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ClinicalTrials.gov public records Last synced Oct 17, 2026, 5:18 AM EDT

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Showing 937–960 of 10,533 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Congenital Diseases Clear all
Not yet recruiting No phase listed Observational

Southeastern ATTR Amyloidosis Consortium: SEATTRAC Family Registry

NCT05974644
View directory record Official record
Conditions
Amyloidosis, Hereditary
Interventions
Registry
Other
Lead sponsor
Virginia Commonwealth University
Other
Eligibility
18 Years and older
Enrollment
1,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2030
U.S. locations
1
States / cities
Richmond, Virginia
Source: ClinicalTrials.gov public record
Updated Aug 12, 2026 · Synced Oct 17, 2026, 5:18 AM EDT
Completed Phase 2 Interventional Results available

A Study of Oral ARD-101 in Patients With Prader-Willi Syndrome

NCT05153434
View directory record Official record
Conditions
Prader-Willi Syndrome
Interventions
ARD-101
Drug
Lead sponsor
Aardvark Therapeutics, Inc.
Industry
Eligibility
17 Years to 65 Years
Enrollment
19 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2024
U.S. locations
2
States / cities
Palo Alto, California • Aurora, Colorado
Source: ClinicalTrials.gov public record
Updated Sep 8, 2026 · Synced Oct 17, 2026, 5:18 AM EDT
Completed Not applicable Interventional Results available

Virtual Reality Distraction During Outpatient Pediatric Orthopedic Procedures

NCT05510232
View directory record Official record
Conditions
Fractures, Bone, Deformity; Bone
Interventions
A treatment group that will watch a non-VR video via an iPad, A treatment group that will have headset on and will watch a non-VR video, A treatment group that will be immersed in the VR game (Bear Blast) via the headset.
Device
Lead sponsor
Hospital for Special Surgery, New York
Other
Eligibility
4 Years to 14 Years
Enrollment
226 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2022
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jan 31, 2025 · Synced Oct 17, 2026, 5:18 AM EDT
Completed No phase listed Observational Accepts healthy volunteers Results available

Neurologic Injuries in Adults With Urea Cycle Disorders

NCT00472732
View directory record Official record
Conditions
Brain Diseases, Metabolic, Inborn, Urea Cycle Disorder, Ornithine Transcarbamylase Deficiency
Interventions
Not listed
Lead sponsor
Andrea Gropman
Other
Eligibility
18 Years to 60 Years
Enrollment
46 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2007 – 2010
U.S. locations
2
States / cities
Washington D.C., District of Columbia
Source: ClinicalTrials.gov public record
Updated Jun 24, 2015 · Synced Oct 17, 2026, 5:18 AM EDT
Terminated Early Phase 1 Interventional

New York Blood Center National Cord Blood Program

NCT00212407
View directory record Official record
Conditions
Leukemia, Lymphoma, Genetic Disease, Severe Aplastic Anemia, Myelodysplasia
Interventions
Umbilical Cord Blood Transplantation
Biological
Lead sponsor
New York Blood Center
Other
Eligibility
Not listed
Enrollment
4,476 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1993 – 2011
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Nov 2, 2018 · Synced Oct 17, 2026, 5:18 AM EDT
Completed Phase 4 Interventional Results available

VANOS Cream and Skin Barrier Function

NCT00819507
View directory record Official record
Conditions
Atopic Dermatitis
Interventions
Fluocinonide
Drug
Lead sponsor
Oregon Health and Science University
Other
Eligibility
12 Years and older
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2010
U.S. locations
1
States / cities
Portland, Oregon
Source: ClinicalTrials.gov public record
Updated Jul 11, 2017 · Synced Oct 17, 2026, 5:18 AM EDT
Not listed Not applicable Interventional

Investigation of Supplemental L-alanine in the Management of Dietary Fructose Intolerance

NCT04022434
View directory record Official record
Conditions
Fructose Intolerance
Interventions
Placedo, Alanine
Dietary Supplement
Lead sponsor
Augusta University
Other
Eligibility
18 Years and older
Enrollment
65 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2020
U.S. locations
1
States / cities
Augusta, Georgia
Source: ClinicalTrials.gov public record
Updated Apr 15, 2020 · Synced Oct 17, 2026, 5:18 AM EDT
Completed Phase 3 Interventional Results available

Long Term Administration of Inhaled Mannitol in Cystic Fibrosis

NCT00630812
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
inhaled mannitol, Placebo comparator
Drug
Lead sponsor
Syntara
Industry
Eligibility
6 Years and older
Enrollment
318 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2010
U.S. locations
28
States / cities
Tucson, Arizona • Hartford, Connecticut • Jacksonville, Florida + 23 more
Source: ClinicalTrials.gov public record
Updated Oct 9, 2020 · Synced Oct 17, 2026, 5:18 AM EDT
Recruiting Phase 1Phase 2 Interventional

ATR 1072 in Participants With PRKAG2 Syndrome

NCT07738107
View directory record Official record
Conditions
Cardiac Glycogenosis, Cardiomyopathy, Hypertrophic, Wolff-Parkinson-White Syndrome
Interventions
ATR 1072
Drug
Lead sponsor
Atrium Therapeutics
Industry
Eligibility
18 Years to 65 Years
Enrollment
37 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2028
U.S. locations
1
States / cities
Kansas City, Missouri
Source: ClinicalTrials.gov public record
Updated Sep 18, 2026 · Synced Oct 17, 2026, 5:18 AM EDT
Completed Phase 1 Interventional Accepts healthy volunteers Results available

Study Of PF-06817024 In Healthy Subjects, In Patients With Chronic Rhinosinusitis With Nasal Polyps And in Patients With Atopic Dermatitis

NCT02743871
View directory record Official record
Conditions
Healthy, Chronic Rhinosinusitis With Nasal Polyps, Atopic Dermatitis
Interventions
PF-06817024, Placebo for PF-06817024
Biological · Other
Lead sponsor
Pfizer
Industry
Eligibility
18 Years to 75 Years
Enrollment
97 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2016 – 2021
U.S. locations
20
States / cities
Sacramento, California • New Haven, Connecticut • Shelton, Connecticut + 12 more
Source: ClinicalTrials.gov public record
Updated May 19, 2022 · Synced Oct 17, 2026, 5:18 AM EDT
Completed Phase 4 Interventional Results available

Guanfacine in Children With Tic Disorders

NCT01547000
View directory record Official record
Conditions
Tourette Disorder, Tourette Syndrome
Interventions
placebo, extended-release guanfacine (Intuniv)
Drug
Lead sponsor
Yale University
Other
Eligibility
6 Years to 17 Years
Enrollment
34 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2016
U.S. locations
3
States / cities
New Haven, Connecticut • Tampa, Florida • New York, New York
Source: ClinicalTrials.gov public record
Updated Aug 13, 2021 · Synced Oct 17, 2026, 5:18 AM EDT
Completed Phase 1 Interventional

Re-administration of Intramuscular AAV9 in Patients With Late-Onset Pompe Disease

NCT02240407
View directory record Official record
Conditions
Pompe Disease
Interventions
Recombinant Adeno-Associated Virus Acid Alpha-Glucosidase, Rapamycin, saline, Rituxan, Diphenhydramine, Acetaminophen, Lidocaine, LMX 4 Topical Cream
Genetic · Drug · Other
Lead sponsor
University of Florida
Other
Eligibility
18 Years to 50 Years
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2021
U.S. locations
1
States / cities
Gainesville, Florida
Source: ClinicalTrials.gov public record
Updated Apr 5, 2022 · Synced Oct 17, 2026, 5:18 AM EDT
Withdrawn Not applicable Interventional

Ranibizumab for Neovascularization in Sickle Cell Retinopathy

NCT00618644
View directory record Official record
Conditions
Sickle Cell Anemia, Retinopathy
Interventions
Ranibizumab
Drug
Lead sponsor
Kresge Eye Institute
Other
Eligibility
18 Years and older
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2011
U.S. locations
1
States / cities
Detroit, Michigan
Source: ClinicalTrials.gov public record
Updated Sep 27, 2012 · Synced Oct 17, 2026, 5:18 AM EDT
Terminated Phase 2 Interventional Results available

Open-label Long-term Safety Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Disease Who Have Completed a Previous AT1001 Study

NCT00526071
View directory record Official record
Conditions
Fabry Disease
Interventions
migalastat HCl
Drug
Lead sponsor
Amicus Therapeutics
Industry
Eligibility
18 Years and older
Enrollment
23 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2012
U.S. locations
3
States / cities
Decatur, Georgia • New York, New York • Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Oct 3, 2018 · Synced Oct 17, 2026, 5:18 AM EDT
Completed Not applicable Interventional Results available

The Brain, Neurological Features and Neuropsychological Functioning in Adults With Phenylketonuria: A Pilot Study

NCT01917344
View directory record Official record
Conditions
Phenylketonuria
Interventions
MRI
Other
Lead sponsor
Boston Children's Hospital
Other
Eligibility
18 Years to 55 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2014
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Jun 19, 2015 · Synced Oct 17, 2026, 5:18 AM EDT
Completed No phase listed Observational

Genetic Linkage Study of Idiopathic Talipes Equinovarus (ITEV) (Clubfoot)

NCT00474344
View directory record Official record
Conditions
Clubfoot
Interventions
Not listed
Lead sponsor
University of British Columbia
Other
Eligibility
Not listed
Enrollment
370 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2013
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated May 18, 2022 · Synced Oct 17, 2026, 5:18 AM EDT
Completed Phase 2Phase 3 Interventional

Supplemental Therapeutic Oxygen for Prethreshold Retinopathy of Prematurity

NCT01203436
View directory record Official record
Conditions
Infant, Newborn, Infant, Low Birth Weight, Infant, Small for Gestational Age, Infant, Premature, Retinopathy of Prematurity, Blindness
Interventions
Supplemental Oxygen Management, Conventional Oxygen Management
Procedure
Lead sponsor
NICHD Neonatal Research Network
Network
Eligibility
Up to 48 Hours
Enrollment
649 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1994 – 1999
U.S. locations
9
States / cities
Palo Alto, California • New Haven, Connecticut • Atlanta, Georgia + 6 more
Source: ClinicalTrials.gov public record
Updated Jun 8, 2015 · Synced Oct 17, 2026, 5:18 AM EDT
Completed Not applicable Interventional Accepts healthy volunteers Results available

A Community-based Assessment of Skin Care, Allergies, and Eczema

NCT03409367
View directory record Official record
Conditions
Atopic Dermatitis, Atopic Disorders, Eczema, Atopic Eczema
Interventions
Participant choice of over-the-counter emollients: Vaseline, Vanicream, CeraVe Healing Ointment, CeraVe cream, Cetaphil cream
Other
Lead sponsor
Oregon Health and Science University
Other
Eligibility
1 Day to 63 Days
Enrollment
1,260 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2018 – 2024
U.S. locations
4
States / cities
Denver, Colorado • Durham, North Carolina • Portland, Oregon + 1 more
Source: ClinicalTrials.gov public record
Updated Aug 15, 2025 · Synced Oct 17, 2026, 5:18 AM EDT
Enrolling by invitation No phase listed Observational Accepts healthy volunteers

Distribution of Ventilation, Respiratory Drive and Gas Exchange: Measurements and Monitoring

NCT05462600
View directory record Official record
Conditions
Pulmonary Disease, Respiratory System Abnormalities, OSA, Respiratory Failure
Interventions
Position change
Other
Lead sponsor
University of California, San Diego
Other
Eligibility
18 Years and older
Enrollment
40 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2022 – 2026
U.S. locations
1
States / cities
La Jolla, California
Source: ClinicalTrials.gov public record
Updated Apr 11, 2025 · Synced Oct 17, 2026, 5:18 AM EDT
Active, not recruiting Phase 1Phase 2 Interventional

Dose-escalation Study to Evaluate the Safety and Tolerability of GS030 in Subjects With Retinitis Pigmentosa

NCT03326336
View directory record Official record
Conditions
Non-syndromic Retinitis Pigmentosa
Interventions
Gene therapy: GS030-DP AND Medical device: GS030-MD
Combination Product
Lead sponsor
GenSight Biologics
Industry
Eligibility
18 Years to 75 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2027
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Mar 2, 2026 · Synced Oct 17, 2026, 5:18 AM EDT
Completed No phase listed Observational

REALITY LHON Registry

NCT03295071
View directory record Official record
Conditions
Leber Hereditary Optic Neuropathy
Interventions
Patient-reported outcomes (PROs)
Other
Lead sponsor
GenSight Biologics
Industry
Eligibility
Not listed
Enrollment
44 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2020
U.S. locations
5
States / cities
Pasadena, California • Atlanta, Georgia • Boston, Massachusetts + 2 more
Source: ClinicalTrials.gov public record
Updated Mar 2, 2026 · Synced Oct 17, 2026, 5:18 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Parkinsonism in Spinocerebellar Ataxia Type 6

NCT01934998
View directory record Official record
Conditions
Spinocerebellar Ataxia Type 6
Interventions
Not listed
Lead sponsor
University of Chicago
Other
Eligibility
18 Years and older
Enrollment
20 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2013 – 2015
U.S. locations
1
States / cities
Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Apr 28, 2015 · Synced Oct 17, 2026, 5:18 AM EDT
Completed Phase 3 Interventional

Botox Injection for Lower Extremity Lengthening and Deformity Correction Surgery

NCT00412035
View directory record Official record
Conditions
Unequal Length of Limbs; Congenital, Lower Extremity Deformities, Congenital
Interventions
Botulinum toxin A injection, saline injection
Drug
Lead sponsor
Shriners Hospitals for Children
Other
Eligibility
5 Years to 21 Years
Enrollment
125 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2012
U.S. locations
4
States / cities
Wilmington, Delaware • Honolulu, Hawaii • Portland, Oregon + 1 more
Source: ClinicalTrials.gov public record
Updated Dec 23, 2014 · Synced Oct 17, 2026, 5:18 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 17, 2026, 5:18 AM EDT