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ClinicalTrials.gov public records Last synced Oct 20, 2026, 12:23 AM EDT

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Showing 1129–1152 of 10,537 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Congenital Diseases Clear all
Recruiting Phase 1 Interventional Accepts healthy volunteers

Treating Primary Sclerosing Cholangitis and Biliary Atresia With Vancomycin

NCT02137668
View directory record Official record
Conditions
Primary Sclerosing Cholangitis, Biliary Atresia
Interventions
Oral Vancomycin
Drug
Lead sponsor
Sacramento Pediatric Gastroenterology
Other
Eligibility
2 Weeks to 40 Years
Enrollment
200 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2010 – 2028
U.S. locations
1
States / cities
Sacramento, California
Source: ClinicalTrials.gov public record
Updated Nov 15, 2016 · Synced Oct 20, 2026, 12:23 AM EDT
Completed Not applicable Interventional Accepts healthy volunteers

SofWave Treatment to Lift Lax Tissue in the Submental and Neck Zones and to Lift the Eyebrow

NCT04969380
View directory record Official record
Conditions
Skin Laxity
Interventions
Sofwave
Device
Lead sponsor
Sofwave Medical LTD
Industry
Eligibility
35 Years to 80 Years
Enrollment
20 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2020 – 2023
U.S. locations
1
States / cities
Irvine, California
Source: ClinicalTrials.gov public record
Updated Nov 18, 2023 · Synced Oct 20, 2026, 12:23 AM EDT
Recruiting No phase listed Observational

Genotype and Phenotype Correlation in Hereditary Thrombotic Thrombocytopenic Purpura (Upshaw-Schulman Syndrome)

NCT01257269
View directory record Official record
Conditions
Thrombotic Thrombocytopenic Purpura, Congenital Thrombotic Thrombocytopenic Purpura, Familial Thrombotic Thrombocytopenic Purpura, Thrombotic Thrombocytopenic Purpura, Congenital, Upshaw-Schulman Syndrome
Interventions
Observation
Other
Lead sponsor
Insel Gruppe AG, University Hospital Bern
Other
Eligibility
Not listed
Enrollment
450 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2030
U.S. locations
1
States / cities
Oklahoma City, Oklahoma
Source: ClinicalTrials.gov public record
Updated Oct 11, 2023 · Synced Oct 20, 2026, 12:23 AM EDT
Terminated Phase 1 Interventional

IGF-1 Therapy in Patients With Cystic Fibrosis

NCT00566241
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
recombinant human IGF-1, Placebo
Drug
Lead sponsor
Stony Brook University
Other
Eligibility
18 Years and older
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2012
U.S. locations
1
States / cities
Stony Brook, New York
Source: ClinicalTrials.gov public record
Updated Jan 28, 2013 · Synced Oct 20, 2026, 12:23 AM EDT
Completed Not applicable Interventional Results available

Corticosteroid Therapy in Neonates Undergoing Cardiopulmonary Bypass

NCT01579513
View directory record Official record
Conditions
Congenital Heart Disease, Disorder of Fetus or Newborn
Interventions
Methylprednisolone, Placebo
Drug
Lead sponsor
Medical University of South Carolina
Other
Eligibility
Up to 30 Days
Enrollment
190 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2018
U.S. locations
2
States / cities
Atlanta, Georgia • Charleston, South Carolina
Source: ClinicalTrials.gov public record
Updated Dec 9, 2019 · Synced Oct 20, 2026, 12:23 AM EDT
Recruiting Not applicable Interventional Accepts healthy volunteers

Maternal Oxygen Supplementation for Intrauterine Resuscitation

NCT05681624
View directory record Official record
Conditions
Fetal Distress, Fetal Hypoxia, Labor and Delivery Complication
Interventions
Maternal oxygen supplementation, Room air
Other
Lead sponsor
Washington University School of Medicine
Other
Eligibility
Female only
Enrollment
2,252 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2023 – 2028
U.S. locations
1
States / cities
St Louis, Missouri
Source: ClinicalTrials.gov public record
Updated Aug 10, 2026 · Synced Oct 20, 2026, 12:23 AM EDT
Completed Phase 2Phase 3 Interventional

A Study of the Safety and Efficacy of rhGAA in Patients With Infantile-onset Pompe Disease

NCT00059280
View directory record Official record
Conditions
Glycogen Storage Disease Type II
Interventions
Myozyme
Biological
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
Up to 26 Weeks
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003 – 2005
U.S. locations
4
States / cities
Gainesville, Florida • Durham, North Carolina • Cincinnati, Ohio + 1 more
Source: ClinicalTrials.gov public record
Updated Feb 5, 2014 · Synced Oct 20, 2026, 12:23 AM EDT
Completed No phase listed Observational

A Study to Assess Variation in Potential Biomarkers in Friedreich Ataxia

NCT04255680
View directory record Official record
Conditions
Friedreich Ataxia
Interventions
Buccal Swabs and Blood Draws
Diagnostic Test
Lead sponsor
Larimar Therapeutics, Inc.
Industry
Eligibility
12 Years to 65 Years
Enrollment
20 participants
Timeline
2020
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Aug 18, 2020 · Synced Oct 20, 2026, 12:23 AM EDT
Active, not recruiting Phase 1Phase 2 Interventional

BEACON: A Study Evaluating the Safety and Efficacy of BEAM-101 in Patients With Severe Sickle Cell Disease

NCT05456880
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
BEAM-101
Biological
Lead sponsor
Beam Therapeutics Inc.
Industry
Eligibility
12 Years to 35 Years
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2028
U.S. locations
19
States / cities
Birmingham, Alabama • Phoenix, Arizona • Jacksonville, Florida + 14 more
Source: ClinicalTrials.gov public record
Updated Dec 16, 2025 · Synced Oct 20, 2026, 12:23 AM EDT
Completed Phase 4 Interventional Results available

Comparing Acute Pain Management Protocols for Patients With Sickle Cell Disease

NCT02222246
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Hydromorphone (Standardized, weight-based dosing), Morphine Sulfate (Standardized, weight-based dosing), Hydromorphone (Patient Specific dosing), Morphine Sulfate (Patient Specific dosing)
Drug
Lead sponsor
Duke University
Other
Eligibility
21 Years and older
Enrollment
106 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2016
U.S. locations
2
States / cities
New York, New York • Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Aug 4, 2017 · Synced Oct 20, 2026, 12:23 AM EDT
Withdrawn Phase 1 Interventional

Aberrations in Carnitine Homeostasis in Congenital Heart Disease With Increased Pulmonary Blood Flow

NCT01825369
View directory record Official record
Conditions
Heart Septal Defects, Ventricular, Atrioventricular Septal Defect
Interventions
IV L-carnitine
Drug
Lead sponsor
University of California, San Francisco
Other
Eligibility
2 Months to 12 Months
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2020
U.S. locations
1
States / cities
San Francisco, California
Source: ClinicalTrials.gov public record
Updated May 6, 2020 · Synced Oct 20, 2026, 12:23 AM EDT
Completed Not applicable Interventional Accepts healthy volunteers

Does Batten Grafting Improve Nasal Outcomes in Septoplasty and Turbinate Reduction?

NCT05287841
View directory record Official record
Conditions
Nasal Obstruction, Septal Defect, Allergic Rhinitis, Nasal Polyps, Nasal Valve Collapse
Interventions
Batten batten graft, Septoplasty, Inferior Turbinate Reduction
Procedure
Lead sponsor
Northwell Health
Other
Eligibility
18 Years and older
Enrollment
13 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2021 – 2026
U.S. locations
3
States / cities
Poplar Bluff, Missouri • New York, New York • Staten Island, New York
Source: ClinicalTrials.gov public record
Updated Oct 13, 2026 · Synced Oct 20, 2026, 12:23 AM EDT
Completed Phase 2 Interventional

A Study of ARRY-371797 in Patients With LMNA-Related Dilated Cardiomyopathy

NCT02057341
View directory record Official record
Conditions
LMNA-Related Dilated Cardiomyopathy
Interventions
ARRY-371797, p38 inhibitor; oral
Drug
Lead sponsor
Pfizer
Industry
Eligibility
18 Years and older
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2016
U.S. locations
6
States / cities
Stanford, California • Aurora, Colorado • Baltimore, Maryland + 3 more
Source: ClinicalTrials.gov public record
Updated Oct 14, 2020 · Synced Oct 20, 2026, 12:23 AM EDT
Completed Phase 3 Interventional Results available

Sickle Cell Disease Treatment With Arginine Therapy (STArT) Trial

NCT04839354
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
L-Arginine Hydrochloride, Normal saline
Drug · Other
Lead sponsor
Claudia R. Morris
Other
Eligibility
3 Years to 21 Years
Enrollment
271 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2024
U.S. locations
10
States / cities
Los Angeles, California • San Francisco, California • Washington D.C., District of Columbia + 6 more
Source: ClinicalTrials.gov public record
Updated Jun 23, 2026 · Synced Oct 20, 2026, 12:23 AM EDT
Withdrawn Phase 2 Interventional

Abciximab (ReoPro) as a Therapeutic Intervention for Sickle Cell Vaso-Occlusive Pain Crisis

NCT01932554
View directory record Official record
Conditions
Sickle Cell Disease, Hb-SS Disease With Vasoocclusive Pain, Hemoglobin SS Disease With Vasoocclusive Crisis, Other Sickle Cell Disease With Vaso-Occlusive Pain, Hemoglobin SS Disease With Crisis
Interventions
Abciximab, Placebo, Intravenous hydration, Ibuprofen, Parenteral narcotic, Incentive spirometry, Supplemental oxygen
Drug · Other
Lead sponsor
St. Louis University
Other
Eligibility
5 Years to 25 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2015
U.S. locations
1
States / cities
St Louis, Missouri
Source: ClinicalTrials.gov public record
Updated Mar 26, 2015 · Synced Oct 20, 2026, 12:23 AM EDT
Completed Phase 3 Interventional

Higher-Dose Ezetimibe to Treat Homozygous Sitosterolemia

NCT00099996
View directory record Official record
Conditions
Heart Diseases, Metabolism, Inborn Errors
Interventions
SCH-58235, Ezetimibe
Drug
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
Not listed
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2005
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 4, 2008 · Synced Oct 20, 2026, 12:23 AM EDT
Terminated Phase 2 Interventional

Cilengitide in Treating Patients With Acute Myeloid Leukemia

NCT00089388
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 Without Maturation (M1), Adult Acute Myeloid Leukemia in Remission, Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities, Adult Acute Myeloid Leukemia With t(15;17)(q22;q12), Adult Erythroleukemia (M6a), Adult Pure Erythroid Leukemia (M6b)
Interventions
cilengitide
Drug
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
50 Years and older
Enrollment
70 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 24, 2013 · Synced Oct 20, 2026, 12:23 AM EDT
Completed Not applicable Interventional Results available

Hemangioma Associated With High Rates of Morbidity

NCT00394888
View directory record Official record
Conditions
Hemangioma
Interventions
MRI, MRI of head and neck, Dermatological Examination, Cardiac examination, Abdominal ultrasound
Device · Other
Lead sponsor
Medical College of Wisconsin
Other
Eligibility
Up to 18 Years
Enrollment
433 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2010
U.S. locations
1
States / cities
Milwaukee, Wisconsin
Source: ClinicalTrials.gov public record
Updated Sep 20, 2013 · Synced Oct 20, 2026, 12:23 AM EDT
Completed Early Phase 1 Interventional

Baclofen Treatment of Ataxia Telangiectasia

NCT00640003
View directory record Official record
Conditions
Ataxia Telangiectasia
Interventions
Baclofen, Placebo
Drug
Lead sponsor
Johns Hopkins University
Other
Eligibility
12 Years and older
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2011
U.S. locations
2
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Dec 20, 2017 · Synced Oct 20, 2026, 12:23 AM EDT
Recruiting Phase 3 Interventional

A Study to Investigate Efficacy and Safety of KP-001 Compared With Placebo in Patients Aged ≥2 Years With Common VM, Common LM, or KTS/CLOVES Syndrome

NCT07285005
View directory record Official record
Conditions
Venous Malformations, Lymphatic Malformations, Klippel-Trenaunay Syndrome, CLOVES Syndrome
Interventions
KP-001, Placebo
Drug
Lead sponsor
Kaken Pharmaceutical
Industry
Eligibility
2 Years and older
Enrollment
150 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2028
U.S. locations
1
States / cities
Charleston, South Carolina
Source: ClinicalTrials.gov public record
Updated Sep 21, 2026 · Synced Oct 20, 2026, 12:23 AM EDT
Completed Phase 2 Interventional Results available

Clofarabine, Cytarabine, and Filgrastim in Treating Patients With Newly Diagnosed Acute Myeloid Leukemia, Advanced Myelodysplastic Syndrome, and/or Advanced Myeloproliferative Neoplasm

NCT01101880
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), Chronic Myelomonocytic Leukemia, de Novo Myelodysplastic Syndromes, Refractory Anemia With Excess Blasts, Untreated Adult Acute Myeloid Leukemia, Myeloproliferative Neoplasm With 10% Blasts or Higher
Interventions
filgrastim, clofarabine, cytarabine
Biological · Drug
Lead sponsor
University of Washington
Other
Eligibility
18 Years to 64 Years
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2017
U.S. locations
3
States / cities
Duarte, California • Stanford, California • Seattle, Washington
Source: ClinicalTrials.gov public record
Updated Oct 19, 2017 · Synced Oct 20, 2026, 12:23 AM EDT
Terminated Phase 2 Interventional

An Open Label Extension Study of STX209 in Subjects With Fragile X Syndrome

NCT01013480
View directory record Official record
Conditions
Fragile X Syndrome
Interventions
Arbaclofen
Drug
Lead sponsor
Seaside Therapeutics, Inc.
Industry
Eligibility
6 Years to 40 Years
Enrollment
45 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2012
U.S. locations
9
States / cities
Los Angeles, California • Sacramento, California • Chicago, Illinois + 6 more
Source: ClinicalTrials.gov public record
Updated Dec 20, 2012 · Synced Oct 20, 2026, 12:23 AM EDT
Terminated Not applicable Interventional

Storybook Reading in Individuals With Down Syndrome

NCT03764761
View directory record Official record
Conditions
Down Syndrome, Augmentative and Alternative Communication
Interventions
AAC Technology - Standard of Care, AAC Technology - non-optimal integrated arrangement
Device
Lead sponsor
Penn State University
Other
Eligibility
7 Years to 35 Years
Enrollment
14 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2021
U.S. locations
1
States / cities
University Park, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Apr 29, 2022 · Synced Oct 20, 2026, 12:23 AM EDT
Completed Not applicable Interventional Results available

Partially Matched Stem Cell Transplantation for Patients With Refractory Severe Aplastic Anemia or Refractory Cytopenias

NCT00244010
View directory record Official record
Conditions
Anemia, Aplastic, Amegakaryocytic Thrombocytopenia, Diamond-Blackfan Anemia, Kostmann Syndrome
Interventions
Allogeneic stem cell transplant
Device
Lead sponsor
St. Jude Children's Research Hospital
Other
Eligibility
Up to 21 Years
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2009
U.S. locations
1
States / cities
Memphis, Tennessee
Source: ClinicalTrials.gov public record
Updated May 30, 2017 · Synced Oct 20, 2026, 12:23 AM EDT