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ClinicalTrials.gov public records Last synced May 21, 2026, 7:22 PM EDT

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

Showing 1–24 of 9,235 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Genetic Disorder Clear all
Active, not recruiting Not applicable Interventional
View directory record Official record
Conditions
Hydrops Fetalis, Birth Defect, Fetal Anomaly
Interventions
Exome sequencing
Diagnostic Test
Lead sponsor
University of California, San Francisco
Other
Eligibility
18 Years to 55 Years
Enrollment
500 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2027
U.S. locations
1
States / cities
San Francisco, California
Source: ClinicalTrials.gov public record
Updated Apr 16, 2026 · Synced May 21, 2026, 7:22 PM EDT
Completed Phase 3 Interventional Results available
View directory record Official record
Conditions
Hemophilia A
Interventions
PEGylated Recombinant Factor VIII
Biological
Lead sponsor
Baxalta now part of Shire
Industry
Eligibility
12 Years to 65 Years
Enrollment
135 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2018
U.S. locations
14
States / cities
Phoenix, Arizona • Tucson, Arizona • Los Angeles, California + 11 more
Source: ClinicalTrials.gov public record
Updated May 24, 2021 · Synced May 21, 2026, 7:22 PM EDT
Not listed No phase listed Observational
View directory record Official record
Conditions
Mucopolysaccharidosis Type IIIB
Interventions
Not listed
Lead sponsor
Allievex Corporation
Industry
Eligibility
Up to 18 Years
Enrollment
44 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2025
U.S. locations
1
States / cities
Oakland, California
Source: ClinicalTrials.gov public record
Updated Oct 25, 2022 · Synced May 21, 2026, 7:22 PM EDT
Terminated Not applicable Interventional Results available
View directory record Official record
Conditions
Blood Pressure, Gout
Interventions
febuxostat
Drug
Lead sponsor
University of Mississippi Medical Center
Other
Eligibility
18 Years to 80 Years
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2011
U.S. locations
1
States / cities
Jackson, Mississippi
Source: ClinicalTrials.gov public record
Updated Jan 11, 2018 · Synced May 21, 2026, 7:22 PM EDT
Terminated No phase listed Observational Accepts healthy volunteers
View directory record Official record
Conditions
Short Stature, Growth Disorder, Syndromic Growth Disorder, Tall Stature
Interventions
Not listed
Lead sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
NIH
Eligibility
Not listed
Enrollment
334 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2014 – 2023
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 15, 2023 · Synced May 21, 2026, 7:22 PM EDT
Completed No phase listed Observational
View directory record Official record
Conditions
Sickle Cell Disease, Thalassemia, Pediatric Cancer
Interventions
Red Blood Cell (RBC) Transfusion
Biological
Lead sponsor
Westat
Other
Eligibility
Not listed
Enrollment
157 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2024
U.S. locations
9
States / cities
Oakland, California • San Francisco, California • Boston, Massachusetts + 2 more
Source: ClinicalTrials.gov public record
Updated May 16, 2024 · Synced May 21, 2026, 7:22 PM EDT
Completed No phase listed Observational
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Not listed
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
18 Years to 80 Years
Enrollment
99 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2019
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 31, 2026 · Synced May 21, 2026, 7:22 PM EDT
Completed Phase 3 Interventional Results available
View directory record Official record
Conditions
Hypophosphatemia, Familial, Hyperparathyroidism
Interventions
Paricalcitol, Placebo
Drug · Other
Lead sponsor
Yale University
Other
Eligibility
9 Years and older
Enrollment
33 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2012
U.S. locations
1
States / cities
New Haven, Connecticut
Source: ClinicalTrials.gov public record
Updated Mar 16, 2020 · Synced May 21, 2026, 7:22 PM EDT
Completed Phase 3 Interventional Results available
View directory record Official record
Conditions
Severe Hemophilia A
Interventions
Human-cl rhFVIII
Biological
Lead sponsor
Octapharma
Industry
Eligibility
12 Years to 65 Years · Male only
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2012
U.S. locations
1
States / cities
Washington D.C., District of Columbia
Source: ClinicalTrials.gov public record
Updated Mar 16, 2020 · Synced May 21, 2026, 7:22 PM EDT
Completed Not applicable Interventional Results available
View directory record Official record
Conditions
Tourette Syndrome, Chronic Tic Disorder
Interventions
Active Oral Orthotic Treatment, Placebo Oral Orthotic Treatment
Device
Lead sponsor
Weill Medical College of Cornell University
Other
Eligibility
7 Years to 25 Years
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2021
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated May 23, 2022 · Synced May 21, 2026, 7:22 PM EDT
Recruiting Phase 1 Interventional
View directory record Official record
Conditions
Von Willebrand Disease, Type 3, Concomitant VWD and Hemophilia
Interventions
Emicizumab
Drug
Lead sponsor
Bleeding and Clotting Disorders Institute Peoria, Illinois
Other
Eligibility
0 Years to 90 Years
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2028
U.S. locations
12
States / cities
Orange, California • Redwood City, California • Coral Gables, Florida + 9 more
Source: ClinicalTrials.gov public record
Updated Apr 2, 2026 · Synced May 21, 2026, 7:22 PM EDT
Not listed No phase listed Observational
View directory record Official record
Conditions
Down Syndrome, Dysphagia, Oropharyngeal
Interventions
Not listed
Lead sponsor
University of Colorado, Denver
Other
Eligibility
3 Years to 18 Years
Enrollment
75 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2024
U.S. locations
1
States / cities
Aurora, Colorado
Source: ClinicalTrials.gov public record
Updated Jan 25, 2024 · Synced May 21, 2026, 7:22 PM EDT
Completed Phase 3 Interventional Results available
View directory record Official record
Conditions
Lennox Gastaut Syndrome (LGS)
Interventions
Placebo, Soticlestat
Drug
Lead sponsor
Takeda
Industry
Eligibility
2 Years to 55 Years
Enrollment
270 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2024
U.S. locations
24
States / cities
Phoenix, Arizona • Tucson, Arizona • Los Angeles, California + 17 more
Source: ClinicalTrials.gov public record
Updated Aug 20, 2024 · Synced May 21, 2026, 7:22 PM EDT
Completed Not applicable Interventional
View directory record Official record
Conditions
Depression, Anxiety
Interventions
CF-CBT: A Cognitive-Behavioral Skills-Based Program to Promote Emotional Well-Being for Adults wtih Cystic Fibrosis, Waitlist Control
Behavioral
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
18 Years and older
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2022
U.S. locations
4
States / cities
Palo Alto, California • Kansas City, Kansas • Boston, Massachusetts + 1 more
Source: ClinicalTrials.gov public record
Updated Jul 24, 2023 · Synced May 21, 2026, 7:22 PM EDT
Not listed Not applicable Interventional Accepts healthy volunteers
View directory record Official record
Conditions
Macular Degeneration, Retinal Degeneration, Retinal Diseases, Eye Diseases
Interventions
Pistachio Group
Dietary Supplement
Lead sponsor
Tufts University
Other
Eligibility
40 Years to 70 Years
Enrollment
40 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2022 – 2023
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Mar 8, 2023 · Synced May 21, 2026, 7:22 PM EDT
Terminated Phase 2 Interventional Accepts healthy volunteers Results available
View directory record Official record
Conditions
Anemia, Sickle Cell, Sickle Cell Disease, Kidney Disease, Hypertension, Proteinuria
Interventions
Losartan
Drug
Lead sponsor
University of Alabama at Birmingham
Other
Eligibility
5 Years to 25 Years
Enrollment
1 participant
Healthy volunteers
Accepts healthy volunteers
Timeline
2015 – 2021
U.S. locations
1
States / cities
Birmingham, Alabama
Source: ClinicalTrials.gov public record
Updated Apr 2, 2024 · Synced May 21, 2026, 7:22 PM EDT
Completed Phase 1 Interventional Accepts healthy volunteers
View directory record Official record
Conditions
Healthy Volunteers, Sickle Cell Disease
Interventions
FT-4202/Placebo
Drug
Lead sponsor
Forma Therapeutics, Inc.
Industry
Eligibility
12 Years to 65 Years
Enrollment
130 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2018 – 2021
U.S. locations
18
States / cities
Little Rock, Arkansas • Long Beach, California • Oakland, California + 12 more
Source: ClinicalTrials.gov public record
Updated Apr 9, 2024 · Synced May 21, 2026, 7:22 PM EDT
Terminated Not applicable Interventional
View directory record Official record
Conditions
Chronic Obstructive Pulmonary Disease, Alpha-1 Antitrypsin Deficiency
Interventions
six-minute walk study indoors, six-minute walk study outdoors
Other
Lead sponsor
University of Florida
Other
Eligibility
18 Years and older
Enrollment
27 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2017
U.S. locations
1
States / cities
Gainesville, Florida
Source: ClinicalTrials.gov public record
Updated Aug 21, 2019 · Synced May 21, 2026, 7:22 PM EDT
Completed No phase listed Observational
View directory record Official record
Conditions
Huntington Disease
Interventions
Not listed
Lead sponsor
University of California, Davis
Other
Eligibility
18 Years and older
Enrollment
29 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2016
U.S. locations
1
States / cities
Sacramento, California
Source: ClinicalTrials.gov public record
Updated Oct 31, 2016 · Synced May 21, 2026, 7:22 PM EDT
Not listed No phase listed Observational
View directory record Official record
Conditions
Nevi, Dysplastic
Interventions
Not listed
Lead sponsor
Orlucent, Inc
Industry
Eligibility
Not listed
Enrollment
500 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2024
U.S. locations
1
States / cities
Salt Lake City, Utah
Source: ClinicalTrials.gov public record
Updated Feb 6, 2024 · Synced May 21, 2026, 7:22 PM EDT
Completed No phase listed Observational
View directory record Official record
Conditions
Chordoma
Interventions
Not listed
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
Up to 110 Years
Enrollment
56 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1999 – 2020
U.S. locations
2
States / cities
Iowa City, Iowa • Rockville, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 9, 2020 · Synced May 21, 2026, 7:22 PM EDT
Completed Not applicable Interventional
View directory record Official record
Conditions
Spinocerebellar Ataxias
Interventions
Aerobic training, Balance training
Behavioral
Lead sponsor
Columbia University
Other
Eligibility
18 Years to 65 Years
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2022
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Oct 11, 2022 · Synced May 21, 2026, 7:22 PM EDT
Completed Phase 4 Interventional Results available
View directory record Official record
Conditions
Cytomegalovirus Infection
Interventions
Valganciclovir
Drug
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
18 Years to 99 Years
Enrollment
205 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2018
U.S. locations
6
States / cities
Los Angeles, California • Atlanta, Georgia • Rochester, Minnesota + 3 more
Source: ClinicalTrials.gov public record
Updated Aug 25, 2021 · Synced May 21, 2026, 7:22 PM EDT
Terminated Phase 3 Interventional Results available
View directory record Official record
Conditions
Wilson Disease
Interventions
ALXN1840, SoC Therapy
Drug
Lead sponsor
Alexion Pharmaceuticals, Inc.
Industry
Eligibility
12 Years and older
Enrollment
214 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2023
U.S. locations
6
States / cities
Los Angeles, California • New Haven, Connecticut • Chicago, Illinois + 3 more
Source: ClinicalTrials.gov public record
Updated Jun 16, 2024 · Synced May 21, 2026, 7:22 PM EDT