Key Insights

Highlights

Success Rate

90% trial completion (above average)

Clinical Risk Assessment

Based on trial outcomes

Low Risk

Score: 27/100

Termination Rate

7.7%

2 terminated out of 26 trials

Success Rate

90.5%

+4.0% vs benchmark

Late-Stage Pipeline

15%

4 trials in Phase 3/4

Results Transparency

42%

8 of 19 completed with results

Key Signals

8 with results90% success

Data Visualizations

Phase Distribution

19Total
Not Applicable (6)
P 1 (2)
P 2 (7)
P 3 (3)
P 4 (1)

Trial Status

Completed19
Recruiting4
Terminated2
Enrolling By Invitation1

Trial Success Rate

90.5%

Benchmark: 86.5%

Based on 19 completed trials

Clinical Trials (26)

Showing 20 of 20 trials
NCT06904027RecruitingPrimary

A Clinical Study of Glycerol Phenylbutyrate in Chinese Patients With Urea Cycle Disorders

NCT05671666Not ApplicableRecruitingPrimary

Ureagenesis Analysis in Healthy Subjects and in Urea Cycle Disorder Patients

NCT06953505Not ApplicableEnrolling By InvitationPrimary

At Home Ammonia Monitoring of Inborn Errors of Ammonia Metabolism

NCT01257737Phase 4CompletedPrimary

To Evaluate the Safety of Long-term Use of HPN-100 in the Management of Urea Cycle Disorders (UCDs)

NCT01347073Phase 3CompletedPrimary

Study of the Safety, Pharmacokinetics and Efficacy of HPN-100, in Pediatric Subjects With Urea Cycle Disorders (UCDs)

NCT00947544Phase 2CompletedPrimary

Study of the Safety and Tolerability of HPN-100 Compared to Sodium Phenylbutyrate in Children With Urea Cycle Disorders

NCT00551200Phase 2CompletedPrimary

Dose-Escalation Safety Study of HPN-100 to Treat Urea Cycle Disorders

NCT00947297Phase 3CompletedPrimary

Study of the Safety of HPN (Hyperion)-100 for the Long-Term Treatment of Urea Cycle Disorders (Treat UCD)

NCT00992459Phase 3CompletedPrimary

Efficacy and Safety of HPN-100 for the Treatment of Adults With Urea Cycle Disorders

NCT02935283RecruitingPrimary

Neuroimaging and Neuropsychological Outcomes in Urea Cycle Disorders

NCT00986895Phase 1Completed

A Study of Glyceryl Tri-(4-phenylbutyrate) Administered Orally as a Single Dose, and Twice Daily for Seven Consecutive Days to Subjects With Hepatic Impairment With Cirrhosis and to a Control Group

NCT00237315Recruiting

Longitudinal Study of Urea Cycle Disorders

NCT02740153CompletedPrimary

PCORI Urea Cycle Disorder Study

NCT01765283Phase 1CompletedPrimary

Safety Study of HepaStem for the Treatment of Urea Cycle Disorders (UCD) and Crigler-Najjar Syndrome (CN)

NCT02489292Phase 2CompletedPrimary

Study to Evaluate the Efficacy of HepaStem in Urea Cycle Disorders Paediatric Patients (HEP002)

NCT02252770Not ApplicableCompleted

Nitric Oxide Supplementation in Argininosuccinic Aciduria

NCT03797131Not ApplicableCompletedPrimary

Clinical Food Study to Evaluate the Effect of KB195 on Gut Nitrogen Metabolism in Patients With Urea Cycle Disorders

NCT02051049CompletedPrimary

Long-term Safety Follow-up Study of Patients Having Received HepaStem (SAF001)

NCT02311283CompletedPrimary

Pilot Study: Urea Cycle Disorders Practice Patterns and Outcomes Assessment

NCT00345605Phase 2Completed

Arginine and Buphenyl in Patients With Argininosuccinic Aciduria (ASA), a Urea Cycle Disorder

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