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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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Phase 1 Study to Evaluate Safety and Antiviral Activity of PBGENE-HBV in Adult Patients with Chronic Hepatitis B
08 November 2024Conditions: HEPATITIS B CHRONIC Interventions: Biological: PBGENE-HBV Sponsors: Precision BioSciences, Inc. Not yet recruiting
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Decision-Making and Quality of Life Surrounding Hematologic Disease and Gene Therapy
29 October 2024Conditions: Transfusion Dependent Beta Thalassemia; Sickle Cell Disease Sponsors: Georgetown University Enrolling by invitation
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An Exploratory Study to Evaluate the Tolerability and Safety of MWAV201 in Subjects With Wilson Disease
29 October 2024Conditions: Wilson Disease Interventions: Genetic: MWAV201 Sponsors: Xinhua Hospital, Shanghai Jiao Tong University School of Medicine; Mabwell (Shanghai) Bioscience Co., Ltd. Not yet recruiting
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JAG201 Gene Therapy Study in Children & Adults with SHANK3 Haploinsufficiency
01 November 2024Conditions: SHANK3 Haploinsufficiency; Phelan-McDermid Syndrome Interventions: Genetic: JAG201; Genetic: JAG201 Sponsors: Jaguar Gene Therapy, LLC Not yet recruiting
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Study of Populations At Risk of Developing Chronic Hepatitis Linked to Chronic Enteric Virus Infection in Patients with Primary Immunodeficiency and Secondary Humoral Deficiency
26 October 2024Conditions: Humoral Primary Immunodeficiencies (PIDs); Secondary Form of Humoral Immunodeficiencies; Combined Immunodeficiency (CID); Severe Combined Immunodeficiency (SCID) Interventions: Other: Plasma, urine and stool collection Sponsors: Assistance Publique - Hôpitaux de Paris Not yet recruiting
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Evaluating Safety and Efficacy of Lentiviral-transduced CD34+ HSCs in Β-thalassaemia Patients.
23 October 2024Conditions: Β-thalassemia Interventions: Other: β-globin restored autologous hematopoietic stem cells Sponsors: Shenzhen Hemogen Recruiting
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Long-term Follow-up of Participants Treated with Galapagos Chimeric Antigen Receptor (CAR) T-cell Therapies
22 October 2024Conditions: Hematological Malignancies Interventions: Genetic: GLPG CAR T-cell therapy Sponsors: Galapagos NV Recruiting
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A Clinical Study to Evaluate the Safety and Efficacy of LY-M003 Injection in Patients with Wilson Disease
21 October 2024Conditions: Wilson Disease Interventions: Biological: intravenous LY-M003 Sponsors: Chaohui Yu Enrolling by invitation
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Hematopoietic Stem Cell BCL11A Enhancer Gene Editing for Severe Β-Hemoglobinopathies
18 October 2024Conditions: Sickle Cell Disease; Sickle Cell Anemia (HbSS, or HbSβ-thalassemia0); Beta-Thalassemia; Transfusion Dependent Beta-Thalassaemia Interventions: Biological: autologous bone marrow derived CD34+ HSPCs electroporated with BCL11A enhancer targeting Cas9 ribonucleoprotein; Device: Sequencing Assay for Variant rs114518452 Sponsors: Daniel Bauer Not yet recruiting
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Long Term Follow-Up Study for Individuals With Hemoglobin Disorders After Hematopoietic Stem Cell Transplant or Gene Therapy
17 October 2024Conditions: Hemoglobin Disorder Sponsors: St. Jude Children's Research Hospital Not yet 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)
17 October 2024Conditions: X-Linked Retinitis Pigmentosa Interventions: Biological: AAV5-hRKp.RPGR; Other: No intervention (Follow-Up assessment) Sponsors: Janssen Research & Development, LLC Not yet recruiting
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An IIT Clinical Study to Evaluate the Safety, Tolerability, and Preliminary Efficacy of a Single Intrathecal Injection of SNUG01 in Patients with Amyotrophic Lateral Sclerosis
16 October 2024Conditions: Amyotrophic Lateral Sclerosis (ALS) Interventions: Drug: SNUG01 Sponsors: Peking University Third Hospital; SineuGene Therapeutics Co., Ltd Not yet recruiting
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Evaluation of the Safety and Efficacy of BBM-D101 to Treat Patients With Duchenne Muscular Dystrophy
15 October 2024Conditions: Duchenne Muscular Dystrophy (DMD) Interventions: Genetic: BBM-D101 Sponsors: Shanghai Jiao Tong University School of Medicine; Shanghai Mianyi Biopharmaceutical Co., Ltd. Recruiting
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Gene Therapy for Crigler Najjar Syndrome Type I
07 November 2024Conditions: 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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Patient Interview Study to Explore the Impact of Gene Therapy in Hemophilia A & B
29 October 2024Conditions: Hemophilia A; Hemophilia B Sponsors: Pfizer Not yet recruiting
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A Gene Transfer Therapy to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) Following Therapeutic Plasma Exchange (Plasmapheresis) in Participants With Duchenne Muscular Dystrophy (DMD) and Pre-existing Antibodies to AAVrh74
16 October 2024Conditions: Duchenne Muscular Dystrophy Interventions: Genetic: delandistrogene moxeparvovec; Procedure: Plasmapheresis Sponsors: Sarepta Therapeutics, Inc. Recruiting
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A Study to Check Liver Health in Boys With XLMTM, a Serious Genetic Muscle Condition
21 October 2024Conditions: X-Linked Myotubular Myopathy Interventions: Other: No Intervention Sponsors: Astellas Gene Therapies Not yet recruiting
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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 October 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
26 October 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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Evaluate the Safety and Preliminary Efficacy of EXG110 in Subjects With Fabry Disease
30 October 2024Conditions: Fabry Disease Interventions: Genetic: EXG110 injection Sponsors: The Children's Hospital of Zhejiang University School of Medicine Recruiting
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Safety of INT2104 in Participants Aged 18 Years and Older Who Have B-cell Cancer That Came Back After Prior Treatment
26 October 2024Conditions: Lymphomas Non-Hodgkin's B-Cell; Precursor Cell Lymphoblastic Leukemia-Lymphoma Interventions: Genetic: INT2104 Sponsors: Interius BioTherapeutics Inc. Recruiting
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Gene Therapy for B-Cell Acute Lymphoblastic Leukemia
22 October 2024Conditions: B-cell Acute Lymphoblastic Leukemia Interventions: Genetic: Dose Level 1, VNX-101; Genetic: Dose Level 2, VNX-101; Genetic: Dose Level 3, VNX-101; Genetic: Dose Level 4, VNX-101 Sponsors: Vironexis Biotherapeutics Inc. 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)
01 November 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
07 November 2024Conditions: Intermediate Risk Non-Muscle Invasive Bladder Cancer Interventions: Drug: Nadofaragene Firadenovec Sponsors: Ferring Pharmaceuticals Recruiting
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DB107-RRV, DB107-FC, and Radiation Therapy With or Without Temozolomide (TMZ) for High Grade Glioma
23 October 2024Conditions: High Grade Glioma; MGMT-Unmethylated Glioblastoma; MGMT-Methylated Glioblastoma Interventions: Genetic: DB107-RRV; Genetic: DB107-FC; Radiation: Radiation Therapy (RT); Drug: Temozolomide; Procedure: Magnetic Resonance Imaging (MRI); Procedure: Surgical resection Sponsors: Nicholas Butowski; California Institute for Regenerative Medicine (CIRM) Not yet recruiting
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A Study of ASP2016 in Adults Who Have Heart Disease Associated With Friedreich Ataxia
31 October 2024Conditions: Friedreich Ataxia; Cardiomyopathy Interventions: Genetic: ASP2016; Drug: Prednisolone Sponsors: Astellas Gene Therapies Not yet recruiting
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Study to Evaluate ACDN-01 in ABCA4-related Retinopathy (STELLAR)
15 October 2024Conditions: Stargardt Disease; Cone Rod Dystrophy; Juvenile Macular Degeneration; Stargardt Disease 1 Interventions: Drug: ACDN-01 Sponsors: Ascidian Therapeutics, Inc Recruiting
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Prescreening Study to Identify Potential Participants for ACDN-01 Clinical Trials
30 October 2024Conditions: 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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Investigation Into the Use of BAH243 Lentiviral Vector for Gene Therapy in Treating Sickle Cell Disease
05 November 2024Conditions: Sickle Cell Disease; Sickle-Cell Disease With Crisis Interventions: Genetic: Drug Product is administered by IV infusion following myeloablative conditioning with busulfan Sponsors: Essen Biotech 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)
05 November 2024Conditions: Age Related Macular Degeneration (ARMD) Interventions: Drug: Elamipretide; Drug: Placebo Sponsors: Stealth BioTherapeutics Inc. 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.