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ClinicalTrials.gov public records Last synced Oct 23, 2026, 2:13 AM EDT

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Showing 841–864 of 8,007 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Genetics/Birth Defects Clear all
Completed Phase 3 Interventional

A Multicenter, Phase 3 Randomized, Double-Blind, Vehicle-Controlled Study Evaluating the Safety and Efficacy of QTORIN 3.9% Rapamycin Anhydrous Gel in the Treatment of Pachyonychia Congenita

NCT05180708
View directory record Official record
Conditions
Pachyonychia Congenita
Interventions
QTORIN 3.9% rapamycin anhydrous gel, Vehicle
Drug
Lead sponsor
Palvella Therapeutics, Inc.
Industry
Eligibility
18 Years and older
Enrollment
87 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2023
U.S. locations
7
States / cities
Palo Alto, California • New Haven, Connecticut • Orange Park, Florida + 4 more
Source: ClinicalTrials.gov public record
Updated Aug 29, 2024 · Synced Oct 23, 2026, 2:13 AM EDT
Enrolling by invitation Not applicable Interventional Accepts healthy volunteers

Rapid Whole Genome Sequencing Study

NCT03385876
View directory record Official record
Conditions
Genetic Diseases, Genetic Syndrome
Interventions
Genomic sequencing and molecular diagnostic results, if any
Genetic
Lead sponsor
Rady Pediatric Genomics & Systems Medicine Institute
Other
Eligibility
Not listed
Enrollment
100,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2017 – 2050
U.S. locations
1
States / cities
San Diego, California
Source: ClinicalTrials.gov public record
Updated Dec 23, 2021 · Synced Oct 23, 2026, 2:13 AM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

Combined Focused Ultrasound and Calcium Hydroxylapatitie Filler for Skin Laxity and Volume Restoration

NCT04176068
View directory record Official record
Conditions
Skin Laxity
Interventions
calcium hydroxylapatite (Radiesse) and IFUS (Ulthera)
Combination Product
Lead sponsor
University of California, Irvine
Other
Eligibility
18 Years to 85 Years
Enrollment
20 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2019 – 2021
U.S. locations
1
States / cities
Irvine, California
Source: ClinicalTrials.gov public record
Updated Feb 2, 2024 · Synced Oct 23, 2026, 2:13 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Mitochondrial Biomarkers in Huntington's Disease

NCT04515550
View directory record Official record
Conditions
Huntington Disease
Interventions
lumbar puncture
Diagnostic Test
Lead sponsor
University Hospitals Cleveland Medical Center
Other
Eligibility
20 Years to 85 Years
Enrollment
27 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2019 – 2022
U.S. locations
1
States / cities
Cleveland, Ohio
Source: ClinicalTrials.gov public record
Updated Apr 12, 2022 · Synced Oct 23, 2026, 2:13 AM 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 23, 2026, 2:13 AM EDT
Recruiting No phase listed Observational

Von Willebrand Factor in Pregnancy (VIP) Study

NCT04146376
View directory record Official record
Conditions
Von Willebrand Diseases
Interventions
Use of a postpartum diary and additional blood draws., VWF replacement therapy with Wilate, Tranexamic acid
Other · Drug
Lead sponsor
University of Washington
Other
Eligibility
18 Years and older · Female only
Enrollment
110 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2026
U.S. locations
11
States / cities
Aurora, Colorado • New Haven, Connecticut • Miami, Florida + 8 more
Source: ClinicalTrials.gov public record
Updated Dec 18, 2025 · Synced Oct 23, 2026, 2:13 AM EDT
Completed Phase 3 Interventional Results available

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

NCT05372419
View directory record Official record
Conditions
Atopic Dermatitis
Interventions
Lebrikizumab
Drug
Lead sponsor
Eli Lilly and Company
Industry
Eligibility
12 Years and older
Enrollment
90 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2025
U.S. locations
36
States / cities
Birmingham, Alabama • Fountain Valley, California • Fremont, California + 30 more
Source: ClinicalTrials.gov public record
Updated Mar 9, 2026 · Synced Oct 23, 2026, 2:13 AM EDT
Completed Phase 2 Interventional Results available

A Study to Evaluate the Anti-pruritic Effectiveness of ASN008 in Adults With Mild to Moderate Atopic Dermatitis

NCT05870865
View directory record Official record
Conditions
Dermatitis, Atopic, Pruritus
Interventions
ASN008, ASN008 Matching Vehicle
Drug · Other
Lead sponsor
Formation Bio, Inc.
Industry
Eligibility
18 Years and older
Enrollment
144 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023
U.S. locations
27
States / cities
Scottsdale, Arizona • Hot Springs, Arkansas • Beverly Hills, California + 23 more
Source: ClinicalTrials.gov public record
Updated May 16, 2025 · Synced Oct 23, 2026, 2:13 AM EDT
Not listed Phase 2 Interventional

Treatment of Mitochondrial Dysfunction in Rett Syndrome With Triheptanoin

NCT02696044
View directory record Official record
Conditions
Rett Syndrome
Interventions
triheptanoin
Drug
Lead sponsor
Center for Rare Neurological Diseases, Norcross, GA
Other
Eligibility
2 Years and older · Female only
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2022
U.S. locations
1
States / cities
Norcross, Georgia
Source: ClinicalTrials.gov public record
Updated Jul 17, 2020 · Synced Oct 23, 2026, 2:13 AM EDT
Completed Phase 1 Interventional

A Pharmacokinetic Study of Ruxolitinib Phosphate Cream in Pediatric Subjects With Atopic Dermatitis

NCT03257644
View directory record Official record
Conditions
Atopic Dermatitis
Interventions
Ruxolitinib phosphate cream
Drug
Lead sponsor
Incyte Corporation
Industry
Eligibility
2 Years to 17 Years
Enrollment
70 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2020
U.S. locations
23
States / cities
Hoover, Alabama • Gilbert, Arizona • Little Rock, Arkansas + 17 more
Source: ClinicalTrials.gov public record
Updated Nov 10, 2020 · Synced Oct 23, 2026, 2:13 AM EDT
Active, not recruiting No phase listed Observational

Alpha-1 Antitrypsin Disease Cohort: Longitudinal Biomarker Study of Disease

NCT05297812
View directory record Official record
Conditions
Alpha 1-Antitrypsin Deficiency, Genetic Disease
Interventions
Not listed
Lead sponsor
Columbia University
Other
Eligibility
18 Years and older
Enrollment
286 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2027
U.S. locations
9
States / cities
Birmingham, Alabama • Los Angeles, California • Denver, Colorado + 6 more
Source: ClinicalTrials.gov public record
Updated Jan 16, 2026 · Synced Oct 23, 2026, 2:13 AM EDT
Completed Phase 2 Interventional Results available

DHA and X-Linked Retinitis Pigmentosa

NCT00100230
View directory record Official record
Conditions
Retinitis Pigmentosa, X-linked Genetic Diseases
Interventions
docosahexaenoic acid OR corn/soy oil placebo
Drug
Lead sponsor
Retina Foundation of the Southwest
Other
Eligibility
7 Years to 32 Years · Male only
Enrollment
78 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2014
U.S. locations
1
States / cities
Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Mar 17, 2015 · Synced Oct 23, 2026, 2:13 AM EDT
Completed Phase 4 Interventional Results available

Saline vs. Lactated Ringers for Emergency Department IV Fluid Resuscitation

NCT03133767
View directory record Official record
Conditions
Fluid Loss, Symptom Complex, Triple, Emergencies
Interventions
Lactated Ringer Solution, Normal Saline 0.9% Infusion Solution Bag
Drug
Lead sponsor
Nicholas M Mohr
Other
Eligibility
18 Years and older
Enrollment
157 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017
U.S. locations
1
States / cities
Iowa City, Iowa
Source: ClinicalTrials.gov public record
Updated Sep 11, 2018 · Synced Oct 23, 2026, 2:13 AM EDT
Terminated Not applicable Interventional Accepts healthy volunteers

Evaluation of the Ulthera® System for Treatment of the Face and Neck

NCT01713907
View directory record Official record
Conditions
Wrinkles, Rhytids, Skin Laxity
Interventions
Ulthera® Treatment
Device
Lead sponsor
Ulthera, Inc
Industry
Eligibility
30 Years to 65 Years
Enrollment
15 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2011 – 2012
U.S. locations
1
States / cities
Rockville, Maryland
Source: ClinicalTrials.gov public record
Updated Nov 24, 2017 · Synced Oct 23, 2026, 2:13 AM EDT
Recruiting No phase listed Observational

Rocket Study: A Study to Characterize Biomarkers and Disease Progression in Participants With Pelizaeus-Merzbacher Disease

NCT05659901
View directory record Official record
Conditions
Pelizaeus-Merzbacher Disease
Interventions
Not listed
Lead sponsor
Ionis Pharmaceuticals, Inc.
Industry
Eligibility
6 Months to 17 Years · Male only
Enrollment
32 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2029
U.S. locations
3
States / cities
Atlanta, Georgia • Baltimore, Maryland • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Oct 2, 2026 · Synced Oct 23, 2026, 2:13 AM EDT
Not listed Phase 2 Interventional

A Randomized Trial of LOVAZA in Pediatric Sickle Cell Disease (SCD)

NCT01202812
View directory record Official record
Conditions
Sickle Cell Disease, HEMOGLOBIN SS, Hemoglobin S Beta-0 Thalassemia, Inflammation, Quality of Life
Interventions
Omega-3 Fatty Acids: Eicosapentaenoic Acid (EPA) and Docosahexaenoic Acid (DHA), Placebo Capsules
Dietary Supplement · Other
Lead sponsor
Thomas Jefferson University
Other
Eligibility
10 Years to 19 Years
Enrollment
48 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2012
U.S. locations
2
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Oct 25, 2010 · Synced Oct 23, 2026, 2:13 AM EDT
Recruiting No phase listed Observational

A Prospective Sub-Study of the Global Hypophosphatasia Registry

NCT05234567
View directory record Official record
Conditions
Hypophosphatasia
Interventions
Asfotase Alfa
Biological
Lead sponsor
Alexion Pharmaceuticals, Inc.
Industry
Eligibility
Not listed
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2028
U.S. locations
12
States / cities
Hartford, Connecticut • Chicago, Illinois • Boston, Massachusetts + 9 more
Source: ClinicalTrials.gov public record
Updated Feb 9, 2026 · Synced Oct 23, 2026, 2:13 AM EDT
Recruiting Phase 4 Interventional

Hemlibra in Mild Hemophilia A

NCT04567511
View directory record Official record
Conditions
Factor VIII Deficiency, Congenital
Interventions
Emicizumab
Drug
Lead sponsor
Indiana Hemophilia &Thrombosis Center, Inc.
Other
Eligibility
5 Years to 45 Years · Male only
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2027
U.S. locations
1
States / cities
Indianapolis, Indiana
Source: ClinicalTrials.gov public record
Updated Mar 15, 2024 · Synced Oct 23, 2026, 2:13 AM EDT
Active, not recruiting Phase 3 Interventional

An Efficacy, Safety, and Tolerability Study of VRDN-003 in Participants With Active Thyroid Eye Disease (TED)

NCT06625411
View directory record Official record
Conditions
Thyroid Eye Disease
Interventions
VRDN-003, Placebo
Drug
Lead sponsor
Viridian Therapeutics, Inc.
Industry
Eligibility
18 Years to 75 Years
Enrollment
132 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
34
States / cities
Scottsdale, Arizona • Canoga Park, California • Huntington Beach, California + 25 more
Source: ClinicalTrials.gov public record
Updated Aug 18, 2026 · Synced Oct 23, 2026, 2:13 AM EDT
Completed No phase listed Observational

The Classification and Cause of Leukodystrophies of Unknown Cause

NCT00001671
View directory record Official record
Conditions
Lysosomal Storage Disease
Interventions
Not listed
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
Not listed
Enrollment
400 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1997 – 2008
U.S. locations
2
States / cities
San Francisco, California • Washington D.C., District of Columbia
Source: ClinicalTrials.gov public record
Updated Jul 2, 2017 · Synced Oct 23, 2026, 2:13 AM EDT
Recruiting No phase listed Observational

Development of Patient-Reported Outcome Measures Assessing Tumor Visibility and Appearance Concerns in Neurofibromatosis Type 1: A Qualitative Study

NCT06880991
View directory record Official record
Conditions
Neurofibromatosis Type 1, Neurofibroma
Interventions
Arm 1
Other
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
5 Years to 120 Years
Enrollment
110 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2028
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 16, 2026 · Synced Oct 23, 2026, 2:13 AM EDT
Recruiting Not applicable Interventional

Well-Formulated Ketogenic Diet Polycystic Kidney Disease

NCT06325644
View directory record Official record
Conditions
Polycystic Kidney Disease
Interventions
Ketogenic Diet, Ketone/Glucose Monitoring, CGM/CKM, Blood Draw, Body Composition, Change in glomerular filtration rate (mg/mmol) via magnetic resonance imaging (MRI) .
Other · Biological
Lead sponsor
Ohio State University
Other
Eligibility
18 Years to 65 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2027
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated May 14, 2026 · Synced Oct 23, 2026, 2:13 AM EDT
Completed Not applicable Interventional

Modified Comprehensive Behavioral Intervention for Tics (M_CBIT)

NCT02900144
View directory record Official record
Conditions
Tourette Syndrome, ADHD
Interventions
Modified Comprehensive Behavioral Intervention for Tics, Comprehensive Behavioral Intervention for Tics
Behavioral
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
10 Years to 17 Years
Enrollment
17 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2019
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Sep 16, 2021 · Synced Oct 23, 2026, 2:13 AM EDT
Withdrawn Phase 2 Interventional

A Trial of Chronotherapy of Corticosteroids in Duchenne Muscular Dystrophy

NCT02036463
View directory record Official record
Conditions
Duchenne Muscular Dystrophy (DMD)
Interventions
Prednisone, Placebo
Drug
Lead sponsor
Ann & Robert H Lurie Children's Hospital of Chicago
Other
Eligibility
3 Years to 6 Years · Male only
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2015
U.S. locations
1
States / cities
Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Feb 18, 2015 · Synced Oct 23, 2026, 2:13 AM EDT