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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 Assess Safety and Tolerability of PCRX-201 in Subjects With Painful Osteoarthritis of the Knee
19 March 2025Conditions: Osteoarthritis (OA) of the Knee Interventions: Biological: Enekinragene Inzadenovec (PCRX-201); Biological: Placebo Sponsors: Pacira Pharmaceuticals, Inc Recruiting
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Long Term Follow-up Study for Subjects Administered CLBR001
18 March 2025Conditions: Long Term Follow-Up Interventions: Other: Long Term Follow-Up Sponsors: Calibr, a division of Scripps Research Not yet recruiting
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Evaluating the Safety and Efficacy of DuoCAR20.19.22-D95 in Adult Patients with Relapsed or Refractory B-cell Malignancies
17 March 2025Conditions: B-Cell Non-Hodgkin Lymphoma; B-cell Acute Lymphoblastic Leukemia Interventions: Drug: DuoCAR20.19.22-D95; Drug: Fludarabine (Conditional therapy); Drug: Cyclophosphamide (Conditional therapy) Sponsors: University of Kansas Medical Center Not yet recruiting
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Study of EN-374 Gene Therapy in Participants with X-Linked Chronic Granulomatous Disease
14 March 2025Conditions: X-Linked Chronic Granulomatous Disease Interventions: Genetic: EN-374 Sponsors: Ensoma Not yet recruiting
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Phenotypic and Molecular Characterisation of Cerebral Amyloid Angiopathy
07 March 2025Conditions: Cerebral Amyloid Aβ Angiopathy Sponsors: University Hospital, Rouen Recruiting
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4D-150 in Patients With Macular Neovascularization Secondary to Age-Related Macular Degeneration
07 March 2025Conditions: Macular Neovascularization Secondary to Age-Related Macular Degeneration Interventions: Biological: 4D-150 IVT (3E10 vg/eye); Biological: EYLEA® (aflibercept) Injection 2 mg (0.05mL) Sponsors: 4D Molecular Therapeutics Recruiting
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Phase I/IIa Study: RR001 Administered Following Chemotherapy Cycles for Patients with Locally Advanced Pancreatic Carcinoma
06 March 2025Conditions: Locally Advanced Pancreatic Adenocarcinoma Interventions: Drug: RR001 Sponsors: EIR Biotherapies s.r.l. Recruiting
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A Long-term Follow up Study of EXG102-031 in Participants With wAMD
05 March 2025Conditions: Wet Age-related Macular Degeneration Interventions: Other: No intervention, only for observational studies Sponsors: Hangzhou Jiayin Biotech Ltd; Peking University People's Hospital Not yet recruiting
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Single-Dose AAV-MECP2 Safety/Tolerability and Efficacy in Rett Syndrome
04 March 2025Conditions: Rett Syndrome Interventions: Genetic: Intrathecal injection of AAV-MECP2 for the treatment of Rett syndrome Sponsors: Guangzhou Women and Children's Medical Center; Xiamen Children's Hospital, Fujian of China; Shanghai Jiao Tong University School of Medicine Recruiting
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Efficacy and Safety Study of Ixoberogene Soroparvovec (Ixo-vec) in Participants With Neovascular Age-Related Macular Degeneration
04 March 2025Conditions: Neovascular Age-Related Macular Degeneration (nAMD); Wet AMD Interventions: Genetic: Ixo-vec; Drug: Aflibercept Sponsors: Adverum Biotechnologies, Inc. Not yet recruiting
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Base-Edited Hematopoietic Stem/Progenitor Cell X-Linked Severe Combined Immunodeficiency Gene Therapy
19 March 2025Conditions: X-linked Severe Combined Immunodeficiency; X-SCID; XSCID Interventions: Genetic: Plerixafor; Drug: Filgrastim; Drug: Palifermin; Drug: Busulfan; Biological: Base-edited hematopoietic stem and progenitor cells Sponsors: National Institute of Allergy and Infectious Diseases (NIAID) Not yet recruiting
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A Study to Evaluate the Tolerability, Safety and Efficacy of VGN-R13 in Patients with ALS
17 March 2025Conditions: Amyotrophic Lateral Sclerosis (ALS) Interventions: Biological: VGN-R13 Sponsors: Hong Chen Recruiting
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A Study to Investigate the Safety, Tolerability, and Efficacy of SAR446268, an Adeno-associated Viral Vector-mediated Gene Therapy in Participants Aged 10 to 50 Years of Age With Non-congenital Myotonic Dystrophy Type 1
12 March 2025Conditions: Myotonic Dystrophy Interventions: Biological: SAR446268 Sponsors: Sanofi Not yet recruiting
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Gene Therapy for Neurofibromatosis Type 2 (NF2) with ST002
19 February 2025Conditions: NF2 Deficiency Interventions: Genetic: ST002 Sponsors: Cancer Institute and Hospital, Chinese Academy of Medical Sciences Not yet recruiting
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To Evaluate the Clinical Study of GS1191-0445 Injection in the Treatment of Hemophilia a
19 February 2025Conditions: Hemophilia a Interventions: Drug: GS1191-0445 injection Sponsors: Gritgen Therapeutics Co., Ltd. Not yet recruiting
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Study Assessing Left Ventricular Administration of a Genetic Medicine Directing Organ Regeneration in Heart Failure
18 February 2025Conditions: Heart Failure With Reduced Ejection Fraction Interventions: Combination Product: YAP101 (AAV9-Sav-shRNA) Sponsors: YAP Therapeutics, Inc. Not yet recruiting
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A Randomized Study of SPK-10001 Gene Therapy in Participants With Huntington's Disease
26 February 2025Conditions: Huntington Disease Interventions: Genetic: SPK-10001; Other: Placebo Surgery Control Sponsors: Spark Therapeutics, Inc. Recruiting
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Study to Evaluate the Safety, Tolerability, and Efficacy of SRP-9004 Administered by Systemic Infusion in Limb Girdle Muscular Dystrophy Type 2D/R3 Participants in the United States
03 March 2025Conditions: Limb Girdle Muscular Dystrophy; Limb Girdle Muscular Dystrophy Type 2D/R3 Interventions: Drug: SRP-9004 Sponsors: Sarepta Therapeutics, Inc. Enrolling by invitation
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GCB-002 in Treatment of Patients with Rett Syndrome
11 March 2025Conditions: RETT Syndrome with Proven MECP2 Mutation Interventions: Genetic: GCB-002 Sponsors: Genecombio Ltd.; Xinhua Hospital, Shanghai Jiao Tong University School of Medicine Enrolling by invitation
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A Study of EH002 Gene Therapy for Otoferlin Gene Mutation-mediated Hearing Loss
11 March 2025Conditions: DFNB9; Congenital Hearing Loss; Hearing Loss, Sensorineural Interventions: Genetic: EH002 administration Sponsors: Yilai Shu 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
19 March 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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JAG201 Gene Therapy Study in Children & Adults with SHANK3 Haploinsufficiency
05 March 2025Conditions: SHANK3 Haploinsufficiency; Phelan-McDermid Syndrome Interventions: Genetic: JAG201; Genetic: JAG201 Sponsors: Jaguar Gene Therapy, LLC Recruiting
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Long Term Follow-Up Study for Individuals With Hemoglobin Disorders After Hematopoietic Stem Cell Transplant or Gene Therapy
03 March 2025Conditions: Hemoglobin Disorder Sponsors: St. Jude Children's Research Hospital Not yet recruiting
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Patient Interview Study to Explore the Impact of Gene Therapy in Hemophilia A & B
05 March 2025Conditions: Hemophilia A; Hemophilia B Sponsors: Pfizer Not yet recruiting
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A Multicentric European Study to Promote Clinical Trial Readiness for STXBP1-related Disorders
04 March 2025Conditions: STXBP1 Encephalopathy with Epilepsy Sponsors: Amsterdam UMC, location VUmc; Filadelfia Epilepsy Hospital; University Hospital Heidelberg; University Hospital, Antwerp; Istituto Giannina Gaslini, Genoa, Italy; Hospital Sant Joan de Deu; Sheba Medical Center; Aix Marseille Université; Hospital Ruber Internacional Not yet recruiting
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A Long-term Follow-up Study Evaluating Intravenous Injection of EXG001-307 in Patients With Type 1 SMA
05 March 2025Conditions: Spinal Muscular Atrophy Interventions: Other: No intervention, only for observational studies Sponsors: Hangzhou Jiayin Biotech Ltd Enrolling by invitation
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5-Azacytidine with Steroids for Gastrointestinal GVHD (5-AZA for GVHD)
04 March 2025Conditions: 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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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
03 March 2025Conditions: 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
05 March 2025Conditions: X-Linked Myotubular Myopathy Interventions: Other: No Intervention Sponsors: Astellas Gene Therapies 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 )
26 February 2025Conditions: Chronic Granulomatous Disease; Granulomatous Disease, Chronic Interventions: Biological: PM359 Sponsors: Prime Medicine, 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.
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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.