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Showing 25–48 of 24 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Urea Cycle Disorders, Inborn Clear all
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, 2:45 AM EDT
Active, not recruiting Phase 3 Interventional

Clinical Study of DTX301 AAV-Mediated Gene Transfer for Ornithine Transcarbamylase (OTC) Deficiency

NCT05345171
View directory record Official record
Conditions
OTC Deficiency
Interventions
DTX301, Placebo, Oral Corticosteroids, Placebo for oral corticosteroids, Sodium Acetate
Genetic · Other · Drug
Lead sponsor
Ultragenyx Pharmaceutical Inc
Industry
Eligibility
12 Years and older
Enrollment
37 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2031
U.S. locations
4
States / cities
Los Angeles, California • Aurora, Colorado • Chicago, Illinois + 1 more
Source: ClinicalTrials.gov public record
Updated Jun 1, 2026 · Synced Jul 23, 2026, 2:45 AM EDT
Completed Not applicable Interventional

Nitric Oxide Supplementation on Neurocognitive Functions in Patients With ASLD

NCT03064048
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Feb 26, 2023 · Synced Jul 23, 2026, 2:45 AM 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, 2:45 AM 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, 2:45 AM EDT
Completed Phase 4 Interventional Results available

A Study of the Safety, Efficacy and Pharmacokinetics of Glycerol Phenylbutyrate in Pediatric Subjects Under 2 Years of Age With Urea Cycle Disorders

NCT02246218
View directory record Official record
Conditions
Urea Cycle Disorder
Interventions
RAVICTI
Drug
Lead sponsor
Amgen
Industry
Eligibility
1 Day to 2 Years
Enrollment
27 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2017
U.S. locations
16
States / cities
Los Angeles, California • Palo Alto, California • Washington D.C., District of Columbia + 13 more
Source: ClinicalTrials.gov public record
Updated Jun 30, 2024 · Synced Jul 23, 2026, 2:45 AM EDT
Not listed No phase listed Observational Accepts healthy volunteers

Neuroimaging and Neuropsychological Outcomes in Urea Cycle Disorders

NCT02935283
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Jun 24, 2024 · Synced Jul 23, 2026, 2:45 AM 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, 2:45 AM EDT
Completed Phase 4 Interventional Results available

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

NCT03335488
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Jun 30, 2024 · Synced Jul 23, 2026, 2:45 AM 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, 2:45 AM 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, 2:45 AM EDT
Recruiting No phase listed Observational

Orphan Europe Carbaglu® Surveillance Protocol

NCT03409003
View directory record Official record
Conditions
N-acetylglutamate Synthase (NAGS) Deficiency
Interventions
Not listed
Lead sponsor
Nicholas Ah Mew
Other
Eligibility
Not listed
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2027
U.S. locations
3
States / cities
Washington D.C., District of Columbia • Boston, Massachusetts • New York, New York
Source: ClinicalTrials.gov public record
Updated Feb 6, 2024 · Synced Jul 23, 2026, 2:45 AM EDT
Completed No phase listed Observational Accepts healthy volunteers Results available

PCORI Urea Cycle Disorder Study

NCT02740153
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Jan 25, 2021 · Synced Jul 23, 2026, 2:45 AM EDT
Not yet recruiting Phase 1Phase 2 Interventional

A Phase I/II Open-label Safety and Efficacy Study of LNP.UCD.ABE in Patients With Urea Cycle Disorders.

NCT07667387
View directory record Official record
Conditions
Urea Cycle Disorders, Carbamoyl-Phosphate Synthase I Deficiency
Interventions
LNP.UCD.ABE
Biological
Lead sponsor
Rebecca Ahrens-Nicklas
Other
Eligibility
24 Hours to 5 Years
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2028
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jun 28, 2026 · Synced Jul 23, 2026, 2:45 AM EDT
Terminated Phase 2 Interventional

Human Heterologous Liver Cells for Infusion in Children With Urea Cycle Disorders

NCT01195753
View directory record Official record
Conditions
Urea Cycle Disorders
Interventions
HHLivC
Biological
Lead sponsor
Cytonet GmbH & Co. KG
Industry
Eligibility
1 Day to 5 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2015
U.S. locations
4
States / cities
Palo Alto, California • San Diego, California • New Haven, Connecticut + 1 more
Source: ClinicalTrials.gov public record
Updated Feb 7, 2016 · Synced Jul 23, 2026, 2:45 AM EDT
Completed Phase 4 Interventional Results available

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

NCT01257737
View directory record Official record
Conditions
Urea Cycle Disorders
Interventions
HPN-100
Drug
Lead sponsor
Amgen
Industry
Eligibility
1 Year and older
Enrollment
88 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2017
U.S. locations
15
States / cities
Los Angeles, California • Palo Alto, California • Aurora, Colorado + 12 more
Source: ClinicalTrials.gov public record
Updated Aug 21, 2024 · Synced Jul 23, 2026, 2:45 AM EDT
Completed Phase 2 Interventional

A Study of AEB1102 (Pegzilarginase) in Patients With Arginase I Deficiency

NCT03378531
View directory record Official record
Conditions
Arginase I Deficiency, Hyperargininemia
Interventions
AEB1102
Drug
Lead sponsor
Aeglea Biotherapeutics
Industry
Eligibility
2 Years and older
Enrollment
14 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2022
U.S. locations
4
States / cities
Stanford, California • Gainesville, Florida • New York, New York + 1 more
Source: ClinicalTrials.gov public record
Updated Jul 26, 2023 · Synced Jul 23, 2026, 2:45 AM EDT
Completed Phase 2 Interventional Results available

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

NCT00947544
View directory record Official record
Conditions
Urea Cycle Disorders
Interventions
HPN-100, NaPBA
Drug
Lead sponsor
Amgen
Industry
Eligibility
6 Years to 17 Years
Enrollment
17 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2011
U.S. locations
7
States / cities
Los Angeles, California • Washington D.C., District of Columbia • New York, New York + 4 more
Source: ClinicalTrials.gov public record
Updated Jul 10, 2024 · Synced Jul 23, 2026, 2:45 AM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

Safety and Tolerability of SYNB1020-CP-001

NCT03179878
View directory record Official record
Conditions
Healthy Volunteer, Urea Cycle Disorder
Interventions
Placebo, SYNB1020
Drug
Lead sponsor
Synlogic
Industry
Eligibility
18 Years to 64 Years
Enrollment
52 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2017 – 2018
U.S. locations
1
States / cities
Brooklyn, Maryland
Source: ClinicalTrials.gov public record
Updated May 11, 2021 · Synced Jul 23, 2026, 2:45 AM EDT
Completed Not applicable Interventional Accepts healthy volunteers

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

NCT01610089
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Mar 19, 2015 · Synced Jul 23, 2026, 2:45 AM 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, 2:45 AM EDT
Recruiting Not applicable Interventional

At Home Ammonia Monitoring of Inborn Errors of Ammonia Metabolism

NCT06953505
View directory record Official record
Conditions
Urea Cycle Disorders, Organic Acidemias, Fatty Acid Oxidation Disorder, Ammonia; Metabolic Disorder
Interventions
Ammonia Study Device
Diagnostic Test
Lead sponsor
Sequitur Health Corp.
Industry
Eligibility
12 Years and older
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
1
States / cities
Portland, Oregon
Source: ClinicalTrials.gov public record
Updated May 26, 2026 · Synced Jul 23, 2026, 2:45 AM 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, 2:45 AM EDT
Completed Phase 3 Interventional Results available

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

NCT01347073
View directory record Official record
Conditions
Urea Cycle Disorders
Interventions
HPN-100
Drug
Lead sponsor
Amgen
Industry
Eligibility
29 Days to 6 Years
Enrollment
23 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2013
U.S. locations
8
States / cities
Los Angeles, California • Washington D.C., District of Columbia • Portland, Maine + 5 more
Source: ClinicalTrials.gov public record
Updated Jul 10, 2024 · Synced Jul 23, 2026, 2:45 AM EDT