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ClinicalTrials.gov public records Last synced Oct 25, 2026, 12:23 PM EDT

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

Showing 673–696 of 10,333 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Congenital, Hereditary, and Neonatal Diseases and Abnormalities Clear all
Completed Not applicable Interventional Results available

Oxygen Consumption-based Assessments of Hemodynamics in Neonates Following Congenital Heart Surgery (Oxy-CAHN Study)

NCT02184169
View directory record Official record
Conditions
Transposition of the Great Vessels, Hypoplastic Left Heart Syndrome, Oxygen Consumption Based Assessment of Hemodynamics
Interventions
General Electric Healthcare E-COVX Gas Monitoring Module for VO2 Measurments, Edwards Continuous SVO2 Catheter and Monitoring System - 4.5 fr
Device
Lead sponsor
Boston Children's Hospital
Other
Eligibility
1 Day to 6 Months
Enrollment
27 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2019
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Jun 4, 2020 · Synced Oct 25, 2026, 12:23 PM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

A First-in-Human Study Evaluating AGA2115 in Adult Healthy Volunteers

NCT06086613
View directory record Official record
Conditions
Osteogenesis Imperfecta
Interventions
AGA2115, Placebo
Drug
Lead sponsor
Angitia Incorporated Limited
Industry
Eligibility
18 Years to 65 Years
Enrollment
72 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2023 – 2024
U.S. locations
1
States / cities
Lake Forest, California
Source: ClinicalTrials.gov public record
Updated Jan 10, 2025 · Synced Oct 25, 2026, 12:23 PM EDT
Recruiting No phase listed Observational

Molecular Genetics of Heterotaxy and Related Congenital Heart Defects

NCT02432079
View directory record Official record
Conditions
Heterotaxy Syndrome, Congenital Heart Defects
Interventions
Not listed
Lead sponsor
Indiana University
Other
Eligibility
Not listed
Enrollment
2,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2030
U.S. locations
1
States / cities
Indianapolis, Indiana
Source: ClinicalTrials.gov public record
Updated Jun 22, 2026 · Synced Oct 25, 2026, 12:23 PM EDT
Completed Not applicable Interventional Results available

Computer-Assisted Rehabilitation Environment Training After Argus Retinal Prosthesis

NCT03444961
View directory record Official record
Conditions
Retinitis Pigmentosa
Interventions
CAREN system training
Device
Lead sponsor
The Cleveland Clinic
Other
Eligibility
25 Years to 100 Years
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018
U.S. locations
1
States / cities
Cleveland, Ohio
Source: ClinicalTrials.gov public record
Updated Jul 14, 2021 · Synced Oct 25, 2026, 12:23 PM EDT
Completed No phase listed Observational

Repository for Inherited Eye Diseases

NCT00378742
View directory record Official record
Conditions
Retinitis Pigmentosa, Inherited Ophthalmic Diseases
Interventions
Not listed
Lead sponsor
National Eye Institute (NEI)
NIH
Eligibility
1 Day to 100 Years
Enrollment
6,618 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2015
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Dec 8, 2025 · Synced Oct 25, 2026, 12:23 PM EDT
Active, not recruiting Not applicable Interventional

Transanastomotic Tube for Proximal Esophageal Atresia With Distal Tracheoesophageal Fistula Repair

NCT03730454
View directory record Official record
Conditions
Esophageal Atresia, Tracheoesophageal Fistula
Interventions
Transanastomotic Tube (5FR), No Transanastomotic Tube
Device · Other
Lead sponsor
Phoenix Children's Hospital
Other
Eligibility
Up to 6 Months
Enrollment
142 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2026
U.S. locations
10
States / cities
Phoenix, Arizona • Los Angeles, California • San Diego, California + 7 more
Source: ClinicalTrials.gov public record
Updated Mar 10, 2025 · Synced Oct 25, 2026, 12:23 PM EDT
Completed Phase 1Phase 2 Interventional

A Phase 1/2 Trial of PTR-01 in Adult Patients With Recessive Dystrophic Epidermolysis Bullosa (RDEB)

NCT03752905
View directory record Official record
Conditions
Recessive Dystrophic Epidermolysis Bullosa
Interventions
PTR-01, Normal saline
Drug
Lead sponsor
Phoenix Tissue Repair, Inc.
Industry
Eligibility
16 Years and older
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2020
U.S. locations
3
States / cities
Redwood City, California • Aurora, Colorado • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Mar 9, 2021 · Synced Oct 25, 2026, 12:23 PM EDT
Completed Phase 3 Interventional Results available

A Double-Blind Study to Evaluate the Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)

NCT01275066
View directory record Official record
Conditions
MPS IV A
Interventions
BMN 110 Weekly, Placebo, BMN 110 Every Other Week
Drug
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
5 Years and older
Enrollment
177 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2012
U.S. locations
6
States / cities
Oakland, California • Wilmington, Delaware • Washington D.C., District of Columbia + 3 more
Source: ClinicalTrials.gov public record
Updated Jul 7, 2014 · Synced Oct 25, 2026, 12:23 PM EDT
Completed Phase 1 Interventional Results available

A Phase I Study to Evaluate the Oral Absorption of Nepadutant in Infants

NCT00655083
View directory record Official record
Conditions
Infantile Colic, Infantile Functional Gastrointestinal Disorders
Interventions
Nepadutant
Drug
Lead sponsor
Menarini Group
Industry
Eligibility
6 Weeks to 24 Weeks
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2009
U.S. locations
3
States / cities
Little Rock, Arkansas • Louisville, Kentucky • Cleveland, Ohio
Source: ClinicalTrials.gov public record
Updated Oct 17, 2011 · Synced Oct 25, 2026, 12:23 PM EDT
Active, not recruiting No phase listed Observational

Artificial Intelligence Patient App for RDEB SCCs

NCT05843994
View directory record Official record
Conditions
Epidermolysis Bullosa Dystrophica
Interventions
online survey
Other
Lead sponsor
Northwestern University
Other
Eligibility
12 Years and older
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2028
U.S. locations
1
States / cities
Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Feb 3, 2026 · Synced Oct 25, 2026, 12:23 PM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Functional Evaluation in Patients With Urea Cycle Disorders (UCD) During a Driving Task

NCT07701694
View directory record Official record
Conditions
Urea Cycle Disorder
Interventions
Not listed
Lead sponsor
St. Jude Children's Research Hospital
Other
Eligibility
16 Years to 40 Years
Enrollment
60 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2026 – 2032
U.S. locations
1
States / cities
Memphis, Tennessee
Source: ClinicalTrials.gov public record
Updated Jul 31, 2026 · Synced Oct 25, 2026, 12:23 PM EDT
Completed Not applicable Interventional Results available

Vitamin B12 and Folate Administration on Homocysteine Concentrations After Nitrous Oxide Anesthesia

NCT00901394
View directory record Official record
Conditions
Cardiovascular Abnormalities
Interventions
B12-Folic Acid, nitrous oxide, Nitrous oxide (NO) and placebo, Placebo, oxygen nitrogen
Drug · Other
Lead sponsor
Washington University School of Medicine
Other
Eligibility
18 Years and older
Enrollment
63 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2010
U.S. locations
1
States / cities
St Louis, Missouri
Source: ClinicalTrials.gov public record
Updated Sep 16, 2020 · Synced Oct 25, 2026, 12:23 PM EDT
Completed Phase 3 Interventional Results available

A Study to Evaluate the Clinical Efficacy and Safety of Subcutaneously Administered C1-esterase Inhibitor in the Prevention of Hereditary Angioedema

NCT01912456
View directory record Official record
Conditions
Hereditary Angioedema Types I and II
Interventions
Low-volume C1-esterase inhibitor, Higher-volume C1-esterase inhibitor, Low-volume placebo, Higher-volume placebo
Biological
Lead sponsor
CSL Behring
Industry
Eligibility
12 Years and older
Enrollment
90 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2015
U.S. locations
19
States / cities
Birmingham, Alabama • Scottsdale, Arizona • Bell Gardens, California + 16 more
Source: ClinicalTrials.gov public record
Updated Jan 29, 2021 · Synced Oct 25, 2026, 12:23 PM EDT
Recruiting Phase 3 Interventional

CHAMPION: A Study to Evaluate the Efficacy and Safety of Obudanersen (ION582) in Children and Adults With Angelman Syndrome (AS)

NCT07782827
View directory record Official record
Conditions
Angelman Syndrome
Interventions
obudanersen
Drug
Lead sponsor
Ionis Pharmaceuticals, Inc.
Industry
Eligibility
2 Years to 50 Years
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2031
U.S. locations
1
States / cities
Carrboro, North Carolina
Source: ClinicalTrials.gov public record
Updated Oct 20, 2026 · Synced Oct 25, 2026, 12:23 PM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Physical Activity and Cognitive Development in Children

NCT02542683
View directory record Official record
Conditions
Congenital Heart Diseases, Children
Interventions
structured physical activity program
Other
Lead sponsor
Columbia University
Other
Eligibility
30 Months to 6 Years
Enrollment
300 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2016 – 2028
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Mar 4, 2026 · Synced Oct 25, 2026, 12:23 PM EDT
Active, not recruiting No phase listed Observational

The REPLACE Registry for Cholbam® (Cholic Acid)

NCT03115086
View directory record Official record
Conditions
Bile Acid Synthesis Disorders
Interventions
Cholbam
Drug
Lead sponsor
Mirum Pharmaceuticals, Inc.
Industry
Eligibility
Not listed
Enrollment
55 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2039
U.S. locations
24
States / cities
Birmingham, Alabama • Palo Alto, California • San Francisco, California + 18 more
Source: ClinicalTrials.gov public record
Updated Jul 31, 2026 · Synced Oct 25, 2026, 12:23 PM EDT
Completed No phase listed Observational

VCRC Patient Contact Registry Patient-Reported Data Validation Study

NCT02190942
View directory record Official record
Conditions
Behcet's Disease, Churg-Strauss Syndrome, Giant Cell Arteritis, Wegener Granulomatosis, Microscopic Polyangiitis, Polyarteritis Nodosa, Takayasu's Arteritis
Interventions
Not listed
Lead sponsor
University of South Florida
Other
Eligibility
Not listed
Enrollment
198 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2019
U.S. locations
1
States / cities
Tampa, Florida
Source: ClinicalTrials.gov public record
Updated Oct 16, 2019 · Synced Oct 25, 2026, 12:23 PM EDT
Active, not recruiting Phase 2 Interventional Results available

Eltrombopag for Fanconi Anemia

NCT03206086
View directory record Official record
Conditions
Fanconi Anemia
Interventions
Eltrombopag
Drug
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
6 Years to 99 Years
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2028
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 16, 2026 · Synced Oct 25, 2026, 12:23 PM EDT
Recruiting Phase 4 Interventional

Efanesoctocog Alfa Prophylaxis in Patients With Hemophilia A With Synovial Hypertrophy

NCT06941870
View directory record Official record
Conditions
Factor VIII Deficiency
Interventions
Efanesoctocog alfa
Drug
Lead sponsor
Sanofi
Industry
Eligibility
12 Years and older
Enrollment
35 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
5
States / cities
Los Angeles, California • Indianapolis, Indiana • Iowa City, Iowa + 2 more
Source: ClinicalTrials.gov public record
Updated Oct 15, 2026 · Synced Oct 25, 2026, 12:23 PM EDT
Terminated Phase 2 Interventional Results available

Treatment of Adult Patients With Hemoglobin SC Disease (SCYTHE)

NCT02640573
View directory record Official record
Conditions
Hemoglobin SC Disease
Interventions
Hydroxyurea
Drug
Lead sponsor
Baylor College of Medicine
Other
Eligibility
18 Years to 69 Years
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2017
U.S. locations
2
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Jan 27, 2020 · Synced Oct 25, 2026, 12:23 PM EDT
Completed Phase 1 Interventional Results available

Safety and Efficacy of Pembrolizumab (MK-3475) Plus Binimetinib Alone or Pembrolizumab Plus Chemotherapy With or Without Binimetinib in Metastatic Colorectal Cancer (mCRC) Participants (MK-3475-651/KEYNOTE-651)

NCT03374254
View directory record Official record
Conditions
Metastatic Colorectal Cancer
Interventions
Pembrolizumab, Binimetinib, Oxaliplatin, Leucovorin, 5-Fluorouracil [5-FU], Irinotecan
Biological · Drug
Lead sponsor
Merck Sharp & Dohme LLC
Industry
Eligibility
18 Years and older
Enrollment
116 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2023
U.S. locations
10
States / cities
Duarte, California • Aurora, Colorado • New Haven, Connecticut + 7 more
Source: ClinicalTrials.gov public record
Updated Nov 21, 2024 · Synced Oct 25, 2026, 12:23 PM EDT
Completed Not applicable Interventional

A Clinical Study to Evaluate the Safety and Efficacy of a Baby Cleanser and a Moisturizer

NCT03719742
View directory record Official record
Conditions
Atopic Dermatitis
Interventions
Sponsor Test Products
Other
Lead sponsor
Burt's Bees Inc.
Industry
Eligibility
6 Months to 7 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2019
U.S. locations
1
States / cities
Richardson, Texas
Source: ClinicalTrials.gov public record
Updated Apr 19, 2019 · Synced Oct 25, 2026, 12:23 PM EDT
Terminated Phase 3 Interventional

LIFE-DSR-Biomarker Sub-study of Biomarkers in Down Syndrome Related Alzheimer's Disease (DS-AD)

NCT06860373
View directory record Official record
Conditions
Down Syndrome
Interventions
[18F]MK-6240
Biological
Lead sponsor
LuMind IDSC Foundation
Other
Eligibility
35 Years to 55 Years
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2024
U.S. locations
1
States / cities
Phoenix, Arizona
Source: ClinicalTrials.gov public record
Updated Mar 6, 2025 · Synced Oct 25, 2026, 12:23 PM EDT
Completed Phase 2 Interventional

Mind-Body Therapy for Pain in Ehlers-Danlos Syndrome

NCT00001966
View directory record Official record
Conditions
Ehlers-Danlos Syndrome, Pain
Interventions
Mind-body therapy
Drug
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
Not listed
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2000 – 2001
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 4, 2008 · Synced Oct 25, 2026, 12:23 PM EDT