Independent directory Public ClinicalTrials.gov records United States
Clinical Trials Finder
Objective public record navigation
Search Results

Search by objective public record fields.

Search by query text, NCT ID, condition, intervention, sponsor, city, state, recruitment status, phase, study type, healthy volunteer eligibility, sex, or age. Results are retrieved from ClinicalTrials.gov and synchronized into the directory. Search pages remain noindex by default.

Clear filters
ClinicalTrials.gov public records Last synced Sep 23, 2026, 12:40 PM EDT

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

Showing 25–48 of 24 matching trials from the live ClinicalTrials.gov search.
Local D1 index available.
Filters: Condition: Genetic Syndrome Clear all
Completed Phase 3 Interventional Results available

A Study of PEGylated Recombinant Factor VIII (BAX855) in Previously Untreated Young Children With Severe Hemophilia A

NCT02615691
View directory record Official record
Conditions
Hemophilia A
Interventions
PEGylated Recombinant Factor VIII, ITI
Biological
Lead sponsor
Baxalta now part of Shire
Industry
Eligibility
Up to 5 Years
Enrollment
120 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2024
U.S. locations
20
States / cities
Phoenix, Arizona • Cupertino, California • Oakland, California + 17 more
Source: ClinicalTrials.gov public record
Updated Jul 27, 2025 · Synced Sep 23, 2026, 12:40 PM EDT
Completed Phase 2 Interventional Results available

Transcranial Magnetic Stimulation (TMS) for Individuals With Tourette's Syndrome

NCT00529308
View directory record Official record
Conditions
Tourette Syndrome
Interventions
Transcranial Magnetic Stimulation (active), Transcranial Magnetic Stimulation (sham)
Device
Lead sponsor
Yale University
Other
Eligibility
18 Years to 70 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2011
U.S. locations
2
States / cities
New Haven, Connecticut • New York, New York
Source: ClinicalTrials.gov public record
Updated Feb 20, 2019 · Synced Sep 23, 2026, 12:40 PM EDT
Completed Early Phase 1 Interventional

Biological Clock Dysfunction in Optic Nerve Hypoplasia

NCT00825591
View directory record Official record
Conditions
Biological Clock Dysfunction, Optic Nerve Hypoplasia
Interventions
Melatonin, Placebo Comparator
Dietary Supplement
Lead sponsor
Yale University
Other
Eligibility
2 Years to 10 Years
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2011
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Sep 24, 2020 · Synced Sep 23, 2026, 12:40 PM EDT
Completed Phase 4 Interventional Results available

EAGLE: Evaluating Genotypes Using Intravitreal Aflibercept Injection

NCT02689518
View directory record Official record
Conditions
Macular Degeneration, Wet Macular Degeneration
Interventions
Intravitreal aflibercept injection
Drug
Lead sponsor
University of California, San Diego
Other
Eligibility
50 Years to 90 Years
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2019
U.S. locations
1
States / cities
La Jolla, California
Source: ClinicalTrials.gov public record
Updated Mar 24, 2021 · Synced Sep 23, 2026, 12:40 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Development of the ORCA Communication Measure for Rett Syndrome

NCT04920110
View directory record Official record
Conditions
Rett Syndrome
Interventions
Not listed
Lead sponsor
Rett Syndrome Research Trust
Other
Eligibility
18 Years and older
Enrollment
272 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2021 – 2022
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Apr 21, 2022 · Synced Sep 23, 2026, 12:40 PM EDT
Active, not recruiting Phase 1Phase 2 Interventional Results available

Feasibility of [11C]Acetate-PET in LAM and TSC

NCT05467397
View directory record Official record
Conditions
Lymphangioleiomyomatosis, Tuberous Sclerosis Complex
Interventions
[11C]acetate
Drug
Lead sponsor
Brigham and Women's Hospital
Other
Eligibility
18 Years and older · Female only
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2026
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Jan 20, 2026 · Synced Sep 23, 2026, 12:40 PM EDT
Terminated Phase 2 Interventional

Vitamin D Supplementation for Adults With Neurofibromatosis Type 1 (NF1)

NCT01968590
View directory record Official record
Conditions
Neurofibromatosis Type 1 (NF1)
Interventions
Cholecalciferol
Drug
Lead sponsor
University of Utah
Other
Eligibility
25 Years to 40 Years
Enrollment
26 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2022
U.S. locations
2
States / cities
Cincinnati, Ohio • Salt Lake City, Utah
Source: ClinicalTrials.gov public record
Updated May 24, 2022 · Synced Sep 23, 2026, 12:40 PM EDT
Completed Phase 2 Interventional Results available

An Extension Study to Evaluate the Long-Term Safety and Durability of Effect of LUM001 in the Treatment of Cholestatic Liver Disease in Pediatric Subjects With Alagille Syndrome

NCT02117713
View directory record Official record
Conditions
Alagille Syndrome
Interventions
LUM001 (Maralixibat)
Drug
Lead sponsor
Mirum Pharmaceuticals, Inc.
Industry
Eligibility
1 Year to 18 Years
Enrollment
34 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2020
U.S. locations
10
States / cities
Los Angeles, California • San Francisco, California • Aurora, Colorado + 7 more
Source: ClinicalTrials.gov public record
Updated Jun 30, 2021 · Synced Sep 23, 2026, 12:40 PM EDT
Completed No phase listed Observational

Correlation of Gene Abnormalities and Clinical Manifestations of Aniridia

NCT00265590
View directory record Official record
Conditions
Aniridia
Interventions
Not listed
Lead sponsor
National Eye Institute (NEI)
NIH
Eligibility
4 Years and older
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2008
U.S. locations
3
States / cities
Chicago, Illinois • Bethesda, Maryland • Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Jul 1, 2017 · Synced Sep 23, 2026, 12:40 PM EDT
Completed Phase 4 Interventional

Replagal Enzyme Replacement Therapy for Adults With Fabry Disease

NCT00097890
View directory record Official record
Conditions
Fabry Disease
Interventions
Replagal (Agalsidase Alfa), Replagal
Drug
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
Male only
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2005
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 3, 2008 · Synced Sep 23, 2026, 12:40 PM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

A Phase 1, Non-Randomized, Open-Label, Single-Dose Study To Evaluate The Pharmacokinetics, Safety, And Tolerability Of Dimebon [PF 01913539] In Subjects With Severely-Impaired And Normal Renal Function

NCT00824590
View directory record Official record
Conditions
Alzheimer's Disease, Huntington's Disease
Interventions
Dimebon
Drug
Lead sponsor
Pfizer
Industry
Eligibility
18 Years to 75 Years
Enrollment
20 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2009
U.S. locations
2
States / cities
Miami, Florida • Saint Paul, Minnesota
Source: ClinicalTrials.gov public record
Updated Dec 29, 2009 · Synced Sep 23, 2026, 12:40 PM EDT
Completed Phase 2 Interventional Results available

Using Topical Sirolimus 2% for Patients With Epidermolysis Bullous Simplex (EBS) Study

NCT02960997
View directory record Official record
Conditions
Epidermolysis Bullosa Simplex, Epidermolysis Bullosa Simplex Kobner, Weber-Cockayne Syndrome
Interventions
Sirolimus, 2%, Vehicle
Drug
Lead sponsor
Stanford University
Other
Eligibility
4 Years and older
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2021
U.S. locations
1
States / cities
Palo Alto, California
Source: ClinicalTrials.gov public record
Updated Mar 8, 2022 · Synced Sep 23, 2026, 12:40 PM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Caregiving Networks Across Disease Context and the Life Course

NCT05007990
View directory record Official record
Conditions
Inherited Metabolic Disorders, Undiagnosed Diseases, Batten's Disease, Tay Sachs, Diabetes
Interventions
Not listed
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
18 Years to 100 Years
Enrollment
2,800 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2022 – 2030
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 14, 2026 · Synced Sep 23, 2026, 12:40 PM EDT
Completed No phase listed Observational

Screening an Orthopedic Population for Mildly-affected Individuals With Morquio Syndrome A and Maroteaux-Lamy Syndrome

NCT01961518
View directory record Official record
Conditions
Morquio Syndrome A, Maroteaux Lamy Syndrome, MPS IVA, MPS VI
Interventions
Not listed
Lead sponsor
Greenwood Genetic Center
Other
Eligibility
Up to 18 Years
Enrollment
17 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2015
U.S. locations
1
States / cities
Greenville, South Carolina
Source: ClinicalTrials.gov public record
Updated Aug 12, 2015 · Synced Sep 23, 2026, 12:40 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Muscle Tissue Bank for Muscular Dystrophy

NCT01950897
View directory record Official record
Conditions
Muscular Dystrophies, Muscular Dystrophy
Interventions
Not listed
Lead sponsor
Wake Forest University Health Sciences
Other
Eligibility
6 Years and older
Enrollment
21 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2005 – 2015
U.S. locations
1
States / cities
Charlotte, North Carolina
Source: ClinicalTrials.gov public record
Updated Apr 25, 2022 · Synced Sep 23, 2026, 12:40 PM EDT
Completed Phase 3 Interventional Results available

Rollover Study of Ivacaftor in Subjects With Cystic Fibrosis and a Non G551D CFTR Mutation

NCT01707290
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
Ivacaftor
Drug
Lead sponsor
Vertex Pharmaceuticals Incorporated
Industry
Eligibility
6 Years and older
Enrollment
125 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2016
U.S. locations
33
States / cities
Birmingham, Alabama • Palo Alto, California • Denver, Colorado + 30 more
Source: ClinicalTrials.gov public record
Updated May 11, 2017 · Synced Sep 23, 2026, 12:40 PM EDT
Recruiting No phase listed Observational

Registry Study to Observe Long-term Safety of Vamorolone (AGAMREE®) in Patients With Duchenne Muscular Dystrophy-SUMMIT

NCT06564974
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Vamorolone
Drug
Lead sponsor
Catalyst Pharmaceuticals, Inc.
Industry
Eligibility
2 Years and older · Male only
Enrollment
250 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2032
U.S. locations
31
States / cities
Phoenix, Arizona • Little Rock, Arkansas • Loma Linda, California + 27 more
Source: ClinicalTrials.gov public record
Updated Jul 19, 2026 · Synced Sep 23, 2026, 12:40 PM EDT
Completed Not applicable Interventional

Marrow Mesenchymal Cell Therapy for Osteogenesis Imperfecta: A Pilot Study

NCT00187018
View directory record Official record
Conditions
Osteogenesis Imperfecta
Interventions
Bone marrow transplant
Biological
Lead sponsor
St. Jude Children's Research Hospital
Other
Eligibility
Not listed
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2007
U.S. locations
1
States / cities
Memphis, Tennessee
Source: ClinicalTrials.gov public record
Updated Mar 3, 2015 · Synced Sep 23, 2026, 12:40 PM EDT
Completed No phase listed Observational

Genetics of Mammographic Appearances and Patterns (The GenMap Study)

NCT01160510
View directory record Official record
Conditions
Benign Breast Disease
Interventions
Not listed
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
18 Years to 100 Years · Female only
Enrollment
106 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2020
U.S. locations
1
States / cities
Burlington, Vermont
Source: ClinicalTrials.gov public record
Updated Mar 11, 2020 · Synced Sep 23, 2026, 12:40 PM EDT
Terminated No phase listed Observational

Observational Study to Characterize Biomarkers and Disease Progression in Participants With Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome

NCT06014541
View directory record Official record
Conditions
Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome
Interventions
Not listed
Lead sponsor
Ionis Pharmaceuticals, Inc.
Industry
Eligibility
1 Month to 65 Years · Male only
Enrollment
29 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2025
U.S. locations
5
States / cities
San Diego, California • Saint Paul, Minnesota • Philadelphia, Pennsylvania + 2 more
Source: ClinicalTrials.gov public record
Updated Apr 8, 2026 · Synced Sep 23, 2026, 12:40 PM EDT
Completed Phase 3 Interventional Results available

The Safety and Tolerability of Alpha-1 Modified Process (MP) In Subjects With Alpha-1-antitrypsin (AAT) Deficiency

NCT00301366
View directory record Official record
Conditions
Alpha 1-Antitrypsin Deficiency
Interventions
alpha-1 proteinase inhibitor (human)
Drug
Lead sponsor
Grifols Therapeutics LLC
Industry
Eligibility
18 Years and older
Enrollment
38 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2007
U.S. locations
8
States / cities
Denver, Colorado • Gainesville, Florida • Miami, Florida + 5 more
Source: ClinicalTrials.gov public record
Updated Aug 18, 2014 · Synced Sep 23, 2026, 12:40 PM EDT
Recruiting No phase listed Observational

MRI as Noninvasive Innovative Approach in Detection and Monitoring of Malignant Oral Lesions in Fanconi Anemia Patients

NCT07649031
View directory record Official record
Conditions
Fanconi Anemia
Interventions
MRIs
Diagnostic Test
Lead sponsor
University of Minnesota
Other
Eligibility
18 Years and older
Enrollment
80 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2030
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Jul 1, 2026 · Synced Sep 23, 2026, 12:40 PM EDT
Completed Phase 4 Interventional

A Multicenter Study of the Efficacy of Cerezyme in Testing Skeletal Disease in Patients With Type I Gaucher Disease.

NCT00365131
View directory record Official record
Conditions
Gaucher Disease Type I, Cerebroside Lipidosis Syndrome, Clucocerebrosidase Deficiency Disease, Glucosylceramide Beta-Glucosidase Deficiency Disease, Gaucher Disease, Non-Neuronopathic Form
Interventions
Cerezyme (imiglucerase for injection)
Drug
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
10 Years to 65 Years
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1997 – 2004
U.S. locations
1
States / cities
Coral Springs, Florida
Source: ClinicalTrials.gov public record
Updated Mar 4, 2015 · Synced Sep 23, 2026, 12:40 PM EDT
Completed No phase listed Observational

Red Blood Cell - IMProving trAnsfusions for Chronically Transfused Recipients

NCT05255445
View directory record Official record
Conditions
Sickle Cell Disease, Thalassemia, Pediatric Cancer
Interventions
Red Blood Cell (RBC) Transfusion
Biological
Lead sponsor
Westat
Other
Eligibility
Not listed
Enrollment
157 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2024
U.S. locations
9
States / cities
Oakland, California • San Francisco, California • Boston, Massachusetts + 2 more
Source: ClinicalTrials.gov public record
Updated May 16, 2024 · Synced Sep 23, 2026, 12:40 PM EDT