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ClinicalTrials.gov public records Last synced Oct 15, 2026, 5:10 AM EDT

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Showing 553–576 of 10,529 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Congenital Diseases Clear all
Active, not recruiting Not applicable Interventional

AMPLATZER PFO Occluder Post Approval Study

NCT03309332
View directory record Official record
Conditions
Stroke, Patent Foramen Ovale
Interventions
AMPLATZER™ PFO Occluder
Device
Lead sponsor
Abbott Medical Devices
Industry
Eligibility
18 Years to 60 Years
Enrollment
1,214 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2030
U.S. locations
78
States / cities
Birmingham, Alabama • Huntsville, Alabama • Chandler, Arizona + 63 more
Source: ClinicalTrials.gov public record
Updated Mar 11, 2026 · Synced Oct 15, 2026, 5:10 AM EDT
Completed Phase 2 Interventional Results available

Pharmacokinetic and Dose Response Study of Asfotase Alfa in Adult Patients With Pediatric-Onset Hypophosphatasia (HPP)

NCT02797821
View directory record Official record
Conditions
Hypophosphatasia
Interventions
Asfotase alfa
Drug
Lead sponsor
Alexion Pharmaceuticals, Inc.
Industry
Eligibility
18 Years and older
Enrollment
27 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2017
U.S. locations
3
States / cities
St Louis, Missouri • Durham, North Carolina • Nashville, Tennessee
Source: ClinicalTrials.gov public record
Updated Sep 17, 2019 · Synced Oct 15, 2026, 5:10 AM EDT
Active, not recruiting Not applicable Interventional Results available

Sickle Cell Disease (SCD) Bone Pain Study

NCT05283148
View directory record Official record
Conditions
Sickle Cell Disease, Sickle Cell Anemia, Low Bone Density, Osteoporosis, Osteopenia, Vertebral Fracture, Vertebral Compression, Osteonecrosis, Ischemic Necrosis, Avascular Necrosis
Interventions
Dual-energy X-ray absorptiometry, Vertebral fracture analysis, Adult Sickle Cell Quality of Life Measurement System pain impact questionnaire
Other
Lead sponsor
University of California, Davis
Other
Eligibility
18 Years to 80 Years
Enrollment
53 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2027
U.S. locations
1
States / cities
Sacramento, California
Source: ClinicalTrials.gov public record
Updated Sep 23, 2026 · Synced Oct 15, 2026, 5:10 AM EDT
Completed Phase 2 Interventional Accepts healthy volunteers Results available

Antioxidant Therapy With N-acetylcysteine for Children With Neurofibromatosis Type 1

NCT04481048
View directory record Official record
Conditions
Neurofibromatosis 1
Interventions
N-Acetyl cysteine, Placebo
Drug · Other
Lead sponsor
Children's Hospital Medical Center, Cincinnati
Other
Eligibility
8 Years to 16 Years
Enrollment
25 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2021 – 2024
U.S. locations
1
States / cities
Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Jun 5, 2026 · Synced Oct 15, 2026, 5:10 AM EDT
Recruiting Not applicable Interventional

BOND: Direct Breastfeeding to Enhance Maternal and Infant Health in Congenital Heart Disease

NCT07582848
View directory record Official record
Conditions
Congenital Heart Disease (CHD)
Interventions
Breastfeeding clinical practice guideline (CPG) for congenital heart disease (CHD)
Other
Lead sponsor
Carelon Research
Other
Eligibility
18 Years and older
Enrollment
266 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2030
U.S. locations
17
States / cities
Phoenix, Arizona • Los Angeles, California • Aurora, Colorado + 13 more
Source: ClinicalTrials.gov public record
Updated Sep 10, 2026 · Synced Oct 15, 2026, 5:10 AM EDT
Recruiting Phase 3 Interventional

Pegtibatinase as a Treatment for Participants With Classical Homocystinuria (HCU) Receiving Standard of Care Treatment

NCT06247085
View directory record Official record
Conditions
Homocystinuria
Interventions
Pegtibatinase, Placebo
Drug · Other
Lead sponsor
Travere Therapeutics, Inc.
Industry
Eligibility
12 Years to 65 Years
Enrollment
70 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2027
U.S. locations
13
States / cities
Phoenix, Arizona • New Haven, Connecticut • Atlanta, Georgia + 10 more
Source: ClinicalTrials.gov public record
Updated Oct 8, 2026 · Synced Oct 15, 2026, 5:10 AM EDT
Completed Phase 1Phase 2 Interventional Accepts healthy volunteers

A Single-ascending Dose (Part A) and Repeat-dose (Part B) Study to Investigate the Safety, Pharmacokinetics and Efficacy (Part B Only) of UCB1381 in Healthy Study Participants (Part A) and in Study Participants With Moderate to Severe Atopic Dermatitis (Part B)

NCT05277571
View directory record Official record
Conditions
Atopic Dermatitis
Interventions
UCB1381, Placebo
Biological · Drug
Lead sponsor
UCB Biopharma SRL
Industry
Eligibility
18 Years to 65 Years
Enrollment
109 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2022 – 2025
U.S. locations
16
States / cities
Beverly Hills, California • Glendale, California • Los Angeles, California + 13 more
Source: ClinicalTrials.gov public record
Updated Jul 22, 2026 · Synced Oct 15, 2026, 5:10 AM EDT
Recruiting Not applicable Interventional Accepts healthy volunteers

Dynamic Critical Congenital Heart Screening With Addition of Perfusion Measurements

NCT05637814
View directory record Official record
Conditions
Congenital Heart Disease
Interventions
SpO2/PIx Measurement and ML Algorithm
Diagnostic Test
Lead sponsor
University of California, Davis
Other
Eligibility
0 Minutes to 21 Days
Enrollment
320 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2023 – 2027
U.S. locations
3
States / cities
Davis, California • Queens, New York • Salt Lake City, Utah
Source: ClinicalTrials.gov public record
Updated Jun 18, 2026 · Synced Oct 15, 2026, 5:10 AM EDT
Recruiting No phase listed Observational

Cognitive Function in Rett Syndrome During Trofinetide Treatment

NCT06705816
View directory record Official record
Conditions
Rett Syndrome
Interventions
None interventional
Other
Lead sponsor
University of Minnesota
Other
Eligibility
18 Years to 99 Years
Enrollment
20 participants
Timeline
2024 – 2027
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Jun 18, 2026 · Synced Oct 15, 2026, 5:10 AM EDT
Completed Not applicable Interventional

Sedation Management in Pediatric Patients With Acute Respiratory Failure (The RESTORE Study)

NCT00814099
View directory record Official record
Conditions
Respiratory Insufficiency, Respiratory Distress Syndrome, Newborn, Lung Diseases
Interventions
Team approach to sedation management, Usual approach to sedation management
Behavioral
Lead sponsor
University of Pennsylvania
Other
Eligibility
2 Weeks to 18 Years
Enrollment
2,449 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2013
U.S. locations
31
States / cities
Birmingham, Alabama • Tucson, Arizona • Oakland, California + 26 more
Source: ClinicalTrials.gov public record
Updated May 1, 2026 · Synced Oct 15, 2026, 5:10 AM EDT
Completed Phase 2 Interventional Results available

Sirolimus and Azacitidine in Treating Patients With High Risk Myelodysplastic Syndrome or Acute Myeloid Leukemia That is Recurrent or Not Eligible for Intensive Chemotherapy

NCT01869114
View directory record Official record
Conditions
Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities, Adult Acute Myeloid Leukemia With Del(5q), Adult Acute Myeloid Leukemia With Inv(16)(p13;q22), Adult Acute Myeloid Leukemia With t(15;17)(q22;q12), Adult Acute Myeloid Leukemia With t(16;16)(p13;q22), Adult Acute Myeloid Leukemia With t(8;21)(q22;q22), de Novo Myelodysplastic Syndromes, Myelodysplastic Syndrome With Isolated Del(5q), Previously Treated Myelodysplastic Syndromes, Recurrent Adult Acute Myeloid Leukemia
Interventions
Sirolimus, Azacitidine
Drug
Lead sponsor
Sidney Kimmel Comprehensive Cancer Center at Thomas Jefferson University
Other
Eligibility
18 Years and older
Enrollment
57 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2024
U.S. locations
5
States / cities
Sewell, New Jersey • Abington, Pennsylvania • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Dec 10, 2025 · Synced Oct 15, 2026, 5:10 AM EDT
Withdrawn No phase listed Observational

Ultrasound to Predict Steal-Syndrome After Arteriovenous-Fistula Creation (UPSAC - Trial)

NCT01468506
View directory record Official record
Conditions
Chronic Kidney Disease
Interventions
Not listed
Lead sponsor
Baptist Health South Florida
Other
Eligibility
18 Years and older
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2014
U.S. locations
1
States / cities
Miami, Florida
Source: ClinicalTrials.gov public record
Updated Sep 25, 2014 · Synced Oct 15, 2026, 5:10 AM EDT
Completed No phase listed Observational

Physical and Mental Assessment of Older Patients With Newly Diagnosed Acute Myeloid Leukemia

NCT00499785
View directory record Official record
Conditions
Cognitive/Functional Effects, Depression, Leukemia
Interventions
Not listed
Lead sponsor
Wake Forest University Health Sciences
Other
Eligibility
60 Years to 120 Years
Enrollment
11 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2012
U.S. locations
1
States / cities
Winston-Salem, North Carolina
Source: ClinicalTrials.gov public record
Updated May 30, 2017 · Synced Oct 15, 2026, 5:10 AM EDT
Completed Phase 3 Interventional

A Randomized Trial of Unruptured Brain AVMs

NCT00389181
View directory record Official record
Conditions
Arteriovenous Malformations, Cerebral
Interventions
Interventional therapy, Medical management
Procedure · Other
Lead sponsor
Columbia University
Other
Eligibility
18 Years and older
Enrollment
226 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2015
U.S. locations
25
States / cities
Phoenix, Arizona • Los Angeles, California • Redwood City, California + 20 more
Source: ClinicalTrials.gov public record
Updated Jun 4, 2015 · Synced Oct 15, 2026, 5:10 AM EDT
Terminated No phase listed Observational

A Long-term Follow-up Study of Patients With MPS IIIB Treated With ABO-101

NCT04655911
View directory record Official record
Conditions
Mucopolysaccharidosis III-B
Interventions
ABO-101
Biological
Lead sponsor
Abeona Therapeutics, Inc
Industry
Eligibility
Not listed
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2022
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated May 31, 2022 · Synced Oct 15, 2026, 5:10 AM EDT
Completed Not applicable Interventional Accepts healthy volunteers Results available

Phone-Based Based Walk With Ease Program for Adults With Arthritis

NCT05289544
View directory record Official record
Conditions
Arthritis, Gout, Fibromyalgia, Rheumatoid Arthritis, Osteoarthritis, Lupus Erythematosus, Systemic
Interventions
Phone-Based Walk With Ease
Behavioral
Lead sponsor
University of South Carolina
Other
Eligibility
18 Years and older
Enrollment
267 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2022 – 2024
U.S. locations
1
States / cities
Columbia, South Carolina
Source: ClinicalTrials.gov public record
Updated Mar 6, 2026 · Synced Oct 15, 2026, 5:10 AM EDT
Completed Phase 3 Interventional Results available

Trial of Eflornithine Plus Sulindac in Patients With Familial Adenomatous Polyposis (FAP)

NCT01483144
View directory record Official record
Conditions
Familial Adenomatous Polyposis
Interventions
Eflornithine, Eflornithine Placebo, Sulindac 150 MG, Sulindac placebo
Drug
Lead sponsor
Cancer Prevention Pharmaceuticals, Inc.
Industry
Eligibility
18 Years and older
Enrollment
171 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2019
U.S. locations
10
States / cities
La Jolla, California • Atlanta, Georgia • Boston, Massachusetts + 7 more
Source: ClinicalTrials.gov public record
Updated Jun 8, 2021 · Synced Oct 15, 2026, 5:10 AM EDT
Active, not recruiting Phase 1 Interventional

CNS10-NPC for the Treatment of RP

NCT04284293
View directory record Official record
Conditions
Retinitis Pigmentosa
Interventions
CNS10-NPC implantation
Biological
Lead sponsor
Cedars-Sinai Medical Center
Other
Eligibility
18 Years and older
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2026
U.S. locations
1
States / cities
Beverly Hills, California
Source: ClinicalTrials.gov public record
Updated Aug 19, 2025 · Synced Oct 15, 2026, 5:10 AM EDT
Recruiting Phase 1 Interventional

B7-H3-Specific Chimeric Antigen Receptor Autologous T-Cell Therapy for Pediatric Patients With Solid Tumors (3CAR)

NCT04897321
View directory record Official record
Conditions
Pediatric Solid Tumor, Osteosarcoma, Rhabdomyosarcoma, Neuroblastoma, Ewing Sarcoma, Wilms Tumor, Adrenocortical Cancer, Desmoplastic Small Round Cell Tumor, Germ Cell Cancer, Rhabdoid Tumor, Clear Cell Sarcoma, Hepatoblastoma, Melanoma, Carcinoma, Malignant Peripheral Nerve Sheath Tumors, Soft Tissue Sarcoma
Interventions
Fludarabine, Cyclophosphamide, MESNA, B7-H3 CAR T cells
Drug
Lead sponsor
St. Jude Children's Research Hospital
Other
Eligibility
Up to 21 Years
Enrollment
48 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2028
U.S. locations
1
States / cities
Memphis, Tennessee
Source: ClinicalTrials.gov public record
Updated May 19, 2026 · Synced Oct 15, 2026, 5:10 AM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

A Trial to Evaluate Pharmacokinetics, Immunogenicity, Safety, and Tolerability of LEO 138559 in Healthy Japanese Subjects

NCT05099133
View directory record Official record
Conditions
Atopic Dermatitis
Interventions
LEO 138559, LEO 138559 Placebo
Drug
Lead sponsor
LEO Pharma
Industry
Eligibility
18 Years to 65 Years
Enrollment
24 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2021 – 2022
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Feb 24, 2025 · Synced Oct 15, 2026, 5:10 AM EDT
Terminated No phase listed Observational

Chylothorax Following Heart Surgery

NCT00215098
View directory record Official record
Conditions
Chylothorax
Interventions
Not listed
Lead sponsor
Children's Healthcare of Atlanta
Other
Eligibility
Not listed
Enrollment
70 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2006
U.S. locations
1
States / cities
Atlanta, Georgia
Source: ClinicalTrials.gov public record
Updated Mar 16, 2012 · Synced Oct 15, 2026, 5:10 AM EDT
Completed Phase 1 Interventional Results available

A Pharmacokinetic and Safety Study of IV Gallium Nitrate (Ganite) in Cystic Fibrosis Patients

NCT01093521
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
100 mg/m2 dose, 200 mg/m2 dose
Drug
Lead sponsor
University of Washington
Other
Eligibility
18 Years to 55 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2013
U.S. locations
3
States / cities
Iowa City, Iowa • Baltimore, Maryland • Seattle, Washington
Source: ClinicalTrials.gov public record
Updated Nov 29, 2022 · Synced Oct 15, 2026, 5:10 AM EDT
Completed Phase 2 Interventional Results available

Probiotic Prophylaxis of Hirschprung's Disease Associated Enterocolitis (HAEC)

NCT00630838
View directory record Official record
Conditions
Hirschsprung Disease
Interventions
VSL#3, Placebo
Drug
Lead sponsor
University of Michigan
Other
Eligibility
1 Month to 12 Months
Enrollment
62 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2010
U.S. locations
2
States / cities
Ann Arbor, Michigan • Buffalo, New York
Source: ClinicalTrials.gov public record
Updated Jul 19, 2016 · Synced Oct 15, 2026, 5:10 AM EDT
Completed No phase listed Observational

Liver Fibrosis in Alpha-1 Antitrypsin Deficiency (Liver AATD)

NCT01810458
View directory record Official record
Conditions
Liver Fibrosis, Alpha-1 Antitrypsin Deficiency, AAT Deficiency, AATD
Interventions
Abdominal ultrasound, History and physical, Intravenous catheter, Blood draw, Liver questionnaire, Liver Biopsy, Midazolam, Fentanyl, Lidocaine, Acetaminophen, Lorazepam, Oxycodone/Acetaminophen, Ondansetron
Device · Procedure · Other + 1 more
Lead sponsor
University of Florida
Other
Eligibility
18 Years to 70 Years
Enrollment
109 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2019
U.S. locations
1
States / cities
Gainesville, Florida
Source: ClinicalTrials.gov public record
Updated Aug 21, 2023 · Synced Oct 15, 2026, 5:10 AM EDT