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ClinicalTrials.gov public records Last synced Oct 19, 2026, 5:57 PM EDT

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Showing 193–216 of 9,568 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Genetics Clear all
Recruiting No phase listed Observational

A Prospective Observational Study of Foam Sclerotherapy .

NCT04111692
View directory record Official record
Conditions
Autosomal Dominant Polycystic Kidney, Renal Cyst, Autosomal Dominant Polycystic Liver Disease, Hepatic Cyst
Interventions
Not listed
Lead sponsor
Mayo Clinic
Other
Eligibility
18 Years to 100 Years
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2027
U.S. locations
1
States / cities
Rochester, Minnesota
Source: ClinicalTrials.gov public record
Updated Jan 26, 2026 · Synced Oct 19, 2026, 5:57 PM EDT
Active, not recruiting Phase 3 Interventional

A Study of Upadacitinib in Adult Participants With Moderate-to-Severe Atopic Dermatitis and Inadequate Response to Dupilumab

NCT06389136
View directory record Official record
Conditions
Atopic Dermatitis
Interventions
Upadacitinib 15mg Dose, Dupilumab 300mg Dose, Upadacitinib 30mg Dose
Drug
Lead sponsor
AbbVie
Industry
Eligibility
18 Years to 63 Years
Enrollment
184 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2027
U.S. locations
84
States / cities
Birmingham, Alabama • Scottsdale, Arizona • Fayetteville, Arkansas + 70 more
Source: ClinicalTrials.gov public record
Updated Oct 16, 2026 · Synced Oct 19, 2026, 5:57 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Genetic Influences Between Cognitive Function and BMI

NCT03116334
View directory record Official record
Conditions
Obesity
Interventions
Not listed
Lead sponsor
Baylor College of Medicine
Other
Eligibility
7 Years to 15 Years
Enrollment
869 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2009 – 2016
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Apr 18, 2017 · Synced Oct 19, 2026, 5:57 PM EDT
Completed Phase 2 Interventional Results available

Safety Study of Eteplirsen to Treat Advanced Stage Duchenne Muscular Dystrophy

NCT02286947
View directory record Official record
Conditions
Muscular Dystrophy, Duchenne
Interventions
Eteplirsen
Drug
Lead sponsor
Sarepta Therapeutics, Inc.
Industry
Eligibility
7 Years to 21 Years · Male only
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2018
U.S. locations
9
States / cities
Los Angeles, California • Sacramento, California • Iowa City, Iowa + 6 more
Source: ClinicalTrials.gov public record
Updated Mar 30, 2020 · Synced Oct 19, 2026, 5:57 PM EDT
Terminated No phase listed Observational

A Follow-up Study in Patients With Inherited Metabolic Disorders (IMD) Who Underwent Hematopoietic Stem Cell Transplantation (HSCT) With MGTA-456

NCT04008849
View directory record Official record
Conditions
Inherited Metabolic Disorders (IMD)
Interventions
Safety and efficacy assessments
Other
Lead sponsor
Magenta Therapeutics, Inc.
Industry
Eligibility
Up to 16 Years
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2020
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Mar 15, 2021 · Synced Oct 19, 2026, 5:57 PM EDT
Terminated Phase 2 Interventional Results available

A Study of Belcesiran in Patients With AATLD

NCT04764448
View directory record Official record
Conditions
Alpha 1-Antitrypsin Deficiency
Interventions
Belcesiran, Placebo
Drug · Other
Lead sponsor
Dicerna Pharmaceuticals, Inc., a Novo Nordisk company
Industry
Eligibility
18 Years to 75 Years
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2024
U.S. locations
3
States / cities
La Jolla, California • Gainesville, Florida • Charleston, South Carolina
Source: ClinicalTrials.gov public record
Updated Apr 7, 2026 · Synced Oct 19, 2026, 5:57 PM EDT
Recruiting No phase listed Observational

Characteristics of Patients With Recessive Dystrophic Epidermolysis Bullosa

NCT01019148
View directory record Official record
Conditions
Epidermolysis Bullosa Dystrophica
Interventions
Not listed
Lead sponsor
Stanford University
Other
Eligibility
Not listed
Enrollment
70 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2029
U.S. locations
1
States / cities
Stanford, California
Source: ClinicalTrials.gov public record
Updated Apr 24, 2026 · Synced Oct 19, 2026, 5:57 PM EDT
Completed Phase 4 Interventional

D-Cycloserine for Serine Palmitoyltransferase Inhibition

NCT07542548
View directory record Official record
Conditions
Hereditary Spastic Paraplegia
Interventions
D-cycloserine
Drug
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
6 Years to 6 Years · Female only
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Apr 21, 2026 · Synced Oct 19, 2026, 5:57 PM EDT
Completed Phase 2 Interventional Results available

High Dose Vitamin D for Sickle Cell Disease

NCT01331148
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Vitamin D, Placebo
Drug · Other
Lead sponsor
Wake Forest University Health Sciences
Other
Eligibility
7 Years to 21 Years
Enrollment
46 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2013
U.S. locations
1
States / cities
Atlanta, Georgia
Source: ClinicalTrials.gov public record
Updated Aug 9, 2022 · Synced Oct 19, 2026, 5:57 PM EDT
Completed Phase 1Phase 2 Interventional

Study of Gene Therapy Using a Lentiviral Vector to Treat X-linked Chronic Granulomatous Disease

NCT02234934
View directory record Official record
Conditions
Granulomatous Disease, Chronic, X-linked
Interventions
Lentiviral G1XCGD Gene Therapy
Biological
Lead sponsor
University of California, Los Angeles
Other
Eligibility
23 Months and older · Male only
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2024
U.S. locations
3
States / cities
Los Angeles, California • Bethesda, Maryland • Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated May 2, 2025 · Synced Oct 19, 2026, 5:57 PM EDT
Completed Phase 2 Interventional

The Nutritional Supplement Phosphatidylserine in Patients With Familial Dysautonomia

NCT02276716
View directory record Official record
Conditions
Familial Dysautonomia
Interventions
Phosphatidylserine
Dietary Supplement
Lead sponsor
NYU Langone Health
Other
Eligibility
12 Years and older
Enrollment
26 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2019
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Feb 21, 2021 · Synced Oct 19, 2026, 5:57 PM EDT
Recruiting Phase 2 Interventional Accepts healthy volunteers

Allo HSCT for High Risk Hemoglobinopathies

NCT06872333
View directory record Official record
Conditions
Graft Failure, Sickle Cell Disease, Hemoglobinopathies
Interventions
Alemtuzumab, Total Body Irradiation, Cell Infusion, Thymoglobulin, Fludarabine, Busulfan, Cyclophosphamide, Sirolimus, Tacrolimus, Mycophenolate Mofetil, Plerixafor (mozobil)
Drug · Radiation · Biological
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
Up to 55 Years
Enrollment
62 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2024 – 2032
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Jun 4, 2026 · Synced Oct 19, 2026, 5:57 PM EDT
Recruiting Phase 1Phase 2 Interventional

A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of NXT007 in Persons With Severe or Moderate Hemophilia A

NCT05987449
View directory record Official record
Conditions
Hemophilia A
Interventions
NXT007
Drug
Lead sponsor
Hoffmann-La Roche
Industry
Eligibility
2 Years to 59 Years · Male only
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2033
U.S. locations
4
States / cities
Sacramento, California • Washington D.C., District of Columbia • Indianapolis, Indiana + 1 more
Source: ClinicalTrials.gov public record
Updated Oct 8, 2026 · Synced Oct 19, 2026, 5:57 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Studies of Patients With Skin Disease, Patients With Neurological Degenerations, and Normal Volunteers

NCT00001164
View directory record Official record
Conditions
Healthy, Muscular Dystrophy, Retinal Degeneration, Skin Diseases, Virus Diseases
Interventions
Not listed
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
Not listed
Healthy volunteers
Accepts healthy volunteers
Timeline
1978 – 2000
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 5, 2008 · Synced Oct 19, 2026, 5:57 PM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

A Study To Evaluate the Relative Bioavailability, Food Effect, and Dose Proportionality of a Granule Formulation of Vanzacaftor/Tezacaftor/Deutivacaftor(VNZ/TEZ/D-IVA)

NCT06299709
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
VNZ/TEZ/D-IVA
Drug
Lead sponsor
Vertex Pharmaceuticals Incorporated
Industry
Eligibility
18 Years to 55 Years
Enrollment
34 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2024
U.S. locations
1
States / cities
Tempe, Arizona
Source: ClinicalTrials.gov public record
Updated Sep 26, 2025 · Synced Oct 19, 2026, 5:57 PM EDT
Recruiting No phase listed Observational

ARPKD Database Study

NCT01401998
View directory record Official record
Conditions
Hepato/Renal Fibrocystic Disease, Autosomal Recessive Polycystic Kidney Disease, Joubert Syndrome, Bardet Biedl Syndrome, Meckel-Gruber Syndrome, Congenital Hepatic Fibrosis, Caroli Syndrome, Oro-Facial-Digital Syndrome Type I, Nephronophthisis, Glomerulocystic Kidney Disease
Interventions
Not listed
Lead sponsor
Children's Hospital of Philadelphia
Other
Eligibility
Up to 18 Years
Enrollment
200 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2030
U.S. locations
6
States / cities
Washington D.C., District of Columbia • Atlanta, Georgia • Boston, Massachusetts + 3 more
Source: ClinicalTrials.gov public record
Updated Jun 15, 2026 · Synced Oct 19, 2026, 5:57 PM EDT
Completed Phase 2 Interventional

VoIP Delivered Behavior Therapy for Tourette Syndrome

NCT02247206
View directory record Official record
Conditions
Tourette Syndrome, Chronic Tic Disorder
Interventions
Behavior Therapy for Tics (CBIT)
Behavioral
Lead sponsor
University of Wisconsin, Milwaukee
Other
Eligibility
8 Years to 17 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2013
U.S. locations
1
States / cities
Milwaukee, Wisconsin
Source: ClinicalTrials.gov public record
Updated Sep 23, 2014 · Synced Oct 19, 2026, 5:57 PM EDT
Completed Phase 2 Interventional Results available

Safety and Efficacy of Intravitreal Injection of Human Retinal Progenitor Cells in Adults With Retinitis Pigmentosa

NCT03073733
View directory record Official record
Conditions
Retinitis Pigmentosa
Interventions
human retinal progenitor cells, Mock injection
Biological · Other
Lead sponsor
jCyte, Inc
Industry
Eligibility
18 Years and older
Enrollment
84 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2020
U.S. locations
3
States / cities
Irvine, California • Los Angeles, California • Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Jun 18, 2024 · Synced Oct 19, 2026, 5:57 PM EDT
Not listed Phase 4 Interventional Accepts healthy volunteers

Naproxen Pharmacogenetic Study (Project 1, Aim 2)

NCT04449471
View directory record Official record
Conditions
Pharmacogenetics
Interventions
Naproxen tablet
Drug
Lead sponsor
University of Washington
Other
Eligibility
18 Years and older
Enrollment
30 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2016 – 2022
U.S. locations
1
States / cities
Portland, Oregon
Source: ClinicalTrials.gov public record
Updated Jun 29, 2020 · Synced Oct 19, 2026, 5:57 PM EDT
Active, not recruiting No phase listed Observational Accepts healthy volunteers

Genetic and Environmental Risk Factors Related to Esophageal Cancer

NCT01035398
View directory record Official record
Conditions
Esophageal Cancer
Interventions
Not listed
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
18 Years to 100 Years
Enrollment
1,200 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2000 – 2028
U.S. locations
2
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Oct 7, 2026 · Synced Oct 19, 2026, 5:57 PM EDT
Active, not recruiting Phase 1Phase 2 Interventional

Study of EN-374 Gene Therapy in Participants With X-Linked Chronic Granulomatous Disease

NCT06876363
View directory record Official record
Conditions
X-Linked Chronic Granulomatous Disease
Interventions
EN-374
Genetic
Lead sponsor
Ensoma
Industry
Eligibility
3 Months and older · Male only
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
8
States / cities
Los Angeles, California • San Francisco, California • St. Petersburg, Florida + 5 more
Source: ClinicalTrials.gov public record
Updated Aug 21, 2026 · Synced Oct 19, 2026, 5:57 PM EDT
Completed Phase 4 Interventional

Response of Children With Atopic Dermatitis (Eczema) to Eucrisa

NCT04023084
View directory record Official record
Conditions
Atopic Dermatitis, Eczema
Interventions
Crisaborole
Drug
Lead sponsor
Ann & Robert H Lurie Children's Hospital of Chicago
Other
Eligibility
4 Months to 17 Years
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2022
U.S. locations
1
States / cities
Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Jul 19, 2022 · Synced Oct 19, 2026, 5:57 PM EDT
Recruiting No phase listed Observational

Defining the Natural History of Squamous Cell Carcinoma in Fanconi Anemia

NCT05687149
View directory record Official record
Conditions
Fanconi Anemia, Inherited Bone Marrow Failure Syndrome
Interventions
Not listed
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
8 Years to 90 Years
Enrollment
200 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2035
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 1, 2026 · Synced Oct 19, 2026, 5:57 PM EDT
Completed Phase 3 Interventional Results available

A Phase 3 Study of VX-445 Combination Therapy in Subjects With Cystic Fibrosis Heterozygous for the F508del Mutation and a Minimal Function Mutation (F/MF)

NCT03525444
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
VX-445/TEZ/IVA, IVA, Placebo
Drug
Lead sponsor
Vertex Pharmaceuticals Incorporated
Industry
Eligibility
12 Years and older
Enrollment
405 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2019
U.S. locations
53
States / cities
Tucson, Arizona • Little Rock, Arkansas • Long Beach, California + 48 more
Source: ClinicalTrials.gov public record
Updated May 19, 2020 · Synced Oct 19, 2026, 5:57 PM EDT