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ClinicalTrials.gov public records Last synced Oct 14, 2026, 10:33 AM EDT

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Showing 265–288 of 1,608 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Metabolism, Inborn Errors Clear all
Withdrawn Not applicable Interventional Accepts healthy volunteers

Effects of Potassium Citrate in Urine of Children With Elevated Calcium in Urine and Kidney Stones

NCT00120731
View directory record Official record
Conditions
Kidney Calculi
Interventions
Potassium Citrate, Acetazolamide, Diet low in oxalate and purines
Drug · Behavioral
Lead sponsor
Children's Mercy Hospital Kansas City
Other
Eligibility
5 Years to 17 Years
Healthy volunteers
Accepts healthy volunteers
Timeline
2005 – 2007
U.S. locations
1
States / cities
Kansas City, Missouri
Source: ClinicalTrials.gov public record
Updated Nov 5, 2020 · Synced Oct 14, 2026, 10:33 AM EDT
Completed Phase 2 Interventional Results available

Nedosiran in Pediatric Patients From Birth to 11 Years of Age With PH and Relatively Intact Renal Function

NCT05001269
View directory record Official record
Conditions
Primary Hyperoxaluria, Primary Hyperoxaluria Type 1, Primary Hyperoxaluria Type 2, Primary Hyperoxaluria Type 3
Interventions
nedosiran
Drug
Lead sponsor
Dicerna Pharmaceuticals, Inc., a Novo Nordisk company
Industry
Eligibility
Up to 11 Years
Enrollment
27 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2025
U.S. locations
1
States / cities
Rochester, Minnesota
Source: ClinicalTrials.gov public record
Updated Apr 16, 2026 · Synced Oct 14, 2026, 10:33 AM EDT
Completed Phase 4 Interventional

Phase IV Study to Gather More Information About the Safety of ACZONE Gel, 5% in Treating Subjects With Acne Who Have G6PD Deficiency

NCT00243542
View directory record Official record
Conditions
Acne Vulgaris
Interventions
ACZONE Gel, 5%, Vehicle
Drug
Lead sponsor
Allergan
Industry
Eligibility
12 Years and older
Enrollment
64 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2006
U.S. locations
29
States / cities
Birmingham, Alabama • San Diego, California • San Francisco, California + 24 more
Source: ClinicalTrials.gov public record
Updated May 30, 2011 · Synced Oct 14, 2026, 10:33 AM EDT
Withdrawn Phase 2 Interventional

Androgen Reduction in Congenital Adrenal Hyperplasia

NCT03548246
View directory record Official record
Conditions
Congenital Adrenal Hyperplasia
Interventions
Abiraterone acetate, Placebo, Hydrocortisone, Fludrocortisone
Drug
Lead sponsor
University of Texas Southwestern Medical Center
Other
Eligibility
2 Years to 9 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2026
U.S. locations
4
States / cities
Los Angeles, California • Bethesda, Maryland • Ann Arbor, Michigan + 1 more
Source: ClinicalTrials.gov public record
Updated Feb 1, 2023 · Synced Oct 14, 2026, 10:33 AM EDT
Completed Phase 3 Interventional Results available

GlycoCholic Acid Treatment for Patients With Inborn Errors in Bile Acid Synthesis

NCT01589523
View directory record Official record
Conditions
Bile Acid Synthesis Defect, Inborn Error of Bile Acid Metabolism, Inborn Error of Bile Acid Conjugation
Interventions
Glycocholic Acid
Drug
Lead sponsor
Children's Hospital Medical Center, Cincinnati
Other
Eligibility
1 Week to 85 Years
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2019
U.S. locations
1
States / cities
Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Jun 8, 2022 · Synced Oct 14, 2026, 10:33 AM EDT
Completed Phase 1Phase 2 Interventional Results available

Hydroxyproline Influence on Oxalate Metabolism

NCT02038543
View directory record Official record
Conditions
Hyperoxaluria
Interventions
Hydroxyproline and Leucine
Drug
Lead sponsor
Mayo Clinic
Other
Eligibility
15 Years to 65 Years
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2017
U.S. locations
1
States / cities
Rochester, Minnesota
Source: ClinicalTrials.gov public record
Updated Jul 2, 2017 · Synced Oct 14, 2026, 10:33 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Primary Hyperoxaluria Mutation Genotyping

NCT00589225
View directory record Official record
Conditions
Primary Hyperoxaluria
Interventions
Genetic Analysis
Genetic
Lead sponsor
Mayo Clinic
Other
Eligibility
Not listed
Enrollment
902 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2003 – 2014
U.S. locations
1
States / cities
Rochester, Minnesota
Source: ClinicalTrials.gov public record
Updated Jul 7, 2016 · Synced Oct 14, 2026, 10:33 AM EDT
Recruiting Phase 1Phase 2 Interventional

DFT383 in Pediatric Participants With Nephropathic Cystinosis

NCT06910813
View directory record Official record
Conditions
Nephropathic Cystinosis
Interventions
DFT383
Genetic
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
2 Years to 5 Years
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2044
U.S. locations
6
States / cities
Phoenix, Arizona • San Diego, California • Stanford, California + 3 more
Source: ClinicalTrials.gov public record
Updated Sep 22, 2026 · Synced Oct 14, 2026, 10:33 AM EDT
Active, not recruiting Phase 3 Interventional

A Study to Investigate the Efficacy and Safety of OTL-203 in Subjects With MPS-IH Compared With Standard of Care With Allogeneic HSCT

NCT06149403
View directory record Official record
Conditions
MPS-IH (Hurler Syndrome)
Interventions
Experimental: OTL-203, Active Comparator: Allo-HSCT
Genetic
Lead sponsor
Orchard Therapeutics
Industry
Eligibility
28 Days to 30 Months
Enrollment
41 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2031
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Dec 22, 2025 · Synced Oct 14, 2026, 10:33 AM EDT
Withdrawn Phase 1 Interventional

Safety and Efficacy Study of HuCNS-SC in Subjects With Neuronal Ceroid Lipofuscinosis

NCT01238315
View directory record Official record
Conditions
Neuronal Ceroid Lipofuscinosis
Interventions
HuCNS-SC
Biological
Lead sponsor
StemCells, Inc.
Industry
Eligibility
6 Months to 6 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2011
U.S. locations
1
States / cities
Portland, Oregon
Source: ClinicalTrials.gov public record
Updated Jan 15, 2015 · Synced Oct 14, 2026, 10:33 AM EDT
Completed Not applicable Interventional

Pompe Disease QMUS and EIM

NCT02742298
View directory record Official record
Conditions
Pompe Disease
Interventions
Muscle ultrasound, Electrical Impedance Myography
Device · Other
Lead sponsor
Duke University
Other
Eligibility
18 Years and older
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2020
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Sep 25, 2020 · Synced Oct 14, 2026, 10:33 AM EDT
Terminated Phase 1Phase 2 Interventional Results available

A Gene Therapy Study for Homozygous Familial Hypercholesterolemia (HoFH)

NCT02651675
View directory record Official record
Conditions
Homozygous Familial Hypercholesterolemia (HoFH)
Interventions
AAV directed hLDLR gene therapy
Genetic
Lead sponsor
REGENXBIO Inc.
Industry
Eligibility
18 Years and older
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2020
U.S. locations
5
States / cities
Boca Raton, Florida • Kansas City, Kansas • Portland, Oregon + 2 more
Source: ClinicalTrials.gov public record
Updated Jul 13, 2023 · Synced Oct 14, 2026, 10:33 AM EDT
Recruiting No phase listed Observational

A Global Prospective Observational Registry of Patients With Pompe Disease

NCT06121011
View directory record Official record
Conditions
Pompe Disease
Interventions
Cipaglucosidase alfa, Miglustat, Alglucosidase alfa or Avalglucosidase alfa, Untreated
Biological · Drug · Other
Lead sponsor
Amicus Therapeutics
Industry
Eligibility
Not listed
Enrollment
500 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2034
U.S. locations
15
States / cities
Little Rock, Arkansas • Irvine, California • Jacksonville, Florida + 10 more
Source: ClinicalTrials.gov public record
Updated Mar 10, 2026 · Synced Oct 14, 2026, 10:33 AM EDT
Recruiting Phase 1 Interventional

UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells

NCT02254863
View directory record Official record
Conditions
Adrenoleukodystrophy, Batten Disease, Mucopolysaccharidosis II, Leukodystrophy, Globoid Cell, Leukodystrophy, Metachromatic, Neimann Pick Disease, Pelizaeus-Merzbacher Disease, Sandhoff Disease, Tay-Sachs Disease, Brain Diseases, Metabolic, Inborn, Alpha-Mannosidosis, Sanfilippo Mucopolysaccharidoses
Interventions
DUOC-01
Biological
Lead sponsor
Joanne Kurtzberg, MD
Other
Eligibility
1 Week to 22 Years
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2027
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Sep 3, 2026 · Synced Oct 14, 2026, 10:33 AM EDT
Completed Phase 1Phase 2 Interventional Results available

Safety and Efficacy of Albuterol in Individuals With Late-onset Pompe Disease

NCT01885936
View directory record Official record
Conditions
Pompe Disease
Interventions
Albuterol, Placebo
Drug
Lead sponsor
Duke University
Other
Eligibility
18 Years and older
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2016
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Jul 15, 2019 · Synced Oct 14, 2026, 10:33 AM EDT
Completed Phase 1Phase 2 Interventional Results available

Safety, Tolerability and Pharmacokinetics of SBC-102 (Sebelipase Alfa) in Adult Participants With Lysosomal Acid Lipase Deficiency

NCT01307098
View directory record Official record
Conditions
Cholesterol Ester Storage Disease(CESD), Lysosomal Acid Lipase Deficiency, LAL-Deficiency
Interventions
Sebelipase alfa 0.35 mg/kg, Sebelipase alfa 1 mg/kg, Sebelipase alfa 3 mg/kg
Drug
Lead sponsor
Alexion Pharmaceuticals, Inc.
Industry
Eligibility
18 Years to 65 Years
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2012
U.S. locations
3
States / cities
Stanford, California • New York, New York • Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Dec 11, 2018 · Synced Oct 14, 2026, 10:33 AM EDT
Completed Not applicable Interventional

Study of NTBC for Tyrosinemia I

NCT00004443
View directory record Official record
Conditions
Tyrosinemia I
Interventions
NTBC
Drug
Lead sponsor
University of Washington
Other
Eligibility
Up to 17 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1998 – 2006
U.S. locations
2
States / cities
Portland, Oregon • Seattle, Washington
Source: ClinicalTrials.gov public record
Updated Mar 25, 2015 · Synced Oct 14, 2026, 10:33 AM EDT
Enrolling by invitation Phase 1 Interventional

PET Imaging With [18F]Fluselenamyl in People With Cardiac Amyloidosis

NCT07771621
View directory record Official record
Conditions
Cardiac Amyloidosis
Interventions
[18F]Fluselenamyl (FSA)
Drug
Lead sponsor
Washington University School of Medicine
Other
Eligibility
18 Years and older
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2030
U.S. locations
1
States / cities
St Louis, Missouri
Source: ClinicalTrials.gov public record
Updated Aug 18, 2026 · Synced Oct 14, 2026, 10:33 AM EDT
Completed Phase 1 Interventional Results available

Administration of IV Laronidase Post Bone Marrow Transplant in Hurler

NCT01173016
View directory record Official record
Conditions
Hurler Syndrome
Interventions
Laronidase
Drug
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
Up to 14 Years
Enrollment
11 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2016
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Mar 20, 2020 · Synced Oct 14, 2026, 10:33 AM EDT
Recruiting No phase listed Observational

PheCheck™ Validation Study

NCT07551921
View directory record Official record
Conditions
Phenylketonuria (PKU) and Hyperphenylalaninemia
Interventions
PheCheck, Dried Blood Spot Cards.
Device · Diagnostic Test
Lead sponsor
Aptatek BioSciences, Inc
Industry
Eligibility
1 Year and older
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2027
U.S. locations
2
States / cities
Chicago, Illinois • Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Aug 25, 2026 · Synced Oct 14, 2026, 10:33 AM EDT
Completed Phase 1Phase 2 Interventional Results available

Oxidative Stress Markers In Inherited Homocystinuria And The Impact Of Taurine

NCT01192828
View directory record Official record
Conditions
Homocystinuria
Interventions
taurine
Drug
Lead sponsor
University of Colorado, Denver
Other
Eligibility
8 Years to 49 Years
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2017
U.S. locations
4
States / cities
Aurora, Colorado • Durham, North Carolina • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jul 9, 2018 · Synced Oct 14, 2026, 10:33 AM EDT
Completed No phase listed Observational

Sjogren-Larsson Syndrome: Natural History, Clinical Variation and Evaluation of Biochemical Markers

NCT01971957
View directory record Official record
Conditions
Sjogren-Larsson Syndrome (SLS)
Interventions
Not listed
Lead sponsor
University of Nebraska
Other
Eligibility
Not listed
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2023
U.S. locations
2
States / cities
Omaha, Nebraska • Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Oct 8, 2024 · Synced Oct 14, 2026, 10:33 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Large-Scale Metabolomic Profiling for the Diagnosis of Inborn Errors of Metabolism

NCT04201067
View directory record Official record
Conditions
Congenital Disorders of Glycosylation
Interventions
Not listed
Lead sponsor
Mayo Clinic
Other
Eligibility
Not listed
Enrollment
240 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2019 – 2025
U.S. locations
1
States / cities
Rochester, Minnesota
Source: ClinicalTrials.gov public record
Updated Oct 3, 2025 · Synced Oct 14, 2026, 10:33 AM EDT
Enrolling by invitation No phase listed Observational

A Long-Term Follow-Up Study of Participants With Cystinosis Who Previously Received CTNS-RD-04

NCT05146830
View directory record Official record
Conditions
Cystinosis
Interventions
Safety and Efficacy Assessments
Other
Lead sponsor
Stephanie Cherqui
Other
Eligibility
14 Years to 50 Years
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2036
U.S. locations
1
States / cities
La Jolla, California
Source: ClinicalTrials.gov public record
Updated Jul 3, 2024 · Synced Oct 14, 2026, 10:33 AM EDT