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ClinicalTrials.gov public records Last synced Oct 19, 2026, 10:24 PM EDT

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Showing 289–312 of 9,568 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Genetics Clear all
Completed Phase 1 Interventional

Haploidentical Stem Cell Transplant for Patients With Sickle Cell Disease and Prior Stroke or Abnormal Transcranial Ultrasound

NCT00152113
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Hematopoietic Stem Cell Transplantation, CliniMACS, See intervention description
Procedure · Device · Drug
Lead sponsor
St. Jude Children's Research Hospital
Other
Eligibility
2 Years to 16 Years
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2009
U.S. locations
1
States / cities
Memphis, Tennessee
Source: ClinicalTrials.gov public record
Updated Apr 26, 2017 · Synced Oct 19, 2026, 10:24 PM 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 19, 2026, 10:24 PM EDT
Completed Not applicable Interventional

Individualizing Hemophilia Prophylaxis Using Thromboelastography

NCT02582060
View directory record Official record
Conditions
Hemophilia A
Interventions
Thromboelastography/Thromboelastometry-Guided Treatment Regimen
Other
Lead sponsor
Children's Hospital Los Angeles
Other
Eligibility
5 Years to 70 Years · Male only
Enrollment
18 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2017
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Feb 18, 2019 · Synced Oct 19, 2026, 10:24 PM EDT
Active, not recruiting Phase 4 Interventional

Long-term Use of Viltolarsen in Boys With Duchenne Muscular Dystrophy in Clinical Practice (VILT-502)

NCT04687020
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Viltolarsen
Drug
Lead sponsor
NS Pharma, Inc.
Industry
Eligibility
Male only
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2032
U.S. locations
4
States / cities
Sacramento, California • Chicago, Illinois • Durham, North Carolina + 1 more
Source: ClinicalTrials.gov public record
Updated Oct 19, 2022 · Synced Oct 19, 2026, 10:24 PM EDT
Completed Phase 1 Interventional

Albuterol in Individuals With Late Onset Pompe Disease (LOPD)

NCT01859624
View directory record Official record
Conditions
Pompe Disease
Interventions
Albuterol
Drug
Lead sponsor
Duke University
Other
Eligibility
18 Years and older
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2013
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Jul 5, 2019 · Synced Oct 19, 2026, 10:24 PM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

Mass Balance Study of [14C]LC350189 in Healthy Volunteers

NCT04070846
View directory record Official record
Conditions
Gout, Hyperuricemia
Interventions
[14C] LC350189
Drug
Lead sponsor
LG Chem
Industry
Eligibility
18 Years to 55 Years · Male only
Enrollment
6 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2020
U.S. locations
1
States / cities
Austin, Texas
Source: ClinicalTrials.gov public record
Updated Jun 26, 2020 · Synced Oct 19, 2026, 10:24 PM EDT
Completed No phase listed Observational

Study of Abnormal Blood Clotting in Children With Stroke

NCT00001927
View directory record Official record
Conditions
Abnormalities, Blood Coagulation Disorder, Brain Disease, Cerebrovascular Accident, Vascular Disease
Interventions
Not listed
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
Up to 45 Years
Enrollment
130 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1999 – 2011
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 2, 2017 · Synced Oct 19, 2026, 10:24 PM EDT
Recruiting Phase 2 Interventional

Hematopoietic Stem Cell Transplantation (HSCT) for Common Variable Immunodeficiency (CVID) and Other Autoimmune Manifestations of Primary Immune Regulatory Disorders (PIRD)

NCT07284641
View directory record Official record
Conditions
Common Variable Immunodeficiency (CVID), Primary Immune Regulatory Disorder, Immune Dysregulation, DiGeorge Syndrome, STAT 1 Gain of Function, STAT 3 Gain of Function, Hypomorphic RAG1 Deficiency, CD40 Ligand Deficiency, Mendelian Susceptibility to Mycobacterial Disease, GATA2 Associated Immunodeficiency, CD40 Deficiency, Hypomorphic RAG2 Deficiency, Immune Dysregulation Polyendocrinopathy Enteropathy X-Linked Syndrome, Omenn Syndrome, Chronic Granulomatous Disease
Interventions
Hematopoietic stem cell transplant (HSCT)
Biological
Lead sponsor
Paul Szabolcs
Other
Eligibility
5 Years to 40 Years
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2031
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated May 15, 2026 · Synced Oct 19, 2026, 10:24 PM EDT
Not listed No phase listed Observational Accepts healthy volunteers

Genetics of Neural Tube Defects

NCT01253746
View directory record Official record
Conditions
Neural Tube Defects
Interventions
Not listed
Lead sponsor
John P. Hussman Institute for Human Genomics
Other
Eligibility
Not listed
Enrollment
6,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
Started 2009
U.S. locations
1
States / cities
Miami, Florida
Source: ClinicalTrials.gov public record
Updated Dec 3, 2010 · Synced Oct 19, 2026, 10:24 PM EDT
Not listed No phase listed Observational Accepts healthy volunteers

Study of Total Energy Expenditure in Infants and Children With Moderate to Severe Cystic Fibrosis

NCT00006273
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
Not listed
Lead sponsor
National Center for Research Resources (NCRR)
NIH
Eligibility
0 Years to 10 Years
Enrollment
60 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
Started 1996
U.S. locations
1
States / cities
Indianapolis, Indiana
Source: ClinicalTrials.gov public record
Updated Jun 24, 2005 · Synced Oct 19, 2026, 10:24 PM EDT
Completed Not applicable Interventional Results available

Tourette Syndrome Deep Brain Stimulation

NCT02056873
View directory record Official record
Conditions
Tourette Syndrome
Interventions
Continuous DBS, Responsive Brain Stimulation (RBS Setting)
Device
Lead sponsor
University of Florida
Other
Eligibility
21 Years and older
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2023
U.S. locations
1
States / cities
Gainesville, Florida
Source: ClinicalTrials.gov public record
Updated Sep 5, 2024 · Synced Oct 19, 2026, 10:24 PM EDT
Suspended No phase listed Observational Accepts healthy volunteers

A Non-Interventional Pilot Study to Explore the Role of Gut Flora in Disease

NCT04031469
View directory record Official record
Conditions
Gut Microbiome, Gastrointestinal Microbiome, Autism, Autism Spectrum Disorder, Autism Spectrum Disorder High-Functioning, Alzheimer Disease, Alzheimer Dementia (AD), Alzheimer Dementia, Alzheimer Disease (AD), Crohn, Crohn Colitis, Crohn Disease (CD), Crohn Disease Colon, Myalgic Encephalomyelitis, ME/CFS, Myalgic Encephalomyelitis (ME), Myalgic Encephalomyelitis/Chronic Fatigue Syndrome, Psoriasis, Psoriasis Annularis, Psoriasis Chronic, Chronic Urinary Tract Infection, Ulcerative Colitis (Disorder), Ulcerative Colitis (UC), Ulcerative Colitis Acute, Ulcerative Colitis Chronic, Multiple Sclerosis, Multiple Sclerosis (MS) - Relapsing-remitting, Multiple Sclerosis (MS) Primary Progressive, Constipation Chronic Idiopathic, Constipation, Celiac, Celiac Disease, Celiac Sprue, Lyme Arthritis, Lyme Borreliosis, Nervous System, Lyme Disease, Chronic, Cholesterol, Cholesterol Level, High, Cancer, Colon Cancer, Amyotrophic Lateral Sclerosis (ALS), Amyotrophic Lateral Sclerosis, Rheumatoid Arthritis (RA), Rheumatoid Arthritis - Rheumatism, Chronic Fatigue Syndrome (CFS), PARKINSON DISEASE (Disorder), Parkinson Disease, Parkinson Disease (PD), Depression, Major Depressive Disorder (MDD), Depression Disorder, Depression in Adults, Anxiety, Anxiety Disorder Generalized, Obsessive Compulsive Disorder (OCD), Obsessive Compulsive Disorder OCD, Obsessive - Compulsive Disorder, Bipolar, Bipolar 1 Disorder, Bipolar Disorder (BD), Bipolar Disorder I and II, Bipolar and Related Disorders, Migraine, Migraine Disorder, Diabetes (DM), Diabetes, Lupus, Lupus Erythematosus, Epidermolysis Bullosa (EB), Mesothelioma, Mesothelioma Malignant, IBS (Irritable Bowel Syndrome), Irritable Bowel, IBS - Irritable Bowel Syndrome, Irritable Bowel Syndrome (IBS), Eczema, Eczema Atopic Dermatitis, Acne, Myasthaenia Gravis, Gout
Interventions
There is no intervention in this study
Other
Lead sponsor
ProgenaBiome
Other
Eligibility
12 Years and older
Enrollment
1,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2019 – 2030
U.S. locations
1
States / cities
Ventura, California
Source: ClinicalTrials.gov public record
Updated Sep 21, 2026 · Synced Oct 19, 2026, 10:24 PM EDT
Completed No phase listed Observational

Insulin Resistance and Breast Cancer

NCT00304941
View directory record Official record
Conditions
Breast Cancer, Insulin Resistance
Interventions
Not listed
Lead sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
NIH
Eligibility
30 Years to 70 Years · Female only
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2010
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 2, 2017 · Synced Oct 19, 2026, 10:24 PM EDT
Not listed No phase listed Observational

Characterization of Human Cancers by Molecular Profiling of Patient Biospecimens

NCT02749838
View directory record Official record
Conditions
Cancer
Interventions
Not listed
Lead sponsor
Pharmatech
Industry
Eligibility
18 Years and older
Enrollment
183 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2017
U.S. locations
1
States / cities
Denver, Colorado
Source: ClinicalTrials.gov public record
Updated Apr 25, 2016 · Synced Oct 19, 2026, 10:24 PM EDT
Active, not recruiting No phase listed Observational

Genetic and Physical Study of Childhood Nerve and Muscle Disorders

NCT01568658
View directory record Official record
Conditions
Muscular Dystrophies, Muscle Myopathies, Hereditary Spastic Paraplegias, Inherited Neuropathies, Inherited Neuromuscular Conditions
Interventions
Not listed
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
1 Day to 100 Years
Enrollment
2,586 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2012
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 9, 2026 · Synced Oct 19, 2026, 10:24 PM EDT
Completed Not applicable Interventional

Stem Cell Transplantation (SCT) for Genetic Diseases

NCT00004378
View directory record Official record
Conditions
Thrombocytopenia, Metachromatic Leukodystrophy, Fanconi's Anemia, Thalassemia Major, Pure Red-Cell Aplasia, Inborn Errors of Metabolism
Interventions
Stem Cell Transplantation
Procedure
Lead sponsor
National Center for Research Resources (NCRR)
NIH
Eligibility
0 Years to 17 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1995
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Jun 24, 2005 · Synced Oct 19, 2026, 10:24 PM EDT
Temporarily Not Available No phase listed Expanded access

Expanded Access Protocol for Gene Therapy Utilizing shmiR Lentivirus Vector to Induce Fetal Hemoglobin in Sickle Cell Disease

NCT07640815
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Autologous CD34+ HSC cells transduced with the lentiviral vector containing a shRNA targeting BCL11a
Biological
Lead sponsor
David Williams
Other
Eligibility
13 Years to 55 Years
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Jun 11, 2026 · Synced Oct 19, 2026, 10:24 PM EDT
Completed Phase 1 Interventional

Study of the Pharmacokinetics and Safety of Tasimelteon in Children and Adolescents

NCT02776215
View directory record Official record
Conditions
Circadian Rhythm Sleep Disorders, Non-24 Hour Sleep-Wake Disorder, Autism Spectrum Disorder, Smith-Magenis Syndrome
Interventions
tasimelteon
Drug
Lead sponsor
Vanda Pharmaceuticals
Industry
Eligibility
3 Years to 17 Years
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2017
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 21, 2024 · Synced Oct 19, 2026, 10:24 PM EDT
Not listed No phase listed Observational Accepts healthy volunteers

Investigating Lysosomal Storage Diseases in Minority Groups

NCT02120235
View directory record Official record
Conditions
Lysosomal Storage Disorders, Gaucher Disease, Fabry Disease, Pompe Disease, Niemann-Pick Disease
Interventions
Not listed
Lead sponsor
O & O Alpan LLC
Other
Eligibility
1 Day to 100 Years
Enrollment
20,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2014 – 2018
U.S. locations
1
States / cities
Fairfax, Virginia
Source: ClinicalTrials.gov public record
Updated Apr 4, 2017 · Synced Oct 19, 2026, 10:24 PM EDT
Recruiting Not applicable Interventional

Elevated Rate Pacing of Cardiac Amyloidosis

NCT07583823
View directory record Official record
Conditions
Cardiac Amyloidosis, Pacemaker
Interventions
Pacemaker Rate
Other
Lead sponsor
University of Minnesota
Other
Eligibility
18 Years and older
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2026
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Oct 13, 2026 · Synced Oct 19, 2026, 10:24 PM EDT
Active, not recruiting Not applicable Interventional

Robotic-Assisted da Vinci System Prophylactic Nipple-Sparing Mastectomy

NCT03892980
View directory record Official record
Conditions
Nipple Sparing Mastectomy
Interventions
Nipple-Sparing Mastectomy
Device
Lead sponsor
Intuitive Surgical
Industry
Eligibility
18 Years to 80 Years · Female only
Enrollment
145 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2029
U.S. locations
6
States / cities
Jacksonville, Florida • Evanston, Illinois • Rochester, Minnesota + 3 more
Source: ClinicalTrials.gov public record
Updated Dec 18, 2025 · Synced Oct 19, 2026, 10:24 PM EDT
Completed Not applicable Interventional

Fitness Trial in Adults With Sickle Cell Disease (SCD Fit): A Feasibility Study

NCT04772391
View directory record Official record
Conditions
Cardiovascular Diseases, Sickle Cell Disease, Exercise
Interventions
Exercise Sessions
Other
Lead sponsor
Memorial Healthcare System
Other
Eligibility
18 Years and older
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2022
U.S. locations
1
States / cities
Hollywood, Florida
Source: ClinicalTrials.gov public record
Updated Dec 8, 2022 · Synced Oct 19, 2026, 10:24 PM EDT
Enrolling by invitation No phase listed Observational

Examining Developmental Outcomes of Children Diagnosed With CLN2 Disease

NCT03862274
View directory record Official record
Conditions
Batten Disease, CLN2, Neuronal Ceroid-Lipofuscinoses
Interventions
CLN2 Treatment
Other
Lead sponsor
Jessica Scherr
Other
Eligibility
Not listed
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2025
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Oct 24, 2025 · Synced Oct 19, 2026, 10:24 PM EDT
Not yet recruiting Not applicable Interventional

Di-PKD: A Pilot Trial of Dietary Intervention in Patients With Autosomal Dominant Polycystic Kidney Disease

NCT06786260
View directory record Official record
Conditions
Autosomal Dominant Polycystic Kidney Disease (ADPKD)
Interventions
Interventional Diet
Other
Lead sponsor
Loma Linda University
Other
Eligibility
18 Years to 65 Years
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2027
U.S. locations
1
States / cities
Loma Linda, California
Source: ClinicalTrials.gov public record
Updated Jan 30, 2026 · Synced Oct 19, 2026, 10:24 PM EDT