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ClinicalTrials.gov public records Last synced Oct 3, 2026, 10:58 AM EDT

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Showing 25–48 of 10,517 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Congenital Disease Clear all
Available No phase listed Expanded access

Post-trial Access Program of Idursulfase-IT Along With Elaprase in Children With Hunter Syndrome

NCT05795361
View directory record Official record
Conditions
Hunter Syndrome
Interventions
Idursulfase-IT
Drug
Lead sponsor
Takeda
Industry
Eligibility
Male only
Healthy volunteers
Healthy volunteers not accepted
U.S. locations
17
States / cities
Phoenix, Arizona • Oakland, California • Wilmington, Delaware + 14 more
Source: ClinicalTrials.gov public record
Updated Apr 13, 2026 · Synced Oct 3, 2026, 10:58 AM EDT
Completed No phase listed Observational

Prospective Study of 2 mm Margins for the Biopsy of Dysplastic Nevi

NCT03094273
View directory record Official record
Conditions
Dysplastic Nevi
Interventions
Saucerization biopsy
Procedure
Lead sponsor
NYU Langone Health
Other
Eligibility
18 Years and older
Enrollment
138 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2016
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated May 8, 2019 · Synced Oct 3, 2026, 10:58 AM EDT
Completed Phase 1 Interventional

Vorinostat, Cytarabine, and Etoposide in Treating Patients With Relapsed and/or Refractory Acute Leukemia or Myelodysplastic Syndromes or Myeloproliferative Disorders

NCT00357305
View directory record Official record
Conditions
Accelerated Phase Chronic Myelogenous Leukemia, 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(15;17)(q22;q12), 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 Acute Promyelocytic Leukemia (M3), Adult Erythroleukemia (M6a), Adult Pure Erythroid Leukemia (M6b), Atypical Chronic Myeloid Leukemia, BCR-ABL1 Negative, Blastic Phase Chronic Myelogenous Leukemia, Chronic Eosinophilic Leukemia, Chronic Myelomonocytic Leukemia, Chronic Neutrophilic Leukemia, de Novo Myelodysplastic Syndromes, Essential Thrombocythemia, Myelodysplastic/Myeloproliferative Neoplasm, Unclassifiable, Polycythemia Vera, Previously Treated Myelodysplastic Syndromes, Primary Myelofibrosis, Recurrent Adult Acute Lymphoblastic Leukemia, Recurrent Adult Acute Myeloid Leukemia, Relapsing Chronic Myelogenous Leukemia, Secondary Acute Myeloid Leukemia, Secondary Myelodysplastic Syndromes
Interventions
vorinostat, cytarabine, etoposide, pharmacological study, laboratory biomarker analysis
Drug · Other
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
18 Years and older
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2006
U.S. locations
2
States / cities
Baltimore, Maryland • Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated May 3, 2013 · Synced Oct 3, 2026, 10:58 AM EDT
Completed No phase listed Observational

Suggamadex and Fast-track Cardiac Anesthesia in a Developing Country

NCT02704351
View directory record Official record
Conditions
Suggamadex, Surgery for Congenital Heart Disease
Interventions
Surgery
Procedure
Lead sponsor
Joseph D. Tobias
Other
Eligibility
0 Years to 18 Years
Enrollment
14 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated May 6, 2023 · Synced Oct 3, 2026, 10:58 AM EDT
Not listed Phase 1 Interventional

Dilated cardiomYopathy iNtervention With Allogeneic MyocardIally-regenerative Cells (DYNAMIC)

NCT02293603
View directory record Official record
Conditions
Dilated Cardiomyopathy (DCM), Ischemic Cardiomyopathy, Nonischemic Cardiomyopathy, Heart Failure
Interventions
Allogeneic Cardiosphere-Derived Cells (CDCs)
Biological
Lead sponsor
Capricor Inc.
Industry
Eligibility
18 Years and older
Enrollment
42 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2020
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Jun 21, 2016 · Synced Oct 3, 2026, 10:58 AM EDT
Recruiting Not applicable Interventional Accepts healthy volunteers

Comparison of a Demand Oxygen Delivery

NCT06609694
View directory record Official record
Conditions
Obstructive Apnea
Interventions
Portable oxygen concentrator Inogen G5 model
Device
Lead sponsor
Children's Hospital Medical Center, Cincinnati
Other
Eligibility
5 Years to 17 Years
Enrollment
10 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2023 – 2028
U.S. locations
1
States / cities
Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Jan 7, 2026 · Synced Oct 3, 2026, 10:58 AM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

Effect of Venglustat in Patients With Renal Impairment

NCT03687554
View directory record Official record
Conditions
Healthy Volunteers, Polycystic Kidney, Autosomal Dominant
Interventions
Venglustat GZ/SAR402671
Drug
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
18 Years to 79 Years
Enrollment
24 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2018 – 2019
U.S. locations
1
States / cities
Miami, Florida
Source: ClinicalTrials.gov public record
Updated Apr 25, 2022 · Synced Oct 3, 2026, 10:58 AM EDT
Not listed Phase 2 Interventional

Phase II Study of Cholesterol- and Cholestanol-Free Diet, Lovastatin, and Chenodeoxycholic Acid for Cerebrotendinous Xanthomatosis

NCT00004346
View directory record Official record
Conditions
Cerebrotendinous Xanthomatosis
Interventions
chenodeoxycholic acid, lovastatin
Drug
Lead sponsor
National Center for Research Resources (NCRR)
NIH
Eligibility
0 Years and older
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1996
U.S. locations
1
States / cities
Portland, Oregon
Source: ClinicalTrials.gov public record
Updated Jun 24, 2005 · Synced Oct 3, 2026, 10:58 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

PIONEER: Intraoperative and Perioperative OCT Study

NCT02423161
View directory record Official record
Conditions
Retinal Detachment, Epiretinal Membrane, Macular Hole, Fuchs Endothelial Dystrophy
Interventions
Not listed
Lead sponsor
The Cleveland Clinic
Other
Eligibility
18 Years and older
Enrollment
750 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2011 – 2017
U.S. locations
1
States / cities
Cleveland, Ohio
Source: ClinicalTrials.gov public record
Updated Aug 20, 2024 · Synced Oct 3, 2026, 10:58 AM EDT
Recruiting Not applicable Interventional

A Feasibility Study to Evaluate Safety and Probable Benefit of the Eclipse XL1 System for Distraction Enterogenesis in Adult and Pediatric Patients With Short Bowel Syndrome

NCT05535361
View directory record Official record
Conditions
Short Bowel Syndrome
Interventions
Distraction Enterogenesis in Adult Patients with Short Bowel Syndrome
Device
Lead sponsor
Eclipse Regenesis, Inc.
Other
Eligibility
3 Months to 65 Years
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2028
U.S. locations
5
States / cities
Palo Alto, California • San Francisco, California • Washington D.C., District of Columbia + 2 more
Source: ClinicalTrials.gov public record
Updated Aug 12, 2026 · Synced Oct 3, 2026, 10:58 AM EDT
Recruiting No phase listed Observational

Molecular and Genetic Studies of Congenital Myopathies

NCT00272883
View directory record Official record
Conditions
Central Core Disease, Centronuclear Myopathy, Congenital Fiber Type Disproportion, Multiminicore Disease, Myotubular Myopathy, Nemaline Myopathy, Rigid Spine Muscular Dystrophy, Undefined Congenital Myopathy
Interventions
Not listed
Lead sponsor
Boston Children's Hospital
Other
Eligibility
Not listed
Enrollment
4,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003 – 2050
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Mar 25, 2026 · Synced Oct 3, 2026, 10:58 AM EDT
Completed Phase 1 Interventional Results available

Dose-Titration and Open-label Extension Study of SRP-4045 in Advanced Stage Duchenne Muscular Dystrophy (DMD) Patients

NCT02530905
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
SRP-4045, Placebo
Drug
Lead sponsor
Sarepta Therapeutics, Inc.
Industry
Eligibility
7 Years to 21 Years · Male only
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2018
U.S. locations
3
States / cities
Los Angeles, California • Chicago, Illinois • Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated May 17, 2021 · Synced Oct 3, 2026, 10:58 AM EDT
Completed Not applicable Interventional Accepts healthy volunteers

Bisphenol A and Muscle Insulin Sensitivity

NCT03771066
View directory record Official record
Conditions
Insulin Sensitivity, Glucose Metabolism Disorders, Microtia
Interventions
bisphenol A, Placebo
Behavioral
Lead sponsor
California Polytechnic State University-San Luis Obispo
Other
Eligibility
18 Years to 50 Years
Enrollment
40 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2019 – 2023
U.S. locations
1
States / cities
San Luis Obispo, California
Source: ClinicalTrials.gov public record
Updated Jul 8, 2024 · Synced Oct 3, 2026, 10:58 AM EDT
Completed Phase 2 Interventional

Use of Ketamine Prior to Cardiopulmonary Bypass in Children

NCT00556361
View directory record Official record
Conditions
Ventricular Septal Defect
Interventions
saline, ketamine
Drug
Lead sponsor
Arkansas Children's Hospital Research Institute
Other
Eligibility
Up to 1 Year
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2007
U.S. locations
1
States / cities
Little Rock, Arkansas
Source: ClinicalTrials.gov public record
Updated Nov 12, 2007 · Synced Oct 3, 2026, 10:58 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Pseudoxanthoma Elasticum (PXE) Natural History Biomarkers in PXE Individuals and Their Biological Non-PXE Siblings

NCT05025722
View directory record Official record
Conditions
Pseudoxanthoma Elasticum
Interventions
Non Interventional
Other
Lead sponsor
Daiichi Sankyo
Industry
Eligibility
18 Years to 75 Years
Enrollment
48 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2021 – 2022
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Sep 6, 2022 · Synced Oct 3, 2026, 10:58 AM EDT
Completed No phase listed Observational

Study of the Metabolism of Pyruvate and Related Problems in Patients With Lactic Acidemia

NCT00004353
View directory record Official record
Conditions
Mitochondrial Myopathy, MELAS Syndrome, Lactic Acidosis
Interventions
Not listed
Lead sponsor
National Center for Research Resources (NCRR)
NIH
Eligibility
Not listed
Enrollment
35 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1978
U.S. locations
1
States / cities
San Diego, California
Source: ClinicalTrials.gov public record
Updated Jul 10, 2008 · Synced Oct 3, 2026, 10:58 AM EDT
Completed No phase listed Observational

Informed Consent for Whole Genome Sequencing: Ideals and Norms Referenced by Early Participants

NCT01369953
View directory record Official record
Conditions
Coronary Artery Disease, Proteus Syndrome, Coffin - Sins Syndrome, Familial Isolated Hyperparathyroidism, Dubouitz Syndrome
Interventions
Not listed
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
18 Years to 65 Years
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2014
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 26, 2018 · Synced Oct 3, 2026, 10:58 AM EDT
Active, not recruiting Phase 2 Interventional

Total Marrow and Lymphoid Irradiation and Chemotherapy Before DSCT in Treating Patients With High-Risk ALL or AML

NCT02094794
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), Recurrent Adult Acute Lymphoblastic Leukemia, Recurrent Adult Acute Myeloid Leukemia, Recurrent Childhood Acute Lymphoblastic Leukemia, Recurrent Childhood Acute Myeloid Leukemia
Interventions
etoposide, cyclophosphamide, total marrow irradiation, allogeneic hematopoietic stem cell transplantation, allogeneic bone marrow transplantation
Drug · Radiation · Procedure
Lead sponsor
City of Hope Medical Center
Other
Eligibility
16 Years to 60 Years
Enrollment
108 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2027
U.S. locations
1
States / cities
Duarte, California
Source: ClinicalTrials.gov public record
Updated Jul 1, 2026 · Synced Oct 3, 2026, 10:58 AM EDT
Completed Phase 3 Interventional Results available

Safety and Efficacy Study of Several Replagal Dosing Regimens on Cardiac Function in Adults With Fabry Disease

NCT00864851
View directory record Official record
Conditions
Fabry Disease
Interventions
Replagal
Biological
Lead sponsor
Shire
Industry
Eligibility
18 Years and older
Enrollment
44 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2012
U.S. locations
4
States / cities
Tucson, Arizona • Iowa City, Iowa • New York, New York + 1 more
Source: ClinicalTrials.gov public record
Updated Jun 9, 2021 · Synced Oct 3, 2026, 10:58 AM EDT
Completed Phase 1Phase 2 Interventional Results available

Chemotherapy and Monoclonal Antibody Therapy in Treating Patients With Advanced Myeloid Cancer

NCT00014495
View directory record Official record
Conditions
Leukemia, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Neoplasms
Interventions
filgrastim, cytarabine, bismuth Bi213 monoclonal antibody M195
Biological · Drug · Radiation
Lead sponsor
Memorial Sloan Kettering Cancer Center
Other
Eligibility
Up to 120 Years
Enrollment
32 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2000 – 2009
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jan 22, 2016 · Synced Oct 3, 2026, 10:58 AM EDT
Completed No phase listed Observational

Arthroereisis Versus Lateral Column Lengthening in the Treatment of Planovalgus Feet

NCT02055495
View directory record Official record
Conditions
Flatfeet
Interventions
Not listed
Lead sponsor
University of Utah
Other
Eligibility
7 Years to 17 Years
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2014
U.S. locations
1
States / cities
Salt Lake City, Utah
Source: ClinicalTrials.gov public record
Updated Aug 8, 2014 · Synced Oct 3, 2026, 10:58 AM EDT
Recruiting Phase 2 Interventional

Study to Evaluate Soquelitinib in Participants With Moderate to Severe AD

NCT07441395
View directory record Official record
Conditions
Atopic Dermatitis, Atopic Dermatitis Eczema, Eczema, Eczema, Atopic
Interventions
Soquelitinib, Placebo
Drug
Lead sponsor
Corvus Pharmaceuticals, Inc.
Industry
Eligibility
18 Years and older
Enrollment
200 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2027
U.S. locations
1
States / cities
South San Francisco, California
Source: ClinicalTrials.gov public record
Updated Mar 5, 2026 · Synced Oct 3, 2026, 10:58 AM EDT
Completed No phase listed Observational Results available

STeP IT UP CF: STimulating ImProved Health And Well-being In CysTic Fibrosis

NCT04018495
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
Not listed
Lead sponsor
Medical College of Wisconsin
Other
Eligibility
18 Years to 100 Years
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2022
U.S. locations
1
States / cities
Milwaukee, Wisconsin
Source: ClinicalTrials.gov public record
Updated Feb 3, 2025 · Synced Oct 3, 2026, 10:58 AM EDT
Terminated Phase 1Phase 2 Interventional Results available

Busulfan, Etoposide, and Intensity-Modulated Radiation Therapy Followed By Donor Stem Cell Transplant in Treating Patients With Advanced Myeloid Cancer

NCT00540995
View directory record Official record
Conditions
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), Blastic Phase Chronic Myelogenous Leukemia, Childhood Acute Myeloid Leukemia in Remission, Childhood Chronic Myelogenous Leukemia, Childhood Myelodysplastic Syndromes, Previously Treated Myelodysplastic Syndromes, Recurrent Adult Acute Myeloid Leukemia, Recurrent Childhood Acute Myeloid Leukemia, Refractory Anemia With Excess Blasts
Interventions
busulfan, etoposide, intensity-modulated radiation therapy, allogeneic hematopoietic stem cell transplantation, allogeneic bone marrow transplantation, peripheral blood stem cell transplantation, tomotherapy
Drug · Radiation · Procedure
Lead sponsor
City of Hope Medical Center
Other
Eligibility
6 Years to 55 Years
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2011
U.S. locations
1
States / cities
Duarte, California
Source: ClinicalTrials.gov public record
Updated Nov 30, 2023 · Synced Oct 3, 2026, 10:58 AM EDT