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Showing 25–48 of 24 matching trials from the live ClinicalTrials.gov search.
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Completed Phase 1Phase 2 Interventional

A Study of Metastatic Gastrointestinal Cancers Treated With Tumor Infiltrating Lymphocytes in Which the Gene Encoding the Intracellular Immune Checkpoint CISH Is Inhibited Using CRISPR Genetic Engineering

NCT04426669
View directory record Official record
Conditions
Gastrointestinal Epithelial Cancer, Gastrointestinal Neoplasms, Cancer of Gastrointestinal Tract, Cancer, Gastrointestinal, Gastrointestinal Cancer, Colo-rectal Cancer, Pancreatic Cancer, Gall Bladder Cancer, Colon Cancer, Esophageal Cancer, Stomach Cancer
Interventions
Cyclophosphamide, Fludarabine, Tumor-Infiltrating Lymphocytes (TIL), Aldesleukin
Drug · Biological
Lead sponsor
Intima Bioscience, Inc.
Industry
Eligibility
18 Years to 70 Years
Enrollment
23 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2026
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Feb 22, 2026 · Synced Sep 4, 2026, 1:56 AM EDT
Enrolling by invitation No phase listed Observational

A Long-Term Follow-Up Study in Severe Hemophilia A Subjects Who Received BMN 270 in a Prior BioMarin Clinical Trial (270-401)

NCT05768386
View directory record Official record
Conditions
Hemophilia A
Interventions
Not listed
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
18 Years and older · Male only
Enrollment
172 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2040
U.S. locations
13
States / cities
Los Angeles, California • Sacramento, California • San Diego, California + 10 more
Source: ClinicalTrials.gov public record
Updated Jan 11, 2026 · Synced Sep 4, 2026, 1:56 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 13, 2017 · Synced Sep 4, 2026, 1:56 AM EDT
Completed Phase 1Phase 2 Interventional Results available

Gene Therapy for Painful Diabetic Neuropathy

NCT01002235
View directory record Official record
Conditions
Painful Diabetic Peripheral Neuropathy
Interventions
VM202
Biological
Lead sponsor
Helixmith Co., Ltd.
Industry
Eligibility
18 Years to 75 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2012
U.S. locations
2
States / cities
Walnut Creek, California • Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Oct 5, 2025 · Synced Sep 4, 2026, 1:56 AM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

In Vitro Fertilization (IVF) and Prenatal Effects Independent of Genetics

NCT07296107
View directory record Official record
Conditions
Maternal Distress, Child Development
Interventions
Prenatal maternal psychosocial and biological assessment protocol
Other
Lead sponsor
Columbia University
Other
Eligibility
18 Years to 50 Years · Female only
Enrollment
360 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2026 – 2030
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jul 6, 2026 · Synced Sep 4, 2026, 1:56 AM EDT
Terminated Phase 1Phase 2 Interventional Results available

Open Label, Study Of Efficacy and Safety Of AVR-RD-01 for Treatment-Naive Subjects With Classic Fabry Disease

NCT03454893
View directory record Official record
Conditions
Fabry Disease
Interventions
AVR-RD-01
Drug
Lead sponsor
AVROBIO
Industry
Eligibility
16 Years to 50 Years · Male only
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2022
U.S. locations
2
States / cities
Hackensack, New Jersey • Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jan 4, 2024 · Synced Sep 4, 2026, 1:56 AM EDT
Active, not recruiting Phase 1 Interventional

A Long-term Follow up Study of EXG102-031 in Patients With wAMD (Everest LTFU)

NCT06817343
View directory record Official record
Conditions
Neovascular Age-Related Macular Degeneration (nAMD), Neovascular (Wet) Age-related Macular Degeneration (AMD)
Interventions
Aflibercept (2.0 mg)
Biological
Lead sponsor
Exegenesis Bio
Industry
Eligibility
Not listed
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2029
U.S. locations
2
States / cities
Reno, Nevada • Erie, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Apr 7, 2026 · Synced Sep 4, 2026, 1:56 AM EDT
Active, not recruiting Phase 3 Interventional

A Trial to Learn More About an Experimental Gene Therapy Called Bidridistrogene Xeboparvovec (SRP-9003) as a Possible Treatment for Limb Girdle Muscular Dystrophy 2E/R4

NCT06246513
View directory record Official record
Conditions
Limb-girdle Muscular Dystrophy
Interventions
SRP-9003, Glucocorticoid
Biological · Drug
Lead sponsor
Sarepta Therapeutics, Inc.
Industry
Eligibility
4 Years and older
Enrollment
17 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2029
U.S. locations
4
States / cities
La Jolla, California • Columbus, Ohio • Philadelphia, Pennsylvania + 1 more
Source: ClinicalTrials.gov public record
Updated Mar 1, 2026 · Synced Sep 4, 2026, 1:56 AM EDT
Enrolling by invitation Phase 1Phase 2 Interventional

Base-Edited Hematopoietic Stem/Progenitor Cell X-Linked Severe Combined Immunodeficiency Gene Therapy

NCT06851767
View directory record Official record
Conditions
X-linked Severe Combined Immunodeficiency, X-SCID, XSCID
Interventions
Plerixafor, Filgrastim, Palifermin, Busulfan, Base-edited hematopoietic stem and progenitor cells
Genetic · Drug · Biological
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
3 Years to 99 Years · Male only
Enrollment
18 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2034
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 2, 2026 · Synced Sep 4, 2026, 1:56 AM EDT
Completed Phase 2 Interventional

Gene Therapy in Treating Patients With Stage III or Stage IV Melanoma

NCT00003646
View directory record Official record
Conditions
Stage IV Melanoma, Stage III Melanoma, Recurrent Melanoma
Interventions
allovectin-7
Drug
Lead sponsor
Vical
Industry
Eligibility
18 Years and older
Enrollment
78 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1998 – 2002
U.S. locations
3
States / cities
Rochester, Minnesota • Buffalo, New York • Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Jul 6, 2011 · Synced Sep 4, 2026, 1:56 AM EDT
Active, not recruiting Phase 3 Interventional

A Study to Investigate the Efficacy and Safety of OTL-203 in Subjects With MPS-IH Compared With Standard of Care With Allogeneic HSCT

NCT06149403
View directory record Official record
Conditions
MPS-IH (Hurler Syndrome)
Interventions
Experimental: OTL-203, Active Comparator: Allo-HSCT
Genetic
Lead sponsor
Orchard Therapeutics
Industry
Eligibility
28 Days to 30 Months
Enrollment
41 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2031
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Dec 21, 2025 · Synced Sep 4, 2026, 1:56 AM EDT
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 4, 2026, 1:56 AM EDT
Enrolling by invitation Phase 1Phase 2 Interventional

Phase 1/2 Study of FRF-001, an AAV-9 Gene Therapy, in Patients With FOXG1 Syndrome (FS)

NCT07293546
View directory record Official record
Conditions
FOXG1 Syndrome
Interventions
FRF-001
Genetic
Lead sponsor
FOXG1 Research Foundation
Other
Eligibility
2 Years to 20 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2029
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Jul 23, 2026 · Synced Sep 4, 2026, 1:56 AM EDT
Completed Phase 3 Interventional Results available

HOPE-B: Trial of AMT-061 in Severe or Moderately Severe Hemophilia B Patients

NCT03569891
View directory record Official record
Conditions
Hemophilia B
Interventions
AAV5-hFIXco-Padua, Factor IX (FIX)
Genetic · Biological
Lead sponsor
CSL Behring
Industry
Eligibility
18 Years and older · Male only
Enrollment
67 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2025
U.S. locations
19
States / cities
Phoenix, Arizona • Little Rock, Arkansas • Los Angeles, California + 14 more
Source: ClinicalTrials.gov public record
Updated Mar 26, 2026 · Synced Sep 4, 2026, 1:56 AM EDT
Withdrawn Phase 3 Interventional

Study of a Gene Therapy Treatment for Hemophilia A

NCT06297486
View directory record Official record
Conditions
Hemophilia A
Interventions
SPK-8011
Genetic
Lead sponsor
Spark Therapeutics, Inc.
Industry
Eligibility
18 Years and older · Male only
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2035
U.S. locations
27
States / cities
Phoenix, Arizona • Loma Linda, California • Los Angeles, California + 23 more
Source: ClinicalTrials.gov public record
Updated Dec 12, 2024 · Synced Sep 4, 2026, 1:56 AM EDT
Terminated Phase 1Phase 2 Interventional Results available

CAR T Cell Receptor Immunotherapy Targeting Mesothelin for Patients With Metastatic Cancer

NCT01583686
View directory record Official record
Conditions
Cervical Cancer, Pancreatic Cancer, Ovarian Cancer, Mesothelioma, Lung Cancer
Interventions
Fludarabine, Anti-mesothelin chimeric T cell receptor (CAR) transduced peripheral blood lymphocytes (PBL), Cyclophosphamide, Aldesleukin
Drug · Biological
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
18 Years to 70 Years
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2018
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 13, 2019 · Synced Sep 4, 2026, 1:56 AM EDT
Completed Phase 1 Interventional

Gene-Modified T Cells, Vaccine Therapy, and Nivolumab in Treating Patients With Stage IV or Locally Advanced Solid Tumors Expressing NY-ESO-1

NCT02775292
View directory record Official record
Conditions
Adult Solid Neoplasm, Childhood Solid Neoplasm, Metastatic Neoplasm
Interventions
Aldesleukin, Cyclophosphamide, Fludarabine Phosphate, Laboratory Biomarker Analysis, Nivolumab, NY-ESO-1 Reactive TCR Retroviral Vector Transduced Autologous PBL, NY-ESO-1(157-165) Peptide-pulsed Autologous Dendritic Cell Vaccine, Positron Emission Tomography
Biological · Drug · Other + 1 more
Lead sponsor
Jonsson Comprehensive Cancer Center
Other
Eligibility
16 Years and older
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2019
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Jul 29, 2025 · Synced Sep 4, 2026, 1:56 AM EDT
Not listed No phase listed Observational Accepts healthy volunteers

Skin Barrier Assessment in Pregnancy and at Birth

NCT04445298
View directory record Official record
Conditions
Pregnancy Related, Allergy, Atopic Dermatitis Eczema, Asthma in Children, Food Allergy
Interventions
Transepidermal water loss, Skin tape stripping, Blood draw, Bacterial PCR swab
Diagnostic Test
Lead sponsor
National Jewish Health
Other
Eligibility
0 Minutes to 55 Years
Enrollment
160 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2020 – 2022
U.S. locations
1
States / cities
Denver, Colorado
Source: ClinicalTrials.gov public record
Updated Sep 9, 2020 · Synced Sep 4, 2026, 1:56 AM EDT
Terminated Phase 1 Interventional Results available

Pilot Study of GVAX in Colorectal Cancer Cells

NCT01952730
View directory record Official record
Conditions
Colorectal Cancer
Interventions
GVAX
Biological
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
18 Years and older
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2020
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Jan 11, 2023 · Synced Sep 4, 2026, 1:56 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 Sep 4, 2026, 1:56 AM EDT
Completed Phase 1 Interventional

Re-administration of Intramuscular AAV9 in Patients With Late-Onset Pompe Disease

NCT02240407
View directory record Official record
Conditions
Pompe Disease
Interventions
Recombinant Adeno-Associated Virus Acid Alpha-Glucosidase, Rapamycin, saline, Rituxan, Diphenhydramine, Acetaminophen, Lidocaine, LMX 4 Topical Cream
Genetic · Drug · Other
Lead sponsor
University of Florida
Other
Eligibility
18 Years to 50 Years
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2021
U.S. locations
1
States / cities
Gainesville, Florida
Source: ClinicalTrials.gov public record
Updated Apr 4, 2022 · Synced Sep 4, 2026, 1:56 AM EDT
Recruiting Phase 1 Interventional

CD4^LVFOXP3 in Participants With IPEX

NCT05241444
View directory record Official record
Conditions
IPEX
Interventions
CD4^LVFOXP3
Biological
Lead sponsor
Bacchetta, Rosa, MD
Other
Eligibility
4 Months to 35 Years · Male only
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2037
U.S. locations
1
States / cities
Palo Alto, California
Source: ClinicalTrials.gov public record
Updated May 21, 2025 · Synced Sep 4, 2026, 1:56 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Peripheral Blood Stem Cells Obtained From Normal Volunteers for Studying Retroviral Vector Mediated Gene Transfer Into Primitive Hematopoietic Cells and Vector Mediated Transgene Expression in Mature Hematopoietic Lineages

NCT00758992
View directory record Official record
Conditions
Immunodeficiency
Interventions
Not listed
Lead sponsor
St. Jude Children's Research Hospital
Other
Eligibility
18 Years to 40 Years
Enrollment
14 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2006 – 2015
U.S. locations
1
States / cities
Memphis, Tennessee
Source: ClinicalTrials.gov public record
Updated Mar 13, 2016 · Synced Sep 4, 2026, 1:56 AM EDT
Active, not recruiting Phase 1Phase 2 Interventional

4D-125 in Patients With X-Linked Retinitis Pigmentosa (XLRP)

NCT04517149
View directory record Official record
Conditions
X-Linked Retinitis Pigmentosa
Interventions
4D-125 IVT Injection, Observational
Biological · Other
Lead sponsor
4D Molecular Therapeutics
Industry
Eligibility
12 Years and older · Male only
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2029
U.S. locations
8
States / cities
Aurora, Colorado • Gainesville, Florida • Ann Arbor, Michigan + 5 more
Source: ClinicalTrials.gov public record
Updated Mar 20, 2025 · Synced Sep 4, 2026, 1:56 AM EDT