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ClinicalTrials.gov public records Last synced Oct 21, 2026, 10:36 PM EDT

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Showing 193–216 of 10,540 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Congenital Disorders Clear all
Recruiting Phase 3 Interventional

Effect of Sacubitril-Valsartan on Cardiac Structure and Function

NCT06693674
View directory record Official record
Conditions
Congenital Heart Disease
Interventions
Entresto Pill, Placebo
Drug · Other
Lead sponsor
Mayo Clinic
Other
Eligibility
18 Years and older
Enrollment
45 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2028
U.S. locations
1
States / cities
Rochester, Minnesota
Source: ClinicalTrials.gov public record
Updated Oct 9, 2026 · Synced Oct 21, 2026, 10:36 PM EDT
Completed No phase listed Observational

The Feasibility of Text Messaging to Assess Secondhand Smoke Exposure Among Youngsters With Cancer or Sickle Cell Disease

NCT01591187
View directory record Official record
Conditions
Cancer, Sickle Cell Disease
Interventions
Text messaging, Questionnaire, Interviews
Other
Lead sponsor
St. Jude Children's Research Hospital
Other
Eligibility
10 Years to 17 Years
Enrollment
77 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2014
U.S. locations
1
States / cities
Memphis, Tennessee
Source: ClinicalTrials.gov public record
Updated Mar 5, 2015 · Synced Oct 21, 2026, 10:36 PM EDT
Recruiting No phase listed Observational

Non-interventional Study of Seroprevalence of Pre-existing Antibodies Against Adenovirus-associated Virus Vector (AAV9) and the Progression of Disease in Patients With Plakophilin 2 (PKP2)-Associated Arrhythmogenic Right Ventricular Cardiomyopathy (ARVC)

NCT06311708
View directory record Official record
Conditions
Arrhythmogenic Right Ventricular Cardiomyopathy
Interventions
Not listed
Lead sponsor
Tenaya Therapeutics
Industry
Eligibility
14 Years to 65 Years
Enrollment
200 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2030
U.S. locations
8
States / cities
San Francisco, California • Aurora, Colorado • Baltimore, Maryland + 5 more
Source: ClinicalTrials.gov public record
Updated Nov 1, 2024 · Synced Oct 21, 2026, 10:36 PM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

A Study to Evaluate Pharmacokinetic Parameters of Eliglustat in Healthy Volunteers Who Are CYP2D6 Extensive or Poor Metabolizers

NCT06188325
View directory record Official record
Conditions
Gaucher's Disease
Interventions
Eliglustat
Drug
Lead sponsor
Sanofi
Industry
Eligibility
18 Years to 65 Years
Enrollment
18 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2018
U.S. locations
1
States / cities
Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Jan 10, 2024 · Synced Oct 21, 2026, 10:36 PM EDT
Completed Phase 2 Interventional Results available

Historically Controlled Trial of Corticosteroids in Young Boys With Duchenne Muscular Dystrophy

NCT02167217
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Prednisolone
Drug
Lead sponsor
Washington University School of Medicine
Other
Eligibility
1 Month to 30 Months · Male only
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2017
U.S. locations
6
States / cities
Sacramento, California • Orlando, Florida • Chicago, Illinois + 3 more
Source: ClinicalTrials.gov public record
Updated Dec 21, 2018 · Synced Oct 21, 2026, 10:36 PM EDT
Not listed Not applicable Interventional

Safety and Efficacy Study of the Draeger Babylog VN500 Device in HFOV Mode in VLBW Neonates

NCT02445040
View directory record Official record
Conditions
Respiratory Distress Syndrome In Premature Infants
Interventions
Babylog VN500 in HFOV Mode
Device
Lead sponsor
Draeger Medical Systems, Inc.
Industry
Eligibility
23 Weeks to 30 Weeks
Enrollment
225 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2020
U.S. locations
14
States / cities
Little Rock, Arkansas • San Diego, California • Iowa City, Iowa + 9 more
Source: ClinicalTrials.gov public record
Updated Sep 19, 2019 · Synced Oct 21, 2026, 10:36 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 21, 2026, 10:36 PM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

National Eye Institute Biorepository for Retinal Diseases

NCT01496625
View directory record Official record
Conditions
Age-Related Macular Degeneration, Diabetic Retinopathy, Von Hippel-Lindau Syndrome, Retinal Disease, Retinal Vein Occlusion
Interventions
Not listed
Lead sponsor
National Eye Institute (NEI)
NIH
Eligibility
2 Years to 120 Years
Enrollment
650 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
Started 2012
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 16, 2026 · Synced Oct 21, 2026, 10:36 PM EDT
Active, not recruiting Phase 1 Interventional

Study of BMN 349 Single Dose in PiZZ and PiMZ/MASH Adult Participants

NCT06738017
View directory record Official record
Conditions
Alpha 1-Antitrypsin Deficiency
Interventions
BMN 349, Placebo
Drug
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
18 Years to 75 Years
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2026
U.S. locations
4
States / cities
San Diego, California • St Louis, Missouri • Cincinnati, Ohio + 1 more
Source: ClinicalTrials.gov public record
Updated May 6, 2026 · Synced Oct 21, 2026, 10:36 PM EDT
Terminated No phase listed Observational

Blood Samples From Patients on a Clinical Trial to CINV During HSCT

NCT00900068
View directory record Official record
Conditions
Breast Cancer, Chronic Myeloproliferative Disorders, Gestational Trophoblastic Tumor, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Neoplasms, Nausea and Vomiting, Neuroblastoma, Ovarian Cancer, Testicular Germ Cell Tumor
Interventions
immunoenzyme technique, laboratory biomarker analysis, medical chart review
Other
Lead sponsor
OHSU Knight Cancer Institute
Other
Eligibility
18 Years to 120 Years
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2009
U.S. locations
1
States / cities
Portland, Oregon
Source: ClinicalTrials.gov public record
Updated May 9, 2017 · Synced Oct 21, 2026, 10:36 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

The Roles of Trust and Respect in Patient Reactions to Race-based and Personalized Medicine Vignettes: An Experimental Study

NCT00911833
View directory record Official record
Conditions
Diabetes, Congenital Heart Disease, Hypertension
Interventions
Not listed
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
18 Years and older
Enrollment
600 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2009 – 2011
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 2, 2017 · Synced Oct 21, 2026, 10:36 PM EDT
Recruiting No phase listed Observational

Extracellular RNA Biomarkers of Duchenne Muscular Dystrophy

NCT05016908
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Not listed
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
5 Years and older
Enrollment
100 participants
Timeline
2019 – 2027
U.S. locations
2
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Nov 24, 2025 · Synced Oct 21, 2026, 10:36 PM EDT
Completed Phase 4 Interventional Results available

Management of Pruritus With Xyzal in Atopic Dermatitis

NCT00884325
View directory record Official record
Conditions
Atopic Dermatitis, Pruritus
Interventions
Levocetirizine dihydrochloride (Xyzal), placebo
Drug
Lead sponsor
Derm Research, PLLC
Other
Eligibility
18 Years and older
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2010
U.S. locations
1
States / cities
Louisville, Kentucky
Source: ClinicalTrials.gov public record
Updated Aug 16, 2013 · Synced Oct 21, 2026, 10:36 PM EDT
Completed Not applicable Interventional Accepts healthy volunteers

Adherence to Colon Cancer Screening (ACCS)

NCT02112747
View directory record Official record
Conditions
Colon Cancer, Rectal Cancer
Interventions
Patient Navigator
Other
Lead sponsor
Ohio State University Comprehensive Cancer Center
Other
Eligibility
25 Years to 75 Years
Enrollment
1,900 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2013 – 2020
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Mar 10, 2021 · Synced Oct 21, 2026, 10:36 PM EDT
Completed Phase 3 Interventional Results available

Efficacy, Safety, and Tolerability of ACZ885 in Patients With Muckle-Wells Syndrome

NCT00465985
View directory record Official record
Conditions
Muckle Wells Syndrome
Interventions
ACZ885, Placebo
Drug
Lead sponsor
Novartis
Industry
Eligibility
4 Years to 75 Years
Enrollment
35 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2007
U.S. locations
3
States / cities
San Francisco, California • Chicago, Illinois • Madison, Wisconsin
Source: ClinicalTrials.gov public record
Updated Aug 28, 2017 · Synced Oct 21, 2026, 10:36 PM EDT
Terminated Phase 1Phase 2 Interventional

Subcutaneous Implant Combination Product (AUR-201) in Patients With Unilateral Microtia

NCT06072040
View directory record Official record
Conditions
Microtia
Interventions
AUR-201
Combination Product
Lead sponsor
Auregen Biotherapeutics, SA
Industry
Eligibility
8 Years to 29 Years
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2025
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Nov 21, 2025 · Synced Oct 21, 2026, 10:36 PM EDT
Suspended Phase 2 Interventional

A Study to Assess the Tolerability of a Single Dose of STX107 in Adults With Fragile X Syndrome

NCT01325740
View directory record Official record
Conditions
Fragile X Syndrome
Interventions
STX107
Drug
Lead sponsor
Seaside Therapeutics, Inc.
Industry
Eligibility
18 Years to 50 Years · Male only
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2013
U.S. locations
5
States / cities
Los Angeles, California • Sacramento, California • Aurora, Colorado + 2 more
Source: ClinicalTrials.gov public record
Updated Dec 20, 2012 · Synced Oct 21, 2026, 10:36 PM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

A Study to Evaluate the Safety, Reactogenicity, and Immunogenicity of Cytomegalovirus (CMV) Vaccine mRNA-1647

NCT05105048
View directory record Official record
Conditions
Cytomegalovirus Infection
Interventions
mRNA-1647, Placebo
Biological
Lead sponsor
ModernaTX, Inc.
Industry
Eligibility
18 Years to 40 Years
Enrollment
9 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2021 – 2023
U.S. locations
3
States / cities
North Hollywood, California • San Diego, California • Honolulu, Hawaii
Source: ClinicalTrials.gov public record
Updated Sep 11, 2023 · Synced Oct 21, 2026, 10:36 PM EDT
Active, not recruiting Not applicable Interventional Results available

Sickle Cell Disease (SCD) Bone Pain Study

NCT05283148
View directory record Official record
Conditions
Sickle Cell Disease, Sickle Cell Anemia, Low Bone Density, Osteoporosis, Osteopenia, Vertebral Fracture, Vertebral Compression, Osteonecrosis, Ischemic Necrosis, Avascular Necrosis
Interventions
Dual-energy X-ray absorptiometry, Vertebral fracture analysis, Adult Sickle Cell Quality of Life Measurement System pain impact questionnaire
Other
Lead sponsor
University of California, Davis
Other
Eligibility
18 Years to 80 Years
Enrollment
53 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2027
U.S. locations
1
States / cities
Sacramento, California
Source: ClinicalTrials.gov public record
Updated Sep 23, 2026 · Synced Oct 21, 2026, 10:36 PM EDT
Completed Not applicable Interventional

Chemotherapy With CD133+ Select Autologous Hematopoietic Stem Cells for Children With Solid Tumors and Lymphomas

NCT00152126
View directory record Official record
Conditions
Neuroblastoma, Central Nervous System Tumors, Lymphomas, Wilms Tumor
Interventions
Stem Cell Transplantation, Busulfan, Melphalan
Procedure · Drug
Lead sponsor
St. Jude Children's Research Hospital
Other
Eligibility
Up to 25 Years
Enrollment
26 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003 – 2009
U.S. locations
1
States / cities
Memphis, Tennessee
Source: ClinicalTrials.gov public record
Updated Feb 13, 2009 · Synced Oct 21, 2026, 10:36 PM EDT
Terminated Phase 1 Interventional

Eltrombopag Olamine in Treating Patients With Relapsed/Refractory Acute Myeloid Leukemia

NCT01550185
View directory record Official record
Conditions
Adult Acute Basophilic Leukemia, Adult Acute Eosinophilic Leukemia, Adult Acute Megakaryoblastic Leukemia (M7), Adult Acute Minimally Differentiated Myeloid Leukemia (M0), Adult Acute Monoblastic Leukemia (M5a), Adult Acute Monocytic Leukemia (M5b), Adult Acute Myeloblastic Leukemia With Maturation (M2), Adult Acute Myeloblastic Leukemia Without Maturation (M1), Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities, Adult Acute Myeloid Leukemia With Del(5q), Adult Acute Myeloid Leukemia With Inv(16)(p13;q22), Adult Acute Myeloid Leukemia With t(16;16)(p13;q22), Adult Acute Myeloid Leukemia With t(8;21)(q22;q22), Adult Acute Myelomonocytic Leukemia (M4), Adult Erythroleukemia (M6a), Adult Pure Erythroid Leukemia (M6b), Recurrent Adult Acute Myeloid Leukemia
Interventions
eltrombopag olamine, standard follow-up care
Drug · Procedure
Lead sponsor
Roswell Park Cancer Institute
Other
Eligibility
18 Years and older
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2014
U.S. locations
2
States / cities
Atlanta, Georgia • Buffalo, New York
Source: ClinicalTrials.gov public record
Updated Jul 22, 2022 · Synced Oct 21, 2026, 10:36 PM EDT
Not listed No phase listed Observational

Management of Esophagitis Following Repair of Esophageal Atresia

NCT03619408
View directory record Official record
Conditions
Esophageal Atresia, Esophagitis
Interventions
Antacids
Drug
Lead sponsor
Boston Children's Hospital
Other
Eligibility
30 Days to 17 Years
Enrollment
50 participants
Timeline
2019 – 2025
U.S. locations
2
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Sep 10, 2021 · Synced Oct 21, 2026, 10:36 PM EDT
Terminated No phase listed Observational

Natural History Study to Characterise the Course of Disease Progression in Participants With Mucopolysaccharidosis Type IIIB

NCT02293408
View directory record Official record
Conditions
MPS IIIB (Sanfilippo B Syndrome)
Interventions
Not listed
Lead sponsor
Alexion Pharmaceuticals, Inc.
Industry
Eligibility
1 Year and older
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2017
U.S. locations
2
States / cities
Minneapolis, Minnesota • Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Sep 13, 2018 · Synced Oct 21, 2026, 10:36 PM EDT
Terminated Phase 3 Interventional Results available

A Study of Nivolumab-relatlimab Fixed-dose Combination Versus Regorafenib or TAS-102 in Participants With Later-lines of Metastatic Colorectal Cancer

NCT05328908
View directory record Official record
Conditions
Colorectal Neoplasms
Interventions
Nivolumab-relatlimab FDC, Regorafenib, TAS-102
Drug
Lead sponsor
Bristol-Myers Squibb
Industry
Eligibility
18 Years and older
Enrollment
769 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2025
U.S. locations
20
States / cities
Springdale, Arkansas • Los Angeles, California • Norwich, Connecticut + 17 more
Source: ClinicalTrials.gov public record
Updated Aug 26, 2026 · Synced Oct 21, 2026, 10:36 PM EDT