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ClinicalTrials.gov public records Last synced Oct 10, 2026, 9:01 AM EDT

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Showing 97–120 of 10,525 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Congenital Diseases Clear all
Recruiting Not applicable Interventional

Encore PFO Closure Device - The PerFOrm Trial

NCT05537753
View directory record Official record
Conditions
Patent Foramen Ovale, Cryptogenic Stroke
Interventions
Encore PFO closure device, FDA-approved PFO closure device
Device
Lead sponsor
Encore Medical Inc.
Industry
Eligibility
18 Years to 60 Years
Enrollment
500 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2030
U.S. locations
8
States / cities
Little Rock, Arkansas • La Jolla, California • Tampa, Florida + 5 more
Source: ClinicalTrials.gov public record
Updated Sep 19, 2024 · Synced Oct 10, 2026, 9:01 AM EDT
Completed Phase 3 Interventional

A Clinical Study of Intravenous Immunoglobulin

NCT00468273
View directory record Official record
Conditions
Immunologic Deficiency Syndromes
Interventions
Immune Globulin Intravenous (Human) Omr-IgG-am IGIV
Drug
Lead sponsor
FFF Enterprises
Industry
Eligibility
3 Years to 75 Years
Enrollment
57 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2009
U.S. locations
8
States / cities
Los Angeles, California • Centennial, Colorado • North Palm Beach, Florida + 5 more
Source: ClinicalTrials.gov public record
Updated Aug 11, 2014 · Synced Oct 10, 2026, 9:01 AM EDT
Terminated Phase 1 Interventional

Yttrium Y 90 Anti-CD45 Monoclonal Antibody AHN-12 in Treating Patients With Advanced Leukemia

NCT00618696
View directory record Official record
Conditions
Leukemia, Myelodysplastic Syndromes
Interventions
anti-CD45 monoclonal antibody AHN-12, yttrium Y 90 anti-CD45 monoclonal antibody AHN-12
Biological · Radiation
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
18 Years and older
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2007
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Nov 29, 2017 · Synced Oct 10, 2026, 9:01 AM EDT
Completed Phase 2 Interventional Results available

Oral Pirfenidone for the Pulmonary Fibrosis of Hermansky-Pudlak Syndrome

NCT00001596
View directory record Official record
Conditions
Albinism, Inborn Errors of Metabolism, Oculocutaneous Albinism, Platelet Storage Pool Deficiency, Pulmonary Fibrosis
Interventions
Pirfenidone, Placebo
Drug
Lead sponsor
William Gahl, M.D.
NIH
Eligibility
18 Years and older
Enrollment
35 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2016
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 16, 2017 · Synced Oct 10, 2026, 9:01 AM EDT
Completed Phase 3 Interventional Results available

Efficacy and Safety of Extended Release and Immediate Release Febuxostat in Participants With Gout

NCT02139046
View directory record Official record
Conditions
Gout
Interventions
Febuxostat IR, Febuxostat XR, Febuxostat placebo, Colchicine, Naproxen, Lansoprazole
Drug
Lead sponsor
Takeda
Industry
Eligibility
18 Years and older
Enrollment
1,790 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2015
U.S. locations
192
States / cities
Binghamton, Alabama • Birmingham, Alabama • Huntsville, Alabama + 189 more
Source: ClinicalTrials.gov public record
Updated Nov 3, 2016 · Synced Oct 10, 2026, 9:01 AM EDT
Completed Phase 2 Interventional Results available

Long-Term Study of Nitisinone to Treat Alkaptonuria

NCT00107783
View directory record Official record
Conditions
Alkaptonuria
Interventions
Nitisinone (NTBC)
Drug
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
30 Years to 80 Years
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2009
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Aug 26, 2021 · Synced Oct 10, 2026, 9:01 AM EDT
Completed Phase 2 Interventional Results available

A Study to Evaluate the Safety and Efficacy of Crizanlizumab in Sickle Cell Disease Related Priapism

NCT03938454
View directory record Official record
Conditions
Priapism
Interventions
Crizanlizumab
Drug
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
12 Years to 100 Years · Male only
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2023
U.S. locations
13
States / cities
Birmingham, Alabama • Farmington, Connecticut • Washington D.C., District of Columbia + 10 more
Source: ClinicalTrials.gov public record
Updated Jan 13, 2026 · Synced Oct 10, 2026, 9:01 AM EDT
Completed No phase listed Observational

Kids Intestinal Dysfunction in Congenital Heart Disease: the Kind Heart Study

NCT02824705
View directory record Official record
Conditions
Congenital Disease
Interventions
Congenital Heart Disease Surgery
Procedure
Lead sponsor
University of Arizona
Other
Eligibility
3 Days to 2 Years
Enrollment
53 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2021
U.S. locations
1
States / cities
Tucson, Arizona
Source: ClinicalTrials.gov public record
Updated Sep 28, 2021 · Synced Oct 10, 2026, 9:01 AM EDT
Terminated Phase 2 Interventional Results available

TKI 258 in Von Hippel-Lindau Syndrome (VHL)

NCT01266070
View directory record Official record
Conditions
Von Hippel-Lindau Syndrome
Interventions
Dovitinib
Drug
Lead sponsor
M.D. Anderson Cancer Center
Other
Eligibility
18 Years and older
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2015
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Feb 13, 2017 · Synced Oct 10, 2026, 9:01 AM EDT
Not listed Phase 2Phase 3 Interventional

An Open Label Dose Escalation Study to Assess the Safety, Tolerability, and Pharmacologic Properties of High Dose Ambroxol Hydrochloride in Adult (≥ 18 Years of Age) Subjects With MPS III

NCT06614894
View directory record Official record
Conditions
Sanfilippo Syndrome, MPS3
Interventions
Ambroxol Hydrochloride 30 mg tablet - 9 mg/kg/day, Ambroxol Hydrochloride 30 mg tablet - 18 mg/kg/day, Ambroxol Hydrochloride 30 mg tablet - 27 mg/kg/day
Drug
Lead sponsor
Ozlem Goker-Alpan
Other
Eligibility
18 Years to 99 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
1
States / cities
Fairfax, Virginia
Source: ClinicalTrials.gov public record
Updated Aug 20, 2025 · Synced Oct 10, 2026, 9:01 AM EDT
Completed Phase 4 Interventional Results available

NAS Treatment - Opiate Versus Non-Opiate

NCT01734551
View directory record Official record
Conditions
Neonatal Abstinence Syndrome
Interventions
Morphine, Clonidine
Drug
Lead sponsor
Henrietta Bada
Other
Eligibility
1 Day to 7 Days
Enrollment
31 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2014
U.S. locations
1
States / cities
Lexington, Kentucky
Source: ClinicalTrials.gov public record
Updated Sep 12, 2017 · Synced Oct 10, 2026, 9:01 AM EDT
Completed Phase 1 Interventional

Montelukast in Very Low Birthweight Infants

NCT00492102
View directory record Official record
Conditions
Bronchopulmonary Dysplasia
Interventions
Montelukast
Drug
Lead sponsor
Children's Hospital Medical Center, Cincinnati
Other
Eligibility
7 Days and older
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2011
U.S. locations
1
States / cities
Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Aug 2, 2012 · Synced Oct 10, 2026, 9:01 AM EDT
Recruiting Not applicable Interventional

The Effects of a Low Glycemic Load Diet on Dysglycemia and Body Composition in Adults With Cystic Fibrosis-Related Diabetes

NCT05723445
View directory record Official record
Conditions
Cystic Fibrosis, Cystic Fibrosis-related Diabetes, Cystic Fibrosis With Intestinal Manifestations
Interventions
Low Glycemic Load Diet
Behavioral
Lead sponsor
Rhode Island Hospital
Other
Eligibility
18 Years to 70 Years
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2026
U.S. locations
2
States / cities
Boston, Massachusetts • Providence, Rhode Island
Source: ClinicalTrials.gov public record
Updated Aug 22, 2025 · Synced Oct 10, 2026, 9:01 AM EDT
Completed No phase listed Observational

Effects Rapid Atrial Pacing Has on the Electrocardiogram (ECG) in Patients With Dual Chamber Pacemakers

NCT00883610
View directory record Official record
Conditions
Prolonged QT Interval
Interventions
Not listed
Lead sponsor
Vanderbilt University
Other
Eligibility
21 Years and older
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2012
U.S. locations
1
States / cities
Nashville, Tennessee
Source: ClinicalTrials.gov public record
Updated Dec 11, 2014 · Synced Oct 10, 2026, 9:01 AM EDT
Completed Phase 2 Interventional Results available

Study of Ataluren in Participants With Nonsense Mutation Aniridia

NCT02647359
View directory record Official record
Conditions
Aniridia
Interventions
Ataluren, Placebo
Drug
Lead sponsor
PTC Therapeutics
Industry
Eligibility
2 Years and older
Enrollment
39 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2021
U.S. locations
2
States / cities
Portland, Oregon • Charlottesville, Virginia
Source: ClinicalTrials.gov public record
Updated May 27, 2022 · Synced Oct 10, 2026, 9:01 AM EDT
Completed Phase 3 Interventional Results available

An Open Label Study to Determine the Safety and Efficacy of Replacement Factor VIII Protein (Known as rFVIIIFc) in Previously Untreated Males With Severe Hemophilia A

NCT02234323
View directory record Official record
Conditions
Hemophilia A
Interventions
rFVIIIFc
Biological
Lead sponsor
Bioverativ, a Sanofi company
Industry
Eligibility
Up to 5 Years · Male only
Enrollment
108 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2019
U.S. locations
20
States / cities
Little Rock, Arkansas • Duarte, California • Sacramento, California + 16 more
Source: ClinicalTrials.gov public record
Updated Apr 11, 2022 · Synced Oct 10, 2026, 9:01 AM EDT
Active, not recruiting No phase listed Observational

A Trial That Evaluates Disease Characteristics in Hemophilia B Adult Male Participants Receiving Prophylaxis With Standard of Care Factor IX Protein (FIX) Replacement Therapy

NCT05568459
View directory record Official record
Conditions
Hemophilia B
Interventions
Non-Interventional
Other
Lead sponsor
Regeneron Pharmaceuticals
Industry
Eligibility
16 Years and older · Male only
Enrollment
11 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
9
States / cities
Aurora, Colorado • New Haven, Connecticut • Washington D.C., District of Columbia + 6 more
Source: ClinicalTrials.gov public record
Updated Aug 7, 2026 · Synced Oct 10, 2026, 9:01 AM EDT
Not listed Not applicable Interventional Accepts healthy volunteers

Pilot Randomized Control Trial of Telehealth Group for Improving Peer Relationships (PEERS) in NF1

NCT04860362
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
30 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2021 – 2022
U.S. locations
1
States / cities
Milwaukee, Wisconsin
Source: ClinicalTrials.gov public record
Updated Apr 26, 2021 · Synced Oct 10, 2026, 9:01 AM EDT
Completed Phase 3 Interventional Results available

A Study of Factor XIII Concentrate in Subjects With Congenital Factor XIII Deficiency

NCT00885742
View directory record Official record
Conditions
Factor XIII Deficiency
Interventions
FXIII Concentrate (Human)
Biological
Lead sponsor
CSL Behring
Industry
Eligibility
Not listed
Enrollment
41 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2011
U.S. locations
22
States / cities
Dothan, Alabama • Oakland, California • Orange, California + 19 more
Source: ClinicalTrials.gov public record
Updated Jul 16, 2012 · Synced Oct 10, 2026, 9:01 AM EDT
Completed No phase listed Observational

Investigation of the Genetic Causes of Kallmann Syndrome and Reproductive Disorders

NCT00494169
View directory record Official record
Conditions
Hypogonadotropic Hypogonadism, Kallmann Syndrome, Puberty, Delayed, Puberty, Precocious, GnRH Deficiency
Interventions
Not listed
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
Not listed
Enrollment
4,042 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1999 – 2022
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Jun 30, 2022 · Synced Oct 10, 2026, 9:01 AM EDT
Active, not recruiting Phase 3 Interventional

Setrusumab vs Bisphosphonates in Pediatric Subjects With Osteogenesis Imperfecta

NCT05768854
View directory record Official record
Conditions
Osteogenesis Imperfecta
Interventions
Bisphosphonate, Setrusumab
Drug · Biological
Lead sponsor
Ultragenyx Pharmaceutical Inc
Industry
Eligibility
2 Years to 6 Years
Enrollment
69 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2027
U.S. locations
13
States / cities
Phoenix, Arizona • Los Angeles, California • Aurora, Colorado + 10 more
Source: ClinicalTrials.gov public record
Updated Feb 25, 2026 · Synced Oct 10, 2026, 9:01 AM EDT
Active, not recruiting Not applicable Interventional

Spina Bifida Healthcare Navigator Program

NCT07531862
View directory record Official record
Conditions
Spina Bifida
Interventions
Behavioral Treatment
Behavioral
Lead sponsor
University of Pittsburgh
Other
Eligibility
18 Years and older
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jun 4, 2026 · Synced Oct 10, 2026, 9:01 AM EDT
Completed Phase 2 Interventional Results available

Study of Apremilast in Atopic or Contact Dermatitis

NCT00931242
View directory record Official record
Conditions
Atopic Dermatitis, Allergic Contact Dermatitis
Interventions
Apremilast
Drug
Lead sponsor
Tufts Medical Center
Other
Eligibility
18 Years and older
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2010
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Dec 20, 2010 · Synced Oct 10, 2026, 9:01 AM EDT
Recruiting No phase listed Observational

Prostate Cancer Genetic Risk Evaluation and Screening Study

NCT05129605
View directory record Official record
Conditions
Prostatic Neoplasm, Prostate Cancer, BRCA2 Mutation, BRCA1 Mutation, ATM Gene Mutation, MMR Mutation, Lynch Syndrome, Genetic Predisposition to Disease
Interventions
Prostate cancer screening
Diagnostic Test
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
35 Years to 74 Years · Male only
Enrollment
400 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2040
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Oct 9, 2024 · Synced Oct 10, 2026, 9:01 AM EDT