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ClinicalTrials.gov public records Last synced Oct 5, 2026, 5:21 AM EDT

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Showing 49–72 of 1,603 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Inborn Errors of Metabolism Clear all
Completed Phase 2 Interventional Results available

Efficacy of Betaine for Reduction of Urine Oxalate in Patients With Type 1 Primary Hyperoxaluria

NCT00283387
View directory record Official record
Conditions
Hyperoxaluria
Interventions
Betaine, Placebo
Drug
Lead sponsor
Mayo Clinic
Other
Eligibility
6 Years to 70 Years
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2011
U.S. locations
1
States / cities
Rochester, Minnesota
Source: ClinicalTrials.gov public record
Updated Dec 16, 2013 · Synced Oct 5, 2026, 5:21 AM EDT
Completed Phase 4 Interventional

Efficacy Study of Extended-Release Niacin/Lovastatin Versus Usual Care

NCT00345657
View directory record Official record
Conditions
Hyperlipidemia, Mixed Hyperlipidemia, Dyslipidemia
Interventions
Niacin Extended Release/Lovastatin
Drug
Lead sponsor
In His Image
Other
Eligibility
18 Years and older
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003 – 2004
U.S. locations
1
States / cities
Tulsa, Oklahoma
Source: ClinicalTrials.gov public record
Updated Jun 28, 2006 · Synced Oct 5, 2026, 5:21 AM EDT
Completed Phase 1 Interventional

Recapturing Immune Tolerance to Pegloticase for the Management of Tophaceous Gout

NCT06186219
View directory record Official record
Conditions
Tophaceous Gout
Interventions
Rituximab
Drug
Lead sponsor
University of California, Los Angeles
Other
Eligibility
18 Years and older
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Nov 20, 2025 · Synced Oct 5, 2026, 5:21 AM EDT
Completed Phase 3 Interventional

Open Label Extension of 2 mg/kg Pegunigalsidase Alfa (PRX-102) Every 4 Weeks in Adult Fabry Disease Patients

NCT03614234
View directory record Official record
Conditions
Fabry Disease
Interventions
pegunigalsidase alfa
Drug
Lead sponsor
Chiesi Farmaceutici S.p.A.
Industry
Eligibility
18 Years and older
Enrollment
29 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2026
U.S. locations
7
States / cities
Birmingham, Alabama • Atlanta, Georgia • Iowa City, Iowa + 4 more
Source: ClinicalTrials.gov public record
Updated May 22, 2026 · Synced Oct 5, 2026, 5:21 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Assessment of Primary Care Providers' Knowledge and Beliefs Regarding Gout Management

NCT01151709
View directory record Official record
Conditions
Gout
Interventions
Not listed
Lead sponsor
University of Massachusetts, Worcester
Other
Eligibility
18 Years and older
Enrollment
838 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2010 – 2012
U.S. locations
1
States / cities
Worcester, Massachusetts
Source: ClinicalTrials.gov public record
Updated Mar 7, 2012 · Synced Oct 5, 2026, 5:21 AM EDT
Completed No phase listed Observational

GM1 and GM2 Gangliosidosis PROspective Neurological Disease TrajectOry Study (PRONTO)

NCT05109793
View directory record Official record
Conditions
GM1 Gangliosidosis, Sandhoff Disease, Tay-Sachs Disease
Interventions
Not listed
Lead sponsor
Azafaros B.V.
Industry
Eligibility
2 Years to 20 Years
Enrollment
31 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2025
U.S. locations
2
States / cities
Oakland, California • Rochester, Minnesota
Source: ClinicalTrials.gov public record
Updated Sep 29, 2025 · Synced Oct 5, 2026, 5:21 AM EDT
Recruiting Phase 4 Interventional

SAVE-Care (Sodium Glucose Cotransporter-2 Inhibitors [SGLT2i] As Novel Gout Care) Trial

NCT06674109
View directory record Official record
Conditions
Gout and Hyperuricemia
Interventions
Empagliflozin 10 mg, Placebo
Drug
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
18 Years to 80 Years
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2028
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Aug 5, 2026 · Synced Oct 5, 2026, 5:21 AM EDT
Completed Phase 2 Interventional

Alpha-Galactosidase A Replacement Therapy for Fabry Disease

NCT00048906
View directory record Official record
Conditions
Fabry Disease
Interventions
DRX005B
Drug
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
Not listed
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2002 – 2003
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 4, 2008 · Synced Oct 5, 2026, 5:21 AM EDT
Terminated Phase 3 Interventional Accepts healthy volunteers Results available

A Multinational, Randomized, Double-blind, Placebo-controlled Study to Assess the Efficacy, Pharmacodynamics, Pharmacokinetics, and Safety of Venglustat in Late-onset GM2

NCT04221451
View directory record Official record
Conditions
Tay-Sachs Disease, Sandhoff Disease
Interventions
venglustat GZ402671, placebo
Drug
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
2 Years and older
Enrollment
75 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2020 – 2024
U.S. locations
5
States / cities
Los Angeles, California • Atlanta, Georgia • Bethesda, Maryland + 2 more
Source: ClinicalTrials.gov public record
Updated Jan 28, 2026 · Synced Oct 5, 2026, 5:21 AM EDT
Active, not recruiting Phase 3 Interventional Results available

Study to Evaluate the Safety and Efficacy of Pegvaliase in Adolescents (Ages 12-17) With Phenylketonuria

NCT05270837
View directory record Official record
Conditions
Phenylketonuria (PKU)
Interventions
Pegvaliase, Diet Only
Drug · Other
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
12 Years to 17 Years
Enrollment
55 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2027
U.S. locations
13
States / cities
Phoenix, Arizona • Little Rock, Arkansas • Aurora, Colorado + 10 more
Source: ClinicalTrials.gov public record
Updated Jul 21, 2026 · Synced Oct 5, 2026, 5:21 AM EDT
Recruiting Phase 4 Interventional

Effect of Theophylline in Pseudohypoparathyroidism

NCT03718403
View directory record Official record
Conditions
PHP Ia, PHP IB, Php1C
Interventions
Theophylline
Drug
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
5 Years to 21 Years
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2032
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Jul 22, 2022 · Synced Oct 5, 2026, 5:21 AM EDT
Completed Phase 2 Interventional Results available

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

NCT01599286
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Feb 15, 2021 · Synced Oct 5, 2026, 5:21 AM EDT
Terminated Phase 3 Interventional Results available

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

NCT03403205
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Jun 17, 2024 · Synced Oct 5, 2026, 5:21 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Early Detection of Neuropathy in ATTRv

NCT05311488
View directory record Official record
Conditions
Hereditary Amyloidosis, Transthyretin-Related
Interventions
neurofilament light chain, In-vivo Meissner Corpuscle imaging, Nerve conduction study
Diagnostic Test
Lead sponsor
University of Pennsylvania
Other
Eligibility
30 Years to 90 Years
Enrollment
47 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2022 – 2026
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Mar 12, 2026 · Synced Oct 5, 2026, 5:21 AM EDT
Completed No phase listed Observational Accepts healthy volunteers Results available

PCORI Urea Cycle Disorder Study

NCT02740153
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Jan 26, 2021 · Synced Oct 5, 2026, 5:21 AM EDT
Completed No phase listed Observational

Clinical and Pathophysiological Investigations Into Erdheim Chester Disease

NCT01417520
View directory record Official record
Conditions
Myelofibrosis, Gaucher Disease, Pulmonary Fibrosis, Hermansky-Pudlak Syndrome (HPS), Cancer
Interventions
Not listed
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
2 Years to 80 Years
Enrollment
88 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2019
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 26, 2019 · Synced Oct 5, 2026, 5:21 AM EDT
Completed Phase 1 Interventional

AAV2/8-LSPhGAA (ACTUS-101) in Late-Onset Pompe Disease

NCT03533673
View directory record Official record
Conditions
Pompe Disease
Interventions
ACTUS-101
Genetic
Lead sponsor
AskBio Inc
Industry
Eligibility
18 Years and older
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2026
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Apr 13, 2026 · Synced Oct 5, 2026, 5:21 AM EDT
Active, not recruiting Phase 3 Interventional

A Study to Evaluate the Effect of Venglustat Tablets on Neuropathic and Abdominal Pain in Male and Female Participants ≥16 Years of Age With Fabry Disease

NCT05206773
View directory record Official record
Conditions
Fabry Disease
Interventions
Venglustat (GZ402671), Placebo
Drug
Lead sponsor
Sanofi
Industry
Eligibility
16 Years and older
Enrollment
122 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2027
U.S. locations
12
States / cities
Birmingham, Alabama • Los Angeles, California • Orange, California + 9 more
Source: ClinicalTrials.gov public record
Updated Feb 4, 2026 · Synced Oct 5, 2026, 5:21 AM EDT
Completed Not applicable Interventional Accepts healthy volunteers Results available

North Carolina Newborn Exome Sequencing for Universal Screening

NCT02826694
View directory record Official record
Conditions
Metabolism, Inborn Errors, Hearing Loss, Hereditary Disease
Interventions
Well infant, whole exome sequencing, Diagnosed, whole exome sequencing
Genetic
Lead sponsor
University of North Carolina, Chapel Hill
Other
Eligibility
1 Hour to 5 Years
Enrollment
106 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2016 – 2019
U.S. locations
1
States / cities
Chapel Hill, North Carolina
Source: ClinicalTrials.gov public record
Updated Jul 8, 2020 · Synced Oct 5, 2026, 5:21 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

A Study to Assess Plasma Ammonia Time-Normalized Area Under the Curve and Rate of Ureagenesis in Healthy Adult Subjects

NCT04269122
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Mar 30, 2020 · Synced Oct 5, 2026, 5:21 AM EDT
Completed Phase 3 Interventional Results available

Study of Phenoptin to Increase Phenylalanine Tolerance in Phenylketonuric Children on a Phenylalanine-restricted Diet

NCT00272792
View directory record Official record
Conditions
Phenylketonurias
Interventions
Sapropterin Dihydrochloride, Placebo
Drug
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
4 Years to 12 Years
Enrollment
45 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006
U.S. locations
9
States / cities
Los Angeles, California • Oakland, California • Sacramento, California + 6 more
Source: ClinicalTrials.gov public record
Updated Aug 13, 2015 · Synced Oct 5, 2026, 5:21 AM EDT
Completed Early Phase 1 Interventional Accepts healthy volunteers

Comparative Bioavailability Study of TAH3311 5 mg Oral Dissolving Film vs ELIQUIS® 5 mg Tablet in Healthy Volunteers

NCT05995119
View directory record Official record
Conditions
Healthy Volunteers, Fasting, Fed
Interventions
Apixaban 5 mg Oral Dissolving Film (TAH3311) and Tablet (Eliquis®)
Drug
Lead sponsor
TAHO Pharmaceuticals Ltd.
Industry
Eligibility
18 Years and older
Enrollment
12 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2022 – 2023
U.S. locations
1
States / cities
Las Vegas, Nevada
Source: ClinicalTrials.gov public record
Updated Aug 18, 2023 · Synced Oct 5, 2026, 5:21 AM EDT
Completed No phase listed Observational

The Natural History of Metachromatic Leukodystrophy Study (HOME Study)

NCT04628364
View directory record Official record
Conditions
Metachromatic Leukodystrophy
Interventions
Not listed
Lead sponsor
National Organization for Rare Disorders
Other
Eligibility
Not listed
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2025
U.S. locations
1
States / cities
Danbury, Connecticut
Source: ClinicalTrials.gov public record
Updated Jun 8, 2025 · Synced Oct 5, 2026, 5:21 AM EDT
Recruiting No phase listed Observational

Hepatic Histopathology in Urea Cycle Disorders

NCT04908319
View directory record Official record
Conditions
Urea Cycle Disorder, Ornithine Transcarbamylase Deficiency, Citrullinemia 1, ARGI Deficiency, ASL Deficiency, Argininosuccinic Aciduria, ASS Deficiency, Hyperargininemia, Carbamyl Phosphate Synthetase Deficiency, NAGS Deficiency
Interventions
Not listed
Lead sponsor
Baylor College of Medicine
Other
Eligibility
Not listed
Enrollment
70 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2026
U.S. locations
2
States / cities
Washington D.C., District of Columbia • Houston, Texas
Source: ClinicalTrials.gov public record
Updated Jul 20, 2025 · Synced Oct 5, 2026, 5:21 AM EDT