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ClinicalTrials.gov public records Last synced Oct 23, 2026, 10:05 AM EDT

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Showing 433–456 of 1,510 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Inborn Errors of Metabolism Disorders Clear all
Completed Not applicable Interventional Accepts healthy volunteers

Habitual Diet and Avocado Trial

NCT03528031
View directory record Official record
Conditions
Intra-abdominal Fat, Metabolic Syndrome, High Cholesterol, Triglycerides High, Diet Habit, High Blood Sugar, Liver Fat, Dietary Modification, HDL Cholesterol, Low Serum, Cardiovascular Diseases, High Density Lipoprotein Deficiency, Low-density-lipoprotein-type, Cardiovascular Risk Factor, Diabetes
Interventions
Intervention Daily Avocado
Other
Lead sponsor
Wake Forest University Health Sciences
Other
Eligibility
25 Years and older
Enrollment
1,008 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2018 – 2020
U.S. locations
4
States / cities
Loma Linda, California • Los Angeles, California • Boston, Massachusetts + 1 more
Source: ClinicalTrials.gov public record
Updated Mar 22, 2023 · Synced Oct 23, 2026, 10:05 AM EDT
Not listed No phase listed Observational Accepts healthy volunteers

Immune Biomarkers Related to Bone Pathology in Patients With Type 1 Gaucher Disease

NCT04055831
View directory record Official record
Conditions
Gaucher Disease Type 1
Interventions
Not listed
Lead sponsor
Lysosomal and Rare Disorders Research and Treatment Center, Inc.
Other
Eligibility
16 Years to 90 Years
Enrollment
40 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2019 – 2020
U.S. locations
1
States / cities
Fairfax, Virginia
Source: ClinicalTrials.gov public record
Updated Aug 15, 2019 · Synced Oct 23, 2026, 10:05 AM EDT
Recruiting Not applicable Interventional

Effects of Whole-body Electrical Muscle Stimulation Exercise on Adults With Neuromuscular Disease

NCT07478172
View directory record Official record
Conditions
Neuromuscular Diseases (NMD), Amyotrophic Lateral Sclerosis, Myasthenia Gravis, Lambert-eaton Myasthenic Syndrome, Primary Lateral Sclerosis, Spinal Muscular Atrophy, Charcot Marie Tooth Disease (CMT), Fascioscapulohumeral Muscular Dystrophy, Inclusion Body Myositis, Mitochondrial Myopathy, Nemaline Myopathy, Centronuclear Myopathy, Postpolio Syndrome, Pompe Disease (Late-onset), Chronic Inflammatory Demyelinating Polyneuropathy, Hereditary Spastic Paraplegia, Postural Orthostatic Tachycardia Syndrome (POTS), Progressive Muscular Atrophy
Interventions
Whole-body Electrical Muscle Stimulation Exercise
Device
Lead sponsor
University of Missouri-Columbia
Other
Eligibility
18 Years and older
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2031
U.S. locations
1
States / cities
Columbia, Missouri
Source: ClinicalTrials.gov public record
Updated Mar 17, 2026 · Synced Oct 23, 2026, 10:05 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 23, 2026, 10:05 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Association Between Lactase Deficiency, and the Small Intestinal Microbiome in Adults.

NCT03860051
View directory record Official record
Conditions
Lactose Intolerance
Interventions
Not listed
Lead sponsor
Indiana University
Other
Eligibility
18 Years to 75 Years
Enrollment
37 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2018 – 2023
U.S. locations
1
States / cities
Indianapolis, Indiana
Source: ClinicalTrials.gov public record
Updated Mar 6, 2024 · Synced Oct 23, 2026, 10:05 AM EDT
Withdrawn No phase listed Observational

A Screening Study Evaluating Disease Status of Gaucher Type I Patients

NCT00795197
View directory record Official record
Conditions
Gaucher Disease, Type 1
Interventions
Not listed
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
16 Years to 65 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2009
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated May 8, 2015 · Synced Oct 23, 2026, 10:05 AM EDT
Completed Not applicable Interventional

Stem Cell Transplantation (SCT) for Genetic Diseases

NCT00004378
View directory record Official record
Conditions
Thrombocytopenia, Metachromatic Leukodystrophy, Fanconi's Anemia, Thalassemia Major, Pure Red-Cell Aplasia, Inborn Errors of Metabolism
Interventions
Stem Cell Transplantation
Procedure
Lead sponsor
National Center for Research Resources (NCRR)
NIH
Eligibility
0 Years to 17 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1995
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Jun 24, 2005 · Synced Oct 23, 2026, 10:05 AM EDT
Completed Phase 3 Interventional Results available

Open-Label Extension Study of the Long-Term Effects of Migalastat HCL in Patients With Fabry Disease

NCT02194985
View directory record Official record
Conditions
Fabry Disease
Interventions
migalastat HCl 150 mg
Drug
Lead sponsor
Amicus Therapeutics
Industry
Eligibility
18 Years and older
Enrollment
84 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2019
U.S. locations
7
States / cities
Atlanta, Georgia • Kansas City, Kansas • Grand Rapids, Michigan + 4 more
Source: ClinicalTrials.gov public record
Updated Dec 21, 2020 · Synced Oct 23, 2026, 10:05 AM EDT
Completed Phase 2 Interventional Results available

A Study to Evaluate Efficacy and Safety of Anakinra in the Treatment of Acute Gouty Arthritis

NCT03002974
View directory record Official record
Conditions
Acute Gouty Arthritis
Interventions
Anakinra 100 mg, Triamcinolone Acetonide 40 mg, Placebo to Anakinra 100 mg, Placebo to Triamcinolone Acetonide 40 mg
Drug
Lead sponsor
Swedish Orphan Biovitrum
Industry
Eligibility
18 Years and older
Enrollment
165 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2019
U.S. locations
37
States / cities
Birmingham, Alabama • Gulf Shores, Alabama • Mobile, Alabama + 32 more
Source: ClinicalTrials.gov public record
Updated Jul 15, 2020 · Synced Oct 23, 2026, 10:05 AM EDT
Recruiting No phase listed Observational

Abbott Vascular Medical Device Registry

NCT04573660
View directory record Official record
Conditions
Acute Myocardial Infarction, Restenoses, Coronary, Coronary Artery Lesions, Venous Embolism, Arterial Embolism
Interventions
Coronary and peripheral stents, Pacing catheters, Vascular plugs, Measurement and imaging (FFR and OCT), Peripheral dilatation catheters, Coronary dilatation catheters, Coronary and peripheral guidewires, Vessel closure/compression devices, Vascular access introducers
Device
Lead sponsor
Abbott Medical Devices
Industry
Eligibility
18 Years and older
Enrollment
3,784 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2031
U.S. locations
7
States / cities
Birmingham, Alabama • Little Rock, Arkansas • Los Angeles, California + 4 more
Source: ClinicalTrials.gov public record
Updated Oct 13, 2026 · Synced Oct 23, 2026, 10:05 AM EDT
Completed Phase 1Phase 2 Interventional

AAVRh.10 Administered to Children With Late Infantile Neuronal Ceroid Lipofuscinosis

NCT01414985
View directory record Official record
Conditions
Late Infantile Neuronal Ceroid Lipofuscinosis, Batten Disease
Interventions
AAVrh.10CUCLN2
Biological
Lead sponsor
Weill Medical College of Cornell University
Other
Eligibility
3 Years to 18 Years
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2017
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Aug 31, 2020 · Synced Oct 23, 2026, 10:05 AM EDT
Completed No phase listed Observational

The Classification and Cause of Leukodystrophies of Unknown Cause

NCT00001671
View directory record Official record
Conditions
Lysosomal Storage Disease
Interventions
Not listed
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
Not listed
Enrollment
400 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1997 – 2008
U.S. locations
2
States / cities
San Francisco, California • Washington D.C., District of Columbia
Source: ClinicalTrials.gov public record
Updated Jul 2, 2017 · Synced Oct 23, 2026, 10:05 AM EDT
Completed No phase listed Observational

Dietary Monosaccharide Supplementation in Patients With Congenital Disorders of Glycosylation

NCT04198987
View directory record Official record
Conditions
Congenital Disorder of Glycosylation
Interventions
Not listed
Lead sponsor
Mayo Clinic
Other
Eligibility
Not listed
Enrollment
20 participants
Timeline
2018 – 2025
U.S. locations
1
States / cities
Rochester, Minnesota
Source: ClinicalTrials.gov public record
Updated Oct 1, 2025 · Synced Oct 23, 2026, 10:05 AM EDT
Active, not recruiting No phase listed Observational

Liver Disease in Urea Cycle Disorders

NCT04612764
View directory record Official record
Conditions
Urea Cycle Disorder, Ornithine Transcarbamylase Deficiency, Citrullinemia 1, ARGI Deficiency, ASL Deficiency, Argininosuccinic Aciduria, ASS Deficiency, Hyperargininemia
Interventions
Not listed
Lead sponsor
Baylor College of Medicine
Other
Eligibility
6 Years to 65 Years
Enrollment
62 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2026
U.S. locations
5
States / cities
Aurora, Colorado • Washington D.C., District of Columbia • Philadelphia, Pennsylvania + 2 more
Source: ClinicalTrials.gov public record
Updated Apr 27, 2026 · Synced Oct 23, 2026, 10:05 AM EDT
Recruiting Phase 1Phase 2 Interventional

64Cu-GRIP B in Patients With Advanced Malignancies

NCT05888532
View directory record Official record
Conditions
Prostate Cancer, Renal Cancer, Urethral Cancer, Advanced Solid Tumor, Metastatic Castration-resistant Prostate Cancer, Solid Tumor, Adult
Interventions
Copper-64 labeled Granzyme B (64Cu-GRIP B), Positron Emission Tomography (PET)
Drug · Procedure
Lead sponsor
Rahul Aggarwal
Other
Eligibility
18 Years and older
Enrollment
91 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2027
U.S. locations
1
States / cities
San Francisco, California
Source: ClinicalTrials.gov public record
Updated Mar 17, 2026 · Synced Oct 23, 2026, 10:05 AM EDT
Completed Phase 3 Interventional Results available

Study in Participants With Homozygous Familial Hypercholesterolemia (HoFH)

NCT03156621
View directory record Official record
Conditions
Homozygous Familial Hypercholesterolemia
Interventions
Alirocumab, Placebo
Drug
Lead sponsor
Regeneron Pharmaceuticals
Industry
Eligibility
18 Years and older
Enrollment
69 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2020
U.S. locations
4
States / cities
Boca Raton, Florida • New York, New York • Cincinnati, Ohio + 1 more
Source: ClinicalTrials.gov public record
Updated Jun 29, 2021 · Synced Oct 23, 2026, 10:05 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 23, 2026, 10:05 AM EDT
Completed Phase 1Phase 2 Interventional Results available

An Extension Study of HGT-HIT-045 Evaluating Long-Term Safety and Clinical Outcomes of Idursulfase-IT in Conjunction With Elaprase in Pediatric Participants With Hunter Syndrome and Cognitive Impairment

NCT01506141
View directory record Official record
Conditions
Hunter Syndrome
Interventions
Idursulfase-IT, Elaprase
Drug
Lead sponsor
Takeda
Industry
Eligibility
3 Years to 18 Years · Male only
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2024
U.S. locations
7
States / cities
Chicago, Illinois • Chapel Hill, North Carolina • Portland, Oregon + 4 more
Source: ClinicalTrials.gov public record
Updated Aug 6, 2025 · Synced Oct 23, 2026, 10:05 AM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

A Phase 1 Open-Label Study to Evaluate the Metabolism and Excretion of CC-223 and the Effect of Food on the Pharmacokinetics of CC-223 in Healthy Male Adult Subjects

NCT01611467
View directory record Official record
Conditions
Safety and Pharmacokinetics in Healthy Volunteer Subjects
Interventions
CC-223
Drug
Lead sponsor
Celgene
Industry
Eligibility
18 Years to 55 Years · Male only
Enrollment
18 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2012
U.S. locations
1
States / cities
Madison, Wisconsin
Source: ClinicalTrials.gov public record
Updated Nov 12, 2019 · Synced Oct 23, 2026, 10:05 AM EDT
Completed Phase 3 Interventional

Tigulixostat, Phase 3 Study, Allopurinol Controlled in Gout Patients

NCT05586971
View directory record Official record
Conditions
Gout, Hyperuricemia, Gout Flare, Tophi
Interventions
Tigulixostat, Allopurinol, Placebo
Drug
Lead sponsor
LG Chem
Industry
Eligibility
18 Years to 85 Years
Enrollment
2,202 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2025
U.S. locations
124
States / cities
Birmingham, Alabama • Daphne, Alabama • Mobile, Alabama + 91 more
Source: ClinicalTrials.gov public record
Updated Jul 22, 2025 · Synced Oct 23, 2026, 10:05 AM EDT
Completed Phase 2 Interventional Results available

Phase 2 Study of Obeticholic Acid for Lipodystrophy Patients

NCT02430077
View directory record Official record
Conditions
Familial Partial Lipodystrophy
Interventions
Obeticholic Acid, Placebo
Drug
Lead sponsor
Abhimanyu Garg
Other
Eligibility
18 Years to 70 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2022
U.S. locations
2
States / cities
Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Aug 27, 2024 · Synced Oct 23, 2026, 10:05 AM EDT
Completed Phase 2 Interventional

Effectiveness, Safety and Tolerability Study of RP-G28 for Symptoms Associated With Lactose Intolerance

NCT01113619
View directory record Official record
Conditions
Lactose Intolerance
Interventions
RP-G28 or placebo
Drug
Lead sponsor
Ritter Pharmaceuticals, Inc.
Industry
Eligibility
18 Years to 64 Years
Enrollment
61 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011
U.S. locations
2
States / cities
Honolulu, Hawaii • Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Dec 8, 2011 · Synced Oct 23, 2026, 10:05 AM EDT
Completed No phase listed Observational

LOWER: Lomitapide Observational Worldwide Evaluation Registry

NCT02135705
View directory record Official record
Conditions
Homozygous Familial Hypercholesterolemia
Interventions
Lomitapide
Drug
Lead sponsor
Amryt Pharma
Industry
Eligibility
Not listed
Enrollment
260 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2026
U.S. locations
46
States / cities
Birmingham, Alabama • Mobile, Alabama • Cottonwood, Arizona + 37 more
Source: ClinicalTrials.gov public record
Updated May 12, 2026 · Synced Oct 23, 2026, 10:05 AM EDT
Completed Phase 3 Interventional Results available

HELIOS-A: A Study of Vutrisiran (ALN-TTRSC02) in Patients With Hereditary Transthyretin Amyloidosis (hATTR Amyloidosis)

NCT03759379
View directory record Official record
Conditions
Amyloidosis, Hereditary, Transthyretin Amyloidosis
Interventions
Patisiran, Vutrisiran
Drug
Lead sponsor
Alnylam Pharmaceuticals
Industry
Eligibility
18 Years to 85 Years
Enrollment
164 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2025
U.S. locations
15
States / cities
La Mesa, California • Aurora, Colorado • Jacksonville, Florida + 12 more
Source: ClinicalTrials.gov public record
Updated Jan 12, 2026 · Synced Oct 23, 2026, 10:05 AM EDT