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Showing 1–24 of 28 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Ornithine Transcarbamylase Deficiency Disease Clear all
Terminated Phase 1Phase 2 Interventional Accepts healthy volunteers Results available

Manipulating the Gut Microbiome Study

NCT03181828
View directory record Official record
Conditions
Urea Cycle Disorder
Interventions
Acetohydroxamic Acid Oral Tablet, No treatment
Drug · Other
Lead sponsor
Nicholas Ah Mew
Other
Eligibility
18 Years to 60 Years
Enrollment
4 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2017 – 2018
U.S. locations
1
States / cities
Washington D.C., District of Columbia
Source: ClinicalTrials.gov public record
Updated Dec 14, 2021 · Synced Jul 23, 2026, 12:39 PM EDT
Terminated No phase listed Observational Accepts healthy volunteers

The NIH UNI Study: Urea Cycle Disorders, Nutrition and Immunity

NCT01421888
View directory record Official record
Conditions
Argininosuccinic Aciduria, Carbamoyl-Phosphate Synthase I Deficiency, Citrullinemia, Ornithine Carbamoyltransferase Deficiency, Hyperargininemia, N-Acetylglutamate Synthase Deficiency
Interventions
Not listed
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
2 Years to 99 Years
Enrollment
4 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2011 – 2013
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Dec 8, 2019 · Synced Jul 23, 2026, 12:39 PM EDT
Completed Phase 2 Interventional Results available

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

NCT01599286
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Feb 14, 2021 · Synced Jul 23, 2026, 12:39 PM EDT
Completed Phase 1 Interventional

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

NCT04442347
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Aug 1, 2024 · Synced Jul 23, 2026, 12:39 PM EDT
Recruiting No phase listed Observational

Systemic Biomarkers of Brain Injury From Hyperammonemia

NCT04602325
View directory record Official record
Conditions
Urea Cycle Disorder, Organic Acidemia, Maple Syrup Urine Disease, Glutaric Acidemia I, Fatty Acid Oxidation Disorder, Hypoxic-Ischemic Encephalopathy
Interventions
Not listed
Lead sponsor
Children's National Research Institute
Other
Eligibility
7 Years to 18 Years
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2027
U.S. locations
1
States / cities
Washington D.C., District of Columbia
Source: ClinicalTrials.gov public record
Updated Feb 6, 2024 · Synced Jul 23, 2026, 12:39 PM EDT
Active, not recruiting No phase listed Observational

Long Term Follow Up to Evaluate DTX301 in Adults With Late-Onset OTC Deficiency

NCT03636438
View directory record Official record
Conditions
Ornithine Transcarbamylase (OTC) Deficiency
Interventions
No Intervention
Other
Lead sponsor
Ultragenyx Pharmaceutical Inc
Industry
Eligibility
18 Years and older
Enrollment
11 participants
Timeline
2018 – 2029
U.S. locations
4
States / cities
Aurora, Colorado • Boston, Massachusetts • New York, New York + 1 more
Source: ClinicalTrials.gov public record
Updated May 31, 2026 · Synced Jul 23, 2026, 12:39 PM EDT
Terminated No phase listed Observational

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

NCT04909346
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Jun 11, 2023 · Synced Jul 23, 2026, 12:39 PM EDT
Terminated No phase listed Observational

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

NCT04717453
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Feb 17, 2022 · Synced Jul 23, 2026, 12:39 PM EDT
Completed No phase listed Observational

Age-related Macular Degeneration: Detection of Onset of New Choroidal Neovascularization (AMD DOC Study)

NCT00417846
View directory record Official record
Conditions
Maculopathy, Age-Related, Choroidal Neovascularization
Interventions
Not listed
Lead sponsor
Johns Hopkins University
Other
Eligibility
50 Years and older
Enrollment
98 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2009
U.S. locations
4
States / cities
Beverly Hills, California • Baltimore, Maryland • Boston, Massachusetts + 1 more
Source: ClinicalTrials.gov public record
Updated Oct 4, 2009 · Synced Jul 23, 2026, 12:39 PM EDT
Completed Not applicable Interventional Results available

Low Cost OCT for Point of Care

NCT03323307
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Dec 4, 2019 · Synced Jul 23, 2026, 12:39 PM EDT
Active, not recruiting No phase listed Observational

Liver Disease in Urea Cycle Disorders

NCT04612764
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Apr 26, 2026 · Synced Jul 23, 2026, 12:39 PM EDT
Completed Phase 2 Interventional

Pilot Study For Hypothermia Treatment In Hyperammonemic Encephalopathy In Neonates And Very Young Infants

NCT01624311
View directory record Official record
Conditions
Urea Cycle Disorders, Organic Acidemias
Interventions
Therapeutic Hypothermia, Standard of Care Therapy
Other
Lead sponsor
Uta Lichter-Konecki
Other
Eligibility
1 Day to 30 Days
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2015
U.S. locations
3
States / cities
Washington D.C., District of Columbia • New York, New York • Milwaukee, Wisconsin
Source: ClinicalTrials.gov public record
Updated May 31, 2015 · Synced Jul 23, 2026, 12:39 PM EDT
Recruiting No phase listed Observational

Abbott Vascular Medical Device Registry

NCT04573660
View directory record Official record
Conditions
Acute Myocardial Infarction, Restenoses, Coronary, Coronary Artery Lesions, Venous Embolism, Arterial Embolism
Interventions
Coronary and peripheral stents, Pacing catheters, Vascular plugs, Measurement and imaging (FFR and OCT), Peripheral dilatation catheters, Coronary dilatation catheters, Coronary and peripheral guidewires, Vessel closure/compression devices, Vascular access introducers
Device
Lead sponsor
Abbott Medical Devices
Industry
Eligibility
18 Years and older
Enrollment
3,784 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2031
U.S. locations
7
States / cities
Birmingham, Alabama • Little Rock, Arkansas • Los Angeles, California + 4 more
Source: ClinicalTrials.gov public record
Updated Jul 2, 2025 · Synced Jul 23, 2026, 12:39 PM EDT
Terminated No phase listed Observational Results available

Observational Study That Will Collect Information on Patients With Urea Cycle Disorders (UCDs)

NCT01948427
View directory record Official record
Conditions
Urea Cycle Disorder
Interventions
Not listed
Lead sponsor
Amgen
Industry
Eligibility
Not listed
Enrollment
203 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2020
U.S. locations
1
States / cities
Blue Bell, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jun 30, 2024 · Synced Jul 23, 2026, 12:39 PM EDT
Terminated Phase 2 Interventional

UNLOCKED: A Phase 2, Open-label Trial With KB195 in Subjects With a Urea Cycle Disorder

NCT03933410
View directory record Official record
Conditions
Urea Cycle Disorder
Interventions
KB195
Drug
Lead sponsor
Kaleido Biosciences
Industry
Eligibility
18 Years to 65 Years
Enrollment
13 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2021
U.S. locations
6
States / cities
Washington D.C., District of Columbia • Tampa, Florida • Baltimore, Maryland + 3 more
Source: ClinicalTrials.gov public record
Updated Jan 24, 2022 · Synced Jul 23, 2026, 12:39 PM EDT
Completed No phase listed Observational Accepts healthy volunteers Results available

Neurologic Injuries in Adults With Urea Cycle Disorders

NCT00472732
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Jun 23, 2015 · Synced Jul 23, 2026, 12:39 PM EDT
Completed Phase 1Phase 2 Interventional Results available

Safety and Dose-Finding Study of DTX301 (scAAV8OTC) in Adults With Late-Onset Ornithine Transcarbamylase (OTC) Deficiency

NCT02991144
View directory record Official record
Conditions
Ornithine Transcarbamylase (OTC) Deficiency
Interventions
scAAV8OTC, Reactive Corticosteroid Taper Regimen, Prophylactic Corticosteroid Taper Regimen
Genetic · Drug
Lead sponsor
Ultragenyx Pharmaceutical Inc
Industry
Eligibility
18 Years and older
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2021
U.S. locations
4
States / cities
Aurora, Colorado • Boston, Massachusetts • New York, New York + 1 more
Source: ClinicalTrials.gov public record
Updated Jan 25, 2023 · Synced Jul 23, 2026, 12:39 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

A Study to Assess Plasma Ammonia Time-Normalized Area Under the Curve and Rate of Ureagenesis in Healthy Adult Subjects

NCT04269122
View directory record Official record
Conditions
Ornithine Transcarbamylase Deficiency
Interventions
No Intervention
Other
Lead sponsor
Ultragenyx Pharmaceutical Inc
Industry
Eligibility
18 Years to 55 Years
Enrollment
120 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2019 – 2020
U.S. locations
1
States / cities
Austin, Texas
Source: ClinicalTrials.gov public record
Updated Mar 29, 2020 · Synced Jul 23, 2026, 12:39 PM EDT
Terminated Phase 1 Interventional

Hepatocyte Transplantation for Liver Based Metabolic Disorders

NCT01345578
View directory record Official record
Conditions
Metabolic Diseases
Interventions
human hepatocyte transplantation, Preparative Radiation Therapy
Drug · Radiation
Lead sponsor
Ira Fox
Other
Eligibility
Up to 21 Years
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2022
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Dec 6, 2022 · Synced Jul 23, 2026, 12:39 PM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

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

NCT01568749
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Nov 23, 2014 · Synced Jul 23, 2026, 12:39 PM EDT
Not listed Phase 1 Interventional Accepts healthy volunteers

Study of Treatment and Metabolism in Patients With Urea Cycle Disorders

NCT00004307
View directory record Official record
Conditions
Amino Acid Metabolism, Inborn Errors
Interventions
Protein and calorie controlled diet, Ornithine transcarbamylase vector
Behavioral · Genetic
Lead sponsor
National Center for Research Resources (NCRR)
NIH
Eligibility
6 Months to 64 Years
Enrollment
66 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
Started 1999
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Jun 23, 2005 · Synced Jul 23, 2026, 12:39 PM EDT
Recruiting Phase 1Phase 2 Interventional

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

NCT06255782
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Apr 14, 2026 · Synced Jul 23, 2026, 12:39 PM EDT
Recruiting Phase 2 Interventional

A Study to Evaluate the Pharmacodynamics and Safety of ARCT-810 in Participants With OTCD

NCT06488313
View directory record Official record
Conditions
OTC Deficiency, Ornithine Transcarbamylase Deficiency, OTCD
Interventions
ARCT-810
Biological
Lead sponsor
Arcturus Therapeutics, Inc.
Industry
Eligibility
12 Years and older
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
1
States / cities
Chevy Chase, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 16, 2025 · Synced Jul 23, 2026, 12:39 PM EDT
Recruiting No phase listed Observational

Hepatic Histopathology in Urea Cycle Disorders

NCT04908319
View directory record Official record
Conditions
Urea Cycle Disorder, Ornithine Transcarbamylase Deficiency, Citrullinemia 1, ARGI Deficiency, ASL Deficiency, Argininosuccinic Aciduria, ASS Deficiency, Hyperargininemia, Carbamyl Phosphate Synthetase Deficiency, NAGS Deficiency
Interventions
Not listed
Lead sponsor
Baylor College of Medicine
Other
Eligibility
Not listed
Enrollment
70 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2026
U.S. locations
2
States / cities
Washington D.C., District of Columbia • Houston, Texas
Source: ClinicalTrials.gov public record
Updated Jul 19, 2025 · Synced Jul 23, 2026, 12:39 PM EDT