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 28, 2026, 9:58 PM EDT

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

Showing 673–696 of 1,511 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Inborn Errors of Metabolism Disorders Clear all
Completed Not applicable Interventional Results available

SToRytelling to Improve DiseasE Outcomes in GOut: The STRIDE-GO2 Study

NCT02741700
View directory record Official record
Conditions
Gout, Low Medication Adherence, Health Related Quality of Life
Interventions
Gout Storytelling Video Intervention
Behavioral
Lead sponsor
VA Office of Research and Development
Federal
Eligibility
19 Years and older
Enrollment
306 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2021
U.S. locations
3
States / cities
Birmingham, Alabama • St Louis, Missouri • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Mar 17, 2022 · Synced Oct 28, 2026, 9:58 PM EDT
Completed No phase listed Observational

A Study to Determine Eligibility for CAH-301 (A Study of Gene Therapy for Classic Congenital Adrenal Hyperplasia (CAH) [NCT04783181])

NCT05101902
View directory record Official record
Conditions
Congenital Adrenal Hyperplasia
Interventions
Pre-Screening
Other
Lead sponsor
Adrenas Therapeutics Inc
Industry
Eligibility
18 Years and older
Enrollment
65 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2024
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Sep 19, 2024 · Synced Oct 28, 2026, 9:58 PM EDT
Completed Phase 2Phase 3 Interventional

A Clinical Study to Evaluate the Efficacy and Safety of MIN-102 (IMP) in Male AMN Patients

NCT03231878
View directory record Official record
Conditions
Adrenoleukodystrophy
Interventions
Leriglitazone, Placebo
Drug
Lead sponsor
Minoryx Therapeutics, S.L.
Industry
Eligibility
18 Years to 65 Years · Male only
Enrollment
116 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2025
U.S. locations
3
States / cities
Stanford, California • Baltimore, Maryland • Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Oct 28, 2026 · Synced Oct 28, 2026, 9:58 PM EDT
Terminated Phase 3 Interventional Results available

Ecopipam Treatment of Self-Injurious Behavior in Subjects With Lesch-Nyhan Disease

NCT01751802
View directory record Official record
Conditions
Lesch-Nyhan Disease, Self-injurious Behavior
Interventions
Ecopipam, Placebo
Drug
Lead sponsor
Emalex Biosciences Inc.
Industry
Eligibility
6 Years to 70 Years
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2014
U.S. locations
1
States / cities
Atlanta, Georgia
Source: ClinicalTrials.gov public record
Updated Apr 22, 2024 · Synced Oct 28, 2026, 9:58 PM EDT
Completed Phase 1Phase 2 Interventional Results available

Safety and Efficacy Study of Asfotase Alfa in Severely Affected Infants With Hypophosphatasia (HPP)

NCT00744042
View directory record Official record
Conditions
Hypophosphatasia (HPP)
Interventions
asfotase alfa
Biological
Lead sponsor
Alexion Pharmaceuticals, Inc.
Industry
Eligibility
Up to 36 Months
Enrollment
11 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2010
U.S. locations
6
States / cities
Little Rock, Arkansas • Wilmington, Delaware • Springfield, Missouri + 3 more
Source: ClinicalTrials.gov public record
Updated Apr 1, 2019 · Synced Oct 28, 2026, 9:58 PM EDT
Completed No phase listed Observational

A Retrospective Natural History Study of Patients With Lysosomal Acid Lipase Deficiency/Wolman Phenotype

NCT01358370
View directory record Official record
Conditions
Lysosomal Acid Lipase Deficiency, Wolman Disease
Interventions
Not listed
Lead sponsor
Alexion Pharmaceuticals, Inc.
Industry
Eligibility
Not listed
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2013
U.S. locations
7
States / cities
Los Angeles, California • Palo Alto, California • Minneapolis, Minnesota + 2 more
Source: ClinicalTrials.gov public record
Updated Jun 27, 2016 · Synced Oct 28, 2026, 9:58 PM EDT
Completed Phase 2 Interventional

CD34+Selection for Partially Matched Family or Matched Unrelated Adult Donor Transplant

NCT01049854
View directory record Official record
Conditions
Leukemia, Lymphoma, Bone Marrow Failure, Immunodeficiencies, Histiocytosis, Sickle Cell Disease, Beta Thalassemia, Inborn Errors of Metabolism
Interventions
Full Intensity with TBI, Full Intensity, Reduced Intensity, Reduced Intensity (Fanconi)
Drug
Lead sponsor
New York Medical College
Other
Eligibility
Up to 70 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2018
U.S. locations
1
States / cities
Valhalla, New York
Source: ClinicalTrials.gov public record
Updated Aug 21, 2018 · Synced Oct 28, 2026, 9:58 PM EDT
Active, not recruiting Phase 1Phase 2 Interventional

A Phase 1/Phase 2 Open-label Study to Evaluate the Safety, Tolerability, and Efficacy of a Single Intravenous Administration of SAR444836 in Adult Participants With Phenylketonuria

NCT05972629
View directory record Official record
Conditions
Phenylketonuria
Interventions
SAR444836
Drug
Lead sponsor
Sanofi
Industry
Eligibility
18 Years to 65 Years
Enrollment
32 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2030
U.S. locations
7
States / cities
San Francisco, California • Aurora, Colorado • Gainesville, Florida + 4 more
Source: ClinicalTrials.gov public record
Updated Sep 23, 2025 · Synced Oct 28, 2026, 9:58 PM EDT
Terminated Phase 2 Interventional

Study of HST5040 in Subjects With Propionic or Methylmalonic Acidemia

NCT04732429
View directory record Official record
Conditions
Methylmalonic Acidemia, Propionic Acidemia
Interventions
HST5040, Placebo
Drug
Lead sponsor
HemoShear Therapeutics
Industry
Eligibility
2 Years and older
Enrollment
26 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2023
U.S. locations
14
States / cities
San Diego, California • New Haven, Connecticut • Washington D.C., District of Columbia + 11 more
Source: ClinicalTrials.gov public record
Updated Jan 5, 2024 · Synced Oct 28, 2026, 9:58 PM EDT
Completed No phase listed Observational Results available

Screening Study to Identify Pediatric Patients With Hunter Syndrome Who Demonstrate Evidence of Central Nervous System (CNS) Involvement and Who Are Currently Receiving Treatment With Elaprase®

NCT00937794
View directory record Official record
Conditions
Hunter Syndrome
Interventions
Neurobehavioral testing, Visual and auditory assessments
Behavioral · Other
Lead sponsor
Shire
Industry
Eligibility
32 Months to 18 Years · Male only
Enrollment
33 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2011
U.S. locations
1
States / cities
Chapel Hill, North Carolina
Source: ClinicalTrials.gov public record
Updated Jun 14, 2021 · Synced Oct 28, 2026, 9:58 PM EDT
Not listed Phase 1 Interventional Accepts healthy volunteers

Safety and Efficacy Study of Intravenous Uricase-PEG 20

NCT01021241
View directory record Official record
Conditions
Gout, Hyperuricemia
Interventions
Uricase-PEG 20
Biological
Lead sponsor
EnzymeRx
Industry
Eligibility
40 Years to 75 Years
Enrollment
20 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2009 – 2010
U.S. locations
2
States / cities
Miami, Florida • Duncansville, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Feb 23, 2010 · Synced Oct 28, 2026, 9:58 PM EDT
Completed Phase 3 Interventional

Preventing Cytomegalovirus (CMV) Organ Damage With Valganciclovir in People With HIV

NCT00006145
View directory record Official record
Conditions
Cytomegalovirus Infections, HIV Infections
Interventions
Valganciclovir
Drug
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
13 Years and older
Enrollment
350 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2000 – 2006
U.S. locations
56
States / cities
Birmingham, Alabama • Los Angeles, California • Palo Alto, California + 33 more
Source: ClinicalTrials.gov public record
Updated Nov 1, 2021 · Synced Oct 28, 2026, 9:58 PM EDT
Completed Phase 3 Interventional Results available

Long Term, Follow-on Study of Lomitapide in Patients With Homozygous Familial Hypercholesterolemia

NCT00943306
View directory record Official record
Conditions
Familial Hypercholesterolemia
Interventions
lomitapide
Drug
Lead sponsor
Aegerion Pharmaceuticals, Inc.
Industry
Eligibility
18 Years and older
Enrollment
19 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2014
U.S. locations
2
States / cities
Los Angeles, California • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jun 13, 2018 · Synced Oct 28, 2026, 9:58 PM EDT
Completed Phase 1Phase 2 Interventional Results available

Safety and Dose-Finding Study of DTX401 (AAV8G6PC) in Adults With Glycogen Storage Disease Type Ia (GSDIa)

NCT03517085
View directory record Official record
Conditions
GSD1
Interventions
DTX401, steroid regimen
Genetic · Drug
Lead sponsor
Ultragenyx Pharmaceutical Inc
Industry
Eligibility
18 Years and older
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2021
U.S. locations
3
States / cities
Farmington, Connecticut • Ann Arbor, Michigan • Houston, Texas
Source: ClinicalTrials.gov public record
Updated Nov 18, 2022 · Synced Oct 28, 2026, 9:58 PM EDT
Completed Phase 3 Interventional

REVEAL - Research With I-124 EVuzamitide to Elucidate Cardiac AmyLoidosis

NCT06788535
View directory record Official record
Conditions
Cardiac Amyloidosis
Interventions
I-124 evuzamitide
Drug
Lead sponsor
Sharmila Dorbala
Other
Eligibility
18 Years and older
Enrollment
204 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2026
U.S. locations
20
States / cities
Scottsdale, Arizona • Duarte, California • San Francisco, California + 16 more
Source: ClinicalTrials.gov public record
Updated Apr 30, 2026 · Synced Oct 28, 2026, 9:58 PM EDT
Completed Phase 1 Interventional

Study of the Pharmacokinetics of Trappsol and Effects on Potential Biomarkers of Niemann-Pick C1 (NPC1)

NCT02939547
View directory record Official record
Conditions
Niemann-Pick Disease, Type C1
Interventions
Hydroxypropyl-beta-cyclodextrin
Drug
Lead sponsor
Cyclo Therapeutics, Inc.
Industry
Eligibility
18 Years and older
Enrollment
13 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2020
U.S. locations
2
States / cities
Oakland, California • Morristown, New Jersey
Source: ClinicalTrials.gov public record
Updated Feb 21, 2021 · Synced Oct 28, 2026, 9:58 PM EDT
Completed Phase 1 Interventional

L-Citrulline Dose Finding Safety Study in MELAS

NCT03952234
View directory record Official record
Conditions
MELAS Syndrome
Interventions
L-Citrulline
Drug
Lead sponsor
Baylor College of Medicine
Other
Eligibility
18 Years to 65 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2024
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Jun 26, 2024 · Synced Oct 28, 2026, 9:58 PM EDT
Completed No phase listed Observational

Pompe Telemedicine Developmental Study

NCT02950298
View directory record Official record
Conditions
Pompe Disease, Glycogen Storage Disease II
Interventions
Not listed
Lead sponsor
Duke University
Other
Eligibility
6 Years to 18 Years
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2021
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Oct 28, 2021 · Synced Oct 28, 2026, 9:58 PM EDT
Completed No phase listed Observational

Diagnosis of Mucopolysaccharidosis Disorders in Patients Presenting With Bilateral Hip Disease

NCT01707433
View directory record Official record
Conditions
Mucopolysaccharidosis IV A, Mucopolysaccharidosis VI
Interventions
Enzyme testing
Other
Lead sponsor
Children's Hospitals and Clinics of Minnesota
Other
Eligibility
Up to 21 Years
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2015
U.S. locations
2
States / cities
Minneapolis, Minnesota • Saint Paul, Minnesota
Source: ClinicalTrials.gov public record
Updated Aug 4, 2015 · Synced Oct 28, 2026, 9:58 PM EDT
Completed Phase 1Phase 2 Interventional Results available

Treatment of the Cholesterol Defect in Smith-Lemli-Opitz Syndrome

NCT00272844
View directory record Official record
Conditions
Smith-Lemli-Opitz Syndrome
Interventions
crystalline cholesterol oil-based suspension
Drug
Lead sponsor
Boston Children's Hospital
Other
Eligibility
Not listed
Enrollment
23 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1998 – 2011
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Oct 20, 2017 · Synced Oct 28, 2026, 9:58 PM EDT
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 Oct 28, 2026, 9:58 PM EDT
Completed Phase 2 Interventional Results available

Cellcept for Treatment of Juvenile Neuronal Ceroid Lipofuscinosis

NCT01399047
View directory record Official record
Conditions
Juvenile Neuronal Ceroid Lipofuscinosis
Interventions
Mycophenolate mofetil, Liquid Placebo
Drug
Lead sponsor
University of Rochester
Other
Eligibility
6 Years to 25 Years
Enrollment
19 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2015
U.S. locations
1
States / cities
Rochester, New York
Source: ClinicalTrials.gov public record
Updated May 21, 2019 · Synced Oct 28, 2026, 9:58 PM EDT
Completed Phase 3 Interventional Results available

Effect of Kuvan on Neurocognitive Function, Blood Phenylalanine Level, Safety, and Pharmacokinetics in Children With PKU

NCT00838435
View directory record Official record
Conditions
Phenylketonuria
Interventions
sapropterin dihydrochloride
Drug
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
Up to 6 Years
Enrollment
95 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2018
U.S. locations
12
States / cities
La Jolla, California • Orange, California • Tampa, Florida + 9 more
Source: ClinicalTrials.gov public record
Updated Dec 22, 2020 · Synced Oct 28, 2026, 9:58 PM EDT
Terminated Phase 4 Interventional Results available

A Study to Evaluate the Efficacy and Safety of Alglucosidase Alfa Produced at the 4000 L Scale for Pompe Disease

NCT01526785
View directory record Official record
Conditions
Pompe Disease
Interventions
Alglucosidase alfa
Drug
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
1 Year and older
Enrollment
113 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2014
U.S. locations
51
States / cities
Birmingham, Alabama • Phoenix, Arizona • Little Rock, Arkansas + 48 more
Source: ClinicalTrials.gov public record
Updated Dec 8, 2015 · Synced Oct 28, 2026, 9:58 PM EDT