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ClinicalTrials.gov public records Last synced Nov 3, 2026, 5:23 PM EST

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Showing 1081–1104 of 10,353 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Congenital, Hereditary, and Neonatal Diseases and Abnormalities Clear all
Not listed Phase 2 Interventional

Stem Cell Transplant in Sickle Cell Disease and Thalassemia

NCT00408447
View directory record Official record
Conditions
Sickle Cell Disease, Beta Thalassemia
Interventions
Busulfan, Fludarabine, Alemtuzumab, Allogeneic stem cell transplant
Drug · Procedure
Lead sponsor
Columbia University
Other
Eligibility
1 Month to 30 Years
Enrollment
53 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2025
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jun 13, 2024 · Synced Nov 3, 2026, 5:23 PM EST
Recruiting No phase listed Observational Accepts healthy volunteers

Quantification & Classification of Inflammatory Cells in Uveitis Using OCT

NCT07065747
View directory record Official record
Conditions
Anterior Uveitis (AU), Birdshot Chorioretinitis, Behcet Disease, Herpes Simplex Virus, Juvenile Idiopathic Arthritis (JIA), Spondyloarthritis (SA), Sarcoidosis, Posterior Vitreous Detachment
Interventions
Not listed
Lead sponsor
Oregon Health and Science University
Other
Eligibility
5 Years to 85 Years
Enrollment
125 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2024 – 2026
U.S. locations
1
States / cities
Portland, Oregon
Source: ClinicalTrials.gov public record
Updated Jul 17, 2025 · Synced Nov 3, 2026, 5:23 PM EST
Completed No phase listed Observational

Redesigning the Care Journey of Patients With Single-ventricle Congenital Heart Disease

NCT04613934
View directory record Official record
Conditions
Single-ventricle, Congenital Heart Disease
Interventions
Not listed
Lead sponsor
University of Texas at Austin
Other
Eligibility
6 Years and older
Enrollment
142 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020
U.S. locations
1
States / cities
Austin, Texas
Source: ClinicalTrials.gov public record
Updated Apr 28, 2021 · Synced Nov 3, 2026, 5:23 PM EST
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 Nov 3, 2026, 5:23 PM EST
Terminated Phase 4 Interventional Results available

Safety and Efficacy of Pimecrolimus Cream 1% in Atopic Disease Modification

NCT00124709
View directory record Official record
Conditions
Atopic Dermatitis
Interventions
Pimecrolimus, Corticosteroid
Drug
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
3 Months to 18 Months
Enrollment
1,091 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003 – 2008
U.S. locations
36
States / cities
Birmingham, Alabama • Fayetteville, Arkansas • Jonesboro, Arkansas + 33 more
Source: ClinicalTrials.gov public record
Updated Mar 23, 2011 · Synced Nov 3, 2026, 5:23 PM EST
Withdrawn Phase 1Phase 2 Interventional

Phase I/II Pilot Study of Mixed Chimerism to Treat Hemoglobinopathies

NCT01419704
View directory record Official record
Conditions
Anemia, Sickle Cell, Complex and Transfusion-dependent Hemoglobinopathies, Thalassemia, Diamond-Blackfan Anemia, Bone Marrow Failure Syndromes, Alpha-Thalassemia, Beta-Thalassemia
Interventions
Enriched Hematopoetic Stem Cell Infusion
Biological
Lead sponsor
Talaris Therapeutics Inc.
Industry
Eligibility
Up to 45 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2016
U.S. locations
3
States / cities
Chicago, Illinois • Louisville, Kentucky • Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Mar 3, 2023 · Synced Nov 3, 2026, 5:23 PM EST
Completed Phase 2 Interventional Results available

Extension Study of Protocol ENB-002-08 - Study of Asfotase Alfa in Infants and Young Children With Hypophosphatasia (HPP)

NCT01205152
View directory record Official record
Conditions
Hypophosphatasia
Interventions
asfotase alfa
Biological
Lead sponsor
Alexion Pharmaceuticals, Inc.
Industry
Eligibility
24 Weeks to 42 Months
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2016
U.S. locations
5
States / cities
Wilmington, Delaware • Springfield, Missouri • Omaha, Nebraska + 2 more
Source: ClinicalTrials.gov public record
Updated Mar 13, 2019 · Synced Nov 3, 2026, 5:23 PM EST
Completed Phase 1Phase 2 Interventional Results available

Clofarabine, Melphalan, and Thiotepa Followed By a Donor Stem Cell Transplant in Treating Patients With High-Risk and/or Advanced Hematologic Cancer or Other Disease

NCT00423514
View directory record Official record
Conditions
Graft Versus Host Disease, Leukemia, Myelodysplastic Syndromes
Interventions
filgrastim, clofarabine, melphalan, mycophenolate mofetil, tacrolimus, thiotepa, allogeneic bone marrow transplantation, allogeneic hematopoietic stem cell transplantation, peripheral blood stem cell transplantation
Biological · Drug · Procedure
Lead sponsor
Memorial Sloan Kettering Cancer Center
Other
Eligibility
0 Years to 54 Years
Enrollment
38 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2021
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Dec 27, 2022 · Synced Nov 3, 2026, 5:23 PM EST
Recruiting No phase listed Observational

Universal Rare Gene Study: A Registry and Natural History Study of Retinal Dystrophies Associated With Rare Disease-Causing Genetic Variants

NCT05589714
View directory record Official record
Conditions
Inherited Retinal Degeneration, Retinitis Pigmentosa
Interventions
Not listed
Lead sponsor
Jaeb Center for Health Research
Other
Eligibility
4 Years and older
Enrollment
1,500 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2030
U.S. locations
19
States / cities
Little Rock, Arkansas • Los Angeles, California • San Francisco, California + 16 more
Source: ClinicalTrials.gov public record
Updated Oct 29, 2026 · Synced Nov 3, 2026, 5:23 PM EST
Completed Phase 2 Interventional Results available

A Study to Evaluate Multiple Doses of GLPG2222 in Adult Subjects With Cystic Fibrosis

NCT03119649
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
GLPG2222 50 mg, GLPG2222 100 mg, Placebo, GLPG2222 200 mg, GLPG2222 400 mg
Drug
Lead sponsor
Lakefront Biotherapeutics NV
Industry
Eligibility
18 Years to 99 Years
Enrollment
59 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017
U.S. locations
7
States / cities
Chatom, Alabama • Little Rock, Arkansas • Orlando, Florida + 4 more
Source: ClinicalTrials.gov public record
Updated Nov 16, 2018 · Synced Nov 3, 2026, 5:23 PM EST
Terminated Phase 2Phase 3 Interventional Results available

A Study to Test Whether Spesolimab Helps People With a Skin Disease Called Netherton Syndrome

NCT05856526
View directory record Official record
Conditions
Netherton Syndrome
Interventions
Spesolimab - solution for infusion, Placebo matching to spesolimab - solution for infusion, Spesolimab - solution for injection, Placebo matching to spesolimab - solution for injection
Drug
Lead sponsor
Boehringer Ingelheim
Industry
Eligibility
12 Years and older
Enrollment
43 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2025
U.S. locations
4
States / cities
Rancho Santa Margarita, California • New Haven, Connecticut • Charleston, South Carolina + 1 more
Source: ClinicalTrials.gov public record
Updated Mar 12, 2026 · Synced Nov 3, 2026, 5:23 PM EST
Completed No phase listed Observational Results available

Study of Long-Term Safety and Efficacy on Gene Therapy in Glycogen Storage Disease Type Ia

NCT03970278
View directory record Official record
Conditions
Glycogen Storage Disease Type IA, Von Gierke's Disease (GSD Type Ia)
Interventions
No intervention
Other
Lead sponsor
Ultragenyx Pharmaceutical Inc
Industry
Eligibility
18 Years and older
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2025
U.S. locations
3
States / cities
Farmington, Connecticut • Ann Arbor, Michigan • Houston, Texas
Source: ClinicalTrials.gov public record
Updated Mar 18, 2026 · Synced Nov 3, 2026, 5:23 PM EST
Active, not recruiting Not applicable Interventional Accepts healthy volunteers Results available

Pulmonary Function Using Non-invasive Forced Oscillometry

NCT03346343
View directory record Official record
Conditions
Infant, Premature, Diseases, Infant, Newborn, Diseases, Bronchopulmonary Dysplasia, Respiratory Distress Syndrome, Newborn, Meconium Aspiration Syndrome, Transient Tachypnea of the Newborn
Interventions
Non-invasive forced airway oscillometry
Device
Lead sponsor
University of Alabama at Birmingham
Other
Eligibility
1 Day to 2 Years
Enrollment
600 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2017 – 2027
U.S. locations
1
States / cities
Birmingham, Alabama
Source: ClinicalTrials.gov public record
Updated Jan 27, 2026 · Synced Nov 3, 2026, 5:23 PM EST
Completed No phase listed Observational

Clinical and Molecular Correlations in Spinocerebellar Ataxia Type 10 (SCA10)

NCT00004306
View directory record Official record
Conditions
Hereditary Ataxia
Interventions
Not listed
Lead sponsor
Office of Rare Diseases (ORD)
NIH
Eligibility
3 Years and older
Enrollment
18 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1999 – 2009
U.S. locations
1
States / cities
Galveston, Texas
Source: ClinicalTrials.gov public record
Updated Mar 6, 2012 · Synced Nov 3, 2026, 5:23 PM EST
Not listed No phase listed Observational Accepts healthy volunteers

Non-Invasive Determination of Fetal Chromosome Abnormalities

NCT00891852
View directory record Official record
Conditions
Down Syndrome (Trisomy 21), Edward's Syndrome (Trisomy 18), Patau Syndrome (Trisomy 13), Klinefelter Syndrome (47, XXY), and Other Chromosome, Abnormalities.
Interventions
Not listed
Lead sponsor
Lenetix Medical Screening Laboratory
Industry
Eligibility
18 Years and older · Female only
Enrollment
1,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2009
U.S. locations
7
States / cities
Rockville, Maryland • Moristown, New Jersey • Mineola, New York + 3 more
Source: ClinicalTrials.gov public record
Updated May 1, 2009 · Synced Nov 3, 2026, 5:23 PM EST
Recruiting Not applicable Interventional

Pharmacist-Led Treat-to-Target Urate Lowering Therapy in Patients With Gout

NCT06887452
View directory record Official record
Conditions
Gout, Gouty Arthritis
Interventions
Pharmacist-Led
Other
Lead sponsor
VA Office of Research and Development
Federal
Eligibility
18 Years and older
Enrollment
310 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2031
U.S. locations
5
States / cities
Omaha, Nebraska • Portland, Oregon • Philadelphia, Pennsylvania + 2 more
Source: ClinicalTrials.gov public record
Updated Oct 21, 2026 · Synced Nov 3, 2026, 5:23 PM EST
Active, not recruiting Phase 2Phase 3 Interventional

A Phase 2/3 Trial to Assess the Efficacy and Safety of OCU410ST for Stargardt Disease

NCT05956626
View directory record Official record
Conditions
Stargardt Disease
Interventions
OCU410ST
Drug
Lead sponsor
Ocugen
Industry
Eligibility
5 Years and older
Enrollment
51 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2026
U.S. locations
14
States / cities
Phoenix, Arizona • Sacramento, California • Gainesville, Florida + 11 more
Source: ClinicalTrials.gov public record
Updated Jul 31, 2026 · Synced Nov 3, 2026, 5:23 PM EST
Completed Phase 3 Interventional Results available

A Study of UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Therapy in Subjects With Mucopolysaccharidosis Type 7, Sly Syndrome (MPS 7)

NCT02432144
View directory record Official record
Conditions
Sly Syndrome, MPS VII, Mucopolysaccharidosis, Mucopolysaccharidosis VII
Interventions
UX003
Drug
Lead sponsor
Ultragenyx Pharmaceutical Inc
Industry
Eligibility
5 Years and older
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2019
U.S. locations
4
States / cities
Oakland, California • Orange, California • Minneapolis, Minnesota + 1 more
Source: ClinicalTrials.gov public record
Updated Jul 30, 2020 · Synced Nov 3, 2026, 5:23 PM EST
Not yet recruiting Not applicable Interventional

Evaluation of the Safety and Effectiveness of the CORUS™ Posterior Cervical Stabilization System 3D (PCSS 3D) for the Treatment of 2-level Cervical Degenerative Disease

NCT07721493
View directory record Official record
Conditions
Degenerative Disc Disease(DDD)
Interventions
Circumferential Cervical Fusion (CCF), Anterior Cervical Discectomy and Fusion
Device · Procedure
Lead sponsor
Providence Medical Technology, Inc.
Industry
Eligibility
18 Years and older
Enrollment
225 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2029
U.S. locations
1
States / cities
Paducah, Kentucky
Source: ClinicalTrials.gov public record
Updated Jul 23, 2026 · Synced Nov 3, 2026, 5:23 PM EST
Recruiting Phase 2 Interventional

An Investigational Drug (TPST-1495) in Patients With Familial Adenomatous Polyposis

NCT06557733
View directory record Official record
Conditions
Colorectal Carcinoma, Familial Adenomatous Polyposis
Interventions
Biopsy Procedure, Biospecimen Collection, EP2/EP4 Antagonist TPST-1495, Esophagogastroduodenoscopy, Gastrointestinal Endoscopy, Questionnaire Administration
Procedure · Drug · Other
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
18 Years and older
Enrollment
38 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2027
U.S. locations
4
States / cities
Phoenix, Arizona • Rochester, Minnesota • Salt Lake City, Utah + 1 more
Source: ClinicalTrials.gov public record
Updated Sep 14, 2026 · Synced Nov 3, 2026, 5:23 PM EST
Recruiting Phase 1 Interventional

A Phase 1 AAV Gene Therapy Trial Evaluating Safety and Preliminary Efficacy of RP-A701 in Subjects With BAG3 Dilated Cardiomyopathy

NCT07137338
View directory record Official record
Conditions
Dilated Cardiomyopathy (DCM)
Interventions
RP-A701 is a recombinant viral vector composed of an AAV serotype rh.74 (AAVrh.74) capsid encapsulating the transgene, BCL2-associated Athanogene 3 (BAG3)
Genetic
Lead sponsor
Rocket Pharmaceuticals Inc.
Industry
Eligibility
18 Years to 65 Years
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2029
U.S. locations
3
States / cities
San Diego, California • Rochester, Minnesota • Charleston, South Carolina
Source: ClinicalTrials.gov public record
Updated Oct 1, 2026 · Synced Nov 3, 2026, 5:23 PM EST
Completed No phase listed Observational

Utility of CD64 and TLR2 Assays to Diagnose Acute Pulmonary Exacerbations in Cystic Fibrosis

NCT04397809
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
Not listed
Lead sponsor
National Jewish Health
Other
Eligibility
18 Years and older
Enrollment
150 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2021
U.S. locations
1
States / cities
Denver, Colorado
Source: ClinicalTrials.gov public record
Updated Sep 2, 2021 · Synced Nov 3, 2026, 5:23 PM EST
Completed Not applicable Interventional Results available

Impact of Cyanoacrylate Glue on PICC Line Dressing Care

NCT05246709
View directory record Official record
Conditions
Central Line Complication, Neonatal Disease
Interventions
Cyanoacrylate glue, Standard transparent film dressing
Device
Lead sponsor
Columbia University
Other
Eligibility
1 Day to 6 Months
Enrollment
32 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jun 26, 2025 · Synced Nov 3, 2026, 5:23 PM EST
Completed No phase listed Observational

Estimation of the Carrier Frequency and Incidence of Smith-Lemli-Opitz Syndrome in African Americans

NCT00017732
View directory record Official record
Conditions
Smith-Lemli-Opitz Syndrome
Interventions
Not listed
Lead sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
NIH
Eligibility
Not listed
Enrollment
2,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2003
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 4, 2008 · Synced Nov 3, 2026, 5:23 PM EST