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 16, 2026, 3:23 AM EDT

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

Showing 625–648 of 1,605 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Inborn Errors of Metabolism Clear all
Withdrawn No phase listed Observational

International Registry for Primary Hyperoxaluria

NCT00875823
View directory record Official record
Conditions
Primary Hyperoxaluria, Nephrocalcinosis, Kidney Stones
Interventions
Not listed
Lead sponsor
Mayo Clinic
Other
Eligibility
Not listed
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003 – 2009
U.S. locations
4
States / cities
Los Angeles, California • Sacramento, California • Chicago, Illinois + 1 more
Source: ClinicalTrials.gov public record
Updated Apr 7, 2015 · Synced Oct 16, 2026, 3:23 AM EDT
Withdrawn No phase listed Observational

Evaluation of Phenotypic Variability in Fabry Disease

NCT03145779
View directory record Official record
Conditions
Fabry Disease
Interventions
Not listed
Lead sponsor
Boston Children's Hospital
Other
Eligibility
1 Year and older
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2030
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Dec 3, 2020 · Synced Oct 16, 2026, 3:23 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Exploration of Immunity in Gaucher Disease

NCT01358188
View directory record Official record
Conditions
Gaucher Disease
Interventions
Not listed
Lead sponsor
O & O Alpan LLC
Other
Eligibility
18 Years and older
Enrollment
33 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2011 – 2016
U.S. locations
1
States / cities
Fairfax, Virginia
Source: ClinicalTrials.gov public record
Updated Apr 4, 2017 · Synced Oct 16, 2026, 3:23 AM EDT
Completed Phase 3 Interventional Results available

Phase 3 Extension Study of the Safety and Efficacy of Aldurazyme® (Laronidase) in Mucopolysaccharidosis I (MPS I) Patients

NCT00146770
View directory record Official record
Conditions
Mucopolysaccharidosis I, Hurler's Syndrome, Hurler-Scheie Syndrome, Scheie Syndrome
Interventions
Aldurazyme, placebo
Biological
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
Not listed
Enrollment
45 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2005
U.S. locations
8
States / cities
Mobile, Alabama • Atlanta, Georgia • Rochester, New York + 5 more
Source: ClinicalTrials.gov public record
Updated Apr 3, 2015 · Synced Oct 16, 2026, 3:23 AM EDT
Recruiting No phase listed Observational

Longitudinal Prospective Natural History Study of Retinopathy in Zellweger Spectrum Disorder

NCT06190626
View directory record Official record
Conditions
Zellweger Spectrum Disorder
Interventions
Not listed
Lead sponsor
McGill University Health Centre/Research Institute of the McGill University Health Centre
Other
Eligibility
Not listed
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2029
U.S. locations
1
States / cities
Rochester, New York
Source: ClinicalTrials.gov public record
Updated Jan 21, 2026 · Synced Oct 16, 2026, 3:23 AM EDT
Completed Not applicable Interventional Accepts healthy volunteers

Does Daily Supplementation of Lactobacillus Acidophilus MPH734, for One Week, Affect Acute (Immediate), Subacute (7 Days), and Post-treatment Discontinuation Lactose Metabolism, Gastrointestinal Symptoms, and Clinical Markers of Inflammation and Safety Compared to a Placebo

NCT04531033
View directory record Official record
Conditions
Lactose Intolerance
Interventions
Lactobacillus acidophilus MPH734, Placebo
Dietary Supplement
Lead sponsor
Lemuel W. Taylor IV
Other
Eligibility
18 Years to 55 Years
Enrollment
50 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2020 – 2022
U.S. locations
1
States / cities
Belton, Texas
Source: ClinicalTrials.gov public record
Updated May 16, 2024 · Synced Oct 16, 2026, 3:23 AM EDT
Recruiting Phase 1Phase 2 Interventional

A Study to Evaluate Safety, Tolerability, and Efficacy of AB-1009 Gene Therapy (GAA Gene) in Adult Participants With Late-Onset Pompe Disease (PROGRESS-GT LOPD)

NCT07282847
View directory record Official record
Conditions
Pompe Disease (Late-onset), Pompe Disease Late-Onset, LOPD
Interventions
AB-1009 (GAA Gene)
Genetic
Lead sponsor
AskBio Inc
Industry
Eligibility
18 Years to 65 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2032
U.S. locations
9
States / cities
Phoenix, Arizona • Irvine, California • Palo Alto, California + 6 more
Source: ClinicalTrials.gov public record
Updated Oct 13, 2026 · Synced Oct 16, 2026, 3:23 AM EDT
Recruiting Phase 2 Interventional

Study of S-606001 as an Add-on to Enzyme Replacement Therapy (ERT) in Participants With Late-onset Pompe Disease (LOPD)

NCT07123155
View directory record Official record
Conditions
Pompe Disease
Interventions
S-606001, Placebo
Drug
Lead sponsor
Shionogi
Industry
Eligibility
18 Years and older
Enrollment
45 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
9
States / cities
Irvine, California • Gainesville, Florida • Atlanta, Georgia + 6 more
Source: ClinicalTrials.gov public record
Updated Jun 23, 2026 · Synced Oct 16, 2026, 3:23 AM EDT
Active, not recruiting Phase 1Phase 2 Interventional

A Clinical Trial of PR001 (LY3884961) in Patients With Peripheral Manifestations of Gaucher Disease (PROCEED)

NCT05487599
View directory record Official record
Conditions
Gaucher Disease, Gaucher Disease, Type 1
Interventions
LY3884961
Genetic
Lead sponsor
Prevail Therapeutics
Industry
Eligibility
18 Years and older
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2031
U.S. locations
2
States / cities
Durham, North Carolina • Fairfax, Virginia
Source: ClinicalTrials.gov public record
Updated Aug 17, 2026 · Synced Oct 16, 2026, 3:23 AM EDT
Recruiting Phase 1Phase 2 Interventional

Phase 1/2: CD45RA Depleted Stem Cell Addback to Prevent Viral or Fungal Infections Post TCRab/CD19 Depleted HSCT

NCT06839456
View directory record Official record
Conditions
Leukemia, High Risk Acute Lymphoblastic Leukemia, High Risk Acute Myeloid Leukemia, Relapse Leukemia, MDS (Myelodysplastic Syndrome), Relapsed Non-Hodgkin Lymphoma, Acquired Aplastic Anemia, Inherited BMF Syndrome, Immunodeficiency, Primary Immune Regulatory Disorder, Hemoglobinopathies, Bone Marrow Failure, Inborn Errors of Metabolism, HLH
Interventions
Phase 1 Dose Level 1, Phase 1 Dose Level 2, Phase 1 Dose Level 3, Phase 2 Maximum Tolerated Dose determined in Phase 1, Phase 2 Established Dose from prior study, NCT03810196
Device
Lead sponsor
Children's Hospital of Philadelphia
Other
Eligibility
1 Month to 25 Years
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2032
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Apr 15, 2026 · Synced Oct 16, 2026, 3:23 AM EDT
Completed Phase 1Phase 2 Interventional Results available

An Open-label Safety, Pharmacokinetic, and Efficacy Study of Miglustat for the Treatment of Subjects With Batten Ceroid Lipofuscinosis, Neuronal 3 (CLN3) Disease

NCT05174039
View directory record Official record
Conditions
Batten Disease
Interventions
Miglustat 100 milligrams (mg) Oral Capsule
Drug
Lead sponsor
Beyond Batten Disease Foundation
Other
Eligibility
17 Years and older
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2024
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Sep 9, 2025 · Synced Oct 16, 2026, 3:23 AM EDT
Recruiting Phase 2Phase 3 Interventional

Study of Cannabidiol in Sanfilippo Syndrome

NCT06333041
View directory record Official record
Conditions
Sanfilippo Syndrome, Mucopolysaccharidosis III
Interventions
Epidiolex, Placebo
Drug
Lead sponsor
Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center
Other
Eligibility
4 Years and older
Enrollment
35 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2028
U.S. locations
1
States / cities
Torrance, California
Source: ClinicalTrials.gov public record
Updated Sep 15, 2026 · Synced Oct 16, 2026, 3:23 AM EDT
Completed Phase 3 Interventional Results available

A Study of Lumasiran in Infants and Young Children With Primary Hyperoxaluria Type 1

NCT03905694
View directory record Official record
Conditions
Primary Hyperoxaluria, Primary Hyperoxaluria Type 1 (PH1)
Interventions
Lumasiran
Drug
Lead sponsor
Alnylam Pharmaceuticals
Industry
Eligibility
0 Years to 5 Years
Enrollment
18 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2024
U.S. locations
2
States / cities
Rochester, Minnesota • Houston, Texas
Source: ClinicalTrials.gov public record
Updated Feb 14, 2025 · Synced Oct 16, 2026, 3:23 AM EDT
Completed Phase 3 Interventional Results available

An Open-Label Phase 3 Study of BMN 165 for Adults With PKU Not Previously Treated w/ BMN 165

NCT01819727
View directory record Official record
Conditions
Phenylketonuria
Interventions
BMN 165
Drug
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
18 Years to 70 Years
Enrollment
261 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2015
U.S. locations
31
States / cities
La Jolla, California • Oakland, California • Aurora, Colorado + 28 more
Source: ClinicalTrials.gov public record
Updated Feb 26, 2019 · Synced Oct 16, 2026, 3:23 AM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

A Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of Oral ABP-745 Administration in Healthy Volunteers

NCT06258213
View directory record Official record
Conditions
Gout, Acute Gout Flare, Gout Flares
Interventions
ABP-745, Placebo
Drug
Lead sponsor
Atom Therapeutics Co., Ltd
Industry
Eligibility
18 Years to 55 Years
Enrollment
90 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2024
U.S. locations
1
States / cities
San Antonio, Texas
Source: ClinicalTrials.gov public record
Updated Aug 17, 2025 · Synced Oct 16, 2026, 3:23 AM EDT
Terminated No phase listed Observational

Oxidative Stress, Inflammation and Acute Decompensation in Urea Cycle Disorders

NCT01541722
View directory record Official record
Conditions
Urea Cycle Disorders
Interventions
Not listed
Lead sponsor
Mark Batshaw
Other
Eligibility
Not listed
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2015
U.S. locations
10
States / cities
Los Angeles, California • Aurora, Colorado • Washington D.C., District of Columbia + 7 more
Source: ClinicalTrials.gov public record
Updated Oct 2, 2015 · Synced Oct 16, 2026, 3:23 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 16, 2026, 3:23 AM EDT
Recruiting Phase 2Phase 3 Interventional

Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH

NCT02716246
View directory record Official record
Conditions
MPS IIIA, Sanfilippo Syndrome, Sanfilippo A, Mucopolysaccharidosis III
Interventions
UX111, Prophylactic Immunomodulatory (IM) Therapy, Optimized Prophylactic IM Therapy, Adjuvant IM Therapy
Biological · Drug
Lead sponsor
Ultragenyx Pharmaceutical Inc
Industry
Eligibility
Not listed
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2029
U.S. locations
2
States / cities
Columbus, Ohio • Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Sep 22, 2026 · Synced Oct 16, 2026, 3:23 AM EDT
Completed Phase 1 Interventional Results available

RDEA3170 and Allopurinol Combination Study in Gout Subjects

NCT02279641
View directory record Official record
Conditions
Gout
Interventions
RDEA3170 10 mg, allopurinol 300 mg
Drug
Lead sponsor
Ardea Biosciences, Inc.
Industry
Eligibility
18 Years to 75 Years · Male only
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2015
U.S. locations
1
States / cities
Overland Park, Kansas
Source: ClinicalTrials.gov public record
Updated Dec 20, 2017 · Synced Oct 16, 2026, 3:23 AM EDT
Completed Phase 2 Interventional Results available

A Long-Term Extension Study of AT2101 (Afegostat Tartrate) in Type 1 Gaucher Patients

NCT00813865
View directory record Official record
Conditions
Gaucher Disease, Type 1 Gaucher Disease, Gaucher Disease, Type 1
Interventions
afegostat tartrate
Drug
Lead sponsor
Amicus Therapeutics
Industry
Eligibility
18 Years and older
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2012
U.S. locations
3
States / cities
Coral Springs, Florida • Decatur, Georgia • Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Aug 15, 2018 · Synced Oct 16, 2026, 3:23 AM EDT
Terminated Phase 3 Interventional Results available

An Extension Study to Evaluate the Long-term Efficacy and Safety of Oxabact in Patients With Primary Hyperoxaluria

NCT03938272
View directory record Official record
Conditions
Hyperoxaluria, Primary
Interventions
Oxabact OC5 - Oxalobacter formigenes Strain HC-1
Biological
Lead sponsor
OxThera
Industry
Eligibility
3 Years and older
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2021
U.S. locations
1
States / cities
Nashville, Tennessee
Source: ClinicalTrials.gov public record
Updated Dec 8, 2021 · Synced Oct 16, 2026, 3:23 AM EDT
Completed Not applicable Interventional

Studies of the Ocular Complications of AIDS (SOCA) CMV Retinitis Trial: Foscarnet-Ganciclovir Component

NCT00000665
View directory record Official record
Conditions
Cytomegalovirus Retinitis, HIV Infections
Interventions
Foscarnet sodium, Ganciclovir
Drug
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
13 Years and older
Enrollment
240 participants
Healthy volunteers
Healthy volunteers not accepted
U.S. locations
9
States / cities
La Jolla, California • Los Angeles, California • San Francisco, California + 4 more
Source: ClinicalTrials.gov public record
Updated Mar 14, 2011 · Synced Oct 16, 2026, 3:23 AM EDT
Terminated Phase 2 Interventional Results available

Study of ARO-ANG3 in Participants With Homozygous Familial Hypercholesterolemia (HOFH)

NCT05217667
View directory record Official record
Conditions
Homozygous Familial Hypercholesterolemia
Interventions
ARO-ANG 3 Injection
Drug
Lead sponsor
Arrowhead Pharmaceuticals
Industry
Eligibility
16 Years and older
Enrollment
18 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2025
U.S. locations
2
States / cities
Mount Sinai, New York • Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Jun 12, 2026 · Synced Oct 16, 2026, 3:23 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 16, 2026, 3:23 AM EDT