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ClinicalTrials.gov public records Last synced Oct 22, 2026, 5:45 PM EDT

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Showing 289–312 of 10,332 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Congenital, Hereditary, and Neonatal Diseases and Abnormalities Clear all
Recruiting Not applicable Interventional

Measuring Impact of Computer Gaming on Arm Use in Rett Syndrome

NCT05012475
View directory record Official record
Conditions
Rett Syndrome
Interventions
Modified Virtual Reality Gaming
Behavioral
Lead sponsor
Georgetown University
Other
Eligibility
4 Years to 60 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2025
U.S. locations
1
States / cities
Washington D.C., District of Columbia
Source: ClinicalTrials.gov public record
Updated Oct 3, 2024 · Synced Oct 22, 2026, 5:45 PM EDT
Recruiting Phase 1 Interventional

Serial Amnioinfusions as Regenerative Therapy for Pulmonary Hypoplasia

NCT03723564
View directory record Official record
Conditions
Renal Failure Congenital, Congenital Renal Anomaly Nos, Renal Agenesis and Dysgenesis, Lower Urinary Tract Obstructive Syndrome
Interventions
Lactated Ringers Solution for Injection
Drug
Lead sponsor
Mayo Clinic
Other
Eligibility
Not listed
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2027
U.S. locations
1
States / cities
Rochester, Minnesota
Source: ClinicalTrials.gov public record
Updated Nov 12, 2025 · Synced Oct 22, 2026, 5:45 PM EDT
Completed Not applicable Interventional

Respiratory Training in Friedreich's Ataxia

NCT06539598
View directory record Official record
Conditions
Friedreich Ataxia
Interventions
Respiratory Strength Training (RST)
Other
Lead sponsor
University of Florida
Other
Eligibility
18 Years to 65 Years
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2025
U.S. locations
1
States / cities
Gainesville, Florida
Source: ClinicalTrials.gov public record
Updated Nov 3, 2025 · Synced Oct 22, 2026, 5:45 PM EDT
Completed Phase 1 Interventional

Therapeutic Application of Intravascular Nitrite for Sickle Cell Disease

NCT00095472
View directory record Official record
Conditions
Sickle Cell Anemia
Interventions
L-NMMA, Sodium Nitrite
Drug
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
18 Years and older
Enrollment
18 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2007
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Nov 5, 2019 · Synced Oct 22, 2026, 5:45 PM EDT
Completed Phase 2 Interventional

Tuvusertib Combined With Niraparib or Lartesertib in Participants With Epithelial Ovarian Cancer (DDRiver EOC 302)

NCT06433219
View directory record Official record
Conditions
Ovarian Cancer
Interventions
Tuvusertib (M1774), Niraparib, Lartesertib (M4076)
Drug
Lead sponsor
EMD Serono Research & Development Institute, Inc.
Industry
Eligibility
18 Years and older · Female only
Enrollment
63 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
9
States / cities
Little Rock, Arkansas • San Francisco, California • Columbus, Georgia + 4 more
Source: ClinicalTrials.gov public record
Updated Sep 14, 2026 · Synced Oct 22, 2026, 5:45 PM EDT
Terminated No phase listed Observational

Chlorhexidine Swabs Effectiveness in Reducing Blood Stream Infections

NCT00485992
View directory record Official record
Conditions
Congenital Disorders
Interventions
Not listed
Lead sponsor
Children's Healthcare of Atlanta
Other
Eligibility
Up to 18 Years
Enrollment
352 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2007
U.S. locations
1
States / cities
Atlanta, Georgia
Source: ClinicalTrials.gov public record
Updated Nov 21, 2011 · Synced Oct 22, 2026, 5:45 PM EDT
Recruiting Phase 2 Interventional

GLP-1 Agonist Therapy in Cystic Fibrosis-Related Glucose Intolerance

NCT04731272
View directory record Official record
Conditions
Cystic Fibrosis, Pancreatic Insufficiency, Abnormal Glucose Tolerance, Diabetes
Interventions
Dulaglutide 0.75Mg/0.5Ml Inj Pen
Drug
Lead sponsor
University of Pennsylvania
Other
Eligibility
18 Years and older
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2028
U.S. locations
2
States / cities
Aurora, Colorado • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated May 18, 2026 · Synced Oct 22, 2026, 5:45 PM EDT
Completed Not applicable Interventional Accepts healthy volunteers Results available

Comparative Efficacy of Phenylbutyrate (PBA) vs. Benzoate in Urea Cycle Disorders

NCT02111200
View directory record Official record
Conditions
Urea Cycle Disorders, Inborn
Interventions
Sodium Benzoate, Sodium Phenylbutyrate
Drug
Lead sponsor
Baylor College of Medicine
Other
Eligibility
18 Years to 64 Years
Enrollment
7 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2014 – 2015
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Jan 9, 2018 · Synced Oct 22, 2026, 5:45 PM EDT
Recruiting Phase 1 Interventional

Sodium/Glucose Cotransporter-2 Inhibitors (SGLT2i) Therapy in Duchenne Cardiomyopathy

NCT07172971
View directory record Official record
Conditions
Duchenne Muscular Dystrophy (DMD)
Interventions
SGLT-2 inhibitor, SGLT2 inhibitor
Drug
Lead sponsor
Vanderbilt University Medical Center
Other
Eligibility
8 Years to 18 Years · Male only
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2028
U.S. locations
1
States / cities
Nashville, Tennessee
Source: ClinicalTrials.gov public record
Updated Aug 24, 2026 · Synced Oct 22, 2026, 5:45 PM EDT
Completed No phase listed Observational

Stigma, Self-management, & Quality of Life in SCD

NCT03488264
View directory record Official record
Conditions
Sickle Cell Disease, Quality of Life, Stigma, Social, Disease Self-Management
Interventions
Not listed
Lead sponsor
Duke University
Other
Eligibility
18 Years and older
Enrollment
102 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Apr 11, 2019 · Synced Oct 22, 2026, 5:45 PM EDT
Active, not recruiting Not applicable Interventional Results available

Haploidentical Hematopoietic Cell Transplantation Using TCR Alpha/Beta and CD19 Depletion

NCT05236764
View directory record Official record
Conditions
Acute Lymphoblastic Leukemia in Remission, Acute Myeloid Leukemia in Remission, Myelodysplastic Syndromes, Chronic Myeloid Leukemia, Hemophagocytic Lymphohistiocytoses, Primary Immunodeficiency Diseases, Hemoglobinopathies, Severe Aplastic Anemia, Cytopenia, Bone Marrow Failure Syndrome, Severe Chronic Active Epstein-Barr Virus Infection
Interventions
CliniMACS
Device
Lead sponsor
Baylor College of Medicine
Other
Eligibility
Up to 55 Years
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2027
U.S. locations
2
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Oct 8, 2026 · Synced Oct 22, 2026, 5:45 PM EDT
Completed Phase 2 Interventional Results available

CAMB/MAT2203 in Patients With Mucocutaneous Candidiasis

NCT02629419
View directory record Official record
Conditions
Candidiasis, Chronic Mucocutaneous
Interventions
Amphotericin B
Drug
Lead sponsor
Matinas BioPharma Nanotechnologies, Inc.
Industry
Eligibility
18 Years to 75 Years
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2022
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Aug 7, 2024 · Synced Oct 22, 2026, 5:45 PM EDT
Completed Phase 4 Interventional

Cefiderocol Pharmacokinetics in Adult Patients With Cystic Fibrosis

NCT05314764
View directory record Official record
Conditions
Cystic Fibrosis, Pneumonia, Bacterial
Interventions
Cefiderocol
Drug
Lead sponsor
Hartford Hospital
Other
Eligibility
18 Years and older
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2024
U.S. locations
4
States / cities
Hartford, Connecticut • Indianapolis, Indiana • Pittsburgh, Pennsylvania + 1 more
Source: ClinicalTrials.gov public record
Updated Dec 16, 2024 · Synced Oct 22, 2026, 5:45 PM EDT
Active, not recruiting No phase listed Observational Accepts healthy volunteers

Intrarenal Microvasculature in ADPKD

NCT05288998
View directory record Official record
Conditions
Autosomal Dominant Polycystic Kidney Disease
Interventions
Not listed
Lead sponsor
Mayo Clinic
Other
Eligibility
18 Years to 40 Years
Enrollment
34 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2021 – 2026
U.S. locations
1
States / cities
Rochester, Minnesota
Source: ClinicalTrials.gov public record
Updated Jan 16, 2026 · Synced Oct 22, 2026, 5:45 PM EDT
Completed No phase listed Observational

VAlidation of a Lower Cost aneUploidy scrEen

NCT03087357
View directory record Official record
Conditions
Down Syndrome, Trisomy 18, Trisomy 13
Interventions
SmartNIPT
Other
Lead sponsor
Women and Infants Hospital of Rhode Island
Other
Eligibility
18 Years and older · Female only
Enrollment
2,443 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2020
U.S. locations
13
States / cities
Los Angeles, California • Aurora, Colorado • Bridgeport, Connecticut + 9 more
Source: ClinicalTrials.gov public record
Updated Sep 22, 2022 · Synced Oct 22, 2026, 5:45 PM EDT
Not yet recruiting Not applicable Interventional

Early Intervention in Infants With Unrepaired Cleft Palate: Language, Palatal Function, and Articulation.

NCT07219901
View directory record Official record
Conditions
Cleft Lip and Cleft Palate, Cleft Lip Palate, Cleft Lip and/or Palate, Cleft Palate, Cleft Palate Children, Cleft Palates, Cleft Palate Repair
Interventions
Early intervention for language, articulation and palatal function skills
Behavioral
Lead sponsor
Old Dominion University
Other
Eligibility
6 Months to 12 Months
Enrollment
200 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
1
States / cities
Norfolk, Virginia
Source: ClinicalTrials.gov public record
Updated Oct 22, 2025 · Synced Oct 22, 2026, 5:45 PM EDT
Completed Phase 2 Interventional

Defibrotide in Sickle Cell Disease-Related Acute Chest Syndrome

NCT03805581
View directory record Official record
Conditions
Sickle Cell Disease, Acute Chest Syndrome
Interventions
Defibrotide
Drug
Lead sponsor
New York Medical College
Other
Eligibility
2 Years to 40 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2023
U.S. locations
1
States / cities
Valhalla, New York
Source: ClinicalTrials.gov public record
Updated Dec 8, 2023 · Synced Oct 22, 2026, 5:45 PM EDT
Enrolling by invitation Not applicable Interventional Accepts healthy volunteers

Parent and Infant Inter(X)Action Intervention (PIXI)

NCT03836300
View directory record Official record
Conditions
Fragile X Syndrome, Angelman Syndrome, Prader-Willi Syndrome, Dup15Q Syndrome, Duchenne Muscular Dystrophy, Phelan-McDermid Syndrome, Rett Syndrome, Smith Magenis Syndrome, Williams Syndrome, Turner Syndrome, Klinefelter Syndrome, Chromosome 22q11.2 Deletion Syndrome, Tuberous Sclerosis, Down Syndrome
Interventions
Parent-Infant Inter(X)action Intervention (PIXI)
Behavioral
Lead sponsor
RTI International
Other
Eligibility
Up to 99 Years
Enrollment
120 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2018 – 2026
U.S. locations
1
States / cities
Research Triangle Park, North Carolina
Source: ClinicalTrials.gov public record
Updated Jul 13, 2026 · Synced Oct 22, 2026, 5:45 PM EDT
Recruiting No phase listed Observational

Delineating the Molecular Spectrum and the Clinical, Imaging and Neuronal Phenotype of Chopra-Amiel-Gordon Syndrome

NCT05528744
View directory record Official record
Conditions
Genetic Disease, Chopra-Amiel-Gordon Syndrome, CAGS, ANKRD17
Interventions
Observational Study, Sample collection only
Other
Lead sponsor
Boston Children's Hospital
Other
Eligibility
Not listed
Enrollment
125 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2030
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Feb 17, 2026 · Synced Oct 22, 2026, 5:45 PM EDT
Completed Not applicable Interventional Results available

SpHincterotomy for Acute Recurrent Pancreatitis

NCT03609944
View directory record Official record
Conditions
Pancreatitis, Pancreas Divisum, Pancreatitis, Acute, Pancreatitis Idiopathic, Pancreas Inflamed
Interventions
ERCP with miES, EUS
Procedure
Lead sponsor
Oregon Health and Science University
Other
Eligibility
18 Years and older
Enrollment
181 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2025
U.S. locations
20
States / cities
Little Rock, Arkansas • Los Angeles, California • San Francisco, California + 16 more
Source: ClinicalTrials.gov public record
Updated Dec 30, 2025 · Synced Oct 22, 2026, 5:45 PM EDT
Recruiting Not applicable Interventional

North American Fetal Therapy Network for Long-term Outcome Following Fetoscopic Endoluminal Tracheal Occlusion in Severe Left and Right Congenital Diaphragmatic Hernia

NCT06739356
View directory record Official record
Conditions
Congenital Diaphragmatic Hernia
Interventions
Fetal Treatment arm (FETO Group)
Device
Lead sponsor
The University of Texas Health Science Center, Houston
Other
Eligibility
18 Years and older · Female only
Enrollment
80 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2031
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Oct 20, 2026 · Synced Oct 22, 2026, 5:45 PM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

Flaxseed Modulates Inflammation and Oxidative Stress in CF

NCT02014181
View directory record Official record
Conditions
Cystic Fibrosis, Oxidative Stress, Inflammation
Interventions
finely ground flaxseed powder
Dietary Supplement
Lead sponsor
University of Pennsylvania
Other
Eligibility
18 Years to 64 Years
Enrollment
10 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2012 – 2013
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jan 7, 2014 · Synced Oct 22, 2026, 5:45 PM EDT
Completed Phase 4 Interventional Results available

Fibrinogen Concentrate vs Cryoprecipitate

NCT03014700
View directory record Official record
Conditions
Congenital Heart Disease
Interventions
Fibrinogen Concentrate, Cryoprecipitate
Biological
Lead sponsor
Stanford University
Other
Eligibility
1 Day to 12 Months
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2018
U.S. locations
2
States / cities
Stanford, California • Emory, Georgia
Source: ClinicalTrials.gov public record
Updated May 7, 2019 · Synced Oct 22, 2026, 5:45 PM EDT
Completed Not applicable Interventional

SoftHand Comparison Study

NCT05328934
View directory record Official record
Conditions
Upper Extremity Injury, Upper Extremity Deformities, Congenital, Amputation, Traumatic, Amputation, Congenital
Interventions
SoftHand Pro, Ossur i-Limb
Device
Lead sponsor
Mayo Clinic
Other
Eligibility
18 Years to 99 Years
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2026
U.S. locations
2
States / cities
Tempe, Arizona • Rochester, Minnesota
Source: ClinicalTrials.gov public record
Updated Jun 12, 2026 · Synced Oct 22, 2026, 5:45 PM EDT