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ClinicalTrials.gov public records Last synced Oct 25, 2026, 4:46 PM EDT

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Showing 697–720 of 10,333 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Congenital, Hereditary, and Neonatal Diseases and Abnormalities Clear all
Recruiting No phase listed Observational

Genetic Markers of Cardiovascular Disease in Epilepsy

NCT02824822
View directory record Official record
Conditions
Epilepsy, Seizures, Syncope, Channelopathy, Cardiomyopathies
Interventions
Not listed
Lead sponsor
Mayo Clinic
Other
Eligibility
18 Years to 50 Years
Enrollment
600 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2031
U.S. locations
1
States / cities
Rochester, Minnesota
Source: ClinicalTrials.gov public record
Updated Sep 8, 2025 · Synced Oct 25, 2026, 4:46 PM EDT
Completed Not applicable Interventional Results available

A Pilot Study of HSCT for Patients With High-risk Hemoglobinopathy Using a Nonmyeloablative Preparative Regimen

NCT00427661
View directory record Official record
Conditions
Sickle Cell Disease, Thalassemia, Hemoglobinopathies
Interventions
Busulfan; Fludarabine; cyclosporine A and MMF
Other
Lead sponsor
University of Pittsburgh
Other
Eligibility
3 Years to 35 Years
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2002 – 2014
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Aug 18, 2016 · Synced Oct 25, 2026, 4:46 PM EDT
Terminated No phase listed Observational

Managing Cyanosis Following Bidirectional Glenn Operation

NCT00327886
View directory record Official record
Conditions
Congenital Disorders
Interventions
Not listed
Lead sponsor
Children's Healthcare of Atlanta
Other
Eligibility
Up to 1 Year
Enrollment
150 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006
U.S. locations
1
States / cities
Atlanta, Georgia
Source: ClinicalTrials.gov public record
Updated Jun 13, 2007 · Synced Oct 25, 2026, 4:46 PM EDT
Completed Phase 1 Interventional

Rebeccamycin Analog in Treating Patients With Relapsed or Refractory Acute Myeloid Leukemia, Myelodysplastic Syndrome, Acute Lymphoblastic Leukemia, or Chronic Myelogenous Leukemia

NCT00087204
View directory record Official record
Conditions
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), Blastic Phase Chronic Myelogenous Leukemia, Chronic Myelomonocytic Leukemia, de Novo Myelodysplastic Syndromes, Previously Treated Myelodysplastic Syndromes, Recurrent Adult Acute Lymphoblastic Leukemia, Recurrent Adult Acute Myeloid Leukemia, Refractory Anemia With Excess Blasts, Refractory Anemia With Excess Blasts in Transformation, Relapsing Chronic Myelogenous Leukemia, Secondary Acute Myeloid Leukemia, Secondary Myelodysplastic Syndromes
Interventions
becatecarin, laboratory biomarker analysis
Drug · Other
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
18 Years and older
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2004
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Jan 23, 2013 · Synced Oct 25, 2026, 4:46 PM EDT
Terminated Phase 3 Interventional Results available

Open-label Extension Study of Pramipexole in the Treatment of Children and Adolescents With Tourette Syndrome

NCT00681863
View directory record Official record
Conditions
Tourette Syndrome
Interventions
pramipexole 0.125 mg BID, pramipexole 0.0625 mg QD, pramipexole 0.125 mg TID, pramipexole 0.25 mg BID, pramipexole 0.0625 mg BID
Drug
Lead sponsor
Boehringer Ingelheim
Industry
Eligibility
6 Years to 17 Years
Enrollment
45 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2008
U.S. locations
13
States / cities
Bradenton, Florida • Tampa, Florida • Columbus, Georgia + 9 more
Source: ClinicalTrials.gov public record
Updated May 23, 2014 · Synced Oct 25, 2026, 4:46 PM EDT
Completed Not applicable Interventional Results available

Palifermin in Preventing Chronic Graft-Versus-Host Disease in Patients Who Have Undergone Donor Stem Cell Transplant for Hematologic Cancer

NCT01233921
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 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), Atypical Chronic Myeloid Leukemia, BCR-ABL1 Negative, Blastic Phase Chronic Myelogenous Leukemia, Chronic Eosinophilic Leukemia, Chronic Myelomonocytic Leukemia, Chronic Neutrophilic Leukemia, Chronic Phase Chronic Myelogenous Leukemia, de Novo Myelodysplastic Syndromes, Extranodal Marginal Zone B-cell Lymphoma of Mucosa-associated Lymphoid Tissue, Graft Versus Host Disease, 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, Primary Myelofibrosis, 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
palifermin, flow cytometry, laboratory biomarker analysis, pharmacological study
Biological · Other
Lead sponsor
Martin, Paul
Other
Eligibility
18 Years and older
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2010
U.S. locations
1
States / cities
Seattle, Washington
Source: ClinicalTrials.gov public record
Updated Apr 2, 2014 · Synced Oct 25, 2026, 4:46 PM EDT
Not listed Not applicable Interventional Accepts healthy volunteers

Open Trial of Telehealth Group for Improving Peer Relationships (PEERS) in NF1

NCT04856514
View directory record Official record
Conditions
Neurofibromatosis 1, Social Skills
Interventions
PEERS
Behavioral
Lead sponsor
University of Wisconsin, Milwaukee
Other
Eligibility
12 Years to 17 Years
Enrollment
8 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2020 – 2021
U.S. locations
1
States / cities
Milwaukee, Wisconsin
Source: ClinicalTrials.gov public record
Updated Apr 27, 2021 · Synced Oct 25, 2026, 4:46 PM EDT
Not yet recruiting No phase listed Observational

Pharmacokinetics of CFTR Modulators in Pregnant Individuals and in Postpartum Breastfeeding Mothers

NCT04940533
View directory record Official record
Conditions
Cystic Fibrosis, Pregnancy
Interventions
Not listed
Lead sponsor
University of Minnesota
Other
Eligibility
18 Years and older · Female only
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2027 – 2028
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Jan 20, 2026 · Synced Oct 25, 2026, 4:46 PM EDT
Completed Phase 3 Interventional Results available

Efficacy & Safety Study of Once-weekly Oral Aripiprazole in Children and Adolescents With Tourette's Disorder (TD)

NCT01418352
View directory record Official record
Conditions
Tourette's Disorder
Interventions
Aripiprazole, Placebo
Drug
Lead sponsor
Otsuka Pharmaceutical Development & Commercialization, Inc.
Industry
Eligibility
7 Years to 17 Years
Enrollment
83 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2014
U.S. locations
13
States / cities
Dothan, Alabama • San Francisco, California • Gainesville, Florida + 9 more
Source: ClinicalTrials.gov public record
Updated Oct 7, 2021 · Synced Oct 25, 2026, 4:46 PM EDT
Completed Phase 1Phase 2 Interventional Results available

Pharmacokinetic and Safety Trial of Intravenous Levetiracetam in the Treatment of Neonatal Seizures

NCT00884052
View directory record Official record
Conditions
Seizures, Disorder of Fetus or Newborn
Interventions
Low dose levetiracetam, High dose levetiracetam
Drug
Lead sponsor
Richard H. Haas
Other
Eligibility
1 Minute to 14 Days
Enrollment
18 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2011
U.S. locations
1
States / cities
San Diego, California
Source: ClinicalTrials.gov public record
Updated Mar 26, 2020 · Synced Oct 25, 2026, 4:46 PM EDT
Completed No phase listed Observational

Pain, Exercise and Psychological Well-being in Pectus Excavatum

NCT02337621
View directory record Official record
Conditions
Pectus Excavatum
Interventions
No intervention
Other
Lead sponsor
Phoenix Children's Hospital
Other
Eligibility
12 Years to 17 Years
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2021
U.S. locations
1
States / cities
Phoenix, Arizona
Source: ClinicalTrials.gov public record
Updated Apr 25, 2022 · Synced Oct 25, 2026, 4:46 PM EDT
Completed Phase 1 Interventional Results available

Safety and Pharmacokinetic Study of N6022 in Subjects With Cystic Fibrosis Homozygous for the F508del-CFTR Mutation

NCT01746784
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
N6022, Normal saline
Drug
Lead sponsor
Nivalis Therapeutics, Inc.
Industry
Eligibility
18 Years and older
Enrollment
66 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014
U.S. locations
17
States / cities
Birmingham, Alabama • Anchorage, Alaska • Palo Alto, California + 14 more
Source: ClinicalTrials.gov public record
Updated Nov 24, 2014 · Synced Oct 25, 2026, 4:46 PM EDT
Completed Phase 2 Interventional Results available

A Long-Term Study of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency

NCT02004704
View directory record Official record
Conditions
Sphingomyelin Lipidosis
Interventions
GZ402665
Drug
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
Not listed
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2023
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jul 3, 2024 · Synced Oct 25, 2026, 4:46 PM EDT
Completed Phase 1 Interventional

Glucocorticoid Administration After Traumatic Birth

NCT04852458
View directory record Official record
Conditions
Traumatic Birth
Interventions
Intravenous (IV) hydrocortisone
Drug
Lead sponsor
University of Michigan
Other
Eligibility
18 Years and older · Female only
Enrollment
133 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2022
U.S. locations
1
States / cities
Ann Arbor, Michigan
Source: ClinicalTrials.gov public record
Updated Nov 9, 2022 · Synced Oct 25, 2026, 4:46 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Frailty in Children With Cardiac Disease

NCT02999438
View directory record Official record
Conditions
Pulmonary Hypertension, Heart Failure, Single Ventricle
Interventions
Actigraph to check activity levels at home
Device
Lead sponsor
Children's Mercy Hospital Kansas City
Other
Eligibility
8 Years to 18 Years
Enrollment
56 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2016 – 2022
U.S. locations
1
States / cities
Kansas City, Missouri
Source: ClinicalTrials.gov public record
Updated Jul 14, 2023 · Synced Oct 25, 2026, 4:46 PM EDT
Completed Not applicable Interventional Accepts healthy volunteers

Dermal Cryotherapy in Patients Undergoing Abdominoplasty

NCT02763306
View directory record Official record
Conditions
Skin Laxity
Interventions
Dermal Cooling System
Device
Lead sponsor
R2 Dermatology
Industry
Eligibility
18 Years and older
Enrollment
28 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2015 – 2017
U.S. locations
2
States / cities
Folsom, California • Fremont, California
Source: ClinicalTrials.gov public record
Updated Mar 29, 2021 · Synced Oct 25, 2026, 4:46 PM EDT
Completed Not applicable Interventional Accepts healthy volunteers

Use of Real-Time Functional Magnetic Resonance Imaging Neurofeedback to Improve Motor Function in Cerebellar Ataxia

NCT05436249
View directory record Official record
Conditions
Healthy
Interventions
Neurofeedback treatment, At-home therapy
Device · Behavioral
Lead sponsor
Johns Hopkins University
Other
Eligibility
18 Years to 100 Years
Enrollment
12 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2022 – 2024
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Apr 5, 2024 · Synced Oct 25, 2026, 4:46 PM EDT
Recruiting Phase 2 Interventional

18FluoroLDOPA PET Imaging for the Detection and Localization of Focal Congenital Hyperinsulinism

NCT04205604
View directory record Official record
Conditions
Congenital Hyperinsulinism
Interventions
18F-Fluoro Dopa Imaging
Drug
Lead sponsor
Miguel Pampaloni
Other
Eligibility
0 Days to 18 Years
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2026
U.S. locations
1
States / cities
San Francisco, California
Source: ClinicalTrials.gov public record
Updated May 1, 2026 · Synced Oct 25, 2026, 4:46 PM EDT
Terminated Phase 2 Interventional Results available

Combination Therapy Compared With Single-Drug Therapy in Patients With Cardiac Diseases

NCT00115349
View directory record Official record
Conditions
Cardiovascular Diseases, Heart Diseases, Beta-Thalassemia
Interventions
Deferoxamine, Deferiprone (L1)
Drug
Lead sponsor
Carelon Research
Other
Eligibility
18 Years to 100 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2009
U.S. locations
6
States / cities
Los Angeles, California • Oakland, California • Chicago, Illinois + 3 more
Source: ClinicalTrials.gov public record
Updated Mar 1, 2018 · Synced Oct 25, 2026, 4:46 PM EDT
Terminated Phase 3 Interventional Results available

Opioid Reduction Initiative During Outpatient Pediatric Urologic Procedures Using Exparel

NCT04826484
View directory record Official record
Conditions
Urologic Diseases, Hypospadias, Undescended Testes, Chordee, Hydrocele, Orchiectomy
Interventions
Exparel 133 miligrams per 10 milliliter injection, Bupivacaine Hydrochloride
Drug
Lead sponsor
Johns Hopkins University
Other
Eligibility
6 Years and older
Enrollment
104 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2023
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Dec 26, 2023 · Synced Oct 25, 2026, 4:46 PM EDT
Completed Phase 1 Interventional

Safety and Tolerability Study of rAvPAL-PEG to Treat Phenylketonuria

NCT00634660
View directory record Official record
Conditions
Phenylketonuria
Interventions
rAvPAL-PEG
Drug
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
16 Years to 50 Years
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2009
U.S. locations
8
States / cities
Chicago, Illinois • Minneapolis, Minnesota • St Louis, Missouri + 5 more
Source: ClinicalTrials.gov public record
Updated Feb 1, 2017 · Synced Oct 25, 2026, 4:46 PM EDT
Active, not recruiting No phase listed Observational

Monitoring Response to Orkambi in Cystic Fibrosis Lung Disease by Inhaled Xenon MRI

NCT02848560
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
Hyperpolarized Xenon
Drug
Lead sponsor
Children's Hospital Medical Center, Cincinnati
Other
Eligibility
6 Years to 12 Years
Enrollment
38 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2025
U.S. locations
1
States / cities
Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Nov 21, 2024 · Synced Oct 25, 2026, 4:46 PM EDT
Completed Phase 3 Interventional Results available

Study of PlasmaCap IG in Adults and Children With PIDD

NCT03238079
View directory record Official record
Conditions
Primary Immune Deficiency Diseases (PIDD)
Interventions
10% IGIV
Biological
Lead sponsor
Evolve Biologics
Industry
Eligibility
2 Years to 70 Years
Enrollment
63 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2020
U.S. locations
11
States / cities
Los Angeles, California • Centennial, Colorado • Tampa, Florida + 7 more
Source: ClinicalTrials.gov public record
Updated Jun 30, 2026 · Synced Oct 25, 2026, 4:46 PM EDT
Not listed No phase listed Observational

Studying Tissue and Blood Samples From Patients With Acute Myeloid Leukemia

NCT00900224
View directory record Official record
Conditions
Leukemia
Interventions
DNA analysis, DNA methylation analysis, gene expression analysis, mutation analysis, polymerase chain reaction, reverse transcriptase-polymerase chain reaction, high performance liquid chromatography, laboratory biomarker analysis
Genetic · Other
Lead sponsor
Alliance for Clinical Trials in Oncology
Other
Eligibility
Not listed
Enrollment
529 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2008
U.S. locations
77
States / cities
Mountain View, California • Lewes, Delaware • Newark, Delaware + 55 more
Source: ClinicalTrials.gov public record
Updated Aug 31, 2023 · Synced Oct 25, 2026, 4:46 PM EDT