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ClinicalTrials.gov public records Last synced Oct 14, 2026, 8:21 PM EDT

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Showing 433–456 of 10,529 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Congenital Diseases Clear all
Recruiting Phase 3 Interventional

A Study of Sepiapterin in Participants With Phenylketonuria (PKU)

NCT06302348
View directory record Official record
Conditions
Phenylketonuria
Interventions
Sepiapterin
Drug
Lead sponsor
PTC Therapeutics
Industry
Eligibility
Up to 9 Years
Enrollment
56 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2031
U.S. locations
1
States / cities
Indianapolis, Indiana
Source: ClinicalTrials.gov public record
Updated Aug 28, 2026 · Synced Oct 14, 2026, 8:21 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Cortical Excitability in Obsessive-Compulsive and Related Disorders

NCT00029068
View directory record Official record
Conditions
Obsessive Compulsive Disorder, Healthy
Interventions
Not listed
Lead sponsor
National Institute of Mental Health (NIMH)
NIH
Eligibility
Not listed
Enrollment
145 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2002 – 2003
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 4, 2008 · Synced Oct 14, 2026, 8:21 PM EDT
Withdrawn No phase listed Observational

Exhaled Carbon Monoxide as a Marker of Hemolysis in Sickle Cell Disease- an Exploratory Study

NCT01888614
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Not listed
Lead sponsor
University Hospitals Cleveland Medical Center
Other
Eligibility
18 Years and older
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2016
U.S. locations
1
States / cities
Cleveland, Ohio
Source: ClinicalTrials.gov public record
Updated Dec 23, 2016 · Synced Oct 14, 2026, 8:21 PM EDT
Recruiting Phase 2 Interventional

Cord Blood Transplant in Children and Young Adults With Blood Cancers and Non-malignant Disorders

NCT04644016
View directory record Official record
Conditions
AML, ALL, MDS, MPD Withou Myelofibrosis, NHL or HL, Inherited Metabolic Disorders, Hemoglobinopathies, Bone Marrow Failure, HLH
Interventions
Clofarabine, Fludarabine, Busulfan, Cyclosporine-A, Mycophenolate Mofetil, Cord Blood Graft
Drug · Biological
Lead sponsor
Memorial Sloan Kettering Cancer Center
Other
Eligibility
Up to 21 Years
Enrollment
31 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2026
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Mar 13, 2026 · Synced Oct 14, 2026, 8:21 PM EDT
Enrolling by invitation No phase listed Observational

Investigation of Filaggrin Gene Mutations Among Latinx Patients With Atopic Dermatitis

NCT06464133
View directory record Official record
Conditions
Atopic Dermatitis, Eczema
Interventions
Not listed
Lead sponsor
University of Pennsylvania
Other
Eligibility
Not listed
Enrollment
300 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Apr 24, 2026 · Synced Oct 14, 2026, 8:21 PM EDT
Completed Phase 1 Interventional

7-Hydroxystaurosporine and Perifosine in Treating Patients With Relapsed or Refractory Acute Leukemia, Chronic Myelogenous Leukemia or High Risk Myelodysplastic Syndromes

NCT00301938
View directory record Official record
Conditions
Accelerated Phase Chronic Myelogenous 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), Blastic Phase Chronic Myelogenous Leukemia, Myelodysplastic/Myeloproliferative Neoplasms, Previously Treated Myelodysplastic Syndromes, Recurrent Adult Acute Lymphoblastic Leukemia, Recurrent Adult Acute Myeloid Leukemia, Relapsing Chronic Myelogenous Leukemia, Secondary Acute Myeloid Leukemia, T-cell Adult Acute Lymphoblastic Leukemia, Untreated Adult Acute Lymphoblastic Leukemia, Untreated Adult Acute Myeloid Leukemia
Interventions
7-hydroxystaurosporine, perifosine, pharmacological study
Drug · Other
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
18 Years and older
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2005
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 30, 2013 · Synced Oct 14, 2026, 8:21 PM EDT
Completed Not applicable Interventional Results available

Using Patient-Centered Guidelines in a Technology Platform to Improve Health Care in Adults With Sickle Cell Disease

NCT03629678
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
mobile health application
Other
Lead sponsor
Vanderbilt University Medical Center
Other
Eligibility
18 Years to 70 Years
Enrollment
67 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2023
U.S. locations
2
States / cities
Columbus, Ohio • Nashville, Tennessee
Source: ClinicalTrials.gov public record
Updated Jan 14, 2025 · Synced Oct 14, 2026, 8:21 PM EDT
Terminated Phase 2 Interventional Accepts healthy volunteers Results available

Safety, Tolerability, and Immunogenicity of One Dose of NDV 3A Vaccine in People With STAT3-Mutated Hyper-IgE Syndrome

NCT02996448
View directory record Official record
Conditions
Autosomal-dominant Hyper-IgE Syndrome
Interventions
NDV-3A
Drug
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
18 Years to 55 Years
Enrollment
3 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2016 – 2018
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jun 30, 2020 · Synced Oct 14, 2026, 8:21 PM EDT
Terminated Phase 1 Interventional

DFF332 as a Single Agent and in Combination With Everolimus & Immuno-Oncology Agents in Advanced/Relapsed Renal Cancer & Other Malignancies

NCT04895748
View directory record Official record
Conditions
Carcinoma, Renal Cell
Interventions
DFF332, RAD001, PDR001, NIR178
Drug
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
18 Years to 100 Years
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2026
U.S. locations
5
States / cities
Duarte, California • Boston, Massachusetts • St Louis, Missouri + 2 more
Source: ClinicalTrials.gov public record
Updated Mar 3, 2026 · Synced Oct 14, 2026, 8:21 PM EDT
Completed Not applicable Interventional

High Intensity Exercise and Improving Physical Activity Among People With Neurologic Dysfunction

NCT06945835
View directory record Official record
Conditions
Cerebral Palsy (CP), Stroke, Spinal Cord Injury, Traumatic Brain Injury, Charcot Marie Tooth Disease (CMT), Spina Bifida
Interventions
Participate in high intensity exercise of at least 20 minutes in 60 minutes session of RPE greater than 6.
Behavioral
Lead sponsor
University of Hartford
Other
Eligibility
8 Years to 99 Years
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025
U.S. locations
1
States / cities
West Hartford, Connecticut
Source: ClinicalTrials.gov public record
Updated Nov 3, 2025 · Synced Oct 14, 2026, 8:21 PM EDT
Completed Phase 4 Interventional

Synchronized Intermittent Mandatory Ventilation (SIMV) Versus Nasal Intermittent Positive Pressure Ventilation (NIPPV) In Preterm Infants With Respiratory Distress

NCT00486850
View directory record Official record
Conditions
Respiratory Distress Syndrome, Newborn
Interventions
Extubation to Nasal IPPV
Other
Lead sponsor
University of Southern California
Other
Eligibility
10 Minutes to 120 Minutes
Enrollment
110 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2009
U.S. locations
4
States / cities
Los Angeles, California • San Diego, California • Augusta, Georgia + 1 more
Source: ClinicalTrials.gov public record
Updated Jul 10, 2009 · Synced Oct 14, 2026, 8:21 PM EDT
Completed Not applicable Interventional Accepts healthy volunteers Results available

Generic vs. Name-Brand Levothyroxine

NCT00403390
View directory record Official record
Conditions
Congenital Hypothyroidism, Hypothyroidism
Interventions
Brand Name Levothyroxine (Synthroid), Generic formulation of Levothyroxine
Drug
Lead sponsor
Boston Children's Hospital
Other
Eligibility
3 Years to 18 Years
Enrollment
34 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2006 – 2010
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Feb 23, 2018 · Synced Oct 14, 2026, 8:21 PM EDT
Terminated Phase 3 Interventional Results available

Sleep Apnea and Fetal Growth Restriction

NCT04084990
View directory record Official record
Conditions
Obstructive Sleep Apnea, Fetal Growth Restriction, Pregnancy Related
Interventions
S9 VPAP Adapt
Device
Lead sponsor
Washington University School of Medicine
Other
Eligibility
18 Years to 50 Years · Female only
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2021
U.S. locations
2
States / cities
St Louis, Missouri • Rochester, New York
Source: ClinicalTrials.gov public record
Updated Mar 9, 2022 · Synced Oct 14, 2026, 8:21 PM EDT
Recruiting Not applicable Interventional

A Pragmatic Clinical Trial of the WE BEAT Well-Being Education Program in Adolescent Congenital Heart Disease: WE BEAT CHD Study

NCT07525843
View directory record Official record
Conditions
Patient-Reported Outcome Measures (PROMs), Pragmatic Trial, Adolescent Congenital Heart Disease, Resiliency-building Intervention, Resilience, Psychological, Telemedicine-Based Education
Interventions
Telemedicine-based resiliency-building intervention
Behavioral
Lead sponsor
Carelon Research
Other
Eligibility
12 Years to 17 Years
Enrollment
390 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2029
U.S. locations
14
States / cities
Stanford, California • Aurora, Colorado • Atlanta, Georgia + 10 more
Source: ClinicalTrials.gov public record
Updated Jul 17, 2026 · Synced Oct 14, 2026, 8:21 PM EDT
Terminated Phase 4 Interventional Results available

Sabril for Complex Partial Seizures in Adult Tolerability Study (TS) Patients

NCT01266291
View directory record Official record
Conditions
Complex Partial Seizures
Interventions
vigabatrin
Drug
Lead sponsor
University of Pennsylvania
Other
Eligibility
18 Years and older
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2013
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jul 12, 2017 · Synced Oct 14, 2026, 8:21 PM EDT
Completed Phase 3 Interventional

Ribavirin With or Without Monoclonal Antibody Therapy in Treating Patients Who Develop RSV Pneumonia Following Peripheral Stem Cell Transplantation

NCT00014391
View directory record Official record
Conditions
Cancer
Interventions
palivizumab, ribavirin
Biological · Drug
Lead sponsor
Fred Hutchinson Cancer Center
Other
Eligibility
Not listed
Healthy volunteers
Healthy volunteers not accepted
Timeline
1999 – 2001
U.S. locations
1
States / cities
Seattle, Washington
Source: ClinicalTrials.gov public record
Updated Apr 2, 2010 · Synced Oct 14, 2026, 8:21 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Clinical Research Core Dental Screening Protocol

NCT00090818
View directory record Official record
Conditions
Oral Mucosal Disease
Interventions
Not listed
Lead sponsor
National Institute of Dental and Craniofacial Research (NIDCR)
NIH
Eligibility
Not listed
Enrollment
441 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2004 – 2010
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 2, 2017 · Synced Oct 14, 2026, 8:21 PM EDT
Completed Not applicable Interventional

Regional Anesthesia for Cardiothoracic Enhanced Recovery (RACER) Study

NCT03611374
View directory record Official record
Conditions
Congenital Heart Defect, Congenital Heart Disease, Anesthesia, Local, Opioid Use
Interventions
Bilateral Erector Spinae Plane Block (ESPB)
Procedure
Lead sponsor
Stanford University
Other
Eligibility
0 Years to 99 Years
Enrollment
38 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2023
U.S. locations
1
States / cities
Palo Alto, California
Source: ClinicalTrials.gov public record
Updated Sep 28, 2023 · Synced Oct 14, 2026, 8:21 PM EDT
Withdrawn Phase 4 Interventional

The Effect of Velaglucerase Alfa (Vpriv) on Skeletal Development in Pediatric Gaucher Disease

NCT02528617
View directory record Official record
Conditions
Gaucher Disease Type 1, Gaucher Disease Type 3
Interventions
Velaglucerase alfa
Drug
Lead sponsor
Baylor Research Institute
Other
Eligibility
4 Years to 14 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2017
U.S. locations
1
States / cities
Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Mar 2, 2018 · Synced Oct 14, 2026, 8:21 PM EDT
Completed No phase listed Observational Results available

Pediatric Hydroxyurea Phase III Clinical Trial (BABY HUG) Follow-up Observational Study II Protocol

NCT01783990
View directory record Official record
Conditions
Sickle Cell Anemia
Interventions
Hydroxyurea
Drug
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
24 Months to 18 Years
Enrollment
150 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2016
U.S. locations
14
States / cities
Birmingham, Alabama • Washington D.C., District of Columbia • Miami, Florida + 9 more
Source: ClinicalTrials.gov public record
Updated Aug 20, 2020 · Synced Oct 14, 2026, 8:21 PM EDT
Recruiting Phase 2Phase 3 Interventional

FORTIFI-HN01: A Study of Ficerafusp Alfa (BCA101) or Placebo in Combination With Pembrolizumab in First-Line PD-L1-pos, R or M HNSCC

NCT06788990
View directory record Official record
Conditions
Metastatic Head and Neck Squamous Cell Carcinoma, Recurrent Head and Neck Squamous Cell Carcinoma
Interventions
Ficerafusp alfa, Pembrolizumab (KEYTRUDA®), Placebo
Drug
Lead sponsor
Bicara Therapeutics
Industry
Eligibility
18 Years and older
Enrollment
650 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2029
U.S. locations
55
States / cities
Birmingham, Alabama • Phoenix, Arizona • La Jolla, California + 41 more
Source: ClinicalTrials.gov public record
Updated Oct 2, 2026 · Synced Oct 14, 2026, 8:21 PM EDT
Completed Phase 2 Interventional Results available

Decitabine for High-Risk Sickle Cell Disease

NCT01375608
View directory record Official record
Conditions
Neutropenia, Sickle Cell Disease
Interventions
Decitabine
Drug
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
18 Years and older
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2016
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 1, 2017 · Synced Oct 14, 2026, 8:21 PM EDT
Completed Phase 4 Interventional Results available

The Efficacy and Population Pharmacokinetics of Tranexamic Acid for Craniosynostosis Surgery

NCT02188576
View directory record Official record
Conditions
Craniosynostosis
Interventions
high dose TXA, Low dose TXA
Drug
Lead sponsor
Boston Children's Hospital
Other
Eligibility
3 Months to 6 Years
Enrollment
66 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2018
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Jan 31, 2020 · Synced Oct 14, 2026, 8:21 PM EDT
Terminated No phase listed Observational

Diagnosis and Treatment of Patients With Cystic Fibrosis

NCT00001223
View directory record Official record
Conditions
Cystic Fibrosis, Liver Disease or Abnormalities, Diabetes, Pancreatic Insufficiency (Exocrine and Endocrine)
Interventions
Not listed
Lead sponsor
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
NIH
Eligibility
4 Years to 120 Years
Enrollment
79 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1988 – 2026
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Feb 4, 2026 · Synced Oct 14, 2026, 8:21 PM EDT