- 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
Search Results
Search by objective public record fields.
Search by query text, NCT ID, condition, intervention, sponsor, city, state, recruitment status, phase, study type, healthy volunteer eligibility, sex, or age. Results are retrieved from ClinicalTrials.gov and synchronized into the directory. Search pages remain noindex by default.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Showing 25–48
of 24
matching trials from the live ClinicalTrials.gov search.
Local D1 index available.
- 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
- 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
- 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
- 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
- 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 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
- 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
- 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
- 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
- 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
- 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, 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
- 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
- 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
- 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
- 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
- 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
- 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
- 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
- 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
- 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
- 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