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ClinicalTrials.gov public records Last synced Sep 30, 2026, 5:22 PM EDT

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Showing 49–72 of 10,514 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Congenital Disorder Clear all
Completed No phase listed Observational

Study of Thiotepa and TEPA Drug Exposure in Pediatric Hematopoietic Stem Cell Transplant Patients

NCT03609840
View directory record Official record
Conditions
Hematologic Malignancies, Nonmalignant Diseases, Immune Deficiency, Hemoglobinopathies, Genetic Inborn Errors of Metabolism, Fanconi Anemia, Thalassemia, Sickle Cell Disease
Interventions
Thiotepa
Drug
Lead sponsor
University of California, San Francisco
Other
Eligibility
Up to 17 Years
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2023
U.S. locations
1
States / cities
San Francisco, California
Source: ClinicalTrials.gov public record
Updated Sep 21, 2023 · Synced Sep 30, 2026, 5:22 PM EDT
Completed Not applicable Interventional

Study of Allogeneic Bone Marrow Transplantation Following Cyclophosphamide and Radiotherapy in Patients With Fanconi's Anemia

NCT00005891
View directory record Official record
Conditions
Fanconi's Anemia
Interventions
cyclophosphamide, Allogeneic Bone Marrow Transplantation
Drug · Procedure
Lead sponsor
Fairview University Medical Center
Other
Eligibility
0 Years to 54 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2000
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Jun 24, 2005 · Synced Sep 30, 2026, 5:22 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Acute Kidney Injury in Patients With Sickle Cell Disease

NCT03105271
View directory record Official record
Conditions
Sickle Cell Disease, Kidney Injury, Kidney Diseases, Kidney Disease, Chronic
Interventions
Not listed
Lead sponsor
University of Alabama at Birmingham
Other
Eligibility
1 Year to 25 Years
Enrollment
60 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2017 – 2022
U.S. locations
1
States / cities
Birmingham, Alabama
Source: ClinicalTrials.gov public record
Updated Feb 7, 2022 · Synced Sep 30, 2026, 5:22 PM EDT
Completed Not applicable Interventional

Randomized Control Trial: Synchronized Non-invasive Positive Pressure Ventilation Versus Non Synchronized Non Invasive Positive Pressure Ventilation in Extremely Low Birth Weight Infants

NCT03613987
View directory record Official record
Conditions
BPD - Bronchopulmonary Dysplasia
Interventions
NAVA technology to synchronize NIPPV
Device
Lead sponsor
New York Medical College
Other
Eligibility
24 Weeks to 30 Weeks
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2022
U.S. locations
1
States / cities
Valhalla, New York
Source: ClinicalTrials.gov public record
Updated Mar 27, 2023 · Synced Sep 30, 2026, 5:22 PM EDT
Active, not recruiting No phase listed Observational

Long Term Follow-Up for Safety of AVR-RD-02

NCT06488261
View directory record Official record
Conditions
Gaucher Disease
Interventions
AVR-RD-02
Biological
Lead sponsor
John Bernat
Other
Eligibility
18 Years and older
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2037
U.S. locations
1
States / cities
Iowa City, Iowa
Source: ClinicalTrials.gov public record
Updated Jul 22, 2025 · Synced Sep 30, 2026, 5:22 PM EDT
Active, not recruiting No phase listed Observational

ALTERRA Post-Approval Study

NCT05378386
View directory record Official record
Conditions
Pulmonary Valve Insufficiency, Complex Congenital Heart Defect, Tetralogy of Fallot, Pulmonary Regurgitation, RVOT Anomaly
Interventions
Edwards Alterra Adaptive Prestent with SAPIEN 3 Transcatheter Pulmonary Valve System
Device
Lead sponsor
Edwards Lifesciences
Industry
Eligibility
Not listed
Enrollment
150 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2035
U.S. locations
23
States / cities
Birmingham, Alabama • Little Rock, Arkansas • Los Angeles, California + 18 more
Source: ClinicalTrials.gov public record
Updated Nov 4, 2025 · Synced Sep 30, 2026, 5:22 PM EDT
Active, not recruiting Phase 3 Interventional

A Study of TAK-755 in Participants With Congenital Thrombotic Thrombocytopenic Purpura

NCT04683003
View directory record Official record
Conditions
Thrombotic Thrombocytopenic Purpura (TTP)
Interventions
TAK-755
Biological
Lead sponsor
Takeda
Industry
Eligibility
0 Years to 70 Years
Enrollment
77 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2027
U.S. locations
5
States / cities
Atlanta, Georgia • Buffalo, New York • Durham, North Carolina + 2 more
Source: ClinicalTrials.gov public record
Updated Apr 2, 2026 · Synced Sep 30, 2026, 5:22 PM EDT
Completed No phase listed Observational

Repository for Inherited Eye Diseases

NCT00378742
View directory record Official record
Conditions
Retinitis Pigmentosa, Inherited Ophthalmic Diseases
Interventions
Not listed
Lead sponsor
National Eye Institute (NEI)
NIH
Eligibility
1 Day to 100 Years
Enrollment
6,618 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2015
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Dec 8, 2025 · Synced Sep 30, 2026, 5:22 PM EDT
Terminated Not applicable Interventional Accepts healthy volunteers Results available

Plerixafor and Filgrastim For Mobilization of Donor Peripheral Blood Stem Cells Before A Donor Peripheral Blood Stem Cell Transplant in Treating Patients With Hematologic Malignancies

NCT01076270
View directory record Official record
Conditions
Accelerated Phase Chronic Myelogenous Leukemia, Adult Acute Lymphoblastic Leukemia in Remission, Adult Acute Myeloid Leukemia in Remission, Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities, 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), Atypical Chronic Myeloid Leukemia, BCR-ABL Negative, Blastic Phase Chronic Myelogenous Leukemia, Chronic Phase Chronic Myelogenous Leukemia, de Novo Myelodysplastic Syndromes, Extranodal Marginal Zone B-cell Lymphoma of Mucosa-associated Lymphoid Tissue, Myelodysplastic/Myeloproliferative Neoplasm, Unclassifiable, Nodal Marginal Zone B-cell Lymphoma, Noncontiguous Stage II Adult Burkitt Lymphoma, Noncontiguous Stage II Adult Diffuse Large Cell Lymphoma, Noncontiguous Stage II Adult Diffuse Mixed Cell Lymphoma, Noncontiguous Stage II Adult Diffuse Small Cleaved Cell Lymphoma, Noncontiguous Stage II Adult Immunoblastic Large Cell Lymphoma, Noncontiguous Stage II Adult Lymphoblastic Lymphoma, Noncontiguous Stage II Grade 1 Follicular Lymphoma, Noncontiguous Stage II Grade 2 Follicular Lymphoma, Noncontiguous Stage II Grade 3 Follicular Lymphoma, Noncontiguous Stage II Mantle Cell Lymphoma, Noncontiguous Stage II Marginal Zone Lymphoma, Noncontiguous Stage II Small Lymphocytic Lymphoma, Previously Treated Myelodysplastic Syndromes, Recurrent Adult Acute Lymphoblastic Leukemia, Recurrent Adult Acute Myeloid Leukemia, Recurrent Adult Burkitt Lymphoma, Recurrent Adult Diffuse Large Cell Lymphoma, Recurrent Adult Diffuse Mixed Cell Lymphoma, Recurrent Adult Diffuse Small Cleaved Cell Lymphoma, Recurrent Adult Hodgkin Lymphoma, Recurrent Adult Immunoblastic Large Cell Lymphoma, Recurrent Adult Lymphoblastic Lymphoma, Recurrent Cutaneous T-cell Non-Hodgkin Lymphoma, Recurrent Grade 1 Follicular Lymphoma, Recurrent Grade 2 Follicular Lymphoma, Recurrent Grade 3 Follicular Lymphoma, Recurrent Mantle Cell Lymphoma, Recurrent Marginal Zone Lymphoma, Recurrent Mycosis Fungoides/Sezary Syndrome, Recurrent Small Lymphocytic Lymphoma, Refractory Chronic Lymphocytic Leukemia, Refractory Hairy Cell Leukemia, Refractory Multiple Myeloma, Relapsing Chronic Myelogenous Leukemia, Secondary Acute Myeloid Leukemia, Secondary Myelodysplastic Syndromes, Splenic Marginal Zone Lymphoma, Stage I Multiple Myeloma, Stage II Multiple Myeloma, Stage III Adult Burkitt Lymphoma, Stage III Adult Diffuse Large Cell Lymphoma, Stage III Adult Diffuse Mixed Cell Lymphoma, Stage III Adult Diffuse Small Cleaved Cell Lymphoma, Stage III Adult Hodgkin Lymphoma, Stage III Adult Immunoblastic Large Cell Lymphoma, Stage III Adult Lymphoblastic Lymphoma, Stage III Chronic Lymphocytic Leukemia, Stage III Grade 1 Follicular Lymphoma, Stage III Grade 2 Follicular Lymphoma, Stage III Grade 3 Follicular Lymphoma, Stage III Mantle Cell Lymphoma, Stage III Marginal Zone Lymphoma, Stage III Multiple Myeloma, Stage III Small Lymphocytic Lymphoma, Stage IV Adult Burkitt Lymphoma, Stage IV Adult Diffuse Large Cell Lymphoma, Stage IV Adult Diffuse Mixed Cell Lymphoma, Stage IV Adult Diffuse Small Cleaved Cell Lymphoma, Stage IV Adult Hodgkin Lymphoma, Stage IV Adult Immunoblastic Large Cell Lymphoma, Stage IV Adult Lymphoblastic Lymphoma, Stage IV Chronic Lymphocytic Leukemia, Stage IV Grade 1 Follicular Lymphoma, Stage IV Grade 2 Follicular Lymphoma, Stage IV Grade 3 Follicular Lymphoma, Stage IV Mantle Cell Lymphoma, Stage IV Marginal Zone Lymphoma, Stage IV Small Lymphocytic Lymphoma
Interventions
plerixafor, filgrastim, peripheral blood stem cell transplantation, allogeneic hematopoietic stem cell transplantation
Drug · Biological · Procedure
Lead sponsor
Fred Hutchinson Cancer Center
Other
Eligibility
18 Years to 65 Years
Enrollment
1 participant
Healthy volunteers
Accepts healthy volunteers
Timeline
2010 – 2011
U.S. locations
1
States / cities
Seattle, Washington
Source: ClinicalTrials.gov public record
Updated Jun 28, 2017 · Synced Sep 30, 2026, 5:22 PM EDT
Completed No phase listed Observational

Technical Development of Cardiovascular Magnetic Resonance Imaging

NCT00027170
View directory record Official record
Conditions
Cardiomyopathy, Congenital Heart Disease, Cardiovascular Disease, Cardiac Magnetic Resonance Imaging, Acute Myocardial Infarction
Interventions
Siemens MRI scanner
Device
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
18 Years to 100 Years
Enrollment
8,781 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2020
U.S. locations
2
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 30, 2021 · Synced Sep 30, 2026, 5:22 PM EDT
Terminated No phase listed Observational

Heart Failure in Adult Patients With a History of Congenital Heart Disease

NCT00208754
View directory record Official record
Conditions
Congenital Heart Defects
Interventions
Not listed
Lead sponsor
Emory University
Other
Eligibility
Not listed
Enrollment
350 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2005
U.S. locations
1
States / cities
Atlanta, Georgia
Source: ClinicalTrials.gov public record
Updated Nov 13, 2013 · Synced Sep 30, 2026, 5:22 PM EDT
Terminated Phase 2Phase 3 Interventional Results available

Study of Inhaled Nitric Oxide (iNO) and Respiratory Outcomes in Late Preterm Infants

NCT01748045
View directory record Official record
Conditions
Bronchopulmonary Dysplasia
Interventions
inhaled nitric oxide, Placebo Comparator - nitrogen gas
Drug
Lead sponsor
Tufts Medical Center
Other
Eligibility
30 Weeks to 36 Weeks
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2014
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Jul 19, 2017 · Synced Sep 30, 2026, 5:22 PM EDT
Completed Phase 2 Interventional Results available

Safety and Efficacy of AFQ056 in Adolescent Patients With Fragile X Syndrome

NCT01357239
View directory record Official record
Conditions
Fragile X Syndrome
Interventions
AFQ056, Placebo
Drug
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
12 Years to 17 Years
Enrollment
139 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2014
U.S. locations
8
States / cities
Sacramento, California • Decatur, Georgia • Chicago, Illinois + 5 more
Source: ClinicalTrials.gov public record
Updated May 12, 2015 · Synced Sep 30, 2026, 5:22 PM EDT
Completed Phase 3 Interventional Results available

A Randomized Study in Children Ages Birth Through Three Years Undergoing Unilateral Cataract Extraction With or Without Lens Replacement

NCT02132312
View directory record Official record
Conditions
Unilateral Cataract Extraction, Congenital Cataract
Interventions
OMS302, Phenylephrine HCl
Drug
Lead sponsor
Omeros Corporation
Industry
Eligibility
1 Day to 47 Months
Enrollment
78 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2016
U.S. locations
11
States / cities
Los Angeles, California • Atlanta, Georgia • Chicago, Illinois + 8 more
Source: ClinicalTrials.gov public record
Updated Jul 22, 2021 · Synced Sep 30, 2026, 5:22 PM EDT
Completed No phase listed Observational

Evaluation of Lung T1-MRI in Pediatric Cystic Fibrosis Patients

NCT04994301
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
Lung T1 MRI
Diagnostic Test
Lead sponsor
University Hospitals Cleveland Medical Center
Other
Eligibility
5 Years to 11 Years
Enrollment
48 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2023
U.S. locations
3
States / cities
Indianapolis, Indiana • Ann Arbor, Michigan • Cleveland, Ohio
Source: ClinicalTrials.gov public record
Updated Sep 14, 2026 · Synced Sep 30, 2026, 5:22 PM EDT
Completed Phase 2 Interventional Results available

Safety Tolerability and Efficacy Study of Cabaletta to Treat Oculopharyngeal Muscular Dystrophy (OPMD) Patients

NCT02015481
View directory record Official record
Conditions
Oculopharyngeal Muscular Dystrophy
Interventions
Cabaletta
Drug
Lead sponsor
Bioblast Pharma Ltd.
Industry
Eligibility
18 Years to 80 Years
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2016
U.S. locations
2
States / cities
Los Angeles, California • Orange, California
Source: ClinicalTrials.gov public record
Updated Oct 17, 2017 · Synced Sep 30, 2026, 5:22 PM EDT
Withdrawn No phase listed Observational

Measurement of Carboxyhemoglobin by Gas Chromatography as an Index of Hemolysis

NCT00917007
View directory record Official record
Conditions
ABO Incompatibility, Hemolytic Disease of Newborn, Neonatal Jaundice, Neonatal Hyperbilirubinemia
Interventions
Not listed
Lead sponsor
State University of New York - Upstate Medical University
Other
Eligibility
Up to 28 Days
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2010
U.S. locations
2
States / cities
Syracuse, New York
Source: ClinicalTrials.gov public record
Updated Oct 25, 2021 · Synced Sep 30, 2026, 5:22 PM EDT
Enrolling by invitation No phase listed Observational

Point of Care Lung Ultrasound in Preterm Infants With Respiratory Distress

NCT05925075
View directory record Official record
Conditions
Bronchopulmonary Dysplasia
Interventions
Lung Ultrasound
Diagnostic Test
Lead sponsor
Phoenix Children's Hospital
Other
Eligibility
0 Days and older
Enrollment
150 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2027
U.S. locations
1
States / cities
Phoenix, Arizona
Source: ClinicalTrials.gov public record
Updated May 22, 2026 · Synced Sep 30, 2026, 5:22 PM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Natural History Evaluation of Charcot Marie Tooth Disease (CMT) Types CMT1B, CMT2A, CMT4A, CMT4C, and Others

NCT01193075
View directory record Official record
Conditions
Charcot Marie Tooth Disease
Interventions
Not listed
Lead sponsor
Michael Shy
Other
Eligibility
Not listed
Enrollment
5,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2010 – 2026
U.S. locations
18
States / cities
Los Angeles, California • Palo Alto, California • Aurora, Colorado + 13 more
Source: ClinicalTrials.gov public record
Updated Oct 7, 2025 · Synced Sep 30, 2026, 5:22 PM EDT
Completed No phase listed Observational

The Impact of Community-based Wellness Programs on The Triple Aim

NCT02938130
View directory record Official record
Conditions
Spina Bifida, Cerebral Palsy, Spinal Cord Injury, Traumatic Brain Injury
Interventions
Wellness Programs
Other
Lead sponsor
University of Pittsburgh
Other
Eligibility
7 Years and older
Enrollment
130 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2023
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Sep 3, 2026 · Synced Sep 30, 2026, 5:22 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Evaluation and Treatment of Patients With Connective Tissue Disease

NCT00076830
View directory record Official record
Conditions
Connective Tissue Disorders, Osteogenesis Imperfecta, Bone Diseases, Metabolic, Melorheostosis
Interventions
Not listed
Lead sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
NIH
Eligibility
Up to 70 Years
Enrollment
180 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
Started 2004
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 28, 2026 · Synced Sep 30, 2026, 5:22 PM EDT
Completed Phase 2 Interventional Results available

Lenalidomide in Treating Older Patients With Acute Myeloid Leukemia

NCT00352365
View directory record Official record
Conditions
Adult Acute Basophilic Leukemia, Adult Acute Eosinophilic Leukemia, Adult Acute Megakaryoblastic Leukemia (M7), Adult Acute Minimally Differentiated Myeloid Leukemia (M0), Adult Acute Monoblastic Leukemia (M5a), Adult Acute Monocytic Leukemia (M5b), Adult Acute Myeloblastic Leukemia With Maturation (M2), Adult Acute Myeloblastic Leukemia Without Maturation (M1), Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities, Adult Acute Myeloid Leukemia With Inv(16)(p13;q22), Adult Acute Myeloid Leukemia With t(16;16)(p13;q22), Adult Acute Myeloid Leukemia With t(8;21)(q22;q22), Adult Acute Myelomonocytic Leukemia (M4), Adult Erythroleukemia (M6a), Adult Pure Erythroid Leukemia (M6b), Secondary Acute Myeloid Leukemia, Untreated Adult Acute Myeloid Leukemia
Interventions
lenalidomide
Drug
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
60 Years and older
Enrollment
41 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2011
U.S. locations
54
States / cities
Little Rock, Arkansas • Redding, California • Roseville, California + 30 more
Source: ClinicalTrials.gov public record
Updated Feb 11, 2022 · Synced Sep 30, 2026, 5:22 PM EDT
Active, not recruiting No phase listed Observational

Genetic Modifiers of Cystic Fibrosis (CF) Liver Disease

NCT00804583
View directory record Official record
Conditions
Cystic Fibrosis, Liver Disease
Interventions
Not listed
Lead sponsor
University of North Carolina, Chapel Hill
Other
Eligibility
2 Years and older
Enrollment
154 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2027
U.S. locations
1
States / cities
Chapel Hill, North Carolina
Source: ClinicalTrials.gov public record
Updated Sep 23, 2025 · Synced Sep 30, 2026, 5:22 PM EDT
Recruiting Phase 1 Interventional

Feasibility and Tolerability of IMLYGIC for the Treatment of Cutaneous Neurofibromas in Adults With NF1

NCT07102394
View directory record Official record
Conditions
NF1, Neurofibromatosis, Cutaneous Neurofibroma, Neurofibroma, Von Recklinghausen Disease, Neurofibromas
Interventions
Imlygic
Drug
Lead sponsor
Johns Hopkins University
Other
Eligibility
18 Years and older
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2027
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated May 8, 2026 · Synced Sep 30, 2026, 5:22 PM EDT