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 come from ClinicalTrials.gov. When the live registry is unavailable, a clearly labeled stored copy may be shown.

Clear filters

These results were retrieved from the live ClinicalTrials.gov registry. Filters and sort order apply to this result list.

ClinicalTrials.gov public records Last synced Oct 30, 2026, 11:16 AM EDT

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

Showing 1537–1560 of 8,019 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Genetics/Birth Defects Clear all
Completed Phase 1Phase 2 Interventional Results available

Safety and Efficacy of Clenbuterol in Individuals With Late-onset Pompe Disease and Receiving Enzyme Replacement Therapy

NCT01942590
View directory record Official record
Conditions
Pompe Disease
Interventions
Clenbuterol, Placebo
Drug
Lead sponsor
Dwight Koeberl, M.D., Ph.D.
Other
Eligibility
18 Years and older
Enrollment
17 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2016
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Jul 2, 2019 · Synced Oct 30, 2026, 11:16 AM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

A Study to Learn How Different Amounts of the Study Medicine Called PF-08049820 Are Tolerated and Act in the Body in Healthy Adults

NCT06686797
View directory record Official record
Conditions
Healthy
Interventions
PF-08049820, Placebo
Drug
Lead sponsor
Pfizer
Industry
Eligibility
18 Years to 65 Years
Enrollment
71 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2024 – 2026
U.S. locations
1
States / cities
New Haven, Connecticut
Source: ClinicalTrials.gov public record
Updated Sep 8, 2026 · Synced Oct 30, 2026, 11:16 AM EDT
Terminated Phase 1Phase 2 Interventional

A Study to Assess the Safety, Pharmacokinetics, and Pharmacodynamics of mRNA-3705 in Participants With Isolated Methylmalonic Acidemia

NCT04899310
View directory record Official record
Conditions
Methylmalonic Acidemia
Interventions
mRNA-3705, Placebo
Biological
Lead sponsor
ModernaTX, Inc.
Industry
Eligibility
1 Year and older
Enrollment
18 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2026
U.S. locations
3
States / cities
Los Angeles, California • Palo Alto, California • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated May 6, 2026 · Synced Oct 30, 2026, 11:16 AM EDT
Terminated No phase listed Observational

The Natural History of Mucolipidosis Type IV

NCT01067742
View directory record Official record
Conditions
Mucolipidosis Type IV
Interventions
Not listed
Lead sponsor
Baylor Research Institute
Other
Eligibility
1 Year to 64 Years
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2021
U.S. locations
1
States / cities
Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Jan 26, 2026 · Synced Oct 30, 2026, 11:16 AM EDT
Completed No phase listed Observational

Studies of Children With Metabolic and Other Genetic Disorders

NCT00025870
View directory record Official record
Conditions
Genetic Disorder, Metabolic Disease
Interventions
Not listed
Lead sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
NIH
Eligibility
Up to 18 Years
Enrollment
1,090 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2016
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Nov 4, 2019 · Synced Oct 30, 2026, 11:16 AM EDT
Active, not recruiting Phase 1 Interventional

Study for the Treatment for CLN7 Disease

NCT04737460
View directory record Official record
Conditions
CLN7
Interventions
AAV9/CLN7
Genetic
Lead sponsor
Benjamin Greenberg
Other
Eligibility
1 Year to 18 Years
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2029
U.S. locations
1
States / cities
Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Dec 31, 2025 · Synced Oct 30, 2026, 11:16 AM EDT
Active, not recruiting Not applicable Interventional

A Study About Antibody Levels and Biomarkers in the Blood in People With Late-onset Pompe Disease

NCT06150820
View directory record Official record
Conditions
Pompe Disease (Late-onset)
Interventions
No Intervention
Other
Lead sponsor
Astellas Gene Therapies
Industry
Eligibility
16 Years to 69 Years
Enrollment
119 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2027
U.S. locations
10
States / cities
Atlanta, Georgia • Kansas City, Kansas • Ann Arbor, Michigan + 7 more
Source: ClinicalTrials.gov public record
Updated Oct 8, 2026 · Synced Oct 30, 2026, 11:16 AM EDT
Completed Phase 3 Interventional Results available

A Study of NewGam, Human Immunoglobulin 10%, in Patients With Primary Immunodeficiency Diseases

NCT01012323
View directory record Official record
Conditions
Primary Immunodeficiency Diseases
Interventions
NewGam
Biological
Lead sponsor
Octapharma
Industry
Eligibility
2 Years to 75 Years
Enrollment
51 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2012
U.S. locations
7
States / cities
Irvine, California • Centennial, Colorado • Chicago, Illinois + 4 more
Source: ClinicalTrials.gov public record
Updated Mar 16, 2017 · Synced Oct 30, 2026, 11:16 AM EDT
Completed Phase 2 Interventional

Study of ATI-1777 in Patients 12 to 65 Years Old With Mild to Severe Atopic Dermatitis

NCT05432596
View directory record Official record
Conditions
Atopic Dermatitis
Interventions
ATI-1777 2.0% w/w, ATI-1777 1.0% w/w, ATI-1777 0.5% w/w, Vehicle
Drug
Lead sponsor
Aclaris Therapeutics, Inc.
Industry
Eligibility
12 Years to 65 Years
Enrollment
250 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2023
U.S. locations
32
States / cities
Birmingham, Alabama • Scottsdale, Arizona • Bryant, Arkansas + 29 more
Source: ClinicalTrials.gov public record
Updated Oct 26, 2024 · Synced Oct 30, 2026, 11:16 AM EDT
Not listed Phase 3 Interventional

Safety, Efficacy and Pharmacokinetics of GreenGene™ F to Previously Treated Patients With Severe Hemophilia A

NCT01619046
View directory record Official record
Conditions
Hemophilia A
Interventions
GreenGene™ F and an approved recombinant Factor VIII product, GreenGene™ F
Biological
Lead sponsor
Green Cross Corporation
Industry
Eligibility
12 Years and older
Enrollment
124 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2015
U.S. locations
10
States / cities
Little Rock, Arkansas • Los Angeles, California • Torrance, California + 7 more
Source: ClinicalTrials.gov public record
Updated Jul 3, 2014 · Synced Oct 30, 2026, 11:16 AM EDT
Recruiting No phase listed Observational

Rare Kidney Stone Consortium Biobank

NCT02026388
View directory record Official record
Conditions
Primary Hyperoxaluria, Dent Disease, APRT Deficiency, Cystinuria
Interventions
Not listed
Lead sponsor
Mayo Clinic
Other
Eligibility
Not listed
Enrollment
2,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2030
U.S. locations
1
States / cities
Rochester, Minnesota
Source: ClinicalTrials.gov public record
Updated Jul 22, 2026 · Synced Oct 30, 2026, 11:16 AM EDT
Recruiting Phase 1 Interventional

Nucleoside Therapy in Patients With Telomere Biology Disorders

NCT06817590
View directory record Official record
Conditions
Telomere Biology Disorders, Dyskeratosis Congenita, Revesz Syndrome, Hoyeraal Hreidarsson Syndrome, Telomere Biology Disorders With Bone Marrow Failure, Interstitial Lung Disease Due to Systemic Disease (Telomere Biology Disorder), Pulmonary Fibrosis, Familial (Telomere Biology Disorder)
Interventions
deoxycytidine, deoxythymidine
Drug
Lead sponsor
Suneet Agarwal
Other
Eligibility
1 Year to 70 Years
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2029
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Aug 4, 2026 · Synced Oct 30, 2026, 11:16 AM EDT
Active, not recruiting No phase listed Observational

International Bicuspid Aortic Valve Consortium (BAVCon)

NCT01980797
View directory record Official record
Conditions
Bicuspid Aortic Valve Disease, Thoracic Aortic Disease in Patients With a Bicuspid Aortic Valve
Interventions
Not listed
Lead sponsor
Boston University
Other
Eligibility
8 Years to 90 Years
Enrollment
6,464 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2028
U.S. locations
5
States / cities
Boston, Massachusetts • Ann Arbor, Michigan • Rochester, Minnesota + 1 more
Source: ClinicalTrials.gov public record
Updated Apr 1, 2026 · Synced Oct 30, 2026, 11:16 AM EDT
Terminated Not applicable Interventional

Hematopoietic Stem Cell Transplant for High Risk Hemoglobinopathies

NCT02179359
View directory record Official record
Conditions
Sickle Cell Disease, Transfusion Dependent Alpha- or Beta- Thalassemia, Diamond Blackfan Anemia, Paroxysmal Nocturnal Hemoglobinuria, Glanzmann Thrombasthenia, Severe Congenital Neutropenia, Shwachman-Diamond Syndrome, Non-Malignant Hematologic Disorders
Interventions
Reduced Toxicity Ablative Regimen, Reduced Intensity Preparative Regimen, Myeloablative Preparative Regimen
Drug
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
Up to 55 Years
Enrollment
38 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2024
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Mar 3, 2025 · Synced Oct 30, 2026, 11:16 AM EDT
Withdrawn Not applicable Interventional

National Wilm's Tumor Study Late Effects

NCT00228696
View directory record Official record
Conditions
Wilm's Tumor
Interventions
none, screening only
Other
Lead sponsor
Emory University
Other
Eligibility
18 Years to 85 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2011
U.S. locations
1
States / cities
Atlanta, Georgia
Source: ClinicalTrials.gov public record
Updated Nov 26, 2013 · Synced Oct 30, 2026, 11:16 AM EDT
Recruiting No phase listed Observational

Rare CFTR Mutation Cell Collection Protocol (RARE)

NCT03161808
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
Not listed
Lead sponsor
George Solomon
Other
Eligibility
17 Years and older
Enrollment
500 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2028
U.S. locations
1
States / cities
Birmingham, Alabama
Source: ClinicalTrials.gov public record
Updated May 29, 2026 · Synced Oct 30, 2026, 11:16 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Genetic Risk: Whether, When, and How to Tell Adolescents

NCT03421327
View directory record Official record
Conditions
Huntington Disease, Hereditary Breast and Ovarian Cancer, Hereditary Cancer, Hereditary Non-polyposis Colon Cancer, Hereditary Non-Polyposis Colorectal Cancer Syndrome
Interventions
Not listed
Lead sponsor
Johns Hopkins University
Other
Eligibility
15 Years and older
Enrollment
85 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2017 – 2018
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Feb 7, 2019 · Synced Oct 30, 2026, 11:16 AM EDT
Not listed Phase 1 Interventional

A Clinical Trial to Evaluate the Safety of RP-L102 in Pediatric Subjects With Fanconi Anemia Subtype A

NCT03814408
View directory record Official record
Conditions
Fanconi Anemia Complementation Group A
Interventions
RP-L102
Biological
Lead sponsor
Rocket Pharmaceuticals Inc.
Industry
Eligibility
1 Year to 12 Years
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2022
U.S. locations
1
States / cities
Stanford, California
Source: ClinicalTrials.gov public record
Updated Nov 24, 2020 · Synced Oct 30, 2026, 11:16 AM EDT
Completed Not applicable Interventional

An Exploratory Study to Evaluate a Digital Intervention to Disrupt Scratching in Atopic Dermatitis and Psoriasis

NCT06275659
View directory record Official record
Conditions
Psoriasis, Atopic Dermatitis
Interventions
CT-100-004-A, CT-100-004-B
Device
Lead sponsor
Click Therapeutics, Inc.
Industry
Eligibility
18 Years and older
Enrollment
120 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2024
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Apr 30, 2025 · Synced Oct 30, 2026, 11:16 AM EDT
Completed No phase listed Observational

The Natural History of Infantile Globoid Cell Leukodystrophy

NCT00983879
View directory record Official record
Conditions
Infantile Globoid Cell Leukodystrophy
Interventions
Not listed
Lead sponsor
Zymenex A/S
Industry
Eligibility
Up to 2 Years
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2014
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Mar 8, 2018 · Synced Oct 30, 2026, 11:16 AM EDT
Completed Phase 2 Interventional Results available

Safety and Efficacy Study of NBI-98854 in Children and Adolescents With Tourette Syndrome

NCT02679079
View directory record Official record
Conditions
Tourette Syndrome
Interventions
NBI-98854, Placebo
Drug
Lead sponsor
Neurocrine Biosciences
Industry
Eligibility
6 Years to 17 Years
Enrollment
98 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2017
U.S. locations
44
States / cities
Sun City, Arizona • Springdale, Arkansas • Long Beach, California + 41 more
Source: ClinicalTrials.gov public record
Updated Jan 15, 2021 · Synced Oct 30, 2026, 11:16 AM EDT
Completed Phase 1 Interventional

The Role of Endothelin-1 in Sickle Cell Disease

NCT02712346
View directory record Official record
Conditions
Sickle Cell Anemia
Interventions
Ambrisentan, Placebo
Drug
Lead sponsor
Augusta University
Other
Eligibility
18 Years to 65 Years
Enrollment
26 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2019
U.S. locations
1
States / cities
Augusta, Georgia
Source: ClinicalTrials.gov public record
Updated Apr 2, 2020 · Synced Oct 30, 2026, 11:16 AM EDT
Completed Phase 2 Interventional Results available

MND-ADA Transduction of CD34+ Cells From Children With ADA-SCID

NCT00794508
View directory record Official record
Conditions
Severe Combined Immunodeficiency
Interventions
ADA gene transfer
Biological
Lead sponsor
Donald B. Kohn, M.D.
Other
Eligibility
1 Month to 18 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2015
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Apr 23, 2021 · Synced Oct 30, 2026, 11:16 AM EDT
Completed Not applicable Interventional Results available

Gait in Children With Down Syndrome While Wearing Orthoses

NCT01598350
View directory record Official record
Conditions
Down Syndrome
Interventions
Supramalleolar Orthoses (Cascade)
Device
Lead sponsor
University of Puget Sound
Other
Eligibility
2 Years to 10 Years
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2011
U.S. locations
1
States / cities
Tacoma, Washington
Source: ClinicalTrials.gov public record
Updated Dec 30, 2013 · Synced Oct 30, 2026, 11:16 AM EDT