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ClinicalTrials.gov public records Last synced Sep 11, 2026, 9:08 PM EDT

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Showing 1–24 of 203 matching trials from the live ClinicalTrials.gov search.
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Filters: Intervention: Gene modified therapy Clear all
Terminated Phase 2 Interventional Results available

VB-111 in Surgically Accessible Recurrent/Progressive GBM

NCT04406272
View directory record Official record
Conditions
Glioblastoma, Recurrent Glioblastoma
Interventions
VB11, Surgery, Placebo, Bevacizumab
Drug · Procedure · Other
Lead sponsor
Dana-Farber Cancer Institute
Other
Eligibility
18 Years and older
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2024
U.S. locations
4
States / cities
Los Angeles, California • Boston, Massachusetts • San Antonio, Texas + 1 more
Source: ClinicalTrials.gov public record
Updated Jun 9, 2025 · Synced Sep 11, 2026, 9:08 PM EDT
Active, not recruiting Phase 1 Interventional

CD4 CAR+ ZFN-modified T Cells in HIV Therapy

NCT03617198
View directory record Official record
Conditions
Hiv
Interventions
CD4 CAR+CCR5 ZFN T-cells
Biological
Lead sponsor
University of Pennsylvania
Other
Eligibility
18 Years and older
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2027
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Aug 26, 2026 · Synced Sep 11, 2026, 9:08 PM EDT
Recruiting Phase 1Phase 2 Interventional

A Phase 1/2 Study of KSQ-004EX, Autologous Tumor Infiltrating Lymphocytes Engineered to Inactivate Genes Encoding SOCS1 and Regnase-1, in Patients With Select Advanced Solid Tumors

NCT06598371
View directory record Official record
Conditions
Select Advanced Solid Tumors
Interventions
Cyclophosphamide, Fludarabine, KSQ-004EX, Interleukin-2
Drug
Lead sponsor
M.D. Anderson Cancer Center
Other
Eligibility
18 Years and older
Enrollment
141 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2041
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated May 19, 2026 · Synced Sep 11, 2026, 9:08 PM EDT
Active, not recruiting Phase 1Phase 2 Interventional

Study of Gene Modified Donor T-cells Following TCR Alpha Beta Positive Depleted Stem Cell Transplant

NCT03301168
View directory record Official record
Conditions
Acute Lymphoblastic Leukemia, Leukemia, Acute Myeloid (AML), Child, Lymphoma, Non-Hodgkin, Myelodysplastic Syndromes, Primary Immune Deficiency Disorder, Osteopetrosis, Cytopenia, Hemoglobinopathy in Children, Anemia, Aplastic
Interventions
BPX-501 T cells, Rimiducid
Biological · Drug
Lead sponsor
Bellicum Pharmaceuticals
Industry
Eligibility
1 Month to 26 Years
Enrollment
120 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2034
U.S. locations
10
States / cities
Los Angeles, California • Palo Alto, California • Washington D.C., District of Columbia + 7 more
Source: ClinicalTrials.gov public record
Updated Jul 11, 2022 · Synced Sep 11, 2026, 9:08 PM EDT
Completed Not applicable Interventional Results available

Molecular-Guided Therapy for Relapsed and Refractory Childhood Cancer

NCT01802567
View directory record Official record
Conditions
Neuroblastoma, Medulloblastoma, Brain Tumors, Rare Tumors
Interventions
Guided Therapy- Pediatric Gene Analysis Platform
Device
Lead sponsor
Giselle Sholler
Other
Eligibility
13 Months and older
Enrollment
52 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2024
U.S. locations
13
States / cities
Phoenix, Arizona • San Diego, California • Hartford, Connecticut + 10 more
Source: ClinicalTrials.gov public record
Updated Aug 5, 2024 · Synced Sep 11, 2026, 9:08 PM 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 11, 2026, 9:08 PM EDT
Active, not recruiting Phase 2 Interventional

Levels of Circulating Tumor DNA as a Predictive Marker for Early Switch in Treatment for Patients With Metastatic (Stage IV) Breast Cancer

NCT05826964
View directory record Official record
Conditions
Breast Cancer, ER-positive Breast Cancer, HER2-negative Breast Cancer, Metastatic Breast Cancer
Interventions
AI+CDK4/6i, SERD+CDK4/6i, mTOR inhibitor + AI, mTOR inhibitor + SERD, mTOR inhibitor + Selective estrogen receptor modulator, PI3K inhibitor + SERD, PI3K inhibitor + AI, Chemotherapy, Oral SERD, PARPi, AKT inhibitor, Step 3 Arm 2
Drug · Other
Lead sponsor
University of Miami
Other
Eligibility
18 Years and older
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2029
U.S. locations
1
States / cities
Miami, Florida
Source: ClinicalTrials.gov public record
Updated Jan 12, 2026 · Synced Sep 11, 2026, 9:08 PM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Long-Term Follow-Up of Recipient of Gene Transfer Research

NCT01492036
View directory record Official record
Conditions
Retroviridae Infections, Cancer
Interventions
Blood Tests, Questionnaires
Other · Behavioral
Lead sponsor
M.D. Anderson Cancer Center
Other
Eligibility
Not listed
Enrollment
130 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2011 – 2027
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Jul 14, 2026 · Synced Sep 11, 2026, 9:08 PM EDT
Recruiting Phase 4 Interventional

A Study Evaluating the Effectiveness and Safety of Risdiplam Administered in Pediatric Patients With Spinal Muscular Atrophy Who Experienced a Plateau or Decline in Function After Gene Therapy

NCT05861999
View directory record Official record
Conditions
Muscular Atrophy, Spinal
Interventions
Risdiplam
Drug
Lead sponsor
Hoffmann-La Roche
Industry
Eligibility
3 Months to 24 Months
Enrollment
28 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2029
U.S. locations
10
States / cities
Little Rock, Arkansas • Madera, California • Palo Alto, California + 7 more
Source: ClinicalTrials.gov public record
Updated Sep 9, 2026 · Synced Sep 11, 2026, 9:08 PM EDT
Active, not recruiting Phase 1 Interventional Accepts healthy volunteers

Novel Gamma-Delta (γδ)T Cell Therapy for Treatment of Patients With Newly Diagnosed Glioblastoma

NCT04165941
View directory record Official record
Conditions
Brain Tumor Adult
Interventions
DRI cell therapy
Biological
Lead sponsor
University of Alabama at Birmingham
Other
Eligibility
18 Years and older
Enrollment
22 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2020 – 2026
U.S. locations
1
States / cities
Birmingham, Alabama
Source: ClinicalTrials.gov public record
Updated Dec 25, 2025 · Synced Sep 11, 2026, 9:08 PM EDT
Terminated Phase 1 Interventional

Modified White Blood Cells That Secrete IL-2 and Express a Protein That Targets the ESO-1tumor Protein for Metastatic Cancer

NCT01457131
View directory record Official record
Conditions
Metastatic Cancer, Metastatic Melanoma, Metastatic Renal Cancer
Interventions
Fludarabine, Cyclophosphamide, IL-12 & Anti-NY ESO1 TCR PBL
Drug · Other
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
18 Years to 99 Years
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2013
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jan 7, 2019 · Synced Sep 11, 2026, 9:08 PM 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 Sep 11, 2026, 9:08 PM EDT
Completed Phase 1 Interventional Results available

Vaccine Therapy in Stage II, III, or IV Epithelial Ovarian, Fallopian Tube, or Primary Peritoneal Cancers

NCT00803569
View directory record Official record
Conditions
Fallopian Tube Cancer, Ovarian Cancer, Peritoneal Cavity Cancer
Interventions
ALVAC(2)-NY-ESO-1(M)/TRICOM vaccine, Sargramostim
Biological
Lead sponsor
Ludwig Institute for Cancer Research
Other
Eligibility
18 Years and older · Female only
Enrollment
13 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2011
U.S. locations
2
States / cities
Buffalo, New York • New York, New York
Source: ClinicalTrials.gov public record
Updated Oct 3, 2023 · Synced Sep 11, 2026, 9:08 PM EDT
Recruiting No phase listed Observational

Transgene Assay Testing Service of Tumor Samples From Patients Who Received a Bristol-Myers Squibb Manufactured Gene Modified Cell Therapy and Have a Qualifying Second Primary Malignancy

NCT06357754
View directory record Official record
Conditions
Non-Hodgkin Lymphoma, Chronic Lymphocytic Leukaemia, Multiple Myeloma
Interventions
Idecabtagene vicleucel, Lisocabtagene maraleucel
Biological
Lead sponsor
Bristol-Myers Squibb
Industry
Eligibility
Not listed
Enrollment
50 participants
Timeline
2023 – 2038
U.S. locations
4
States / cities
Portland, Oregon • Dallas, Texas • Salt Lake City, Utah + 1 more
Source: ClinicalTrials.gov public record
Updated Jan 21, 2026 · Synced Sep 11, 2026, 9:08 PM EDT
Completed Phase 1Phase 2 Interventional Results available

Stem Cell Gene Therapy for Cystinosis

NCT03897361
View directory record Official record
Conditions
Lysosomal Storage Diseases, Cystinosis
Interventions
CTNS-RD-04 (including CTNS-RD-04-LB manufactured with LentiBOOST)
Genetic
Lead sponsor
University of California, San Diego
Other
Eligibility
18 Years and older
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2024
U.S. locations
1
States / cities
La Jolla, California
Source: ClinicalTrials.gov public record
Updated Aug 5, 2026 · Synced Sep 11, 2026, 9:08 PM EDT
Completed Phase 1 Interventional

Neuroblastoma Vaccine for Treatment of High-Risk Neuroblastoma After Chemotherapy

NCT00048386
View directory record Official record
Conditions
Neuroblastoma
Interventions
autologous neuroblastoma vaccine
Biological
Lead sponsor
Malcolm Brenner
Other
Eligibility
Up to 64 Years
Enrollment
13 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1999 – 2009
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Jul 26, 2012 · Synced Sep 11, 2026, 9:08 PM EDT
Terminated Phase 4 Interventional Results available

A Study of Fuzeon (Enfuvirtide) With an Integrase Inhibitor Plus Optimized Background in Treatment-Experienced HIV-1 Infected Patients

NCT00488059
View directory record Official record
Conditions
HIV Infections
Interventions
enfuvirtide [Fuzeon], Optimized background ARV, Integrase inhibitor
Drug
Lead sponsor
Hoffmann-La Roche
Industry
Eligibility
18 Years and older
Enrollment
29 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2008
U.S. locations
41
States / cities
Hobson City, Alabama • Phoenix, Arizona • Los Angeles, California + 33 more
Source: ClinicalTrials.gov public record
Updated Jul 21, 2011 · Synced Sep 11, 2026, 9:08 PM EDT
Completed No phase listed Observational

Biomarkers in Patients With Rectal Cancer Undergoing Chemotherapy and Radiation Therapy

NCT00280761
View directory record Official record
Conditions
Colorectal Cancer
Interventions
capecitabine, 5-fluorouracil, Surgical Resection, Radiation therapy
Drug · Procedure · Radiation
Lead sponsor
UNC Lineberger Comprehensive Cancer Center
Other
Eligibility
18 Years and older
Enrollment
47 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003 – 2021
U.S. locations
1
States / cities
Chapel Hill, North Carolina
Source: ClinicalTrials.gov public record
Updated Aug 10, 2022 · Synced Sep 11, 2026, 9:08 PM EDT
Terminated Phase 2Phase 3 Interventional

The Toca 5 Trial: Toca 511 & Toca FC Versus Standard of Care in Patients With Recurrent High Grade Glioma

NCT02414165
View directory record Official record
Conditions
Glioblastoma Multiforme, Anaplastic Astrocytoma
Interventions
Toca 511, Toca FC, Lomustine, Temozolomide, Bevacizumab
Biological · Drug
Lead sponsor
Tocagen Inc.
Industry
Eligibility
18 Years to 75 Years
Enrollment
403 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2019
U.S. locations
50
States / cities
Phoenix, Arizona • Irvine, California • La Jolla, California + 41 more
Source: ClinicalTrials.gov public record
Updated Feb 6, 2020 · Synced Sep 11, 2026, 9:08 PM EDT
Completed Phase 1Phase 2 Interventional

A Clinical Trial to Evaluate the Safety and Efficacy of RP-L201 in Subjects With Leukocyte Adhesion Deficiency-I

NCT03812263
View directory record Official record
Conditions
Leukocyte Adhesion Defect - Type I
Interventions
RP-L201
Biological
Lead sponsor
Rocket Pharmaceuticals Inc.
Industry
Eligibility
3 Months and older
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2023
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Nov 14, 2023 · Synced Sep 11, 2026, 9:08 PM EDT
Active, not recruiting No phase listed Observational

Safety and Survival of Genetically Modified White Blood Cells in HIV-infected Twins The Gemini Study

NCT04799483
View directory record Official record
Conditions
HIV
Interventions
Not listed
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
21 Years and older
Enrollment
32 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2030
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 21, 2024 · Synced Sep 11, 2026, 9:08 PM EDT
Terminated Phase 1 Interventional

A Study of Toca 511, a Retroviral Replicating Vector, Combined With Toca FC in Patients With Solid Tumors or Lymphoma (Toca 6)

NCT02576665
View directory record Official record
Conditions
Colorectal Cancer, Triple Negative Breast Cancer, Pancreatic Cancer, Non-Small Cell Lung Cancer, Head and Neck Cancer, Ovarian Cancer, Lymphoma, Sarcoma, Bladder Cancer, Melanoma, IDH1 Mutated Solid Tumors, IDH1 Mutated or MGMT Methylated Recurrent HGG (Not Recruiting)
Interventions
Toca 511, Toca FC
Biological · Drug
Lead sponsor
Tocagen Inc.
Industry
Eligibility
18 Years to 75 Years
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2019
U.S. locations
3
States / cities
Denver, Colorado • Miami, Florida • Houston, Texas
Source: ClinicalTrials.gov public record
Updated Feb 6, 2020 · Synced Sep 11, 2026, 9:08 PM EDT
Recruiting Phase 1 Interventional

A Safety and Efficacy Study Evaluating CTX112 in Adult Subjects With Refractory Autoimmune Disease

NCT06925542
View directory record Official record
Conditions
SLE (Systemic Lupus), Lupus Erythematosus, Systemic, Lupus Nephritis, Systemic Sclerosis, Inflammatory Myopathy, Idiopathic, Myositis, Diffuse Cutaneous Systemic Sclerosis
Interventions
CTX112
Biological
Lead sponsor
CRISPR Therapeutics
Industry
Eligibility
18 Years to 70 Years
Enrollment
80 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2031
U.S. locations
9
States / cities
La Jolla, California • Redwood City, California • Chicago, Illinois + 6 more
Source: ClinicalTrials.gov public record
Updated Aug 27, 2026 · Synced Sep 11, 2026, 9:08 PM EDT
Completed No phase listed Observational

Molecular Determinants of Acquired Clinical Resistance to Crizotinib in Non-small Cell Lung Cancer Harboring a Translocation or Inversion Event Involving the ALK Gene Locus

NCT01300429
View directory record Official record
Conditions
Lung Cancer
Interventions
obtain tissue specimens
Genetic
Lead sponsor
Memorial Sloan Kettering Cancer Center
Other
Eligibility
18 Years and older
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2021
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jan 6, 2021 · Synced Sep 11, 2026, 9:08 PM EDT