Clinical Trial Search

Primary: Phenylalanine Level in the Brain as Determined by MR Spectroscopy and in Blood — 130; 137; 961 umol/L

Primary: Change in the Plasma Phenylalanine Concentration of PKU Subjects Fed the Glycomacropeptide Diet Compared With the Change When Fed the Amino Acid Diet — 62; -85 micro…

Primary: Amount of Dietary Supplemented Phenylalanine (Phe)Tolerated in Children With Phenylketonuria — 20.9; 2.9 mg/kg/day — p=<0.001

Primary: Change in Blood Phenylalanine Levels From Baseline to Week 6. — 843; 888; 607; 891 micromole per liter — p=<0.001

Primary: Plasma Phenylalanine Level (mg/dl) — 18.0; 17.2 mg/dl — p=>0.05

Primary: Tabulation of the Incidence and Frequency of All AEs and SAEs That Occur Throughout the Study. — 83.8; 33.3; 6.3; 0.9 percentage of subjects reporting events

Primary: Dietary Phenylalanine (Phe) Tolerance at Week 26 — 80.6; 50.1 mg/kg/day

Primary: Percent Change in Blood Phenylalanine — -13.71; -8.13; 48.75 Percent change in blood phenylalanine

Primary: Blood Phenylalanine Concentration — 1482.1; 1566.0 umol/L

Primary: Blood Phe After 7 Days of Each Treatment — 357.5; 294.0; 346.8; 442.4 µmol/L

Primary: Change in Attention-Deficit Hyperactivity Disorder Rating Scale-IV (ADHD-RS) / Adult ADHD Self-Report Scale (ASRS) Total Score From Baseline to Week 13 — -4.9; -9.1…

Primary: Change From Baseline in BMI at 12 Months — -0.65574 kg/m^2 — p=0.692782

Primary: Part 2 Double-blind Phase: Mean Change From Baseline in Blood Phenylketonuria (Phe) Level to Weeks 5 and 6 (Averaged Over a 2-week Period) in Participants With Phe…

Primary: Blood Phenylalanine(Phe) Levels Measured at Specified Timepoints — 132.9; 72.2; 315.0; 104.1 μmol/L

Primary: Number of Participants With 20% Decrease in Phenylalanine Levels From Baseline (Positive Response) — 0; 0; 0 Participants

Primary: Percentage of Participants With at Least 30 Percent Reduction From Baseline in Blood Phenylalanine (Phe) Level — 75 Percentage of participants

Primary: Number of Participants With Treatment-Emergent Adverse Events (TEAEs) — 2; 2; 4; 3 Participants

Primary: Blood Phenylalanine Concentrations — 1382.7; 1336.1; 1290.5; 846.0 umol/L

Primary: Full-Scale Intelligence Quotient (FSIQ) Score — 101.06; 103.64; 104.69; 103.78 score on a scale

Primary: Percentage of Participants With Response to Kuvan® (Sapropterin Dihydrochloride) Treatment — 33.3 percentage of participants

Primary: Change From Baseline in Rapid Visual Processing (RVP) Mean Response Latency (CANTAB Task) — -24.23; 18.03 Millisec

Primary: Blood Phenylalanine Concentration — 1168.8; 617.6 umol/L

Primary: Number of Treatment-emergent Adverse Events Assessed Using CTCAE v4.0 — 1.15; 1.38; 1.14; 0.86 Events per person — p=0.50

Primary: 0-24 Hour AUC Leucine (Samples Collected at 0, 2, 4, 8, 12, 16, 20, and 24 Hours) — 6217; 4616 micromoles*hour/L

Primary: Change From Baseline in the Blood Plasma Phenylalanine (Phe) Concentration at Part 2 — 596.8; 563.9; 410.9; 508.2 µmol/L — p=<0.0001

Primary: Change From Baseline in the Blood Phenylalanine (Phe) Concentration — 1022.4; -553.5; -653.3; -672.3 μmol/L

Primary: Number of Participants With Treatment-emergent Adverse Events (TEAEs) as Assessed by CTCAE v5.0 — 36; 17; 7; 1 Participants

Primary: Preschool Language Scales — 7.6 Raw score units / 16 weeks

Primary: Serum Melatonin at the End of 4 Weeks — 266.9; 205.7; 220.4; 301.2 pg/ml

Primary: Number of Participants With Hypersensitivity Adverse Reaction — 111; 119 Participants