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ClinicalTrials.gov public records Last synced Oct 22, 2026, 5:16 AM EDT

Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.

Showing 289–312 of 10,540 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Congenital Disorders Clear all
Completed No phase listed Observational

Genetic Analysis of Left-Right Axis Formations

NCT00341133
View directory record Official record
Conditions
Congenital Heart Disease
Interventions
Not listed
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
Not listed
Enrollment
900 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1999
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 2, 2017 · Synced Oct 22, 2026, 5:16 AM EDT
Not listed No phase listed Observational

Evaluation of Abdominal Tissue Oxygenation in Premature Infants Using Near Infrared Spectroscopy

NCT01855347
View directory record Official record
Conditions
Infant, Premature, Diseases, Hypoxia, Necrotizing Enterocolitis
Interventions
Near Infrared Spectroscopy
Device
Lead sponsor
Brigham and Women's Hospital
Other
Eligibility
Up to 3 Weeks
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Aug 25, 2015 · Synced Oct 22, 2026, 5:16 AM EDT
Recruiting Phase 1Phase 2 Interventional

T-Cell Depleted Alternative Donor Bone Marrow Transplant for Sickle Cell Disease (SCD) and Other Anemias

NCT03653338
View directory record Official record
Conditions
Sickle Cell Anemia, Beta-thalassemia Major, Diamond-blackfan Anemia
Interventions
CD3/CD19 depleted leukocytes, CD45RA depleted leukocytes, Hydroxyurea, Rituximab, Alemtuzumab, Fludarabine, Thiotepa
Biological · Drug
Lead sponsor
Paul Szabolcs
Other
Eligibility
5 Years to 40 Years
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2028
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Aug 13, 2026 · Synced Oct 22, 2026, 5:16 AM EDT
Completed No phase listed Observational

Screening Protocol for Genetic Diseases of Allergic Inflammation

NCT00852943
View directory record Official record
Conditions
Eosinophilic Disease, Immune Deficiency, HaTS, Urticaria Anaphylaxis, Elevated IgE Level
Interventions
Not listed
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
1 Day to 99 Years
Enrollment
834 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2010
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 13, 2026 · Synced Oct 22, 2026, 5:16 AM EDT
Completed Phase 1Phase 2 Interventional Results available

Safety and Dose-Finding Study of DTX401 (AAV8G6PC) in Adults With Glycogen Storage Disease Type Ia (GSDIa)

NCT03517085
View directory record Official record
Conditions
GSD1
Interventions
DTX401, steroid regimen
Genetic · Drug
Lead sponsor
Ultragenyx Pharmaceutical Inc
Industry
Eligibility
18 Years and older
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2021
U.S. locations
3
States / cities
Farmington, Connecticut • Ann Arbor, Michigan • Houston, Texas
Source: ClinicalTrials.gov public record
Updated Nov 18, 2022 · Synced Oct 22, 2026, 5:16 AM EDT
Completed Phase 2 Interventional

Study of SANGUINATE™ In the Treatment of Sickle Cell Disease Patients With Vaso-Occlusive Crisis

NCT02411708
View directory record Official record
Conditions
Anemia, Sickle Cell
Interventions
SANGUINATE, Placebo
Drug
Lead sponsor
Prolong Pharmaceuticals
Industry
Eligibility
18 Years and older
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2017
U.S. locations
9
States / cities
Hollywood, Florida • Tampa, Florida • Baltimore, Maryland + 5 more
Source: ClinicalTrials.gov public record
Updated May 23, 2018 · Synced Oct 22, 2026, 5:16 AM EDT
Completed Not applicable Interventional

Intervention Effectiveness on the Neurocognitive Functioning of Children and Adolescents With Neurofibromatosis Type 1

NCT05377008
View directory record Official record
Conditions
Neurocognitive Deficit, Mental Health Impairment, Neurofibromatosis 1
Interventions
OPTIMAL-SSI
Behavioral
Lead sponsor
Virginia Commonwealth University
Other
Eligibility
6 Years to 16 Years
Enrollment
38 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2023
U.S. locations
1
States / cities
Richmond, Virginia
Source: ClinicalTrials.gov public record
Updated Sep 22, 2023 · Synced Oct 22, 2026, 5:16 AM EDT
Recruiting Not applicable Interventional

Impact of Hospital to Home: Optimizing Preterm Infant Environment for Surgical Neonates and Their Parents (H-HOPE)

NCT07372898
View directory record Official record
Conditions
Oral Feeding Outcomes, Neurodevelopment Outcome
Interventions
H-HOPE
Behavioral
Lead sponsor
Ann & Robert H Lurie Children's Hospital of Chicago
Other
Eligibility
1 Week to 8 Weeks
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
1
States / cities
Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Jan 28, 2026 · Synced Oct 22, 2026, 5:16 AM EDT
Completed Phase 2Phase 3 Interventional Results available

Efficacy and Safety of Inotersen in Familial Amyloid Polyneuropathy

NCT01737398
View directory record Official record
Conditions
FAP, Familial Amyloid Polyneuropathy, TTR, Transthyretin, Amyloidosis
Interventions
Inotersen, Placebo
Drug
Lead sponsor
Ionis Pharmaceuticals, Inc.
Industry
Eligibility
18 Years to 82 Years
Enrollment
173 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2017
U.S. locations
9
States / cities
Orange, California • Indianapolis, Indiana • Baltimore, Maryland + 5 more
Source: ClinicalTrials.gov public record
Updated Jul 17, 2019 · Synced Oct 22, 2026, 5:16 AM EDT
Completed No phase listed Observational

FGF23 and Angiotensin-(1-7) in Hypophosphatemia (GAP)

NCT03489993
View directory record Official record
Conditions
Hypophosphatemia, X-linked Hypophosphatemia, Renin-angiotensin System, Left Ventricular Hypertrophy
Interventions
Ang II and Ang-(1-7), FGF23 and klotho
Diagnostic Test
Lead sponsor
Wake Forest University Health Sciences
Other
Eligibility
2 Years to 24 Years
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2020
U.S. locations
1
States / cities
Winston-Salem, North Carolina
Source: ClinicalTrials.gov public record
Updated Aug 5, 2022 · Synced Oct 22, 2026, 5:16 AM EDT
Completed Not applicable Interventional Results available

Does a Daily Iron Tablet Improve Anemia in Cystic Fibrosis

NCT01755455
View directory record Official record
Conditions
Cystic Fibrosis, Anemia, Iron-Deficiency
Interventions
Ferrous sulfate 325mg, Placebo
Drug
Lead sponsor
Dartmouth-Hitchcock Medical Center
Other
Eligibility
18 Years to 70 Years
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2012
U.S. locations
2
States / cities
Portland, Maine • Lebanon, New Hampshire
Source: ClinicalTrials.gov public record
Updated Mar 16, 2017 · Synced Oct 22, 2026, 5:16 AM EDT
Active, not recruiting Phase 1 Interventional

Intramyocardial Injection of Autologous UCB-MNC During Fontan Surgery for SRV Dependent CHD

NCT04907526
View directory record Official record
Conditions
Congenital Heart Disease, SRV Dependent
Interventions
Autologous mononuclear cells
Biological
Lead sponsor
Timothy J Nelson, MD, PhD
Other
Eligibility
2 Years to 5 Years
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2025
U.S. locations
6
States / cities
Birmingham, Alabama • Los Angeles, California • Aurora, Colorado + 3 more
Source: ClinicalTrials.gov public record
Updated Jun 27, 2025 · Synced Oct 22, 2026, 5:16 AM EDT
Not listed Not applicable Interventional Accepts healthy volunteers

Comparing the Inflammation, Maldigestion and Symptoms Due to Commercial Milk and A2 Milk

NCT05660278
View directory record Official record
Conditions
Lactose Intolerance
Interventions
Milk containing A1 and A2 beta-casein, Milk containing only A2 beta-casein
Other
Lead sponsor
Purdue University
Other
Eligibility
18 Years to 65 Years
Enrollment
35 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2022 – 2025
U.S. locations
1
States / cities
West Lafayette, Indiana
Source: ClinicalTrials.gov public record
Updated Dec 21, 2022 · Synced Oct 22, 2026, 5:16 AM EDT
Terminated No phase listed Observational

Hemophilia Non-Interventional Study Prior to SerpinPC Intervention (PRESent-5)

NCT05605678
View directory record Official record
Conditions
Hemophilia a, Hemophilia B
Interventions
No Intervention
Other
Lead sponsor
ApcinteX Ltd
Industry
Eligibility
12 Years to 65 Years · Male only
Enrollment
108 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2024
U.S. locations
2
States / cities
Ann Arbor, Michigan • Greenville, South Carolina
Source: ClinicalTrials.gov public record
Updated Feb 7, 2025 · Synced Oct 22, 2026, 5:16 AM EDT
Completed Phase 2Phase 3 Interventional

Phase 2b/3 Study to Assess ABP-671 a Novel URAT1 Inhibitor in Participants With Gout

NCT05818085
View directory record Official record
Conditions
Gout
Interventions
ABP-671, Allopurinol, Placebo
Drug · Other
Lead sponsor
Atom Therapeutics Co., Ltd
Industry
Eligibility
19 Years to 75 Years
Enrollment
300 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2025
U.S. locations
35
States / cities
Tempe, Arizona • Tuscon, Arizona • Anaheim, California + 27 more
Source: ClinicalTrials.gov public record
Updated Dec 22, 2025 · Synced Oct 22, 2026, 5:16 AM EDT
Completed Phase 3 Interventional Results available

A Randomized Controlled Trial of Cannabidiol (GWP42003-P, CBD) for Seizures in Tuberous Sclerosis Complex (GWPCARE6)

NCT02544763
View directory record Official record
Conditions
Tuberous Sclerosis Complex, Seizures
Interventions
GWP42003-P, Placebo
Drug
Lead sponsor
Jazz Pharmaceuticals
Industry
Eligibility
1 Year to 65 Years
Enrollment
224 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2019
U.S. locations
23
States / cities
Birmingham, Alabama • Little Rock, Arkansas • Los Angeles, California + 20 more
Source: ClinicalTrials.gov public record
Updated Sep 28, 2022 · Synced Oct 22, 2026, 5:16 AM EDT
Completed Phase 3 Interventional

Study to Assess Batoclimab in Participants With Active Thyroid Eye Disease

NCT05517421
View directory record Official record
Conditions
Thyroid Eye Disease
Interventions
Batoclimab, Placebo
Drug
Lead sponsor
Immunovant Sciences GmbH
Industry
Eligibility
18 Years and older
Enrollment
114 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2025
U.S. locations
29
States / cities
Beverly Hills, California • Carlsbad, California • Los Angeles, California + 24 more
Source: ClinicalTrials.gov public record
Updated Dec 3, 2025 · Synced Oct 22, 2026, 5:16 AM EDT
Completed No phase listed Observational

Clinical Utility of the Addition of a SNP-based NIPT Zygosity Determination in Twin Pregnancy Management.

NCT05312814
View directory record Official record
Conditions
Twin to Twin Transfusion Syndrome, Pregnancy Complications, Multiple Gestation; Maternal Care
Interventions
Not listed
Lead sponsor
Natera, Inc.
Industry
Eligibility
18 Years to 55 Years · Female only
Enrollment
137 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2023
U.S. locations
8
States / cities
San Diego, California • Aurora, Colorado • Annapolis, Maryland + 5 more
Source: ClinicalTrials.gov public record
Updated Sep 26, 2024 · Synced Oct 22, 2026, 5:16 AM EDT
Terminated Phase 1Phase 2 Interventional Accepts healthy volunteers Results available

Safety, Tolerability, Pharmacokinetics, and Preliminary Pharmacodynamics of QBW251 in Healthy Subjects and Cystic Fibrosis Patients

NCT02190604
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
Placebo, QBW251
Drug
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
18 Years to 65 Years
Enrollment
153 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2012 – 2015
U.S. locations
9
States / cities
Birmingham, Alabama • Denver, Colorado • Chicago, Illinois + 5 more
Source: ClinicalTrials.gov public record
Updated Dec 30, 2020 · Synced Oct 22, 2026, 5:16 AM EDT
Completed Phase 2 Interventional

Allopurinol Combination Study

NCT01001338
View directory record Official record
Conditions
Gout
Interventions
RDEA594, Placebo, Allopurinol
Drug
Lead sponsor
Ardea Biosciences, Inc.
Industry
Eligibility
18 Years to 80 Years
Enrollment
227 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2016
U.S. locations
25
States / cities
Phoenix, Arizona • La Jolla, California • Los Angeles, California + 22 more
Source: ClinicalTrials.gov public record
Updated Jan 24, 2017 · Synced Oct 22, 2026, 5:16 AM EDT
Recruiting Phase 2 Interventional

Rifampin in CYP24A1-related Hypercalcemia and Hypercalciuria

NCT03301038
View directory record Official record
Conditions
Idiopathic Infantile Hypercalcaemia - Severe Form, Genetic Disease, Hypercalcemia, Idiopathic, of Infancy, Hypercalciuric Hypercalcemia, Idiopathic Infantile Hypercalcemia - Mild Form, Hypercalciuria
Interventions
Rifampin
Drug
Lead sponsor
Children's Hospital of Philadelphia
Other
Eligibility
6 Months to 70 Years
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2030
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Oct 13, 2026 · Synced Oct 22, 2026, 5:16 AM EDT
Completed Early Phase 1 Interventional Accepts healthy volunteers

Hyaluronidase Via LADD Scleroderma-induced Microstomia

NCT05995626
View directory record Official record
Conditions
Microstomia, Scleroderma
Interventions
Hyaluronidase, CO2 laser
Drug · Device
Lead sponsor
University of Nebraska
Other
Eligibility
19 Years and older
Enrollment
3 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2024
U.S. locations
2
States / cities
Omaha, Nebraska
Source: ClinicalTrials.gov public record
Updated Apr 15, 2026 · Synced Oct 22, 2026, 5:16 AM EDT
Completed No phase listed Observational

Learning and Behavior Problems in Children With Chronic Granulomatous Disease and Related Disorders

NCT00005933
View directory record Official record
Conditions
Chediak Higashi Syndrome, Chronic Granulomatous Disease, Job's Syndrome, Leukocyte Disorder
Interventions
Not listed
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
Not listed
Enrollment
150 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2000 – 2005
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 4, 2008 · Synced Oct 22, 2026, 5:16 AM EDT
Completed Phase 1 Interventional

Evaluation of Hydroxyurea Plus L-arginine or Sildenafil to Treat Sickle Cell Anemia

NCT00056433
View directory record Official record
Conditions
Sickle Cell Anemia
Interventions
Hydroxyurea, L-Arginine, Sildenafil
Drug
Lead sponsor
National Institutes of Health Clinical Center (CC)
NIH
Eligibility
16 Years and older
Enrollment
39 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003 – 2006
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Nov 22, 2019 · Synced Oct 22, 2026, 5:16 AM EDT