VivoSeq
Metabolic & Endocrine

Urea Cycle Disorders

1

Pipeline Programs↗

2

Active Trials↗

2

Companies Involved↗

Urea Cycle Disorders is an early-stage landscape with 1 program across 2 companies. The most active players are Rebecca Ahrens-Nicklas, Amgen.

Intelligence

Pipeline Metrics

Competitive Density

16/100

Pipeline Velocity

11/100

Mech. Concentration

N/A

Completion Rate

85/100

Attrition Rate

13/100

Competition

Competitive Landscape

Bubble size = clinical trials

Approved
Phase 3
Phase 2
Phase 1
Preclinical

Key Players

Top Companies Companies ranked by the number of pipeline programs they run for this disease.

R

Rebecca Ahrens-Nicklas

1 program

PreclinicalAmgen logo

Amgen

0 programs

Preclinical

Pipeline

Programs by Phase

Preclinical1

Lnp.ucd.abe

Rebecca Ahrens-Nicklas

Phase 1
No programs
Phase 2
No programs
Phase 3
No programs
Approved
No programs

Analysis

Trial Status

15

clinical trials

Completed11 · 73%
Withdrawn1 · 7%
Recruiting1 · 7%
Terminated1 · 7%
Not yet recruiting1 · 7%

Clinical Trials

Trial Activity

15 trials

A Clinical Study of Glycerol Phenylbutyrate in Chinese Patients With Urea Cycle Disorders
Recruiting·TTongji Hospital·N/A·NCT06904027·40 enrolled
Master Protocol for a Phase I/II Open-label Safety and Efficacy Study of LNP.UCD.ABE, a Lipid Nanoparticle-delivered Base Editing Therapy, in Patients With Urea Cycle Disorders Due to Variants Amenable to Corrective Editing by LNP.UCD.ABE
Not yet recruiting·—·Preclinical·NCT07667387·7 enrolled
Comparative Efficacy of Phenylbutyrate (PBA) vs. Benzoate in Urea Cycle Disorders
Completed·—·N/A·NCT02111200·7 enrolled
Study of the Safety of HPN (Hyperion)-100 for the Long-Term Treatment of Urea Cycle Disorders (Treat UCD)
Completed·Amgen logoAmgen·Phase 3·NCT00947297·60 enrolled
To Evaluate the Safety of Long-term Use of HPN-100 in the Management of Urea Cycle Disorders (UCDs)
Completed·Amgen logoAmgen·Phase 4·NCT01257737·88 enrolled
Study of Glycerol Phenylbutyrate & Sodium Phenylbutyrate in Phenylbutyrate Naïve Patients With Urea Cycle Disorders (UCDs)
Completed·Amgen logoAmgen·Phase 4·NCT03335488·16 enrolled
Study of the Safety and Tolerability of HPN-100 Compared to Sodium Phenylbutyrate in Children With Urea Cycle Disorders
Completed·Amgen logoAmgen·Phase 2·NCT00947544·17 enrolled
Efficacy and Safety of HPN-100 for the Treatment of Adults With Urea Cycle Disorders
Completed·Amgen logoAmgen·Phase 3·NCT00992459·46 enrolled
Dose-Escalation Safety Study of HPN-100 to Treat Urea Cycle Disorders
Completed·Amgen logoAmgen·Phase 2·NCT00551200·14 enrolled
Study to Evaluate the Efficacy of HepaStem in Urea Cycle Disorders Paediatric Patients (HEP002)
Completed·CCellaion SA·Phase 2·NCT02489292·5 enrolled
Study of the Safety, Pharmacokinetics and Efficacy of HPN-100, in Pediatric Subjects With Urea Cycle Disorders (UCDs)
Completed·Amgen logoAmgen·Phase 3·NCT01347073·23 enrolled
A Study of the Safety, Efficacy and Pharmacokinetics of Glycerol Phenylbutyrate in Pediatric Subjects Under 2 Years of Age With Urea Cycle Disorders
Completed·Amgen logoAmgen·Phase 4·NCT02246218·27 enrolled
Human Heterologous Liver Cells for Infusion in Children With Urea Cycle Disorders
Completed·CCytonet GmbH & Co. KG·Phase 2·NCT00718627·12 enrolled
Human Heterologous Liver Cells for Infusion in Children With Urea Cycle Disorders
Terminated·CCytonet GmbH & Co. KG·Phase 2·NCT01195753·10 enrolled
A Safety and Efficacy Study of Infusions of HepaStem in Urea Cycle Disorders Pediatric Patients
Withdrawn·HHLB Cell Co., Ltd.·Phase 2·NCT03884959

Data sources: ClinicalTrials.gov (NIH) · OpenFDA (FDA) · ChEMBL (EMBL-EBI) · Open Targets (EBI/Sanger) · MedlinePlus (NIH) · PubMed (NIH) · SEC EDGAR (SEC)