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ClinicalTrials.gov public records Last synced Oct 21, 2026, 1:42 PM EDT

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

Showing 409–432 of 8,007 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Genetics/Birth Defects Clear all
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 21, 2026, 1:42 PM EDT
Approved For Marketing No phase listed Expanded access

Sapropterin Expanded Access Program

NCT00484991
View directory record Official record
Conditions
Phenylketonuria
Interventions
Sapropterin dihydrochloride
Drug
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
9 Years and older
U.S. locations
36
States / cities
Los Angeles, California • Orange, California • San Diego, California + 33 more
Source: ClinicalTrials.gov public record
Updated Apr 15, 2008 · Synced Oct 21, 2026, 1:42 PM EDT
Active, not recruiting Phase 1 Interventional

TGF-beta Resistant Cytotoxic T-lymphocytes in Treatment of EBV-positive Nasopharyngeal Carcinoma / RESIST-NPC

NCT02065362
View directory record Official record
Conditions
EBV-positive Nasopharyngeal Carcinoma
Interventions
DNR.NPC-specific T cells, DNR.NPC-specific T cells + cyclophosphamide + fludarabine
Biological
Lead sponsor
Baylor College of Medicine
Other
Eligibility
Not listed
Enrollment
14 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2033
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Jan 29, 2026 · Synced Oct 21, 2026, 1:42 PM EDT
Completed Phase 3 Interventional Results available

Study to Assess the Safety and Efficacy of Lebrikizumab (LY3650150) in Adolescent Participants With Moderate-to-Severe Atopic Dermatitis

NCT04250350
View directory record Official record
Conditions
Atopic Dermatitis
Interventions
Lebrikizumab
Biological
Lead sponsor
Eli Lilly and Company
Industry
Eligibility
12 Years to 17 Years
Enrollment
206 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2022
U.S. locations
48
States / cities
Anniston, Alabama • North Little Rock, Arkansas • Canoga Park, California + 38 more
Source: ClinicalTrials.gov public record
Updated Feb 24, 2023 · Synced Oct 21, 2026, 1:42 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Improving Scientific Rigor of Renal Clinical Endpoints for Sickle Cell Anemia

NCT04380610
View directory record Official record
Conditions
Sickle Cell Disease, Renal Disease, Glomerular Disease
Interventions
iohexol GFR
Diagnostic Test
Lead sponsor
University of Alabama at Birmingham
Other
Eligibility
5 Years to 50 Years
Enrollment
120 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2021 – 2025
U.S. locations
4
States / cities
Birmingham, Alabama • Chicago, Illinois • Memphis, Tennessee
Source: ClinicalTrials.gov public record
Updated Sep 18, 2025 · Synced Oct 21, 2026, 1:42 PM EDT
Completed Phase 4 Interventional Results available

Vitamin D and Prebiotics for Intestinal Health in Cystic Fibrosis

NCT04118010
View directory record Official record
Conditions
Cystic Fibrosis, Dysbiosis
Interventions
Vitamin D3, Placebo vitamin D3, Inulin, Placebo Inulin
Drug
Lead sponsor
Emory University
Other
Eligibility
18 Years and older
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2022
U.S. locations
1
States / cities
Atlanta, Georgia
Source: ClinicalTrials.gov public record
Updated Feb 7, 2024 · Synced Oct 21, 2026, 1:42 PM EDT
Completed Not applicable Interventional

Milk Beta Caseins on Digestive Discomfort, Gastric Emptying

NCT07884123
View directory record Official record
Conditions
Lactose Intolerants, Lactose Malabsorption, Caseins
Interventions
A1/A2 milk with oral naloxone, A1PF milk with oral naloxone
Other
Lead sponsor
Purdue University
Other
Eligibility
18 Years to 65 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2026
U.S. locations
1
States / cities
West Lafayette, Indiana
Source: ClinicalTrials.gov public record
Updated Oct 20, 2026 · Synced Oct 21, 2026, 1:42 PM 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 21, 2026, 1:42 PM EDT
Completed Not applicable Interventional Accepts healthy volunteers

Clinical Evaluation of Acuity 200 (Fluoroxyfocon A) RGP Contact Lens

NCT03971227
View directory record Official record
Conditions
Refractive Errors
Interventions
Acuity 200 (Fluoroxyfocon A) contact lens, Acuity 100 (Hexafocon A) contact lens
Device
Lead sponsor
Acuity Polymers, Inc.
Industry
Eligibility
18 Years and older
Enrollment
74 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2019 – 2020
U.S. locations
3
States / cities
Honolulu, Hawaii • Edina, Minnesota • Pittsford, New York
Source: ClinicalTrials.gov public record
Updated May 12, 2020 · Synced Oct 21, 2026, 1:42 PM EDT
Terminated No phase listed Observational Accepts healthy volunteers

Studies of Disorders in Antibody Production and Related Primary Immunodeficiency States

NCT00266513
View directory record Official record
Conditions
Hyper-IgM Syndrome, Ectodermal Dysplasia
Interventions
Not listed
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
Not listed
Enrollment
119 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2005 – 2013
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 6, 2017 · Synced Oct 21, 2026, 1:42 PM EDT
Completed No phase listed Observational

Genetic Regulation of Surfactant Deficiency

NCT00828243
View directory record Official record
Conditions
Respiratory Distress Syndrome, Newborn
Interventions
Nutrient
Drug
Lead sponsor
Washington University School of Medicine
Other
Eligibility
1 Day to 6 Months
Enrollment
525 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2013
U.S. locations
1
States / cities
St Louis, Missouri
Source: ClinicalTrials.gov public record
Updated Jun 7, 2021 · Synced Oct 21, 2026, 1:42 PM EDT
Recruiting No phase listed Observational

Long Term Follow-Up Study for Individuals With Hemoglobin Disorders After Hematopoietic Cell Transplant or Gene Therapy

NCT06646640
View directory record Official record
Conditions
Hemoglobin Disorder
Interventions
Not listed
Lead sponsor
St. Jude Children's Research Hospital
Other
Eligibility
Not listed
Enrollment
200 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2050
U.S. locations
1
States / cities
Memphis, Tennessee
Source: ClinicalTrials.gov public record
Updated Sep 15, 2026 · Synced Oct 21, 2026, 1:42 PM EDT
Completed Not applicable Interventional

Prader-Willi Syndrome Macronutrient Study

NCT02011360
View directory record Official record
Conditions
Prader Willi Syndrome, Syndromic Obesity, Childhood Obesity
Interventions
Low Carbohydrate diet, Low Fat diet
Other
Lead sponsor
Duke University
Other
Eligibility
5 Years to 17 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2016
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Dec 14, 2016 · Synced Oct 21, 2026, 1:42 PM EDT
Completed Phase 3 Interventional Results available

Safety/Effectiveness Study of Cysteamine Bitartrate Delayed-release Capsules (RP103) in Cysteamine Treatment Naive Patients With Cystinosis

NCT01744782
View directory record Official record
Conditions
Cystinosis
Interventions
RP103
Drug
Lead sponsor
Amgen
Industry
Eligibility
Up to 6 Years
Enrollment
17 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2016
U.S. locations
1
States / cities
Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Dec 27, 2024 · Synced Oct 21, 2026, 1:42 PM EDT
Completed Phase 4 Interventional Results available

A Prospective Study to Investigate Safety and Tolerability of Shorter Infusion of Fabrazyme

NCT06019728
View directory record Official record
Conditions
Fabry's Disease
Interventions
AGALSIDASE BETA (GZ419828), Acetaminophen, Diphenhydramine, Dexamethasone, Montelukast, Loratadine, Cetirizine, Fexofenadine
Drug
Lead sponsor
Sanofi
Industry
Eligibility
2 Years to 65 Years
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2024
U.S. locations
5
States / cities
Santa Monica, California • Atlanta, Georgia • Grand Rapids, Michigan + 2 more
Source: ClinicalTrials.gov public record
Updated Oct 21, 2025 · Synced Oct 21, 2026, 1:42 PM EDT
Terminated Phase 1Phase 2 Interventional Results available

Safety & Efficacy of Encapsulated Allogeneic FVIII Cell Therapy in Haemophilia A

NCT04541628
View directory record Official record
Conditions
Hemophilia A
Interventions
SIG-001
Combination Product
Lead sponsor
Eli Lilly and Company
Industry
Eligibility
18 Years and older · Male only
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2023
U.S. locations
3
States / cities
Indianapolis, Indiana • Boston, Massachusetts • Seattle, Washington
Source: ClinicalTrials.gov public record
Updated Sep 4, 2024 · Synced Oct 21, 2026, 1:42 PM EDT
Completed Phase 1 Interventional

A Phase I Safety, Pharmacokinetics and Pharmacodynamics Study of Recombinant Factor VIIa in Adult Patients With Hemophilia A or B

NCT01708564
View directory record Official record
Conditions
Hemophilia
Interventions
rhFVIIa
Biological
Lead sponsor
rEVO Biologics
Industry
Eligibility
18 Years to 75 Years · Male only
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2013
U.S. locations
2
States / cities
Sacramento, California • Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Jul 30, 2013 · Synced Oct 21, 2026, 1:42 PM EDT
Completed Phase 3 Interventional Results available

Efficacy and Safety Study of Prophylactic Versus On-Demand Treatment With Feiba NF in Subjects With Hemophilia A or B and a High Titer Inhibitor

NCT00851721
View directory record Official record
Conditions
Hemophilia A or B With Inhibitors, Hemophilia A, Hemophilia B
Interventions
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated)
Biological
Lead sponsor
Baxalta now part of Shire
Industry
Eligibility
4 Years to 65 Years
Enrollment
52 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2012
U.S. locations
2
States / cities
Chicago, Illinois • Cleveland, Ohio
Source: ClinicalTrials.gov public record
Updated May 19, 2021 · Synced Oct 21, 2026, 1:42 PM EDT
Completed Phase 4 Interventional

Safety Study in Subjects ≥ 12 Years of Age With Hereditary Angioedema Switching to Garadacimab

NCT06806657
View directory record Official record
Conditions
Hereditary Angioedema
Interventions
Garadacimab
Biological
Lead sponsor
CSL Behring
Industry
Eligibility
12 Years and older
Enrollment
18 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2026
U.S. locations
6
States / cities
Litchfield Park, Arizona • Bentonville, Arkansas • Orange, California + 3 more
Source: ClinicalTrials.gov public record
Updated Jul 23, 2026 · Synced Oct 21, 2026, 1:42 PM EDT
Not listed No phase listed Observational

Non-Invasive Screening for Fetal Aneuploidy

NCT02317965
View directory record Official record
Conditions
Down Syndrome, Edwards Syndrome
Interventions
Maternal Blood Draw
Other
Lead sponsor
Progenity, Inc.
Industry
Eligibility
18 Years to 54 Years · Female only
Enrollment
340 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2018
U.S. locations
10
States / cities
Campbell, California • Long Beach, California • Denver, Colorado + 6 more
Source: ClinicalTrials.gov public record
Updated Aug 28, 2018 · Synced Oct 21, 2026, 1:42 PM EDT
Withdrawn No phase listed Observational Accepts healthy volunteers

COVID-19 In-vitro Diagnostic Test and Androgen Receptor Gene Expression

NCT04530500
View directory record Official record
Conditions
Covid19, Androgen Receptor Gene Overexpression
Interventions
COVID-19 Androgen Sensitivity Test (CoVAST)
Genetic
Lead sponsor
University of California, Irvine
Other
Eligibility
18 Years to 90 Years · Male only
Healthy volunteers
Accepts healthy volunteers
Timeline
2020 – 2023
U.S. locations
2
States / cities
Irvine, California
Source: ClinicalTrials.gov public record
Updated May 8, 2024 · Synced Oct 21, 2026, 1:42 PM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

A Phase 1, Randomized, Open-Label, Two-Way Crossover Study To Evaluate The Steady-State Effect Of Dimebon (PF 01913539) On The Single-Dose Pharmacokinetics And Pharmacodynamics Of Warfarin In Healthy Subjects

NCT00827034
View directory record Official record
Conditions
Alzheimer's Disease, Huntington's Disease
Interventions
Warfarin, Dimebon
Drug
Lead sponsor
Pfizer
Industry
Eligibility
18 Years to 55 Years
Enrollment
14 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2009
U.S. locations
1
States / cities
New Haven, Connecticut
Source: ClinicalTrials.gov public record
Updated Oct 16, 2018 · Synced Oct 21, 2026, 1:42 PM EDT
Completed No phase listed Observational

Advanced Grandparental Age as a Risk Factor for Autism

NCT00464477
View directory record Official record
Conditions
Autistic Disorder, Pervasive Developmental Disorder, Asperger Syndrome, Childhood Disintegrative Disorder, Rett Syndrome
Interventions
Not listed
Lead sponsor
University of Mississippi Medical Center
Other
Eligibility
Not listed
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007
U.S. locations
1
States / cities
Jackson, Mississippi
Source: ClinicalTrials.gov public record
Updated Nov 7, 2007 · Synced Oct 21, 2026, 1:42 PM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

A Phase 1, First-in-human Study of VX-668

NCT05727800
View directory record Official record
Conditions
Alpha-1 Antitrypsin Deficiency
Interventions
VX-668, Placebo
Drug
Lead sponsor
Vertex Pharmaceuticals Incorporated
Industry
Eligibility
18 Years to 55 Years
Enrollment
114 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2023 – 2024
U.S. locations
5
States / cities
Tempe, Arizona • Gainesville, Florida • Orlando, Florida + 2 more
Source: ClinicalTrials.gov public record
Updated Mar 15, 2024 · Synced Oct 21, 2026, 1:42 PM EDT