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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 Prospective, Single-center, Open-label Clinical Study to Evaluate the Safety and Efficacy of Inhaled BMD003(CFTR mRNA) in Chinese Adult Patients With Cystic Fibrosis
08 May 2025Conditions: Cystic Fibrosis (CF); Gene Therapy Interventions: Drug: inhaled BMD003(CFTR mRNA) Sponsors: Peking Union Medical College Hospital Not yet 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)
08 May 2025Conditions: Cystic Fibrosis Interventions: Genetic: BI 3720931 Sponsors: Boehringer Ingelheim Not yet recruiting
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Base Editing Hematopoietic Stem Cell Gene Therapy for CD40L-HyperIgM Syndrome: Single Patient Study
13 May 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
01 May 2025Conditions: Muscular Dystrophies, Limb-Girdle Interventions: Biological: SRP-9005; Drug: Corticosteroid Sponsors: Sarepta Therapeutics, Inc. Not yet recruiting
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Safety and Efficacy of AAV9/AP4B1 (BFB-101) For Patients With AP4B1-related Hereditary Spastic Paraplegia Type 47 (SPG47)
28 April 2025Conditions: HSP; Hereditary Spastic Paraplegia; Hereditary Spastic Paraparesis; Hereditary Spastic Paraplegia Type 50; Hereditary Spastic Paraplegia Type 47; Hereditary Spastic Paraplegia Type 51; Hereditary Spastic Paraplegia Type 52; SPG47; AP4B1; Neurogenetic Disorders; Neurodevelopmental Conditions; Movement Disorders; Gene Therapy Interventions: Biological: BFB-101 (AAV9-CBh-AP4B1) Sponsors: BlackfinBio Ltd; Boston Children's Hospital Not yet recruiting
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Phase 2 Trial of RGX-314 in Adults With Center Involved - Diabetic Macular Edema (CI - DME)
24 April 2025Conditions: Diabetic Macular Edema Interventions: Genetic: RGX-314 Dose 1; Genetic: RGX-314 Dose 2; Biological: Aflibercept (2.0 mg) Sponsors: Sierra Eye Associates; REGENXBIO Inc. Recruiting
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A Phase 1/2, First-in-Human Dose Escalation/Expansion Study to Evaluate the Safety, Tolerability and Preliminary Efficacy of a Subretinal Injection of SB-007 in Subjects With Stargardt Disease (STGD1)
29 April 2025Conditions: Stargardt Disease; Stargardt Macular Degeneration; Stargardt Macular Dystrophy Interventions: Genetic: SB-007 Sponsors: Splice Bio Recruiting
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A First-in-human Clinical Trial Using a Gene Therapy With Patient's Own Stem Cells to Treat Early Type 1 Diabetes
22 April 2025Conditions: Diabetes, Type I Interventions: Genetic: Autologous CD34+ cell enriched population containing HSPCs transduced ex vivo using a LVV encoding the hPD-L1 DNA Sponsors: Altheia Science; Evidenze Health S.r.l. Not yet recruiting
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Anti-CD7 CAR-T Cells in Relapsed/Refractory T-Cell Acute Lymphoblastic Leukemia or Lymphoma
02 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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DFT383 in Pediatric Participants With Nephropathic Cystinosis
07 May 2025Conditions: Nephropathic Cystinosis Interventions: Genetic: DFT383 Sponsors: Novartis Pharmaceuticals Recruiting
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Autologous T Cells Transduced With Retroviral Vectors Expressing TCRs for Participant-specific Neoantigens in Patients With Hematologic Malignancies
13 May 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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4D-150 in Patients With Macular Neovascularization Secondary to Age-Related Macular Degeneration
08 May 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
08 May 2025Conditions: Locally Advanced Pancreatic Adenocarcinoma Interventions: Drug: RR001 Sponsors: EIR Biotherapies s.r.l. Recruiting
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Efficacy and Safety Study of Ixoberogene Soroparvovec (Ixo-vec) in Participants With Neovascular Age-Related Macular Degeneration
27 April 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
13 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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To Evaluate the Clinical Study of GS1191-0445 Injection in the Treatment of Hemophilia A
24 April 2025Conditions: Hemophilia A Interventions: Drug: GS1191-0445 injection Sponsors: Gritgen Therapeutics Co., Ltd. Recruiting
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Study Assessing Left Ventricular Administration of a Genetic Medicine Directing Organ Regeneration in Heart Failure
27 April 2025Conditions: Heart Failure With Reduced Ejection Fraction Interventions: Combination Product: YAP101 (AAV9-Sav-shRNA) Sponsors: YAP 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)
24 April 2025Conditions: Duchenne Muscular Dystrophy Interventions: Genetic: INS1201 Sponsors: Insmed Gene Therapy LLC Recruiting
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A Safety and Efficacy Long-Term Follow-up Study of Adult Participants Treated With Gene Modified T Cells
11 May 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
27 April 2025Conditions: Limb Girdle Muscular Dystrophy; Limb Girdle Muscular Dystrophy Type 2D/R3 Interventions: Drug: SRP-9004 Sponsors: Sarepta Therapeutics, Inc. Active, not recruiting
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Impact of COL7A1 Gene Therapy on SCC Recurrence in RDEB Skin
16 April 2025Conditions: Squamous Cell Carcinoma Interventions: Drug: BVEC Sponsors: Stanford University; Epidermolysis Bullosa Research Partnership Recruiting
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A Study Assessing KB304 for the Treatment of Wrinkles in Women
13 May 2025Conditions: Wrinkles in Decolletage; Pigmentation; Décolleté Wrinkles Interventions: Biological: KB304; Drug: 0.9% Normal Saline (NS) Sponsors: Krystal Biotech, Inc. Active, not 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
13 May 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
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Long Term Follow-Up Study for Individuals With Hemoglobin Disorders After Hematopoietic Cell Transplant or Gene Therapy
23 April 2025Conditions: Hemoglobin Disorder Sponsors: St. Jude Children's Research Hospital Recruiting
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Glycogen Storage Disease Type Ia (GSDIa) Disease Monitoring Program
11 May 2025Conditions: Glycogen Storage Disease Type Ia Interventions: Other: No Intervention Sponsors: Ultragenyx Pharmaceutical 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
06 May 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 CD19-Positive Hematologic Malignancies
11 May 2025Conditions: B-cell Acute Lymphoblastic Leukemia; Large B-cell Lymphoma; Chronic Lymphocytic Leukemia; Small Lymphocytic Lymphoma; Marginal Zone Lymphoma; Follicular Lymphoma; Mantle Cell Lymphoma; Diffuse Large B Cell Lymphoma; High-grade B-cell Lymphoma; Burkitt Lymphoma; Primary Mediastinal Large B-cell Lymphoma (PMBCL); Non Hodgkin Lymphoma; Mixed Phenotype Acute 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 Study to Test How Well BI 3720931 is Tolerated and Whether it Improves Lung Function in People With Cystic Fibrosis (Lenticlair™ 1)
16 April 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
13 May 2025Conditions: Intermediate Risk Non-Muscle Invasive Bladder Cancer Interventions: Drug: Nadofaragene Firadenovec Sponsors: Ferring Pharmaceuticals Recruiting
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Long Term Follow-up for RGX-202
13 May 2025Conditions: Duchenne Muscular Dystrophy Interventions: Genetic: No Intervention Sponsors: REGENXBIO Inc. Enrolling by invitation
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.