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ClinicalTrials.gov public records Last synced Oct 1, 2026, 1:21 PM EDT

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Showing 49–72 of 9,394 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Genetic Disease Clear all
Completed Not applicable Interventional Accepts healthy volunteers

Relationship Between Down Syndrome (DS) and Alzheimer's Disease (AD)

NCT02759887
View directory record Official record
Conditions
Down Syndrome, Alzheimer's Dementia
Interventions
biospecimen collection, cognitive assessments, caregiver questionnaire, Florbetapir F18 imaging, MRI, Fludeoxyglucose F18 (FDG), Tau Pet, Actigraphy
Procedure · Other
Lead sponsor
St. Joseph's Hospital and Medical Center, Phoenix
Other
Eligibility
21 Years and older
Enrollment
19 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2016 – 2018
U.S. locations
1
States / cities
Phoenix, Arizona
Source: ClinicalTrials.gov public record
Updated Oct 29, 2019 · Synced Oct 1, 2026, 1:21 PM EDT
Completed Phase 3 Interventional Results available

Tolvaptan Extension Study in Participants With ADPKD

NCT01214421
View directory record Official record
Conditions
Autosomal Dominant Polycystic Kidney Disease (ADPKD)
Interventions
Tolvaptan
Drug
Lead sponsor
Otsuka Pharmaceutical Development & Commercialization, Inc.
Industry
Eligibility
18 Years and older
Enrollment
1,083 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2016
U.S. locations
38
States / cities
Mobile, Alabama • Peoria, Arizona • Tempe, Arizona + 33 more
Source: ClinicalTrials.gov public record
Updated Oct 25, 2021 · Synced Oct 1, 2026, 1:21 PM EDT
Completed No phase listed Observational

A Novel Assay to Quantify Treatment Response in Cystic Fibrosis (CF)

NCT01626157
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
Not listed
Lead sponsor
Milene Saavedra
Other
Eligibility
18 Years and older
Enrollment
122 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2018
U.S. locations
1
States / cities
Denver, Colorado
Source: ClinicalTrials.gov public record
Updated Mar 23, 2020 · Synced Oct 1, 2026, 1:21 PM EDT
Completed Phase 2 Interventional Results available

Nitrous Oxide Analgesia Vaso-occlusive Crisis

NCT01891812
View directory record Official record
Conditions
Sickle Cell Disease, Vaso-occlusive Crisis
Interventions
Nitrous oxide 50%
Drug
Lead sponsor
Columbia University
Other
Eligibility
8 Years to 18 Years
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2018
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jan 28, 2026 · Synced Oct 1, 2026, 1:21 PM EDT
Completed Phase 3 Interventional

Phase 3 Evaluation of Efficacy and Safety of AR882 in Gout Patients (AR882-302)

NCT06439602
View directory record Official record
Conditions
Gout, Arthritis, Gouty, Hyperuricemia, Gout Chronic
Interventions
AR882 50 mg, AR882 75 mg, Placebo
Drug
Lead sponsor
Arthrosi Therapeutics
Industry
Eligibility
18 Years to 85 Years
Enrollment
818 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
52
States / cities
Foley, Alabama • Anchorage, Alaska • Peoria, Arizona + 47 more
Source: ClinicalTrials.gov public record
Updated Aug 31, 2026 · Synced Oct 1, 2026, 1:21 PM EDT
Active, not recruiting No phase listed Observational

Genotypic Influences on Network Progression in Parkinson's Disease

NCT04228172
View directory record Official record
Conditions
Parkinson's Disease
Interventions
DNA/GeneticTesting, FDG PET scan, MRI scan, Clinical and neuropsychological assessments
Genetic · Radiation · Other
Lead sponsor
Northwell Health
Other
Eligibility
40 Years to 75 Years
Enrollment
32 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2026
U.S. locations
1
States / cities
Manhasset, New York
Source: ClinicalTrials.gov public record
Updated Apr 13, 2026 · Synced Oct 1, 2026, 1:21 PM EDT
Recruiting Phase 1 Interventional Accepts healthy volunteers

A Study of the Safety, Tolerability, Pharmacokinetics, and Immunogenicity of BCX17725

NCT06539507
View directory record Official record
Conditions
Netherton Syndrome
Interventions
BCX17725, Placebo
Drug
Lead sponsor
BioCryst Pharmaceuticals
Industry
Eligibility
12 Years to 65 Years
Enrollment
78 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2024 – 2026
U.S. locations
5
States / cities
Palo Alto, California • San Diego, California • New Haven, Connecticut + 2 more
Source: ClinicalTrials.gov public record
Updated Aug 27, 2026 · Synced Oct 1, 2026, 1:21 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Exploring Men s Prostate Cancer Genetic Risk Perceptions

NCT06764992
View directory record Official record
Conditions
Prostate Cancer
Interventions
Questionnaire, Saliva Sample
Other
Lead sponsor
H. Lee Moffitt Cancer Center and Research Institute
Other
Eligibility
40 Years and older · Male only
Enrollment
100 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2022 – 2024
U.S. locations
1
States / cities
Tampa, Florida
Source: ClinicalTrials.gov public record
Updated Feb 4, 2026 · Synced Oct 1, 2026, 1:21 PM EDT
Completed Phase 3 Interventional

Open Label Extension of 2 mg/kg Pegunigalsidase Alfa (PRX-102) Every 4 Weeks in Adult Fabry Disease Patients

NCT03614234
View directory record Official record
Conditions
Fabry Disease
Interventions
pegunigalsidase alfa
Drug
Lead sponsor
Chiesi Farmaceutici S.p.A.
Industry
Eligibility
18 Years and older
Enrollment
29 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2026
U.S. locations
7
States / cities
Birmingham, Alabama • Atlanta, Georgia • Iowa City, Iowa + 4 more
Source: ClinicalTrials.gov public record
Updated May 22, 2026 · Synced Oct 1, 2026, 1:21 PM EDT
Completed No phase listed Observational Accepts healthy volunteers Results available

Neurologic Injuries in Adults With Urea Cycle Disorders

NCT00472732
View directory record Official record
Conditions
Brain Diseases, Metabolic, Inborn, Urea Cycle Disorder, Ornithine Transcarbamylase Deficiency
Interventions
Not listed
Lead sponsor
Andrea Gropman
Other
Eligibility
18 Years to 60 Years
Enrollment
46 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2007 – 2010
U.S. locations
2
States / cities
Washington D.C., District of Columbia
Source: ClinicalTrials.gov public record
Updated Jun 24, 2015 · Synced Oct 1, 2026, 1:21 PM EDT
Completed Phase 4 Interventional

Efficacy Study of Extended-Release Niacin/Lovastatin Versus Usual Care

NCT00345657
View directory record Official record
Conditions
Hyperlipidemia, Mixed Hyperlipidemia, Dyslipidemia
Interventions
Niacin Extended Release/Lovastatin
Drug
Lead sponsor
In His Image
Other
Eligibility
18 Years and older
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003 – 2004
U.S. locations
1
States / cities
Tulsa, Oklahoma
Source: ClinicalTrials.gov public record
Updated Jun 28, 2006 · Synced Oct 1, 2026, 1:21 PM EDT
Completed Phase 3 Interventional Results available

A Study of (LY3650150) Lebrikizumab to Assess the Safety and Efficacy of Adult and Adolescent Participants With Moderate-to-Severe Atopic Dermatitis and Skin of Color

NCT05372419
View directory record Official record
Conditions
Atopic Dermatitis
Interventions
Lebrikizumab
Drug
Lead sponsor
Eli Lilly and Company
Industry
Eligibility
12 Years and older
Enrollment
90 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2025
U.S. locations
36
States / cities
Birmingham, Alabama • Fountain Valley, California • Fremont, California + 30 more
Source: ClinicalTrials.gov public record
Updated Mar 9, 2026 · Synced Oct 1, 2026, 1:21 PM EDT
Recruiting Not applicable Interventional

Implementation of Population Cancer Genetic Services in Federally Qualified Health Centers (FQHC)

NCT05664867
View directory record Official record
Conditions
Hereditary Cancer Syndrome
Interventions
Mainstream Genetic Testing Model, Enhanced Standard of Care Model
Other
Lead sponsor
University of Illinois at Chicago
Other
Eligibility
25 Years and older
Enrollment
80 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2027
U.S. locations
1
States / cities
Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Jan 14, 2026 · Synced Oct 1, 2026, 1:21 PM EDT
Completed Phase 2 Interventional

Safety and Efficacy Study of A0001 in Subjects With Friedreich's Ataxia

NCT01035671
View directory record Official record
Conditions
Friedreich's Ataxia
Interventions
alpha-tocopherolquinone (A0001), placebo
Drug
Lead sponsor
Penwest Pharmaceuticals Co.
Industry
Eligibility
18 Years to 60 Years
Enrollment
42 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2011
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Apr 22, 2011 · Synced Oct 1, 2026, 1:21 PM EDT
Recruiting Phase 1 Interventional

Phase IA and IB Study of AAVrh.10hFXN Gene Therapy for the Cardiomyopathy of Friedreich's Ataxia

NCT05302271
View directory record Official record
Conditions
Friedreich Ataxia, Cardiomyopathies, Cardiac Hypertrophy, Myocardial Fibrosis
Interventions
AAVrh.10hFXN, serotype rh.10 adeno-associated virus (AAV) gene transfer vector expressing the cDNA coding for human FXN, Prednisone
Biological · Drug
Lead sponsor
Weill Medical College of Cornell University
Other
Eligibility
12 Years to 50 Years
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2029
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Sep 28, 2026 · Synced Oct 1, 2026, 1:21 PM EDT
Completed Not applicable Interventional

Measuring Reading Rehabilitation Outcomes

NCT00125632
View directory record Official record
Conditions
Age Related Maculopathy, Retinal Degeneration
Interventions
Eccentric Viewing Training for Reading
Procedure
Lead sponsor
US Department of Veterans Affairs
Federal
Eligibility
18 Years and older
Enrollment
90 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2008
U.S. locations
1
States / cities
Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Feb 5, 2009 · Synced Oct 1, 2026, 1:21 PM EDT
Not yet recruiting Not applicable Interventional

Clearance in Primary Ciliary Dyskinesia

NCT07699302
View directory record Official record
Conditions
Primary Ciliary Dyskinesia, PCD
Interventions
Exercise, HS pretreatment with Exercise
Behavioral · Other
Lead sponsor
University of North Carolina, Chapel Hill
Other
Eligibility
18 Years and older
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2027
U.S. locations
1
States / cities
Chapel Hill, North Carolina
Source: ClinicalTrials.gov public record
Updated Jul 13, 2026 · Synced Oct 1, 2026, 1:21 PM EDT
Recruiting Not applicable Interventional

Impact of Bright Light Therapy on Prader-Willi Syndrome

NCT05939453
View directory record Official record
Conditions
Prader-Willi Syndrome, Excessive Daytime Sleepiness, Hyperphagia, Body Weight, Mood, Behavior
Interventions
Sham Light, Bright Light Therapy
Other
Lead sponsor
Maimonides Medical Center
Other
Eligibility
6 Years to 88 Years
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2026
U.S. locations
1
States / cities
Brooklyn, New York
Source: ClinicalTrials.gov public record
Updated May 15, 2026 · Synced Oct 1, 2026, 1:21 PM EDT
Completed No phase listed Observational

Outcome of Embryos Transferred After Initial Chaotic Results on PGT-A

NCT06373237
View directory record Official record
Conditions
Infertility
Interventions
Not listed
Lead sponsor
Inception Fertility Research Institute, LLC
Industry
Eligibility
Female only
Enrollment
600 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2025
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Jul 20, 2025 · Synced Oct 1, 2026, 1:21 PM EDT
Completed Phase 2 Interventional Results available

Trial of RAD001 and Neurocognition in Tuberous Sclerosis Complex (TSC)

NCT01289912
View directory record Official record
Conditions
Tuberous Sclerosis Complex
Interventions
RAD001, Placebo
Drug
Lead sponsor
Mustafa Sahin
Other
Eligibility
6 Years to 21 Years
Enrollment
52 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2014
U.S. locations
2
States / cities
Boston, Massachusetts • Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Jan 25, 2018 · Synced Oct 1, 2026, 1:21 PM EDT
Completed No phase listed Observational

A Study Following People With Haemophilia A and B, With or Without Inhibitors, When on Usual Treatment (Explorer™6)

NCT03741881
View directory record Official record
Conditions
Haemophilia A, Haemophilia A With Inhibitors, Haemophilia B, Haemophilia B With Inhibitors
Interventions
No treatment given
Other
Lead sponsor
Novo Nordisk A/S
Industry
Eligibility
12 Years and older · Male only
Enrollment
231 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2021
U.S. locations
29
States / cities
Los Angeles, California • Sacramento, California • Aurora, Colorado + 24 more
Source: ClinicalTrials.gov public record
Updated Nov 23, 2021 · Synced Oct 1, 2026, 1:21 PM EDT
Completed Phase 1Phase 2 Interventional

Phase 1 and 2 Study of PX-866 and Cetuximab

NCT01252628
View directory record Official record
Conditions
Incurable Metastatic Colorectal Carcinoma, Incurable Progressive, Recurrent or Metastatic Squamous Cell Carcinoma of the Head and Neck
Interventions
PX-866 (SCCHN), Cetuximab (SCCHN), PX-866 (CRC), Cetuximab (CRC)
Drug
Lead sponsor
Cascadian Therapeutics Inc.
Industry
Eligibility
18 Years and older
Enrollment
178 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2014
U.S. locations
31
States / cities
Birmingham, Alabama • Escondido, California • Monterey, California + 24 more
Source: ClinicalTrials.gov public record
Updated May 16, 2018 · Synced Oct 1, 2026, 1:21 PM EDT
Completed No phase listed Observational

Hereditary Colorectal and Associated Tumor Registry Study

NCT00633607
View directory record Official record
Conditions
Lynch Syndrome, FAP, Hereditary Diffuse Gastric Cancer, Juvenile Polyposis Syndrome, Peutz-Jeghers Syndrome
Interventions
Research Registry
Other
Lead sponsor
University of Pittsburgh
Other
Eligibility
8 Years to 100 Years
Enrollment
114 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2018
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Feb 19, 2018 · Synced Oct 1, 2026, 1:21 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 1, 2026, 1:21 PM EDT