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ClinicalTrials.gov public records Last synced Oct 29, 2026, 6:01 PM EDT

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Showing 1489–1512 of 8,017 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Genetics/Birth Defects Clear all
Active, not recruiting Phase 2Phase 3 Interventional

Open Label Extension to Assess the Long-Term Safety and Tolerability of ZYN002 in Children and Adolescents With FXS

NCT03802799
View directory record Official record
Conditions
Fragile X Syndrome
Interventions
ZYN002 - Cannabidiol Transdermal Gel
Drug
Lead sponsor
Zynerba Pharmaceuticals, Inc.
Industry
Eligibility
3 Years to 29 Years
Enrollment
476 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2026
U.S. locations
19
States / cities
Phoenix, Arizona • Culver City, California • Irvine, California + 16 more
Source: ClinicalTrials.gov public record
Updated May 27, 2026 · Synced Oct 29, 2026, 6:01 PM EDT
Completed Phase 2 Interventional Results available

Study to Evaluate the Efficacy, Safety and Pharmacokinetics of ASB17061 Capsules in Adult Subjects With Atopic Dermatitis

NCT01756898
View directory record Official record
Conditions
Atopic Dermatitis
Interventions
5 mg ASB17061, Placebo, 10 mg ASB17061, 20 mg ASB17061
Drug
Lead sponsor
Daiichi Sankyo
Industry
Eligibility
18 Years to 65 Years
Enrollment
370 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2014
U.S. locations
40
States / cities
Birmingham, Alabama • Phoenix, Arizona • Rogers, Arkansas + 35 more
Source: ClinicalTrials.gov public record
Updated Feb 16, 2021 · Synced Oct 29, 2026, 6:01 PM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Skin Microbial Ecology in Atopic Dermatitis

NCT04170244
View directory record Official record
Conditions
Atopic Dermatitis, Psoriasis, Healthy
Interventions
AD subject visit sampling procedures, PS subject visit sampling procedures, Healthy control visit sampling procedures
Other
Lead sponsor
University of Rochester
Other
Eligibility
13 Years and older
Enrollment
240 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2019 – 2027
U.S. locations
1
States / cities
Rochester, New York
Source: ClinicalTrials.gov public record
Updated Jul 14, 2026 · Synced Oct 29, 2026, 6:01 PM EDT
Terminated No phase listed Observational Accepts healthy volunteers

Establishment of Functional MRI Imaging Parameters for Use in the Evaluation of Sickle Cell Disease

NCT02445144
View directory record Official record
Conditions
Sickle Cell Anemia
Interventions
Not listed
Lead sponsor
Albert Einstein College of Medicine
Other
Eligibility
18 Years to 55 Years
Enrollment
26 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2014 – 2020
U.S. locations
2
States / cities
The Bronx, New York
Source: ClinicalTrials.gov public record
Updated Dec 14, 2020 · Synced Oct 29, 2026, 6:01 PM EDT
Active, not recruiting Phase 1 Interventional Results available

Interleukin-15 Armored Glypican 3-specific Chimeric Antigen Receptor Expressed in T Cells for Pediatric Solid Tumors

NCT04377932
View directory record Official record
Conditions
Liver Cancer, Rhabdomyosarcoma, Malignant Rhabdoid Tumor, Liposarcoma, Wilms Tumor, Yolk Sac Tumor
Interventions
AGAR T cells
Genetic
Lead sponsor
Baylor College of Medicine
Other
Eligibility
1 Year to 21 Years
Enrollment
14 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2040
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Jan 21, 2026 · Synced Oct 29, 2026, 6:01 PM EDT
Completed No phase listed Observational

A Natural History Study of Canavan Disease

NCT02851563
View directory record Official record
Conditions
Canavan Disease
Interventions
Not listed
Lead sponsor
NYU Langone Health
Other
Eligibility
Up to 99 Years
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2019
U.S. locations
2
States / cities
Boston, Massachusetts • New York, New York
Source: ClinicalTrials.gov public record
Updated Nov 1, 2019 · Synced Oct 29, 2026, 6:01 PM EDT
Completed Phase 1 Interventional Accepts healthy volunteers Results available

Study Of PF-06817024 In Healthy Subjects, In Patients With Chronic Rhinosinusitis With Nasal Polyps And in Patients With Atopic Dermatitis

NCT02743871
View directory record Official record
Conditions
Healthy, Chronic Rhinosinusitis With Nasal Polyps, Atopic Dermatitis
Interventions
PF-06817024, Placebo for PF-06817024
Biological · Other
Lead sponsor
Pfizer
Industry
Eligibility
18 Years to 75 Years
Enrollment
97 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2016 – 2021
U.S. locations
20
States / cities
Sacramento, California • New Haven, Connecticut • Shelton, Connecticut + 12 more
Source: ClinicalTrials.gov public record
Updated May 19, 2022 · Synced Oct 29, 2026, 6:01 PM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

Safety and Tolerability of XmAb®7195 in Adult Healthy Volunteers and Adult Subjects With a History of Allergic Rhinitis and/or Allergic Conjunctivitis and/or Atopic Dermatitis

NCT02148744
View directory record Official record
Conditions
Allergic Rhinitis, Allergic Conjunctivitis, Atopic Dermatitis
Interventions
XmAb7195, Placebo
Biological
Lead sponsor
Xencor, Inc.
Industry
Eligibility
18 Years to 50 Years
Enrollment
72 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2014 – 2015
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Jan 24, 2017 · Synced Oct 29, 2026, 6:01 PM EDT
Completed Early Phase 1 Interventional

Rigosertib in Patients With Recessive Dystrophic Epidermolysis Bullosa Associated SCC

NCT04177498
View directory record Official record
Conditions
Recessive Dystrophic Epidermolysis Bullosa
Interventions
Rigosertib Sodium, Quality-of-Life Assessment
Drug · Other
Lead sponsor
Thomas Jefferson University
Other
Eligibility
18 Years to 79 Years
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2025
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Aug 1, 2025 · Synced Oct 29, 2026, 6:01 PM EDT
Completed Phase 1Phase 2 Interventional Results available

A Study of JR-171 in Patients With Mucopolysaccharidosis I

NCT04227600
View directory record Official record
Conditions
Mucopolysaccharidosis I
Interventions
JR-171 (lepunafusp alfa)
Drug
Lead sponsor
JCR Pharmaceuticals Co., Ltd.
Industry
Eligibility
0 Years and older
Enrollment
18 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2022
U.S. locations
1
States / cities
Oakland, California
Source: ClinicalTrials.gov public record
Updated Mar 19, 2025 · Synced Oct 29, 2026, 6:01 PM EDT
Completed Phase 1 Interventional

Efficacy of Albuterol in the Treatment of Congenital Myasthenic Syndromes

NCT01203592
View directory record Official record
Conditions
Congenital Myasthenic Syndrome
Interventions
Albuterol
Drug
Lead sponsor
Mayo Clinic
Other
Eligibility
2 Years and older
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2013
U.S. locations
1
States / cities
Rochester, Minnesota
Source: ClinicalTrials.gov public record
Updated Jan 1, 2016 · Synced Oct 29, 2026, 6:01 PM EDT
Recruiting Phase 1 Interventional

Phase 1 Study of C.001 in Retinal Degeneration

NCT07594236
View directory record Official record
Conditions
Geographic Atrophy, Stargardt Disease, RPE-mediated Maculopathy, Age Related Macular Degeneration
Interventions
Injection
Biological
Lead sponsor
Cellio Therapeutics Inc
Industry
Eligibility
18 Years and older
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2029
U.S. locations
3
States / cities
Beverly Hills, California • Palo Alto, California • Sacramento, California
Source: ClinicalTrials.gov public record
Updated Aug 17, 2026 · Synced Oct 29, 2026, 6:01 PM EDT
Enrolling by invitation Phase 3 Interventional

An Extension Study of JR-141 to Evaluate the Long-term Safety and Efficacy in MPS II (Hunter Syndrome) Subjects

NCT05594992
View directory record Official record
Conditions
Mucopolysaccharidosis II
Interventions
JR-141
Drug
Lead sponsor
JCR Pharmaceuticals Co., Ltd.
Industry
Eligibility
Not listed
Enrollment
80 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2028
U.S. locations
2
States / cities
Oakland, California • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jan 21, 2026 · Synced Oct 29, 2026, 6:01 PM EDT
Completed Phase 1 Interventional

Bioequivalence of Two Somatropin Products (Norditropin® Versus Genotropin®) in Healthy Adult Volunteers

NCT01401244
View directory record Official record
Conditions
Genetic Disorder, Prader-Willi Syndrome, Growth Disorder, Idiopathic Short Stature, Healthy
Interventions
somatropin
Drug
Lead sponsor
Novo Nordisk A/S
Industry
Eligibility
18 Years to 40 Years
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011
U.S. locations
1
States / cities
Evansville, Indiana
Source: ClinicalTrials.gov public record
Updated Feb 24, 2017 · Synced Oct 29, 2026, 6:01 PM EDT
Completed Phase 3 Interventional Results available

Treatment of Graves' Orbitopathy to Reduce Proptosis With Teprotumumab Infusions in an Open-Label Clinical Extension Study

NCT03461211
View directory record Official record
Conditions
Thyroid Eye Disease
Interventions
Teprotumumab
Biological
Lead sponsor
Amgen
Industry
Eligibility
18 Years to 80 Years
Enrollment
51 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2021
U.S. locations
8
States / cities
Beverly Hills, California • Los Angeles, California • Miami, Florida + 5 more
Source: ClinicalTrials.gov public record
Updated Jun 28, 2024 · Synced Oct 29, 2026, 6:01 PM EDT
Completed Phase 2 Interventional Results available

A 48-Week, Open Label, Study to Evaluate the Efficacy and Safety of AMONDYS 45, EXONDYS 51, VYONDYS 53 in Subjects With DuchenneMuscular Dystrophy Carrying Eligible DMD Duplications.

NCT04179409
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Amondys 45, Exondys 51, Vyondys 53
Drug
Lead sponsor
Kevin Flanigan
Other
Eligibility
6 Months and older · Male only
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2023
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Oct 26, 2023 · Synced Oct 29, 2026, 6:01 PM EDT
Completed Phase 3 Interventional Results available

Hyper- and Hypokalemic Periodic Paralysis Study

NCT00494507
View directory record Official record
Conditions
Hyperkalemic Periodic Paralysis, Hypokalemic Periodic Paralysis
Interventions
Dichlorphenamide (double-blind), Placebo (double-blind), Dichlorphenamide (open-label)
Drug
Lead sponsor
University of Rochester
Other
Eligibility
18 Years and older
Enrollment
71 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2013
U.S. locations
10
States / cities
Los Angeles, California • San Francisco, California • Kansas City, Kansas + 7 more
Source: ClinicalTrials.gov public record
Updated Jun 14, 2017 · Synced Oct 29, 2026, 6:01 PM EDT
Completed No phase listed Observational

Developing Newborn Screening for Infants With Primary Immunodeficiency

NCT00113464
View directory record Official record
Conditions
Immune System Diseases
Interventions
Not listed
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
Not listed
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2007
U.S. locations
1
States / cities
New Haven, Connecticut
Source: ClinicalTrials.gov public record
Updated Jul 2, 2017 · Synced Oct 29, 2026, 6:01 PM EDT
Completed Phase 2Phase 3 Interventional Results available

Safety, Pharmacokinetics and Efficacy of Dupilumab in Patients ≥6 Months to <6 Years With Moderate-to-Severe Atopic Dermatitis (Liberty AD PRESCHOOL)

NCT03346434
View directory record Official record
Conditions
Dermatitis, Atopic
Interventions
Dupilumab, Matching placebo
Drug
Lead sponsor
Regeneron Pharmaceuticals
Industry
Eligibility
6 Months to 5 Years
Enrollment
202 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2021
U.S. locations
33
States / cities
Birmingham, Alabama • Gilbert, Arizona • Long Beach, California + 30 more
Source: ClinicalTrials.gov public record
Updated Jul 28, 2022 · Synced Oct 29, 2026, 6:01 PM EDT
Recruiting No phase listed Observational

A Personalized Surveillance and Intervention Protocol for Duodenal and Gastric Polyposis in Patients With Familial Adenomatous Polyposis

NCT04677998
View directory record Official record
Conditions
Familial Adenomatous Polyposis
Interventions
Personalized surveillance and intervention protocol
Procedure
Lead sponsor
Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)
Other
Eligibility
18 Years and older
Enrollment
1,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2027
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Feb 23, 2026 · Synced Oct 29, 2026, 6:01 PM EDT
Completed Not applicable Interventional

A Prospective and Retrospective Cohort Study in Patients With Chronic Forms of Acid Sphingomyelinase Deficiency (ASMD)

NCT04106544
View directory record Official record
Conditions
Sphingomyelin Lipidosis
Interventions
Investigational Procedures
Procedure
Lead sponsor
Sanofi
Industry
Eligibility
Not listed
Enrollment
84 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2023
U.S. locations
3
States / cities
Atlanta, Georgia • The Bronx, New York • Valhalla, New York
Source: ClinicalTrials.gov public record
Updated Aug 16, 2023 · Synced Oct 29, 2026, 6:01 PM EDT
Completed Not applicable Interventional Results available

Increasing Documentation and Disclosure of Sickle Cell Trait Status: An Implementation Science Approach

NCT05387564
View directory record Official record
Conditions
Sickle Cell Trait
Interventions
SCT Documentation and Disclosure Toolkit (SCT-DD)
Behavioral
Lead sponsor
Nemours Children's Clinic
Other
Eligibility
Not listed
Enrollment
114 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024
U.S. locations
1
States / cities
Wilmington, Delaware
Source: ClinicalTrials.gov public record
Updated Jan 7, 2026 · Synced Oct 29, 2026, 6:01 PM EDT
Completed Phase 4 Interventional Accepts healthy volunteers

Tadalafil in Becker Muscular Dystrophy

NCT01070511
View directory record Official record
Conditions
Becker Muscular Dystrophy
Interventions
Tadalafil, Placebo
Drug
Lead sponsor
Cedars-Sinai Medical Center
Other
Eligibility
18 Years to 55 Years · Male only
Enrollment
48 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2010 – 2012
U.S. locations
2
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Aug 20, 2013 · Synced Oct 29, 2026, 6:01 PM EDT
Terminated No phase listed Observational

Cardiovasculorenal Phenotyping in Fabry Disease Through Noninvasive Testing

NCT05699265
View directory record Official record
Conditions
Fabry Disease
Interventions
Measures of arterial stiffness and endothelial function, Ambulatory blood pressure monitoring, Cardiopulmonary exercise testing (CPET), Serum and urine biomarkers
Diagnostic Test
Lead sponsor
Children's Hospital Medical Center, Cincinnati
Other
Eligibility
8 Years and older
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2025
U.S. locations
1
States / cities
Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Jun 19, 2025 · Synced Oct 29, 2026, 6:01 PM EDT