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Showing 1–24 of 1,024 matching trials from the live ClinicalTrials.gov search.
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Completed Phase 1 Interventional

Intrapleural AdV-tk Therapy in Patients With Malignant Pleural Effusion

NCT01997190
View directory record Official record
Conditions
Malignant Pleural Effusion, Lung Cancer, Mesothelioma, Breast Cancer, Ovarian Cancer
Interventions
AdV-tk + valacyclovir
Biological
Lead sponsor
Candel Therapeutics, Inc.
Industry
Eligibility
18 Years and older
Enrollment
19 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2018
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Aug 24, 2023 · Synced Sep 12, 2026, 10:38 AM EDT
Recruiting Phase 2 Interventional

TAPUR: Testing the Use of Food and Drug Administration (FDA) Approved Drugs That Target a Specific Abnormality in a Tumor Gene in People With Advanced Stage Cancer

NCT02693535
View directory record Official record
Conditions
Lymphoma, Non-Hodgkin, Multiple Myeloma, Advanced Solid Tumors
Interventions
Sunitinib, Temsirolimus, Trastuzumab and Pertuzumab, Vemurafenib and Cobimetinib, Olaparib, Nivolumab and Ipilimumab, Abemaciclib, Talazoparib, Atezolizumab and Talazoparib, Tucatinib plus Trastuzumab Subcutaneous (SC), Futibatinib, Dabrafenib plus Trametinib, Fam-Trastuzumab Deruxtecan-Nxki (TDxD)
Drug
Lead sponsor
American Society of Clinical Oncology
Other
Eligibility
12 Years and older
Enrollment
4,200 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2028
U.S. locations
174
States / cities
Birmingham, Alabama • Phoenix, Arizona • Auburn, California + 137 more
Source: ClinicalTrials.gov public record
Updated Aug 31, 2026 · Synced Sep 12, 2026, 10:38 AM EDT
Approved For Marketing No phase listed Expanded access

An Intermediate Access Protocol for Selumetinib for Treatment of Neurofibromatosis Type 1

NCT03259633
View directory record Official record
Conditions
NF type1 With Inoperable Plexiform Neurofibromas
Interventions
Selumetinib
Drug
Lead sponsor
AstraZeneca
Industry
Eligibility
2 Years to 130 Years
Healthy volunteers
Healthy volunteers not accepted
U.S. locations
1
States / cities
New Orleans, Louisiana
Source: ClinicalTrials.gov public record
Updated Apr 30, 2020 · Synced Sep 12, 2026, 10:38 AM EDT
Terminated Phase 1 Interventional

Phase 1/2 Study of Metastatic Renal Cancer Using T-Cells Transduced With a T-Cell Receptor Which Recognizes TRAIL Bound to the DR4 Receptor

NCT00923390
View directory record Official record
Conditions
Renal Cancer, Kidney Cancer
Interventions
2G-1 TCR Retroviral Vector-Transduced lyn, Aldesleukin, Cyclophosphamide, Fludarabine, (PG13-A(F/K-F-SGSG-T2a-B (opt) (2G-1 TCR) retroviral vector-transduced lymphocyte
Genetic · Drug
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
18 Years to 99 Years
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2012
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Apr 8, 2019 · Synced Sep 12, 2026, 10:38 AM EDT
Completed Phase 2 Interventional Results available

Cilengitide, Temozolomide, and Radiation Therapy in Treating Patients With Newly Diagnosed Glioblastoma and Unmethylated Gene Promoter Status

NCT00813943
View directory record Official record
Conditions
Glioblastoma
Interventions
Cilengitide (2-times weekly), cilengitide (5-times weekly), Temozolomide, Radiotherapy
Drug · Radiation
Lead sponsor
EMD Serono
Industry
Eligibility
18 Years and older
Enrollment
265 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2013
U.S. locations
1
States / cities
Rockland, Massachusetts
Source: ClinicalTrials.gov public record
Updated Jan 29, 2017 · Synced Sep 12, 2026, 10:38 AM EDT
Recruiting Phase 1Phase 2 Interventional

ALS20-101 Lentiviral Gene Therapy for Beta Thalassemia

NCT06364774
View directory record Official record
Conditions
Beta-Thalassemia
Interventions
ALS20
Biological
Lead sponsor
Children's Hospital of Philadelphia
Other
Eligibility
18 Years to 40 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Apr 14, 2026 · Synced Sep 12, 2026, 10:38 AM EDT
Completed Phase 2 Interventional Results available

Treatment of Chronic Lymphocytic Leukemia/Small Lymphocytic Lymphoma (CLL/SLL): DNA Microarray Gene Expression Analysis

NCT00001586
View directory record Official record
Conditions
Chronic Lymphocytic Leukemia
Interventions
Rituximab, Fludarabine phosphate, Leukemic or stroma cells
Biological · Drug · Other
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
18 Years and older
Enrollment
105 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1997 – 2011
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated May 14, 2013 · Synced Sep 12, 2026, 10:38 AM EDT
Not yet recruiting Phase 1Phase 2 Interventional

Personalized Antisense Oligonucleotide Therapy for A Single Patient With CHCHD10 ALS (nL18576)

NCT07423494
View directory record Official record
Conditions
Amyotrophic Lateral Sclerosis
Interventions
nL-CHCHD-001
Drug
Lead sponsor
n-Lorem Foundation
Other
Eligibility
18 Years and older
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2027
U.S. locations
1
States / cities
Rochester, Minnesota
Source: ClinicalTrials.gov public record
Updated Feb 19, 2026 · Synced Sep 12, 2026, 10:38 AM EDT
Completed Phase 2 Interventional Results available

ADVM-022 Intravitreal Gene Therapy for DME

NCT04418427
View directory record Official record
Conditions
Diabetic Macular Edema, Diabetic Retinopathy
Interventions
6E11 vg/eye of ADVM-022, 2E11 vg/eye of ADVM-022, Aflibercept
Biological
Lead sponsor
Adverum Biotechnologies, Inc.
Industry
Eligibility
18 Years and older
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2023
U.S. locations
13
States / cities
Phoenix, Arizona • Bakersfield, California • Beverly Hills, California + 10 more
Source: ClinicalTrials.gov public record
Updated Jul 9, 2025 · Synced Sep 12, 2026, 10:38 AM EDT
Completed No phase listed Observational

Long Term Follow Up Of Patients Who Have Received Gene Therapy Or Gene Marked Products

NCT00695279
View directory record Official record
Conditions
Severe Combined Immunodeficiency, Malignancy, Hematologic, Neuroblastoma, Neoplasm, Mucopolysaccharidosis I
Interventions
Venipuncture
Procedure
Lead sponsor
St. Jude Children's Research Hospital
Other
Eligibility
Not listed
Enrollment
41 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2024
U.S. locations
1
States / cities
Memphis, Tennessee
Source: ClinicalTrials.gov public record
Updated Oct 29, 2024 · Synced Sep 12, 2026, 10:38 AM EDT
Terminated Phase 1Phase 2 Interventional Results available

A Factor IX Gene Therapy Study (FIX-GT)

NCT03369444
View directory record Official record
Conditions
Hemophilia B
Interventions
FLT180a
Biological
Lead sponsor
University College, London
Other
Eligibility
18 Years and older · Male only
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2020
U.S. locations
1
States / cities
Memphis, Tennessee
Source: ClinicalTrials.gov public record
Updated Dec 1, 2022 · Synced Sep 12, 2026, 10:38 AM EDT
Active, not recruiting Phase 2 Interventional

A Study of Oral Erdafitinib in People With Recurrent Non-Invasive Bladder Cancer

NCT04917809
View directory record Official record
Conditions
Bladder Cancer, Recurrent Bladder Cancer, FGFR3 Gene Mutation
Interventions
Erdafitinib
Drug
Lead sponsor
Memorial Sloan Kettering Cancer Center
Other
Eligibility
18 Years and older
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2027
U.S. locations
7
States / cities
Basking Ridge, New Jersey • Middletown, New Jersey • Montvale, New Jersey + 4 more
Source: ClinicalTrials.gov public record
Updated Sep 1, 2026 · Synced Sep 12, 2026, 10:38 AM EDT
Active, not recruiting Phase 3 Interventional

Follow-up Gene Therapy Trial for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene

NCT04794101
View directory record Official record
Conditions
X-Linked Retinitis Pigmentosa
Interventions
Genetic: AAV5-hRKp.RPGR Intermediate Dose, Genetic: AAV5-hRKp.RPGR Low Dose
Biological
Lead sponsor
Janssen Research & Development, LLC
Industry
Eligibility
3 Years and older
Enrollment
97 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2029
U.S. locations
10
States / cities
La Jolla, California • Los Angeles, California • Palo Alto, California + 7 more
Source: ClinicalTrials.gov public record
Updated Aug 27, 2026 · Synced Sep 12, 2026, 10:38 AM EDT
Completed Phase 1 Interventional

Safety and Tolerability Study of AAV2-sFLT01 in Patients With Neovascular Age-Related Macular Degeneration (AMD)

NCT01024998
View directory record Official record
Conditions
Macular Degeneration, Age-Related Maculopathies, Age-Related Maculopathy, Maculopathies, Age-Related, Maculopathy, Age-Related, Retinal Degeneration, Retinal Neovascularization, Gene Therapy, Therapy, Gene, Eye Diseases
Interventions
AAV2-sFLT01
Biological
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
50 Years and older
Enrollment
19 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2018
U.S. locations
4
States / cities
Baltimore, Maryland • Boston, Massachusetts • Worcester, Massachusetts + 1 more
Source: ClinicalTrials.gov public record
Updated Aug 21, 2018 · Synced Sep 12, 2026, 10:38 AM EDT
Recruiting Phase 1 Interventional

Anti-EGFRvIII synNotch Receptor Induced Anti-EphA2/IL-13Ralpha2 CAR (E-SYNC) T Cells

NCT06186401
View directory record Official record
Conditions
EGFR Gene Mutation, Glioblastoma, MGMT-Unmethylated Glioblastoma, Recurrent Glioblastoma
Interventions
E-SYNC T Cells, Cyclophosphamide (non-investigational), Fludarabine (non-investigational), Leukapheresis, Surgical resection
Biological · Drug · Procedure
Lead sponsor
Hideho Okada, MD, PhD
Other
Eligibility
18 Years and older
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2027
U.S. locations
1
States / cities
San Francisco, California
Source: ClinicalTrials.gov public record
Updated Aug 12, 2026 · Synced Sep 12, 2026, 10:38 AM EDT
Withdrawn No phase listed Observational

Mucopolysaccharidosis Type II Observational

NCT04591834
View directory record Official record
Conditions
Mucopolysaccharidosis II
Interventions
Observational
Other
Lead sponsor
REGENXBIO Inc.
Industry
Eligibility
1 Month to 8 Years · Male only
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2025
U.S. locations
2
States / cities
Oakland, California • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Oct 9, 2022 · Synced Sep 12, 2026, 10:38 AM EDT
Active, not recruiting Phase 2 Interventional

A Study Comparing Two Doses of AGTC-501 in Male Subjects With X-linked Retinitis Pigmentosa Caused by RPGR Mutations (SKYLINE)

NCT06333249
View directory record Official record
Conditions
X-Linked Retinitis Pigmentosa
Interventions
rAAV2tYF-GRK1-RPGR
Biological
Lead sponsor
Beacon Therapeutics
Industry
Eligibility
8 Years to 50 Years · Male only
Enrollment
14 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2027
U.S. locations
6
States / cities
Jacksonville, Florida • Boston, Massachusetts • Cincinnati, Ohio + 3 more
Source: ClinicalTrials.gov public record
Updated Aug 11, 2024 · Synced Sep 12, 2026, 10:38 AM EDT
Completed Phase 1 Interventional

Atorvastatin Therapy to Improve Endothelial Function in Sickle Cell Disease

NCT00072826
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Oxypurinol, L-NMMA, Acetylcholine
Drug
Lead sponsor
National Institutes of Health Clinical Center (CC)
NIH
Eligibility
18 Years to 65 Years
Enrollment
44 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003 – 2007
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Dec 11, 2019 · Synced Sep 12, 2026, 10:38 AM EDT
Completed Phase 1 Interventional

Enasidenib in Combination With Cobimetinib for the Treatment of Relapsed or Refractory Acute Myeloid Leukemia

NCT05441514
View directory record Official record
Conditions
Recurrent Acute Myeloid Leukemia, Refractory Acute Myeloid Leukemia
Interventions
Cobimetinib, Enasidenib Mesylate
Drug
Lead sponsor
City of Hope Medical Center
Other
Eligibility
18 Years and older
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2026
U.S. locations
1
States / cities
Duarte, California
Source: ClinicalTrials.gov public record
Updated May 4, 2026 · Synced Sep 12, 2026, 10:38 AM EDT
Recruiting No phase listed Observational

Non-interventional Study of Seroprevalence of Pre-existing Antibodies Against Adenovirus-associated Virus Vector (AAV9) and the Progression of Disease in Patients With Plakophilin 2 (PKP2)-Associated Arrhythmogenic Right Ventricular Cardiomyopathy (ARVC)

NCT06311708
View directory record Official record
Conditions
Arrhythmogenic Right Ventricular Cardiomyopathy
Interventions
Not listed
Lead sponsor
Tenaya Therapeutics
Industry
Eligibility
14 Years to 65 Years
Enrollment
200 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2030
U.S. locations
8
States / cities
San Francisco, California • Aurora, Colorado • Baltimore, Maryland + 5 more
Source: ClinicalTrials.gov public record
Updated Oct 31, 2024 · Synced Sep 12, 2026, 10:38 AM EDT
Terminated Phase 1 Interventional

Evaluation of Safety of Rexin-G Gene Transfer for Advanced Pancreatic Cancer

NCT00121745
View directory record Official record
Conditions
Pancreatic Cancer
Interventions
Rexin-G Dose 1, Rexin-G Dose 2, Rexin-G Dose 3, Rexin-G Dose 4
Genetic
Lead sponsor
Epeius Biotechnologies
Industry
Eligibility
18 Years and older
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2007
U.S. locations
1
States / cities
Rochester, Minnesota
Source: ClinicalTrials.gov public record
Updated Dec 19, 2007 · Synced Sep 12, 2026, 10:38 AM EDT
Withdrawn Phase 1Phase 2 Interventional

Gene Therapy for the Treatment of Chronic Stable Angina

NCT01002495
View directory record Official record
Conditions
Myocardial Ischemia
Interventions
VM202
Biological
Lead sponsor
Helixmith Co., Ltd.
Industry
Eligibility
21 Years to 75 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2016
U.S. locations
2
States / cities
Chicago, Illinois • Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Sep 24, 2025 · Synced Sep 12, 2026, 10:38 AM EDT
Recruiting Phase 2 Interventional

A Precision Medicine Approach Using Gene Silencing to Treat a Chronic Liver Disease Called Metabolic Dysfunction-Associated Steatohepatitis (MASH) in Adult Participants at Increased Genetic Risk for This Condition

NCT05519475
View directory record Official record
Conditions
Metabolic Dysfunction-Associated SteatoHepatitis (MASH)
Interventions
ALN-HSD, Placebo
Drug
Lead sponsor
Regeneron Pharmaceuticals
Industry
Eligibility
18 Years to 75 Years
Enrollment
120 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2028
U.S. locations
61
States / cities
Chandler, Arizona • Flagstaff, Arizona • Peoria, Arizona + 48 more
Source: ClinicalTrials.gov public record
Updated Jul 7, 2026 · Synced Sep 12, 2026, 10:38 AM EDT
Completed No phase listed Observational

Safety and Survival of Genetically Modified White Blood Cells in HIV-Infected Persons - A Study in Identical Twin Pairs

NCT00001353
View directory record Official record
Conditions
Acquired Immunodeficiency Syndrome, HIV Infection
Interventions
Genetically Marked Syngeneic T Lymphocytes
Drug
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
Not listed
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1993 – 2002
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 3, 2008 · Synced Sep 12, 2026, 10:38 AM EDT