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ClinicalTrials.gov public records Last synced Oct 22, 2026, 4:55 AM EDT

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Showing 529–552 of 8,007 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Genetics/Birth Defects Clear all
Completed Phase 2 Interventional

A Study of Lebrikizumab in Participants With Persistent Moderate to Severe Atopic Dermatitis

NCT02340234
View directory record Official record
Conditions
Atopic Dermatitis
Interventions
Lebrikizumab, Placebo, TCS Cream
Drug
Lead sponsor
Hoffmann-La Roche
Industry
Eligibility
18 Years to 75 Years
Enrollment
212 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2016
U.S. locations
16
States / cities
Los Angeles, California • San Diego, California • San Francisco, California + 13 more
Source: ClinicalTrials.gov public record
Updated Oct 2, 2017 · Synced Oct 22, 2026, 4:55 AM EDT
Terminated Phase 2 Interventional Results available

A Study to Evaluate Efficacy and Safety of VX-864 in Participants With the PiZZ Genotype

NCT05643495
View directory record Official record
Conditions
Alpha-1 Antitrypsin Deficiency
Interventions
VX-864
Drug
Lead sponsor
Vertex Pharmaceuticals Incorporated
Industry
Eligibility
18 Years to 80 Years
Enrollment
14 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2024
U.S. locations
10
States / cities
Gainesville, Florida • Orlando, Florida • Iowa City, Iowa + 7 more
Source: ClinicalTrials.gov public record
Updated Sep 8, 2025 · Synced Oct 22, 2026, 4:55 AM EDT
Completed Phase 3 Interventional

Three Times Weekly (TIW) Growth Hormone Therapy in Children on Hemodialysis

NCT00943995
View directory record Official record
Conditions
Kidney Failure, Chronic, Renal Dialysis
Interventions
somatropin
Drug
Lead sponsor
Nationwide Children's Hospital
Other
Eligibility
1 Month to 16 Years
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2012
U.S. locations
5
States / cities
Atlanta, Georgia • Kansas City, Missouri • The Bronx, New York + 1 more
Source: ClinicalTrials.gov public record
Updated May 5, 2015 · Synced Oct 22, 2026, 4:55 AM EDT
Completed Phase 3 Interventional Results available

Hydroxyurea to Prevent Organ Damage in Children With Sickle Cell Anemia

NCT00006400
View directory record Official record
Conditions
Hematologic Diseases, Anemia, Sickle Cell
Interventions
Hydroxyurea, Placebo
Drug
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
9 Months to 18 Months
Enrollment
193 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2000 – 2009
U.S. locations
14
States / cities
Birmingham, Alabama • Washington D.C., District of Columbia • Miami, Florida + 10 more
Source: ClinicalTrials.gov public record
Updated Aug 19, 2020 · Synced Oct 22, 2026, 4:55 AM EDT
Completed Phase 1 Interventional

Seneca Valley Virus-001 and Cyclophosphamide in Treating Young Patients With Relapsed or Refractory Neuroblastoma, Rhabdomyosarcoma, or Rare Tumors With Neuroendocrine Features

NCT01048892
View directory record Official record
Conditions
Adrenocortical Carcinoma, Gastrointestinal Carcinoid Tumor, Kidney Cancer, Neuroblastoma, Retinoblastoma, Sarcoma
Interventions
Seneca Valley virus-001, cyclophosphamide, laboratory biomarker analysis, pharmacological study
Biological · Drug · Other
Lead sponsor
Children's Oncology Group
Network
Eligibility
3 Years to 21 Years
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2009
U.S. locations
18
States / cities
Birmingham, Alabama • Orange, California • San Francisco, California + 15 more
Source: ClinicalTrials.gov public record
Updated Jan 30, 2014 · Synced Oct 22, 2026, 4:55 AM EDT
Completed Not applicable Interventional

Pilot Study to Evaluate a Voice Journaling Application in Adolescents With Cystic Fibrosis

NCT05420844
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
Kintsugi voice journaling application
Other
Lead sponsor
University of Colorado, Denver
Other
Eligibility
14 Years to 18 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2024
U.S. locations
1
States / cities
Aurora, Colorado
Source: ClinicalTrials.gov public record
Updated Nov 13, 2025 · Synced Oct 22, 2026, 4:55 AM EDT
Completed Not applicable Interventional Accepts healthy volunteers

Motiv82Change: an HIV Prevention Study for Young Latino Men

NCT00877695
View directory record Official record
Conditions
HIV Infections
Interventions
Entre Culturas: Navigating Culture, Identity, and HIV Risk
Behavioral
Lead sponsor
Nova Southeastern University
Other
Eligibility
18 Years to 29 Years · Male only
Enrollment
200 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2010 – 2012
U.S. locations
1
States / cities
Miami, Florida
Source: ClinicalTrials.gov public record
Updated Mar 4, 2013 · Synced Oct 22, 2026, 4:55 AM EDT
Recruiting Phase 2 Interventional

A Study to Evaluate the Efficacy and Safety of IMG-007 in Adult Participants With Moderate-to-Severe Atopic Dermatitis

NCT07037901
View directory record Official record
Conditions
Atopic Dermatitis, Atopic Dermatitis Eczema, Eczema
Interventions
IMG-007, Placebo
Drug
Lead sponsor
Inmagene LLC
Industry
Eligibility
18 Years to 74 Years
Enrollment
405 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2028
U.S. locations
16
States / cities
Lancaster, California • Los Angeles, California • Ocala, Florida + 13 more
Source: ClinicalTrials.gov public record
Updated Aug 12, 2026 · Synced Oct 22, 2026, 4:55 AM EDT
Recruiting No phase listed Observational

Nutritional Impacts of Palynziq on Patients With Phenylketonuria (PKU)

NCT04404530
View directory record Official record
Conditions
Phenylketonurias
Interventions
Palynziq
Drug
Lead sponsor
Emory University
Other
Eligibility
16 Years and older
Enrollment
45 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2028
U.S. locations
2
States / cities
Atlanta, Georgia
Source: ClinicalTrials.gov public record
Updated Jan 14, 2026 · Synced Oct 22, 2026, 4:55 AM EDT
Completed Not applicable Interventional Accepts healthy volunteers

Feasibility Study: Lifting and Tightening Neck Skin in Patients

NCT01708928
View directory record Official record
Conditions
Skin Laxity
Interventions
Ulthera System Treatment
Device
Lead sponsor
Ulthera, Inc
Industry
Eligibility
30 Years to 65 Years
Enrollment
30 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2010 – 2012
U.S. locations
1
States / cities
Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Nov 24, 2017 · Synced Oct 22, 2026, 4:55 AM EDT
Completed Phase 2 Interventional Results available

Vaccine Responses in Tralokinumab-Treated Atopic Dermatitis - ECZTRA 5 (ECZema TRAlokinumab Trial No. 5)

NCT03562377
View directory record Official record
Conditions
Atopic Dermatitis
Interventions
Tralokinumab, Placebo, Tdap vaccine, Meningococcal vaccine
Drug · Biological
Lead sponsor
LEO Pharma
Industry
Eligibility
18 Years to 54 Years
Enrollment
215 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2019
U.S. locations
39
States / cities
Fort Smith, Arkansas • Bakersfield, California • Beverly Hills, California + 33 more
Source: ClinicalTrials.gov public record
Updated Mar 11, 2025 · Synced Oct 22, 2026, 4:55 AM EDT
Completed Phase 3 Interventional Results available

Roll-Over Study of Ivacaftor in Cystic Fibrosis Pediatric Subjects With a CF Transmembrane Conductance Regulator Gene (CFTR) Gating Mutation

NCT01946412
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
Ivacaftor
Drug
Lead sponsor
Vertex Pharmaceuticals Incorporated
Industry
Eligibility
2 Years and older
Enrollment
33 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2015
U.S. locations
12
States / cities
Birmingham, Alabama • Denver, Colorado • Atlanta, Georgia + 9 more
Source: ClinicalTrials.gov public record
Updated Feb 1, 2017 · Synced Oct 22, 2026, 4:55 AM EDT
Active, not recruiting No phase listed Observational

MElanoma Research Lymph Node Prediction Implementation National_001

NCT04759781
View directory record Official record
Conditions
Melanoma
Interventions
Not listed
Lead sponsor
SkylineDx
Industry
Eligibility
18 Years and older
Enrollment
1,820 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2029
U.S. locations
9
States / cities
Tampa, Florida • Atlanta, Georgia • Lexington, Kentucky + 6 more
Source: ClinicalTrials.gov public record
Updated Apr 23, 2026 · Synced Oct 22, 2026, 4:55 AM EDT
Recruiting No phase listed Observational

Long-term Follow-up (LTFU) Study of Participants in Any iECURE Protocol Using an Investigational Product (IP)

NCT06805695
View directory record Official record
Conditions
Ornithine Transcarbamylase Deficiency, Ornithine Transcarbamylase Deficiency Disease, Urea Cycle Disorders, Inborn
Interventions
No Intervention
Other
Lead sponsor
iECURE, Inc.
Industry
Eligibility
7 Months to 15 Months
Enrollment
13 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2041
U.S. locations
2
States / cities
Aurora, Colorado • New York, New York
Source: ClinicalTrials.gov public record
Updated Aug 31, 2026 · Synced Oct 22, 2026, 4:55 AM EDT
Completed No phase listed Observational

Adaptation Among Adolescents and Adults With Klinefelter Syndrome

NCT00896272
View directory record Official record
Conditions
Klinefelter Syndrome
Interventions
Not listed
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
14 Years to 80 Years · Male only
Enrollment
302 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2015
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Dec 17, 2019 · Synced Oct 22, 2026, 4:55 AM EDT
Terminated No phase listed Observational

Disease Natural History and Biomarkers of SPG3A, SPG4A, and SPG31

NCT02859428
View directory record Official record
Conditions
Hereditary Spastic Paraplegia
Interventions
Not listed
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
7 Years and older
Enrollment
51 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2020
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 20, 2020 · Synced Oct 22, 2026, 4:55 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 22, 2026, 4:55 AM EDT
Completed Phase 2 Interventional

A Study of the Efficacy and Safety of ICA-17043 (With or Without Hydroxyurea) in Patients With Sickle Cell Anemia.

NCT00040677
View directory record Official record
Conditions
Sickle Cell Disease, Sickle Cell Anemia
Interventions
Low Dose ICA-17043, High dose ICA-17043, Placebo
Drug
Lead sponsor
Icagen
Industry
Eligibility
18 Years to 60 Years
Enrollment
90 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2002 – 2004
U.S. locations
19
States / cities
Birmingham, Alabama • Oakland, California • San Francisco, California + 16 more
Source: ClinicalTrials.gov public record
Updated Jul 18, 2011 · Synced Oct 22, 2026, 4:55 AM EDT
Completed Phase 3 Interventional Results available

An Study to Evaluate Rosuvastatin in Children and Adolescents With Familial Hypercholesterolaemia

NCT01078675
View directory record Official record
Conditions
Familial Hypercholesterolaemia
Interventions
rosuvastatin calcium
Drug
Lead sponsor
AstraZeneca
Industry
Eligibility
6 Years to 17 Years
Enrollment
315 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2013
U.S. locations
1
States / cities
Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Apr 7, 2015 · Synced Oct 22, 2026, 4:55 AM EDT
Completed Phase 1Phase 2 Interventional

A Phase I/II Study of BGT226 in Adult Patients With Advanced Solid Malignancies Including Patients With Advanced Breast Cancer

NCT00600275
View directory record Official record
Conditions
Solid Tumors, Breast Cancer, Cowden Syndrome
Interventions
BGT226
Drug
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
18 Years and older
Enrollment
57 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2007
U.S. locations
4
States / cities
Boston, Massachusetts • Las Vegas, Nevada • San Antonio, Texas
Source: ClinicalTrials.gov public record
Updated Dec 17, 2020 · Synced Oct 22, 2026, 4:55 AM EDT
Withdrawn No phase listed Observational Accepts healthy volunteers

Stem Cells in NF1 Patients With Tumors of the Central Nervous System

NCT03332030
View directory record Official record
Conditions
Neurofibromatosis Type 1, Tumors of the Central Nervous System
Interventions
Collection of Stem Cells
Diagnostic Test
Lead sponsor
Roger Packer
Other
Eligibility
Not listed
Healthy volunteers
Accepts healthy volunteers
Timeline
2015 – 2025
U.S. locations
1
States / cities
Washington D.C., District of Columbia
Source: ClinicalTrials.gov public record
Updated Feb 7, 2024 · Synced Oct 22, 2026, 4:55 AM EDT
Terminated Phase 3 Interventional Results available

Study of Ataluren in Previously Treated Participants With Nonsense Mutation Dystrophinopathy (nmDBMD)

NCT01247207
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Ataluren
Drug
Lead sponsor
PTC Therapeutics
Industry
Eligibility
Male only
Enrollment
265 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2026
U.S. locations
29
States / cities
Phoenix, Arizona • Los Angeles, California • Palo Alto, California + 25 more
Source: ClinicalTrials.gov public record
Updated Oct 8, 2026 · Synced Oct 22, 2026, 4:55 AM EDT
Completed No phase listed Observational

Viral Pathogenesis of Early Cystic Fibrosis Lung Disease

NCT01973192
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
Not listed
Lead sponsor
Indiana University
Other
Eligibility
2 Months to 4 Months
Enrollment
65 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2016
U.S. locations
2
States / cities
Indianapolis, Indiana • St Louis, Missouri
Source: ClinicalTrials.gov public record
Updated Apr 4, 2017 · Synced Oct 22, 2026, 4:55 AM EDT
Recruiting Phase 1 Interventional

A Phase 1, Dose Escalation Trial of RP-A601 in Subjects With PKP2 Variant-Mediated Arrhythmogenic Cardiomyopathy (PKP2-ACM)

NCT05885412
View directory record Official record
Conditions
PKP2 Arrhythmogenic Cardiomyopathy (PKP2-ACM)
Interventions
RP-A601
Genetic
Lead sponsor
Rocket Pharmaceuticals Inc.
Industry
Eligibility
18 Years and older
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2029
U.S. locations
3
States / cities
La Jolla, California • Durham, North Carolina • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Apr 16, 2026 · Synced Oct 22, 2026, 4:55 AM EDT