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ClinicalTrials.gov public records Last synced Oct 22, 2026, 4:28 AM EDT

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Showing 265–288 of 10,540 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Congenital Disorders Clear all
Completed Phase 3 Interventional Results available

Study to Evaluate Efficacy and Safety of Inclisiran in Adolescents With Homozygous Familial Hypercholesterolemia

NCT04659863
View directory record Official record
Conditions
Familial Hypercholesterolemia - Homozygous
Interventions
Inclisiran, Placebo
Drug
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
12 Years to 17 Years
Enrollment
13 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2024
U.S. locations
1
States / cities
Boca Raton, Florida
Source: ClinicalTrials.gov public record
Updated Jan 13, 2026 · Synced Oct 22, 2026, 4:28 AM EDT
Completed No phase listed Observational

Inadvertent Hyperventilation During Intraoperative Care in Neonates

NCT03823716
View directory record Official record
Conditions
Neonatal Disorder
Interventions
Not listed
Lead sponsor
Nationwide Children's Hospital
Other
Eligibility
Up to 36 Weeks
Enrollment
112 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2019
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Feb 17, 2020 · Synced Oct 22, 2026, 4:28 AM EDT
Completed Phase 1 Interventional Results available

A Study to Describe the Pharmacokinetics of Acyclovir in Premature Infants (PTN_Acyclo)

NCT00942084
View directory record Official record
Conditions
Herpes Simplex Virus, Neonatal Sepsis
Interventions
Acyclovir
Drug
Lead sponsor
Phillip Brian Smith
Other
Eligibility
Up to 45 Days
Enrollment
32 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2012
U.S. locations
3
States / cities
Wichita, Kansas • New Orleans, Louisiana • Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Jan 8, 2019 · Synced Oct 22, 2026, 4:28 AM EDT
Terminated Phase 2 Interventional Results available

Sirolimus, Tacrolimus, Thymoglobulin and Rituximab as Graft-versus-Host-Disease Prophylaxis in Patients Undergoing Haploidentical and HLA Partially Matched Donor Hematopoietic Cell Transplantation

NCT01116232
View directory record Official record
Conditions
Chronic Myeloproliferative Disorders, Graft Versus Host Disease, Leukemia, Lymphoma, Lymphoproliferative Disorder, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Neoplasms
Interventions
anti-thymocyte globulin, rituximab, sirolimus, tacrolimus, laboratory biomarker analysis, allogeneic hematopoietic stem cell transplantation, management of therapy complications, peripheral blood stem cell transplantation
Biological · Drug · Other + 1 more
Lead sponsor
Barbara Ann Karmanos Cancer Institute
Other
Eligibility
18 Years to 120 Years
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2013
U.S. locations
1
States / cities
Detroit, Michigan
Source: ClinicalTrials.gov public record
Updated Mar 26, 2019 · Synced Oct 22, 2026, 4:28 AM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

A Phase 1 Study To Evaluate The Pharmacokinetics, Safety, And Tolerability Of Dimebon [PF-01913539] In Japanese And Western Healthy Subjects

NCT00825084
View directory record Official record
Conditions
Alzheimer's Disease, Huntington's Disease
Interventions
Dimebon
Drug
Lead sponsor
Pfizer
Industry
Eligibility
18 Years to 55 Years
Enrollment
45 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2009
U.S. locations
1
States / cities
Glendale, California
Source: ClinicalTrials.gov public record
Updated Apr 26, 2011 · Synced Oct 22, 2026, 4:28 AM EDT
Terminated Not applicable Interventional Results available

Methacetin Breath Test in Patients With Liver Disease Secondary to Heart Disease

NCT04176458
View directory record Official record
Conditions
Dilated Cardiomyopathy, Tricuspid Atresia
Interventions
Methacetin Breath Test (MBT)
Device
Lead sponsor
Northwestern University
Other
Eligibility
18 Years and older
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2021
U.S. locations
1
States / cities
Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Apr 5, 2023 · Synced Oct 22, 2026, 4:28 AM EDT
Completed Early Phase 1 Interventional Accepts healthy volunteers

Atorvastatin ± Aspirin in Lynch Syndrome Syndrome

NCT04379999
View directory record Official record
Conditions
Lynch Syndrome
Interventions
Atorvastatin 20mg, Atorvastatin 20mg AND Aspirin 325 mg
Drug
Lead sponsor
Fox Chase Cancer Center
Other
Eligibility
18 Years to 75 Years
Enrollment
43 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2018 – 2025
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Aug 7, 2025 · Synced Oct 22, 2026, 4:28 AM EDT
Completed Phase 2Phase 3 Interventional Results available

Trial in Adult Participants With Spinocerebellar Ataxia (SCA)

NCT02960893
View directory record Official record
Conditions
Spinocerebellar Ataxias, Spinocerebellar Ataxia Genotype Type 1, Spinocerebellar Ataxia Genotype Type 2, Spinocerebellar Ataxia Genotype Type 3, Spinocerebellar Ataxia Genotype Type 6, Spinocerebellar Ataxia Genotype Type 7, Spinocerebellar Ataxia Genotype Type 8, Spinocerebellar Ataxia Genotype Type 10
Interventions
Troriluzole, Placebo
Drug
Lead sponsor
Biohaven Pharmaceuticals, Inc.
Industry
Eligibility
18 Years to 75 Years
Enrollment
141 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2024
U.S. locations
18
States / cities
Phoenix, Arizona • Long Beach, California • Los Angeles, California + 13 more
Source: ClinicalTrials.gov public record
Updated Oct 8, 2025 · Synced Oct 22, 2026, 4:28 AM EDT
Completed Not applicable Interventional

Patent Ductus Arteriosus (PDA) Screening Trial

NCT01031316
View directory record Official record
Conditions
Ductus Arteriosus, Patent
Interventions
Nondisclosure of screening echocardiogram results, Disclosure of screening echocardiogram results
Other
Lead sponsor
University of Pennsylvania
Other
Eligibility
Up to 72 Hours
Enrollment
88 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2011
U.S. locations
2
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jan 3, 2014 · Synced Oct 22, 2026, 4:28 AM EDT
Completed Phase 3 Interventional Results available

Pharmacokinetics and Safety of rFVIIIFc Manufactured at 15,000 L (15K) Scale

NCT02502149
View directory record Official record
Conditions
Severe Hemophilia A
Interventions
rFVIIIFc
Biological
Lead sponsor
Bioverativ Therapeutics Inc.
Industry
Eligibility
12 Years and older · Male only
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2017
U.S. locations
8
States / cities
Los Angeles, California • Chicago, Illinois • Indianapolis, Indiana + 5 more
Source: ClinicalTrials.gov public record
Updated Dec 19, 2020 · Synced Oct 22, 2026, 4:28 AM EDT
Completed No phase listed Observational

Immune Profiles in CF Fungal Infection

NCT04476758
View directory record Official record
Conditions
Cystic Fibrosis, Fungal Infection, Allergic Bronchopulmonary Aspergillosis
Interventions
Not listed
Lead sponsor
University of Alabama at Birmingham
Other
Eligibility
8 Years to 25 Years
Enrollment
24 participants
Timeline
2021 – 2023
U.S. locations
1
States / cities
Highlands Ranch, Colorado
Source: ClinicalTrials.gov public record
Updated Jul 18, 2024 · Synced Oct 22, 2026, 4:28 AM EDT
Completed No phase listed Observational

Association Between Fuchs' Endothelial Corneal Dystrophy Severity and Estrogen Exposure

NCT04564378
View directory record Official record
Conditions
Fuchs Dystrophy
Interventions
This is an observational study. There is no therapeutic intervention.
Other
Lead sponsor
State University of New York at Buffalo
Other
Eligibility
55 Years and older
Enrollment
43 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2023
U.S. locations
1
States / cities
Buffalo, New York
Source: ClinicalTrials.gov public record
Updated Oct 1, 2024 · Synced Oct 22, 2026, 4:28 AM EDT
Recruiting No phase listed Observational

Tissue, Blood, and Body Fluid Sample Collection From Patients With Hematologic Cancer

NCT01137643
View directory record Official record
Conditions
Chronic Myeloproliferative Disorders, Leukemia, Lymphoma, Lymphoproliferative Disorder, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Neoplasms, Nonmalignant Neoplasm
Interventions
biologic sample preservation procedure, cytology specimen collection procedure
Other
Lead sponsor
UNC Lineberger Comprehensive Cancer Center
Other
Eligibility
18 Years to 120 Years
Enrollment
15,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2040
U.S. locations
1
States / cities
Chapel Hill, North Carolina
Source: ClinicalTrials.gov public record
Updated Oct 27, 2025 · Synced Oct 22, 2026, 4:28 AM EDT
Completed No phase listed Observational

Regulation of Cerebral Blood Flow in Very Low Birth Weight Infants

NCT00579943
View directory record Official record
Conditions
Infant, Very Low Birth Weight, Infant, Premature, Diseases, Cerebral Hemorrhage
Interventions
Not listed
Lead sponsor
University of Arkansas
Other
Eligibility
1 Hour to 7 Days
Enrollment
200 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2008
U.S. locations
1
States / cities
Little Rock, Arkansas
Source: ClinicalTrials.gov public record
Updated Jun 4, 2008 · Synced Oct 22, 2026, 4:28 AM EDT
Terminated No phase listed Observational

Clinical Performance of the MaterniT21 PLUS LDT in Multiple Gestation Pregnancies

NCT02226315
View directory record Official record
Conditions
Down Syndrome, Edwards Syndrome, Patau Syndrome, Turner Syndrome
Interventions
Not listed
Lead sponsor
Sequenom, Inc.
Industry
Eligibility
18 Years and older · Female only
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2015
U.S. locations
1
States / cities
Denver, Colorado
Source: ClinicalTrials.gov public record
Updated Sep 17, 2015 · Synced Oct 22, 2026, 4:28 AM EDT
Terminated Phase 1Phase 2 Interventional Results available

Ascending Dose Study of Genome Editing by the Zinc Finger Nuclease (ZFN) Therapeutic SB-318 in Subjects With MPS I

NCT02702115
View directory record Official record
Conditions
MPS I
Interventions
SB-318
Biological
Lead sponsor
Sangamo Therapeutics
Industry
Eligibility
5 Years and older
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2021
U.S. locations
1
States / cities
Oakland, California
Source: ClinicalTrials.gov public record
Updated Jan 26, 2023 · Synced Oct 22, 2026, 4:28 AM EDT
Completed Phase 2 Interventional Accepts healthy volunteers Results available

Antioxidant Therapy With N-acetylcysteine for Learning and Motor Behavior in Children With Neurofibromatosis Type 1

NCT04481035
View directory record Official record
Conditions
Neurofibromatosis 1
Interventions
N-acetylcysteine (NAC), Placebo
Drug · Other
Lead sponsor
Children's Hospital Medical Center, Cincinnati
Other
Eligibility
8 Years to 16 Years
Enrollment
5 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2019 – 2021
U.S. locations
1
States / cities
Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Apr 21, 2026 · Synced Oct 22, 2026, 4:28 AM EDT
Active, not recruiting Not applicable Interventional

Grand Valley State University (GVSU) Skills on Wheels

NCT05339932
View directory record Official record
Conditions
Spina Bifida, Cerebral Palsy, Genetic Disease, Spinal Cord Injuries, Gross Motor Development Delay, Motor Skills Disorders
Interventions
Manual Wheelchair Skills Training
Other
Lead sponsor
Grand Valley State University
Other
Eligibility
5 Years to 17 Years
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2027
U.S. locations
1
States / cities
Grand Rapids, Michigan
Source: ClinicalTrials.gov public record
Updated Sep 15, 2025 · Synced Oct 22, 2026, 4:28 AM EDT
Completed Phase 2 Interventional Results available

A Study to Assess the Safety, Tolerability, and Pharmacokinetics of Brensocatib Tablets in Adults With Cystic Fibrosis

NCT05090904
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
Brensocatib, Placebo
Drug
Lead sponsor
Insmed Incorporated
Industry
Eligibility
18 Years and older
Enrollment
29 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2022
U.S. locations
14
States / cities
Gainesville, Florida • Augusta, Georgia • Glenview, Illinois + 10 more
Source: ClinicalTrials.gov public record
Updated Mar 12, 2026 · Synced Oct 22, 2026, 4:28 AM EDT
Recruiting Phase 3 Interventional

A Research Study Looking at Long-term Treatment With Etavopivat in People With Sickle Cell Disease or Thalassaemia

NCT06609226
View directory record Official record
Conditions
Sickle Cell Disease, Thalassemia
Interventions
Etavopivat A, Etavopivat B, Etavopivat C
Drug
Lead sponsor
Novo Nordisk A/S
Industry
Eligibility
2 Years and older
Enrollment
480 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2030
U.S. locations
36
States / cities
Birmingham, Alabama • Phoenix, Arizona • Los Angeles, California + 27 more
Source: ClinicalTrials.gov public record
Updated Sep 18, 2026 · Synced Oct 22, 2026, 4:28 AM EDT
Completed Not applicable Interventional

Treatment and Management of Women With Bleeding Disorders

NCT00111215
View directory record Official record
Conditions
Menorrhagia, Blood Coagulation Disorders, Blood Platelet Disorders, Von Willebrand Disease, Hematologic Disease
Interventions
Tranexamic Acid, Desmopressin Acetate
Drug
Lead sponsor
Centers for Disease Control and Prevention
Federal
Eligibility
18 Years to 50 Years · Female only
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2006
U.S. locations
6
States / cities
Atlanta, Georgia • East Lansing, Michigan • Rochester, Minnesota + 3 more
Source: ClinicalTrials.gov public record
Updated Mar 13, 2007 · Synced Oct 22, 2026, 4:28 AM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

A Safety, Tolerability and Pharmacokinetics Study of Topical Ointment YR001 in Adult Healthy Volunteers

NCT05718921
View directory record Official record
Conditions
Atopic Dermatitis, Psoriasis
Interventions
YR001 Dose A on low body surface area, YR001 Dose A on middle body surface area, YR001 Dose A on high body surface area, YR001 Dose B on low body surface area, YR001 Dose B on middle body surface area, YR001 Dose B on high body surface area, Placebo on low body surface area, Placebo on middle body surface area, Placebo on high body surface area, YR001 Dose A on high body surface area twice daily, YR001 Dose B on high body surface area twice daily, Placebo on high body surface area twice daily
Drug
Lead sponsor
Hangzhou Yirui Pharmaceutical Technology Co., Ltd
Industry
Eligibility
18 Years to 65 Years
Enrollment
54 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2023
U.S. locations
1
States / cities
Encinitas, California
Source: ClinicalTrials.gov public record
Updated Jan 24, 2024 · Synced Oct 22, 2026, 4:28 AM EDT
Recruiting Phase 1 Interventional

Safety and Preliminary Efficacy of ULSC in Facioscapulohumeral Muscular Dystrophy (FSHD)

NCT07086521
View directory record Official record
Conditions
FSHD - Facioscapulohumeral Muscular Dystrophy
Interventions
ULSC, Placebo
Biological
Lead sponsor
Restem, LLC.
Industry
Eligibility
15 Years and older
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2030
U.S. locations
1
States / cities
Palo Alto, California
Source: ClinicalTrials.gov public record
Updated Oct 21, 2026 · Synced Oct 22, 2026, 4:28 AM EDT
Completed Phase 2 Interventional Accepts healthy volunteers Results available

Phase II Study of Florbetaben (BAY94-9172) PET Imaging for Detection/Exclusion of Cerebral β-amyloid.

NCT00928304
View directory record Official record
Conditions
Down Syndrome, Amyloid Beta-protein
Interventions
Florbetaben (BAY94-9172)
Drug
Lead sponsor
Lantheus Biosciences Ltd.
Industry
Eligibility
21 Years and older
Enrollment
109 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2009 – 2011
U.S. locations
2
States / cities
Sun City, Arizona • New Haven, Connecticut
Source: ClinicalTrials.gov public record
Updated Mar 5, 2014 · Synced Oct 22, 2026, 4:28 AM EDT