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ClinicalTrials.gov public records Last synced Oct 23, 2026, 2:15 PM EDT

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

Showing 985–1008 of 8,007 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Genetics/Birth Defects Clear all
Active, not recruiting Phase 2 Interventional

A Study Evaluating APG777 in Atopic Dermatitis

NCT06395948
View directory record Official record
Conditions
Atopic Dermatitis
Interventions
APG777, Placebo
Drug
Lead sponsor
Apogee Therapeutics, Inc.
Industry
Eligibility
18 Years and older
Enrollment
470 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2028
U.S. locations
29
States / cities
Fountain Valley, California • Los Angeles, California • San Diego, California + 25 more
Source: ClinicalTrials.gov public record
Updated Oct 13, 2026 · Synced Oct 23, 2026, 2:15 PM EDT
Completed Phase 3 Interventional Results available

A Phase III Safety and Efficacy Study of L-Glutamine to Treat Sickle Cell Disease or Sickle βo-thalassemia

NCT01179217
View directory record Official record
Conditions
Sickle Cell Anemia, Sickle ß0-Thalassemia
Interventions
L-glutamine, Placebo
Drug
Lead sponsor
Emmaus Medical, Inc.
Industry
Eligibility
5 Years and older
Enrollment
230 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2014
U.S. locations
31
States / cities
Mobile, Alabama • Phoenix, Arizona • Inglewood, California + 23 more
Source: ClinicalTrials.gov public record
Updated Aug 19, 2020 · Synced Oct 23, 2026, 2:15 PM EDT
Terminated Phase 3 Interventional Results available

A Trial of Bardoxolone Methyl in Patients With ADPKD - FALCON

NCT03918447
View directory record Official record
Conditions
Autosomal Dominant Polycystic Kidney, ADPKD
Interventions
Bardoxolone methyl oral capsule, Placebo oral capsule
Drug
Lead sponsor
Biogen
Industry
Eligibility
12 Years to 70 Years
Enrollment
667 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2023
U.S. locations
83
States / cities
Birmingham, Alabama • Homewood, Alabama • Huntsville, Alabama + 69 more
Source: ClinicalTrials.gov public record
Updated Jun 3, 2025 · Synced Oct 23, 2026, 2:15 PM EDT
Completed No phase listed Observational

Cystic Fibrosis and Cognitive Function

NCT03820349
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
Not listed
Lead sponsor
University of Minnesota
Other
Eligibility
18 Years and older
Enrollment
28 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2019
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Jul 2, 2021 · Synced Oct 23, 2026, 2:15 PM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

Study 102: Multiple Ascending Dose Study of ALLN-346 (Engineered Urate Oxidase)

NCT04829435
View directory record Official record
Conditions
Hyperuricemia, Gout
Interventions
ALLN-346, Placebo
Drug
Lead sponsor
Allena Pharmaceuticals
Industry
Eligibility
18 Years to 55 Years
Enrollment
18 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2021
U.S. locations
1
States / cities
Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Jul 1, 2021 · Synced Oct 23, 2026, 2:15 PM EDT
Completed Phase 2Phase 3 Interventional Results available

TBI Dose De-escalation for Fanconi Anemia

NCT00352976
View directory record Official record
Conditions
Fanconi Anemia
Interventions
Cyclophosphamide, Fludarabine, Total Body Irradiation, Bone Marrow Transplantation, Mycophenolate Mofetil, Sirolimus
Drug · Procedure
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
Not listed
Enrollment
83 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2020
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Nov 24, 2021 · Synced Oct 23, 2026, 2:15 PM EDT
Recruiting No phase listed Observational

Remote Assessments and Genetic Determinants of Congenital and Childhood Myotonic Dystrophy

NCT07630389
View directory record Official record
Conditions
Myotonic Dystrophy Type 1, Congenital DM1, Juvenile DM1, Childhood DM1
Interventions
Not listed
Lead sponsor
University of Rochester
Other
Eligibility
0 Years to 17 Years
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2030
U.S. locations
1
States / cities
Rochester, New York
Source: ClinicalTrials.gov public record
Updated Jun 5, 2026 · Synced Oct 23, 2026, 2:15 PM EDT
Completed Not applicable Interventional

A Single Center Pilot Study to Assess the Intra-observer Reliability of Measuring Muscle Strength Using a Hand Held Dynamometer in Children and Adults With Neurofibromatosis Type 1 (NF1) and Type 2 (NF2)

NCT03672838
View directory record Official record
Conditions
Neurofibromatosis 1 and 2 (NF1 and NF2)
Interventions
hand-held dynamometer
Behavioral
Lead sponsor
NYU Langone Health
Other
Eligibility
5 Years and older
Enrollment
33 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2019
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Mar 18, 2021 · Synced Oct 23, 2026, 2:15 PM EDT
Completed Phase 2 Interventional

Hydroxyurea to Treat Beta-Thalassemia (Cooley's Anemia)

NCT00001958
View directory record Official record
Conditions
Beta Thalassemia, Hemoglobinopathy
Interventions
Hydroxyurea
Drug
Lead sponsor
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
NIH
Eligibility
Not listed
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1999 – 2002
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 4, 2008 · Synced Oct 23, 2026, 2:15 PM EDT
Completed Not applicable Interventional Accepts healthy volunteers Results available

Safety and Effectiveness Evaluation of the Device in Achieving Submental Lift

NCT02832674
View directory record Official record
Conditions
Skin Laxity
Interventions
Percutaneous Radiofrequency single treatment
Device
Lead sponsor
ThermiGen, LLC
Industry
Eligibility
35 Years to 65 Years
Enrollment
72 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2016 – 2017
U.S. locations
1
States / cities
Montclair, New Jersey
Source: ClinicalTrials.gov public record
Updated May 1, 2018 · Synced Oct 23, 2026, 2:15 PM EDT
Recruiting No phase listed Observational

Monogenic Kidney Stone - Genetic Testing

NCT03305835
View directory record Official record
Conditions
Rare Kidney Stone Diseases
Interventions
Not listed
Lead sponsor
Mayo Clinic
Other
Eligibility
Not listed
Enrollment
6,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2028
U.S. locations
1
States / cities
Rochester, Minnesota
Source: ClinicalTrials.gov public record
Updated Apr 13, 2026 · Synced Oct 23, 2026, 2:15 PM EDT
Recruiting Phase 1Phase 2 Interventional

NORTHERA (DROXIDOPA) for Dysautonomia in Pediatric Survivors of Menkes Disease

NCT07398508
View directory record Official record
Conditions
Menkes Disease
Interventions
Droxidopa Oral Product, Placebo Control
Drug · Other
Lead sponsor
Stephen G. Kaler
Other
Eligibility
7 Years to 17 Years
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2030
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Oct 5, 2026 · Synced Oct 23, 2026, 2:15 PM EDT
Completed Phase 2Phase 3 Interventional Accepts healthy volunteers Results available

The Role of Mineralocorticoid Receptors in Vascular Function

NCT00759525
View directory record Official record
Conditions
Apparent Mineralocorticoid Excess (AME)
Interventions
Glycyrrhetic Acid, Placebo
Drug
Lead sponsor
Brigham and Women's Hospital
Other
Eligibility
18 Years to 85 Years
Enrollment
15 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2002 – 2009
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Oct 26, 2016 · Synced Oct 23, 2026, 2:15 PM EDT
Completed Not applicable Interventional Accepts healthy volunteers

Radiofrequency and Ultrasound for Improvement of Skin Laxity and Wrinkles: Efficacy and Safety Evaluation

NCT05929625
View directory record Official record
Conditions
Skin Laxity
Interventions
BTL-585-2 RF+US, BTL-585-2 RF
Device
Lead sponsor
BTL Industries Ltd.
Industry
Eligibility
21 Years and older
Enrollment
41 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2020 – 2022
U.S. locations
2
States / cities
Birmingham, Michigan • Houston, Texas
Source: ClinicalTrials.gov public record
Updated Jul 3, 2023 · Synced Oct 23, 2026, 2:15 PM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Neurophysiologic Biomarkers in Rett Syndrome

NCT05932589
View directory record Official record
Conditions
Rett Syndrome, RTT, Rett Syndrome, Atypical
Interventions
EEG and Auditory and Visual Evoked Potentials (AEP and VEP), Clinical assessment
Other
Lead sponsor
Children's Hospital of Philadelphia
Other
Eligibility
1 Year to 18 Years · Female only
Enrollment
202 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2023 – 2029
U.S. locations
6
States / cities
Los Angeles, California • Aurora, Colorado • Brookline, Massachusetts + 3 more
Source: ClinicalTrials.gov public record
Updated Oct 20, 2025 · Synced Oct 23, 2026, 2:15 PM EDT
Recruiting No phase listed Observational

Lysosomal Acid Lipase (LAL) Deficiency Registry

NCT01633489
View directory record Official record
Conditions
Lysosomal Acid Lipase Deficiency, Cholesterol Ester Storage Disease, Wolman Disease, Acid Cholesteryl Ester Hydrolase Deficiency, Type 2, Acid Lipase Deficiency, LIPA Deficiency, LAL-Deficiency
Interventions
Not listed
Lead sponsor
Alexion Pharmaceuticals, Inc.
Industry
Eligibility
Not listed
Enrollment
300 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2029
U.S. locations
19
States / cities
Phoenix, Arizona • Stanford, California • Miramar, Florida + 14 more
Source: ClinicalTrials.gov public record
Updated Mar 6, 2026 · Synced Oct 23, 2026, 2:15 PM EDT
Not listed Phase 3 Interventional

Independent Studies of Dextromethorphan and of Donepezil Hydrochloride for Rett Syndrome

NCT00069550
View directory record Official record
Conditions
Rett Syndrome
Interventions
dextromethorphan, donepezil hydrochloride
Drug
Lead sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
NIH
Eligibility
1 Year to 15 Years
Enrollment
90 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2008
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Jun 24, 2005 · Synced Oct 23, 2026, 2:15 PM EDT
Completed Phase 4 Interventional

Exercise Versus DDAVP in Patients With Mild Hemophilia A

NCT03379974
View directory record Official record
Conditions
Hemophilia A
Interventions
DDAVP Inhalant Product, Exercise Intervention
Drug · Behavioral
Lead sponsor
Nationwide Children's Hospital
Other
Eligibility
13 Years to 21 Years · Male only
Enrollment
32 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2019
U.S. locations
4
States / cities
Indianapolis, Indiana • Akron, Ohio • Columbus, Ohio + 1 more
Source: ClinicalTrials.gov public record
Updated Feb 25, 2020 · Synced Oct 23, 2026, 2:15 PM EDT
Completed Phase 1 Interventional Results available

Safety of Fresolimumab in the Treatment of Osteogenesis Imperfecta

NCT03064074
View directory record Official record
Conditions
Osteogenesis Imperfecta
Interventions
Fresolimumab
Drug
Lead sponsor
Baylor College of Medicine
Other
Eligibility
18 Years and older
Enrollment
11 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2022
U.S. locations
2
States / cities
Portland, Oregon • Houston, Texas
Source: ClinicalTrials.gov public record
Updated Dec 4, 2024 · Synced Oct 23, 2026, 2:15 PM EDT
Completed Phase 2 Interventional Results available

Effect of Atorvastatin on Endothelial Dysfunction and Albuminuria in Sickle Cell Disease

NCT01732718
View directory record Official record
Conditions
Sickle Cell Disease, Sickle Cell Nephropathy
Interventions
Atorvastatin, Placebo
Drug
Lead sponsor
University of North Carolina, Chapel Hill
Other
Eligibility
18 Years to 60 Years
Enrollment
13 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2018
U.S. locations
1
States / cities
Chapel Hill, North Carolina
Source: ClinicalTrials.gov public record
Updated Mar 18, 2020 · Synced Oct 23, 2026, 2:15 PM EDT
Recruiting Not applicable Interventional

Exercise Intervention and Immunodeficiency

NCT07698197
View directory record Official record
Conditions
Primary Antibody Deficiency Disorders
Interventions
App-based exercise intervention
Behavioral
Lead sponsor
Yale University
Other
Eligibility
18 Years to 65 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2027
U.S. locations
2
States / cities
Milford, Connecticut • North Haven, Connecticut
Source: ClinicalTrials.gov public record
Updated Jul 23, 2026 · Synced Oct 23, 2026, 2:15 PM EDT
Completed Not applicable Interventional Accepts healthy volunteers Results available

Feasibility Study: Heavy Water Tissue Labeling Protocol

NCT01708525
View directory record Official record
Conditions
Skin Laxity
Interventions
Ulthera® System Treatment, Heavy Water
Device · Other
Lead sponsor
Ulthera, Inc
Industry
Eligibility
30 Years to 65 Years
Enrollment
2 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2012 – 2013
U.S. locations
1
States / cities
Pasadena, California
Source: ClinicalTrials.gov public record
Updated Dec 11, 2017 · Synced Oct 23, 2026, 2:15 PM EDT
Completed Not applicable Interventional

Study of Albuterol and Oxandrolone in Patients With Facioscapulohumeral Dystrophy (FSHD)

NCT00027391
View directory record Official record
Conditions
Muscular Dystrophies
Interventions
Albuterol, Oxandrolone
Drug
Lead sponsor
FDA Office of Orphan Products Development
Federal
Eligibility
18 Years to 80 Years
Enrollment
160 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2004
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Mar 25, 2015 · Synced Oct 23, 2026, 2:15 PM EDT
Completed No phase listed Observational

Socialization of Adult Men With Congenital Hemophilia A or B

NCT01510418
View directory record Official record
Conditions
Hemophilia A, Hemophilia B
Interventions
Not listed
Lead sponsor
Dana-Farber Cancer Institute
Other
Eligibility
21 Years and older
Enrollment
27 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2013
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Aug 3, 2016 · Synced Oct 23, 2026, 2:15 PM EDT