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ClinicalTrials.gov public records Last synced Nov 15, 2026, 5:24 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 1129–1152 of 1,513 matching trials from the live ClinicalTrials.gov search.

Filters: Condition: Inborn Errors of Metabolism Disorders Clear all

Conditions: Gout, Hyperuricemia
Interventions: [14C] LC350189; Drug
Lead sponsor: LG Chem; Industry
Eligibility: 18 Years to 55 Years · Male only
Enrollment: 6 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2020
U.S. locations: 1
States / cities: Austin, Texas

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: Krabbe Disease
Interventions: FBX-101; Biological
Lead sponsor: Duke University; Other
Eligibility: Not listed
Enrollment: 2 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2024 – 2025
U.S. locations: 1
States / cities: Durham, North Carolina

Conditions: Glycogen Storage Disease Type IA, Glycogen Storage Disease Type I, Glycogen Storage Disease Type IB, Glycogen Storage Disease Xi
Interventions: Lactate Meter, Accu Chek; Other · Device
Lead sponsor: Connecticut Children's Medical Center; Other
Eligibility: Up to 60 Years
Enrollment: 10 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2026 – 2027
U.S. locations: 1
States / cities: Hartford, Connecticut

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: Pompe Disease, Glycogen Storage Disease Type II (GSD-II), Glycogenesis 2 Acid Maltase Deficiency
Interventions: alglucosidase alfa; Biological
Lead sponsor: Genzyme, a Sanofi Company; Industry
Eligibility: 6 Months and older
Enrollment: 13 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2007 – 2010
U.S. locations: 9
States / cities: Birmingham, Alabama • Stanford, California • Washington D.C., District of Columbia + 6 more

Conditions: Gout
Interventions: Febuxostat IR, Febuxostat XR, Febuxostat placebo, Colchicine, Naproxen, Lansoprazole; Drug
Lead sponsor: Takeda; Industry
Eligibility: 18 Years and older
Enrollment: 1,790 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2014 – 2015
U.S. locations: 192
States / cities: Binghamton, Alabama • Birmingham, Alabama • Huntsville, Alabama + 189 more

Conditions: Jaundice, Neonatal, Hyperbilirubinemia, Neonatal
Interventions: Phototherapy, Stannsoporfin, Placebo; Procedure · Drug
Lead sponsor: InfaCare Pharmaceuticals Corporation, a Mallinckrodt Company; Industry
Eligibility: 1 Hour to 72 Hours
Enrollment: 91 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2013 – 2016
U.S. locations: 18
States / cities: Colton, California • San Diego, California • San Francisco, California + 14 more

Conditions: Wilson Disease
Interventions: ALXN1840, SoC Therapy; Drug
Lead sponsor: Alexion Pharmaceuticals, Inc.; Industry
Eligibility: 12 Years and older
Enrollment: 214 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018 – 2023
U.S. locations: 6
States / cities: Los Angeles, California • New Haven, Connecticut • Chicago, Illinois + 3 more

Conditions: Pseudohypoparathyroidism, Albright Hereditary Osteodystrophy
Interventions: Theophylline, Placebos; Drug
Lead sponsor: Vanderbilt University Medical Center; Other
Eligibility: 13 Years to 99 Years
Enrollment: 29 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018 – 2026
U.S. locations: 1
States / cities: Nashville, Tennessee

Conditions: Parkinson Disease, Gaucher Disease
Interventions: 15-0 H20; Drug
Lead sponsor: National Human Genome Research Institute (NHGRI); NIH
Eligibility: 18 Years to 110 Years
Enrollment: 64 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: Started 2006
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Mucopolysaccharidosis I, Lysosomal Storage Diseases, Spinal Cord Compression
Interventions: laronidase; Drug
Lead sponsor: Patricia I. Dickson, M.D.; Individual
Eligibility: 8 Years and older
Enrollment: 4 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2005 – 2011
U.S. locations: 1
States / cities: Torrance, California

Conditions: Gaucher Disease
Interventions: Velaglucerase alfa, Vitamin D; Drug · Dietary Supplement
Lead sponsor: Shire; Industry
Eligibility: 18 Years to 70 Years
Enrollment: 21 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2016 – 2020
U.S. locations: 8
States / cities: Beverly Hills, California • Los Angeles, California • Atlanta, Georgia + 5 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: Healthy Volunteers, Propionic Acidemia, Methylmalonic Acidemia, Organic Acidemia
Interventions: BBP-671, Placebo; Drug
Lead sponsor: CoA Therapeutics, Inc., a BridgeBio company; Industry
Eligibility: 15 Years to 55 Years
Enrollment: 79 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2021 – 2023
U.S. locations: 3
States / cities: Topeka, Indiana • Pittsburgh, Pennsylvania • Austin, Texas

Conditions: Phenylketonurias, PAH Deficiency
Interventions: HMI-102; Genetic
Lead sponsor: Homology Medicines, Inc; Industry
Eligibility: 18 Years to 55 Years
Enrollment: 10 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2019 – 2023
U.S. locations: 13
States / cities: Los Angeles, California • Orange, California • Tampa, Florida + 10 more

Conditions: Gut Health
Interventions: Butyrate Formulation, Butyrate + Polyphenol Formulation, Placebo; Dietary Supplement
Lead sponsor: Supplement Formulators, Inc.; Industry
Eligibility: 21 Years to 70 Years
Enrollment: 143 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2023 – 2025
U.S. locations: 1
States / cities: Fort Lauderdale, Florida

Conditions: MPS 7, Sly Syndrome, Mucopolysaccharidosis, MPS VII
Interventions: UX003, Placebo; Drug · Other
Lead sponsor: Ultragenyx Pharmaceutical Inc; Industry
Eligibility: 5 Years to 35 Years
Enrollment: 12 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2014 – 2016
U.S. locations: 4
States / cities: Oakland, California • Orange, California • Miami, Florida + 1 more

Conditions: Small Intestinal Bacterial Overgrowth, Lactose Intolerance, Fructose Intolerance, Glucose Intolerance
Interventions: Not listed
Lead sponsor: Augusta University; Other
Eligibility: 18 Years and older
Enrollment: 1,080 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2011 – 2017
U.S. locations: 1
States / cities: Augusta, Georgia

Conditions: Lysosomal Acid Lipase Deficiency
Interventions: Not listed
Lead sponsor: Alexion Pharmaceuticals, Inc.; Industry
Eligibility: 2 Years and older
Enrollment: 640 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2014 – 2015
U.S. locations: 15
States / cities: Birmingham, Alabama • Phoenix, Arizona • Los Angeles, California + 12 more

Conditions: Diabetes Mellitus, Heart Disease, Hemochromatosis, Thalassemia
Interventions: Not listed
Lead sponsor: National Heart, Lung, and Blood Institute (NHLBI); NIH
Eligibility: 4 Years and older
Enrollment: 151 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 1985 – 2015
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Severe Familial Hypercholesterolemia
Interventions: Evolocumab; Biological
Lead sponsor: Amgen; Industry
Eligibility: 12 Years to 80 Years
Enrollment: 300 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2012 – 2018
U.S. locations: 6
States / cities: Los Angeles, California • New York, New York • Cincinnati, Ohio + 1 more

Conditions: FAP, Familial Amyloid Polyneuropathy, TTR, Transthyretin, Amyloidosis
Interventions: Inotersen, Placebo; Drug
Lead sponsor: Ionis Pharmaceuticals, Inc.; Industry
Eligibility: 18 Years to 82 Years
Enrollment: 173 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2013 – 2017
U.S. locations: 9
States / cities: Orange, California • Indianapolis, Indiana • Baltimore, Maryland + 5 more

Conditions: Inborn Errors of Metabolism
Interventions: N-carbamylglutamate; Drug
Lead sponsor: Mendel Tuchman; Other
Eligibility: 1 Day to 70 Years
Enrollment: 52 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2008 – 2020
U.S. locations: 1
States / cities: Washington D.C., District of Columbia

Search by objective public record fields.

Mass Balance Study of [14C]LC350189 in Healthy Volunteers

Efficacy and Safety of Pegzilarginase in Patients With Arginase 1 Deficiency

Long-term Follow-up Study to Evaluate Safety and Efficacy of FBX-101 in Krabbe Patients

Accuracy of Home Lactate Meter and Accu-chek Glucometer in Patients With Glycogen Storage Disease

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

High Dose or High Dose Frequency Study of Alglucosidase Alfa

Efficacy and Safety of Extended Release and Immediate Release Febuxostat in Participants With Gout

Stannsoporfin With Light Therapy for Newborn Babies With Jaundice

Efficacy and Safety of ALXN1840 Administered for 48 Weeks Versus Standard of Care in Participants With Wilson Disease

Theophylline Treatment for Pseudohypoparathyroidism

Positron Emission Tomography (PET) Imaging in People With Gaucher Mutations

Intrathecal Enzyme Replacement Therapy for Spinal Cord Compression in Mucopolysaccharidosis (MPS) I

Study of the Effect of Velaglucerase Alfa (VPRIV®) on Bone-related Pathology in Treatment-naïve Participants With Type 1 Gaucher Disease

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

A First in Human, Dose Escalation Study to Evaluate the Safety and Tolerability of BBP-671 in Healthy Volunteers and Patients With Propionic Acidemia or Methylmalonic Acidemia

Gene Therapy Clinical Study in Adult PKU

Effectiveness of a Butyrate Formulation and Butyrate + Polyphenol Formulation on Gut Health, Permeability and Associated Symptoms

A Phase 3 Study of UX003 Recombinant Human Betaglucuronidase (rhGUS) Enzyme Replacement Therapy in Patients With Mucopolysaccharidosis Type 7 (MPS 7)

Clinical Utility of Breath Tests in GI

A Study to Identify and Characterize LAL-D Patients in High-risk Populations

Deferoxamine for the Treatment of Hemochromatosis

Trial Assessing Long Term USe of PCSK9 Inhibition in Subjects With Genetic LDL Disorders

Efficacy and Safety of Inotersen in Familial Amyloid Polyneuropathy

N-Carbamylglutamate (Carbaglu) In The Treatment Of Hyperammonemia