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ClinicalTrials.gov public records Last synced Nov 15, 2026, 5:11 AM EST

All trial information on this site comes from the official ClinicalTrials.gov public API (clinicaltrials.gov/api/v2), run by the U.S. National Library of Medicine. We only display that public information; we do not add opinions, endorsements, or any other intent. Always review the official ClinicalTrials.gov record for the latest information.

Showing 25–48 of 60 matching trials from the live ClinicalTrials.gov search.

Filters: Condition: Urea Cycle Disorders, Inborn Clear all

Conditions: Ornithine Transcarbamylase Deficiency
Interventions: No Intervention; Other
Lead sponsor: Ultragenyx Pharmaceutical Inc; Industry
Eligibility: 18 Years and older
Enrollment: 1 participant
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2020 – 2021
U.S. locations: 1
States / cities: Orlando, Florida

Conditions: Urea Cycle Disorders
Interventions: MRI, Behavioral; Procedure · Behavioral
Lead sponsor: Children's National Research Institute; Other
Eligibility: 7 Years to 50 Years
Enrollment: 56 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2016 – 2025
U.S. locations: 1
States / cities: Washington D.C., District of Columbia

Conditions: Urea Cycle Disorders
Interventions: No Intervention Given; Other
Lead sponsor: Children's National Research Institute; Other
Eligibility: Up to 18 Years
Enrollment: 187 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2016 – 2020
U.S. locations: 1
States / cities: Washington D.C., District of Columbia

Conditions: Urea Cycle Disorders
Interventions: Not listed
Lead sponsor: Mark Batshaw; Other
Eligibility: Not listed
Enrollment: 10 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2012 – 2015
U.S. locations: 10
States / cities: Los Angeles, California • Aurora, Colorado • Washington D.C., District of Columbia + 7 more

Conditions: Urea Cycle Disorder
Interventions: RAVICTI, NaPBA; Drug
Lead sponsor: Amgen; Industry
Eligibility: Up to 99 Years
Enrollment: 16 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018 – 2022
U.S. locations: 6
States / cities: Gainesville, Florida • New York, New York • Cleveland, Ohio + 3 more

Conditions: Optical Coherence Tomography (OCT) of the Retina
Interventions: OCT imaging; Device
Lead sponsor: Duke University; Other
Eligibility: 18 Years and older
Enrollment: 70 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018
U.S. locations: 1
States / cities: Chapel Hill, North Carolina

Conditions: Analgesia
Interventions: Paracetamol, Paracetamol formulation 1, Paracetamol Formulation 2, Paracetamol formulation 3, Paracetamol formulation 4; Drug
Lead sponsor: GlaxoSmithKline; Industry
Eligibility: 18 Years to 50 Years
Enrollment: 14 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2008 – 2009
U.S. locations: 1
States / cities: Phoenix, Arizona

Conditions: Urea Cycle Disorder, Ornithine Transcarbamylase Deficiency, Citrullinemia 1, ARGI Deficiency, ASL Deficiency, Argininosuccinic Aciduria, ASS Deficiency, Hyperargininemia
Interventions: Not listed
Lead sponsor: Baylor College of Medicine; Other
Eligibility: 6 Years to 65 Years
Enrollment: 62 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2026
U.S. locations: 5
States / cities: Aurora, Colorado • Washington D.C., District of Columbia • Philadelphia, Pennsylvania + 2 more

Conditions: Ornithine Transcarbamylase Deficiency
Interventions: ARCT-810, Placebo; Biological · Other
Lead sponsor: Arcturus Therapeutics, Inc.; Industry
Eligibility: 18 Years and older
Enrollment: 16 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2020 – 2024
U.S. locations: 8
States / cities: Gainesville, Florida • Minneapolis, Minnesota • New York, New York + 5 more

Conditions: Argininosuccinate Lyase Deficiency, Urea Cycle Disorder, Urea Cycle Disorders, Inborn, Argininosuccinic Aciduria
Interventions: Neo-ASA, Placebo; Dietary Supplement
Lead sponsor: Baylor College of Medicine; Other
Eligibility: 6 Years to 50 Years
Enrollment: 16 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2017 – 2023
U.S. locations: 1
States / cities: Houston, Texas

Conditions: Brain Diseases, Metabolic, Inborn, Urea Cycle Disorder, Ornithine Transcarbamylase Deficiency
Interventions: Not listed
Lead sponsor: Andrea Gropman; Other
Eligibility: 18 Years to 60 Years
Enrollment: 46 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2007 – 2010
U.S. locations: 2
States / cities: Washington D.C., District of Columbia

Conditions: Citrullinemia
Interventions: stable isotope infusion; Other
Lead sponsor: Baylor College of Medicine; Other
Eligibility: 18 Years and older
Enrollment: 6 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2012 – 2013
U.S. locations: 1
States / cities: Houston, Texas

Conditions: Coronary Artery Disease
Interventions: OCT stent guidance; Device
Lead sponsor: Abbott Medical Devices; Industry
Eligibility: 18 Years and older
Enrollment: 418 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2012 – 2015
U.S. locations: 15
States / cities: Birmingham, Alabama • La Jolla, California • Orlando, Florida + 12 more

Conditions: Ornithine Transcarbamylase Deficiency, Ornithine Transcarbamylase Deficiency Disease, Ornithine Carbamoyltransferase Deficiency (Disorder), Urea Cycle Disorders, Inborn
Interventions: ECUR-506; Genetic
Lead sponsor: iECURE, Inc.; Industry
Eligibility: 24 Hours to 7 Months · Male only
Enrollment: 20 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2024 – 2027
U.S. locations: 6
States / cities: Los Angeles, California • Aurora, Colorado • Atlanta, Georgia + 3 more

Conditions: Urea Cycle Disorder
Interventions: Not listed
Lead sponsor: St. Jude Children's Research Hospital; Other
Eligibility: 16 Years to 40 Years
Enrollment: 60 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2026 – 2032
U.S. locations: 1
States / cities: Memphis, Tennessee

Conditions: Ornithine Transcarbamylase Deficiency, Ornithine Transcarbamylase Deficiency Disease, Urea Cycle Disorders, Inborn
Interventions: No Intervention; Other
Lead sponsor: iECURE, Inc.; Industry
Eligibility: 7 Months to 15 Months
Enrollment: 13 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2024 – 2041
U.S. locations: 2
States / cities: Aurora, Colorado • New York, New York

Conditions: Cancers
Interventions: Laparoscopic surgery; Procedure
Lead sponsor: Erin Rowell; Other
Eligibility: Up to 11 Years · Female only
Enrollment: 250 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018 – 2035
U.S. locations: 1
States / cities: Chicago, Illinois

Conditions: Arginase I Deficiency, Hyperargininemia
Interventions: Pegzilarginase, Placebo; Drug
Lead sponsor: Immedica Pharma AB; Industry
Eligibility: 2 Years and older
Enrollment: 32 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2019 – 2023
U.S. locations: 17
States / cities: Rogers, Arkansas • Orange, California • Stanford, California + 14 more

Conditions: Urea Cycle Disorders
Interventions: HPN-100, Buphenyl (NaPBA); Drug
Lead sponsor: Amgen; Industry
Eligibility: 18 Years and older
Enrollment: 46 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2009 – 2010
U.S. locations: 21
States / cities: Long Beach, California • Los Angeles, California • Stanford, California + 18 more

Conditions: Propionic Acidemia, Type I and/or Type II, Methylmalonic Acidemia, Carbamoyl-Phosphate Synthase I Deficiency Disease, Ornithine Carbamoyltransferase Deficiency
Interventions: Carbaglu, Placebo; Drug
Lead sponsor: Mendel Tuchman; Other
Eligibility: 1 Week to 99 Years
Enrollment: 35 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2012 – 2020
U.S. locations: 9
States / cities: Los Angeles, California • Palo Alto, California • Aurora, Colorado + 6 more

Conditions: Urea Cycle Disorders
Interventions: HPN-100, BUPHENYL®; Drug
Lead sponsor: Amgen; Industry
Eligibility: 18 Years and older
Enrollment: 14 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2007 – 2008
U.S. locations: 2
States / cities: Minneapolis, Minnesota • Houston, Texas

Conditions: Urea Cycle Disorders, Inborn
Interventions: Sodium Benzoate, Sodium Phenylbutyrate; Drug
Lead sponsor: Baylor College of Medicine; Other
Eligibility: 18 Years to 64 Years
Enrollment: 7 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2014 – 2015
U.S. locations: 1
States / cities: Houston, Texas

Conditions: Urea Cycle Disorders
Interventions: HPN-100; Drug
Lead sponsor: Amgen; Industry
Eligibility: 6 Years and older
Enrollment: 60 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2009 – 2011
U.S. locations: 21
States / cities: Long Beach, California • Los Angeles, California • Stanford, California + 18 more

Conditions: Ornithine Transcarbamylase Deficiency, Wilson Disease, Glycogen Storage Disease Type IA
Interventions: Not listed
Lead sponsor: Ultragenyx Pharmaceutical Inc; Industry
Eligibility: 18 Years and older
Enrollment: 51 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2022
U.S. locations: 1
States / cities: Akron, Ohio

Search by objective public record fields.

Study to Characterize Rate of Ureagenesis in Patients With Ornithine Transcarbamylase (OTC) Deficiency

Neuroimaging and Neuropsychological Outcomes in Urea Cycle Disorders

PCORI Urea Cycle Disorder Study

Oxidative Stress, Inflammation and Acute Decompensation in Urea Cycle Disorders

Study of Glycerol Phenylbutyrate & Sodium Phenylbutyrate in Phenylbutyrate Naïve Patients With Urea Cycle Disorders (UCDs)

Low Cost OCT for Point of Care

A Study Investigating the Pharmacokinetic Profiles of Four Extended Release Paracetamol Formulations

Liver Disease in Urea Cycle Disorders

Phase 1b Study to Assess Safety, Tolerability, and Pharmacokinetics of ARCT-810 in Stable Adult Subjects With Ornithine Transcarbamylase Deficiency

Nitric Oxide Supplementation on Neurocognitive Functions in Patients With ASLD

Neurologic Injuries in Adults With Urea Cycle Disorders

Nitric Oxide Flux and Ureagenesis in Argininosuccinate Synthetase Deficiency (ASSD)(Citrullinemia I)

Observational Study of OCT in a Patients Undergoing FFR

An Open-label Study to Investigate ECUR-506 in Male Babies Less Than 9 Months of Age With Neonatal Onset OTC Deficiency

Functional Evaluation in Patients With Urea Cycle Disorders (UCD) During a Driving Task

Long-term Follow-up (LTFU) Study of Participants in Any iECURE Protocol Using an Investigational Product (IP)

Ovarian Tissue Cryopreservation in Pre-Pubertal (OTC-Pre Pubertal)

Efficacy and Safety of Pegzilarginase in Patients With Arginase 1 Deficiency

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

Short-Term Outcome of N-Carbamylglutamate in the Treatment of Acute Hyperammonemia

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

Comparative Efficacy of Phenylbutyrate (PBA) vs. Benzoate in Urea Cycle Disorders

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

Adeno-Associated Virus (AAV) Antibody Study in Subjects OTC Deficiency, GSDIa, and Wilson Disease