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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 Study to Check Liver Health in Boys With XLMTM, a Serious Genetic Muscle Condition
03 September 2024Conditions: X-Linked Myotubular Myopathy Interventions: Other: No Intervention Sponsors: Astellas Gene Therapies Not yet recruiting
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Expanded Access Program for RP in Adults
28 August 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 )
19 August 2024Conditions: Chronic Granulomatous Disease; Granulomatous Disease, Chronic Interventions: Biological: PM359 Sponsors: Prime Medicine, Inc. Not yet 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
09 August 2024Conditions: Non-muscle Invasive Bladder Cancer With Carcinoma in Situ Interventions: Drug: Nadofaragene Firadenovec; Drug: Gemcitabine; Drug: Docetaxel; Drug: Pembrolizumab Sponsors: Ferring Pharmaceuticals Not yet 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
06 August 2024Conditions: Lymphomas Non-Hodgkin's B-Cell; Precursor Cell Lymphoblastic Leukemia-Lymphoma Interventions: Genetic: INT2104 Sponsors: Interius 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)
23 July 2024Conditions: Cystic Fibrosis Interventions: Genetic: BI 3720931; Drug: Placebo; Device: Inhaler Sponsors: Boehringer Ingelheim Not yet recruiting
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Clinical Exploration Study of YOLT-203 in the Treatment of Type 1 Primary Hyperoxaluria (PH1)
19 July 2024Conditions: Type 1 Primary Hyperoxaluria Interventions: Drug: YOLT-203 Sponsors: RenJi Hospital 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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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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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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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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Safety and Efficacy Study of Novel Gene Therapy ZM-02 for Retinitis Pigmentosa Patients
05 March 2024Conditions: Retinitis Pigmentosa Interventions: Drug: ZM-02-L; Drug: ZM-02-H; Procedure: ZM-02-S Sponsors: Zhongmou Therapeutics Recruiting
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A Study of AAV2-GDNF in Adults with Moderate Parkinson's Disease (REGENERATE-PD)
29 February 2024Conditions: Parkinson Disease Sponsors: Asklepios Biopharmaceutical, Inc. Recruiting
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A Clinical Study to Evaluate the Safety and Efficacy of ETX101, an AAV9-Delivered Gene Therapy in Children With SCN1A-positive Dravet Syndrome
28 February 2024Conditions: Dravet Syndrome Interventions: Drug: ETX101 Sponsors: Encoded Therapeutics Recruiting
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An Observational Study Comparing Delandistrogene Moxeparvovec With Standard of Care in Participants With Duchenne Muscular Dystrophy
21 February 2024Conditions: Duchenne Muscular Dystrophy Interventions: Genetic: delandistrogene moxeparvovec; Drug: Standard of Care Sponsors: Sarepta Therapeutics, Inc. Recruiting
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Safety, PK/PD, and Exploratory Efficacy Study of AMT-191 in Classic Fabry Disease
21 February 2024Conditions: Fabry Disease Interventions: Drug: AMT-191 Sponsors: UniQure Biopharma B.V. 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
12 February 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
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pCCLCHIM-p47 (Lentiviral Vector Transduced CD34 Plus Cells) in Patients With p47 Autosomal Recessive Chronic Granulomatous Disease (AR-CGD)
12 February 2024Conditions: 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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A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) Following Imlifidase Infusion in Participants With Duchenne Muscular Dystrophy (DMD) Determined to Have Pre-existing Antibodies to Recombinant Adeno-Associated Virus Serotype (rAAVrh74)
05 February 2024Conditions: Duchenne Muscular Dystrophy Interventions: Genetic: delandistrogene moxeparvovec; Biological: imlifidase Sponsors: Sarepta Therapeutics, Inc.; Hansa Biopharma AB Enrolling by invitation
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Open-label, Dose Escalation Study of Safety and Preliminary Efficacy of TN-401 in Adults With PKP2 Mutation-associated ARVC
29 January 2024Conditions: Arrhythmogenic Right Ventricular Cardiomyopathy Interventions: Genetic: TN-401 Sponsors: Tenaya Therapeutics Recruiting
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Autologous CAR-T Cells Targeting B7-H3 in PDAC
06 December 2023Conditions: Pancreas Cancer; Relapse; Resistant Cancer Interventions: Biological: iC9-CAR.B7-H3 T cell infusion Sponsors: UNC Lineberger Comprehensive Cancer Center Recruiting
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A Study to Investigate the Efficacy and Safety of OTL-203 in Subjects With MPS-IH Compared With Standard of Care With Allogeneic HSCT
29 November 2023Conditions: MPS-IH (Hurler Syndrome) Interventions: Genetic: Experimental: OTL-203; Genetic: Active Comparator: Allo-HSCT Sponsors: Orchard Therapeutics Recruiting
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A Clinical Study to Evaluate the Safety and Efficacy of ETX101, an AAV9-Delivered Gene Therapy in Children With SCN1A-positive Dravet Syndrome (Australia Only)
01 November 2023Conditions: Dravet Syndrome Interventions: Drug: ETX101 Sponsors: Encoded Therapeutics Recruiting
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Gene Therapy for ACM Due to a PKP2 Pathogenic Variant
31 October 2023Conditions: Arrhythmogenic Cardiomyopathy; PKP2-ACM; PKP2-ARVC Interventions: Genetic: LX2020 Sponsors: Lexeo Therapeutics Recruiting
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Liver Biopsy Following Gene Therapy For Hemophilia
06 July 2023Conditions: Hemophilia A; Hemophilia B Interventions: Procedure: Liver Biopsy Sponsors: St. Jude Children's Research Hospital Not yet recruiting
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A Gene Transfer Study to Evaluate the Safety, Tolerability and Efficacy of SRP-6004 in Ambulatory Participants With Limb Girdle Muscular Dystrophy, Type 2B/R2 (LGMD2B/R2, Dysferlin [DYSF] Related)
15 June 2023Conditions: Limb Girdle Muscular Dystrophy Interventions: Genetic: SRP-6004 Sponsors: Sarepta Therapeutics, Inc. Active, not recruiting
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A Novel, Regulated Gene Therapy (NGN-401) Study for Female Children With Rett Syndrome
12 June 2023Conditions: Rett Syndrome Interventions: Genetic: NGN-401 Sponsors: Neurogene Inc. Recruiting
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A Study Evaluating the Effectiveness and Safety of Risdiplam Administered as an Early Intervention in Pediatric Participants With Spinal Muscular Atrophy After Gene Therapy
17 May 2023Conditions: Muscular Atrophy, Spinal Interventions: Drug: risdiplam Sponsors: Hoffmann-La Roche Recruiting
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A Study Evaluating the Effectiveness and Safety of Risdiplam Administered in Pediatric Patients With Spinal Muscular Atrophy Who Experienced a Plateau or Decline in Function After Gene Therapy
17 May 2023Conditions: Muscular Atrophy, Spinal Interventions: Drug: risdiplam Sponsors: Hoffmann-La Roche Recruiting
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Trial of Anti-CD19 and Anti-CD20 Bicistronic Chimeric Antigen Receptor T Cells for Treating B-Cell Malignancies
04 April 2023Conditions: Chronic Lymphocytic Leukemia; B-Cell Chronic Lymphocytic Leukemia; Lymphoma, B-Cell Interventions: Biological: Anti-CD19 and anti-CD20 bicistronic CAR T- cells; Drug: Cyclophosphamide; Drug: Fludarabine Sponsors: National Cancer Institute (NCI) Active, not 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.