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ClinicalTrials.gov public records Last synced Oct 6, 2026, 4:13 PM EDT

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Showing 25–48 of 10,522 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Congenital Diseases Clear all
Completed Phase 3 Interventional Results available

Pharmacokinetic, Efficacy, and Safety Study of Recombinant Factor VIII Single Chain (rVIII-SingleChain) in Children With Severe Hemophilia A

NCT02093897
View directory record Official record
Conditions
Congenital Hemophilia A
Interventions
rVIII-SingleChain
Biological
Lead sponsor
CSL Behring
Industry
Eligibility
Up to 11 Years · Male only
Enrollment
84 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2015
U.S. locations
2
States / cities
Aurora, Colorado • Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Jan 27, 2017 · Synced Oct 6, 2026, 4:13 PM EDT
Completed Phase 1Phase 2 Interventional

Reduced Intensity Transplant Using Extracorporeal Photopheresis

NCT00179790
View directory record Official record
Conditions
Leukemia, Cancer
Interventions
Extracorporeal Photopheresis
Procedure
Lead sponsor
Ann & Robert H Lurie Children's Hospital of Chicago
Other
Eligibility
Not listed
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2015
U.S. locations
1
States / cities
Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Mar 6, 2017 · Synced Oct 6, 2026, 4:13 PM EDT
Completed Phase 3 Interventional Results available

A Phase III, Crossover Trial Evaluating the Efficacy and Safety of KVD900 (Sebetralstat) for On-Demand Treatment of Angioedema Attacks in Adolescent and Adult Patients With Hereditary Angioedema (HAE)

NCT05259917
View directory record Official record
Conditions
Hereditary Angioedema
Interventions
Placebo, KVD900 600 mg, KVD900 300 mg
Drug
Lead sponsor
KalVista Pharmaceuticals, Ltd.
Industry
Eligibility
12 Years and older
Enrollment
136 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2023
U.S. locations
22
States / cities
Birmingham, Alabama • Scottsdale, Arizona • Little Rock, Arkansas + 18 more
Source: ClinicalTrials.gov public record
Updated May 2, 2025 · Synced Oct 6, 2026, 4:13 PM EDT
Completed Phase 1 Interventional

Pyrimethamine to Treat Autoimmune Lymphoproliferative Syndrome

NCT00065390
View directory record Official record
Conditions
Autoimmune Disease, Lymphatic Disease, Lymphoproliferative Disorder
Interventions
Pyrimethamine
Drug
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
Not listed
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003 – 2005
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 4, 2008 · Synced Oct 6, 2026, 4:13 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Osteogenesis Imperfecta Blood Pressure Study

NCT04169568
View directory record Official record
Conditions
Osteogenesis Imperfecta
Interventions
Manual cuff blood pressure
Procedure
Lead sponsor
Nemours Children's Clinic
Other
Eligibility
1 Year to 35 Years
Enrollment
50 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2019 – 2025
U.S. locations
1
States / cities
Wilmington, Delaware
Source: ClinicalTrials.gov public record
Updated May 14, 2025 · Synced Oct 6, 2026, 4:13 PM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

A PET Brain Imaging Study of mGluR5 in Subjects With Neuropsychiatric Conditions

NCT00870974
View directory record Official record
Conditions
Parkinson Disease, Huntington Disease, Autistic Spectrum Disorders, Fragile X Syndrome, Alzheimer Disease, Mild Cognitive Impairment
Interventions
[18F]FPEB
Drug
Lead sponsor
Institute for Neurodegenerative Disorders
Other
Eligibility
18 Years to 85 Years
Enrollment
48 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2009 – 2016
U.S. locations
1
States / cities
New Haven, Connecticut
Source: ClinicalTrials.gov public record
Updated Jan 6, 2017 · Synced Oct 6, 2026, 4:13 PM EDT
Recruiting Phase 1Phase 2 Interventional

ALS20-101 Lentiviral Gene Therapy for Beta Thalassemia

NCT06364774
View directory record Official record
Conditions
Beta-Thalassemia
Interventions
ALS20
Biological
Lead sponsor
Children's Hospital of Philadelphia
Other
Eligibility
18 Years to 40 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Sep 23, 2026 · Synced Oct 6, 2026, 4:13 PM EDT
Completed Phase 3 Interventional Results available

Study of Obicetrapib & Ezetimibe Fixed Dose Combination on Top of Maximum Tolerated Lipid-Modifying Therapies

NCT06005597
View directory record Official record
Conditions
Dyslipidemias, Hypercholesterolemia, Familial Hypercholesterolemia, ASCVD, High Cholesterol
Interventions
Combination Therapy, Monotherapy obicetrapib, Monotherapy ezetimibe, Combination Therapy placebo, Obicetrapib Placebo, Ezetimibe Placebo
Combination Product · Drug · Other
Lead sponsor
NewAmsterdam Pharma
Industry
Eligibility
18 Years and older
Enrollment
407 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024
U.S. locations
45
States / cities
Gilbert, Arizona • Jacksonville, Florida • Port Orange, Florida + 37 more
Source: ClinicalTrials.gov public record
Updated Oct 8, 2025 · Synced Oct 6, 2026, 4:13 PM EDT
Completed Phase 2 Interventional

Radiolabeled Monoclonal Antibody, Cyclophosphamide, and Total Body Irradiation Followed By Donor Stem Cell Transplantation in Treating Patients With Advanced Acute Myeloid Leukemia

NCT00003868
View directory record Official record
Conditions
Leukemia
Interventions
cyclophosphamide, methotrexate, allogeneic bone marrow transplantation, peripheral blood stem cell transplantation, iodine I 131 monoclonal antibody BC8, radiation therapy
Drug · Procedure · Radiation
Lead sponsor
Fred Hutchinson Cancer Center
Other
Eligibility
2 Years to 55 Years
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1999 – 2005
U.S. locations
1
States / cities
Seattle, Washington
Source: ClinicalTrials.gov public record
Updated Aug 24, 2010 · Synced Oct 6, 2026, 4:13 PM EDT
Terminated No phase listed Observational

Whole Blood Biospecimen Collection for Subjects With Chronic Granulomatous Disease (CGD)

NCT05915897
View directory record Official record
Conditions
Chronic Granulomatous Disease
Interventions
Specimen Donation
Diagnostic Test
Lead sponsor
Sanguine Biosciences
Industry
Eligibility
18 Years to 85 Years
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2024
U.S. locations
1
States / cities
Woburn, Massachusetts
Source: ClinicalTrials.gov public record
Updated Apr 18, 2024 · Synced Oct 6, 2026, 4:13 PM EDT
Active, not recruiting Not applicable Interventional

Electro-acupuncture and Transcorneal Electrical Stimulation (TES) for Retinitis Pigmentosa

NCT02086890
View directory record Official record
Conditions
Retinitis Pigmentosa
Interventions
Electro-acupuncture, Laser Acupuncture, Transcorneal Electrical Stimulation, Sham Electro-acupuncture, Sham laser acupuncture, Sham transcorneal electrical stimulation
Device
Lead sponsor
Nova Southeastern University
Other
Eligibility
18 Years and older
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2027
U.S. locations
1
States / cities
Fort Lauderdale, Florida
Source: ClinicalTrials.gov public record
Updated Feb 27, 2026 · Synced Oct 6, 2026, 4:13 PM EDT
Completed No phase listed Observational

Endolymphatic Sac Tumors in a Population of Patients With Von Hippel-Lindau Disease:The Natural History and Pathobiology, and a Prospective Non-Randomized Clinical Trial of Hearing Preservation Surgery in Patients With Early Stage Endolymphatic Sac Tumors

NCT00001668
View directory record Official record
Conditions
Deafness, Kidney Diseases, Kidney Neoplasms, Neoplasms, Retinal Diseases
Interventions
Not listed
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
Not listed
Enrollment
75 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1997 – 2000
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 4, 2008 · Synced Oct 6, 2026, 4:13 PM EDT
Completed No phase listed Observational

The Relationship Between Having a Positive Mindset and Exercise Capacity in Patients With Congenital Heart Disease

NCT03912025
View directory record Official record
Conditions
Congenital Heart Disease
Interventions
Relationship between positive mindset and exercise capacity
Other
Lead sponsor
Boston Children's Hospital
Other
Eligibility
8 Years to 17 Years
Enrollment
90 participants
Timeline
2020 – 2021
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Aug 4, 2021 · Synced Oct 6, 2026, 4:13 PM EDT
Recruiting Phase 1Phase 2 Interventional Accepts healthy volunteers

Harmony-HHT: ATV-1601 in Participants With Hereditary Hemorrhagic Telangiectasia (HHT)

NCT07601425
View directory record Official record
Conditions
Hereditary Hemorrhagic Telangiectasia (HHT), Osler Weber Rendu Disease
Interventions
ATV-1601, Placebo
Drug
Lead sponsor
Atavistik Bio, Inc
Industry
Eligibility
18 Years and older
Enrollment
100 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2026 – 2030
U.S. locations
6
States / cities
Phoenix, Arizona • Augusta, Georgia • Indianapolis, Indiana + 3 more
Source: ClinicalTrials.gov public record
Updated Oct 5, 2026 · Synced Oct 6, 2026, 4:13 PM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Collection of Human Biospecimens for Basic and Clinical Research Into Globin Variants

NCT03937817
View directory record Official record
Conditions
Alpha and Beta Thalassemia, Sickle Cell Disease, Malaria, Human Physiology
Interventions
Not listed
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
18 Years to 70 Years
Enrollment
300 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2019 – 2029
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 2, 2026 · Synced Oct 6, 2026, 4:13 PM EDT
Completed No phase listed Observational

Prenatal Effects of Congenital Heart Disease (CHD) on Neurodevelopmental Outcome

NCT00713635
View directory record Official record
Conditions
Congenital Heart Disease
Interventions
Not listed
Lead sponsor
Columbia University
Other
Eligibility
Up to 50 Years
Enrollment
51 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2016
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jul 18, 2017 · Synced Oct 6, 2026, 4:13 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 6, 2026, 4:13 PM EDT
Completed No phase listed Observational

Computer Modeling of Congenital Heart Disease

NCT00366847
View directory record Official record
Conditions
Congenital Disorders
Interventions
Not listed
Lead sponsor
Children's Healthcare of Atlanta
Other
Eligibility
Up to 18 Years
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2012
U.S. locations
1
States / cities
Atlanta, Georgia
Source: ClinicalTrials.gov public record
Updated May 8, 2014 · Synced Oct 6, 2026, 4:13 PM EDT
Terminated Phase 2 Interventional Results available

Asymptomatic Congenital CMV Treatment

NCT03301415
View directory record Official record
Conditions
Congenital Cytomegalovirus Infection
Interventions
Valganciclovir
Drug
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
1 Day to 30 Days
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2020
U.S. locations
9
States / cities
Birmingham, Alabama • Little Rock, Arkansas • Louisville, Kentucky + 6 more
Source: ClinicalTrials.gov public record
Updated Jan 26, 2023 · Synced Oct 6, 2026, 4:13 PM EDT
Completed Phase 1 Interventional

Study to Determine the Maximum Tolerated Dose, Safety and Effectiveness of Pomalidomide for Patients With Sickle Cell Disease

NCT01522547
View directory record Official record
Conditions
Anemia, Sickle Cell
Interventions
pomalidomide
Drug
Lead sponsor
Celgene
Industry
Eligibility
18 Years to 60 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2013
U.S. locations
1
States / cities
Detroit, Michigan
Source: ClinicalTrials.gov public record
Updated Nov 8, 2019 · Synced Oct 6, 2026, 4:13 PM EDT
Withdrawn Phase 2 Interventional

Adjunctive Everolimus (RAD 001) Therapy for Epilepsy in Children With Sturge-Weber Syndrome (SWS)

NCT01997255
View directory record Official record
Conditions
Sturge Weber Syndrome
Interventions
Everolimus
Drug
Lead sponsor
Baylor College of Medicine
Other
Eligibility
2 Years to 18 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2016
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated May 5, 2016 · Synced Oct 6, 2026, 4:13 PM EDT
Completed Phase 3 Interventional

Efficacy and Safety of Apitegromab in Patients With Later-Onset Spinal Muscular Atrophy Treated With Nusinersen or Risdiplam

NCT05156320
View directory record Official record
Conditions
Spinal Muscular Atrophy, Spinal Muscular Atrophy Type 3, Spinal Muscular Atrophy Type 2, SMA, Neuromuscular Diseases, Muscular Atrophy, Atrophy, Muscular Atrophy, Spinal, Neuromuscular Manifestations, Anti-myostatin
Interventions
Apitegromab, Placebo
Drug
Lead sponsor
Scholar Rock, Inc.
Industry
Eligibility
2 Years to 21 Years
Enrollment
188 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2024
U.S. locations
26
States / cities
Birmingham, Alabama • Phoenix, Arizona • Los Angeles, California + 23 more
Source: ClinicalTrials.gov public record
Updated Jan 22, 2026 · Synced Oct 6, 2026, 4:13 PM EDT
Completed No phase listed Observational

A Study of the Epidemiology and Hospital Management of Patients With PROS in France

NCT07222423
View directory record Official record
Conditions
PIK3CA-Related Overgrowth Spectrum
Interventions
Not listed
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
2 Years and older
Enrollment
3,605 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024
U.S. locations
1
States / cities
East Hanover, New Jersey
Source: ClinicalTrials.gov public record
Updated Oct 29, 2025 · Synced Oct 6, 2026, 4:13 PM EDT
Completed Not applicable Interventional

Feasibility and Efficacy of a Home-based, Computerized Cognitive Training Program in Pediatric Sickle Cell Disease

NCT02857023
View directory record Official record
Conditions
Anemia, Sickle Cell
Interventions
Cogmed RM
Behavioral
Lead sponsor
Steven J. Hardy
Other
Eligibility
7 Years to 16 Years
Enrollment
91 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2018
U.S. locations
1
States / cities
Washington D.C., District of Columbia
Source: ClinicalTrials.gov public record
Updated Nov 23, 2018 · Synced Oct 6, 2026, 4:13 PM EDT