Phenylketonuria (PKU)
27
7
10
10
Key Insights
Highlights
Success Rate
91% trial completion (above average)
Clinical Risk Assessment
Based on trial outcomes
Low Risk
Score: 27/100
3.7%
1 terminated out of 27 trials
90.9%
+4.4% vs benchmark
19%
5 trials in Phase 3/4
50%
5 of 10 completed with results
Key Signals
Data Visualizations
Phase Distribution
Trial Status
Trial Success Rate
Benchmark: 86.6%
Based on 10 completed trials
Clinical Trials (27)
Study to Evaluate the Safety and Efficacy of Pegvaliase in Adolescents (Ages 12-17) With Phenylketonuria
A Study of MZE782 in Adults With PKU
Prevalence and Determinants of Obesity in PKU Patients
A Clinical Study on the Safety and Tolerability of PL54 Injection in Adult Patients With Phenylketonuria (PKU)
PKU Microtablets Case Studies
A Phase 2 Study of JNT-517 in Adolescent Participants With Phenylketonuria
Multiomics Approach in Adult Patients With Phenylketonuria
A Long-Term Study of JNT-517 in Participants With Phenylketonuria
A Long-term, Post-marketing Safety Study of Palynziq in Patients With PKU (PALace)
A Long Term, Post-marketing Study of Immune Response in Patients Receiving Palynziq Treatment for PKU (PALisade)
The Psychosocial Functioning of Adults With Phenylketonuria.
To Evaluate the Safety and Efficacy of GS1168 Injection in Adult Phenylketonuria
Evaluation of BH4 Responsiveness in Our PKU Patients
PKU EASY Microtabs Plus - Acceptability and Tolerance
Brain Aging in Phenylketonuria
A Self-test Home-use Blood Phenylalanine Monitoring System Under the Brand Name Egoo Phe System Has Been Developed for Measurement of Phenylalanine (Phe) in Individuals Diagnosed With Phenylketonuria (PKU): The Study Purpose is to Evaluate Accuracy and Usability
Iron Supplements Effect on B-Phe Levels in PKU Patients During 3 Years Study
AAV Gene Therapy Study for Subjects with PKU
PANDA: PKU Amino Acid Evaluation
Safety and Efficacy of Self Administered Injections of Pegvaliase (>40mg/Day Dose) in Adults With PKU