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New and Updated Clinical Gene Therapy Trials
Gene therapy studies updated or received in the last 30 days at ClinicalTrials.gov. Only open studies, recruiting or not yet recruiting, are shown found by a search using the keyword: "gene therapy OR gene transfer OR virus delivery"
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Safety and Preliminary Efficacy of NXL-001 in Patients with Ischemic Stroke
07 January 2025Conditions: Ischemic Stroke Interventions: Genetic: intracerebral stereotactic injection of NXL-001 Sponsors: Beijing Tiantan Hospital Not yet recruiting
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"Studying The Role of Key Epigenetic Mediators in Breast Cancer Patients"
06 January 2025Conditions: Breast Cancer Sponsors: Ain Shams University Recruiting
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Safety and Efficacy of NRG-103 Injection in the Treatment of Recurrent Glioblastoma Patients
03 January 2025Conditions: Glioblastoma (GBM) Interventions: Drug: NRG-103 Sponsors: Zhongnan Hospital Recruiting
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Safety and Efficacy Study of PUMCH-E101 Injection in Subjects with RDH12 Retinopathy
31 December 2024Conditions: Inherited Retinal Diseases Interventions: Genetic: PUMCH-E101 Injection(Low dose); Genetic: PUMCH-E101 Injection(High dose) Sponsors: Peking Union Medical College Hospital Recruiting
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A Clinical Study of Ultra-transplantation for the Treatment of Major Thalassemia Scheme
22 January 2025Conditions: Stem Cell Transplantation Interventions: Other: Supergraft Sponsors: Hu Peng Not yet recruiting
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A Phase II Study of Adjuvant Immunotherapy Targeting KRAS G12D, KRAS G12V, or TP53 R175H for Participants With Advanced Gastrointestinal Malignancies
24 January 2025Conditions: Gastrointestinal Carcinoma; Pancreatic Cancer; Hepatocellular Cancer; Cholangiocarcinoma; Duodenal Cancer; Colorectal Cancer; Small Bowel Cancer; Metastatic Cancers Interventions: Biological: KRAS TCR-Transduced PBL; Drug: Aldesleukin; Drug: Fludarabine; Drug: Cyclophosphamide Sponsors: National Cancer Institute (NCI) Not yet recruiting
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Phase 1 Study to Evaluate Safety and Antiviral Activity of PBGENE-HBV in Adult Patients with Chronic Hepatitis B
15 January 2025Conditions: HEPATITIS B CHRONIC Interventions: Biological: PBGENE-HBV Sponsors: Precision BioSciences, Inc. Recruiting
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JAG201 Gene Therapy Study in Children & Adults with SHANK3 Haploinsufficiency
09 January 2025Conditions: SHANK3 Haploinsufficiency; Phelan-McDermid Syndrome Interventions: Genetic: JAG201; Genetic: JAG201 Sponsors: Jaguar Gene Therapy, LLC Recruiting
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A Follow-on Study for Second-Eye Treatment for Participants Previously Treated With Gene Therapy for X-Linked Retinitis Pigmentosa (XLRP)
22 January 2025Conditions: X-Linked Retinitis Pigmentosa Interventions: Biological: AAV5-hRKp.RPGR; Other: No intervention (Follow-Up assessment) Sponsors: Janssen Research & Development, LLC Recruiting
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Gene Therapy for Crigler Najjar Syndrome Type I (AlphaCN)
13 January 2025Conditions: Crigler-Najjar Syndrome Type I Interventions: Drug: GT-UGT1A1-AAV8-02 Sponsors: Federal State Budget Institution Research Center for Obstetrics, Gynecology and Perinatology Ministry of Healthcare Recruiting
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Glycogen Storage Disease Type Ia (GSDIa) Disease Monitoring Program
22 January 2025Conditions: Glycogen Storage Disease Type Ia Interventions: Other: No Intervention Sponsors: Ultragenyx Pharmaceutical Inc Recruiting
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Expanded Access Program for RP in Adults
23 January 2025Conditions: Retinitis Pigmentosa Interventions: Drug: OCU400 Sponsors: Ocugen Available
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A Study to Test How Well BI 3720931 is Tolerated and Whether it Improves Lung Function in People With Cystic Fibrosis (Lenticlair™ 1)
24 January 2025Conditions: Cystic Fibrosis Interventions: Genetic: BI 3720931; Drug: Placebo; Device: Inhaler Sponsors: Boehringer Ingelheim Recruiting
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A Study of ASP2016 in Adults Who Have Heart Disease Associated With Friedreich Ataxia
08 January 2025Conditions: Friedreich Ataxia; Cardiomyopathy Interventions: Genetic: ASP2016; Drug: Prednisolone Sponsors: Astellas Gene Therapies Suspended
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Study to Evaluate ACDN-01 in ABCA4-related Retinopathy (STELLAR)
22 January 2025Conditions: Stargardt Disease; Cone Rod Dystrophy; Juvenile Macular Degeneration; Stargardt Disease 1 Interventions: Drug: ACDN-01 Sponsors: Ascidian Therapeutics, Inc Recruiting
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Study to Evaluate Safety of RTx-015 Injection in Retinitis Pigmentosa Patients (ENVISION)
08 January 2025Conditions: Retinitis Pigmentosa Interventions: Genetic: RTx-015 Sponsors: Ray Therapeutics, Inc. Recruiting
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Clinical Exploration Trial of YOLT-101 in the Treatment of Familial Hypercholesterolemia (FH)
01 January 2025Conditions: Familial Hypercholesterolemia Interventions: Drug: YOLT-101 Sponsors: RenJi Hospital Recruiting
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Prescreening Study to Identify Potential Participants for ACDN-01 Clinical Trials
22 January 2025Conditions: Stargardt Disease; Stargardt Disease 1; Cone Rod Dystrophy; Juvenile Macular Degeneration Interventions: Diagnostic Test: Prescreening Assessments Sponsors: Ascidian Therapeutics, Inc Recruiting
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A Phase 3 Study Of OCU400 Gene Therapy for the Treatment Of Retinitis Pigmentosa
22 January 2025Conditions: Retinitis Pigmentosa Interventions: Genetic: Sub-Retinal Administration of OCU400-301 Sponsors: Ocugen Recruiting
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ReNEW:Phase 3 Study of Efficacy, Safety & Pharmacokinetics of Subcutaneous Injections of Elamipretide in Subjects With Dry Age-Related Macular Degeneration (Dry AMD)
22 January 2025Conditions: Age Related Macular Degeneration (ARMD) Interventions: Drug: Elamipretide; Drug: Placebo Sponsors: Stealth BioTherapeutics Inc. Recruiting
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ALS20-101 Lentiviral Gene Therapy for Beta Thalassemia
31 December 2024Conditions: Beta-Thalassemia Interventions: Biological: ALS20 Sponsors: Children's Hospital of Philadelphia Recruiting
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Anti-CD19 Chimeric Antigen Receptor T-Cell Immunotherapy for Chronic Lymphocytic Leukemia (CLL)
16 January 2025Conditions: B-Cell Chronic Lymphocytic Leukemia; Leukemia, Lymphocytic, Chronic, B-Cell; B-Lymphocytic Leukemia, Chronic Interventions: Biological: Autologous HuCD19 ( Anti-CD19)CAR T cells; Drug: Cyclophosphamide; Drug: Fludarabine; Drug: Rituximab Sponsors: National Cancer Institute (NCI) Recruiting
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AAV Gene Therapy Clinical Study in Adult Classic PKU (PHEdom)
08 January 2025Conditions: Phenylketonurias Interventions: Genetic: NGGT002 Sponsors: NGGT INC. Recruiting
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A Study of AAV2-GDNF in Adults With Moderate Parkinson's Disease (REGENERATE-PD)
08 January 2025Conditions: Parkinson Disease Interventions: Drug: AAV2-GDNF Gene therapy; Procedure: Control Surgery Sponsors: AskBio Inc; Bayer Recruiting
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Autologous T-cells Genetically Engineered to Express Receptors Reactive Against KRAS Mutations in Conjunction With a Vaccine Directed Against These Antigens in Participants With Metastatic Cancer
01 January 2025Conditions: Metastatic Solid Cancers; Colorectal Cancer; Breast Cancer; Non-Small Cell Lung Cancer; Gastrointestinal Cancer; Ovarian Cancer; Genitourinary Cancer Interventions: Drug: Aldesleukin; Drug: Fludarabine; Drug: Cyclophosphamide; Biological: KRAS TCR-Transduced PBL; Biological: GRT-C903/GRT-R904 Sponsors: National Cancer Institute (NCI) Recruiting
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pCCLCHIM-p47 (Lentiviral Vector Transduced CD34 Plus Cells) in Patients With p47 Autosomal Recessive Chronic Granulomatous Disease (AR-CGD)
16 January 2025Conditions: p47; Autosomal Recessive; Chronic Granulomatous Disease Interventions: Biological: Cryopreserved Autologous CD34+ cells transduced with pCCLCHIM-p47 Sponsors: National Institute of Allergy and Infectious Diseases (NIAID) Enrolling by invitation
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Immunotherapy for Adults with GPC3-Positive Solid Tumors Using IL-15 and IL-21 Armored GPC3-CAR T Cells
10 January 2025Conditions: Hepatoblastoma; Hepatocellular Carcinoma; Wilms Tumor; Malignant Rhabdoid Tumor; Yolk Sac Tumor; Rhabdomyosarcoma; Liposarcoma; Embryonal Sarcoma of Liver Interventions: Genetic: 21.15.GPC3-CAR T cells Sponsors: Baylor College of Medicine; Center for Cell and Gene Therapy, Baylor College of Medicine Not yet recruiting
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A Study About Antibody Levels and Biomarkers in the Blood in People With Late-onset Pompe Disease
23 January 2025Conditions: Pompe Disease (Late-onset) Interventions: Other: No Intervention Sponsors: Astellas Gene Therapies Recruiting
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A Study of SGT-003 Gene Therapy in Duchenne Muscular Dystrophy (INSPIRE DUCHENNE)
17 January 2025Conditions: Duchenne Muscular Dystrophy Interventions: Genetic: SGT-003 Sponsors: Solid Biosciences Inc. Recruiting
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Gene Therapy for ACM Due to a PKP2 Pathogenic Variant
23 January 2025Conditions: Arrhythmogenic Cardiomyopathy; PKP2-ACM; PKP2-ARVC Interventions: Genetic: LX2020 Sponsors: Lexeo Therapeutics Recruiting
Search Clinical Gene Therapy Trials
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Clinical gene therapy trials recently received at ClinicalTrials.gov database.
The U.S. National Institutes of Health, through its National Library of Medicine, has developed ClinicalTrials.gov to provide patients, family members and members of the public current information about clinical research studies. The database is a registry of federally and privately supported clinical trials conducted in the United States and around the world. ClinicalTrials.gov gives you information about a trial's purpose, who may participate, locations, and phone numbers for more details.
Latest clinical trials | |
Search clinical trials |
The U.S. National Institutes of Health, through its National Library of Medicine, has developed ClinicalTrials.gov to provide patients, family members and members of the public current information about clinical research studies. The database is a registry of federally and privately supported clinical trials conducted in the United States and around the world. ClinicalTrials.gov gives you information about a trial's purpose, who may participate, locations, and phone numbers for more details.
Participate in Trial
Are you a patient and considering to participate in a clinical gene therapy trial? Your doctor may be an excellent resource to help identify clinical trials for a particular disease. There are a number of online resources that can also assist in locating clinical trials that are accepting patients:
ClinicalTrials.gov | |
Cancer.gov | |
CenterWatch |
When looking for a clinical trial, remember to make sure the trial is appropriate for the disease. Most trials also specify an age range for the participants. Additional eligibility requirements must also be met, but these will vary depending on the individual study. Contact the health professionals if they are not listed. It is always best to discuss these issues before you travel to gene therapy center. More clinical trial information.
Be aware that Gene Therapy Net is not intended to replace or constitute the giving of medical treatments or advice.