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ClinicalTrials.gov public records Last synced Oct 27, 2026, 5:47 PM EDT

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Showing 457–480 of 2,422 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Metabolic Abnormalities Clear all
Completed Phase 1 Interventional

Gene Therapy for Male Patients With Danon Disease (DD) Using RP-A501; AAV9.LAMP2B

NCT03882437
View directory record Official record
Conditions
Danon Disease
Interventions
RP-A501
Biological
Lead sponsor
Rocket Pharmaceuticals Inc.
Industry
Eligibility
8 Years and older · Male only
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2025
U.S. locations
3
States / cities
La Jolla, California • Aurora, Colorado • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Oct 6, 2026 · Synced Oct 27, 2026, 5:47 PM EDT
Completed Phase 4 Interventional Results available

An Exploratory Study of the Safety and Efficacy of Prophylactic Immunomodulatory Treatment in Myozyme-naive Cross-Reacting Immunologic Material (CRIM[-]) Patients With Infantile-Onset Pompe Disease

NCT00701129
View directory record Official record
Conditions
Pompe Disease, Glycogen Storage Disease Type II
Interventions
Alglucosidase Alfa, Methotrexate, Rituximab
Biological · Drug
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
Not listed
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2013
U.S. locations
2
States / cities
Louisville, Kentucky • Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated May 13, 2014 · Synced Oct 27, 2026, 5:47 PM 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 27, 2026, 5:47 PM EDT
Terminated Phase 3 Interventional

ALD-101 Adjuvant Therapy of Unrelated Umbilical Cord Blood Transfusion (UCBT) in Patients With Inherited Metabolic Diseases

NCT00654433
View directory record Official record
Conditions
Inherited Metabolic Diseases, Lysosomal Storage Disorders, Peroxisomal Storage Diseases, Inborn Errors of Metabolism, Mucopolysaccharidosis
Interventions
ALD-101
Biological
Lead sponsor
Aldagen
Industry
Eligibility
Up to 16 Years
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2011
U.S. locations
3
States / cities
Los Angeles, California • New York, New York • Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Jul 8, 2014 · Synced Oct 27, 2026, 5:47 PM EDT
Not yet recruiting Phase 3 Interventional

Long-Term Efficacy Study of Vatiquinone for the Treatment of Friedreich's Ataxia (FA)

NCT07681713
View directory record Official record
Conditions
Friedreich's Ataxia
Interventions
Vatiquinone
Drug
Lead sponsor
PTC Therapeutics
Industry
Eligibility
7 Years to 21 Years
Enrollment
120 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2029
U.S. locations
4
States / cities
Los Angeles, California • Tampa, Florida • Iowa City, Iowa + 1 more
Source: ClinicalTrials.gov public record
Updated Aug 14, 2026 · Synced Oct 27, 2026, 5:47 PM EDT
Active, not recruiting Phase 2 Interventional

A Study of Setmelanotide in Patients With Prader-Willi Syndrome

NCT06772597
View directory record Official record
Conditions
Prader-Willi Syndrome, Obesity, Hyperphagia
Interventions
Setmelanotide
Drug
Lead sponsor
Rhythm Pharmaceuticals, Inc.
Industry
Eligibility
6 Years to 65 Years
Enrollment
18 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
1
States / cities
Gainesville, Florida
Source: ClinicalTrials.gov public record
Updated Feb 12, 2026 · Synced Oct 27, 2026, 5:47 PM EDT
Completed Phase 2 Interventional Results available

A Study of LJPC-401 for the Treatment of Iron Overload in Adult Patients With Hereditary Hemochromatosis

NCT03395704
View directory record Official record
Conditions
Hereditary Hemochromatosis
Interventions
LJPC-401, Placebo
Drug
Lead sponsor
La Jolla Pharmaceutical Company
Industry
Eligibility
18 Years and older
Enrollment
70 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2019
U.S. locations
18
States / cities
North Little Rock, Arkansas • Los Angeles, California • Palo Alto, California + 15 more
Source: ClinicalTrials.gov public record
Updated Jun 9, 2022 · Synced Oct 27, 2026, 5:47 PM 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 27, 2026, 5:47 PM EDT
Active, not recruiting Phase 1Phase 2 Interventional

A Study of mRNA-3745 in Adult and Pediatric Participants With Glycogen Storage Disease Type 1a (GSD1a)

NCT05095727
View directory record Official record
Conditions
Glycogen Storage Disease
Interventions
mRNA-3745
Drug
Lead sponsor
ModernaTX, Inc.
Industry
Eligibility
6 Years and older
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2026
U.S. locations
8
States / cities
Farmington, Connecticut • Boston, Massachusetts • Durham, North Carolina + 4 more
Source: ClinicalTrials.gov public record
Updated Dec 15, 2025 · Synced Oct 27, 2026, 5:47 PM EDT
Completed Not applicable Interventional Accepts healthy volunteers

Evaluation of GIMate Handheld Hydrogen Breath Monitor for Diagnosis of Lactose Malabsorption

NCT04754724
View directory record Official record
Conditions
Lactose Intolerance
Interventions
GIMate, H2 Check
Diagnostic Test
Lead sponsor
Vivante Health
Industry
Eligibility
18 Years to 55 Years
Enrollment
31 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2021
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Sep 28, 2021 · Synced Oct 27, 2026, 5:47 PM EDT
Completed Not applicable Interventional Accepts healthy volunteers

Clinical Evaluation of Acuity 200 (Fluoroxyfocon A) RGP Contact Lens

NCT03971227
View directory record Official record
Conditions
Refractive Errors
Interventions
Acuity 200 (Fluoroxyfocon A) contact lens, Acuity 100 (Hexafocon A) contact lens
Device
Lead sponsor
Acuity Polymers, Inc.
Industry
Eligibility
18 Years and older
Enrollment
74 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2019 – 2020
U.S. locations
3
States / cities
Honolulu, Hawaii • Edina, Minnesota • Pittsford, New York
Source: ClinicalTrials.gov public record
Updated May 12, 2020 · Synced Oct 27, 2026, 5:47 PM EDT
Completed Phase 2 Interventional

Non-Ablative Allo HSCT For Hematologic Malignancies or SAA

NCT00006379
View directory record Official record
Conditions
Chronic Myeloproliferative Disorders, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Diseases, Precancerous/Nonmalignant Condition, Small Intestine Cancer
Interventions
anti-thymocyte globulin, graft-versus-tumor induction therapy, cyclophosphamide, fludarabine phosphate, peripheral blood stem cell transplantation
Biological · Drug · Procedure
Lead sponsor
Case Comprehensive Cancer Center
Other
Eligibility
Up to 70 Years
Enrollment
58 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2000 – 2011
U.S. locations
1
States / cities
Cleveland, Ohio
Source: ClinicalTrials.gov public record
Updated Dec 8, 2011 · Synced Oct 27, 2026, 5:47 PM EDT
Completed Phase 1 Interventional

An Open-Label Maintenance Study of the Enzyme Replacement Therapy Replagal in Patients With Fabry Disease

NCT00357786
View directory record Official record
Conditions
Fabry Disease
Interventions
Replagal agalsidase alfa, Replagal
Drug
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
39 Years to 45 Years · Male only
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2003
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Aug 17, 2011 · Synced Oct 27, 2026, 5:47 PM EDT
Completed Phase 1 Interventional

Safety and Pharmacodynamics of SEL-037 (Pegsiticase) in Subjects With Elevated Blood Uric Acid Levels

NCT02464605
View directory record Official record
Conditions
Gout
Interventions
SEL-037 (pegsiticase)
Biological
Lead sponsor
Selecta Biosciences, Inc.
Industry
Eligibility
21 Years to 75 Years
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2016
U.S. locations
2
States / cities
Duncansville, Pennsylvania • Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Oct 21, 2016 · Synced Oct 27, 2026, 5:47 PM EDT
Recruiting No phase listed Observational

Natural History Study of Patients With Excess Androgen

NCT00250159
View directory record Official record
Conditions
Congenital Adrenal Hyperplasia (CAH), Familial Male-Limited Precocious Puberty (FMPP)
Interventions
Not listed
Lead sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
NIH
Eligibility
1 Day to 99 Years
Enrollment
3,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2006
U.S. locations
2
States / cities
Washington D.C., District of Columbia • Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 8, 2026 · Synced Oct 27, 2026, 5:47 PM EDT
Recruiting Phase 3 Interventional

A Study of JNT-517 in Participants With Phenylketonuria (PKU)

NCT06971731
View directory record Official record
Conditions
Phenylketonuria
Interventions
JNT-517, JNT-517 Matching Placebo
Drug
Lead sponsor
Otsuka Pharmaceutical Development & Commercialization, Inc.
Industry
Eligibility
4 Years and older
Enrollment
120 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2028
U.S. locations
10
States / cities
Los Angeles, California • Gainesville, Florida • Tampa, Florida + 7 more
Source: ClinicalTrials.gov public record
Updated Oct 21, 2026 · Synced Oct 27, 2026, 5:47 PM 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 27, 2026, 5:47 PM EDT
Recruiting Phase 3 Interventional

A Study of Pitolisant in Patients With Prader-Willi Syndrome

NCT06366464
View directory record Official record
Conditions
Prader-Willi Syndrome
Interventions
Pitolisant tablet, Placebo tablet
Drug · Other
Lead sponsor
Harmony Biosciences Management, Inc.
Industry
Eligibility
6 Years and older
Enrollment
134 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2028
U.S. locations
23
States / cities
Los Angeles, California • Sacramento, California • San Diego, California + 16 more
Source: ClinicalTrials.gov public record
Updated Aug 25, 2026 · Synced Oct 27, 2026, 5:47 PM EDT
Completed Phase 3 Interventional Results available

Efficacy and Safety of Lucerastat Oral Monotherapy in Adult Subjects With Fabry Disease

NCT03425539
View directory record Official record
Conditions
Fabry Disease
Interventions
Lucerastat, Placebo
Drug
Lead sponsor
Idorsia Pharmaceuticals Ltd.
Industry
Eligibility
18 Years and older
Enrollment
118 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2021
U.S. locations
17
States / cities
Birmingham, Alabama • Irvine, California • Oakland, California + 14 more
Source: ClinicalTrials.gov public record
Updated Aug 9, 2024 · Synced Oct 27, 2026, 5:47 PM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

Renal PK Study of LC350189

NCT04066712
View directory record Official record
Conditions
Gout, Hyperuricemia
Interventions
LC350189 200 mg
Drug
Lead sponsor
LG Chem
Industry
Eligibility
18 Years to 80 Years
Enrollment
37 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2019 – 2020
U.S. locations
1
States / cities
Orlando, Florida
Source: ClinicalTrials.gov public record
Updated Sep 21, 2020 · Synced Oct 27, 2026, 5:47 PM EDT
Completed Phase 4 Interventional Results available

A Prospective Study to Investigate Safety and Tolerability of Shorter Infusion of Fabrazyme

NCT06019728
View directory record Official record
Conditions
Fabry's Disease
Interventions
AGALSIDASE BETA (GZ419828), Acetaminophen, Diphenhydramine, Dexamethasone, Montelukast, Loratadine, Cetirizine, Fexofenadine
Drug
Lead sponsor
Sanofi
Industry
Eligibility
2 Years to 65 Years
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2024
U.S. locations
5
States / cities
Santa Monica, California • Atlanta, Georgia • Grand Rapids, Michigan + 2 more
Source: ClinicalTrials.gov public record
Updated Oct 21, 2025 · Synced Oct 27, 2026, 5:47 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Uncertain Genetic Test Results for Lynch Syndrome

NCT01646112
View directory record Official record
Conditions
HNPCC
Interventions
Not listed
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
18 Years to 100 Years
Enrollment
27 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2012 – 2016
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Dec 17, 2019 · Synced Oct 27, 2026, 5:47 PM EDT
Active, not recruiting No phase listed Observational Accepts healthy volunteers

Diffusion Tensor Imaging (DTI) in Infants With Krabbe Disease

NCT00787865
View directory record Official record
Conditions
Krabbe Disease
Interventions
Not listed
Lead sponsor
University of Pittsburgh
Other
Eligibility
Up to 17 Years
Enrollment
100 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2008 – 2027
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Mar 6, 2026 · Synced Oct 27, 2026, 5:47 PM EDT
Not listed No phase listed Observational

Study of Pulmonary Complications in Pediatric Patients With Storage Disorders Undergoing Allogeneic Hematopoietic Stem Cell Transplantation

NCT00005900
View directory record Official record
Conditions
I Cell Disease, Fucosidosis, Globoid Cell Leukodystrophy, Adrenoleukodystrophy, Mannosidosis, Niemann-Pick Disease, Pulmonary Complications, Mucopolysaccharidosis I, Mucopolysaccharidosis VI, Metachromatic Leukodystrophy, Gaucher's Disease, Wolman Disease
Interventions
Not listed
Lead sponsor
Fairview University Medical Center
Other
Eligibility
0 Years and older
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1999
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Jun 24, 2005 · Synced Oct 27, 2026, 5:47 PM EDT