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ClinicalTrials.gov public records Last synced May 22, 2026, 2:46 AM EDT

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

Showing 25–48 of 24 matching trials from the live ClinicalTrials.gov search.
Local D1 index available.
Filters: Condition: Genetic Syndrome Clear all
Terminated Phase 2 Interventional Results available
View directory record Official record
Conditions
Urothelial Cancer
Interventions
Bintrafusp alfa (M7824)
Drug
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
18 Years and older
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2021
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 21, 2022 · Synced May 22, 2026, 2:46 AM EDT
Completed Phase 2 Interventional Results available
View directory record Official record
Conditions
Chronic Iron Overload Due to Transfusion-dependant Anemias
Interventions
Deferasirox dispersible tablet, Defearisox film-coated tablet
Drug
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
10 Years and older
Enrollment
173 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2016
U.S. locations
5
States / cities
Orange, California • Chicago, Illinois • Boston, Massachusetts + 2 more
Source: ClinicalTrials.gov public record
Updated Jul 24, 2017 · Synced May 22, 2026, 2:46 AM EDT
Completed No phase listed Observational Accepts healthy volunteers Results available
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
MRI, Transthoracic Echocardiography, tonometry, EKG
Procedure
Lead sponsor
University of Chicago
Other
Eligibility
18 Years and older
Enrollment
51 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2009 – 2021
U.S. locations
1
States / cities
Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Feb 22, 2022 · Synced May 22, 2026, 2:46 AM EDT
Completed No phase listed Observational
View directory record Official record
Conditions
MPS IVA, Morquio Syndrome
Interventions
Not listed
Lead sponsor
Oregon Health and Science University
Other
Eligibility
1 Year and older
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2017
U.S. locations
1
States / cities
Portland, Oregon
Source: ClinicalTrials.gov public record
Updated Oct 16, 2019 · Synced May 22, 2026, 2:46 AM EDT
Completed Not applicable Interventional
View directory record Official record
Conditions
Sickle Cell Disease, COVID-19 Vaccine, Vaccine Hesitancy
Interventions
SCD-specific COVID-19 vaccination information (SCVI) video
Behavioral
Lead sponsor
Duke University
Other
Eligibility
18 Years to 100 Years
Enrollment
33 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2024
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Dec 23, 2024 · Synced May 22, 2026, 2:46 AM EDT
Active, not recruiting Phase 2 Interventional
View directory record Official record
Conditions
Mitochondrial Encephalopathy, Lactic Acidosis and Stroke-Like Episodes (MELAS Syndrome)
Interventions
zagociguat 15mg, zagociguat 30mg, Placebo
Drug
Lead sponsor
Tisento Therapeutics
Industry
Eligibility
18 Years to 75 Years
Enrollment
43 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
10
States / cities
La Jolla, California • Aurora, Colorado • Atlanta, Georgia + 5 more
Source: ClinicalTrials.gov public record
Updated Mar 24, 2026 · Synced May 22, 2026, 2:46 AM EDT
Completed Phase 3 Interventional Results available
View directory record Official record
Conditions
Mucopolysaccharidosis I, Hurler's Syndrome, Hurler-Scheie Syndrome, Scheie Syndrome
Interventions
Aldurazyme, placebo
Biological
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
Not listed
Enrollment
45 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2005
U.S. locations
8
States / cities
Mobile, Alabama • Atlanta, Georgia • Rochester, New York + 5 more
Source: ClinicalTrials.gov public record
Updated Apr 2, 2015 · Synced May 22, 2026, 2:46 AM EDT
Active, not recruiting Phase 2 Interventional
View directory record Official record
Conditions
Pseudohypoparathyroidism, Albright Hereditary Osteodystrophy, Pseudohypoparathyroidism Type 1a
Interventions
Theophylline, Placebo
Drug
Lead sponsor
Vanderbilt University Medical Center
Other
Eligibility
2 Years to 12 Years
Enrollment
34 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2026
U.S. locations
1
States / cities
Nashville, Tennessee
Source: ClinicalTrials.gov public record
Updated Apr 28, 2026 · Synced May 22, 2026, 2:46 AM EDT
Completed Not applicable Interventional
View directory record Official record
Conditions
Heart Failure, Dilated Cardiomyopathy
Interventions
PITA
Device
Lead sponsor
Johns Hopkins University
Other
Eligibility
18 Years and older
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2023
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Feb 13, 2023 · Synced May 22, 2026, 2:46 AM EDT
Completed Phase 3 Interventional Results available
View directory record Official record
Conditions
Hemophilia A, Hemophilia B
Interventions
Recombinant Factor VIIa BI (rFVIIa BI)
Biological
Lead sponsor
Baxalta now part of Shire
Industry
Eligibility
12 Years to 65 Years · Male only
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2014
U.S. locations
1
States / cities
Tampa, Florida
Source: ClinicalTrials.gov public record
Updated May 10, 2021 · Synced May 22, 2026, 2:46 AM EDT
Completed Phase 3 Interventional
View directory record Official record
Conditions
Achondroplasia
Interventions
Infigratinib 0.25 mg/kg/day, Placebo Comparator 0.25 mg/kg/day
Drug
Lead sponsor
QED Therapeutics, a BridgeBio company
Industry
Eligibility
3 Years to 17 Years
Enrollment
114 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2025
U.S. locations
7
States / cities
San Francisco, California • Aurora, Colorado • Baltimore, Maryland + 4 more
Source: ClinicalTrials.gov public record
Updated Mar 16, 2026 · Synced May 22, 2026, 2:46 AM EDT
Withdrawn No phase listed Observational
View directory record Official record
Conditions
Dystrophic Epidermolysis Bullosa, DEB - Dystrophic Epidermolysis Bullosa
Interventions
Not listed
Lead sponsor
Krystal Biotech, Inc.
Industry
Eligibility
6 Months and older
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020
U.S. locations
1
States / cities
Redwood City, California
Source: ClinicalTrials.gov public record
Updated Sep 16, 2021 · Synced May 22, 2026, 2:46 AM EDT
Terminated No phase listed Observational Accepts healthy volunteers
View directory record Official record
Conditions
Hemoglobinopathies, Hemolysis, Iron Deficiency and Overload, Anemias
Interventions
Not listed
Lead sponsor
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
NIH
Eligibility
1 Year and older
Enrollment
334 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2005 – 2017
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Feb 13, 2018 · Synced May 22, 2026, 2:46 AM EDT
Active, not recruiting Phase 2 Interventional
View directory record Official record
Conditions
PIK3CA-Related Overgrowth Spectrum (PROS)/Proteus Syndrome (PS)
Interventions
Miransertib
Drug
Lead sponsor
Merck Sharp & Dohme LLC
Industry
Eligibility
2 Years to 120 Years
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2030
U.S. locations
5
States / cities
Atlanta, Georgia • Boston, Massachusetts • Cincinnati, Ohio + 2 more
Source: ClinicalTrials.gov public record
Updated Feb 6, 2025 · Synced May 22, 2026, 2:46 AM EDT
Completed Phase 4 Interventional Accepts healthy volunteers
View directory record Official record
Conditions
Cystic Fibrosis, Pancreatic Insufficiency
Interventions
Pentadecanoic acid (PA) and Triheptadecanoin (THA)
Other
Lead sponsor
Children's Hospital of Philadelphia
Other
Eligibility
8 Years and older
Enrollment
9 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2006 – 2008
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Sep 29, 2008 · Synced May 22, 2026, 2:46 AM EDT
Active, not recruiting No phase listed Observational
View directory record Official record
Conditions
Hematopoietic Cell Transplantation, Mucopolysaccharidosis Type I
Interventions
Laronidase therapy and a stem cell transplant
Drug
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
0 Years to 3 Years
Enrollment
13 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2026
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Oct 29, 2025 · Synced May 22, 2026, 2:46 AM EDT
Terminated Phase 1 Interventional Results available
View directory record Official record
Conditions
Muscular Dystrophy, Duchenne
Interventions
Spironolactone, Prednisolone
Drug
Lead sponsor
Kevin Flanigan
Other
Eligibility
4 Years to 7 Years · Male only
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2021
U.S. locations
4
States / cities
Iowa City, Iowa • Columbus, Ohio • Philadelphia, Pennsylvania + 1 more
Source: ClinicalTrials.gov public record
Updated Oct 22, 2023 · Synced May 22, 2026, 2:46 AM EDT
Completed Phase 3 Interventional Accepts healthy volunteers Results available
View directory record Official record
Conditions
Gout
Interventions
High Dose Colchicine (4.8 mg total dose), Low Dose Colchicine (1.8mg total dose), Placebo Control
Drug · Other
Lead sponsor
Takeda
Industry
Eligibility
18 Years and older
Enrollment
185 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2007
U.S. locations
83
States / cities
Birmingham, Alabama • Columbiana, Alabama • Huntsville, Alabama + 78 more
Source: ClinicalTrials.gov public record
Updated Oct 31, 2012 · Synced May 22, 2026, 2:46 AM EDT
Completed Phase 4 Interventional Results available
View directory record Official record
Conditions
Leber's Hereditary Optic Neuropathy (LHON)
Interventions
Idebenone
Drug
Lead sponsor
Santhera Pharmaceuticals
Industry
Eligibility
12 Years and older
Enrollment
199 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2021
U.S. locations
9
States / cities
Phoenix, Arizona • Palo Alto, California • Stanford, California + 6 more
Source: ClinicalTrials.gov public record
Updated Apr 20, 2023 · Synced May 22, 2026, 2:46 AM EDT
Terminated Phase 1 Interventional
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
delandistrogene moxeparvovec, Plasmapheresis
Genetic · Procedure
Lead sponsor
Sarepta Therapeutics, Inc.
Industry
Eligibility
4 Years to 8 Years · Male only
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2025
U.S. locations
3
States / cities
Gainesville, Florida • St Louis, Missouri • Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Sep 3, 2025 · Synced May 22, 2026, 2:46 AM EDT
Recruiting No phase listed Observational Accepts healthy volunteers
View directory record Official record
Conditions
Food Allergy, Loeys-Dietz Syndrome, Atopic Dermatitis, Eosinophilic Esophagitis
Interventions
Not listed
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
1 Day to 99 Years
Enrollment
1,800 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2015 – 2026
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated May 19, 2026 · Synced May 22, 2026, 2:46 AM EDT
Completed Phase 2 Interventional Results available
View directory record Official record
Conditions
Neuroendocrine Tumors, Von Hippel-Lindau Syndrome, Hippel-Lindau Disease
Interventions
68Gallium DOTATATE, Radio-guided surgery
Drug · Procedure
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
10 Years to 99 Years
Enrollment
341 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2018
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Nov 18, 2019 · Synced May 22, 2026, 2:46 AM EDT
Completed No phase listed Observational Accepts healthy volunteers
View directory record Official record
Conditions
Alzheimer's Disease, Brain Neoplasm, Niemann Pick Disease
Interventions
15 O Water
Drug
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
Not listed
Enrollment
123 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
1994 – 2002
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 3, 2008 · Synced May 22, 2026, 2:46 AM EDT
Completed No phase listed Observational
View directory record Official record
Conditions
Rett Syndrome, MECP2 Duplication, CDKL5, FOXG1 Disorders
Interventions
Not listed
Lead sponsor
University of Alabama at Birmingham
Other
Eligibility
Not listed
Enrollment
752 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2021
U.S. locations
12
States / cities
Birmingham, Alabama • Oakland, California • San Diego, California + 9 more
Source: ClinicalTrials.gov public record
Updated Aug 4, 2021 · Synced May 22, 2026, 2:46 AM EDT