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ClinicalTrials.gov public records Last synced Oct 17, 2026, 11:43 AM EDT

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Showing 1009–1032 of 10,533 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Congenital Diseases Clear all
Completed Phase 1 Interventional

Decitabine and Valproic Acid in Treating Patients With Refractory or Relapsed Acute Myeloid Leukemia or Previously Treated Chronic Lymphocytic Leukemia or Small Lymphocytic Lymphoma

NCT00079378
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 Myeloid Leukemia, Recurrent Small Lymphocytic Lymphoma, Refractory Chronic Lymphocytic Leukemia, Untreated Adult Acute Myeloid Leukemia
Interventions
decitabine, valproic acid, pharmacological study, laboratory biomarker analysis
Drug · Other
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
18 Years and older
Enrollment
84 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2004
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Sep 30, 2013 · Synced Oct 17, 2026, 11:43 AM EDT
Recruiting Phase 1Phase 2 Interventional

Cellular Therapy for In Utero Repair of Myelomeningocele - The CuRe Trial

NCT04652908
View directory record Official record
Conditions
Myelomeningocele
Interventions
Placental Mesenchymal Stem Cells seeded on a commercially available dural graft extracellular matrix, Untreated contemporaneous cohort
Biological · Other
Lead sponsor
University of California, Davis
Other
Eligibility
19 Weeks to 25 Weeks
Enrollment
55 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2027
U.S. locations
1
States / cities
Sacramento, California
Source: ClinicalTrials.gov public record
Updated Jan 20, 2026 · Synced Oct 17, 2026, 11:43 AM EDT
Recruiting No phase listed Observational

Study of Inherited Neurological Disorders

NCT00004568
View directory record Official record
Conditions
Motor Neuron Disease, Muscular Disease, Muscular Dystrophy, Peripheral Nervous System Disease
Interventions
Not listed
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
2 Years to 120 Years
Enrollment
3,500 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2000
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 16, 2026 · Synced Oct 17, 2026, 11:43 AM EDT
Recruiting Not applicable Interventional Accepts healthy volunteers

Mobile Health Self-Management and Support System for Chronic and Complex Health Conditions

NCT02592291
View directory record Official record
Conditions
Spinal Cord Injuries, Cerebral Palsy, Spinal Dysraphism, Brain Injuries, Spina Bifida, Traumatic Brain Injury
Interventions
MHealth
Device
Lead sponsor
University of Pittsburgh
Other
Eligibility
12 Years and older
Enrollment
160 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2019 – 2026
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Sep 3, 2026 · Synced Oct 17, 2026, 11:43 AM EDT
Completed Phase 2 Interventional Results available

Safety and Tolerability Trial of Inhaled Alpha1-Proteinase Inhibitor (Human), Hydrophobic Chromatography Process (Alpha-1 HC) in Subjects With Cystic Fibrosis

NCT01684410
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
Alpha-1 HC 100 mg, Placebo, Alpha-1 HC 200 mg
Biological
Lead sponsor
Grifols Therapeutics LLC
Industry
Eligibility
18 Years and older
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2013
U.S. locations
6
States / cities
Birmingham, Alabama • Denver, Colorado • Boston, Massachusetts + 3 more
Source: ClinicalTrials.gov public record
Updated Feb 8, 2016 · Synced Oct 17, 2026, 11:43 AM EDT
Completed Not applicable Interventional Results available

Ellipsys Vascular Access System Post Market Surveillance (PS) Study

NCT04484220
View directory record Official record
Conditions
Renal Disease, End Stage, Kidney Disease, End-Stage, AV Fistula, Fistulas Arteriovenous
Interventions
Ellipsys Vascular Access System
Device
Lead sponsor
Medtronic Endovascular
Industry
Eligibility
18 Years to 80 Years
Enrollment
142 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2025
U.S. locations
12
States / cities
Riverside, California • New Haven, Connecticut • Jacksonville, Florida + 8 more
Source: ClinicalTrials.gov public record
Updated Jun 1, 2026 · Synced Oct 17, 2026, 11:43 AM EDT
Completed Not applicable Interventional Accepts healthy volunteers

Increased Gluconeogenesis is One Cause of Cystic Fibrosis Related Diabetes (CFRD)

NCT00082238
View directory record Official record
Conditions
Cystic Fibrosis, Diabetes Mellitus
Interventions
Stable isotopes
Other
Lead sponsor
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
NIH
Eligibility
18 Years to 45 Years
Enrollment
42 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2003 – 2005
U.S. locations
1
States / cities
Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Mar 14, 2018 · Synced Oct 17, 2026, 11:43 AM EDT
Enrolling by invitation No phase listed Observational

Long-term Follow-up of Subjects with Sickle Cell Disease Treated with Ex Vivo Gene Therapy

NCT04628585
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Safety and efficacy assessments
Other
Lead sponsor
Genetix Biotherapeutics Inc.
Industry
Eligibility
2 Years to 53 Years
Enrollment
85 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2038
U.S. locations
15
States / cities
Birmingham, Alabama • Oakland, California • Atlanta, Georgia + 12 more
Source: ClinicalTrials.gov public record
Updated Mar 20, 2025 · Synced Oct 17, 2026, 11:43 AM EDT
Completed Phase 4 Interventional Results available

A Study Evaluating TEPEZZA® Treatment in Patients With Chronic (Inactive) Thyroid Eye Disease

NCT04583735
View directory record Official record
Conditions
Thyroid Eye Disease, Chronic (Inactive) Thyroid Eye Disease
Interventions
TEPEZZA, Placebo
Biological · Drug
Lead sponsor
Amgen
Industry
Eligibility
18 Years and older
Enrollment
62 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2023
U.S. locations
12
States / cities
Beverly Hills, California • La Jolla, California • Los Angeles, California + 9 more
Source: ClinicalTrials.gov public record
Updated Jul 1, 2024 · Synced Oct 17, 2026, 11:43 AM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

A Phase 1, Non-Randomized, Open-Label, Single-Dose Study To Evaluate The Pharmacokinetics, Safety, And Tolerability Of Dimebon [PF 01913539] In Subjects With Severely-Impaired And Normal Renal Function

NCT00824590
View directory record Official record
Conditions
Alzheimer's Disease, Huntington's Disease
Interventions
Dimebon
Drug
Lead sponsor
Pfizer
Industry
Eligibility
18 Years to 75 Years
Enrollment
20 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2009
U.S. locations
2
States / cities
Miami, Florida • Saint Paul, Minnesota
Source: ClinicalTrials.gov public record
Updated Dec 30, 2009 · Synced Oct 17, 2026, 11:43 AM EDT
Recruiting Phase 2 Interventional

A Study of ROC-101 in Patients With Pulmonary Arterial Hypertension (PAH) and Pulmonary Hypertension Associated With Interstitial Lung Disease (ILD-PH) (ROCSTAR STUDY)

NCT07175038
View directory record Official record
Conditions
Pulmonary Arterial Hypertension (PAH), Pulmonary Hypertension, Interstitial Lung Disease
Interventions
ROC-101
Drug
Lead sponsor
AllRock Bio, Inc.
Industry
Eligibility
18 Years to 80 Years
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2029
U.S. locations
11
States / cities
Phoenix, Arizona • Rancho Cordova, California • Aurora, Colorado + 8 more
Source: ClinicalTrials.gov public record
Updated Oct 8, 2026 · Synced Oct 17, 2026, 11:43 AM EDT
Completed Not applicable Interventional

A Standardized Nursing Intervention Protocol for HCT Patients

NCT00951626
View directory record Official record
Conditions
Chronic Myeloproliferative Disorders, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Neoplasms, Psychosocial Effects of Cancer and Its Treatment, Therapy-related Toxicity
Interventions
exercise intervention, telephone-based intervention, dietary intervention, educational intervention, questionnaire administration, allogeneic bone marrow transplantation, allogeneic hematopoietic stem cell transplantation, assessment of therapy complications, peripheral blood stem cell transplantation, psychosocial assessment and care, quality-of-life assessment, standard follow-up care
Behavioral · Dietary Supplement · Other + 1 more
Lead sponsor
City of Hope Medical Center
Other
Eligibility
18 Years and older
Enrollment
282 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2011
U.S. locations
1
States / cities
Duarte, California
Source: ClinicalTrials.gov public record
Updated Jun 8, 2015 · Synced Oct 17, 2026, 11:43 AM EDT
Recruiting No phase listed Observational

Specimen Collection From Pregnant Women at Increased Risk for Fetal Aneuploidy

NCT01429389
View directory record Official record
Conditions
Down Syndrome, Fetal Aneuploidy
Interventions
Not listed
Lead sponsor
Sequenom, Inc.
Industry
Eligibility
18 Years and older · Female only
Enrollment
2,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2028
U.S. locations
12
States / cities
Birmingham, Alabama • Los Angeles, California • San Diego, California + 6 more
Source: ClinicalTrials.gov public record
Updated Sep 9, 2026 · Synced Oct 17, 2026, 11:43 AM EDT
Not listed Phase 1 Interventional

Emergency Use of Donor Lymphocytes in Treating Patients Who Have Undergone Donor Stem Cell Transplant and Have Cytomegalovirus Infections

NCT00769613
View directory record Official record
Conditions
Cancer
Interventions
cytomegalovirus IE-1-specific cytotoxic T lymphocytes, cytomegalovirus pp65-specific cytotoxic T lymphocytes, therapeutic allogeneic lymphocytes, polymerase chain reaction, flow cytometry, immunological diagnostic method, laboratory biomarker analysis
Biological · Genetic · Other
Lead sponsor
Milton S. Hershey Medical Center
Other
Eligibility
2 Years and older
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2008
U.S. locations
1
States / cities
Hershey, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Dec 18, 2013 · Synced Oct 17, 2026, 11:43 AM EDT
Completed Phase 3 Interventional Results available

A Study of VX-445 Combination Therapy in CF Subjects Homozygous for F508del (F/F)

NCT03525548
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
VX-445/TEZ/IVA, TEZ/IVA, IVA, Placebo
Drug
Lead sponsor
Vertex Pharmaceuticals Incorporated
Industry
Eligibility
12 Years and older
Enrollment
113 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018
U.S. locations
28
States / cities
Tucson, Arizona • Little Rock, Arkansas • Long Beach, California + 24 more
Source: ClinicalTrials.gov public record
Updated Jan 27, 2020 · Synced Oct 17, 2026, 11:43 AM EDT
Completed Not applicable Interventional Accepts healthy volunteers

Sweat Evaporimeter Measurement

NCT01708655
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
Sweat Evaporimeter measurement
Other
Lead sponsor
University of Colorado, Denver
Other
Eligibility
18 Years to 65 Years
Enrollment
16 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2011 – 2014
U.S. locations
1
States / cities
Aurora, Colorado
Source: ClinicalTrials.gov public record
Updated Dec 3, 2014 · Synced Oct 17, 2026, 11:43 AM EDT
Completed Not applicable Interventional Accepts healthy volunteers

High Intensity Functional Training for Individuals With Neurologic Diagnoses and Their Care Partners

NCT05951985
View directory record Official record
Conditions
Neurologic Disorder, Parkinson Disease, Spinal Cord Injuries, Spina Bifida, Stroke, Poliomyelitis, Multiple Sclerosis, Brain Injuries
Interventions
HIFT
Behavioral
Lead sponsor
Arkansas Colleges of Health Education
Other
Eligibility
18 Years and older
Enrollment
100 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2023 – 2025
U.S. locations
1
States / cities
Fort Smith, Arkansas
Source: ClinicalTrials.gov public record
Updated Aug 22, 2025 · Synced Oct 17, 2026, 11:43 AM EDT
Completed Not applicable Interventional

Study to Evaluate the Use of Ultrasound in the Diagnosis of Hemophilic Joint Disease

NCT00212485
View directory record Official record
Conditions
Inherited Bleeding Disorders
Interventions
Power Doppler Sonography, MRI
Procedure
Lead sponsor
New York Presbyterian Hospital
Other
Eligibility
1 Year to 60 Years
Enrollment
43 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2009
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jul 1, 2010 · Synced Oct 17, 2026, 11:43 AM EDT
Completed Phase 2 Interventional Results available

Efficacy and Safety of Pemigatinib in Subjects With Advanced/Metastatic or Surgically Unresectable Cholangiocarcinoma Who Failed Previous Therapy - (FIGHT-202)

NCT02924376
View directory record Official record
Conditions
Cholangiocarcinoma
Interventions
Pemigatinib
Drug
Lead sponsor
Incyte Corporation
Industry
Eligibility
18 Years and older
Enrollment
147 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2022
U.S. locations
49
States / cities
Anchorage, Alaska • Phoenix, Arizona • Tempe, Arizona + 46 more
Source: ClinicalTrials.gov public record
Updated Aug 14, 2025 · Synced Oct 17, 2026, 11:43 AM EDT
Terminated Not applicable Interventional

Efficacy of a Decision Aid for Hydroxyurea in Sickle Cell Disease

NCT03577691
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Training for use of web based Decision Aid, No training for use of web based Decision Aid, Pretest surveys and posttest surveys, Only posttest surveys
Other
Lead sponsor
Emory University
Other
Eligibility
18 Years to 80 Years
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2019
U.S. locations
3
States / cities
Atlanta, Georgia
Source: ClinicalTrials.gov public record
Updated Jul 23, 2019 · Synced Oct 17, 2026, 11:43 AM EDT
Completed No phase listed Observational

Psychological Burden of Thyroid Eye Disease

NCT07444190
View directory record Official record
Conditions
Thyroid Eye Disease, TED
Interventions
focus groups and individual interviews as well as filling out questionnaires
Other
Lead sponsor
Thomas Jefferson University
Other
Eligibility
18 Years and older
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Mar 2, 2026 · Synced Oct 17, 2026, 11:43 AM EDT
Completed Not applicable Interventional Accepts healthy volunteers

Nitric Oxide Flux and Ureagenesis in Argininosuccinate Synthetase Deficiency (ASSD)(Citrullinemia I)

NCT01610089
View directory record Official record
Conditions
Citrullinemia
Interventions
stable isotope infusion
Other
Lead sponsor
Baylor College of Medicine
Other
Eligibility
18 Years and older
Enrollment
6 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2012 – 2013
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Mar 20, 2015 · Synced Oct 17, 2026, 11:43 AM EDT
Recruiting No phase listed Observational

Verifying Antibodies After Live Immunization Delivery (VALID): A Study of Measles Vaccine Immunogenicity in Children With Sickle Cell Disease

NCT07356050
View directory record Official record
Conditions
Sickle Cell Disease, Measles Vaccination, Sickle Cell Anemia
Interventions
Not listed
Lead sponsor
Children's Hospital Medical Center, Cincinnati
Other
Eligibility
6 Months to 6 Years
Enrollment
130 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2028
U.S. locations
1
States / cities
Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Jan 21, 2026 · Synced Oct 17, 2026, 11:43 AM EDT
Terminated No phase listed Observational

Whole Blood Biospecimen Collection for Subjects With Chronic Granulomatous Disease (CGD)

NCT05915897
View directory record Official record
Conditions
Chronic Granulomatous Disease
Interventions
Specimen Donation
Diagnostic Test
Lead sponsor
Sanguine Biosciences
Industry
Eligibility
18 Years to 85 Years
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2024
U.S. locations
1
States / cities
Woburn, Massachusetts
Source: ClinicalTrials.gov public record
Updated Apr 18, 2024 · Synced Oct 17, 2026, 11:43 AM EDT