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ClinicalTrials.gov public records Last synced Oct 15, 2026, 3:22 PM EDT

Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.

Showing 505–528 of 9,409 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Genetic Disease Clear all
Completed Phase 2 Interventional Results available

Effect of Double Dose of Alpha 1-antitrypsin Augmentation Therapy on Lung Inflammation.

NCT01669421
View directory record Official record
Conditions
Alpha 1 Antitrypsin Deficiency
Interventions
Alpha-1 Antitrypsin (human)
Drug
Lead sponsor
Michael Campos, MD
Other
Eligibility
18 Years to 75 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2016
U.S. locations
1
States / cities
Miami, Florida
Source: ClinicalTrials.gov public record
Updated May 8, 2019 · Synced Oct 15, 2026, 3:22 PM EDT
Completed No phase listed Observational

Patients Treated for Wiskott-Aldrich Syndrome (WAS) Since 1990

NCT02064933
View directory record Official record
Conditions
Wiskott-Aldrich Syndrome
Interventions
Not listed
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
Male only
Enrollment
305 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2019
U.S. locations
38
States / cities
Birmingham, Alabama • Phoenix, Arizona • Los Angeles, California + 33 more
Source: ClinicalTrials.gov public record
Updated Aug 10, 2020 · Synced Oct 15, 2026, 3:22 PM EDT
Completed Phase 3 Interventional Results available

Therapeutic Potential for Aldosterone Inhibition in Duchenne Muscular Dystrophy

NCT02354352
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Eplerenone, Spironolactone
Drug
Lead sponsor
Ohio State University
Other
Eligibility
7 Years and older · Male only
Enrollment
52 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2018
U.S. locations
6
States / cities
Los Angeles, California • Aurora, Colorado • Kansas City, Kansas + 3 more
Source: ClinicalTrials.gov public record
Updated Oct 7, 2019 · Synced Oct 15, 2026, 3:22 PM EDT
Active, not recruiting Phase 2 Interventional

A Study of Barzolvolimab in Patients With Atopic Dermatitis

NCT06727552
View directory record Official record
Conditions
Atopic Dermatitis
Interventions
Barzolvolimab, Matching placebo
Biological · Drug
Lead sponsor
Celldex Therapeutics
Industry
Eligibility
18 Years and older
Enrollment
131 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
33
States / cities
Birmingham, Alabama • Beverly Hills, California • Burbank, California + 28 more
Source: ClinicalTrials.gov public record
Updated Aug 11, 2026 · Synced Oct 15, 2026, 3:22 PM EDT
Completed No phase listed Observational

Positive Exposure: A Photography and Video Intervention for Individuals With Craniofacial Differences

NCT00340964
View directory record Official record
Conditions
Craniofacial Differences
Interventions
Not listed
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
12 Years to 20 Years
Enrollment
44 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2014
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Dec 12, 2019 · Synced Oct 15, 2026, 3:22 PM EDT
Completed Phase 2 Interventional Results available

A Study of Topical NS2 Cream to Treat Ichthyosis in Sjögren-Larsson Syndrome (SLS)

NCT02402309
View directory record Official record
Conditions
Sjögren-Larsson Syndrome
Interventions
Active topical NS2 1% dermatologic cream, Vehicle placebo 0.0% NS2 dermatologic cream
Drug
Lead sponsor
Aldeyra Therapeutics, Inc.
Industry
Eligibility
6 Years and older
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2016
U.S. locations
3
States / cities
Omaha, Nebraska • Hershey, Pennsylvania • Fairfax, Virginia
Source: ClinicalTrials.gov public record
Updated Dec 13, 2022 · Synced Oct 15, 2026, 3:22 PM EDT
Completed Not applicable Interventional

Tolerability of Inhaled Hypertonic Saline in Infants With Cystic Fibrosis

NCT00546663
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
inhaled 7% hypertonic saline (HS)
Drug
Lead sponsor
CF Therapeutics Development Network Coordinating Center
Network
Eligibility
12 Months to 30 Months
Enrollment
19 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2008
U.S. locations
2
States / cities
Chapel Hill, North Carolina • Seattle, Washington
Source: ClinicalTrials.gov public record
Updated Aug 24, 2011 · Synced Oct 15, 2026, 3:22 PM EDT
Completed No phase listed Observational

GM1 and GM2 Gangliosidosis PROspective Neurological Disease TrajectOry Study (PRONTO)

NCT05109793
View directory record Official record
Conditions
GM1 Gangliosidosis, Sandhoff Disease, Tay-Sachs Disease
Interventions
Not listed
Lead sponsor
Azafaros B.V.
Industry
Eligibility
2 Years to 20 Years
Enrollment
31 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2025
U.S. locations
2
States / cities
Oakland, California • Rochester, Minnesota
Source: ClinicalTrials.gov public record
Updated Sep 29, 2025 · Synced Oct 15, 2026, 3:22 PM EDT
Active, not recruiting Not applicable Interventional Accepts healthy volunteers

The Electronic Medical Records and GEnomics (eMERGE) Network Genomic Risk Assessment

NCT05277116
View directory record Official record
Conditions
Genetic Disease
Interventions
Genome Informed Risk Assessment (GIRA) report
Other
Lead sponsor
Vanderbilt University Medical Center
Other
Eligibility
3 Years to 75 Years
Enrollment
26,877 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2022 – 2030
U.S. locations
12
States / cities
Birmingham, Alabama • Tempe, Arizona • Chicago, Illinois + 7 more
Source: ClinicalTrials.gov public record
Updated Aug 18, 2026 · Synced Oct 15, 2026, 3:22 PM EDT
Terminated Phase 4 Interventional Results available

Use of Tirosint®-SOL or Tablet Formulations of Levothyroxine in Pediatric Patients With Congenital Hypothyroidism (CH)

NCT05228184
View directory record Official record
Conditions
Congenital Hypothyroidism
Interventions
Tirosint®-SOL, Levothyroxine Sodium
Drug
Lead sponsor
IBSA Institut Biochimique SA
Industry
Eligibility
1 Day to 9 Months
Enrollment
34 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2025
U.S. locations
10
States / cities
Los Angeles, California • Orange, California • San Francisco, California + 7 more
Source: ClinicalTrials.gov public record
Updated Jun 26, 2026 · Synced Oct 15, 2026, 3:22 PM EDT
Completed Phase 3 Interventional Results available

Trientine Tetrahydrochloride (TETA 4HCL) for the Treatment of Wilson's Disease

NCT03539952
View directory record Official record
Conditions
Wilson Disease
Interventions
Penicillamine (D1-W12), TETA 4HCL (W12-60), Penicillamine (W12-W60), TETA 4HCL (60-<W108)
Drug
Lead sponsor
Orphalan
Industry
Eligibility
18 Years to 75 Years
Enrollment
77 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2022
U.S. locations
1
States / cities
New Haven, Connecticut
Source: ClinicalTrials.gov public record
Updated Jul 2, 2025 · Synced Oct 15, 2026, 3:22 PM EDT
Completed No phase listed Observational

A Study of Potential Treatment-Responsive Biomarkers and Clinical Outcomes in Hunter Syndrome

NCT04007536
View directory record Official record
Conditions
Mucopolysaccharidosis II
Interventions
No Intervention
Other
Lead sponsor
Denali Therapeutics Inc.
Industry
Eligibility
Up to 30 Years
Enrollment
18 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2024
U.S. locations
3
States / cities
Oakland, California • Chapel Hill, North Carolina • Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jun 10, 2024 · Synced Oct 15, 2026, 3:22 PM EDT
Withdrawn Not applicable Interventional

Ranibizumab for Neovascularization in Sickle Cell Retinopathy

NCT00618644
View directory record Official record
Conditions
Sickle Cell Anemia, Retinopathy
Interventions
Ranibizumab
Drug
Lead sponsor
Kresge Eye Institute
Other
Eligibility
18 Years and older
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2011
U.S. locations
1
States / cities
Detroit, Michigan
Source: ClinicalTrials.gov public record
Updated Sep 27, 2012 · Synced Oct 15, 2026, 3:22 PM EDT
Recruiting No phase listed Observational

Trial-Ready Cohort-Down Syndrome (TRC-DS)

NCT04165109
View directory record Official record
Conditions
Down Syndrome, Alzheimer Disease, Dementia
Interventions
Not listed
Lead sponsor
University of Southern California
Other
Eligibility
25 Years to 55 Years
Enrollment
550 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2027
U.S. locations
18
States / cities
Phoenix, Arizona • Orange, California • Aurora, Colorado + 15 more
Source: ClinicalTrials.gov public record
Updated May 18, 2026 · Synced Oct 15, 2026, 3:22 PM EDT
Completed No phase listed Observational

A Study of Mucopolysaccharidosis Type IIIB (MPS IIIB)

NCT02493998
View directory record Official record
Conditions
Mucopolysaccharidosis Type IIIB, Mucopolysaccharidosis Type 3 B, MPS III B, MPS 3 B
Interventions
Not listed
Lead sponsor
Allievex Corporation
Industry
Eligibility
1 Year to 10 Years
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2019
U.S. locations
1
States / cities
Oakland, California
Source: ClinicalTrials.gov public record
Updated Mar 6, 2020 · Synced Oct 15, 2026, 3:22 PM EDT
Recruiting Phase 1 Interventional

Safety and Preliminary Efficacy of ULSC in Facioscapulohumeral Muscular Dystrophy (FSHD)

NCT07086521
View directory record Official record
Conditions
FSHD - Facioscapulohumeral Muscular Dystrophy
Interventions
ULSC, Placebo
Biological
Lead sponsor
Restem, LLC.
Industry
Eligibility
15 Years and older
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2029
U.S. locations
1
States / cities
Palo Alto, California
Source: ClinicalTrials.gov public record
Updated Dec 8, 2025 · Synced Oct 15, 2026, 3:22 PM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Ascertainment of Families for Genetic Studies of Familial Lymphoproliferative Disorders

NCT00582621
View directory record Official record
Conditions
Lymphoma, Leukemia, Multiple Myeloma, Colon Cancer, Renal Cancer
Interventions
Not listed
Lead sponsor
Memorial Sloan Kettering Cancer Center
Other
Eligibility
Not listed
Enrollment
500 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2000 – 2026
U.S. locations
6
States / cities
Basking Ridge, New Jersey • Middletown, New Jersey • Commack, New York + 3 more
Source: ClinicalTrials.gov public record
Updated Jan 23, 2026 · Synced Oct 15, 2026, 3:22 PM EDT
Completed No phase listed Observational

Lysosomal Storage Disease: Health, Development, and Functional Outcome Surveillance in Preschool Children

NCT01938014
View directory record Official record
Conditions
Mucopolysaccharidosis Type I (MPS I), Mucopolysaccharidosis Type II (MPS II), Mucopolysaccharidosis Type III (MPS III), Mucopolysaccharidosis Type VI (MPS VI), Krabbe Disease
Interventions
Not listed
Lead sponsor
University of Chicago
Other
Eligibility
1 Day to 18 Years
Enrollment
19 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2016
U.S. locations
3
States / cities
Chicago, Illinois • Minneapolis, Minnesota • Buffalo, New York
Source: ClinicalTrials.gov public record
Updated Oct 4, 2019 · Synced Oct 15, 2026, 3:22 PM EDT
Completed Not applicable Interventional Accepts healthy volunteers

Impact of Color Correcting Lenses on Color Vision Deficiency

NCT05463016
View directory record Official record
Conditions
Color Blindness, Color Vision Defects, Color Blindness, Green, Color Blindness, Red
Interventions
Experimental Group 1: Color Correcting Lenses, Placebo Group 1: Placebo Lenses, Placebo Group 1 Crosses over to become Experimental Group 2: Color Correcting Lenses, Experimental Group 1 Crosses over to become Placebo Group 2: Placebo Lenses, Color Correcting Lenses in Subjects with Normal Color Vision
Device
Lead sponsor
University of the Incarnate Word
Other
Eligibility
18 Years and older
Enrollment
35 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2022 – 2024
U.S. locations
1
States / cities
San Antonio, Texas
Source: ClinicalTrials.gov public record
Updated Apr 3, 2024 · Synced Oct 15, 2026, 3:22 PM EDT
Completed Not applicable Interventional

Effects of Vitamin D Supplementation on Lung Function in an Acute Pulmonary Exacerbation of Cystic Fibrosis

NCT00788138
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
Vitamin D3, Placebo
Dietary Supplement
Lead sponsor
Emory University
Other
Eligibility
18 Years to 70 Years
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2010
U.S. locations
1
States / cities
Atlanta, Georgia
Source: ClinicalTrials.gov public record
Updated Oct 11, 2010 · Synced Oct 15, 2026, 3:22 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Epigenetic Regulation of Immunity in Alpha-1 Anti-trypsin Deficiency

NCT02691611
View directory record Official record
Conditions
Alpha 1-Antitrypsin Deficiency
Interventions
Not listed
Lead sponsor
National Jewish Health
Other
Eligibility
18 Years to 85 Years
Enrollment
39 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2015 – 2020
U.S. locations
1
States / cities
Denver, Colorado
Source: ClinicalTrials.gov public record
Updated Jun 6, 2025 · Synced Oct 15, 2026, 3:22 PM EDT
Recruiting Phase 1 Interventional Accepts healthy volunteers

Virtual Reality Mobility Assessment of Functional Vision in Retinal Disease

NCT04289571
View directory record Official record
Conditions
Cone-Rod Degeneration, Rod-Cone Degeneration
Interventions
VR Mobility Tool
Diagnostic Test
Lead sponsor
National Eye Institute (NEI)
NIH
Eligibility
5 Years to 120 Years
Enrollment
165 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2023 – 2030
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Aug 10, 2026 · Synced Oct 15, 2026, 3:22 PM EDT
Recruiting No phase listed Observational

CDH1-associated Blepharocheilodontic Syndrome Registry

NCT07133464
View directory record Official record
Conditions
Blepharocheilodontic Syndrome
Interventions
Not listed
Lead sponsor
Ohio State University
Other
Eligibility
Not listed
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2040
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Aug 21, 2025 · Synced Oct 15, 2026, 3:22 PM EDT
Completed Phase 2 Interventional Results available

A Study to Evaluate the Safety and Efficacy of VX-371 in Subjects With Cystic Fibrosis Who Are Homozygous for the F508del-CFTR Mutation

NCT02709109
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
VX-371 + HS, Hypertonic saline, Placebo, Orkambi, VX-371
Drug
Lead sponsor
Parion Sciences
Industry
Eligibility
12 Years and older
Enrollment
147 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2017
U.S. locations
30
States / cities
Little Rock, Arkansas • Oakland, California • Gainesville, Florida + 27 more
Source: ClinicalTrials.gov public record
Updated Jul 29, 2021 · Synced Oct 15, 2026, 3:22 PM EDT