; Urea Cycle Disorders, In | 2021 |
Inborn Errors of Metabolism with Hyperammonemia: Urea Cycle Defects and Related Disorders.
Topics: Ammonia; Emergency Treatment; Humans; Hyperammonemia; Infant; Infant, Newborn; Urea; Urea Cycle Diso | 2018 |
The impact of ammonia levels and dialysis on outcome in 202 patients with neonatal onset urea cycle disorders.
Topics: Age of Onset; Ammonia; Humans; Hyperammonemia; Infant, Newborn; Renal Dialysis; Treatment Outcome; U | 2018 |
Ammonia and autophagy: An emerging relationship with implications for disorders with hyperammonemia.
Topics: Ammonia; Animals; Autophagy; Glutamate-Ammonia Ligase; Glutamine; Homeostasis; Humans; Hyperammonemi | 2019 |
α-Ketoglutaramate: an overlooked metabolite of glutamine and a biomarker for hepatic encephalopathy and inborn errors of the urea cycle.
Topics: Amidohydrolases; Aminohydrolases; Ammonia; Animals; Biomarkers; Carbon; Glutamine; Hepatic Encephalo | 2014 |
The Measurement of Ammonia in Human Breath and its Potential in Clinical Diagnostics.
Topics: Amino Acid Metabolism, Inborn Errors; Ammonia; Breath Tests; Humans; Hyperinsulinism; Hypoglycemia; | 2016 |
An update on the use of benzoate, phenylacetate and phenylbutyrate ammonia scavengers for interrogating and modifying liver nitrogen metabolism and its implications in urea cycle disorders and liver disease.
Topics: Ammonia; Animals; Benzoates; Drug Design; Glutamine; Humans; Liver Cirrhosis; Liver Diseases; Liver | 2017 |
Ammonia toxicity and its prevention in inherited defects of the urea cycle.
Topics: Adult; Ammonia; Animals; Arginine; Genetic Therapy; Humans; Hyperammonemia; Ornithine Carbamoyltrans | 2009 |
Severe hyperammonaemia in adults not explained by liver disease.
Topics: Adult; Amino Acids; Ammonia; Biological Transport; Blood-Brain Barrier; Brain; Glutamine; Humans; Hy | 2012 |
Recurrent encephalopathy: NAGS (N-acetylglutamate synthase) deficiency in adults.
Topics: Adult; Amino-Acid N-Acetyltransferase; Ammonia; Brain Diseases; Female; Humans; Male; Metabolic Netw | 2013 |
8 trials available for ammonium hydroxide and Urea Cycle Disorders, Inborn
| Article | Year |
|---|---|
Human heterologous liver cells transiently improve hyperammonemia and ureagenesis in individuals with severe urea cycle disorders.
Topics: Ammonia; Biomarkers; Cell Transplantation; Europe; Female; Humans; Hyperammonemia; Infant; Infant, N | 2018 |
Glycerol phenylbutyrate treatment in children with urea cycle disorders: pooled analysis of short and long-term ammonia control and outcomes.
Topics: Adolescent; Ammonia; Child; Child, Preschool; Cross-Over Studies; Female; Glutamine; Humans; Infant; | 2014 |
Glutamine and hyperammonemic crises in patients with urea cycle disorders.
Topics: Adolescent; Adult; Ammonia; Biomarkers; Child; Child, Preschool; Fasting; Female; Glutamine; Glycero | 2016 |
Phase 2 comparison of a novel ammonia scavenging agent with sodium phenylbutyrate in patients with urea cycle disorders: safety, pharmacokinetics and ammonia control.
Topics: Adult; Aged; Ammonia; Cross-Over Studies; Female; Glutamine; Glycerol; Humans; Male; Middle Aged; Ph | 2010 |
Ammonia control in children with urea cycle disorders (UCDs); phase 2 comparison of sodium phenylbutyrate and glycerol phenylbutyrate.
Topics: Adolescent; Ammonia; Child; Dose-Response Relationship, Drug; Glycerol; Humans; Male; Phenylbutyrate | 2011 |
Urinary phenylacetylglutamine as dosing biomarker for patients with urea cycle disorders.
Topics: Adolescent; Adult; Ammonia; Biomarkers, Pharmacological; Child; Cross-Over Studies; Drug Administrat | 2012 |
Ammonia control and neurocognitive outcome among urea cycle disorder patients treated with glycerol phenylbutyrate.
Topics: Adolescent; Adult; Ammonia; Child; Cross-Over Studies; Double-Blind Method; Female; Glutamine; Glyce | 2013 |
Ammonia control and neurocognitive outcome among urea cycle disorder patients treated with glycerol phenylbutyrate.
Topics: Adolescent; Adult; Ammonia; Child; Cross-Over Studies; Double-Blind Method; Female; Glutamine; Glyce | 2013 |
Ammonia control and neurocognitive outcome among urea cycle disorder patients treated with glycerol phenylbutyrate.
Topics: Adolescent; Adult; Ammonia; Child; Cross-Over Studies; Double-Blind Method; Female; Glutamine; Glyce | 2013 |
Ammonia control and neurocognitive outcome among urea cycle disorder patients treated with glycerol phenylbutyrate.
Topics: Adolescent; Adult; Ammonia; Child; Cross-Over Studies; Double-Blind Method; Female; Glutamine; Glyce | 2013 |
Ammonia control and neurocognitive outcome among urea cycle disorder patients treated with glycerol phenylbutyrate.
Topics: Adolescent; Adult; Ammonia; Child; Cross-Over Studies; Double-Blind Method; Female; Glutamine; Glyce | 2013 |
Ammonia control and neurocognitive outcome among urea cycle disorder patients treated with glycerol phenylbutyrate.
Topics: Adolescent; Adult; Ammonia; Child; Cross-Over Studies; Double-Blind Method; Female; Glutamine; Glyce | 2013 |
Ammonia control and neurocognitive outcome among urea cycle disorder patients treated with glycerol phenylbutyrate.
Topics: Adolescent; Adult; Ammonia; Child; Cross-Over Studies; Double-Blind Method; Female; Glutamine; Glyce | 2013 |
Ammonia control and neurocognitive outcome among urea cycle disorder patients treated with glycerol phenylbutyrate.
Topics: Adolescent; Adult; Ammonia; Child; Cross-Over Studies; Double-Blind Method; Female; Glutamine; Glyce | 2013 |
Ammonia control and neurocognitive outcome among urea cycle disorder patients treated with glycerol phenylbutyrate.
Topics: Adolescent; Adult; Ammonia; Child; Cross-Over Studies; Double-Blind Method; Female; Glutamine; Glyce | 2013 |
Ammonia control and neurocognitive outcome among urea cycle disorder patients treated with glycerol phenylbutyrate.
Topics: Adolescent; Adult; Ammonia; Child; Cross-Over Studies; Double-Blind Method; Female; Glutamine; Glyce | 2013 |
Ammonia control and neurocognitive outcome among urea cycle disorder patients treated with glycerol phenylbutyrate.
Topics: Adolescent; Adult; Ammonia; Child; Cross-Over Studies; Double-Blind Method; Female; Glutamine; Glyce | 2013 |
Ammonia control and neurocognitive outcome among urea cycle disorder patients treated with glycerol phenylbutyrate.
Topics: Adolescent; Adult; Ammonia; Child; Cross-Over Studies; Double-Blind Method; Female; Glutamine; Glyce | 2013 |
Ammonia control and neurocognitive outcome among urea cycle disorder patients treated with glycerol phenylbutyrate.
Topics: Adolescent; Adult; Ammonia; Child; Cross-Over Studies; Double-Blind Method; Female; Glutamine; Glyce | 2013 |
Ammonia control and neurocognitive outcome among urea cycle disorder patients treated with glycerol phenylbutyrate.
Topics: Adolescent; Adult; Ammonia; Child; Cross-Over Studies; Double-Blind Method; Female; Glutamine; Glyce | 2013 |
Ammonia control and neurocognitive outcome among urea cycle disorder patients treated with glycerol phenylbutyrate.
Topics: Adolescent; Adult; Ammonia; Child; Cross-Over Studies; Double-Blind Method; Female; Glutamine; Glyce | 2013 |
Ammonia control and neurocognitive outcome among urea cycle disorder patients treated with glycerol phenylbutyrate.
Topics: Adolescent; Adult; Ammonia; Child; Cross-Over Studies; Double-Blind Method; Female; Glutamine; Glyce | 2013 |
Ammonia control in children ages 2 months through 5 years with urea cycle disorders: comparison of sodium phenylbutyrate and glycerol phenylbutyrate.
Topics: Ammonia; Child; Child, Preschool; Drug Substitution; Female; Glutamine; Glycerol; Humans; Infant; Li | 2013 |
31 other studies available for ammonium hydroxide and Urea Cycle Disorders, Inborn
| Article | Year |
|---|---|
Glutaminase 2 knockdown reduces hyperammonemia and associated lethality of urea cycle disorder mouse model.
Topics: Ammonia; Animals; Disease Models, Animal; Glutaminase; Glutamine; Humans; Hyperammonemia; Liver; Mic | 2022 |
Clinical findings of patients with hyperammonemia affected by urea cycle disorders with hepatic encephalopathy.
Topics: Ammonia; Hepatic Encephalopathy; Humans; Hyperammonemia; Ornithine Carbamoyltransferase Deficiency D | 2022 |
Relationship between longitudinal changes in neuropsychological outcome and disease biomarkers in urea cycle disorders.
Topics: Ammonia; Biomarkers; Glutamine; Humans; Hyperammonemia; Longitudinal Studies; Neuropsychological Tes | 2023 |
Citrulline in the management of patients with urea cycle disorders.
Topics: Ammonia; Arginine; Citrulline; Drug-Related Side Effects and Adverse Reactions; Humans; Retrospectiv | 2023 |
Transition to glycerol phenylbutyrate for the management of urea cycle disorders: clinical experiences.
Topics: Ammonia; Glutamine; Humans; Quality of Life; Urea; Urea Cycle Disorders, Inborn | 2023 |
Ammonia uptake by transmembrane pH gradient poly(isoprene)-block-poly(ethylene glycol) polymersomes.
Topics: Ammonia; Butadienes; Drug Carriers; Fluorescein-5-isothiocyanate; Hemiterpenes; Hepatic Encephalopat | 2020 |
Neonatal factors related to survival and intellectual and developmental outcome of patients with early-onset urea cycle disorders.
Topics: Age of Onset; Ammonia; Argininosuccinate Synthase; Carbamoyl-Phosphate Synthase (Ammonia); Developme | 2020 |
ECMO as a Platform for Rapid Ammonia Removal in a Neonate with Multienzyme Urea Cycle Disorder.
Topics: Adult; Ammonia; Cesarean Section; Extracorporeal Membrane Oxygenation; Female; Humans; Infant, Newbo | 2020 |
Perioperative management of children with urea cycle disorders.
Topics: Ammonia; Anesthesia, General; Child; Humans; Hyperammonemia; Prognosis; Urea Cycle Disorders, Inborn | 2020 |
A retrospective study of adult patients with noncirrhotic hyperammonemia.
Topics: Adult; Age of Onset; Aged; Ammonia; Female; Humans; Hyperammonemia; Male; Middle Aged; Retrospective | 2020 |
Glycerol phenylbutyrate efficacy and safety from an open label study in pediatric patients under 2 months of age with urea cycle disorders.
Topics: Age of Onset; Ammonia; Child, Preschool; Female; Glycerol; Humans; Hyperammonemia; Infant; Infant, N | 2021 |
Long-term outcome of urea cycle disorders: Report from a nationwide study in Japan.
Topics: Adolescent; Adult; Ammonia; Child; Child, Preschool; Female; Humans; Hyperammonemia; Japan; Liver Tr | 2021 |
Reversible Leukoencephalopathy in a Man with Childhood-onset Hyperornithinemia-Hyperammonemia-Homocitrullinuria Syndrome.
Topics: Ammonia; Child; Humans; Hyperammonemia; Leukoencephalopathies; Male; Middle Aged; Ornithine; Urea Cy | 2022 |
Quantitation of phenylbutyrate metabolites by UPLC-MS/MS demonstrates inverse correlation of phenylacetate:phenylacetylglutamine ratio with plasma glutamine levels.
Topics: Ammonia; Argininosuccinic Aciduria; Chromatography, Liquid; Female; Glutamine; Glycerol; Humans; Lim | 2017 |
Safety and efficacy of glycerol phenylbutyrate for management of urea cycle disorders in patients aged 2months to 2years.
Topics: Ammonia; Child, Preschool; Cough; Disease Management; Drug-Related Side Effects and Adverse Reaction | 2017 |
Efficacy of peritoneal dialysis in neonates presenting with hyperammonaemia due to urea cycle defects and organic acidaemia.
Topics: Amino Acid Metabolism, Inborn Errors; Ammonia; Female; Humans; Hyperammonemia; Infant, Newborn; Male | 2019 |
Encephalopathy mimicking non-convulsive status Epilepticus.
Topics: Adult; Ammonia; Brain Diseases; Diagnosis, Differential; Electroencephalography; Female; Humans; Mal | 2018 |
Ammonia Scavenging Prevents Progression of Fibrosis in Experimental Nonalcoholic Fatty Liver Disease.
Topics: Ammonia; Animals; Cells, Cultured; Disease Models, Animal; Disease Progression; Female; Humans; Live | 2020 |
Urea cycle disorders: a life-threatening yet treatable cause of metabolic encephalopathy in adults.
Topics: Adult; Ammonia; Arginine; Brain Diseases, Metabolic; Diet, Protein-Restricted; Fat Emulsions, Intrav | 2015 |
Blood ammonia and glutamine as predictors of hyperammonemic crises in patients with urea cycle disorder.
Topics: Adolescent; Adult; Ammonia; Child; Child, Preschool; Female; Glutamine; Humans; Hyperammonemia; Infa | 2015 |
Unusual cause of general malaise: a young woman with ornithine transcarbamylase deficiency.
Topics: Adult; Ammonia; Arginine; Female; Humans; Ornithine Carbamoyltransferase Deficiency Disease; Phenylb | 2016 |
A rare cause of postpartum coma: isolated hyperammonemia due to urea cycle disorder.
Topics: Ammonia; Coma; Female; Humans; Hyperammonemia; Ornithine Carbamoyltransferase Deficiency Disease; Po | 2016 |
Fatal hyperammonemia after renal transplant due to late-onset urea cycle deficiency: a case report.
Topics: Age of Onset; Ammonia; Circle of Willis; Encephalocele; Exons; Gene Amplification; Humans; Immunosup | 2010 |
Urinary 2-hydroxy-5-oxoproline, the lactam form of α-ketoglutaramate, is markedly increased in urea cycle disorders.
Topics: Adult; Ammonia; Child; Child, Preschool; Gas Chromatography-Mass Spectrometry; Humans; Hydroxyprolin | 2011 |
Assessment of plasma ammonia and glutamine concentrations in urea cycle disorders.
Topics: Ammonia; Glutamine; Humans; Infant, Newborn; Urea Cycle Disorders, Inborn | 2011 |
Threshold for toxicity from hyperammonemia in critically ill children.
Topics: Ammonia; Brain Edema; Child; Child, Preschool; Critical Illness; Encephalocele; Female; Humans; Hype | 2012 |
Long-term outcome and intervention of urea cycle disorders in Japan.
Topics: Age of Onset; Ammonia; Female; Humans; Japan; Male; Ornithine Carbamoyltransferase Deficiency Diseas | 2012 |
A rare case of hyperammonemia complication of high-protein parenteral nutrition.
Topics: Adult; Ammonia; Brain Edema; Dietary Proteins; Fatal Outcome; Humans; Hyperammonemia; Male; Parenter | 2013 |
Rare disease clinical research network's urea cycle consortium delivers a successful clinical trial to improve alternate pathway therapy.
Topics: Ammonia; Female; Glycerol; Humans; Male; Phenylbutyrates; Urea Cycle Disorders, Inborn | 2013 |
Integrating cellular metabolism into a multiscale whole-body model.
Topics: Acetaminophen; Allopurinol; Ammonia; Chemical and Drug Induced Liver Injury; Computational Biology; | 2012 |
Efficacy of clonidine in hyperammonemia induced hyperexcitability syndrome.
Topics: Adrenergic alpha-Agonists; Ammonia; Brain Chemistry; Clonidine; Glucose; Glutamic Acid; Humans; Hype | 2013 |