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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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A Clinical Gene Therapy Study with Hematopoietic Stem Cells for the Treatment, with Single Dose of Temferon, of Patients Suffering from Metastatic Renal Cell Carcinoma
04 December 2024Conditions: Clear Cell RCC Interventions: Genetic: Temferon; Biological: Pembrolizumab; Drug: Cabozantinib Sponsors: Genenta Science; Alira Health; Arithmos srl; Ospedale San Raffaele Recruiting
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Safety and Efficacy Study of NGGT001 in Bietti Crystalline Corneoretinal Dystrophy Subjects
26 November 2024Conditions: Bietti Crystalline Corneoretinal Dystrophy Interventions: Biological: NGGT001 Sponsors: NGGT (Suzhou) Biotechnology Co., Ltd. Active, not recruiting
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Identifying Tissue-of-origin in Transplant Patients and Patients with Malignancies.
25 November 2024Conditions: Malignancies Sponsors: Hadassah Medical Organization Active, not recruiting
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Study to Evaluate the Efficacy and Safety of VGR-R01 Gene Therapy in Patients with Bietti Crystalline Dystrophy
21 November 2024Conditions: Bietti Crystalline Dystrophy; Inherited Retinal Diseases Interventions: Drug: VGR-R01 Sponsors: Shanghai Vitalgen BioPharma Co., Ltd. Not yet recruiting
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Long Term Follow-up Study of AAVAnc80-hOTOF Gene Therapy
20 November 2024Conditions: Otoferlin Gene-mediated Hearing Loss Sponsors: Akouos, Inc.; Eli Lilly and Company Enrolling by invitation
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Phase 3, Open-label Study to Assess the Efficacy and Safety of a Single Lumbar Intrathecal Administration of MELPIDA in Individuals with Hereditary Spastic Paraplegia Type 50 (SPG50) Versus Matched Prospective Concurrent Controls.
18 November 2024Conditions: Hereditary Spastic Paraplegia Type 50 Interventions: Genetic: MELPIDA Sponsors: Elpida Therapeutics SPC 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
26 November 2024Conditions: 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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A Study to Evaluate the Safety and Efficacy of A2B395, an Allogeneic Logic-gated CAR T, in Participants With Solid Tumors That Express EGFR and Have Lost HLA-A*02 Expression
12 November 2024Conditions: Solid Tumor, Adult; Colorectal Cancer; Non-Small Cell Lung; NSCLC (Non-small Cell Lung Cancer); Cancer; Colon Cancer; Rectal Cancer; Lung Cancer; CRC; Head and Neck Squamous Cell Cancer; HNSCC; Renal Cell Carcinoma; RCC; Kidney Cancer; Triple Negative Breast Cancer; TNBC; Colorectal Adenocarcinoma Interventions: Biological: A2B395; Diagnostic Test: xT CDx with HLA-LOH assay Sponsors: A2 Biotherapeutics Inc. 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
14 November 2024Conditions: HEPATITIS B CHRONIC Interventions: Biological: PBGENE-HBV Sponsors: Precision BioSciences, Inc. Recruiting
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Patient Interview Study to Explore the Impact of Gene Therapy in Hemophilia A & B
13 November 2024Conditions: Hemophilia A; Hemophilia B Sponsors: Pfizer Not yet recruiting
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Glycogen Storage Disease Type Ia (GSDIa) Disease Monitoring Program
25 November 2024Conditions: Glycogen Storage Disease Type Ia Interventions: Other: No Intervention Sponsors: Ultragenyx Pharmaceutical Inc Recruiting
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A Multicentric European Study to Promote Clinical Trial Readiness for STXBP1-related Disorders
18 November 2024Conditions: STXBP1 Encephalopathy with Epilepsy Sponsors: Kelsey Ax; Filadelfia Epilepsy Hospital; University Hospital Heidelberg; University Hospital Antwerp, BE; Istituto Giannina Gaslini, Genoa, Italy; Hospital Sant Joan de Deu; Amsterdam UMC, location VUmc; Sheba Medical Center; Aix Marseille Université Not yet recruiting
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5-Azacytidine with Steroids for Gastrointestinal GVHD (5-AZA for GVHD)
04 December 2024Conditions: Graft-Versus-Host Disease(GVHD) Interventions: Drug: 5-Azacytidine Sponsors: Baylor College of Medicine; The Methodist Hospital Research Institute; Center for Cell and Gene Therapy, Baylor College of Medicine Not yet recruiting
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Expanded Access Program for RP in Adults
22 November 2024Conditions: Retinitis Pigmentosa Interventions: Drug: OCU400 Sponsors: Ocugen Available
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A Study of the Safety and Efficacy of Prime Editing (PM359) in Participants with P47phox Autosomal Recessive Chronic Granulomatous Disease (CGD )
15 November 2024Conditions: Chronic Granulomatous Disease; Granulomatous Disease, Chronic Interventions: Biological: PM359 Sponsors: Prime Medicine, Inc. Recruiting
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A Trial to Evaluate Intravesical Nadofaragene Firadenovec Alone or in Combination With Chemotherapy or Immunotherapy in Participants With High-grade BCG Unresponsive Non-muscle Invasive Bladder Cancer
14 November 2024Conditions: Non-muscle Invasive Bladder Cancer With Carcinoma in Situ Interventions: Drug: Nadofaragene Firadenovec; Drug: Gemcitabine; Drug: Docetaxel; Drug: Pembrolizumab Sponsors: Ferring Pharmaceuticals Recruiting
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A Phase 1/2 Trial of SP-101 for the Treatment of Cystic Fibrosis (CF)
25 November 2024Conditions: Cystic Fibrosis Interventions: Combination Product: SP-101 and doxorubicin Cohort 1; Combination Product: SP-101 and doxorubicin Cohort 2 Sponsors: Spirovant Sciences, Inc. Recruiting
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Efficacy and Safety of GNT0003 Following Imlifidase Pre-treatment in Severe Crigler-Najjar Syndrome
03 December 2024Conditions: Crigler-Najjar Syndrome Interventions: Drug: Imlifidase; Drug: GNT0003 Sponsors: Genethon; Hansa Biopharma AB Recruiting
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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)
02 December 2024Conditions: Cystic Fibrosis Interventions: Genetic: BI 3720931; Drug: Placebo; Device: Inhaler Sponsors: Boehringer Ingelheim Recruiting
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Trial of Nadofaragene Firadenovec vs. Observation in Participants With Intermediate Risk Non-Muscle Invasive Bladder Cancer
26 November 2024Conditions: Intermediate Risk Non-Muscle Invasive Bladder Cancer Interventions: Drug: Nadofaragene Firadenovec Sponsors: Ferring Pharmaceuticals Recruiting
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A Study of ASP2016 in Adults Who Have Heart Disease Associated With Friedreich Ataxia
20 November 2024Conditions: Friedreich Ataxia; Cardiomyopathy Interventions: Genetic: ASP2016; Drug: Prednisolone Sponsors: Astellas Gene Therapies Recruiting
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Safety and Tolerability of KH658 Gene Therapy in Subjects With Neovascular Age-related Macular Degeneration (nAMD)
29 November 2024Conditions: Age-related Macular Degeneration Interventions: Drug: KH658 Sponsors: Chengdu Origen Biotechnology Co., Ltd. Recruiting
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Covid-19 Long Haul Preventative and Health Promotion Care Clinical Trial Acceleration Program.
27 November 2024Conditions: COVID-19, Long Haul Interventions: Drug: Ritonavir-Boosted Nirmatrelvir (Paxlovid); Diagnostic Test: Physiological Evaluation; Biological: Moderna COVID-19 Vaccine; Behavioral: Biopsychological; Behavioral: Behavioral (e.g., Psychotherapy, Lifestyle Counseling); Genetic: Genetic (including gene transfer, stem cell and recombinant DNA); Combination Product: Multidisciplinary approach Sponsors: Well- Konnect Healthcare Services and Research Firm; All of Us Research Program at the National Institute of Health 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)
26 November 2024Conditions: 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
26 November 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)
03 December 2024Conditions: 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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Base Editing for Mutation Repair in Hematopoietic Stem & Progenitor Cells for X-Linked Chronic Granulomatous Disease
06 December 2024Conditions: Chronic Granulomatous Disease (CGD); X-Linked Chronic Granulomatous Disease Interventions: Biological: Base-edited hematopoietic stem and progenitor cells; Drug: Busulfan; Drug: Palifermin; Drug: Filgrastim; Drug: Plerixafor Sponsors: National Institute of Allergy and Infectious Diseases (NIAID) Recruiting
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A Study of AAV2-GDNF in Adults With Moderate Parkinson's Disease (REGENERATE-PD)
25 November 2024Conditions: Parkinson Disease Interventions: Drug: AAV2-GDNF Gene therapy; Procedure: Control Surgery Sponsors: Asklepios Biopharmaceutical, Inc.; Bayer Recruiting
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An Observational Study Comparing Delandistrogene Moxeparvovec With Standard of Care in Participants With Duchenne Muscular Dystrophy
02 December 2024Conditions: Duchenne Muscular Dystrophy Interventions: Genetic: Delandistrogene Moxeparvovec; Drug: Standard of Care Sponsors: Sarepta Therapeutics, Inc. 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
25 November 2024Conditions: 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
Search Clinical Gene Therapy Trials
View clinical gene therapy trials available in the ClinicalTrials.gov database by using the following preset search terms:
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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.
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Latest clinical trials |
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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:
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ClinicalTrials.gov |
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Cancer.gov |
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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.