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ClinicalTrials.gov public records Last synced Oct 20, 2026, 5:07 AM EDT

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Showing 145–168 of 8,004 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Genetics/Birth Defects Clear all
Not listed Not applicable Interventional

Study of an Intervention to Improve Problem List Accuracy and Use

NCT01105923
View directory record Official record
Conditions
Attention Deficit Disorder With Hyperactivity, Asthma, COPD, Breast Cancer, Coronary Artery Disease, Congestive Heart Failure, Diabetes, Glaucoma, Hemophilia, Hypertension, Hyperthyroidism, Hypothyroidism, Myasthenia Gravis, Osteoporosis, Osteopenia, Renal Failure, Renal Insufficiency, Sickle Cell Disease, Stroke
Interventions
MAPLE
Other
Lead sponsor
Brigham and Women's Hospital
Other
Eligibility
18 Years and older
Enrollment
140 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2017
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Feb 2, 2015 · Synced Oct 20, 2026, 5:07 AM EDT
Completed Phase 2 Interventional Results available

Pirfenidone to Treat Kidney Disease (Focal Segmental Glomerulosclerosis)

NCT00001959
View directory record Official record
Conditions
Fibrosis, Focal Glomerulosclerosis, Kidney Failure, Nephrotic Syndrome, Proteinuria
Interventions
Pirfenidone
Drug
Lead sponsor
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
NIH
Eligibility
18 Years and older
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1999 – 2008
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated May 26, 2014 · Synced Oct 20, 2026, 5:07 AM EDT
Completed Phase 3 Interventional Results available

Efficacy and Safety Study of DX-88 to Treat Acute Attacks of Hereditary Angioedema (HAE)

NCT00262080
View directory record Official record
Conditions
Hereditary Angioedema (HAE)
Interventions
ecallantide, Phosphate Buffer Saline (PBS),
Drug
Lead sponsor
Shire
Industry
Eligibility
10 Years and older
Enrollment
91 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2007
U.S. locations
1
States / cities
Wheaton, Maryland
Source: ClinicalTrials.gov public record
Updated Jun 11, 2021 · Synced Oct 20, 2026, 5:07 AM EDT
Terminated No phase listed Observational

Von Hippel-Lindau Disease Genetic Epidemiology Study

NCT00001803
View directory record Official record
Conditions
Von Hippel Lindau Disease, Kidney Neoplasms
Interventions
Not listed
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
13 Years to 100 Years
Enrollment
546 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1999 – 2020
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jan 13, 2020 · Synced Oct 20, 2026, 5:07 AM EDT
Completed Phase 1 Interventional

A Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of a Single Dose of SRP-5051 (Vesleteplirsen) in Patients With Duchenne Muscular Dystrophy (DMD)

NCT03375255
View directory record Official record
Conditions
Muscular Dystrophy, Duchenne
Interventions
SRP-5051
Drug
Lead sponsor
Sarepta Therapeutics, Inc.
Industry
Eligibility
12 Years and older · Male only
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2019
U.S. locations
7
States / cities
Sacramento, California • Gulf Breeze, Florida • Atlanta, Georgia + 4 more
Source: ClinicalTrials.gov public record
Updated Jul 6, 2022 · Synced Oct 20, 2026, 5:07 AM EDT
Completed Phase 3 Interventional Results available

Open Label, Phase III Study of NABI-IGIV 10% [Immune Globulin Intravenous(Human), 10%] In Subjects With Primary Immune Deficiency Disorders (PIDD)

NCT00538915
View directory record Official record
Conditions
Primary Immune Deficiency Disorders (PIDD)
Interventions
Nabi-IGIV 10% [Immune Globulin Intravenous (Human). 10%]
Biological
Lead sponsor
ADMA Biologics, Inc.
Industry
Eligibility
6 Years to 75 Years
Enrollment
63 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2009
U.S. locations
17
States / cities
Birmingham, Alabama • Phoenix, Arizona • Los Angeles, California + 13 more
Source: ClinicalTrials.gov public record
Updated Jul 30, 2021 · Synced Oct 20, 2026, 5:07 AM EDT
Completed Phase 1Phase 2 Interventional

Scotoma Reduction in AMD Patients Treated With Ranibizumab

NCT00467935
View directory record Official record
Conditions
Macular Degeneration
Interventions
intravitreal injection Lucentis (ranibizumab)
Drug
Lead sponsor
Retina Research Foundation
Other
Eligibility
50 Years to 90 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007
U.S. locations
1
States / cities
Slingerlands, New York
Source: ClinicalTrials.gov public record
Updated Jul 29, 2011 · Synced Oct 20, 2026, 5:07 AM EDT
Withdrawn Not applicable Interventional Accepts healthy volunteers

Evaluating Skin Barrier Dysfunction in Infants at High Risk of Atopy

NCT03089476
View directory record Official record
Conditions
Atopic Dermatitis, Eczema, Food Allergy
Interventions
Evaluating atopy in infants, Evaluating TEWL and STS in adults
Other
Lead sponsor
National Jewish Health
Other
Eligibility
34 Weeks to 50 Years
Healthy volunteers
Accepts healthy volunteers
Timeline
2017 – 2018
U.S. locations
1
States / cities
Denver, Colorado
Source: ClinicalTrials.gov public record
Updated Jun 12, 2018 · Synced Oct 20, 2026, 5:07 AM EDT
Recruiting Phase 3 Interventional

SAFE Study: Safety of aPCC Following Emicizumab Prophylaxis

NCT04563520
View directory record Official record
Conditions
Hemophilia A
Interventions
Emicizumab, FEIBA, rFVIIa
Drug
Lead sponsor
Emory University
Other
Eligibility
6 Years and older
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2027
U.S. locations
2
States / cities
Atlanta, Georgia
Source: ClinicalTrials.gov public record
Updated Aug 21, 2026 · Synced Oct 20, 2026, 5:07 AM EDT
Completed Phase 2 Interventional Results available

A Study Evaluating the Safety and Efficacy of VX-440 Combination Therapy in Subjects With Cystic Fibrosis

NCT02951182
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
TEZ, IVA, VX-440, Matched Placebo
Drug
Lead sponsor
Vertex Pharmaceuticals Incorporated
Industry
Eligibility
12 Years and older
Enrollment
74 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2017
U.S. locations
22
States / cities
Tucson, Arizona • Palo Alto, California • San Francisco, California + 18 more
Source: ClinicalTrials.gov public record
Updated Aug 28, 2020 · Synced Oct 20, 2026, 5:07 AM EDT
Completed Phase 1Phase 2 Interventional

A Study to Determine the Safety, Tolerability and Efficacy of KP-413 in Subjects With Atopic Dermatitis(AD)

NCT00932074
View directory record Official record
Conditions
Atopic Dermatitis
Interventions
KP-413 Ointment
Drug
Lead sponsor
Kaken Pharmaceutical
Industry
Eligibility
18 Years to 64 Years
Enrollment
58 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2010
U.S. locations
6
States / cities
Clinton Twp., Michigan • Portland, Oregon • Austin, Texas + 3 more
Source: ClinicalTrials.gov public record
Updated Apr 6, 2012 · Synced Oct 20, 2026, 5:07 AM EDT
Completed Phase 2 Interventional Results available

A Trial Evaluating Efficacy and Safety of Prophylactic Administration of Concizumab in Patients With Severe Haemophilia A Without Inhibitors

NCT03196297
View directory record Official record
Conditions
Haemostasis, Haemophilia A
Interventions
Concizumab, Turoctocog alfa
Drug
Lead sponsor
Novo Nordisk A/S
Industry
Eligibility
18 Years and older · Male only
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2020
U.S. locations
5
States / cities
Los Angeles, California • Indianapolis, Indiana • Oklahoma City, Oklahoma + 2 more
Source: ClinicalTrials.gov public record
Updated Nov 16, 2021 · Synced Oct 20, 2026, 5:07 AM EDT
Completed Phase 3 Interventional Results available

Efficacy and Safety of Rivipansel (GMI-1070) in the Treatment of Vaso-Occlusive Crisis in Hospitalized Subjects With Sickle Cell Disease

NCT02187003
View directory record Official record
Conditions
Anemia, Sickle Cell
Interventions
Rivipansel, Placebo
Drug · Other
Lead sponsor
Biossil Inc.
Industry
Eligibility
6 Years and older
Enrollment
345 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2019
U.S. locations
126
States / cities
Mobile, Alabama • Little Rock, Arkansas • Oakland, California + 40 more
Source: ClinicalTrials.gov public record
Updated Mar 11, 2025 · Synced Oct 20, 2026, 5:07 AM EDT
Not listed Phase 1 Interventional

Phase I Study of Amifostine in Patients With Bone Marrow Failure Related to Fanconi's Anemia

NCT00006127
View directory record Official record
Conditions
Fanconi's Anemia
Interventions
amifostine
Drug
Lead sponsor
Dana-Farber Cancer Institute
Other
Eligibility
2 Years and older
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2000
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Jun 24, 2005 · Synced Oct 20, 2026, 5:07 AM EDT
Completed No phase listed Observational

Goal Attainment Scaling (GAS) Sub-Study

NCT06484426
View directory record Official record
Conditions
Alzheimer's Disease in Down Syndrome
Interventions
Not listed
Lead sponsor
LuMind IDSC Foundation
Other
Eligibility
35 Years and older
Enrollment
46 participants
Timeline
2021 – 2024
U.S. locations
5
States / cities
San Diego, California • Park Ridge, Illinois • Baltimore, Maryland + 2 more
Source: ClinicalTrials.gov public record
Updated Jul 3, 2024 · Synced Oct 20, 2026, 5:07 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

NeuroSeq: A Prospective Trial to Evaluate the Diagnostic Yield of Whole Genome Sequencing (WGS) in Adult Neurology

NCT04170985
View directory record Official record
Conditions
Neurologic Deficits, Neurologic Disorder, Neurologic Abnormalities
Interventions
clinical Whole Genome Sequencing
Other
Lead sponsor
Illumina, Inc.
Industry
Eligibility
18 Years and older
Enrollment
160 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2019 – 2024
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated May 1, 2024 · Synced Oct 20, 2026, 5:07 AM EDT
Completed No phase listed Observational

Identifying Barriers and Strategies to Support Self-efficacy for Medication Adherence With Text Messaging

NCT02785848
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Interviews and Surveys
Other
Lead sponsor
Vanderbilt University
Other
Eligibility
12 Years to 70 Years
Enrollment
70 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2026
U.S. locations
1
States / cities
Nashville, Tennessee
Source: ClinicalTrials.gov public record
Updated Sep 23, 2026 · Synced Oct 20, 2026, 5:07 AM EDT
Completed Phase 2 Interventional Results available

A Study to Learn About The Study Medicine (PF-07038124) In Patients With Mild To Moderate Atopic Dermatitis Or Mild To Severe Plaque Psoriasis.

NCT05375955
View directory record Official record
Conditions
Atopic Dermatitis, Plaque Psoriasis
Interventions
PF-07038124 ointment 0.01%, Vehicle ointment, PF-07038124 ointment 0.03%, PF-07038124 ointment 0.06%
Drug
Lead sponsor
Pfizer
Industry
Eligibility
12 Years and older
Enrollment
263 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2023
U.S. locations
23
States / cities
Encinitas, California • Los Angeles, California • Cape Coral, Florida + 17 more
Source: ClinicalTrials.gov public record
Updated Oct 2, 2024 · Synced Oct 20, 2026, 5:07 AM EDT
Terminated Not applicable Interventional Results available

Naglazyme After Allo Transplant for Maroteaux-Lamy Syndrome

NCT02156674
View directory record Official record
Conditions
Maroteaux-Lamy Syndrome
Interventions
Naglazyme®
Drug
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
3 Years and older
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2019
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Dec 23, 2020 · Synced Oct 20, 2026, 5:07 AM EDT
Terminated No phase listed Observational

Down Syndrome Clinical Trials - Study of Alzheimer's Disease in Down Syndrome

NCT04149197
View directory record Official record
Conditions
Alzheimer's Disease in Down Syndrome
Interventions
Not listed
Lead sponsor
LuMind IDSC Foundation
Other
Eligibility
25 Years and older
Enrollment
252 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2024
U.S. locations
14
States / cities
Phoenix, Arizona • Irvine, California • La Jolla, California + 11 more
Source: ClinicalTrials.gov public record
Updated Jun 27, 2024 · Synced Oct 20, 2026, 5:07 AM EDT
Not listed No phase listed Observational Accepts healthy volunteers

PatientSpot Formerly Known as ArthritisPower

NCT03840928
View directory record Official record
Conditions
Rheumatoid Arthritis, Ankylosing Spondylitis, Fibromyalgia, Gout, Crohn Disease, Juvenile Idiopathic Arthritis, Lupus Erythematosus, Myositis, Osteoarthritis, Osteoporosis, Psoriasis, Psoriatic Arthritis, Scleroderma, Dermatomyositis, Inflammatory Bowel Diseases, Polymyositis, Axial Spondyloarthritis, Diffuse Idiopathic Skeletal Hyperostosis, Polymyalgia Rheumatica, Giant Cell Arteritis, Temporal Arteritis, Wegener, Relapsing Polychondritis, Undifferentiated Connective Tissue Disease, Spinal Cord Injuries, Alzheimer Disease, Amyotrophic Lateral Sclerosis, Ataxia, Bell Palsy, Brain Tumor, Cerebral Aneurysm, Epilepsy, Guillain-Barre Syndrome, Headache, Head Injury, Hydrocephalus, Lumbar Disc Disease, Meningitis, Multiple Sclerosis, Muscular Dystrophy, Neurocutaneous Syndromes, Parkinson Disease, Stroke, Cluster Headache, Tension-Type Headache, Chronic Obstructive Pulmonary Disease, Asthma, Lung Cancer, Cystic Fibrosis, Sleep Apnea, Eczema, Alopecia, Chronic Inflammation, Unstable Angina, Heart Attack, Heart Failure, Arrythmia, Valve Heart Disease, High Blood Pressure, Congenital Heart Disease, Peripheral Arterial Disease, Diabetes, Chronic Liver Disease, Obesity
Interventions
Not listed
Lead sponsor
Global Healthy Living Foundation
Other
Eligibility
19 Years and older
Enrollment
40,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2015 – 2025
U.S. locations
1
States / cities
Upper Nyack, New York
Source: ClinicalTrials.gov public record
Updated Dec 13, 2023 · Synced Oct 20, 2026, 5:07 AM EDT
Completed Phase 3 Interventional Results available

Effect of Statin Therapy on Disease Progression in Autosomal Dominant Polycystic Kidney Disease (ADPKD)

NCT00456365
View directory record Official record
Conditions
Polycystic Kidney, Autosomal Dominant
Interventions
pravastatin, Placebo
Drug
Lead sponsor
University of Colorado, Denver
Other
Eligibility
8 Years to 22 Years
Enrollment
110 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2012
U.S. locations
1
States / cities
Denver, Colorado
Source: ClinicalTrials.gov public record
Updated Mar 9, 2018 · Synced Oct 20, 2026, 5:07 AM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

NIAID Centralized Sequencing Protocol

NCT03206099
View directory record Official record
Conditions
Atopy, Primary Immunodeficiency, Autoimmunity, Autoinflammation
Interventions
Not listed
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
1 Day to 100 Years
Enrollment
20,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2017 – 2029
U.S. locations
2
States / cities
Washington D.C., District of Columbia • Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 2, 2026 · Synced Oct 20, 2026, 5:07 AM EDT
Completed Not applicable Interventional

A Comparison Between Full Thickness and Partial Thickness Corneal Transplantation for Corneal Edema

NCT00346138
View directory record Official record
Conditions
Fuchs Dystrophy, Corneal Edema
Interventions
Corneal transplantation
Procedure
Lead sponsor
Mayo Clinic
Other
Eligibility
21 Years and older
Enrollment
29 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2009
U.S. locations
1
States / cities
Rochester, Minnesota
Source: ClinicalTrials.gov public record
Updated Jan 15, 2010 · Synced Oct 20, 2026, 5:07 AM EDT