Key Insights

Highlights

Success Rate

90% trial completion (above average)

Clinical Risk Assessment

Based on trial outcomes

Low Risk

Score: 27/100

Termination Rate

4.3%

1 terminated out of 23 trials

Success Rate

90.0%

+3.5% vs benchmark

Late-Stage Pipeline

22%

5 trials in Phase 3/4

Results Transparency

44%

4 of 9 completed with results

Key Signals

4 with results90% success

Data Visualizations

Phase Distribution

13Total
Not Applicable (3)
Early P 1 (1)
P 1 (2)
P 2 (2)
P 3 (4)
P 4 (1)

Trial Status

Completed9
Recruiting6
Active Not Recruiting3
Not Yet Recruiting2
Unknown1
Enrolling By Invitation1

Trial Success Rate

90.0%

Benchmark: 86.5%

Based on 9 completed trials

Clinical Trials (23)

Showing 20 of 20 trials
NCT07484945RecruitingPrimary

Multiomics Approach in Adult Patients With Phenylketonuria

NCT06628128Phase 3RecruitingPrimary

A Long-Term Study of JNT-517 in Participants With Phenylketonuria

NCT06637514Phase 2RecruitingPrimary

A Phase 2 Study of JNT-517 in Adolescent Participants With Phenylketonuria

NCT05813678RecruitingPrimary

A Long-term, Post-marketing Safety Study of Palynziq in Patients With PKU (PALace)

NCT06305234RecruitingPrimary

A Long Term, Post-marketing Study of Immune Response in Patients Receiving Palynziq Treatment for PKU (PALisade)

NCT07406009Not Yet RecruitingPrimary

The Psychosocial Functioning of Adults With Phenylketonuria.

NCT07318909Early Phase 1Not Yet RecruitingPrimary

To Evaluate the Safety and Efficacy of GS1168 Injection in Adult Phenylketonuria

NCT07255599Phase 1Active Not RecruitingPrimary

Evaluation of BH4 Responsiveness in Our PKU Patients

NCT06694662Not ApplicableCompletedPrimary

PKU EASY Microtabs Plus - Acceptability and Tolerance

NCT05270837Phase 3Active Not RecruitingPrimary

Study to Evaluate the Safety and Efficacy of Pegvaliase in Adolescents (Ages 12-17) With Phenylketonuria

NCT06969209RecruitingPrimary

Brain Aging in Phenylketonuria

NCT06940193Enrolling By InvitationPrimary

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

NCT06792240Phase 2CompletedPrimary

Iron Supplements Effect on B-Phe Levels in PKU Patients During 3 Years Study

NCT04480567Phase 1Active Not RecruitingPrimary

AAV Gene Therapy Study for Subjects with PKU

NCT04086511CompletedPrimary

PANDA: PKU Amino Acid Evaluation

NCT03694353Phase 3CompletedPrimary

Safety and Efficacy of Self Administered Injections of Pegvaliase (>40mg/Day Dose) in Adults With PKU

NCT01889862Phase 3CompletedPrimary

Phase 3 Study to Evaluate the Efficacy & Safety of Self-Administered Injections of BMN165 by Adults With PKU

NCT02009904CompletedPrimary

Simple Breath Test to Examine Phenylalanine Metabolism

NCT03505125CompletedPrimary

A Non-Interventional Interview Study of Phenylketonuria (PKU) Adults

NCT03309345UnknownPrimary

Body Composition, Energy Intake and Expenditure in People With Phenylketonuria

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