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ClinicalTrials.gov public records Last synced Oct 13, 2026, 12:37 AM EDT

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Showing 193–216 of 10,525 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Congenital Diseases Clear all
Active, not recruiting Phase 3 Interventional

Study to Evaluate the Efficacy and Safety of Venglustat in Adult and Pediatric Patients With Gaucher Disease Type 3

NCT05222906
View directory record Official record
Conditions
Gaucher's Disease Type III
Interventions
Venglustat, imiglucerase
Drug
Lead sponsor
Sanofi
Industry
Eligibility
12 Years and older
Enrollment
43 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2026
U.S. locations
4
States / cities
New Haven, Connecticut • Iowa City, Iowa • Dallas, Texas + 1 more
Source: ClinicalTrials.gov public record
Updated Jul 13, 2026 · Synced Oct 13, 2026, 12:37 AM EDT
Withdrawn Phase 1Phase 2 Interventional

Mobile Phone App for Depression and Anxiety in Young Men Who Are Attracted to Men

NCT02072252
View directory record Official record
Conditions
Depressive Symptoms, Anxiety Generalized
Interventions
Mobile Phone Application
Behavioral
Lead sponsor
Northwestern University
Other
Eligibility
17 Years to 20 Years · Male only
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017
U.S. locations
1
States / cities
Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Aug 17, 2017 · Synced Oct 13, 2026, 12:37 AM EDT
Completed Phase 2 Interventional

Study of GMI-1070 for the Treatment of Sickle Cell Pain Crisis

NCT01119833
View directory record Official record
Conditions
Sickle Cell Disease, Vaso-occlusive Crisis, Pain Crisis
Interventions
GMI-1070, Placebo
Drug
Lead sponsor
GlycoMimetics Incorporated
Industry
Eligibility
12 Years to 60 Years
Enrollment
81 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2013
U.S. locations
21
States / cities
Birmingham, Alabama • Berkeley, California • Sacramento, California + 18 more
Source: ClinicalTrials.gov public record
Updated May 13, 2020 · Synced Oct 13, 2026, 12:37 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Lung Ultrasound to Diagnose Transient Tachypnea of the Newborn (TTN) Versus Respiratory Distress Syndrome (RDS) in Neonates

NCT01517958
View directory record Official record
Conditions
Transient Tachypnea of the Newborn, TTN, Respiratory Distress Syndrome, RDS
Interventions
Respiratory Distress Group, Control Group
Other
Lead sponsor
Icahn School of Medicine at Mount Sinai
Other
Eligibility
28 Weeks and older
Enrollment
36 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2011 – 2013
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Mar 25, 2016 · Synced Oct 13, 2026, 12:37 AM EDT
Terminated Phase 2 Interventional

PTC299 for Treatment of Neurofibromatosis Type 2

NCT00911248
View directory record Official record
Conditions
Neurofibromatosis 2
Interventions
PTC299
Drug
Lead sponsor
PTC Therapeutics
Industry
Eligibility
18 Years and older
Enrollment
11 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2012
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Apr 12, 2019 · Synced Oct 13, 2026, 12:37 AM EDT
Completed No phase listed Observational

Identifying Barriers and Strategies to Support Self-efficacy for Medication Adherence With Text Messaging

NCT02785848
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Interviews and Surveys
Other
Lead sponsor
Vanderbilt University
Other
Eligibility
12 Years to 70 Years
Enrollment
70 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2026
U.S. locations
1
States / cities
Nashville, Tennessee
Source: ClinicalTrials.gov public record
Updated Sep 23, 2026 · Synced Oct 13, 2026, 12:37 AM EDT
Completed Phase 1 Interventional

A Randomized Comparative Pharmacokinetic Study of Oral Ganciclovir After Treatment With Intravenous Ganciclovir for Cytomegalovirus Gastrointestinal Disease in AIDS Patients

NCT00000768
View directory record Official record
Conditions
Colitis, HIV Infections
Interventions
Glutamic acid hydrochloride, Ganciclovir
Drug
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
13 Years and older
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Ends 1998
U.S. locations
5
States / cities
Birmingham, Alabama • San Francisco, California • St Louis, Missouri + 2 more
Source: ClinicalTrials.gov public record
Updated Nov 4, 2021 · Synced Oct 13, 2026, 12:37 AM EDT
Terminated Phase 2 Interventional Results available

Topical Sodium Nitrite in Sickle Cell Disease and Leg Ulcers

NCT02863068
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Topical Sodium Nitrite, Placebo
Drug · Other
Lead sponsor
caterina P minniti
Other
Eligibility
18 Years and older
Enrollment
18 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2023
U.S. locations
3
States / cities
The Bronx, New York • Durham, North Carolina • Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated May 7, 2025 · Synced Oct 13, 2026, 12:37 AM EDT
Completed Not applicable Interventional

Improving Neurodevelopment in Adolescents With Congenital Heart Disease

NCT02759263
View directory record Official record
Conditions
Congenital Heart Disease, Neurodevelopment, Executive Function, Working Memory Training, Infant Open-heart Surgery
Interventions
Cogmed Working Memory Training
Behavioral
Lead sponsor
Boston Children's Hospital
Other
Eligibility
13 Years to 16 Years
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2018
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Oct 23, 2018 · Synced Oct 13, 2026, 12:37 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

New Quantitive MRI Parameters in Assessing Kidneys of Autosomal Dominant Polycystic Kidney Disease

NCT02250287
View directory record Official record
Conditions
Autosomal Dominant Polycystic Kidney Disease, Kidney Disease
Interventions
Not listed
Lead sponsor
Mayo Clinic
Other
Eligibility
18 Years to 30 Years
Enrollment
20 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2014 – 2016
U.S. locations
1
States / cities
Rochester, Minnesota
Source: ClinicalTrials.gov public record
Updated Jun 17, 2016 · Synced Oct 13, 2026, 12:37 AM EDT
Terminated Phase 4 Interventional Results available

The Effects of Eplerenone on Markers of Myocardial Fibrosis in Adult Congenital Heart Disease

NCT01971593
View directory record Official record
Conditions
Tetralogy of Fallot, Transposition of the Great Vessels With an Arterial Switch, Single Ventricle With a Fontan Palliation
Interventions
Eplerenone
Drug
Lead sponsor
Washington University School of Medicine
Other
Eligibility
18 Years to 80 Years
Enrollment
26 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2016
U.S. locations
1
States / cities
St Louis, Missouri
Source: ClinicalTrials.gov public record
Updated May 4, 2018 · Synced Oct 13, 2026, 12:37 AM EDT
Active, not recruiting No phase listed Observational

Metagenomic Evaluation of the Gut Microbiome in Patients With Lynch Syndrome and Other Hereditary Colonic Polyposis Syndromes

NCT02371135
View directory record Official record
Conditions
Lynch Syndrome, Hereditary Colonic Polyposis Syndromes
Interventions
colonic biopsies, questionnaires
Genetic
Lead sponsor
Memorial Sloan Kettering Cancer Center
Other
Eligibility
18 Years and older
Enrollment
77 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2027
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Feb 5, 2026 · Synced Oct 13, 2026, 12:37 AM EDT
Terminated Phase 2 Interventional Results available

Early Signs of Efficacy Study With Riociguat in Adult Homozygous Delta F508 Cystic Fibrosis Patients

NCT02170025
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
Riociguat (Adempas, BAY63-2521), Placebo
Drug
Lead sponsor
Bayer
Industry
Eligibility
18 Years and older
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2017
U.S. locations
3
States / cities
Birmingham, Alabama • Denver, Colorado • St Louis, Missouri
Source: ClinicalTrials.gov public record
Updated Nov 7, 2023 · Synced Oct 13, 2026, 12:37 AM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

A Study of Invasive Hemodynamic for MACHD Registry

NCT05849025
View directory record Official record
Conditions
Healthy, Congenital Heart Disease
Interventions
Not listed
Lead sponsor
Mayo Clinic
Other
Eligibility
18 Years and older
Enrollment
50 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2023 – 2026
U.S. locations
1
States / cities
Rochester, Minnesota
Source: ClinicalTrials.gov public record
Updated May 5, 2026 · Synced Oct 13, 2026, 12:37 AM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

A Study to Compare the Relative Bioavailability of Two Iberdomide (CC-220) Formulations and to Assess The Effect Of Food on the Drug Levels of Iberdomide in Healthy Adult Participants

NCT05899738
View directory record Official record
Conditions
Healthy Volunteers
Interventions
Iberdomide
Drug
Lead sponsor
Bristol-Myers Squibb
Industry
Eligibility
18 Years to 65 Years
Enrollment
32 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2023
U.S. locations
1
States / cities
Daytona Beach, Florida
Source: ClinicalTrials.gov public record
Updated Jul 26, 2024 · Synced Oct 13, 2026, 12:37 AM EDT
Completed Phase 1Phase 2 Interventional Results available

Study of PI3Kinase Inhibition (Copanlisib) and Anti-PD-1 Antibody Nivolumab in Relapsed/Refractory Solid Tumors With Expansions in Mismatch-repair Proficient (MSS) Colorectal Cancer

NCT03711058
View directory record Official record
Conditions
Unresectable or Metastatic Microsatellite Stable (MSS) Solid Tumor, Microsatellite Stable (MSS) Colon Cancer
Interventions
Copanlisib, Nivolumab
Drug
Lead sponsor
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
Other
Eligibility
18 Years and older
Enrollment
48 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2025
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 16, 2025 · Synced Oct 13, 2026, 12:37 AM EDT
Active, not recruiting Phase 1Phase 2 Interventional

A Study of Tividenofusp Alfa (DNL310) in Pediatric Participants With Hunter Syndrome

NCT04251026
View directory record Official record
Conditions
Mucopolysaccharidosis II
Interventions
tividenofusp alfa
Drug
Lead sponsor
Denali Therapeutics Inc.
Industry
Eligibility
Up to 18 Years · Male only
Enrollment
47 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2031
U.S. locations
4
States / cities
Oakland, California • Chicago, Illinois • Chapel Hill, North Carolina + 1 more
Source: ClinicalTrials.gov public record
Updated Aug 7, 2025 · Synced Oct 13, 2026, 12:37 AM EDT
Completed No phase listed Observational

Application of a Systematic Developmental Assessment to a Novel Population: Infants With Rare Genetic Disorders

NCT03967743
View directory record Official record
Conditions
Genetic Disease, Genetic Syndrome, Genetic Predisposition to Disease, Development, Infant, Development, Child
Interventions
Not listed
Lead sponsor
Boston Children's Hospital
Other
Eligibility
Up to 4 Years
Enrollment
43 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2026
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Jun 2, 2026 · Synced Oct 13, 2026, 12:37 AM EDT
Completed Phase 2 Interventional

Choline Supplementation in Children With Fetal Alcohol Spectrum Disorders

NCT01911299
View directory record Official record
Conditions
Fetal Alcohol Spectrum Disorders, Fetal Alcohol Syndrome, Partial Fetal Alcohol Syndrome, Alcohol-related Neurodevelopmental Disorder, Prenatal Alcohol Exposure
Interventions
Choline, placebo supplementation consisting of vegetable glycerin (50% by volume) and deionized water
Dietary Supplement
Lead sponsor
San Diego State University
Other
Eligibility
5 Years to 10 Years
Enrollment
55 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2014
U.S. locations
1
States / cities
San Diego, California
Source: ClinicalTrials.gov public record
Updated Aug 1, 2014 · Synced Oct 13, 2026, 12:37 AM EDT
Completed Phase 1Phase 2 Interventional

Phase I/II Study of Total Body Irradiation, Cyclophosphamide, and Fludarabine Followed by Alternate Donor Hematopoietic Cell Transplantation in Patients With Fanconi's Anemia

NCT00005898
View directory record Official record
Conditions
Fanconi's Anemia
Interventions
anti-thymocyte globulin, cyclophosphamide, cyclosporine, filgrastim, fludarabine, methylprednisolone
Drug
Lead sponsor
University of Minnesota
Other
Eligibility
0 Years to 34 Years
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2000
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Jun 24, 2005 · Synced Oct 13, 2026, 12:37 AM EDT
Recruiting No phase listed Observational

Sensory Symptoms in Tourette Syndrome

NCT03851484
View directory record Official record
Conditions
Tourette Syndrome
Interventions
Not listed
Lead sponsor
Vanderbilt University Medical Center
Other
Eligibility
18 Years and older
Enrollment
214 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2028
U.S. locations
2
States / cities
Nashville, Tennessee
Source: ClinicalTrials.gov public record
Updated Mar 13, 2026 · Synced Oct 13, 2026, 12:37 AM EDT
Terminated Early Phase 1 Interventional

68Ga-DOTA-TOC PET/CT in Imaging Participants With Neuroendocrine Tumors

NCT03001349
View directory record Official record
Conditions
Meningioma, Metastatic Well Differentiated Neuroendocrine Neoplasm, Multiple Endocrine Neoplasia Type 1, Neuroendocrine Neoplasm, Somatostatin Positive Neoplastic Cells Present, Von Hippel-Lindau Syndrome
Interventions
Computed Tomography, Gallium Ga 68-Edotreotide, Positron Emission Tomography
Procedure · Drug
Lead sponsor
M.D. Anderson Cancer Center
Other
Eligibility
Not listed
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2021
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Feb 15, 2021 · Synced Oct 13, 2026, 12:37 AM EDT
Active, not recruiting Phase 2 Interventional

A Study of Zenocutuzumab (MCLA-128) in Patients With Solid Tumors Harboring an NRG1 Fusion (eNRGy)

NCT02912949
View directory record Official record
Conditions
Solid Tumours Harboring NRG1 Fusion, NSCLC Harboring NRG1 Fusion, Pancreatic Cancer Harboring NRG1 Fusion, NRG1 Fusion
Interventions
zenocutuzumab (MCLA-128)
Drug
Lead sponsor
Partner Therapeutics, Inc.
Industry
Eligibility
18 Years and older
Enrollment
250 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2026
U.S. locations
26
States / cities
Phoenix, Arizona • Cerritos, California • Irvine, California + 21 more
Source: ClinicalTrials.gov public record
Updated Apr 29, 2025 · Synced Oct 13, 2026, 12:37 AM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

Dose Response to Recombinant Factor VIIa When Administered for Bleed

NCT00104455
View directory record Official record
Conditions
Congenital Bleeding Disorder, Healthy
Interventions
activated recombinant human factor VII
Drug
Lead sponsor
Novo Nordisk A/S
Industry
Eligibility
18 Years to 60 Years · Male only
Enrollment
64 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2004
U.S. locations
1
States / cities
Overland Park, Kansas
Source: ClinicalTrials.gov public record
Updated Jan 12, 2017 · Synced Oct 13, 2026, 12:37 AM EDT