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Showing 25–48 of 24 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Therapy, Gene Clear all
Completed Phase 1 Interventional

Gene Therapy in Treating Patients With Colon Cancer That Has Spread to the Liver

NCT00012155
View directory record Official record
Conditions
Colorectal Cancer, Metastatic Cancer
Interventions
NV1020
Biological
Lead sponsor
Memorial Sloan Kettering Cancer Center
Other
Eligibility
18 Years and older
Healthy volunteers
Healthy volunteers not accepted
Timeline
2000 – 2009
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jun 25, 2013 · Synced Jul 24, 2026, 1:00 AM EDT
Recruiting No phase listed Observational

Observe Change in Nasal Swab DRD2 Gene Expression in Restless Legs Syndrome (RLS) Patients on Prolonged Dopamine Agonist Treatment

NCT06726785
View directory record Official record
Conditions
Restless Leg Syndrome (RLS)
Interventions
Not listed
Lead sponsor
HBC Immunology Inc
Industry
Eligibility
18 Years to 60 Years
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2026
U.S. locations
1
States / cities
San Francisco, California
Source: ClinicalTrials.gov public record
Updated Sep 10, 2025 · Synced Jul 24, 2026, 1:00 AM EDT
Completed No phase listed Observational

Follow-up of Breast Cancer and Multiple Myeloma Patients Previously Enrolled in NIH Gene Therapy Studies

NCT00427726
View directory record Official record
Conditions
Gene Transfer
Interventions
Not listed
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
Not listed
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003 – 2014
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Dec 11, 2019 · Synced Jul 24, 2026, 1:00 AM EDT
Completed Phase 1 Interventional

Gene Therapy for Alzheimer's Disease Clinical Trial

NCT00017940
View directory record Official record
Conditions
Alzheimer Disease
Interventions
Human Nerve Growth Factor
Genetic
Lead sponsor
The Shiley Family Trust
Other
Eligibility
50 Years and older
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Ends 2003
U.S. locations
1
States / cities
La Jolla, California
Source: ClinicalTrials.gov public record
Updated Dec 10, 2009 · Synced Jul 24, 2026, 1:00 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 Jul 24, 2026, 1:00 AM EDT
Active, not recruiting No phase listed Observational Accepts healthy volunteers

The Pancreas Interception Center (PIC) for Early Detection, Prevention, and Novel Therapeutics

NCT05692596
View directory record Official record
Conditions
Pancreatic Ductal Adenocarcinoma, Pancreatic Cyst, Chronic Pancreatitis, Fatty Pancreas, Genetic Pancreatic Cancer, Genetic Pancreatitis, BRCA Mutation, Lynch Syndrome, FAP, Familial Atypical Multiple Mole-Melanoma, PALB2 Gene Mutation, Peutz-Jeghers Syndrome, Ataxia Telangiectasia
Interventions
Data collection
Other
Lead sponsor
H. Lee Moffitt Cancer Center and Research Institute
Other
Eligibility
18 Years and older
Enrollment
317 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2022 – 2026
U.S. locations
1
States / cities
Tampa, Florida
Source: ClinicalTrials.gov public record
Updated Mar 31, 2026 · Synced Jul 24, 2026, 1:00 AM EDT
Completed Phase 1Phase 2 Interventional Results available

Gene Transfer Clinical Trial to Deliver rAAVrh74.MCK.GALGT2 for Duchenne Muscular Dystrophy

NCT03333590
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
rAAVrh74.MCK.GALGT2
Biological
Lead sponsor
Kevin Flanigan
Other
Eligibility
4 Years and older · Male only
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2023
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Mar 12, 2025 · Synced Jul 24, 2026, 1:00 AM EDT
Terminated Phase 2 Interventional

Study of AAV-GAD Gene Transfer Into the Subthalamic Nucleus for Parkinson's Disease

NCT00643890
View directory record Official record
Conditions
Parkinson's Disease
Interventions
Bilateral surgical infusion of AAV-GAD into the subthalamic nucleus
Genetic
Lead sponsor
Neurologix, Inc.
Industry
Eligibility
30 Years to 75 Years
Enrollment
44 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2008
U.S. locations
7
States / cities
Stanford, California • Aurora, Colorado • Boston, Massachusetts + 4 more
Source: ClinicalTrials.gov public record
Updated Feb 21, 2012 · Synced Jul 24, 2026, 1:00 AM EDT
Recruiting Phase 1Phase 2 Interventional

HSV-tk and XRT and Chemotherapy for Newly Diagnosed GBM

NCT03603405
View directory record Official record
Conditions
Glioblastoma, Anaplastic Astrocytoma
Interventions
ADV/HSV-tk (gene therapy)
Drug
Lead sponsor
The Methodist Hospital Research Institute
Other
Eligibility
18 Years and older
Enrollment
62 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2026
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Mar 29, 2026 · Synced Jul 24, 2026, 1:00 AM EDT
Completed Phase 1 Interventional

Treatment of B-CLL With Human IL-2 and CD40 Ligand and Plasmid Gene Modified Autologous Tumor Cells

NCT00078520
View directory record Official record
Conditions
Leukemia, Leukemia, B-Cell, Chronic
Interventions
Dose Level 1, Dose Level 2, Dose Level 2- Fixed Dose
Biological
Lead sponsor
Baylor College of Medicine
Other
Eligibility
18 Years and older
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003 – 2010
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Jan 20, 2020 · Synced Jul 24, 2026, 1:00 AM EDT
Active, not recruiting No phase listed Observational

Long-Term Safety and Efficacy of Spark-Sponsored Gene Therapies in Males With Hemophilia A

NCT03432520
View directory record Official record
Conditions
Hemophilia A
Interventions
SPK-8011, SPK-8016
Genetic
Lead sponsor
Spark Therapeutics, Inc.
Industry
Eligibility
18 Years and older · Male only
Enrollment
29 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2032
U.S. locations
9
States / cities
Boston, Massachusetts • Madison, Mississippi • Kansas City, Missouri + 5 more
Source: ClinicalTrials.gov public record
Updated Nov 28, 2024 · Synced Jul 24, 2026, 1:00 AM EDT
Completed Phase 1 Interventional Results available

Experimental Gene Transfer Procedure to Treat Alpha 1-Antitrypsin (AAT) Deficiency

NCT00430768
View directory record Official record
Conditions
Alpha 1-Antitrypsin Deficiency
Interventions
rAAV1-CB-hAAT
Biological
Lead sponsor
University of Massachusetts, Worcester
Other
Eligibility
18 Years and older
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2015
U.S. locations
2
States / cities
Gainesville, Florida • Worcester, Massachusetts
Source: ClinicalTrials.gov public record
Updated Dec 14, 2016 · Synced Jul 24, 2026, 1:00 AM EDT
Terminated Phase 1 Interventional Results available

Study of Intrathecal Administration of Onasemnogene Abeparvovec-xioi for Spinal Muscular Atrophy

NCT03381729
View directory record Official record
Conditions
Spinal Muscular Atrophy
Interventions
Onasemnogene Abeparvovec-xioi
Biological
Lead sponsor
Novartis Gene Therapies
Industry
Eligibility
6 Months to 60 Months
Enrollment
32 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2021
U.S. locations
11
States / cities
Los Angeles, California • Stanford, California • Orlando, Florida + 8 more
Source: ClinicalTrials.gov public record
Updated Jan 25, 2026 · Synced Jul 24, 2026, 1:00 AM EDT
Active, not recruiting Phase 2 Interventional Results available

Ibrutinib + Venetoclax in Untreated WM

NCT04273139
View directory record Official record
Conditions
Waldenstrom Macroglobulinemia, MYD88 Gene Mutation
Interventions
IBRUTINIB, Venetoclax
Drug
Lead sponsor
Dana-Farber Cancer Institute
Other
Eligibility
18 Years and older
Enrollment
45 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2028
U.S. locations
2
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated May 10, 2026 · Synced Jul 24, 2026, 1:00 AM EDT
Terminated Phase 1 Interventional

Hemophilia B Gene Therapy With AAV8 Vector

NCT01620801
View directory record Official record
Conditions
Hemophilia B
Interventions
AAV8-hFIX19
Biological
Lead sponsor
Spark Therapeutics, Inc.
Industry
Eligibility
18 Years and older · Male only
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2016
U.S. locations
2
States / cities
Philadelphia, Pennsylvania • Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Mar 11, 2019 · Synced Jul 24, 2026, 1:00 AM EDT
Recruiting Phase 2 Interventional

Impact of COL7A1 Gene Therapy on SCC Recurrence in RDEB Skin

NCT06731933
View directory record Official record
Conditions
Squamous Cell Carcinoma
Interventions
BVEC
Drug
Lead sponsor
Stanford University
Other
Eligibility
18 Years and older
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
1
States / cities
Redwood City, California
Source: ClinicalTrials.gov public record
Updated Apr 19, 2026 · Synced Jul 24, 2026, 1:00 AM EDT
Active, not recruiting Phase 1 Interventional

Local Consolidative Therapy and Brigatinib in Treating Patients With Stage IV or Recurrent Non-small Cell Lung Cancer

NCT03707938
View directory record Official record
Conditions
Advanced Lung Carcinoma, ALK Gene Rearrangement, Lung Non-Small Cell Carcinoma, Recurrent Lung Non-Small Cell Carcinoma, Stage IV Lung Cancer AJCC v8, Stage IVA Lung Cancer AJCC v8, Stage IVB Lung Cancer AJCC v8
Interventions
Brigatinib, Local Consolidation Therapy
Drug · Procedure
Lead sponsor
M.D. Anderson Cancer Center
Other
Eligibility
18 Years and older
Enrollment
35 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2026
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Nov 2, 2025 · Synced Jul 24, 2026, 1:00 AM EDT
Recruiting Phase 1 Interventional

A Study to Evaluate a Novel Gene Therapy in Patients With Relapsed and Refractory Multiple Myeloma

NCT07075185
View directory record Official record
Conditions
Multiple Myeloma in Relapse, Myeloma Multiple, Multiple Myeloma Progression, Neoplasms by Histologic Type, Neoplasm, Hemostatic Disorders, Vascular Disorder, Paraproteinemias, Blood Protein Disorders, Hematologic Disease and Disorders, Lymphoproliferative Disorders, Immunoproliferative Disorders, Immune System Disease, Gene Therapy
Interventions
KLN-1010
Drug
Lead sponsor
Kelonia Therapeutics, Inc.
Industry
Eligibility
18 Years and older
Enrollment
70 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2042
U.S. locations
7
States / cities
Duarte, California • Palo Alto, California • San Francisco, California + 4 more
Source: ClinicalTrials.gov public record
Updated Jul 7, 2026 · Synced Jul 24, 2026, 1:00 AM EDT
Recruiting Phase 3 Interventional

Efficacy and Safety of CSL222 (Etranacogene Dezaparvovec) Gene Therapy in Adults With Hemophilia B With Pretreatment Adeno-associated Virus Serotype 5 (AAV5) Neutralizing Antibodies (Nabs)

NCT06003387
View directory record Official record
Conditions
Hemophilia B
Interventions
CSL222 (AAV5-hFIXco-Padua)
Genetic
Lead sponsor
CSL Behring
Industry
Eligibility
18 Years and older
Enrollment
35 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2032
U.S. locations
3
States / cities
San Diego, California • Ann Arbor, Michigan • Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jun 15, 2026 · Synced Jul 24, 2026, 1:00 AM EDT
Active, not recruiting Phase 2Phase 3 Interventional

A Study to Evaluate Efficacy, Safety, Tolerability and Exposure After a Repeat-dose of Sepofarsen (QR-110) in LCA10 (ILLUMINATE)

NCT03913143
View directory record Official record
Conditions
Leber Congenital Amaurosis 10, Blindness, Leber Congenital Amaurosis, Vision Disorders, Sensation Disorders, Neurologic Manifestations, Eye Diseases, Eye Diseases, Hereditary, Eye Disorders Congenital, Retinal Disease
Interventions
sepofarsen, Sham
Drug · Other
Lead sponsor
ProQR Therapeutics
Industry
Eligibility
8 Years and older
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2023
U.S. locations
1
States / cities
Iowa City, Iowa
Source: ClinicalTrials.gov public record
Updated Mar 16, 2022 · Synced Jul 24, 2026, 1:00 AM EDT
Active, not recruiting Phase 1 Interventional

3rd Generation GD-2 Chimeric Antigen Receptor and iCaspase Suicide Safety Switch, Neuroblastoma, GRAIN

NCT01822652
View directory record Official record
Conditions
Neuroblastoma
Interventions
iC9-GD2 T Cells - frozen, iC9-GD2 T Cells - fresh, Cytoxan, Fludara, Keytruda, iC9-GD2 T cells
Genetic · Drug
Lead sponsor
Baylor College of Medicine
Other
Eligibility
Not listed
Enrollment
11 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2030
U.S. locations
2
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Apr 5, 2026 · Synced Jul 24, 2026, 1:00 AM EDT
Completed Phase 3 Interventional Results available

AZD9291 (Osimertinib) Versus Platinum-Based Doublet-Chemotherapy in Locally Advanced or Metastatic Non-Small Cell Lung Cancer

NCT02151981
View directory record Official record
Conditions
Anticancer Treatment
Interventions
Chemotherapy, Cross-over to Osimertinib
Drug
Lead sponsor
AstraZeneca
Industry
Eligibility
18 Years to 130 Years
Enrollment
421 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2023
U.S. locations
21
States / cities
Anaheim, California • Orange, California • Santa Rosa, California + 18 more
Source: ClinicalTrials.gov public record
Updated Jan 6, 2025 · Synced Jul 24, 2026, 1:00 AM EDT
Completed Phase 1 Interventional

Genetically Modified Lymphocytes to Treat HIV-Infected Identical Twins - Study Modifications

NCT00001409
View directory record Official record
Conditions
Acquired Immunodeficiency Syndrome, HIV Infection
Interventions
Interleukin-2
Drug
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
Not listed
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1994 – 2002
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 3, 2008 · Synced Jul 24, 2026, 1:00 AM EDT
Completed Phase 1Phase 2 Interventional Results available

A Double-blind Study to Evaluate the Safety of Glutamic Acid Decarboxylase Gene Transfer in Parkinson's Participants

NCT05603312
View directory record Official record
Conditions
Parkinson's Disease
Interventions
AAV-GAD Low Dose, AAV-GAD High Dose, Sham Surgery
Genetic · Procedure
Lead sponsor
MeiraGTx, LLC
Industry
Eligibility
25 Years to 85 Years
Enrollment
14 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2024
U.S. locations
6
States / cities
New Haven, Connecticut • West Bloomfield, Michigan • New York, New York + 2 more
Source: ClinicalTrials.gov public record
Updated Sep 4, 2025 · Synced Jul 24, 2026, 1:00 AM EDT