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 Oct 14, 2026, 8:41 PM EDT

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

Showing 481–501 of 501 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Lipid Metabolism, Inborn Errors Clear all
Recruiting Phase 3 Interventional

A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease, GM1 Gangliosidosis or GM2 Gangliosidosis

NCT07054515
View directory record Official record
Conditions
Niemann-Pick Type C Disease, GM1 Gangliosidosis, GM2 Gangliosidosis
Interventions
AZ-3102, Placebo
Drug
Lead sponsor
Azafaros B.V.
Industry
Eligibility
4 Years and older
Enrollment
147 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2028
U.S. locations
5
States / cities
Oakland, California • Minneapolis, Minnesota • Rochester, Minnesota + 2 more
Source: ClinicalTrials.gov public record
Updated Sep 24, 2026 · Synced Oct 14, 2026, 8:41 PM EDT
Active, not recruiting Phase 2 Interventional

Venglustat in Combination With Cerezyme in Adult Patients With Gaucher Disease Type 3 With Venglustat Monotherapy Extension

NCT02843035
View directory record Official record
Conditions
Gaucher Disease Type 1, Gaucher Disease Type 3
Interventions
venglustat (GZ402671), imiglucerase
Drug
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
18 Years and older
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2026
U.S. locations
3
States / cities
New Haven, Connecticut • Dallas, Texas • Fairfax, Virginia
Source: ClinicalTrials.gov public record
Updated Jul 10, 2026 · Synced Oct 14, 2026, 8:41 PM EDT
Terminated No phase listed Observational

Cardiovasculorenal Phenotyping in Fabry Disease Through Noninvasive Testing

NCT05699265
View directory record Official record
Conditions
Fabry Disease
Interventions
Measures of arterial stiffness and endothelial function, Ambulatory blood pressure monitoring, Cardiopulmonary exercise testing (CPET), Serum and urine biomarkers
Diagnostic Test
Lead sponsor
Children's Hospital Medical Center, Cincinnati
Other
Eligibility
8 Years and older
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2025
U.S. locations
1
States / cities
Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Jun 19, 2025 · Synced Oct 14, 2026, 8:41 PM EDT
Not listed Phase 2 Interventional

Phase II Study of Cholesterol- and Cholestanol-Free Diet, Lovastatin, and Chenodeoxycholic Acid for Cerebrotendinous Xanthomatosis

NCT00004346
View directory record Official record
Conditions
Cerebrotendinous Xanthomatosis
Interventions
chenodeoxycholic acid, lovastatin
Drug
Lead sponsor
National Center for Research Resources (NCRR)
NIH
Eligibility
0 Years and older
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1996
U.S. locations
1
States / cities
Portland, Oregon
Source: ClinicalTrials.gov public record
Updated Jun 24, 2005 · Synced Oct 14, 2026, 8:41 PM EDT
Terminated Phase 2 Interventional Accepts healthy volunteers Results available

Analyzing Childhood Recall Antigens in Patients With Pancreatic Cancer

NCT03848182
View directory record Official record
Conditions
Pancreatic Cancer
Interventions
Gemcitabine, TT vaccine booster
Drug · Biological
Lead sponsor
Albert Einstein College of Medicine
Other
Eligibility
18 Years to 100 Years
Enrollment
10 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2017 – 2019
U.S. locations
1
States / cities
The Bronx, New York
Source: ClinicalTrials.gov public record
Updated Apr 18, 2024 · Synced Oct 14, 2026, 8:41 PM EDT
Terminated Phase 2 Interventional Results available

Open-label Long-term Safety Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Disease Who Have Completed a Previous AT1001 Study

NCT00526071
View directory record Official record
Conditions
Fabry Disease
Interventions
migalastat HCl
Drug
Lead sponsor
Amicus Therapeutics
Industry
Eligibility
18 Years and older
Enrollment
23 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2012
U.S. locations
3
States / cities
Decatur, Georgia • New York, New York • Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Oct 3, 2018 · Synced Oct 14, 2026, 8:41 PM 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 14, 2026, 8:41 PM EDT
Completed Phase 4 Interventional Results available

A Study Evaluating Glycosphingolipid Clearance in Patients Treated With Agalsidase Alfa Who Switch to Agalsidase Beta

NCT01650779
View directory record Official record
Conditions
Fabry Disease
Interventions
Agalsidase beta
Biological
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
Male only
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2013
U.S. locations
6
States / cities
Coral Springs, Florida • Decatur, Georgia • Baltimore, Maryland + 3 more
Source: ClinicalTrials.gov public record
Updated Jul 10, 2014 · Synced Oct 14, 2026, 8:41 PM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

Safety and Pharmacokinetics of AT1001 (Migalastat HCl) in Healthy Subjects and Subjects With Impaired Renal Function

NCT01730469
View directory record Official record
Conditions
Fabry Disease
Interventions
AT1001 150 mg
Drug
Lead sponsor
Amicus Therapeutics
Industry
Eligibility
18 Years to 75 Years
Enrollment
32 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2011 – 2012
U.S. locations
4
States / cities
Costa Mesa, California • Miami, Florida • Orlando, Florida
Source: ClinicalTrials.gov public record
Updated Aug 3, 2017 · Synced Oct 14, 2026, 8:41 PM EDT
Completed Phase 3 Interventional Results available

An Study to Evaluate Rosuvastatin in Children and Adolescents With Familial Hypercholesterolaemia

NCT01078675
View directory record Official record
Conditions
Familial Hypercholesterolaemia
Interventions
rosuvastatin calcium
Drug
Lead sponsor
AstraZeneca
Industry
Eligibility
6 Years to 17 Years
Enrollment
315 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2013
U.S. locations
1
States / cities
Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Apr 7, 2015 · Synced Oct 14, 2026, 8:41 PM EDT
Withdrawn Not applicable Interventional

Cholestanol in Humans

NCT00018694
View directory record Official record
Conditions
Cerebrotendinous Xanthomatosis
Interventions
Chenodeoxycholic Acid
Drug
Lead sponsor
US Department of Veterans Affairs
Federal
Eligibility
5 Years to 80 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
1999 – 2009
U.S. locations
1
States / cities
East Orange, New Jersey
Source: ClinicalTrials.gov public record
Updated Oct 14, 2013 · Synced Oct 14, 2026, 8:41 PM EDT
Active, not recruiting Phase 1Phase 2 Interventional

Study of Safety, Tolerability and Efficacy of PBGM01 in Pediatric Participants With GM1 Gangliosidosis

NCT04713475
View directory record Official record
Conditions
GM1 Gangliosidosis, GM1 Gangliosidosis, Type I, GM1 Gangliosidosis, Type 2, Beta-Galactosidase-1 (GLB1) Deficiency
Interventions
PBGM01
Biological
Lead sponsor
Gemma Biotherapeutics
Industry
Eligibility
1 Month to 24 Months
Enrollment
26 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2029
U.S. locations
4
States / cities
Oakland, California • Minneapolis, Minnesota • New Brunswick, New Jersey + 1 more
Source: ClinicalTrials.gov public record
Updated Jun 30, 2026 · Synced Oct 14, 2026, 8:41 PM EDT
Active, not recruiting Phase 1Phase 2 Interventional

Intravitreal ERT to Prevent Retinal Disease Progression in Children With CLN2

NCT05152914
View directory record Official record
Conditions
Neuronal Ceroid Lipofuscinosis Type 2
Interventions
Cerliponase Alfa
Drug
Lead sponsor
David L Rogers, MD
Other
Eligibility
24 Months to 72 Months
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2027
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Apr 30, 2026 · Synced Oct 14, 2026, 8:41 PM EDT
Completed No phase listed Observational

Immune Response in Subjects With Fabry Disease Who Are Switching From Agalsidase Alfa to Agalsidase Beta

NCT01745185
View directory record Official record
Conditions
Fabry Disease
Interventions
Not listed
Lead sponsor
O & O Alpan LLC
Other
Eligibility
7 Years and older
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2016
U.S. locations
1
States / cities
Fairfax, Virginia
Source: ClinicalTrials.gov public record
Updated Apr 4, 2017 · Synced Oct 14, 2026, 8:41 PM EDT
Completed Not applicable Interventional

Effect of Warfarin in the Treatment of Metachromatic Leukodystrophy

NCT00683189
View directory record Official record
Conditions
Metachromatic Leukodystrophy
Interventions
Warfarin
Drug
Lead sponsor
The Cooper Health System
Other
Eligibility
1 Year to 10 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2008
U.S. locations
1
States / cities
Camden, New Jersey
Source: ClinicalTrials.gov public record
Updated Mar 21, 2011 · Synced Oct 14, 2026, 8:41 PM EDT
Completed No phase listed Observational

Evaluation of Lipoproteins

NCT00001168
View directory record Official record
Conditions
Evaluation of Plasma Lipoproteins
Interventions
Balanced diet
Behavioral
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
2 Years to 99 Years
Enrollment
1,017 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1979
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Aug 18, 2026 · Synced Oct 14, 2026, 8:41 PM EDT
Completed No phase listed Observational

Antiproteinuric Agents and Fabry Disease

NCT00343577
View directory record Official record
Conditions
Fabry Disease, Proteinuria
Interventions
Not listed
Lead sponsor
University of Alabama at Birmingham
Other
Eligibility
14 Years to 95 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2006
U.S. locations
1
States / cities
Birmingham, Alabama
Source: ClinicalTrials.gov public record
Updated Nov 19, 2013 · Synced Oct 14, 2026, 8:41 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

IN-TANDEM Familial Hypercholesterolemia Pilot Study

NCT03253432
View directory record Official record
Conditions
Familial Hypercholesterolemia
Interventions
Genetic testing
Other
Lead sponsor
University of Pennsylvania
Other
Eligibility
18 Years and older
Enrollment
378 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2017 – 2018
U.S. locations
2
States / cities
Lancaster, Pennsylvania • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Feb 28, 2019 · Synced Oct 14, 2026, 8:41 PM EDT
Completed Not applicable Interventional

The Use of Mobile Health Technology and Behavioral Economics to Encourage Adherence in Adolescents

NCT04458766
View directory record Official record
Conditions
Familial Hypercholesterolemia, Adherence, Medication
Interventions
Monetary incentives
Behavioral
Lead sponsor
Boston Children's Hospital
Other
Eligibility
12 Years to 21 Years
Enrollment
11 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2024
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Aug 21, 2024 · Synced Oct 14, 2026, 8:41 PM EDT