Independent directory Public ClinicalTrials.gov records United States
Clear filters

These results were retrieved from the live ClinicalTrials.gov registry. Filters and sort order apply to this result list.

ClinicalTrials.gov public records Last synced Oct 24, 2026, 12:58 AM EDT

Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.

Showing 1–24 of 61 matching trials from the live ClinicalTrials.gov search.
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
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
Terminated No phase listed Observational Accepts healthy volunteers

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

NCT01421888
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
Completed No phase listed Observational Accepts healthy volunteers Results available

PCORI Urea Cycle Disorder Study

NCT02740153
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 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 (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
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
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
Conditions
Arginase I Deficiency, Hyperargininemia
Interventions
AEB1102
Drug
Lead sponsor
Aeglea Biotherapeutics
Industry
Eligibility
2 Years and older
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2019
U.S. locations
6
States / cities
Stanford, California • Gainesville, Florida • Detroit, Michigan + 3 more
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
Completed Not applicable Interventional Accepts healthy volunteers Results available

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

NCT02111200
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
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
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
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
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
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
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
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
Completed No phase listed Observational Results available

Observational Study of OCT in a Patients Undergoing FFR

NCT01663896
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
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
Not listed No phase listed Observational Accepts healthy volunteers

Neuroimaging and Neuropsychological Outcomes in Urea Cycle Disorders

NCT02935283
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
Recruiting No phase listed Observational

Abbott Vascular Medical Device Registry

NCT04573660
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
Active, not recruiting No phase listed Observational

Liver Disease in Urea Cycle Disorders

NCT04612764
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
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