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ClinicalTrials.gov public records Last synced Oct 14, 2026, 7:50 AM EDT

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Showing 193–216 of 1,608 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Metabolism, Inborn Errors Clear all
Completed Phase 3 Interventional Results available

Study of Obicetrapib & Ezetimibe Fixed Dose Combination on Top of Maximum Tolerated Lipid-Modifying Therapies

NCT06005597
View directory record Official record
Conditions
Dyslipidemias, Hypercholesterolemia, Familial Hypercholesterolemia, ASCVD, High Cholesterol
Interventions
Combination Therapy, Monotherapy obicetrapib, Monotherapy ezetimibe, Combination Therapy placebo, Obicetrapib Placebo, Ezetimibe Placebo
Combination Product · Drug · Other
Lead sponsor
NewAmsterdam Pharma
Industry
Eligibility
18 Years and older
Enrollment
407 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024
U.S. locations
45
States / cities
Gilbert, Arizona • Jacksonville, Florida • Port Orange, Florida + 37 more
Source: ClinicalTrials.gov public record
Updated Oct 8, 2025 · Synced Oct 14, 2026, 7:50 AM EDT
Completed No phase listed Observational

The Fabrazyme® and Arbs and ACE Inhibitor Treatment (FAACET) Study

NCT00446862
View directory record Official record
Conditions
Fabry Disease, Proteinuria
Interventions
enalapril and other angiotensin converting enzyme inhibitors; losartan and other angiotensin receptor blockers
Drug
Lead sponsor
University of Alabama at Birmingham
Other
Eligibility
19 Years to 85 Years
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2012
U.S. locations
5
States / cities
Birmingham, Alabama • Atlanta, Georgia • Chicago, Illinois + 2 more
Source: ClinicalTrials.gov public record
Updated Nov 19, 2013 · Synced Oct 14, 2026, 7:50 AM EDT
Completed No phase listed Observational

Long Term Follow Up Of Patients Who Have Received Gene Therapy Or Gene Marked Products

NCT00695279
View directory record Official record
Conditions
Severe Combined Immunodeficiency, Malignancy, Hematologic, Neuroblastoma, Neoplasm, Mucopolysaccharidosis I
Interventions
Venipuncture
Procedure
Lead sponsor
St. Jude Children's Research Hospital
Other
Eligibility
Not listed
Enrollment
41 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2024
U.S. locations
1
States / cities
Memphis, Tennessee
Source: ClinicalTrials.gov public record
Updated Oct 30, 2024 · Synced Oct 14, 2026, 7:50 AM EDT
Completed Phase 3 Interventional Results available

Safety and Efficacy of Self Administered Injections of Pegvaliase (>40mg/Day Dose) in Adults With PKU

NCT03694353
View directory record Official record
Conditions
Phenylketonuria (PKU)
Interventions
Pegvaliase
Drug
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
18 Years to 70 Years
Enrollment
37 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2021
U.S. locations
17
States / cities
Gainesville, Florida • Tampa, Florida • Chicago, Illinois + 14 more
Source: ClinicalTrials.gov public record
Updated Feb 17, 2022 · Synced Oct 14, 2026, 7:50 AM EDT
Terminated Not applicable Interventional Results available

Extension Study of Intrathecal Enzyme Replacement for Cognitive Decline in MPS I

NCT02232477
View directory record Official record
Conditions
Mucopolysaccharidosis I, Cognitive Decline
Interventions
Intrathecal recombinant human alpha iduronidase
Drug
Lead sponsor
agnes chen
Other
Eligibility
6 Years and older
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2020
U.S. locations
1
States / cities
Torrance, California
Source: ClinicalTrials.gov public record
Updated Oct 31, 2023 · Synced Oct 14, 2026, 7:50 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 14, 2026, 7:50 AM EDT
Completed No phase listed Observational

Lipoprotein Metabolism in Normal Volunteers and Patients With High Levels of Lipoproteins

NCT00001154
View directory record Official record
Conditions
Hypolipoproteinemia, Hypercholesterolemia, Dyslipidemia, Hyperlipidemia
Interventions
Not listed
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
18 Years to 80 Years
Enrollment
759 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1976
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 13, 2026 · Synced Oct 14, 2026, 7:50 AM EDT
Recruiting No phase listed Observational

Long-term Follow-up (LTFU) Study of Participants in Any iECURE Protocol Using an Investigational Product (IP)

NCT06805695
View directory record Official record
Conditions
Ornithine Transcarbamylase Deficiency, Ornithine Transcarbamylase Deficiency Disease, Urea Cycle Disorders, Inborn
Interventions
No Intervention
Other
Lead sponsor
iECURE, Inc.
Industry
Eligibility
7 Months to 15 Months
Enrollment
13 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2041
U.S. locations
2
States / cities
Aurora, Colorado • New York, New York
Source: ClinicalTrials.gov public record
Updated Aug 31, 2026 · Synced Oct 14, 2026, 7:50 AM EDT
Completed Phase 1Phase 2 Interventional

RGX-121 Gene Therapy in Children 5 Years of Age and Over With MPS II (Hunter Syndrome)

NCT04571970
View directory record Official record
Conditions
Mucopolysaccharidosis Type II (MPS II)
Interventions
RGX-121
Genetic
Lead sponsor
REGENXBIO Inc.
Industry
Eligibility
5 Years to 17 Years · Male only
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2024
U.S. locations
1
States / cities
Oakland, California
Source: ClinicalTrials.gov public record
Updated Jan 28, 2025 · Synced Oct 14, 2026, 7:50 AM EDT
Not listed Phase 2Phase 3 Interventional

Study of AAVrh10-h.SGSH Gene Therapy in Patients With Mucopolysaccharidosis Type IIIA (MPS IIIA)

NCT03612869
View directory record Official record
Conditions
Mucopolysaccharidosis Type IIIA
Interventions
LYS-SAF302
Drug
Lead sponsor
LYSOGENE
Industry
Eligibility
6 Months and older
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2022
U.S. locations
4
States / cities
Orange, California • Minneapolis, Minnesota • New York, New York + 1 more
Source: ClinicalTrials.gov public record
Updated Aug 31, 2021 · Synced Oct 14, 2026, 7:50 AM EDT
Completed No phase listed Observational

Study to Evaluate Blood Cell Lines From Patients With Gaucher Disease

NCT00351156
View directory record Official record
Conditions
Gaucher Disease
Interventions
Blood sample
Procedure
Lead sponsor
Amicus Therapeutics
Industry
Eligibility
Not listed
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2007
U.S. locations
7
States / cities
San Francisco, California • Coral Springs, Florida • Decatur, Georgia + 4 more
Source: ClinicalTrials.gov public record
Updated Aug 19, 2010 · Synced Oct 14, 2026, 7:50 AM EDT
Completed Phase 2 Interventional Results available

A Study to Assess the Effect of Intensive Uric Acid (UA) Lowering Therapy With RDEA3170, Febuxostat, Dapagliflozin on Urinary Excretion of UA

NCT03316131
View directory record Official record
Conditions
Asymptomatic Hyperuricemia
Interventions
Verinurad, Febuxostat, Dapagliflozin, Dapagliflozin matched placebo
Drug · Other
Lead sponsor
AstraZeneca
Industry
Eligibility
18 Years to 99 Years
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2018
U.S. locations
2
States / cities
Glendale, California • Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Aug 28, 2019 · Synced Oct 14, 2026, 7:50 AM EDT
No Longer Available No phase listed Expanded access

Single Patient Expanded Access Protocol: Metabolic Boost

NCT02021266
View directory record Official record
Conditions
Metachromatic Leukodystrophy
Interventions
Enriched Hematopoetic Stem Cell Infusion
Biological
Lead sponsor
Talaris Therapeutics Inc.
Industry
Eligibility
Female only
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Oct 8, 2020 · Synced Oct 14, 2026, 7:50 AM EDT
Not yet recruiting No phase listed Observational

PET Imaging Study Using Evuzamitide to Detect Cardiac Amyloidosis in Patients With Inconclusive Nuclear Scans and Elevated TAD1 Levels

NCT07538518
View directory record Official record
Conditions
Amyloidogenic Transthyretin (ATTR) Amyloidosis, Cardiac Amyloidosis
Interventions
Evuzamitide
Drug
Lead sponsor
Lorena Saelices
Other
Eligibility
Not listed
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2028
U.S. locations
3
States / cities
Boston, Massachusetts • New York, New York • Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Sep 3, 2026 · Synced Oct 14, 2026, 7:50 AM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

A Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ABP-671 in Healthy Subjects

NCT03906006
View directory record Official record
Conditions
Gout, Hyperuricemia
Interventions
ABP-671, Placebo
Drug · Other
Lead sponsor
Atom Therapeutics Co., Ltd
Industry
Eligibility
18 Years to 45 Years
Enrollment
32 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2018 – 2019
U.S. locations
1
States / cities
Tempe, Arizona
Source: ClinicalTrials.gov public record
Updated Jul 29, 2020 · Synced Oct 14, 2026, 7:50 AM EDT
Completed No phase listed Observational

Investigating Pompe Prevalence in Neuromuscular Medicine Academic Practices

NCT02838368
View directory record Official record
Conditions
Pompe Disease
Interventions
Not listed
Lead sponsor
University of California, Irvine
Other
Eligibility
8 Years and older
Enrollment
921 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2018
U.S. locations
1
States / cities
Irvine, California
Source: ClinicalTrials.gov public record
Updated Feb 25, 2019 · Synced Oct 14, 2026, 7:50 AM EDT
Completed Not applicable Interventional Accepts healthy volunteers

Hmong Microbiome ANd Gout, Obesity, Vitamin C (HMANGO-C)

NCT04938024
View directory record Official record
Conditions
Hyperuricemia, Gout
Interventions
Vitamin C
Dietary Supplement
Lead sponsor
University of Minnesota
Other
Eligibility
18 Years and older
Enrollment
135 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2021 – 2023
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Sep 7, 2023 · Synced Oct 14, 2026, 7:50 AM EDT
Completed Phase 3 Interventional Results available

Efficacy and Safety Study of ISIS 301012 (Mipomersen) as Add-on in Familial Hypercholesterolemic Patients With Coronary Artery Disease

NCT00706849
View directory record Official record
Conditions
Heterozygous Familial Hypercholesterolemia, Coronary Artery Disease
Interventions
mipomersen sodium, placebo
Drug
Lead sponsor
Kastle Therapeutics, LLC
Industry
Eligibility
18 Years and older
Enrollment
124 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2010
U.S. locations
37
States / cities
La Jolla, California • Los Angeles, California • Mission Viejo, California + 31 more
Source: ClinicalTrials.gov public record
Updated Sep 9, 2016 · Synced Oct 14, 2026, 7:50 AM EDT
Terminated Phase 1Phase 2 Interventional Results available

Combined Intrathecal and Intravenous VTS-270 Therapy for Liver and Neurological Disease Associated With Niemann-Pick Disease, Type C1

NCT03887533
View directory record Official record
Conditions
Niemann-Pick Disease, Type C1
Interventions
VTS-270
Drug
Lead sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
NIH
Eligibility
3 Years to 60 Years
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2021
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Aug 30, 2022 · Synced Oct 14, 2026, 7:50 AM EDT
Completed Phase 1 Interventional

A Pilot Study of Vitamin D in Boys With X-linked Adrenoleukodystrophy

NCT02595489
View directory record Official record
Conditions
X-linked Adrenoleukodystrophy
Interventions
vitamin D3
Dietary Supplement
Lead sponsor
Stanford University
Other
Eligibility
18 Months to 25 Years · Male only
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2020
U.S. locations
1
States / cities
Palo Alto, California
Source: ClinicalTrials.gov public record
Updated Jun 9, 2022 · Synced Oct 14, 2026, 7:50 AM EDT
Completed Phase 1Phase 2 Interventional Results available

Dose-Ranging Study of ST-920, an AAV2/6 Human Alpha Galactosidase A Gene Therapy in Subjects With Fabry Disease (STAAR)

NCT04046224
View directory record Official record
Conditions
Fabry Disease
Interventions
ST-920
Biological
Lead sponsor
Sangamo Therapeutics
Industry
Eligibility
18 Years and older
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2025
U.S. locations
9
States / cities
Irvine, California • Tampa, Florida • Atlanta, Georgia + 6 more
Source: ClinicalTrials.gov public record
Updated Apr 14, 2026 · Synced Oct 14, 2026, 7:50 AM EDT
Completed Phase 2 Interventional

Evaluate the Efficacy and Safety of Arhalofenate for Preventing Flares and Reducing Serum Uric Acid in Gout Patients

NCT02063997
View directory record Official record
Conditions
Gout
Interventions
Arhalofenate 600 mg, Allopurinol 300 mg, Colchicine 0.6 mg, Placebo, Arhalofenate 800 mg
Drug
Lead sponsor
Gilead Sciences
Industry
Eligibility
18 Years to 75 Years
Enrollment
248 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2015
U.S. locations
52
States / cities
Birmingham, Alabama • Scottsdale, Arizona • Tucson, Arizona + 49 more
Source: ClinicalTrials.gov public record
Updated Jan 29, 2018 · Synced Oct 14, 2026, 7:50 AM EDT
Recruiting No phase listed Observational

Extracellular Vesicles as Potential Biomarkers and Therapeutic Target in Gaucher Disease

NCT05843552
View directory record Official record
Conditions
Gaucher Disease
Interventions
no intervention
Other
Lead sponsor
University of Minnesota
Other
Eligibility
18 Years to 80 Years
Enrollment
30 participants
Timeline
2023 – 2026
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Apr 6, 2026 · Synced Oct 14, 2026, 7:50 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 14, 2026, 7:50 AM EDT