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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 Phase I, Open-label, Multicenter, Dose-Escalating Study to Evaluate the Safety and Tolerability of KH658 Gene Therapy in Participants with Neovascular Age-related Macular Degeneration
13 February 2025Conditions: Age-Related Macular Degeneration Interventions: Drug: KH658 Sponsors: Chengdu Origen Biotechnology Co., Ltd. Not yet recruiting
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A Clinical Study Evaluating LY-M001 Injection in the Treatment of Adult Patients with Type I Gaucher Disease
13 February 2025Conditions: Gaucher Disease Type 1 Interventions: Biological: LY-M001 injection Sponsors: Lingyi Biotech Co., Ltd. Recruiting
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The Safety and Efficacy of Intravenous EXG110 in Patients With Fabry Disease
11 February 2025Conditions: Fabry Disease Interventions: Drug: EXG110 Injection Sponsors: Hangzhou Jiayin Biotech Ltd Not yet 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)
10 February 2025Conditions: Duchenne Muscular Dystrophy Interventions: Genetic: INS1201 Sponsors: Insmed Gene Therapy LLC Not yet recruiting
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A Long-term Follow Up Study of EXG102-031 in Patients with WAMD (Everest LTFU)
10 February 2025Conditions: Neovascular Age-Related Macular Degeneration (nAMD); Neovascular (wet) Age-related Macular Degeneration (AMD) Interventions: Biological: Aflibercept (2.0 mg) Sponsors: Exegenesis Bio Enrolling by invitation
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GPC-3 CAR T CELLS for Recurrent GPC-3 Positive Glioblastoma
07 February 2025Conditions: Glioblastoma Multiforme of Brain Interventions: Genetic: 15.GPC3-CAR T cells Sponsors: Baylor College of Medicine; Center for Cell and Gene Therapy, Baylor College of Medicine; Baylor St. Luke's Medical Center Not yet recruiting
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A Safety and Efficacy Long-Term Follow-up Study of Adult Participants Treated With Gene Modified T Cells
29 January 2025Conditions: Long Term Safety Following GM T Cell Therapy Interventions: Genetic: Non-interventional Sponsors: Regeneron Pharmaceuticals Not yet 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
10 February 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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A Clinical Study of Ultra-transplantation for the Treatment of Major Thalassemia Scheme
22 January 2025Conditions: Stem Cell Transplantation Interventions: Other: Supergraft Sponsors: Hu Peng Not yet recruiting
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Long Term Follow-up Study of AAVAnc80-hOTOF Gene Therapy
30 January 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 February 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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A Study to Evaluate the Safety and Efficacy of A2B395, an Allogeneic Logic-gated CAR T, in Participants with Solid Tumors That Express EGFR and Have Lost HLA-A*02 Expression
30 January 2025Conditions: Solid Tumor, Adult; Colorectal Cancer; Non-Small Cell Lung; NSCLC (non-small Cell Lung Cancer); Cancer; Colon Cancer; Rectal Cancer; Lung Cancer; CRC; Head and Neck Squamous Cell Cancer; HNSCC; Renal Cell Carcinoma; RCC; Kidney Cancer; Triple Negative Breast Cancer; TNBC; Colorectal Adenocarcinoma Interventions: Biological: A2B395; Diagnostic Test: xT CDx with HLA-LOH assay Sponsors: A2 Biotherapeutics Inc. Not yet recruiting
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Phase 1 Study to Evaluate Safety and Antiviral Activity of PBGENE-HBV in Adult Patients with Chronic Hepatitis B
10 February 2025Conditions: HEPATITIS B CHRONIC Interventions: Biological: PBGENE-HBV Sponsors: Precision BioSciences, Inc. Recruiting
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A Clinical Study to Evaluate the Safety and Efficacy of LY-M003 Injection in Patients with Wilson Disease
13 February 2025Conditions: Wilson Disease Interventions: Biological: intravenous LY-M003 Sponsors: Chaohui Yu Recruiting
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Hematopoietic Stem Cell BCL11A Enhancer Gene Editing for Severe Β-Hemoglobinopathies
12 February 2025Conditions: 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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A Follow-on Study for Second-Eye Treatment for Participants Previously Treated With Gene Therapy for X-Linked Retinitis Pigmentosa (XLRP)
11 February 2025Conditions: X-Linked Retinitis Pigmentosa Interventions: Biological: AAV5-hRKp.RPGR; Other: No intervention (Follow-Up assessment) Sponsors: Janssen Research & Development, LLC Recruiting
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Patient Interview Study to Explore the Impact of Gene Therapy in Hemophilia A & B
06 February 2025Conditions: Hemophilia A; Hemophilia B Sponsors: Pfizer Not yet recruiting
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Glycogen Storage Disease Type Ia (GSDIa) Disease Monitoring Program
22 January 2025Conditions: Glycogen Storage Disease Type Ia Interventions: Other: No Intervention Sponsors: Ultragenyx Pharmaceutical Inc Recruiting
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Expanded Access Program for RP in Adults
23 January 2025Conditions: 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 )
31 January 2025Conditions: 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
10 February 2025Conditions: 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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Gene Therapy for B-Cell Acute Lymphoblastic Leukemia
07 February 2025Conditions: 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 Phase 1 Study of UB-VV111 With and Without Rapamycin in Relapsed/Refractory CD19+ B-cell Malignancies
13 February 2025Conditions: Lymphoma, B-Cell; Lymphoma, Non-Hodgkin (NHL); Lymphoma, Large B-Cell, Diffuse (DLBCL); Chronic Lymphocytic Leukemia (CLL) Interventions: Genetic: UB-VV111; Drug: rapamycin Sponsors: Umoja Biopharma Recruiting
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Anti-VEGF Gene Therapy Trial for Vestibular Schwannoma
30 January 2025Conditions: 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)
24 January 2025Conditions: 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
05 February 2025Conditions: 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
12 February 2025Conditions: 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); Denovo Biopharma LLC Recruiting
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Study to Evaluate ACDN-01 in ABCA4-related Retinopathy (STELLAR)
22 January 2025Conditions: 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
22 January 2025Conditions: 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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A Phase 3 Study Of OCU400 Gene Therapy for the Treatment Of Retinitis Pigmentosa
11 February 2025Conditions: Retinitis Pigmentosa Interventions: Genetic: Sub-Retinal Administration of OCU400-301 Sponsors: Ocugen 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.