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ClinicalTrials.gov public records Last synced Oct 17, 2026, 5:20 AM EDT

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Showing 1201–1224 of 5,852 matching trials from the live ClinicalTrials.gov search.
Filters: "gene therapy" Clear all
Not yet recruiting Phase 3 Interventional

A Long-Term Extension (LTE) Study Of Enpatoran In Cutaneous Manifestations Of Lupus With Or Without Systemic Disease [Elowen-LTE]

NCT07840287
View directory record Official record
Conditions
Systemic Lupus Erythematosus (SLE), Cutaneous Lupus Erythematosus (CLE)
Interventions
Enpatoran
Drug
Lead sponsor
EMD Serono Research & Development Institute, Inc.
Industry
Eligibility
18 Years to 76 Years
Enrollment
404 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2031
U.S. locations
1
States / cities
Billerica, Massachusetts
Source: ClinicalTrials.gov public record
Updated Sep 25, 2026 · Synced Oct 17, 2026, 5:20 AM EDT
Terminated Phase 1Phase 2 Interventional

Gene Therapy Clinical Trial for the Treatment of Leber's Hereditary Optic Neuropathy Associated With ND1 Mutations

NCT05820152
View directory record Official record
Conditions
Leber Hereditary Optic Neuropathy (LHON)
Interventions
NFS-02 Injection
Drug
Lead sponsor
Neurophth Therapeutics Inc
Other
Eligibility
18 Years to 75 Years
Enrollment
11 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2024
U.S. locations
1
States / cities
Aurora, Colorado
Source: ClinicalTrials.gov public record
Updated Sep 20, 2024 · Synced Oct 17, 2026, 5:20 AM EDT
Completed Phase 2 Interventional Results available

Study to Assess Efficacy, Safety, Tolerability and Pharmacokinetics of Simeprevir, Daclatasvir and Sofosbuvir in Treatment-naive Participants With Chronic Hepatitis C Virus Genotype 1 Infection

NCT02349048
View directory record Official record
Conditions
Hepatitis C Virus
Interventions
Simeprevir 150 mg, Daclatasvir 60 mg, Sofosbuvir 400 mg
Drug
Lead sponsor
Janssen Research & Development, LLC
Industry
Eligibility
18 Years to 70 Years
Enrollment
68 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2016
U.S. locations
7
States / cities
Bakersfield, California • Jacksonville, Florida • Lutherville, Maryland + 4 more
Source: ClinicalTrials.gov public record
Updated Mar 1, 2017 · Synced Oct 17, 2026, 5:20 AM EDT
Completed No phase listed Observational

Screening Protocol for a Gene Therapy Trial in Subjects With Homozygous Familial Hypercholesterolemia

NCT03018678
View directory record Official record
Conditions
Hypercholesterolemia, Familial
Interventions
Not listed
Lead sponsor
University of Pennsylvania
Other
Eligibility
18 Years and older
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2019
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Mar 6, 2020 · Synced Oct 17, 2026, 5:20 AM EDT
Active, not recruiting Phase 3 Interventional

A Trial Comparing Unrelated Donor BMT With IST for Pediatric and Young Adult Patients With Severe Aplastic Anemia (TransIT, BMT CTN 2202)

NCT05600426
View directory record Official record
Conditions
Severe Aplastic Anemia
Interventions
cyclosporine, Matched Unrelated Donor Hematopoetic Stem Cell Transplant, horse anti-thymocyte globulin (ATG), rabbit anti-thymocyte globulin (ATG), Methotrexate, Fludarabine, Cyclophosphamide, low-dose total body irradiation (TBI), Immunosuppressive Therapy (IST)
Drug · Procedure · Radiation
Lead sponsor
Boston Children's Hospital
Other
Eligibility
0 Years to 25 Years
Enrollment
53 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2029
U.S. locations
50
States / cities
Birmingham, Alabama • Phoenix, Arizona • Little Rock, Arkansas + 44 more
Source: ClinicalTrials.gov public record
Updated Sep 25, 2026 · Synced Oct 17, 2026, 5:20 AM EDT
Terminated Phase 1Phase 2 Interventional Results available

Study of MK-3475 Alone or in Combination With Copanlisib in Relapsed or Refractory NK and T-cell Non-Hodgkin Lymphoma

NCT02535247
View directory record Official record
Conditions
Lymphoma, T-Cell, Peripheral
Interventions
MK-3475, Copanlisib
Drug
Lead sponsor
Fox Chase Cancer Center
Other
Eligibility
18 Years and older
Enrollment
17 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2020
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jul 8, 2022 · Synced Oct 17, 2026, 5:20 AM EDT
Completed Phase 1Phase 2 Interventional

AAVRh.10 Administered to Children With Late Infantile Neuronal Ceroid Lipofuscinosis

NCT01414985
View directory record Official record
Conditions
Late Infantile Neuronal Ceroid Lipofuscinosis, Batten Disease
Interventions
AAVrh.10CUCLN2
Biological
Lead sponsor
Weill Medical College of Cornell University
Other
Eligibility
3 Years to 18 Years
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2017
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Aug 31, 2020 · Synced Oct 17, 2026, 5:20 AM EDT
Completed No phase listed Observational

Host and Bacterial Mechanisms During Cystic Fibrosis Pulmonary Exacerbations

NCT04354038
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
Not listed
Lead sponsor
National Jewish Health
Other
Eligibility
18 Years and older
Enrollment
29 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2023
U.S. locations
1
States / cities
Denver, Colorado
Source: ClinicalTrials.gov public record
Updated Apr 13, 2026 · Synced Oct 17, 2026, 5:20 AM EDT
Completed Phase 1Phase 2 Interventional Results available

Safety and Tolerability of RGX-314 (Investigational Product) Gene Therapy for Neovascular AMD Trial

NCT03066258
View directory record Official record
Conditions
Neovascular Age-related Macular Degeneration, Wet Age-related Macular Degeneration
Interventions
RGX-314
Genetic
Lead sponsor
REGENXBIO Inc.
Industry
Eligibility
50 Years to 89 Years
Enrollment
42 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2021
U.S. locations
8
States / cities
Santa Barbara, California • Baltimore, Maryland • Boston, Massachusetts + 4 more
Source: ClinicalTrials.gov public record
Updated May 16, 2023 · Synced Oct 17, 2026, 5:20 AM EDT
Recruiting Phase 2 Interventional

Study of the ITK Inhibitor Soquelitinib to Reduce Lymphoproliferation and Improve Cytopenias in Autoimmune Lymphoproliferative Syndrome (ALPS)-FAS Patients

NCT06730126
View directory record Official record
Conditions
Autoimmune Lymphoproliferative Syndrome
Interventions
Soquelitinib
Drug
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
16 Years to 120 Years
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2026
U.S. locations
3
States / cities
Bethesda, Maryland • Philadelphia, Pennsylvania • Houston, Texas
Source: ClinicalTrials.gov public record
Updated Sep 8, 2026 · Synced Oct 17, 2026, 5:20 AM EDT
Not yet recruiting Phase 1Phase 2 Interventional

A Study to Evaluate ANS014004 in Combination With EGFR-TKI in Non-Small Cell Lung Cancer

NCT07285148
View directory record Official record
Conditions
Non-Small Cell Lung Cancer, EGFR Mutation, Metastatic Lung Cancer, MET Alteration
Interventions
ANS014004 + PLB1004, ANS014004 + Osimertinib
Drug
Lead sponsor
Beijing Pearl Biotechnology Limited Liability Company
Industry
Eligibility
18 Years and older
Enrollment
253 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2029
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Dec 16, 2025 · Synced Oct 17, 2026, 5:20 AM EDT
Recruiting Phase 4 Interventional

Surgical Debulking Prior to Peptide Receptor Radionuclide Therapy in Well Differentiated Gastroenteropancreatic Neuroendocrine Tumors

NCT06016855
View directory record Official record
Conditions
Digestive System Neuroendocrine Tumor G1, Digestive System Neuroendocrine Tumor G2, Metastatic Digestive System Neuroendocrine Neoplasm, Metastatic Malignant Neoplasm in the Liver, Pancreatic Neuroendocrine Tumor G1, Pancreatic Neuroendocrine Tumor G2
Interventions
Tumor Debulking, Lutetium Lu 177 Dotatate, Computed Tomography, Magnetic Resonance Imaging, Copper Cu 64 Dotatate, Positron Emission Tomography
Procedure · Drug
Lead sponsor
Vanderbilt-Ingram Cancer Center
Other
Eligibility
18 Years and older
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2028
U.S. locations
1
States / cities
Nashville, Tennessee
Source: ClinicalTrials.gov public record
Updated Jun 24, 2026 · Synced Oct 17, 2026, 5:20 AM EDT
Active, not recruiting Phase 3 Interventional

RGX-121-3102 Gene Therapy in Participants With MPS II (Hunter Syndrome)

NCT07236606
View directory record Official record
Conditions
MPS II, Hunter Syndrome (MPS II)
Interventions
RGX-121-3102
Genetic
Lead sponsor
REGENXBIO Inc.
Industry
Eligibility
4 Months to 5 Years · Male only
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2031
U.S. locations
1
States / cities
New Brunswick, New Jersey
Source: ClinicalTrials.gov public record
Updated Jun 4, 2026 · Synced Oct 17, 2026, 5:20 AM EDT
Terminated Phase 1Phase 2 Interventional

PTK787 + Trastuzumab for HER2 Overexpressing Metastatic Breast Cancer

NCT00216047
View directory record Official record
Conditions
Breast Cancer
Interventions
PTK787, Trastuzumab
Drug
Lead sponsor
Hoosier Cancer Research Network
Other
Eligibility
18 Years and older · Female only
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2006
U.S. locations
8
States / cities
Elkhart, Indiana • Fort Wayne, Indiana • Goshen, Indiana + 5 more
Source: ClinicalTrials.gov public record
Updated Dec 9, 2015 · Synced Oct 17, 2026, 5:20 AM EDT
Completed Phase 1 Interventional

Study of PDR001 and/or MBG453 in Combination With Decitabine in Patients With AML or High Risk MDS

NCT03066648
View directory record Official record
Conditions
Leukemia, Leukemia, Myeloid, Leukemia, Myeloid, Acute, Myelodysplastic Syndromes, Preleukemia, Bone Marrow Diseases, Hematologic Diseases, Chronic Myelomonocytic Leukemia
Interventions
Decitabine, PDR001, MBG453, Azacitidine
Drug
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
18 Years and older
Enrollment
241 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2023
U.S. locations
3
States / cities
Boston, Massachusetts • Portland, Oregon • Houston, Texas
Source: ClinicalTrials.gov public record
Updated May 18, 2025 · Synced Oct 17, 2026, 5:20 AM EDT
Completed Phase 1Phase 2 Interventional Results available

Phase I/II Study of Celebrex and EPO906 in Patients With Metastatic Colorectal Cancer

NCT00159484
View directory record Official record
Conditions
Colon Cancer, Colorectal Cancer
Interventions
EPO906, Celecoxib
Drug
Lead sponsor
University of Southern California
Other
Eligibility
18 Years and older
Enrollment
75 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2023
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated May 28, 2026 · Synced Oct 17, 2026, 5:20 AM EDT
Completed Phase 1 Interventional Results available

Evaluation of Tofacitinib in Prevention of Photosensitivity in Lupus

NCT05048238
View directory record Official record
Conditions
Cutaneous Lupus
Interventions
Tofacitinib
Drug
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
18 Years to 65 Years
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2024
U.S. locations
2
States / cities
Worcester, Massachusetts • Ann Arbor, Michigan
Source: ClinicalTrials.gov public record
Updated Aug 22, 2025 · Synced Oct 17, 2026, 5:20 AM EDT
Withdrawn Phase 4 Interventional

A Study of ELAPRASE in Treatment-naïve Participants With Hunter Syndrome (Mucopolysaccharidosis [MPS] II)

NCT05494593
View directory record Official record
Conditions
Mucopolysaccharidosis (MPS), Hunter Syndrome
Interventions
ELAPRASE, Rituximab, Methotrexate, Intravenous Immunoglobulin (IVIG)
Drug
Lead sponsor
Takeda
Industry
Eligibility
Up to 6 Years · Male only
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2025
U.S. locations
8
States / cities
Phoenix, Arizona • Oakland, California • San Diego, California + 5 more
Source: ClinicalTrials.gov public record
Updated Dec 9, 2025 · Synced Oct 17, 2026, 5:20 AM EDT
Withdrawn Phase 2 Interventional

PREDATOR: Neoadjuvant Gene Prediction for Breast Cancer

NCT02067416
View directory record Official record
Conditions
Breast Cancer
Interventions
Anthracycline based chemotherapy, taxane-based chemotherapy
Drug
Lead sponsor
Jenny C. Chang, MD
Other
Eligibility
18 Years and older
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2014
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Feb 2, 2023 · Synced Oct 17, 2026, 5:20 AM EDT
Completed Phase 3 Interventional Results available

Study of Lumacaftor in Combination With Ivacaftor in Subjects 6 Through 11 Years of Age With Cystic Fibrosis, Homozygous for the F508del-CFTR Mutation

NCT01897233
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
Lumacaftor, Ivacaftor
Drug
Lead sponsor
Vertex Pharmaceuticals Incorporated
Industry
Eligibility
6 Years to 11 Years
Enrollment
62 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2015
U.S. locations
19
States / cities
Birmingham, Alabama • Tucson, Arizona • Long Beach, California + 16 more
Source: ClinicalTrials.gov public record
Updated Jun 20, 2017 · Synced Oct 17, 2026, 5:20 AM EDT
Completed Phase 1Phase 2 Interventional Results available

HIV-1-Gag Conserved-Element DNA Vaccine (p24CE) Vaccine Study

NCT03560258
View directory record Official record
Conditions
HIV Infections
Interventions
p24CE1/2 pDNA vaccine, p24CE1/2 pDNA vaccine admixed with full-length p55^gag pDNA vaccine, Full-length p55^gag pDNA vaccine, Placebo
Biological
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
18 Years to 65 Years
Enrollment
45 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2021
U.S. locations
14
States / cities
Birmingham, Alabama • Los Angeles, California • San Francisco, California + 9 more
Source: ClinicalTrials.gov public record
Updated Apr 26, 2022 · Synced Oct 17, 2026, 5:20 AM EDT
Completed Phase 1 Interventional Results available

Safety Study of TissueGene-C in Degenerative Joint Disease of the Knee

NCT00599248
View directory record Official record
Conditions
Osteoarthritis, Knee
Interventions
TissueGene-C, Placebo
Biological
Lead sponsor
Kolon TissueGene, Inc.
Industry
Eligibility
18 Years and older
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2010
U.S. locations
2
States / cities
Baltimore, Maryland • Arlington, Virginia
Source: ClinicalTrials.gov public record
Updated Jul 7, 2017 · Synced Oct 17, 2026, 5:20 AM EDT
Active, not recruiting Phase 2 Interventional Results available

Tamoxifen Citrate or Z-Endoxifen Hydrochloride in Treating Patients With Locally Advanced or Metastatic, Estrogen Receptor-Positive, HER2-Negative Breast Cancer

NCT02311933
View directory record Official record
Conditions
Recurrent Breast Carcinoma, Stage III Breast Cancer AJCC v7, Stage IIIA Breast Cancer AJCC v7, Stage IIIB Breast Cancer AJCC v7, Stage IIIC Breast Cancer AJCC v7, Stage IV Breast Cancer AJCC v6 and v7
Interventions
Endoxifen Hydrochloride, Laboratory Biomarker Analysis, Pharmacological Study, Tamoxifen Citrate
Drug · Other
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
18 Years and older · Female only
Enrollment
81 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2027
U.S. locations
559
States / cities
Mobile, Alabama • Anchorage, Alaska • Fairbanks, Alaska + 356 more
Source: ClinicalTrials.gov public record
Updated Aug 28, 2026 · Synced Oct 17, 2026, 5:20 AM EDT
Active, not recruiting Phase 4 Interventional

Investigating Dupilumab's Effect in Asthma by Genotype

NCT03694158
View directory record Official record
Conditions
Asthma
Interventions
Dupilumab, Placebo
Drug · Other
Lead sponsor
Boston Children's Hospital
Other
Eligibility
12 Years and older
Enrollment
150 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2027
U.S. locations
6
States / cities
Boston, Massachusetts • Detroit, Michigan • The Bronx, New York + 2 more
Source: ClinicalTrials.gov public record
Updated Feb 23, 2026 · Synced Oct 17, 2026, 5:20 AM EDT