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ClinicalTrials.gov public records Last synced Oct 22, 2026, 5:31 AM EDT

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

Showing 1–24 of 2,434 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Genetic Therapy Clear all
Recruiting No phase listed Observational

Registry for Systemic Eczema Treatments

NCT06136767
View directory record Official record
Conditions
Atopic Dermatitis
Interventions
Not listed
Lead sponsor
Johns Hopkins University
Other
Eligibility
1 Year to 26 Years
Enrollment
400 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2030
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Apr 7, 2026 · Synced Oct 22, 2026, 5:31 AM EDT
Completed Not applicable Interventional Accepts healthy volunteers

Treatment for Improvement of Cellulite Appearance Using Form Applicator

NCT05882721
View directory record Official record
Conditions
Cellulite
Interventions
Sofwave
Device
Lead sponsor
Sofwave Medical LTD
Industry
Eligibility
18 Years to 60 Years · Female only
Enrollment
60 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2023
U.S. locations
5
States / cities
Sacramento, California • Hackensack, New Jersey • New York, New York + 2 more
Source: ClinicalTrials.gov public record
Updated Nov 18, 2023 · Synced Oct 22, 2026, 5:31 AM EDT
Completed Phase 2 Interventional

Pilot Study For Hypothermia Treatment In Hyperammonemic Encephalopathy In Neonates And Very Young Infants

NCT01624311
View directory record Official record
Conditions
Urea Cycle Disorders, Organic Acidemias
Interventions
Therapeutic Hypothermia, Standard of Care Therapy
Other
Lead sponsor
Uta Lichter-Konecki
Other
Eligibility
1 Day to 30 Days
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2015
U.S. locations
3
States / cities
Washington D.C., District of Columbia • New York, New York • Milwaukee, Wisconsin
Source: ClinicalTrials.gov public record
Updated Jun 1, 2015 · Synced Oct 22, 2026, 5:31 AM EDT
Terminated Phase 2Phase 3 Interventional Results available

Trial of Netarsudil for Acceleration of Corneal Endothelial Restoration

NCT03971357
View directory record Official record
Conditions
Fuchs' Endothelial Dystrophy
Interventions
Netarsudil, Placebo
Drug
Lead sponsor
Price Vision Group
Industry
Eligibility
18 Years and older
Enrollment
19 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2021
U.S. locations
1
States / cities
Indianapolis, Indiana
Source: ClinicalTrials.gov public record
Updated Feb 6, 2023 · Synced Oct 22, 2026, 5:31 AM EDT
Terminated Phase 1Phase 2 Interventional Results available

Glycerol Phenylbutyrate Corrector Therapy For CF (Cystic Fibrosis)

NCT02323100
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
Ravicti low dose, Placebo
Drug
Lead sponsor
National Jewish Health
Other
Eligibility
18 Years and older
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2022
U.S. locations
3
States / cities
Denver, Colorado • Baltimore, Maryland • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Apr 17, 2026 · Synced Oct 22, 2026, 5:31 AM EDT
Recruiting No phase listed Observational

INTERogating Cancer for Etiology, Prevention and Therapy Navigation

NCT06008392
View directory record Official record
Conditions
Cancer, Cancer Gene Mutation, PAN Gene Mutation, Hematopoietic and Lymphoid System Neoplasm, Malignant Solid Neoplasm
Interventions
Pan-genomic Testing
Genetic
Lead sponsor
Mayo Clinic
Other
Eligibility
18 Years and older
Enrollment
500 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2033
U.S. locations
5
States / cities
Scottsdale, Arizona • Jacksonville, Florida • Rochester, Minnesota + 2 more
Source: ClinicalTrials.gov public record
Updated Oct 16, 2026 · Synced Oct 22, 2026, 5:31 AM EDT
Completed No phase listed Observational

iCAT for Recurrent/Refractory/HR Solid Tumors

NCT01853345
View directory record Official record
Conditions
Pediatric Solid Tumor, Sarcoma, Neuroblastoma, Wilms Tumor
Interventions
Not listed
Lead sponsor
Dana-Farber Cancer Institute
Other
Eligibility
Up to 30 Years
Enrollment
101 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2012
U.S. locations
5
States / cities
San Francisco, California • Washington D.C., District of Columbia • Boston, Massachusetts + 1 more
Source: ClinicalTrials.gov public record
Updated Feb 10, 2015 · Synced Oct 22, 2026, 5:31 AM EDT
Recruiting Not applicable Interventional

Treatment of Obstructive Sleep Apnea With Personalized Surgery in Children With Down Syndrome (TOPS-DS)

NCT05508971
View directory record Official record
Conditions
Obstructive Sleep Apnea, Down Syndrome
Interventions
DISE-Directed Surgery, Adenotonsillectomy
Procedure
Lead sponsor
Oregon Health and Science University
Other
Eligibility
2 Years to 17 Years
Enrollment
304 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2028
U.S. locations
7
States / cities
Aurora, Colorado • Ann Arbor, Michigan • Cincinnati, Ohio + 4 more
Source: ClinicalTrials.gov public record
Updated Sep 11, 2026 · Synced Oct 22, 2026, 5:31 AM EDT
Not listed Phase 2Phase 3 Interventional

N-Carbamylglutamate (Carbaglu) In The Treatment Of Hyperammonemia

NCT00843921
View directory record Official record
Conditions
Inborn Errors of Metabolism
Interventions
N-carbamylglutamate
Drug
Lead sponsor
Mendel Tuchman
Other
Eligibility
1 Day to 70 Years
Enrollment
52 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2020
U.S. locations
1
States / cities
Washington D.C., District of Columbia
Source: ClinicalTrials.gov public record
Updated Feb 17, 2020 · Synced Oct 22, 2026, 5:31 AM EDT
Active, not recruiting Phase 2Phase 3 Interventional Results available

SGLT2 Inhibitor Therapy in Cystic Fibrosis-related Diabetes

NCT06149793
View directory record Official record
Conditions
Cystic Fibrosis-related Diabetes, Cystic Fibrosis
Interventions
Empagliflozin, Placebo Control
Drug · Other
Lead sponsor
University of Minnesota
Other
Eligibility
18 Years and older
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Sep 21, 2026 · Synced Oct 22, 2026, 5:31 AM EDT
Completed Not applicable Interventional

Response Inhibition Training for Children With Williams Syndrome

NCT02212314
View directory record Official record
Conditions
Williams Syndrome
Interventions
Response Inhibition Training
Behavioral
Lead sponsor
University of Wisconsin, Milwaukee
Other
Eligibility
10 Years to 17 Years
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2016
U.S. locations
1
States / cities
Milwaukee, Wisconsin
Source: ClinicalTrials.gov public record
Updated May 31, 2017 · Synced Oct 22, 2026, 5:31 AM EDT
Completed Phase 3 Interventional

Genetic Counseling or Usual Care in Helping Women With Newly Diagnosed Ductal Carcinoma In Situ or Stage I, Stage II, or Stage IIIA Breast Cancer Make Treatment Decisions

NCT00262899
View directory record Official record
Conditions
Breast Cancer, Psychosocial Effects of Cancer and Its Treatment
Interventions
counseling intervention, educational intervention, psychosocial assessment and care
Behavioral · Other · Procedure
Lead sponsor
Georgetown University
Other
Eligibility
18 Years and older · Female only
Enrollment
331 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2011
U.S. locations
4
States / cities
Washington D.C., District of Columbia • Chevy Chase, Maryland • Hackensack, New Jersey + 1 more
Source: ClinicalTrials.gov public record
Updated Jan 7, 2013 · Synced Oct 22, 2026, 5:31 AM EDT
Active, not recruiting Not applicable Interventional Accepts healthy volunteers

In Vivo Study to Evaluate Tissue Remodeling Via Fibroblast Stimulation Following a Single SofWave Treatment

NCT07685821
View directory record Official record
Conditions
Lax Skin, Improve Facial Wrinkles
Interventions
Sofwave
Device
Lead sponsor
Sofwave Medical LTD
Industry
Eligibility
35 Years to 60 Years
Enrollment
10 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2025 – 2026
U.S. locations
1
States / cities
San Diego, California
Source: ClinicalTrials.gov public record
Updated Jul 8, 2026 · Synced Oct 22, 2026, 5:31 AM EDT
Completed No phase listed Observational

Resynchronization Therapy in Young Patients With and Without CHD

NCT00208806
View directory record Official record
Conditions
Dilated Cardiomyopathy, Pediatric
Interventions
congestive heart failure patients
Other
Lead sponsor
Emory University
Other
Eligibility
1 Day to 18 Years
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2006
U.S. locations
1
States / cities
Atlanta, Georgia
Source: ClinicalTrials.gov public record
Updated Dec 10, 2013 · Synced Oct 22, 2026, 5:31 AM EDT
Recruiting Phase 1Phase 2 Interventional

Study to Evaluate ACDN-01 in ABCA4-related Stargardt Retinopathy (STELLAR)

NCT06467344
View directory record Official record
Conditions
Stargardt Disease, Cone Rod Dystrophy, Juvenile Macular Degeneration, Stargardt Disease 1
Interventions
ACDN-01
Drug
Lead sponsor
Ascidian Therapeutics, Inc
Industry
Eligibility
12 Years and older
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2030
U.S. locations
11
States / cities
San Francisco, California • Gainesville, Florida • Miami, Florida + 8 more
Source: ClinicalTrials.gov public record
Updated Oct 7, 2026 · Synced Oct 22, 2026, 5:31 AM EDT
Completed Phase 3 Interventional Results available

Comparison of Two Medications Aimed at Slowing Aortic Root Enlargement in Individuals With Marfan Syndrome

NCT00429364
View directory record Official record
Conditions
Marfan Syndrome
Interventions
Losartan Potassium, Atenolol
Drug
Lead sponsor
Carelon Research
Other
Eligibility
6 Months to 25 Years
Enrollment
608 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2014
U.S. locations
24
States / cities
Los Angeles, California • Palo Alto, California • San Diego, California + 18 more
Source: ClinicalTrials.gov public record
Updated Mar 7, 2025 · Synced Oct 22, 2026, 5:31 AM EDT
Recruiting No phase listed Observational

Next Generation Sequencing of Normal Tissues Prospectively in Pediatric Oncology Patients

NCT02530658
View directory record Official record
Conditions
Solid, Liquid, Central Nervous System Tumors
Interventions
Study Introduction Visit, Informed Consent Visit, Informed Consent Follow-Up Visit, Return of Results Conversation, Return of Results Follow-Up Visits, Blood Sample, Skin Biopsy
Other · Procedure
Lead sponsor
St. Jude Children's Research Hospital
Other
Eligibility
Not listed
Enrollment
2,500 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2053
U.S. locations
1
States / cities
Memphis, Tennessee
Source: ClinicalTrials.gov public record
Updated Jul 22, 2026 · Synced Oct 22, 2026, 5:31 AM EDT
Completed Phase 2 Interventional

Hormone Therapy in Preventing Endometrial Cancer in Patients With a Genetic Risk For Hereditary Nonpolyposis Colon Cancer

NCT00033358
View directory record Official record
Conditions
Endometrial Cancer
Interventions
medroxyprogesterone, ethinyl estradiol, norgestrel, laboratory biomarker analysis
Drug · Other
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
25 Years to 50 Years · Female only
Enrollment
52 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2002
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated May 3, 2013 · Synced Oct 22, 2026, 5:31 AM EDT
Completed Early Phase 1 Interventional

Rosuvastatin (Crestor) in Friedreich Ataxia

NCT02705547
View directory record Official record
Conditions
Friedreich Ataxia
Interventions
Rosuvastatin
Drug
Lead sponsor
Children's Hospital of Philadelphia
Other
Eligibility
18 Years to 65 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2017
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Mar 25, 2021 · Synced Oct 22, 2026, 5:31 AM EDT
Withdrawn Phase 3 Interventional

Pulmozyme in Cystic Fibrosis With Sinusitis

NCT01155752
View directory record Official record
Conditions
Sinusitis, Cystic Fibrosis
Interventions
Pulmozyme single use ampule, placebo
Drug
Lead sponsor
Milton S. Hershey Medical Center
Other
Eligibility
12 Years to 65 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2015
U.S. locations
1
States / cities
Hershey, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jul 21, 2017 · Synced Oct 22, 2026, 5:31 AM EDT
Completed Phase 3 Interventional Results available

Long-term Safety of Lumacaftor/Ivacaftor in Participants With Cystic Fibrosis Who Are Homozygous for F508del and 12 to <24 Months of Age at Treatment Initiation

NCT04235140
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
LUM/IVA
Drug
Lead sponsor
Vertex Pharmaceuticals Incorporated
Industry
Eligibility
12 Months and older
Enrollment
52 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2023
U.S. locations
20
States / cities
Birmingham, Alabama • Little Rock, Arkansas • Aurora, Colorado + 17 more
Source: ClinicalTrials.gov public record
Updated Sep 19, 2024 · Synced Oct 22, 2026, 5:31 AM EDT
Terminated Phase 2 Interventional Results available

Study to Evaluate the Safety of AT2220 (Duvoglustat) in Pompe Disease

NCT00688597
View directory record Official record
Conditions
Pompe Disease
Interventions
Duvoglustat
Drug
Lead sponsor
Amicus Therapeutics
Industry
Eligibility
18 Years to 74 Years
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2009
U.S. locations
1
States / cities
Decatur, Georgia
Source: ClinicalTrials.gov public record
Updated Sep 11, 2025 · Synced Oct 22, 2026, 5:31 AM EDT
No Longer Available No phase listed Expanded access

Expanded Access to Voxelotor for Patients With Sickle Cell Disease Who Have No Alternative Treatment Options

NCT03943615
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Voxelotor
Drug
Lead sponsor
Pfizer
Industry
Eligibility
12 Years and older
Healthy volunteers
Healthy volunteers not accepted
U.S. locations
18
States / cities
Birmingham, Alabama • Mobile, Alabama • Atlanta, Georgia + 15 more
Source: ClinicalTrials.gov public record
Updated Jul 18, 2023 · Synced Oct 22, 2026, 5:31 AM EDT
Active, not recruiting Phase 1Phase 2 Interventional

Gene Therapy for Cardiomyopathy Associated With Friedreich's Ataxia

NCT05445323
View directory record Official record
Conditions
Friedreich Ataxia, Cardiomyopathy, Secondary
Interventions
Low dose LX2006, Mid Dose LX2006, High Dose LX2006
Genetic
Lead sponsor
Lexeo Therapeutics
Industry
Eligibility
18 Years to 50 Years
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2029
U.S. locations
3
States / cities
Los Angeles, California • Tampa, Florida • Rochester, Minnesota
Source: ClinicalTrials.gov public record
Updated Dec 23, 2025 · Synced Oct 22, 2026, 5:31 AM EDT