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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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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
14 October 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 Not yet recruiting
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Safety and Efficacy of CAR T Cell Therapy in Patients with R/r B-ALL
10 October 2024Conditions: Relapse/Refractory B-cell Acute Lymphoblastic Leukemia Interventions: Biological: anti-CD19 CAR T cell therapy Sponsors: Kara Yakhteh Tajhiz Azma Company Recruiting
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Patient Interview Study to Explore the Impact of Gene Therapy in Hemophilia A & B
10 October 2024Conditions: Hemophilia A; Hemophilia B Sponsors: Pfizer Not yet recruiting
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Glycogen Storage Disease Type Ia (GSDIa) Disease Monitoring Program
10 October 2024Conditions: Glycogen Storage Disease Type Ia Interventions: Other: No Intervention Sponsors: Ultragenyx Pharmaceutical Inc Not yet recruiting
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A Multicentric European Study to Promote Clinical Trial Readiness for STXBP1-related Disorders
03 October 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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Long-Term Follow-Up of Subjects Treated With AXO-AAV-GM2 for Tay-Sachs or Sandhoff Disease
26 September 2024Conditions: GM2 Gangliosidosis; Tay Sachs Disease; Sandhoff Disease Interventions: Drug: AXO-AAV-GM2 Sponsors: Terence Flotte Active, not recruiting
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Multimodal and Multidisciplinary Approach to Optimize Diagnostic, Prognostic, and Therapeutic Management of Patients with Non-ischemic Cardiomyopathies and Arrhythmogenic-inflammatory Phenotypes: a Multicenter, Observational, Retrospective and Prospective Registry Study.
23 September 2024Conditions: Non-ischemic Cardiomyopathy; Dilated Cardiomyopathy (DCM); Hypertrophic Cardiomyopathy (HCM); Restrictive Cardiomyopathy; Arrhythmogenic Cardiomyopathy (AC, ARVD/C); Left Ventricular Noncompaction; Arrhythmogenic Mitral Valve Prolapse; Peripartum Cardiomyopathy; Anderson-Fabry Disease; Arrhythmic and Inflammatory Non-ischemic Cardiomyopathy; Inflammatory (Non-Arrhythmic) Non-ischemic Cardiomyopathy; Nonischemic Cardiomyopathy Sensu Strictu (Non-inflammatory, Non-arrhythmic); Major Ventricular Arrhythmias, I.e. Sustained Ventricular Tachycardia, Ventricular Fibrillation, or Appropriate Therapy of Cardiac Device (defibrillators); Overlapping Phenotype; Undefined Phenotypes Interventions: Other: Support treatment, cardiac medical treatment, aetiology-specific treatment, device implant, arrhythmia ablation; Other: Support treatment, cardiac medical treatment, aetiology-specific treatment Sponsors: Scientific Institute San Raffaele Recruiting
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A Long-term Follow-up Study Evaluating Intravenous Injection of EXG001-307 in Patients With Type 1 SMA
19 September 2024Conditions: Spinal Muscular Atrophy Interventions: Other: No intervention, only for observational studies Sponsors: Hangzhou Jiayin Biotech Ltd Not yet recruiting
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Walking ANalysis Interest in Persons with FacioscapulohumEral Muscular Dystrophies
19 September 2024Conditions: Facioscapulohumeral Dystrophy Interventions: Other: Walk tests; Other: ecological (real-life) assessment of walking ability Sponsors: Centre Hospitalier Universitaire Dijon 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
19 September 2024Conditions: Duchenne Muscular Dystrophy Interventions: Genetic: delandistrogene moxeparvovec; Procedure: Plasmapheresis Sponsors: Sarepta Therapeutics, Inc. Not yet recruiting
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5-Azacytidine with Steroids for Gastrointestinal GVHD (5-AZA for GVHD)
19 September 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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CAR T Cells in the Treatment of Refractory and Relapsed CD19+ B Cell Neoplasms
19 September 2024Conditions: Acute Lymphoblastic Leukemia (ALL); Mantle Cell Lymphoma (MCL); Large B-cell Lymphoma Interventions: Biological: FCTX-CL19-1 (Tarcidomgen Kimleucel) Sponsors: FamiCordTx 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 )
19 August 2024Conditions: Chronic Granulomatous Disease; Granulomatous Disease, Chronic Interventions: Biological: PM359 Sponsors: Prime Medicine, Inc. Recruiting
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A Phase 1/2 Trial of SP-101 for the Treatment of Cystic Fibrosis (CF)
30 July 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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Anti-VEGF Gene Therapy Trial for Vestibular Schwannoma
24 July 2024Conditions: Vestibular Schwannoma Interventions: Combination Product: AAVAnc80-antiVEGF via Akouos Delivery Device Sponsors: Akouos, Inc.; Eli Lilly and Company 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)
23 July 2024Conditions: 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
03 July 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)
21 June 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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Study to Evaluate Safety of RTx-015 Injection in Retinitis Pigmentosa Patients (ENVISION)
14 June 2024Conditions: Retinitis Pigmentosa Interventions: Genetic: RTx-015 Sponsors: Ray Therapeutics, Inc. Recruiting
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Prescreening Study to Identify Potential Participants for ACDN-01 Clinical Trials
06 June 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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Evaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal Muscular Atrophy (SMA) Type 3 Patients
20 May 2024Conditions: Spinal Muscular Atrophy Type 3 Interventions: Genetic: GC101 Sponsors: GeneCradle Inc Recruiting
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A Phase 3 Study Of OCU400 Gene Therapy for the Treatment Of Retinitis Pigmentosa
29 April 2024Conditions: 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)
18 April 2024Conditions: Age Related Macular Degeneration (ARMD) Interventions: Drug: Elamipretide; Drug: Placebo Sponsors: Stealth BioTherapeutics Inc. Recruiting
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A Phase I/II Clinical Trial with SENS-501 in Children Suffering from Severe to Profound Hearing Loss Due to Otoferlin (OTOF) Mutations
17 April 2024Conditions: OTOF Gene Mutation; DFNB9; Congenital Deafness; Hearing Disorders; Ear Diseases; Otorhinolaryngologic Diseases; Deafness; Hearing Loss, Sensorineural Interventions: Combination Product: SENS-501 administration Sponsors: Sensorion Recruiting
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ALS20-101 Lentiviral Gene Therapy for Beta Thalassemia
15 April 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)
15 April 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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AAV Gene Therapy Clinical Study in Adult Classic PKU (PHEdom)
27 March 2024Conditions: Phenylketonurias Interventions: Genetic: NGGT002 Sponsors: NGGT INC. Recruiting
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Base Editing for Mutation Repair in Hematopoietic Stem & Progenitor Cells for X-Linked Chronic Granulomatous Disease
22 March 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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Non-interventional Study of Seroprevalence of Pre-existing Antibodies Against Adenovirus-associated Virus Vector (AAV9) and the Progression of Disease in Patients with Plakophilin 2 (PKP2)-associated Arrhythmogenic Right Ventricular Cardiomyopathy (ARVC)
15 March 2024Conditions: Arrhythmogenic Right Ventricular Cardiomyopathy Sponsors: Tenaya Therapeutics; University of California, San Francisco; Brigham and Women's Hospital; New York University; University of Colorado, Denver; Johns Hopkins University; The Cleveland Clinic; Mayo Clinic; Medical University of South Carolina; Hopital Louis Pradel; Istituti Clinici Scientifici Maugeri SpA; The Queen Elizabeth Hospital; St George's University Hospitals NHS Foundation Trust; Royal Brompton & Harefield NHS Foundation Trust; Barts & The London NHS Trust; Skane University Hospital; Centro Cardiologico Monzino; Hôpital Haut-Lévêque; Nantes University Hospital; Pitié-Salpêtrière Hospital; Wuerzburg University Hospital; University Hospital Muenster Recruiting
Info ClinicalTrials.gov
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.