Phenylketonurias
56
8
9
31
Key Insights
Highlights
Success Rate
82% trial completion
Clinical Risk Assessment
Based on trial outcomes
High Risk
Score: 72/100
12.5%
7 terminated out of 56 trials
81.6%
-4.9% vs benchmark
5%
3 trials in Phase 3/4
13%
4 of 31 completed with results
Key Signals
Data Visualizations
Phase Distribution
Trial Status
Trial Success Rate
Benchmark: 86.5%
Based on 31 completed trials
Clinical Trials (56)
Evaluation of PKU Sphere in Italy
Open-label, Randomized, 2-way Crossover, Monocentric, Controlled Study to Evaluate the Effect on Daily PHE Fluctuation of PKU GOLIKE Versus SoC in Patients With PKU.
Safety and Efficacy Study of NGGT002 in PKU Adult Subjects
Evaluation of Phe Fluctuation in PKU Pts Treated With PKU GOLIKE Versus Standard Amino Acid Protein Substitute.
Evaluation of the Express Plus Range
Nutritional Impacts of Palynziq on Patients With Phenylketonuria (PKU)
Preliminary Study of Brain Effects of Palynziq-Related Changes in Phenylalanine in Individuals With PKU
Development of Novel Psychological Assessment Tools and Anxiety Intervention for Phenylketonuria
AAV Gene Therapy Clinical Study in Adult Classic PKU (PHEdom)
Evaluation of PKU Explore France
PheCheck Feasibility Study
The Effects of CGMP in Children and Adults With PKU
Metabolic Control and Patient Well-being in Phenylketonuria: do Guidelines Make a Difference?
Effect of Large Neutral Amino Acids in Adults With Classical Phenylketonuria
Early Check: Expanded Screening in Newborns
Amino Acid Kinetics of GMP-AA in Healthy Human Volunteers
PKU Low Calorie Drink Study
Walking Program in Fatty Liver Children With Phenylketonuria
Safety and Efficacy Study of NGGT002 in cPKU Adult Subjects
Evaluation of Low Phenylalanine Formulas