Phenylketonuria
82
12
15
53
Key Insights
Highlights
Success Rate
85% trial completion
Published Results
23 trials with published results (28%)
Research Maturity
53 completed trials (65% of total)
Clinical Risk Assessment
Based on trial outcomes
Moderate Risk
Score: 49/100
11.0%
9 terminated out of 82 trials
85.5%
-1.1% vs benchmark
22%
18 trials in Phase 3/4
43%
23 of 53 completed with results
Key Signals
Data Visualizations
Phase Distribution
Trial Status
Trial Success Rate
Benchmark: 86.5%
Based on 53 completed trials
Clinical Trials (82)
A Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of AG-181 in Subjects With Phenylketonuria
Impact Of A Phe-Restricted Diet On Gut Health In Children With PKU
A Study of Sepiapterin in Participants With Phenylketonuria (PKU)
A Trial to Examine if Repinatrabit is Processed Differently in Adults With Reduced Liver or Kidney Function Compared to Adults With Normal Liver and Kidney Function
First-in-Human, Multiple Part Clinical Study of JNT-517 in Healthy Participants and in Participants With Phenylketonuria
Eating Disorders in Patients With Phenylketonuria
A Study of JNT-517 in Participants With Phenylketonuria (PKU)
GenSci144 Tablets Phase I Clinical Trial
Announcement of Rare Metabolic Diseases in Systematic Newborn Screening: the Phenylketonuria Experience.
A Long-Term Safety Study of PTC923 in Participants With Phenylketonuria
Immune Modulation During Palynziq® Treatment in Adults (IMPALA)
Educational, Social Support, and Nutritional Interventions and Their Cumulative Effect on Pregnancy Outcomes and Quality of Life in Teen and Adult Women With Phenylketonuria
Effect of Different Meal Types Given Before Exercise on Plasma Amino Acid Levels and Metabolic Control Parameters in Classical Phenylketonuria Patients Undergoing Aerobic and Resistance Exercises
Efficacy and Safety of Orally Administered Engineered Probiotics (CBT102-A) for the Treatment of Children With Phenylketonuria
Study of Low-grade Systemic Inflammation in Adult Patients With Phenylketonuria
GMP Powdered Substitutes in PKU and TYR
A Phase 1/Phase 2 Open-label Study to Evaluate the Safety, Tolerability, and Efficacy of a Single Intravenous Administration of SAR444836 in Adult Participants With Phenylketonuria
Effect of L-carnitine Supplementation on Phenylalanine and Brain-derived Neurotrophic Factor Levels in Infants and Children With Phenylketonuria
Rapid Drug Desensitization Study in Adults Experiencing Hypersensitivity Reactions to Palynziq
A Dose-finding Study to Evaluate mRNA-3210 in Participants With Phenylketonuria