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ClinicalTrials.gov public records Last synced Oct 15, 2026, 1:42 AM EDT

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Showing 553–576 of 1,608 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Metabolism, Inborn Errors Clear all
Terminated Phase 2Phase 3 Interventional

Extension Study of ABP-671 in Participants With Gout

NCT06276556
View directory record Official record
Conditions
Gout
Interventions
ABP-671, Allopurinol
Drug
Lead sponsor
Atom Therapeutics Co., Ltd
Industry
Eligibility
19 Years to 75 Years
Enrollment
43 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024
U.S. locations
33
States / cities
Tempe, Arizona • Tucson, Arizona • Anaheim, California + 24 more
Source: ClinicalTrials.gov public record
Updated Jul 16, 2025 · Synced Oct 15, 2026, 1:42 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

The Effects of Kuvan on Functional Brain Connectivity in Individuals With Phenylketonuria (PKU)

NCT00964236
View directory record Official record
Conditions
Phenylketonuria
Interventions
Sapropterin
Drug
Lead sponsor
University of Missouri-Columbia
Other
Eligibility
6 Years and older
Enrollment
20 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2009 – 2011
U.S. locations
1
States / cities
Columbia, Missouri
Source: ClinicalTrials.gov public record
Updated Jan 4, 2018 · Synced Oct 15, 2026, 1:42 AM EDT
Completed No phase listed Observational

Natural History Study of Infantile and Juvenile GM1 Gangliosidosis (GM1) Patients

NCT04041102
View directory record Official record
Conditions
GM1 Gangliosidosis
Interventions
Not listed
Lead sponsor
University of Pennsylvania
Other
Eligibility
Not listed
Enrollment
31 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2024
U.S. locations
2
States / cities
Oakland, California • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Oct 24, 2025 · Synced Oct 15, 2026, 1:42 AM EDT
Completed Phase 3 Interventional Results available

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

NCT02230566
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Jul 30, 2020 · Synced Oct 15, 2026, 1:42 AM EDT
Completed Not applicable Interventional Results available

Cardiac Amyloidosis Discovery Trial

NCT06469372
View directory record Official record
Conditions
Cardiac Amyloidosis
Interventions
Cardiac amyloidosis deep learning model
Device
Lead sponsor
Pierre Elias
Other
Eligibility
50 Years and older
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2025
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Dec 4, 2025 · Synced Oct 15, 2026, 1:42 AM EDT
Completed Phase 2 Interventional

An Open-Label Clinical Trial of Replagal Enzyme Therapy in Children Ages 7-17 Years With Fabry Disease

NCT00071877
View directory record Official record
Conditions
Fabry Disease
Interventions
Replagal
Drug
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
Not listed
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003 – 2005
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 4, 2008 · Synced Oct 15, 2026, 1:42 AM EDT
Completed Phase 3 Interventional Results available

Lesinurad Monotherapy in Gout Subjects Intolerant to Xanthine Oxidase Inhibitors

NCT01508702
View directory record Official record
Conditions
Gout
Interventions
lesinurad, Placebo
Drug
Lead sponsor
Ardea Biosciences, Inc.
Industry
Eligibility
18 Years to 85 Years
Enrollment
214 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2013
U.S. locations
79
States / cities
Birmingham, Alabama • Glendale, Arizona • Phoenix, Arizona + 71 more
Source: ClinicalTrials.gov public record
Updated Feb 12, 2016 · Synced Oct 15, 2026, 1:42 AM EDT
Completed No phase listed Observational Results available

Response to Diaphragmatic Pacing in Subjects With Pompe Disease

NCT02354651
View directory record Official record
Conditions
Pompe Disease
Interventions
Diaphragm conditioning via phrenic nerve stimulation
Other
Lead sponsor
University of Florida
Other
Eligibility
2 Years to 65 Years
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2023
U.S. locations
1
States / cities
Gainesville, Florida
Source: ClinicalTrials.gov public record
Updated Aug 1, 2023 · Synced Oct 15, 2026, 1:42 AM EDT
Terminated Phase 1Phase 2 Interventional

VAL-1221 Delivered Intravenously in Ambulatory and Ventilator-free Participants With Late-Onset Pompe Disease

NCT02898753
View directory record Official record
Conditions
Pompe Disease
Interventions
VAL-1221, RhGAA
Drug
Lead sponsor
Valerion Therapeutics, LLC
Industry
Eligibility
18 Years and older
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2020
U.S. locations
2
States / cities
Orange, California • Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Jun 2, 2020 · Synced Oct 15, 2026, 1:42 AM EDT
Completed Not applicable Interventional

Study of Total Body Irradiation and Fludarabine Followed By Allogeneic Peripheral Blood Stem Cell or Bone Marrow Transplantation in Combination With Cyclosporine and Mycophenolate Mofetil in Patients With Inherited Disorders

NCT00010361
View directory record Official record
Conditions
Metabolism, Inborn Errors, Granulomatous Disease, Chronic
Interventions
cyclosporine, fludarabine, mycophenolate mofetil
Drug
Lead sponsor
Fred Hutchinson Cancer Center
Other
Eligibility
Up to 55 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2000
U.S. locations
1
States / cities
Seattle, Washington
Source: ClinicalTrials.gov public record
Updated Dec 9, 2014 · Synced Oct 15, 2026, 1:42 AM EDT
Completed No phase listed Observational

Mucopolysaccharidosis I (MPS I) Registry

NCT00144794
View directory record Official record
Conditions
Mucopolysaccharidosis I (MPS I)
Interventions
Not listed
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
Not listed
Enrollment
1,406 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003 – 2024
U.S. locations
53
States / cities
Phoenix, Arizona • Little Rock, Arkansas • Los Angeles, California + 44 more
Source: ClinicalTrials.gov public record
Updated Feb 27, 2026 · Synced Oct 15, 2026, 1:42 AM EDT
Completed No phase listed Observational

Longitudinal Studies of the Glycoproteinoses

NCT01891422
View directory record Official record
Conditions
Aspartylglucosaminuria, Fucosidosis, Galactosialidosis, Alpha Mannosidosis, Beta Mannosidosis, Mucolipidosis II, Mucolipidosis III, Schindler Disease, Sialidosis
Interventions
Non-interventional.
Other
Lead sponsor
Greenwood Genetic Center
Other
Eligibility
Not listed
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2020
U.S. locations
1
States / cities
North Charleston, South Carolina
Source: ClinicalTrials.gov public record
Updated Sep 13, 2023 · Synced Oct 15, 2026, 1:42 AM EDT
Not listed Not applicable Interventional

Investigation of Alanine in Fructose Intolerance: A Dose Ranging Study

NCT01185210
View directory record Official record
Conditions
Fructose Intolerance
Interventions
Placebo, Alanine
Dietary Supplement
Lead sponsor
Augusta University
Other
Eligibility
18 Years to 70 Years
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2024
U.S. locations
1
States / cities
Iowa City, Iowa
Source: ClinicalTrials.gov public record
Updated Jul 21, 2022 · Synced Oct 15, 2026, 1:42 AM EDT
Not listed No phase listed Observational Accepts healthy volunteers

Biomarker Analysis for GBA Associated Parkinson's Disease

NCT03811496
View directory record Official record
Conditions
Parkinson Disease, Gaucher Disease
Interventions
Not listed
Lead sponsor
Lysosomal and Rare Disorders Research and Treatment Center, Inc.
Other
Eligibility
50 Years and older
Enrollment
100 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2018 – 2020
U.S. locations
1
States / cities
Fairfax, Virginia
Source: ClinicalTrials.gov public record
Updated Jan 23, 2019 · Synced Oct 15, 2026, 1:42 AM EDT
Not listed No phase listed Observational

SRT in Comparison to ERT on Immune Aspects and Bone Involvement in Gaucher Disease

NCT02605603
View directory record Official record
Conditions
Gaucher Disease
Interventions
Not listed
Lead sponsor
Lysosomal and Rare Disorders Research and Treatment Center, Inc.
Other
Eligibility
18 Years and older
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2020
U.S. locations
1
States / cities
Fairfax, Virginia
Source: ClinicalTrials.gov public record
Updated Aug 13, 2019 · Synced Oct 15, 2026, 1:42 AM EDT
Completed Phase 1 Interventional

Anaplerotic Therapy in Propionic Acidemia

NCT00645879
View directory record Official record
Conditions
Propionic Acidemia
Interventions
ornithine alpha ketoglutarate, glutamine, disodium citrate
Drug
Lead sponsor
Nicola Longo
Other
Eligibility
5 Years to 12 Years
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2010
U.S. locations
1
States / cities
Salt Lake City, Utah
Source: ClinicalTrials.gov public record
Updated Jan 21, 2015 · Synced Oct 15, 2026, 1:42 AM EDT
Completed Not applicable Interventional Accepts healthy volunteers

Pre-Clinical White Matter Changes and Associated Connectivity Effects in Fabry Disease

NCT03678324
View directory record Official record
Conditions
Fabry Disease
Interventions
Functional MRI and fast bound-pool fraction imaging
Other
Lead sponsor
University of Utah
Other
Eligibility
18 Years and older
Enrollment
40 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2020 – 2022
U.S. locations
1
States / cities
Salt Lake City, Utah
Source: ClinicalTrials.gov public record
Updated May 26, 2022 · Synced Oct 15, 2026, 1:42 AM EDT
Completed Phase 1Phase 2 Interventional Results available

Safety and Efficacy Study of Asfotase Alfa in Severely Affected Infants With Hypophosphatasia (HPP)

NCT00744042
View directory record Official record
Conditions
Hypophosphatasia (HPP)
Interventions
asfotase alfa
Biological
Lead sponsor
Alexion Pharmaceuticals, Inc.
Industry
Eligibility
Up to 36 Months
Enrollment
11 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2010
U.S. locations
6
States / cities
Little Rock, Arkansas • Wilmington, Delaware • Springfield, Missouri + 3 more
Source: ClinicalTrials.gov public record
Updated Apr 1, 2019 · Synced Oct 15, 2026, 1:42 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 Jul 1, 2024 · Synced Oct 15, 2026, 1:42 AM EDT
Terminated Phase 2 Interventional Results available

A Study to Examine the Effects of the Leptin Receptor (LEPR) Agonist Antibody REGN4461 in Adult Patients With Familial Partial Lipodystrophy (FPLD)

NCT05088460
View directory record Official record
Conditions
Familial Partial Lipodystrophy, Metabolic Abnormalities
Interventions
REGN4461, Matching Placebo
Drug
Lead sponsor
Regeneron Pharmaceuticals
Industry
Eligibility
18 Years and older
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2024
U.S. locations
5
States / cities
Boca Raton, Florida • Bethesda, Maryland • Ann Arbor, Michigan + 2 more
Source: ClinicalTrials.gov public record
Updated Oct 16, 2025 · Synced Oct 15, 2026, 1:42 AM EDT
Completed No phase listed Observational Accepts healthy volunteers Results available

Nutritional and Neurotransmitter Changes in PKU Subjects on BH4

NCT00688844
View directory record Official record
Conditions
Phenylketonuria
Interventions
KuvanTM Therapy
Drug
Lead sponsor
Emory University
Other
Eligibility
4 Years and older
Enrollment
58 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2008 – 2010
U.S. locations
2
States / cities
Atlanta, Georgia • Decatur, Georgia
Source: ClinicalTrials.gov public record
Updated Jun 8, 2015 · Synced Oct 15, 2026, 1:42 AM EDT
Recruiting Phase 4 Interventional

Treat-to-Target Serum Urate Versus Treat-to-Avoid Symptoms in Gout

NCT04875702
View directory record Official record
Conditions
Gout
Interventions
Allopurinol, Naproxen 250 MG, Colchicine 0.6 mg, Colchicine 1.2 mg, Naproxen 500 Mg, Prednisone 40 mg
Drug
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
18 Years to 90 Years
Enrollment
650 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2028
U.S. locations
7
States / cities
South Birmingham, Alabama • Santa Monica, California • Boston, Massachusetts + 2 more
Source: ClinicalTrials.gov public record
Updated Sep 25, 2025 · Synced Oct 15, 2026, 1:42 AM 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 5, 2019 · Synced Oct 15, 2026, 1:42 AM EDT
Completed Phase 1Phase 2 Interventional Results available

A Study of PTC923 (CNSA-001) in Primary Tetrahydrobiopterin (BH4) Deficient Participants With Hyperphenylalaninemia

NCT03519711
View directory record Official record
Conditions
BH4 Deficiency, Hyperphenylalaninemia
Interventions
PTC923
Drug
Lead sponsor
PTC Therapeutics
Industry
Eligibility
12 Months and older
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2020
U.S. locations
4
States / cities
Minneapolis, Minnesota • Dallas, Texas • Salt Lake City, Utah + 1 more
Source: ClinicalTrials.gov public record
Updated Nov 14, 2023 · Synced Oct 15, 2026, 1:42 AM EDT