Phenylketonuria (PKU)
23
6
9
9
Key Insights
Highlights
Success Rate
90% trial completion (above average)
Clinical Risk Assessment
Based on trial outcomes
Low Risk
Score: 27/100
4.3%
1 terminated out of 23 trials
90.0%
+3.5% vs benchmark
22%
5 trials in Phase 3/4
44%
4 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 (23)
Multiomics Approach in Adult Patients With Phenylketonuria
A Long-Term Study of JNT-517 in Participants With Phenylketonuria
A Phase 2 Study of JNT-517 in Adolescent 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
Study to Evaluate the Safety and Efficacy of Pegvaliase in Adolescents (Ages 12-17) With Phenylketonuria
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
Phase 3 Study to Evaluate the Efficacy & Safety of Self-Administered Injections of BMN165 by Adults With PKU
Simple Breath Test to Examine Phenylalanine Metabolism
A Non-Interventional Interview Study of Phenylketonuria (PKU) Adults
Body Composition, Energy Intake and Expenditure in People With Phenylketonuria