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ClinicalTrials.gov public records Last synced Oct 5, 2026, 4:52 AM EDT

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Showing 73–96 of 10,558 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Congenital Anomalies Clear all
Completed No phase listed Observational Accepts healthy volunteers

A Study of IMMray™ PanCan-d Test for Early Detection of Pancreatic Cancer in High-risk Groups

NCT03693378
View directory record Official record
Conditions
Pancreatic Ductal Adenocarcinoma, Familial Pancreatic Cancer, FAMMM - Familial Atypical Mole Malignant Melanoma Syndrome
Interventions
Not listed
Lead sponsor
Immunovia, Inc.
Industry
Eligibility
Not listed
Enrollment
1,349 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2016 – 2021
U.S. locations
13
States / cities
Stanford, California • New Haven, Connecticut • Chicago, Illinois + 8 more
Source: ClinicalTrials.gov public record
Updated Mar 31, 2022 · Synced Oct 5, 2026, 4:52 AM EDT
Terminated Phase 1Phase 2 Interventional Results available

An Open-Label Extension Study for Patients With Duchenne Muscular Dystrophy Who Participated in Studies of SRP-5051 (Vesleteplirsen)

NCT03675126
View directory record Official record
Conditions
Muscular Dystrophy, Duchenne
Interventions
SRP-5051
Drug
Lead sponsor
Sarepta Therapeutics, Inc.
Industry
Eligibility
4 Years and older · Male only
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2021
U.S. locations
7
States / cities
Hartford, Connecticut • Gulf Breeze, Florida • Atlanta, Georgia + 4 more
Source: ClinicalTrials.gov public record
Updated Sep 19, 2024 · Synced Oct 5, 2026, 4:52 AM EDT
Active, not recruiting Phase 3 Interventional Results available

A Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Subcutaneous Emicizumab in Participants From Birth to 12 Months of Age With Hemophilia A Without Inhibitors

NCT04431726
View directory record Official record
Conditions
Severe Hemophilia A
Interventions
Emicizumab
Drug
Lead sponsor
Hoffmann-La Roche
Industry
Eligibility
0 Months to 12 Months
Enrollment
55 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2030
U.S. locations
6
States / cities
Phoenix, Arizona • Los Angeles, California • Aurora, Colorado + 3 more
Source: ClinicalTrials.gov public record
Updated Oct 1, 2026 · Synced Oct 5, 2026, 4:52 AM EDT
Recruiting No phase listed Observational

MAGNETIC RESONANCE SPECTROSCOPY BIOMARKERS IN TYPE 3 GAUCHER DISEASE (GD3)

NCT05586243
View directory record Official record
Conditions
Gaucher Disease, Type 3
Interventions
No intervention
Other
Lead sponsor
University of Minnesota
Other
Eligibility
18 Years to 80 Years
Enrollment
5 participants
Timeline
2023 – 2026
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Aug 4, 2026 · Synced Oct 5, 2026, 4:52 AM EDT
Recruiting Not applicable Interventional

NobleStitch EL STITCH Trial is a PFO Comparative Trial

NCT04339699
View directory record Official record
Conditions
Foramen Ovale, Patent, Stroke, Ischemic
Interventions
NobleStitch™EL Suture Mediated Closure System, Amplatzer PFO Occluder
Device
Lead sponsor
Nobles Medical Technologies II Inc
Industry
Eligibility
18 Years to 60 Years
Enrollment
640 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2027
U.S. locations
1
States / cities
Tucson, Arizona
Source: ClinicalTrials.gov public record
Updated Mar 23, 2026 · Synced Oct 5, 2026, 4:52 AM EDT
Active, not recruiting Phase 2 Interventional

Phase 2 Open-label Extension Study of AOC 1020 in Participants With Facioscapulohumeral Muscular Dystrophy (FSHD)

NCT06547216
View directory record Official record
Conditions
FSHD, FSHD1, FSHD2, FMD, FMD2, Fascioscapulohumeral Muscular Dystrophy, Fascioscapulohumeral Muscular Dystrophy Type 1, Fascioscapulohumeral Muscular Dystrophy Type 2, Dystrophies, Facioscapulohumeral Muscular, Dystrophy, Facioscapulohumeral Muscular, Facioscapulohumeral Muscular Dystrophy 1, Facioscapulohumeral Muscular Dystrophy 2, Facio-Scapulo-Humeral Dystrophy, Atrophy, Facioscapulohumeral, Atrophies, Facioscapulohumeral, Facioscapulohumeral Atrophy, Muscular Dystrophies, Muscular Dystrophy, Facioscapulohumeral, FSH Muscular Dystrophy, Landouzy Dejerine Dystrophy, Landouzy-Dejerine Muscular Dystrophy, Dystrophies, Landouzy-Dejerine, Dystrophy, Landouzy-Dejerine, Landouzy-Dejerine Syndrome, Muscular Dystrophy, Landouzy Dejerine, Progressive Muscular Dystrophy, FSH
Interventions
AOC 1020
Drug
Lead sponsor
Avidity Biosciences, Inc.
Industry
Eligibility
16 Years to 72 Years
Enrollment
84 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2030
U.S. locations
14
States / cities
Los Angeles, California • Palo Alto, California • San Diego, California + 11 more
Source: ClinicalTrials.gov public record
Updated Jul 14, 2026 · Synced Oct 5, 2026, 4:52 AM EDT
Completed Phase 3 Interventional Results available

Rilonacept for Treatment of Cryopyrin-Associated Periodic Syndromes (CAPS)

NCT00288704
View directory record Official record
Conditions
Familial Cold Autoinflammatory Syndrome (FCAS), Familial Cold Urticaria, Muckle-Wells Syndrome (MWS), Genetic Diseases, Inborn
Interventions
rilonacept 160 mg, Placebo
Drug
Lead sponsor
Regeneron Pharmaceuticals
Industry
Eligibility
7 Years and older
Enrollment
104 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2008
U.S. locations
24
States / cities
Little Rock, Arkansas • Palm Desert, California • Upland, California + 21 more
Source: ClinicalTrials.gov public record
Updated Dec 6, 2011 · Synced Oct 5, 2026, 4:52 AM EDT
Completed No phase listed Observational

Ultrasound Shear Wave Elastography Evaluation of Suspected and Known Biliary Atresia

NCT02652533
View directory record Official record
Conditions
Biliary Atresia
Interventions
Ultrasound Shear Wave Elastography
Device
Lead sponsor
Children's Hospital Medical Center, Cincinnati
Other
Eligibility
Up to 3 Months
Enrollment
41 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2019
U.S. locations
4
States / cities
Stanford, California • Ann Arbor, Michigan • Cincinnati, Ohio + 1 more
Source: ClinicalTrials.gov public record
Updated Sep 23, 2019 · Synced Oct 5, 2026, 4:52 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Research Study in Healthy Volunteers of Patients With Fanconi Anemia, Myeloproliferative Disorders, or Myeloma

NCT00900055
View directory record Official record
Conditions
Chronic Myeloproliferative Disorders, Fanconi Anemia, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic/Myeloproliferative Neoplasms
Interventions
microarray analysis, polyacrylamide gel electrophoresis, polymerase chain reaction, protein expression analysis, reverse transcriptase-polymerase chain reaction, western blotting, chromatography, high performance liquid chromatography, immunoenzyme technique
Genetic · Other
Lead sponsor
OHSU Knight Cancer Institute
Other
Eligibility
1 Year to 55 Years
Enrollment
213 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
1975 – 2016
U.S. locations
1
States / cities
Portland, Oregon
Source: ClinicalTrials.gov public record
Updated Jul 5, 2022 · Synced Oct 5, 2026, 4:52 AM EDT
Active, not recruiting Phase 1 Interventional

Intramyocardial Injection of Autologous UCB-MNC During Fontan Surgery for SRV Dependent CHD

NCT04907526
View directory record Official record
Conditions
Congenital Heart Disease, SRV Dependent
Interventions
Autologous mononuclear cells
Biological
Lead sponsor
Timothy J Nelson, MD, PhD
Other
Eligibility
2 Years to 5 Years
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2025
U.S. locations
6
States / cities
Birmingham, Alabama • Los Angeles, California • Aurora, Colorado + 3 more
Source: ClinicalTrials.gov public record
Updated Jun 27, 2025 · Synced Oct 5, 2026, 4:52 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Cancer of the Pancreas Screening Study (CAPS 3)

NCT00438906
View directory record Official record
Conditions
Pancreatic Neoplasm, Peutz-Jeghers Syndrome
Interventions
Biopsy, Fine Needle Aspiration (FNA), Secretin (human synthetic) - ChiRhoClin
Procedure · Drug
Lead sponsor
Johns Hopkins University
Other
Eligibility
40 Years to 80 Years
Enrollment
200 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2006 – 2009
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Jun 18, 2021 · Synced Oct 5, 2026, 4:52 AM EDT
Recruiting Phase 4 Interventional

Optimizing Perioperative Analgesia to Lower Pain Following Cleft Palate Surgery

NCT06962306
View directory record Official record
Conditions
Cleft Palate, Pain, Postoperative Care, Perioperative Care, Children
Interventions
Methadone hydrochloride, Fentanyl/Hydromorphone
Drug
Lead sponsor
Duke University
Other
Eligibility
6 Months to 4 Years
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Apr 14, 2026 · Synced Oct 5, 2026, 4:52 AM EDT
Completed Phase 2 Interventional

A Single-Dose Open-Label Study of XOMA 358 in Subjects With Congenital Hyperinsulinism

NCT02604485
View directory record Official record
Conditions
Congenital Hyperinsulinism
Interventions
Cohort 1, Cohort 2, Cohort 3, Cohort 4
Drug
Lead sponsor
XOMA (US) LLC
Industry
Eligibility
18 Years and older
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2017
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Feb 6, 2017 · Synced Oct 5, 2026, 4:52 AM EDT
Recruiting Not applicable Interventional

Comparing Direct vs Indirect Methods for Cascade Screening

NCT05348564
View directory record Official record
Conditions
Long QT Syndrome, Familial Hypercholesterolemia, Ethics, Genetic Testing
Interventions
Direct contact of family members for cascade screening of relevant variants
Behavioral
Lead sponsor
University of Maryland, Baltimore
Other
Eligibility
18 Years and older
Enrollment
200 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2027
U.S. locations
1
States / cities
Lancaster, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Apr 14, 2026 · Synced Oct 5, 2026, 4:52 AM EDT
Completed Phase 1 Interventional Accepts healthy volunteers Results available

Pharmacokinetics of Eleclazine in Adults With Normal and Impaired Hepatic Function

NCT02412098
View directory record Official record
Conditions
Long QT Syndrome
Interventions
Eleclazine
Drug
Lead sponsor
Gilead Sciences
Industry
Eligibility
18 Years to 65 Years
Enrollment
49 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2015 – 2016
U.S. locations
4
States / cities
Miami, Florida • Orlando, Florida • Minneapolis, Minnesota + 1 more
Source: ClinicalTrials.gov public record
Updated Jul 29, 2019 · Synced Oct 5, 2026, 4:52 AM EDT
Completed Not applicable Interventional Accepts healthy volunteers

3-D Correction of Adolescent Idiopathic Scoliosis (AIS) With Differential Metals

NCT05631821
View directory record Official record
Conditions
Juvenile and Adolescent Idiopathic Scoliosis
Interventions
differential rod bending with rod contours (MESA Spinal System)
Procedure
Lead sponsor
Rush University Medical Center
Other
Eligibility
10 Years to 18 Years
Enrollment
20 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2014 – 2022
U.S. locations
1
States / cities
Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Dec 2, 2022 · Synced Oct 5, 2026, 4:52 AM EDT
Active, not recruiting Phase 2Phase 3 Interventional

A Phase 2/3 Trial to Assess the Efficacy and Safety of OCU410ST for Stargardt Disease

NCT05956626
View directory record Official record
Conditions
Stargardt Disease
Interventions
OCU410ST
Drug
Lead sponsor
Ocugen
Industry
Eligibility
5 Years and older
Enrollment
51 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2026
U.S. locations
14
States / cities
Phoenix, Arizona • Sacramento, California • Gainesville, Florida + 11 more
Source: ClinicalTrials.gov public record
Updated Jul 31, 2026 · Synced Oct 5, 2026, 4:52 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Evaluating Cognitive Outcomes in Down Syndrome

NCT04208685
View directory record Official record
Conditions
Down Syndrome
Interventions
Not listed
Lead sponsor
Children's Hospital Medical Center, Cincinnati
Other
Eligibility
6 Years to 35 Years
Enrollment
202 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2018 – 2024
U.S. locations
2
States / cities
Fort Collins, Colorado • Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Aug 12, 2026 · Synced Oct 5, 2026, 4:52 AM EDT
Not yet recruiting Phase 4 Interventional

Congenital Syphilis Treatment Trial (CONSISTENT) in Neonates

NCT07457385
View directory record Official record
Conditions
Syphilis, Congenital
Interventions
Benzathine Penicillin, Amoxicillin
Drug
Lead sponsor
University of Alabama at Birmingham
Other
Eligibility
0 Days to 30 Days
Enrollment
374 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2031
U.S. locations
11
States / cities
Birmingham, Alabama • Mobile, Alabama • Orange, California + 8 more
Source: ClinicalTrials.gov public record
Updated Sep 2, 2026 · Synced Oct 5, 2026, 4:52 AM EDT
Completed Phase 2 Interventional Results available

A Study of Pridopidine (ACR16) for the Treatment of Participants With Huntington's Disease

NCT00724048
View directory record Official record
Conditions
Huntington Disease
Interventions
ACR16, Placebo
Drug · Other
Lead sponsor
Teva Branded Pharmaceutical Products R&D, Inc.
Industry
Eligibility
30 Years and older
Enrollment
227 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2010
U.S. locations
22
States / cities
Birmingham, Alabama • San Diego, California • Littleton, Colorado + 18 more
Source: ClinicalTrials.gov public record
Updated Aug 31, 2023 · Synced Oct 5, 2026, 4:52 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Impact of Elastin Mediated Vascular Stiffness on End Organs

NCT02840448
View directory record Official record
Conditions
Williams Syndrome, Supravalvular Aortic Stenosis, Cardiovascular Disease
Interventions
Not listed
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
3 Years to 85 Years
Enrollment
159 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2016 – 2024
U.S. locations
2
States / cities
Bethesda, Maryland • St Louis, Missouri
Source: ClinicalTrials.gov public record
Updated Oct 2, 2026 · Synced Oct 5, 2026, 4:52 AM EDT
Not listed No phase listed Observational

Validation of Optical Genome Mapping for the Identification of Constitutional Genomic Variants in a Postnatal Cohort

NCT05295277
View directory record Official record
Conditions
Developmental Disability, Intellectual Disability, Autism Spectrum Disorder, Congenital Anomaly, Fragile X Syndrome, Facioscapulohumeral Muscular Dystrophy 1
Interventions
Standard of care genetic testing group
Other
Lead sponsor
Bionano Genomics
Industry
Eligibility
Not listed
Enrollment
1,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2024
U.S. locations
8
States / cities
Atlanta, Georgia • Augusta, Georgia • Iowa City, Iowa + 5 more
Source: ClinicalTrials.gov public record
Updated Aug 7, 2023 · Synced Oct 5, 2026, 4:52 AM EDT
Completed Phase 2 Interventional Results available

Safety and Efficacy Study of Recombinant Human Insulin-Like Growth Factor-I/Recombinant Human Insulin-Like Growth Factor Binding Protein-3 (rhIGF-I/rhIGFBP-3) In Myotonic Dystrophy Type 1

NCT00577577
View directory record Official record
Conditions
Myotonic Dystrophy Type 1
Interventions
rhIGF-I/rhIGFBP-3, placebo
Drug
Lead sponsor
Insmed Incorporated
Industry
Eligibility
21 Years to 65 Years
Enrollment
69 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2008
U.S. locations
12
States / cities
Orange, California • Sacramento, California • Kansas City, Kansas + 9 more
Source: ClinicalTrials.gov public record
Updated Jan 6, 2022 · Synced Oct 5, 2026, 4:52 AM EDT
Completed No phase listed Observational

Diagnostic and Screening Study of Genetic Disorders

NCT00006057
View directory record Official record
Conditions
Tay-Sachs Disease, Porphyria, Erythropoietic, Leukodystrophy, Globoid Cell, Metabolism, Inborn Errors
Interventions
Not listed
Lead sponsor
National Center for Research Resources (NCRR)
NIH
Eligibility
0 Years and older
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1999
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jun 24, 2005 · Synced Oct 5, 2026, 4:52 AM EDT