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ClinicalTrials.gov public records Last synced Oct 14, 2026, 2:10 PM EDT

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

Showing 337–360 of 10,529 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Congenital Diseases Clear all
Completed Phase 4 Interventional

Replagal Enzyme Replacement Therapy for Adults With Fabry Disease

NCT00097890
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
Male only
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2005
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 4, 2008 · Synced Oct 14, 2026, 2:10 PM EDT
Terminated Phase 3 Interventional Results available

Mental Stress Reduction in Defibrillator Patients

NCT00624520
View directory record Official record
Conditions
Cardiomyopathy, Dilated, Arrhythmia, Anger, Stress
Interventions
Cognitive Behavioral Stress Management (CBSM), Patient Education
Behavioral · Other
Lead sponsor
US Department of Veterans Affairs
Federal
Eligibility
21 Years to 85 Years
Enrollment
129 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2014
U.S. locations
2
States / cities
Madison, Wisconsin • Milwaukee, Wisconsin
Source: ClinicalTrials.gov public record
Updated Dec 22, 2014 · Synced Oct 14, 2026, 2:10 PM EDT
Completed Phase 2 Interventional Results available

Efficacy and Safety Study of Apremilast in Subjects With Moderate to Severe Atopic Dermatitis

NCT02087943
View directory record Official record
Conditions
Dermatitis, Atopic Dermatitis
Interventions
Apremilast, Placebo
Drug
Lead sponsor
Amgen
Industry
Eligibility
18 Years and older
Enrollment
191 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2016
U.S. locations
15
States / cities
Phoenix, Arizona • Bakersfield, California • Los Angeles, California + 10 more
Source: ClinicalTrials.gov public record
Updated May 7, 2020 · Synced Oct 14, 2026, 2:10 PM EDT
Completed No phase listed Observational

Next Generation to Identify Genetic Causes of Disease in Patients Participating in NICHD Clinical Protocols

NCT01375543
View directory record Official record
Conditions
Genetic Predisposition
Interventions
Not listed
Lead sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
NIH
Eligibility
Not listed
Enrollment
128 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2019
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jan 6, 2020 · Synced Oct 14, 2026, 2:10 PM EDT
Completed Phase 2 Interventional

Gemtuzumab Ozogamicin and Cyclosporine in Treating Older Patients With Relapsed Acute Myeloid Leukemia

NCT00089050
View directory record Official record
Conditions
Leukemia
Interventions
cyclosporine, gemtuzumab ozogamicin
Drug
Lead sponsor
Fred Hutchinson Cancer Center
Other
Eligibility
60 Years and older
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2006
U.S. locations
1
States / cities
Seattle, Washington
Source: ClinicalTrials.gov public record
Updated Nov 30, 2011 · Synced Oct 14, 2026, 2:10 PM EDT
Terminated No phase listed Observational Accepts healthy volunteers

Global and Regional Myocardial Strain and Power Output In Patients With Single Ventricles Using Novel MRI Techniques

NCT01107990
View directory record Official record
Conditions
Fontan Procedure, Hypoplastic Left Heart Syndrome, Tricuspid Atresia, Double Inlet Left Ventricle
Interventions
Not listed
Lead sponsor
University of California, San Francisco
Other
Eligibility
10 Years and older
Enrollment
13 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2009 – 2012
U.S. locations
1
States / cities
San Francisco, California
Source: ClinicalTrials.gov public record
Updated Jan 21, 2020 · Synced Oct 14, 2026, 2:10 PM EDT
Terminated Phase 3 Interventional Results available

CoQ10 and Prednisone in Non-Ambulatory DMD

NCT00308113
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Prednisone, Coenzyme Q10
Drug · Dietary Supplement
Lead sponsor
Cooperative International Neuromuscular Research Group
Network
Eligibility
10 Years to 18 Years · Male only
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2010
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Nov 8, 2013 · Synced Oct 14, 2026, 2:10 PM EDT
Completed Phase 2 Interventional Results available

Clonidine Versus Phenobarbital as Adjunctive Therapy for Neonatal Abstinence Syndrome

NCT03670160
View directory record Official record
Conditions
Neonatal Abstinence Syndrome
Interventions
Phenobarbital, Clonidine
Drug
Lead sponsor
University of Tennessee Medical Center
Other
Eligibility
35 Weeks and older
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2020
U.S. locations
1
States / cities
Knoxville, Tennessee
Source: ClinicalTrials.gov public record
Updated Nov 10, 2021 · Synced Oct 14, 2026, 2:10 PM EDT
Active, not recruiting Phase 3 Interventional

A Study Evaluating Gene Therapy With BB305 Lentiviral Vector in Sickle Cell Disease

NCT04293185
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
bb1111
Genetic
Lead sponsor
Genetix Biotherapeutics Inc.
Industry
Eligibility
2 Years to 50 Years
Enrollment
35 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2027
U.S. locations
9
States / cities
Birmingham, Alabama • Washington D.C., District of Columbia • Boston, Massachusetts + 6 more
Source: ClinicalTrials.gov public record
Updated Dec 10, 2024 · Synced Oct 14, 2026, 2:10 PM EDT
Completed Phase 2 Interventional

Study of ISIS 301012 (Mipomersen) in Heterozygous Familial Hypercholesterolemia Subjects on Lipid Lowering Therapy

NCT00281008
View directory record Official record
Conditions
Hypercholesterolemia, Familial
Interventions
ISIS 301012 or Placebo
Drug
Lead sponsor
Kastle Therapeutics, LLC
Industry
Eligibility
18 Years and older
Enrollment
44 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2007
U.S. locations
7
States / cities
Chicago, Illinois • Auburn, Maine • Scarborough, Maine + 4 more
Source: ClinicalTrials.gov public record
Updated Aug 3, 2016 · Synced Oct 14, 2026, 2:10 PM EDT
Terminated No phase listed Observational Accepts healthy volunteers

Brain Tissue Collection for Neuropathological Studies

NCT00001260
View directory record Official record
Conditions
Bipolar Disorder, Depression, Anxiety Disorders, Schizophrenia, Tourette's Syndrome, Brain Diseases
Interventions
Not listed
Lead sponsor
National Institute of Mental Health (NIMH)
NIH
Eligibility
Not listed
Enrollment
2,161 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
1990 – 2017
U.S. locations
3
States / cities
Washington D.C., District of Columbia • Bethesda, Maryland • Fairfax, Virginia
Source: ClinicalTrials.gov public record
Updated Dec 9, 2019 · Synced Oct 14, 2026, 2:10 PM EDT
Completed No phase listed Observational

Clinical Outcomes of Triple Combination Therapy in Severe Cystic Fibrosis Disease.

NCT04038710
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
Triple combination therapy
Drug
Lead sponsor
National Jewish Health
Other
Eligibility
12 Years and older
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2021
U.S. locations
1
States / cities
Denver, Colorado
Source: ClinicalTrials.gov public record
Updated Mar 11, 2021 · Synced Oct 14, 2026, 2:10 PM EDT
Recruiting Phase 3 Interventional

CHAMPION: A Study to Evaluate the Efficacy and Safety of Obudanersen (ION582) in Children and Adults With Angelman Syndrome (AS)

NCT07782827
View directory record Official record
Conditions
Angelman Syndrome
Interventions
obudanersen
Drug
Lead sponsor
Ionis Pharmaceuticals, Inc.
Industry
Eligibility
2 Years to 50 Years
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2031
U.S. locations
1
States / cities
Carrboro, North Carolina
Source: ClinicalTrials.gov public record
Updated Sep 28, 2026 · Synced Oct 14, 2026, 2:10 PM EDT
Completed Early Phase 1 Interventional Accepts healthy volunteers

IL-4 and Insulin Resistance for Treatment of Patients With Atopic Dermatitis

NCT05372003
View directory record Official record
Conditions
Atopic Dermatitis
Interventions
Dupilumab, Photo therapy
Drug · Other
Lead sponsor
Mayo Clinic
Other
Eligibility
18 Years to 60 Years
Enrollment
7 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2022 – 2024
U.S. locations
1
States / cities
Scottsdale, Arizona
Source: ClinicalTrials.gov public record
Updated Feb 26, 2024 · Synced Oct 14, 2026, 2:10 PM EDT
Completed No phase listed Observational

Pregnancy in Osteogenesis Imperfecta (OI) Registry

NCT03072303
View directory record Official record
Conditions
Osteogenesis Imperfecta, Brittle Bone Disorders
Interventions
Web-based Survey
Other
Lead sponsor
University of South Florida
Other
Eligibility
Female only
Enrollment
170 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2019
U.S. locations
1
States / cities
Tampa, Florida
Source: ClinicalTrials.gov public record
Updated Oct 16, 2019 · Synced Oct 14, 2026, 2:10 PM EDT
Completed No phase listed Observational

Natural History Study of CEP290-Related Retinal Degeneration

NCT03396042
View directory record Official record
Conditions
Blindness, Leber Congenital Amaurosis 10, Vision Disorders, Eye Diseases, Eye Diseases, Hereditary, Eye Disorders Congenital, Retinal Disease, Retinal Degeneration
Interventions
Not listed
Lead sponsor
Editas Medicine, Inc.
Industry
Eligibility
3 Years to 99 Years
Enrollment
26 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2022
U.S. locations
4
States / cities
Miami, Florida • Boston, Massachusetts • Ann Arbor, Michigan + 1 more
Source: ClinicalTrials.gov public record
Updated May 19, 2022 · Synced Oct 14, 2026, 2:10 PM EDT
Terminated No phase listed Observational

Intra-Atrial vs. Extra-Cardiac Technique During Fontan Operation

NCT00266942
View directory record Official record
Conditions
Congenital Disorders
Interventions
Not listed
Lead sponsor
Children's Healthcare of Atlanta
Other
Eligibility
Up to 18 Years
Enrollment
400 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1990 – 2007
U.S. locations
1
States / cities
Atlanta, Georgia
Source: ClinicalTrials.gov public record
Updated May 23, 2007 · Synced Oct 14, 2026, 2:10 PM EDT
Completed Not applicable Interventional

Study of Tauroursodeoxycholic Acid for Hepatobiliary Disease in Cystic Fibrosis

NCT00004441
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
tauroursodeoxycholic acid, ursodiol
Drug
Lead sponsor
Children's Hospital Medical Center, Cincinnati
Other
Eligibility
Not listed
Enrollment
39 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1997 – 2001
U.S. locations
2
States / cities
Denver, Colorado • Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Mar 25, 2015 · Synced Oct 14, 2026, 2:10 PM EDT
Completed Not applicable Interventional Results available

Apollo™ Onyx™ Delivery Microcatheter Post Market Safety Study

NCT02378883
View directory record Official record
Conditions
Arteriovenous Malformations
Interventions
Apollo™ Onyx™ Delivery Micro Catheter
Device
Lead sponsor
Medtronic Neurovascular Clinical Affairs
Industry
Eligibility
Not listed
Enrollment
112 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2018
U.S. locations
2
States / cities
Englewood, Colorado • Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Apr 29, 2019 · Synced Oct 14, 2026, 2:10 PM EDT
Completed Phase 2 Interventional Results available

A Study of INCB050465 in Participants With Autoimmune Hemolytic Anemia

NCT03538041
View directory record Official record
Conditions
Autoimmune Hemolytic Anemia
Interventions
Parsaclisib
Drug
Lead sponsor
Incyte Corporation
Industry
Eligibility
18 Years and older
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2024
U.S. locations
6
States / cities
Washington D.C., District of Columbia • Minneapolis, Minnesota • St Louis, Missouri + 3 more
Source: ClinicalTrials.gov public record
Updated Jul 11, 2025 · Synced Oct 14, 2026, 2:10 PM EDT
Completed Phase 3 Interventional Results available

A Study to Evaluate the Efficacy and Safety of Oxabact in Patients With Primary Hyperoxaluria

NCT03116685
View directory record Official record
Conditions
Primary Hyperoxaluria
Interventions
Oxabact OC5 - Oxalobacter formigenes HC-1, Placebo
Biological · Other
Lead sponsor
OxThera
Industry
Eligibility
2 Years and older
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2021
U.S. locations
1
States / cities
Nashville, Tennessee
Source: ClinicalTrials.gov public record
Updated Dec 10, 2021 · Synced Oct 14, 2026, 2:10 PM EDT
Withdrawn Not applicable Interventional

Utility of Bowel Ultrasound in Diagnosing Necrotizing Eneterocolitis in Congenital Heart Disease

NCT03142685
View directory record Official record
Conditions
Enterocolitis, Necrotizing, Congenital Heart Disease
Interventions
Bowel Ultrasound
Device
Lead sponsor
Children's Mercy Hospital Kansas City
Other
Eligibility
Up to 6 Months
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2018
U.S. locations
1
States / cities
Kansas City, Missouri
Source: ClinicalTrials.gov public record
Updated Jul 18, 2018 · Synced Oct 14, 2026, 2:10 PM EDT
Completed No phase listed Observational

Psychological Burden of Thyroid Eye Disease

NCT07444190
View directory record Official record
Conditions
Thyroid Eye Disease, TED
Interventions
focus groups and individual interviews as well as filling out questionnaires
Other
Lead sponsor
Thomas Jefferson University
Other
Eligibility
18 Years and older
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Mar 2, 2026 · Synced Oct 14, 2026, 2:10 PM EDT
Recruiting Phase 1 Interventional

A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of DNL952 in Adult Participants With Late-Onset Pompe Disease

NCT07354724
View directory record Official record
Conditions
Late-onset Pompe Disease
Interventions
DNL952
Drug
Lead sponsor
Denali Therapeutics Inc.
Industry
Eligibility
18 Years to 75 Years
Enrollment
32 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2028
U.S. locations
4
States / cities
Irvine, California • St Louis, Missouri • Durham, North Carolina + 1 more
Source: ClinicalTrials.gov public record
Updated Aug 17, 2026 · Synced Oct 14, 2026, 2:10 PM EDT