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ClinicalTrials.gov public records Last synced Oct 30, 2026, 1:57 AM EDT

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Showing 1–24 of 1,031 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Therapy, Gene Clear all
Terminated Phase 1 Interventional

Gene Therapy and Chemotherapy in Treating Patients With Advanced Solid Tumors or Non-Hodgkin's Lymphoma

NCT00003567
View directory record Official record
Conditions
Brain and Central Nervous System Tumors, Lymphoma, Unspecified Adult Solid Tumor, Protocol Specific
Interventions
filgrastim, sargramostim, therapeutic autologous lymphocytes, O6-benzylguanine, carmustine, temozolomide, in vitro-treated peripheral blood stem cell transplantation
Biological · Drug · Procedure
Lead sponsor
Case Comprehensive Cancer Center
Other
Eligibility
18 Years to 70 Years
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1999 – 2007
U.S. locations
1
States / cities
Cleveland, Ohio
Source: ClinicalTrials.gov public record
Updated Jun 11, 2010 · Synced Oct 30, 2026, 1:57 AM EDT
Completed Phase 1 Interventional

Assessing the Suitability of an Imaging Probe for Use in Clinical Cell and Gene Therapy Trials in Cancer and Rheumatoid Arthritis

NCT00185848
View directory record Official record
Conditions
Brain Cancer, Arthritis, Rheumatoid, Central Nervous System Neoplasms, Glioma
Interventions
[18F]FHBG
Radiation
Lead sponsor
Sanjiv Sam Gambhir
Other
Eligibility
18 Years and older
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2013
U.S. locations
2
States / cities
Los Angeles, California • Stanford, California
Source: ClinicalTrials.gov public record
Updated Mar 14, 2017 · Synced Oct 30, 2026, 1:57 AM EDT
Completed Phase 1 Interventional

Gene Therapy in Treating Patients With Advanced Recurrent or Persistent Ovarian Cancer or Primary Peritoneal Cancer

NCT00003450
View directory record Official record
Conditions
Ovarian Cancer, Peritoneal Cavity Cancer
Interventions
Ad5CMV-p53 gene
Biological
Lead sponsor
University of Texas Southwestern Medical Center
Other
Eligibility
18 Years to 120 Years · Female only
Healthy volunteers
Healthy volunteers not accepted
Timeline
1998 – 1999
U.S. locations
1
States / cities
Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Feb 2, 2021 · Synced Oct 30, 2026, 1:57 AM EDT
Enrolling by invitation No phase listed Observational

Long-term Follow-up of Subjects with Sickle Cell Disease Treated with Ex Vivo Gene Therapy

NCT04628585
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Safety and efficacy assessments
Other
Lead sponsor
Genetix Biotherapeutics Inc.
Industry
Eligibility
2 Years to 53 Years
Enrollment
85 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2038
U.S. locations
15
States / cities
Birmingham, Alabama • Oakland, California • Atlanta, Georgia + 12 more
Source: ClinicalTrials.gov public record
Updated Mar 20, 2025 · Synced Oct 30, 2026, 1:57 AM EDT
Recruiting Phase 1Phase 2 Interventional

AMT-260 Gene Therapy Study in Adults With Unilateral Refractory Mesial Temporal Lobe Epilepsy

NCT06063850
View directory record Official record
Conditions
Mesial Temporal Lobe Epilepsy
Interventions
AAV9-hSyn1-miGRIK2
Genetic
Lead sponsor
UniQure Biopharma B.V.
Industry
Eligibility
18 Years to 75 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2031
U.S. locations
18
States / cities
Birmingham, Alabama • Phoenix, Arizona • Palo Alto, California + 15 more
Source: ClinicalTrials.gov public record
Updated Aug 24, 2026 · Synced Oct 30, 2026, 1:57 AM EDT
Terminated Phase 1 Interventional

Interleukin-12 Gene Therapy in Treating Patients With Skin Metastases

NCT00028652
View directory record Official record
Conditions
Metastatic Cancer
Interventions
interleukin-12 gene
Biological
Lead sponsor
University of Wisconsin, Madison
Other
Eligibility
18 Years to 120 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2002
U.S. locations
1
States / cities
Madison, Wisconsin
Source: ClinicalTrials.gov public record
Updated Dec 13, 2019 · Synced Oct 30, 2026, 1:57 AM EDT
Not listed Phase 1 Interventional

Vaccine Therapy and Ganciclovir in Treating Patients With Mesothelioma

NCT00006216
View directory record Official record
Conditions
Malignant Mesothelioma
Interventions
PA-1-STK ovarian carcinoma vaccine, ganciclovir
Biological · Drug
Lead sponsor
Louisiana State University Health Sciences Center in New Orleans
Other
Eligibility
Not listed
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1997
U.S. locations
1
States / cities
New Orleans, Louisiana
Source: ClinicalTrials.gov public record
Updated Sep 20, 2013 · Synced Oct 30, 2026, 1:57 AM EDT
Completed Phase 2 Interventional Results available

Tazemetostat in Treating Patients With Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphoma, or Histiocytic Disorders With EZH2, SMARCB1, or SMARCA4 Gene Mutations (A Pediatric MATCH Treatment Trial)

NCT03213665
View directory record Official record
Conditions
Advanced Malignant Solid Neoplasm, Ann Arbor Stage III Hodgkin Lymphoma, Ann Arbor Stage III Non-Hodgkin Lymphoma, Ann Arbor Stage IV Hodgkin Lymphoma, Ann Arbor Stage IV Non-Hodgkin Lymphoma, Ewing Sarcoma/Peripheral Primitive Neuroectodermal Tumor, Low Grade Glioma, Recurrent Ependymoma, Recurrent Ewing Sarcoma, Recurrent Glioma, Recurrent Hepatoblastoma, Recurrent Hodgkin Lymphoma, Recurrent Langerhans Cell Histiocytosis, Recurrent Malignant Germ Cell Tumor, Recurrent Malignant Glioma, Recurrent Malignant Solid Neoplasm, Recurrent Medulloblastoma, Recurrent Neuroblastoma, Recurrent Non-Hodgkin Lymphoma, Recurrent Osteosarcoma, Recurrent Peripheral Primitive Neuroectodermal Tumor, Recurrent Primary Central Nervous System Neoplasm, Recurrent Rhabdoid Tumor, Recurrent Rhabdomyosarcoma, Recurrent Soft Tissue Sarcoma, Refractory Hodgkin Lymphoma, Refractory Langerhans Cell Histiocytosis, Refractory Malignant Germ Cell Tumor, Refractory Malignant Glioma, Refractory Malignant Solid Neoplasm, Refractory Medulloblastoma, Refractory Neuroblastoma, Refractory Non-Hodgkin Lymphoma, Refractory Osteosarcoma, Refractory Peripheral Primitive Neuroectodermal Tumor, Refractory Rhabdoid Tumor, Refractory Soft Tissue Sarcoma, Rhabdoid Tumor, Stage III Soft Tissue Sarcoma AJCC v7, Stage IV Soft Tissue Sarcoma AJCC v7, Wilms Tumor
Interventions
Tazemetostat
Drug
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
12 Months to 21 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2024
U.S. locations
115
States / cities
Birmingham, Alabama • Anchorage, Alaska • Mesa, Arizona + 91 more
Source: ClinicalTrials.gov public record
Updated Apr 13, 2026 · Synced Oct 30, 2026, 1:57 AM EDT
Completed Phase 1 Interventional

Gene Therapy in Treating Patients With Advanced Bladder Cancer

NCT00003167
View directory record Official record
Conditions
Recurrent Bladder Cancer, Stage I Bladder Cancer, Stage II Bladder Cancer, Stage III Bladder Cancer, Stage IV Bladder Cancer, Transitional Cell Carcinoma of the Bladder
Interventions
Ad5CMV-p53 gene
Biological
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
18 Years and older
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1998
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Jan 23, 2013 · Synced Oct 30, 2026, 1:57 AM EDT
Recruiting Phase 1Phase 2 Interventional

A Study of DB-OTO, an Adeno-Associated Virus (AAV) Based Gene Therapy, in Children/Infants, Adolescents and Adults With Hearing Loss Due to Otoferlin Mutations

NCT05788536
View directory record Official record
Conditions
Congenital Hearing Loss Secondary to Biallelic Mutations of the Otoferlin Gene (OTOF)
Interventions
DB-OTO
Genetic
Lead sponsor
Regeneron Pharmaceuticals
Industry
Eligibility
Not listed
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2032
U.S. locations
9
States / cities
Los Angeles, California • San Diego, California • Jacksonville, Florida + 6 more
Source: ClinicalTrials.gov public record
Updated Aug 5, 2026 · Synced Oct 30, 2026, 1:57 AM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

Gene Therapy for Gaucher's and Fabry Disease Using Viruses and Blood-Forming Cells

NCT00001234
View directory record Official record
Conditions
Gaucher's Disease
Interventions
human glucocerebrosidase cDNA
Genetic
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
Not listed
Enrollment
120 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
1988 – 2002
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 4, 2008 · Synced Oct 30, 2026, 1:57 AM EDT
Recruiting Phase 1Phase 2 Interventional

Safety, PK/PD, and Exploratory Efficacy Study of AMT-191 in Classic Fabry Disease

NCT06270316
View directory record Official record
Conditions
Fabry Disease
Interventions
AMT-191
Drug
Lead sponsor
UniQure Biopharma B.V.
Industry
Eligibility
18 Years to 65 Years · Male only
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2031
U.S. locations
10
States / cities
Birmingham, Alabama • Los Angeles, California • Atlanta, Georgia + 7 more
Source: ClinicalTrials.gov public record
Updated Sep 24, 2026 · Synced Oct 30, 2026, 1:57 AM EDT
Recruiting Phase 2Phase 3 Interventional

Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH

NCT02716246
View directory record Official record
Conditions
MPS IIIA, Sanfilippo Syndrome, Sanfilippo A, Mucopolysaccharidosis III
Interventions
UX111, Prophylactic Immunomodulatory (IM) Therapy, Optimized Prophylactic IM Therapy, Adjuvant IM Therapy
Biological · Drug
Lead sponsor
Ultragenyx Pharmaceutical Inc
Industry
Eligibility
Not listed
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2029
U.S. locations
2
States / cities
Columbus, Ohio • Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Sep 22, 2026 · Synced Oct 30, 2026, 1:57 AM EDT
Recruiting Phase 4 Interventional

ASsessing The REAl-world Safety & Effectiveness of Spinal Muscular Atrophy Participants Treated With Intrathecal Onasemnogene Abeparvovec-brve (OAV101B) (ITVISMA®): A U.S. Pragmatic Multicenter Study (STREAM)

NCT07448610
View directory record Official record
Conditions
Spinal Muscular Atrophy
Interventions
Onasemnogene Abeparvovec-brve
Drug
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
2 Years to 100 Years
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2032
U.S. locations
6
States / cities
Little Rock, Arkansas • Stanford, California • Boston, Massachusetts + 3 more
Source: ClinicalTrials.gov public record
Updated Oct 29, 2026 · Synced Oct 30, 2026, 1:57 AM EDT
Terminated Phase 1 Interventional

Gene Therapy for Fanconi Anemia

NCT01331018
View directory record Official record
Conditions
Fanconi Anemia
Interventions
Bone Marrow Aspiration, Filgrastim, Genetically Engineered Hematopoietic Stem Progenitor Cells, Laboratory Biomarker Analysis, Leukapheresis, Methylprednisolone, Plerixafor, Prednisone
Procedure · Biological · Other + 1 more
Lead sponsor
Fred Hutchinson Cancer Center
Other
Eligibility
4 Years and older
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2024
U.S. locations
1
States / cities
Seattle, Washington
Source: ClinicalTrials.gov public record
Updated May 17, 2024 · Synced Oct 30, 2026, 1:57 AM EDT
Withdrawn No phase listed Observational

Prognostic Value of Androgen Receptor Expression and Mutations Within Oncogenes and Tumor Suppressor Genes in Patients Treated for High Risk Prostate Cancer With Proton Therapy (PRX32)

NCT03296124
View directory record Official record
Conditions
Prostate Cancer
Interventions
Not listed
Lead sponsor
University of Florida
Other
Eligibility
18 Years and older · Male only
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2020
U.S. locations
1
States / cities
Jacksonville, Florida
Source: ClinicalTrials.gov public record
Updated Nov 16, 2018 · Synced Oct 30, 2026, 1:57 AM EDT
Completed Phase 1 Interventional Results available

Safety Study of Adenovirus/PNP Coupled With Fludarabine Phosphate to Treat Solid Tumors

NCT01310179
View directory record Official record
Conditions
Head and Neck Cancer
Interventions
Ad/PNP and fludarabine monophosphate
Genetic
Lead sponsor
PNP Therapeutics, Inc.
Industry
Eligibility
19 Years and older
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2014
U.S. locations
2
States / cities
Birmingham, Alabama • Nashville, Tennessee
Source: ClinicalTrials.gov public record
Updated Jun 10, 2015 · Synced Oct 30, 2026, 1:57 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 30, 2026, 1:57 AM EDT
Active, not recruiting No phase listed Observational

Blood Biomarkers in Pediatric Kidney Transplant Recipients

NCT05477082
View directory record Official record
Conditions
Rejection of Kidney Allograft
Interventions
Observational group
Other
Lead sponsor
University of Minnesota
Other
Eligibility
Up to 21 Years
Enrollment
27 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2026
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Dec 17, 2025 · Synced Oct 30, 2026, 1:57 AM EDT
Completed Phase 1 Interventional

Stem Cell (Modified Bone Marrow) Transplantation in HIV-Infected Patients With Blood Cancer

NCT00005785
View directory record Official record
Conditions
Hematologic Neoplasm, HIV Infection
Interventions
GCSF Mobilized Allogeneic PBSC Cultured w/Cytokines; Transduced w/RV
Drug
Lead sponsor
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
NIH
Eligibility
Not listed
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1999 – 2001
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 4, 2008 · Synced Oct 30, 2026, 1:57 AM EDT
Completed Phase 3 Interventional Results available

A Study of ASP2215 (Gilteritinib) by Itself, ASP2215 Combined With Azacitidine or Azacitidine by Itself to Treat Adult Patients Who Have Recently Been Diagnosed With Acute Myeloid Leukemia With a FLT3 Gene Mutation and Who Cannot Receive Standard Chemotherapy

NCT02752035
View directory record Official record
Conditions
Acute Myeloid Leukemia (AML), Acute Myeloid Leukemia With FMS-like Tyrosine Kinase (FLT3) Mutation
Interventions
gilteritinib, azacitidine
Drug
Lead sponsor
Astellas Pharma Global Development, Inc.
Industry
Eligibility
18 Years and older
Enrollment
183 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2024
U.S. locations
12
States / cities
Los Angeles, California • Orange, California • Chicago, Illinois + 7 more
Source: ClinicalTrials.gov public record
Updated Jul 14, 2026 · Synced Oct 30, 2026, 1:57 AM EDT
Completed Phase 1 Interventional

Safety Study of a Gene Transfer Vector for Children With Late Infantile Neuronal Ceroid Lipofuscinosis

NCT00151216
View directory record Official record
Conditions
Batten Disease, Late Infantile Neuronal Ceroid Lipofuscinosis
Interventions
AAV2CUhCLN2 (3x10^12 particle units)
Biological
Lead sponsor
Weill Medical College of Cornell University
Other
Eligibility
3 Years to 18 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2019
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jul 24, 2020 · Synced Oct 30, 2026, 1:57 AM EDT
Recruiting Phase 1 Interventional

Study Assessing Left Ventricular Administration of a Genetic Medicine Directing Organ Regeneration in Heart Failure

NCT06831825
View directory record Official record
Conditions
Heart Failure With Reduced Ejection Fraction
Interventions
YAP101 (AAV9-Sav-shRNA)
Combination Product
Lead sponsor
Medley Therapeutics
Industry
Eligibility
18 Years to 79 Years
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Apr 27, 2025 · Synced Oct 30, 2026, 1:57 AM EDT
Completed No phase listed Observational Results available

Changes in Gene Transcription and Immunophenotypes Following Mepolizumab Treatment for Asthma

NCT05002621
View directory record Official record
Conditions
Eosinophilic Asthma
Interventions
Observational
Other
Lead sponsor
Mayo Clinic
Other
Eligibility
18 Years and older
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2025
U.S. locations
1
States / cities
Rochester, Minnesota
Source: ClinicalTrials.gov public record
Updated Mar 18, 2026 · Synced Oct 30, 2026, 1:57 AM EDT