PKU
15
1
2
9
Key Insights
Highlights
Success Rate
82% trial completion
Clinical Risk Assessment
Based on trial outcomes
High Risk
Score: 72/100
13.3%
2 terminated out of 15 trials
81.8%
-4.7% vs benchmark
7%
1 trials in Phase 3/4
0%
0 of 9 completed with results
Key Signals
Data Visualizations
Phase Distribution
Trial Status
Trial Success Rate
Benchmark: 86.5%
Based on 9 completed trials
Clinical Trials (15)
Evaluation of PKU Sphere in Italy
Evaluation of PKU UP
Evaluation of the Express Plus Range
PKU Protein Requirements
Rapid Drug Desensitization Study in Adults Experiencing Hypersensitivity Reactions to Palynziq
Determining the Most Efficient Screening Cut Off Levels for Phenylketonuria (PKU)
GMP Case Studies of Tolerance, Safety and Acceptability in PKU and TYR
Natural History Clinical Study in Adult PKU
Study of a National Cohort of Adult Patients With Phenylketonuria
Feeding Patterns and Practices in Young Children With PKU During the First Two Years of Life
Market Research - Acceptability Study for a New PKU Protein Substitute
Market Research - Acceptability Trial for a New PKU Amino Acid Based Protein Substitute
Antioxidant Signature in Adult Patients With Phenylketonuria
Neuroimaging and Neurocognitive Assessment and Response to Sapropterin Dihydrochloride Treatment in Phenylketonuria
Moderate Intensity Exercise and Phenylketonuria