27 closest matches · ranked by relevance
Hyperammonemia
Primary: Safety of TNP-2092 by Assessment of the Number of Participants With Adverse Events (AEs) — 6; 6; 7; 8 participants
Argininosuccinic Aciduria · Amino Acid Metabolism, Inborn Errors · Urea Cycle Disorders
Primary: Measures of Liver Function: AST and ALT — 36.2; 52; 31.7; 57.86 IU/L
Propionic Acidemia, Type I and/or Type II · Methylmalonic Acidemia · Carbamoyl-Phosphate Synthase I Deficiency Disease
Primary: Time to the Primary Outcome (Earlier of Ammonia <50 µmol/L or Hospital Discharge) — 1.37; 0.79 Hazard Ratio
Acute Liver Failure · Acute Liver Injury
Primary: Number of Participants That do Not Tolerate the Administered Dose and Had Grade 3 or 4 Treatment Emergent Adverse Events as a Measure of Safety and Tolerability — 0; 0…
Urea Cycle Disorders
Primary: Rate of Adverse Events (Number of Participants Who Experienced Any AE Considered Related to Study Drug) — 33 participants
Acute Episode of Overt Hepatic Encephalopathy · Hepatic Encephalopathy
Primary: Percentage of Participants in Each HE Stage — 11; 6; 30; 40 percentage of participants
Brain Diseases, Metabolic, Inborn · Urea Cycle Disorder · Ornithine Transcarbamylase Deficiency
Primary: Concentration of Glutamine and Myoinositol by MRS — 4.97; 3.66; 3.78; 4.50 mM — p=0.003
Hyperinsulinism-Hyperammonemia Syndrome
Primary: Tolerability of Vitamin E Based on Responses to a Subject/Parent-reported Symptom Questionnaire After Vitamin E Supplementation Compared to Baseline — 2 Participants
Urea Cycle Disorders
Primary: Mortality — 18.4; 20.7 participant per 1000 person-years
Urea Cycle Disorders
Primary: Venous Ammonia Levels at the Peak and Mean TNUAC Time-normalized Area Under the Curve) — 79.1; 56.3; 38.4; 26.5 μmol/L
Urea Cycle Disorders
Primary: Rate of Adverse Events During the Switchover Part of the Study Rate of Adverse Events (Number of Participants Showing Adverse Events) — 4; 2 participants
Urea Cycle Disorder
Primary: Rate of Treatment Success (Percentage of Participants Defined as Treatment Success at Week 4) During the Initial Treatment Period — 81.8; 80.0 percentage of participants…
Urea Cycle Disorders
Primary: The Primary Endpoint Was the 24-hour Area Under the Curve for Blood Ammonia (NH324-hour AUC) on Days 14 and 28. — 976.6; 865.35 μmol∙h/L
Urea Cycle Disorder
Primary: Liver Stiffness as Measured by Shear Wave Elastography — 13; 14 Participants
Maple Syrup Urine Disease
Primary: 0-24 Hour AUC Leucine (Samples Collected at 0, 2, 4, 8, 12, 16, 20, and 24 Hours) — 6217; 4616 micromoles*hour/L
BH4 Deficiency · Hyperphenylalaninemia
Primary: Number of Participants With Treatment-Emergent Adverse Events (TEAEs) — 2; 2; 4; 3 Participants
Urea Cycle Disorders, Inborn
Primary: Urinary Hippuric Acid — 16.4; 0; 10.5 mmol/24 hours
Urea Cycle Disorders
Primary: Adverse Events — 0; 6 participants
Cirrhosis · Hepatic Encephalopathy
Primary: Serious Adverse Events Related to FMT — 0; 0; 0; 0 Participants
Hyperoxaluria
Primary: Mean Percent Conversion of Hydroxyproline (Hyp) to Urinary Oxalate (UOx) — 12.8; 32.9; 14.8 percent converted
Chronic Traumatic Brain Injury
Primary: Change From Baseline of Concentrations of Essential Amino Acid at 1.5 Hours After Eating — 487.28; 553.70; 1224.19; 1339.54 µM
Ornithine Transcarbamylase Deficiency
Primary: Concentration of Glutamine and Myoinositol — 2.61; 1.66; 2.23; 2.69 mM — p=0.001
Tetrahydrobiopterin Deficiencies · Hyperphenylalaninemia, Non-Phenylketonuric
Primary: Blood Phenylalanine(Phe) Levels Measured at Specified Timepoints — 132.9; 72.2; 315.0; 104.1 μmol/L
Phenylketonuria
Primary: Number of Participants With 20% Decrease in Phenylalanine Levels From Baseline (Positive Response) — 0; 0; 0 Participants
Parenteral Nutrition-related Hepatitis
Primary: Disease Progression as Measured by Serum Levels of Hepatic Enzymes
Phenylketonuria
Primary: Phenylalanine Level in the Brain as Determined by MR Spectroscopy and in Blood — 130; 137; 961 umol/L
Pulmonary Hypertension · Sickle Cell Disease · Thalassemia
Primary: Erythrocyte Glutamine/Glutamate Ratio at 8 Weeks — 2.40 Ratio