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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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RB001 Gene Therapy Study in Children With SHANK3-related Phelan McDermid Syndrome (PMS)
10 June 2025Conditions: Phelan-McDermid Syndrome; SHANK3 Haploinsufficiency Interventions: Genetic: RB001 Sponsors: Peking University First Hospital; Shenzhen Reborngene Therapeutics Co., Ltd.; Shenzhen Institutes of Advanced Technology ,Chinese Academy of Sciences Not yet recruiting
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A Clinical Trial of CAP-003 Gene Therapy in Adult Patients With GBA1 Associated Parkinson's Disease
10 June 2025Conditions: GBA1 Parkinson Disease Interventions: Drug: gene therapy; Drug: gene therapy Sponsors: Capsida Biotherapeutics, Inc. Not yet recruiting
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Study to Assess the Injection Burden, Adverse Events, Change in Disease Activity, and Long-Term Preservation of Visual Acuity of Surabgene Lomparvovec in Adult Participants With Neovascular Age-Related Macular Degeneration (nAMD)
05 June 2025Conditions: Neovascular Age-related Macular Degeneration Interventions: Drug: Surabgene Lomparvovec (ABBV-RGX-314); Drug: Ranibizumab Control Sponsors: AbbVie Not yet recruiting
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PHOX2B PC-CAR T Cells for Relapsed Neuroblastoma
05 June 2025Conditions: Refractory Neuroblastoma; Relapsed Neuroblastoma; High-Risk Neuroblastoma Interventions: Biological: PHOX2B PC-CAR T Cells Sponsors: Stephan Grupp MD PhD; Children's Hospital of Philadelphia; Alliance for Cancer Gene Therapy; Children's Cancer Research Fund Not yet recruiting
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Ethical Issues of Using Gene Therapy in the Treatment of AMD
04 June 2025Conditions: Exudative Age-related Macular Degeneration Sponsors: Nantes University Hospital Not yet recruiting
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Safety and Preliminary Efficacy of VG801 in Patients With ABCA4 Mutation-associated Retinal Dystrophy (Stargardt Disease)
03 June 2025Conditions: Retinal Dystrophy Due to Biallelic ABCA4 Mutations; Stargardt Disease 1 Interventions: Drug: VG801 Sponsors: ViGeneron GmbH Recruiting
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Gene Therapy for Alpha-1 Antitrypsin Deficiency
30 May 2025Conditions: Alpha 1-Antitrypsin Deficiency Interventions: Biological: AAV8hAAT(AVL) Sponsors: Weill Medical College of Cornell University; National Heart, Lung, and Blood Institute (NHLBI) Not yet recruiting
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A Clinical Trial of CAP-002 Gene Therapy in Pediatric Patients With Syntaxin-Binding Protein 1 (STXBP1) Encephalopathy
10 June 2025Conditions: Developmental and Epileptic Encephalopathy Interventions: Drug: gene therapy Sponsors: Capsida Biotherapeutics, Inc. Not yet recruiting
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InsightRP2 Registry
21 May 2025Conditions: RP2-associated Retinitis Pigmentosa; X-Linked Retinitis Pigmentosa (XLRP); Retinitis Pigmentosa 2 Sponsors: University of Göttingen Recruiting
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Safety and Tolerability Study of ST-503 for Small Fiber Neuropathy-associated Refractory Pain
08 June 2025Conditions: Chronic Neuropathic Pain Interventions: Genetic: ST-503; Procedure: Sham (No Treatment) Sponsors: Sangamo Therapeutics Not yet recruiting
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A Study to Assess Real-world Patient Characteristics and Clinical Course for Symptomatic Patients With PKP2-ACM
16 May 2025Conditions: Arrhythmogenic Cardiomyopathy; PKP2-ACM; PKP2-ARVC Sponsors: Lexeo Therapeutics Recruiting
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Anti CD19/BCMA CAR Gene Therapy for Relapsed/Refractory Immune Thrombocytopenia
15 May 2025Conditions: Immune Thrombocytopenia (ITP) Interventions: Drug: Anti-CD19/BCMA CAR gene vector injection will be injected intravenously on a one-time basis. Sponsors: Anhui Provincial Hospital Recruiting
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Safety and Efficacy Evaluation of GC101 Gene Therapy Via Intrathecal (IT) Injectionin the Treatment of Patients With Type 2 Spinal Muscular Atrophy (SMA) - Phase III
05 June 2025Conditions: SMA - Spinal Muscular Atrophy Interventions: Genetic: GC101 adeno-associated virus injection Sponsors: GeneCradle Inc Recruiting
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A Long-term Study to Monitor the Health Status of People With Cystic Fibrosis Who Took Part in a Previous Study With BI 3720931 (Lenticlair™-ON)
28 May 2025Conditions: Cystic Fibrosis Interventions: Genetic: BI 3720931 Sponsors: Boehringer Ingelheim Recruiting
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Base Editing Hematopoietic Stem Cell Gene Therapy for CD40L-HyperIgM Syndrome: Single Patient Study
13 June 2025Conditions: CD40L-HyperIgM Syndrome Interventions: Drug: Busulfan; Drug: Palifermin; Drug: Sirolimus; Drug: Alemtuzumab; Biological: Base-edited hematopoietic stem and progenitor cells Sponsors: National Institute of Allergy and Infectious Diseases (NIAID) Enrolling by invitation
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A Study of SRP-9005 in Limb Girdle Muscular Dystrophy Type 2C/R5 Pediatric and Adult Participants
04 June 2025Conditions: Muscular Dystrophies, Limb-Girdle Interventions: Biological: SRP-9005; Drug: Corticosteroid Sponsors: Sarepta Therapeutics, Inc. Recruiting
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Anti-CD7 CAR-T Cells in Relapsed/Refractory T-Cell Acute Lymphoblastic Leukemia or Lymphoma
31 May 2025Conditions: T-Cell Acute Lymphoblastic Leukemia/Lymphoma Interventions: Biological: Allogeneic anti-CD7 CAR-T cells (BEAM-201) Sponsors: Stephan Grupp MD PhD; Beam Therapeutics Inc. Recruiting
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GVB-2001 Gene Therapy Via Intracameral Injection for the Treatment of Primary Open Angle Glaucoma
08 June 2025Conditions: Primary Open-Angle Glaucoma (POAG) Interventions: Drug: GVB-2001-high dose; Drug: GVB-2001-low dose Sponsors: IVIEW Therapeutics Inc.; The First Affiliated Hospital of Soochow University Not yet recruiting
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Autologous T Cells Transduced With Retroviral Vectors Expressing TCRs for Participant-specific Neoantigens in Patients With Hematologic Malignancies
13 June 2025Conditions: Malignancy, Hematologic; Neoplasms, Hematologic; Neoplasms, Hematopoietic; Blood Cancer; Hematological Neoplasms; Hematopoietic Malignancies; Dysmyelopoietic Syndromes; Hematopoetic Myelodysplasia; Myeloid Leukemia, Acute; Nonlymphoblastic Leukemia, Acute; Leukemia, Lymphocytic, Acute Interventions: Drug: aldesleukin; Drug: cyclophosphamide; Drug: fludarabine phosphate; Biological: Individual Patient TCR-Transduced PBL; Device: TruSight Oncology (TSO) 500 Sponsors: National Cancer Institute (NCI) Not yet recruiting
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Anti-Mesothelin TNaive/SCM hYP218 (TNhYP218) CAR T Cells in Participants With Mesothelin-Expressing Solid Tumors Including Mesothelioma
13 June 2025Conditions: Mesothelioma; Neoplasms; Stomach Neoplasms; Pancreatic Neoplasms; Ovarian Neoplasms; Lung Neoplasms; Thymus Neoplasms; Colonic Neoplasms Interventions: Device: mesothelin expression testing; Biological: TNhYP217 CAR T Cells; Drug: fludarabine; Drug: cyclophosphamide Sponsors: National Cancer Institute (NCI) Not yet recruiting
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A Study to Assess Safety and Tolerability of PCRX-201 in Subjects With Painful Osteoarthritis of the Knee
30 May 2025Conditions: Osteoarthritis (OA) of the Knee Interventions: Biological: Enekinragene Inzadenovec (PCRX-201); Biological: Placebo Sponsors: Pacira Pharmaceuticals, Inc Recruiting
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Phase I/IIa Study: RR001 Administered Following Chemotherapy Cycles for Patients With Locally Advanced Pancreatic Carcinoma
25 May 2025Conditions: Locally Advanced Pancreatic Adenocarcinoma Interventions: Drug: RR001 Sponsors: EIR Biotherapies s.r.l. Recruiting
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Single-Dose AAV-MECP2 Safety/Tolerability and Efficacy in Rett Syndrome
31 May 2025Conditions: Rett Syndrome Interventions: Genetic: Intrathecal injection of AAV-MECP2 for the treatment of Rett syndrome Sponsors: Guangzhou Women and Children's Medical Center Recruiting
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Efficacy and Safety Study of Ixoberogene Soroparvovec (Ixo-vec) in Participants With Neovascular Age-Related Macular Degeneration
18 May 2025Conditions: Neovascular Age-Related Macular Degeneration (nAMD); Wet AMD Interventions: Genetic: Ixo-vec; Drug: Aflibercept Sponsors: Adverum Biotechnologies, Inc. Recruiting
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Base-Edited Hematopoietic Stem/Progenitor Cell X-Linked Severe Combined Immunodeficiency Gene Therapy
18 May 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) Enrolling by invitation
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A Randomized Study of SPK-10001 Gene Therapy in Participants With Huntington's Disease
22 May 2025Conditions: Huntington Disease Interventions: Genetic: SPK-10001; Other: Placebo Surgery Control Sponsors: Spark Therapeutics, Inc. Recruiting
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A Study to Investigate the Safety and Biodistribution of a Single Intrathecal (IT) Injection of INS1201 in Ambulatory Males With Duchenne Muscular Dystrophy (DMD)
03 June 2025Conditions: Duchenne Muscular Dystrophy Interventions: Genetic: INS1201 Sponsors: Insmed Gene Therapy LLC Recruiting
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Safety and Efficacy Study of NGGT001 in Bietti Crystalline Corneoretinal Dystrophy Subjects
25 May 2025Conditions: Bietti Crystalline Corneoretinal Dystrophy Interventions: Biological: NGGT001 Sponsors: NGGT (Suzhou) Biotechnology Co., Ltd. Active, not recruiting
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Long Term Follow-up Study of AAVAnc80-hOTOF Gene Therapy
31 May 2025Conditions: Otoferlin Gene-mediated Hearing Loss Sponsors: Akouos, Inc.; Eli Lilly and Company Enrolling by invitation
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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
13 June 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) 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.