30 closest matches · ranked by relevance
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
Arginase I Deficiency · Hyperargininemia
Primary: Change From Baseline in Plasma Arginine Concentration After 24 Weeks of Treatment — 0.244; 0.918 micromolar — p=<0.0001
Arginase 1 Deficiency
Primary: Change From Baseline in Plasma Arginine Concentrations in Subjects <24 Months of Age With Arginase 1 Deficiency (ARG1-D). — -221.53 μM
Anemia, Sickle Cell
Primary: Gardos Channel Activity — -0.0342; 0.0043; 0.1076 mmol/10^13 cells x min — p=0.080
Sickle Cell Disease · Vaso-occlusive Pain Episode
Primary: Total Parenteral Opioid Use in IV Morphine Equivalents — 1.77; 1.95; 2.36 mg/Kg
Asthma
Primary: Number of Asthma Exacerbations in Three Months — 30; 31 exacerbations
Asthma · Inflammation
Primary: Number of Acute Exacerbation at 3 Months — 2.7; 2.2; 1.6; 2.2 Events — p=0.78
Non Small Cell Lung Cancer · Urothelial Carcinoma · Malignant Melanoma
Primary: Adverse Events Evaluated by CTCAE 4.0 — 1; 7; 2; 0 Participants
Pulmonary Hypertension · Sickle Cell Disease · Thalassemia
Primary: Erythrocyte Glutamine/Glutamate Ratio at 8 Weeks — 2.40 Ratio
Urea Cycle Disorders
Primary: Mortality — 18.4; 20.7 participant per 1000 person-years
Glucose Transporter Type 1 Deficiency Syndrome · GLUT1 Deficiency Syndrome
Primary: Number of Participants With Reduction in Spike-wave Fraction of the EEG Recording Time — 13 Participants
Sickle Cell Disease
Primary: Time-to-crisis Resolution — 81.5; 88.6 hours
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
Metastatic Cancer · Solid Tumors · Colorectal Cancer (CRC)
Primary: Number of Participants With Any Treatment-emergent Adverse Event (TEAE) — 7; 7; 81; 7 Participants
Alpha 1-Antitrypsin Deficiency
Primary: Adverse Events Possibly, Probably or Definitely Related to Study Drug — 2; 2; 3; 2 participants
Schizophrenia
Primary: Change From Baseline in Mean Positive and Negative Syndrome Scale (PANSS) Total and Positive, Negative and General Psychopathology Subscale Scores at 3 Weeks — 71.6…
Medium-chain Acyl-CoA Dehydrogenase (MCAD) Deficiency
Primary: Metabolic Stress
Insulin Resistance · Endothelial Dysfunction
Primary: Change in Microvascular Blood Volume (MBV) During Insulin Infusion — 13.16; 11.3 ml/kg
Peroxisome Biogenesis Disorders
Primary: Peroxisome Biochemical Functions as Measured by Plasma Very Long Chain Fatty Acid — 0.180; 0.188 ratio
GTP Cyclohydrolase Deficiency
Primary: Change in BH4 Levels in Cerebral Spinal Fluid — 6; 10.7; 5.3; 13.2 nmol/L
Kidney Disease · Albuminuria
Primary: The Primary Outcome Measure is Level of Albuminuria. — 927.9; 897.1 ratio
Atrial Septal Defect · Ventricular Septal Defect · Atrioventricular Septal Defect
Primary: L-citrulline Plasma Levels — 200.9; 34.1; 2405.9; 29.5 µmol/L
Acute Lung Injury
Primary: A Composite Variable Consisting of the Longer of Either (1) Length of Time on Mechanical Ventilation or (2) Length of Inotrope Use. — 3351.1; 3549.6 Minutes — p=0.4930
Healthy
Primary: Change in Plasma 2-AAA Concentration From Baseline — -9.35; 7.02 ng/ml
Sickle Cell Disease
Primary: Number of Participants With Adverse Events — 4 Participants
Ornithine Transcarbamylase Deficiency
Primary: Concentration of Glutamine and Myoinositol — 2.61; 1.66; 2.23; 2.69 mM — p=0.001
Pompe Disease
Primary: Safety Assessments of the rAAV1-CMV-GAA (Study Agent), Changes Post Study Agent Administration. — 40,031; 29,638; 5,509,882; 1,907,161 mU/mL
Alkaptonuria
Primary: Homogentisic Acid Excretion — 1425.9; 113.1; 34.0 uM
Severe Sepsis · Acute Lung Injury
Primary: Vasopressor Dependency Index — 50.0; 44.4; 49.0 mmHg/catecholamine index — p=0.86
Hereditary Sensory and Autonomic Neuropathy Type I
Primary: Charcot Marie Tooth Neuropathy Score — 25.67; 20.22 scores on a scale