Independent directory Public ClinicalTrials.gov records United States
Clinical Trials Finder
Objective public record navigation
Search Results

Search by objective public record fields.

Search by query text, NCT ID, condition, intervention, sponsor, city, state, recruitment status, phase, study type, healthy volunteer eligibility, sex, or age. Results come from ClinicalTrials.gov. When the live registry is unavailable, a clearly labeled stored copy may be shown.

Clear filters

These results were retrieved from the live ClinicalTrials.gov registry. Filters and sort order apply to this result list.

ClinicalTrials.gov public records Last synced Nov 1, 2026, 5:19 AM EST

Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.

Showing 817–840 of 1,511 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Inborn Errors of Metabolism Disorders Clear all
Completed No phase listed Observational Accepts healthy volunteers

Intracardiac Flow Assessment in Cardiac Amyloidosis

NCT05379101
View directory record Official record
Conditions
Amyloidosis Cardiac, Healthy Adults
Interventions
Cardiac magnetic resonance image (CMR), Transthoracic Echocardiogram (TTE), Six-minute Walk Test
Diagnostic Test
Lead sponsor
Mayo Clinic
Other
Eligibility
40 Years and older
Enrollment
81 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2022 – 2026
U.S. locations
1
States / cities
Rochester, Minnesota
Source: ClinicalTrials.gov public record
Updated Jun 29, 2026 · Synced Nov 1, 2026, 5:19 AM EST
Terminated Phase 2 Interventional

Human Heterologous Liver Cells for Infusion in Children With Urea Cycle Disorders

NCT01195753
View directory record Official record
Conditions
Urea Cycle Disorders
Interventions
HHLivC
Biological
Lead sponsor
Cytonet GmbH & Co. KG
Industry
Eligibility
1 Day to 5 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2015
U.S. locations
4
States / cities
Palo Alto, California • San Diego, California • New Haven, Connecticut + 1 more
Source: ClinicalTrials.gov public record
Updated Feb 8, 2016 · Synced Nov 1, 2026, 5:19 AM EST
Completed Phase 2 Interventional Results available

Simvastatin Therapy in Smith-Lemli-Opitz Syndrome

NCT00064792
View directory record Official record
Conditions
Smith-Lemli-Opitz Syndrome
Interventions
Simvastatin Susp., OraPlus
Drug
Lead sponsor
Forbes Porter, M.D.
NIH
Eligibility
4 Years to 18 Years
Enrollment
23 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003 – 2010
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jun 6, 2014 · Synced Nov 1, 2026, 5:19 AM EST
Completed No phase listed Observational

Study of Clofarabine and Fludarabine Drug Exposure in Pediatric Bone Marrow Transplantation (HCT)

NCT03609814
View directory record Official record
Conditions
Hematologic Malignancies, Nonmalignant Diseases, Immunodeficiencies, Hemoglobinopathies, Genetic Inborn Errors of Metabolism, Fanconi's Anemia, Thalassemia, Sickle Cell Disease
Interventions
Clofarabine, Fludarabine Injection
Drug
Lead sponsor
University of California, San Francisco
Other
Eligibility
Up to 17 Years
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2020
U.S. locations
1
States / cities
San Francisco, California
Source: ClinicalTrials.gov public record
Updated Oct 4, 2021 · Synced Nov 1, 2026, 5:19 AM EST
Active, not recruiting Phase 3 Interventional Results available

HELIOS-B: A Study to Evaluate Vutrisiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy

NCT04153149
View directory record Official record
Conditions
Transthyretin Amyloidosis (ATTR) With Cardiomyopathy
Interventions
Vutrisiran, Sterile Normal Saline (0.9% NaCl)
Drug
Lead sponsor
Alnylam Pharmaceuticals
Industry
Eligibility
18 Years to 85 Years
Enrollment
655 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2026
U.S. locations
17
States / cities
La Mesa, California • Los Angeles, California • Stanford, California + 13 more
Source: ClinicalTrials.gov public record
Updated Jan 12, 2026 · Synced Nov 1, 2026, 5:19 AM EST
Active, not recruiting Phase 3 Interventional

A Study to Evaluate Migalastat in Fabry Subjects With Amenable GLA Variant and Renal Disease

NCT04020055
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
14 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2026
U.S. locations
3
States / cities
Atlanta, Georgia • Cleveland, Ohio • Fairfax, Virginia
Source: ClinicalTrials.gov public record
Updated Jul 6, 2026 · Synced Nov 1, 2026, 5:19 AM EST
Completed Phase 2 Interventional Results available

Ponatinib for Patients Whose Advanced Solid Tumor Cancer Has Activating Mutations Involving the Following Genes: FGFR1, FGFR2, FGFR3, FGFR4, RET, KIT.

NCT02272998
View directory record Official record
Conditions
Malignant Neoplasm
Interventions
ponatinib hydrochloride, laboratory biomarker analysis
Drug · Other
Lead sponsor
Sameek Roychowdhury
Other
Eligibility
18 Years and older
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2022
U.S. locations
2
States / cities
Ann Arbor, Michigan • Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Mar 24, 2025 · Synced Nov 1, 2026, 5:19 AM EST
Not listed Phase 2 Interventional

Phase II Study of Glucocerebrosidase in Patients With Gaucher Disease

NCT00004293
View directory record Official record
Conditions
Gaucher's Disease
Interventions
glucocerebrosidase
Drug
Lead sponsor
National Center for Research Resources (NCRR)
NIH
Eligibility
18 Years to 65 Years
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1999
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jun 24, 2005 · Synced Nov 1, 2026, 5:19 AM EST
Completed Phase 3 Interventional Results available

Celebrex In Acute Gouty Arthritis Study

NCT00549549
View directory record Official record
Conditions
Arthritis, Gouty
Interventions
Indomethacin, Celecoxib
Drug
Lead sponsor
Pfizer's Upjohn has merged with Mylan to form Viatris Inc.
Industry
Eligibility
18 Years and older
Enrollment
402 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2009
U.S. locations
43
States / cities
Glendale, Arizona • Mesa, Arizona • Paradise Valley, Arizona + 37 more
Source: ClinicalTrials.gov public record
Updated Feb 21, 2021 · Synced Nov 1, 2026, 5:19 AM EST
Completed Phase 2Phase 3 Interventional Results available

Efficacy and Safety of Inotersen in Familial Amyloid Polyneuropathy

NCT01737398
View directory record Official record
Conditions
FAP, Familial Amyloid Polyneuropathy, TTR, Transthyretin, Amyloidosis
Interventions
Inotersen, Placebo
Drug
Lead sponsor
Ionis Pharmaceuticals, Inc.
Industry
Eligibility
18 Years to 82 Years
Enrollment
173 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2017
U.S. locations
9
States / cities
Orange, California • Indianapolis, Indiana • Baltimore, Maryland + 5 more
Source: ClinicalTrials.gov public record
Updated Jul 17, 2019 · Synced Nov 1, 2026, 5:19 AM EST
Recruiting Phase 4 Interventional

Isturisa Treatment in Mild Autonomous Cortisol Secretion( MACS)

NCT07247058
View directory record Official record
Conditions
Mild Autonomous Cortisol Secretion (MACS)
Interventions
Osilodrostat (Isturisa)
Drug
Lead sponsor
Johns Hopkins University
Other
Eligibility
18 Years and older
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2029
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 23, 2026 · Synced Nov 1, 2026, 5:19 AM EST
Recruiting Phase 2 Interventional

A Study to Investigate the Clinical Effect and the Safety of PRX-115 Infused Intravenously at Different Dosing Regimens, With and Without Methotrexate, Versus Placebo in Adults Gout Patients (RELEASE)

NCT07280156
View directory record Official record
Conditions
Gout
Interventions
PRX-115, Methotrexate (MTX), PRX-115 placebo, Placebo-Methotrexate
Biological · Drug · Other
Lead sponsor
Protalix
Industry
Eligibility
18 Years and older
Enrollment
150 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2028
U.S. locations
26
States / cities
Glendale, Arizona • Little Rock, Arkansas • Anaheim, California + 20 more
Source: ClinicalTrials.gov public record
Updated Sep 29, 2026 · Synced Nov 1, 2026, 5:19 AM EST
Active, not recruiting Phase 3 Interventional Accepts healthy volunteers

Molecular Imaging of Myocardial Fibrosis in Cardiac Amyloidosis

NCT05619302
View directory record Official record
Conditions
Cardiac Amyloidosis
Interventions
[68Ga]CBP8 PET/MRI
Drug
Lead sponsor
Brigham and Women's Hospital
Other
Eligibility
18 Years and older
Enrollment
18 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2023 – 2026
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Jul 6, 2026 · Synced Nov 1, 2026, 5:19 AM EST
Completed Phase 2Phase 3 Interventional Results available

Safety and Efficacy Study of Fx-1006A in Patients With Familial Amyloidosis

NCT00409175
View directory record Official record
Conditions
Familial Amyloid Polyneuropathy
Interventions
Fx-1006A, Placebo
Drug
Lead sponsor
Pfizer
Industry
Eligibility
18 Years to 75 Years
Enrollment
128 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2009
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Dec 17, 2012 · Synced Nov 1, 2026, 5:19 AM EST
Completed Phase 2 Interventional Results available

Copper Concentration & Histopathologic Changes in Liver Biopsy in Participants With Wilson Disease Treated With ALXN1840

NCT04422431
View directory record Official record
Conditions
Wilson Disease
Interventions
Bis-Choline Tetrathiomolybdate
Drug
Lead sponsor
Alexion Pharmaceuticals, Inc.
Industry
Eligibility
18 Years and older
Enrollment
31 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2023
U.S. locations
3
States / cities
Sacramento, California • Ann Arbor, Michigan • Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Oct 18, 2024 · Synced Nov 1, 2026, 5:19 AM EST
Active, not recruiting No phase listed Observational

Evaluation of Intravenous Laronidase Pharmacokinetics Before and After Hematopoietic Cell Transplantation in Patients With Mucopolysaccharidosis Type IH.

NCT05634512
View directory record Official record
Conditions
Hematopoietic Cell Transplantation, Mucopolysaccharidosis Type I
Interventions
Laronidase therapy and a stem cell transplant
Drug
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
0 Years to 3 Years
Enrollment
13 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2026
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Oct 30, 2025 · Synced Nov 1, 2026, 5:19 AM EST
Completed Phase 2 Interventional Accepts healthy volunteers

A Study to Evaluate OPTI-BIOME™ Bacillus Subtilis MB40 on Abdominal Discomfort, Gas and Bloating in a Healthy Population

NCT02950012
View directory record Official record
Conditions
Bloating
Interventions
OPTI-BIOME™ Bacillus subtilis MB40, Placebo
Dietary Supplement · Other
Lead sponsor
BIO-CAT Microbials, LLC
Industry
Eligibility
18 Years to 75 Years
Enrollment
100 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2016 – 2017
U.S. locations
2
States / cities
Foothill Ranch, California • Orlando, Florida
Source: ClinicalTrials.gov public record
Updated Oct 29, 2021 · Synced Nov 1, 2026, 5:19 AM EST
Completed No phase listed Observational

Wolman/CESD Natural History Chart Review and Longitudinal Follow-Up

NCT01884220
View directory record Official record
Conditions
Wolman Disease, Cholesterol Ester Storage Disease, Acid Cholesteryl Ester Hydrolase Deficiency, Type 2
Interventions
There are no interventions in this study.
Other
Lead sponsor
Children's Hospital Medical Center, Cincinnati
Other
Eligibility
Not listed
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2014
U.S. locations
1
States / cities
Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Jul 28, 2015 · Synced Nov 1, 2026, 5:19 AM EST
Completed No phase listed Observational

Validating a New Severity Score System for Adults With Type 1 Gaucher Disease (GD1)

NCT01136304
View directory record Official record
Conditions
Gaucher Disease
Interventions
Imiglucerase
Drug
Lead sponsor
University Research Foundation for Lysosomal Storage Diseases, Inc.
Other
Eligibility
18 Years and older
Enrollment
173 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2013
U.S. locations
2
States / cities
Beverly Hills, California • Coral Springs, Florida
Source: ClinicalTrials.gov public record
Updated Jun 1, 2015 · Synced Nov 1, 2026, 5:19 AM EST
Terminated Phase 1 Interventional

Hepatocyte Transplantation for Liver Based Metabolic Disorders

NCT01345578
View directory record Official record
Conditions
Metabolic Diseases
Interventions
human hepatocyte transplantation, Preparative Radiation Therapy
Drug · Radiation
Lead sponsor
Ira Fox
Other
Eligibility
Up to 21 Years
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2022
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Dec 7, 2022 · Synced Nov 1, 2026, 5:19 AM EST
Completed Phase 2 Interventional Results available

A Study of AT2101 (Afegostat Tartrate) in Adult Patients With Type 1 Gaucher Disease Currently Receiving Enzyme Replacement Therapy

NCT00433147
View directory record Official record
Conditions
Gaucher Disease, Type 1, Type 1 Gaucher Disease, Gaucher Disease
Interventions
Afegostat tartrate
Drug
Lead sponsor
Amicus Therapeutics
Industry
Eligibility
18 Years to 74 Years
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2008
U.S. locations
10
States / cities
San Francisco, California • Coral Springs, Florida • Decatur, Georgia + 7 more
Source: ClinicalTrials.gov public record
Updated Sep 25, 2018 · Synced Nov 1, 2026, 5:19 AM EST
Completed Phase 3 Interventional Results available

Efficacy and Safety of AG10 in Subjects With Transthyretin Amyloid Cardiomyopathy

NCT03860935
View directory record Official record
Conditions
Amyloidosis, Amyloid Cardiomyopathy, Transthyretin Amyloidosis, Cardiomyopathies, Heart Diseases
Interventions
acoramidis, Placebo Oral Tablet
Drug
Lead sponsor
Eidos Therapeutics, a BridgeBio company
Industry
Eligibility
18 Years to 90 Years
Enrollment
632 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2023
U.S. locations
40
States / cities
Los Angeles, California • Santa Monica, California • Aurora, Colorado + 32 more
Source: ClinicalTrials.gov public record
Updated Jun 27, 2024 · Synced Nov 1, 2026, 5:19 AM EST
Completed Phase 2Phase 3 Interventional Results available

Phenylbutyrate Therapy for Maple Syrup Urine Disease

NCT01529060
View directory record Official record
Conditions
Maple Syrup Urine Disease
Interventions
Phenylbutyrate, Placebo powder
Drug
Lead sponsor
Brendan Lee
Other
Eligibility
3 Years and older
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2017
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Mar 19, 2019 · Synced Nov 1, 2026, 5:19 AM EST
Terminated Phase 1 Interventional Results available

Androgen Reduction in Congenital Adrenal Hyperplasia, Phase 1

NCT02574910
View directory record Official record
Conditions
Congenital Adrenal Hyperplasia
Interventions
Abiraterone acetate
Drug
Lead sponsor
University of Texas Southwestern Medical Center
Other
Eligibility
2 Years to 9 Years
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2023
U.S. locations
4
States / cities
Los Angeles, California • Bethesda, Maryland • Ann Arbor, Michigan + 1 more
Source: ClinicalTrials.gov public record
Updated Nov 4, 2025 · Synced Nov 1, 2026, 5:19 AM EST