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Urea Cycle Disorders

6
Pipeline Programs
3
Companies
10
Clinical Trials
0
Approved Products

Pipeline by Development Stage

Preclinical
Phase 1
Phase 1/2
Phase 2
Phase 2/3
Phase 3
On Market
0
0
0
2
0
3
1
Early DiscoveryClinical DevelopmentMarket

Competitive Landscape

3 companies ranked by most advanced pipeline stage

Amgen
AmgenTHOUSAND OAKS, CA
6 programs
2
3
1
HPN-100Phase 41 trial
HPN-100Phase 31 trial
HPN-100Phase 33 trials
HPN-100Phase 31 trial
HPN-100Phase 21 trial
+1 more programs
Active Trials
NCT00551200Completed14Est. Dec 2008
NCT00947544Completed17Est. Aug 2011
NCT00992459Completed46Est. Sep 2010
+5 more trials
Human BioSciences
Human BioSciencesWV - Martinsburg
2 programs
Ammonia Study DeviceN/A1 trial
Oxidative Stress, Inflammation and Acute Decompensation in Urea Cycle DisordersN/A1 trial
Active Trials
NCT06953505Enrolling By Invitation30Est. Aug 2027
NCT01541722Terminated10Est. Jul 2015
Oregon Therapeutics
Oregon TherapeuticsFrance - Paris
1 program
Ammonia Study DeviceN/A

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
2027
AmgenHPN-100
AmgenHPN-100
AmgenHPN-100
AmgenHPN-100
AmgenHPN-100
AmgenHPN-100
AmgenHPN-100
AmgenHPN-100
Human BioSciencesAmmonia Study Device
Human BioSciencesOxidative Stress, Inflammation and Acute Decompensation in Urea Cycle Disorders

Clinical Trials (10)

Total enrollment: 575 patients across 10 trials

NCT01257737AmgenHPN-100

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

Start: Oct 2010Est. completion: Feb 201788 patients
Phase 4Completed
NCT01347073AmgenHPN-100

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

Start: Jul 2011Est. completion: Mar 201323 patients
Phase 3Completed
NCT00947297AmgenHPN-100

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

Start: Nov 2009Est. completion: Nov 201160 patients
Phase 3Completed
NCT00992459AmgenHPN-100

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

Start: Oct 2009Est. completion: Sep 201046 patients
Phase 3Completed
NCT00947544AmgenHPN-100

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

Start: Mar 2010Est. completion: Aug 201117 patients
Phase 2Completed
NCT00999167AmgenHPN-100

A Study of Safety and Efficacy of HPN-100 in Subjects With Cirrhosis and Episodic Hepatic Encephalopathy

Start: Dec 2009Est. completion: Apr 2012189 patients
Phase 2Completed
NCT00551200AmgenHPN-100

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

Start: Oct 2007Est. completion: Dec 200814 patients
Phase 2Completed
NCT01135680AmgenHPN-100

Double-Blind Randomized Crossover Trial to Access Electrocardiogram Effects of HPN-100

Start: May 2010Est. completion: Sep 201098 patients
Phase 1Completed
NCT06953505Human BioSciencesAmmonia Study Device

At Home Ammonia Monitoring of Inborn Errors of Ammonia Metabolism

Start: Sep 2025Est. completion: Aug 202730 patients
N/AEnrolling By Invitation
NCT01541722Human BioSciencesOxidative Stress, Inflammation and Acute Decompensation in Urea Cycle Disorders

Oxidative Stress, Inflammation and Acute Decompensation in Urea Cycle Disorders

Start: Feb 2012Est. completion: Jul 201510 patients
N/ATerminated

Phase Legend

Preclinical— Lab & animal studies
Phase 1— Safety & dosing
Phase 2— Efficacy testing
Phase 3— Large-scale trials
On Market— Approved & available

Key Insights

3 late-stage (Phase 3) programs, potential near-term approvals
3 companies competing in this space

The information on this page is for informational purposes only and should not be used as a substitute for professional medical advice. Drug information is sourced from FDA, DailyMed, and other government databases. Adverse event data from FAERS does not establish causation. Always consult a healthcare professional for medical decisions.