New and Updated Clinical Gene Therapy Trials
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rAAV-Olig001-ASPA Gene Therapy for Treatment of Children With Typical Canavan Disease
Apr 6, 2021 | 18:00 pmCondition: Canavan DiseaseInterventions: Drug: rAAV-Olig001-ASPA; Drug: Levetiracetam; Drug: PrednisoloneSponsor: CureRareDisease LLCRecruiting
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RGX-314 Gene Therapy Pharmacodynamic Study for Neovascular Age-related Macular Degeneration (nAMD)
Apr 6, 2021 | 18:00 pmConditions: Neovascular Age-related Macular Degeneration; Wet Macular Degeneration; Wet Age-related Macular DegenerationIntervention: Genetic: RGX-314Sponsor: Regenxbio Inc.Recruiting
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Phase I/II Clinical Trial Stem Cell Gene Therapy in RAG1-Deficient SCID
Mar 15, 2021 | 17:00 pmCondition: Severe Combined Immunodeficiency Due to RAG1 DeficiencyIntervention: Genetic: Gene therapySponsors: Leiden University Medical Center; ZonMw: The Netherlands Organisation for Health Research and DevelopmentRecruiting
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Study to Evaluate the Efficacy and Safety of PF-07055480 in Moderately Severe to Severe Hemophilia A Adults
Apr 30, 2020 | 18:00 pmCondition: Hemophilia AIntervention: Biological: Recombinant AAV2/6 Human Factor VIII Gene TherapySponsor: PfizerRecruiting
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TAC Chemotherapy and Pembrolizumab Plus Interleukin-12 Gene Therapy and L-NMMA in Triple Negative Breast Cancer
Sep 19, 2019 | 18:00 pmCondition: Triple Negative Breast CancerInterventions: Drug: Docetaxel; Drug: Doxorubicin; Drug: Cyclophosphamide; Drug: Pembrolizumab; Drug: IL-12 gene therapy; Drug: L-NMMASponsors: The Methodist Hospital System; Merck Sharp & Dohme Corp.Recruiting
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A Phase 1/2 Study of Intravenous Gene Transfer With an AAV9 Vector Expressing Human <=-Galactosidase in Type II GM1 Gangliosidosis
May 16, 2019 | 18:00 pmConditions: Lysosomal Diseases; Gangliosidosis; GM1Interventions: Biological: AAV9-GLB1; Drug: Rituximab; Drug: Sirolimus; Drug: Methylprednisolone; Drug: Prednisone; Diagnostic Test: Audiology assessmentwith ABR; Diagnostic Test: Bone density scan (DEXA; Diagnostic Test: Electrocardiogram (EKG); Diagnostic Test: Echocardiogram; Other: Electroencephalogram (EEG) awake andextended overnight; Diagnostic Test: Laboratory tests; Procedure: Lumbar puncture; Procedure: Brain MRI/MRS/fMRI; Behavioral: Neurocognitive testing; Other: Neurology exam; Behavioral: PICC line placement; Procedure: Skeletal survey; Procedure: Skin biopsy; Procedure: Speech and modified barium swallow study; Procedure: Ophthalmology examSponsors: National Human Genome Research Institute (NHGRI); Axovant Sciences, Inc.Recruiting
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Gene Therapy for APOE4 Homozygote of Alzheimer's Disease
Aug 16, 2018 | 18:00 pmConditions: Alzheimer Disease; Early Onset Alzheimer DiseaseIntervention: Biological: LX1001Sponsors: Lexeo Therapeutics; Alzheimer's Drug Discovery Foundation; Weill Medical College of Cornell UniversityRecruiting
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Phase II High Risk Prostate Cancer Trial Using Gene & Androgen Deprivation Therapies, Radiotherapy, & Surgery
May 31, 2018 | 18:00 pmConditions: High-risk Prostate Cancer; Prostate CancerInterventions: Drug: HSV-Tk; Drug: Valacyclovir; Drug: Bicalutamide; Drug: Leuprolide Acetate; Radiation: Brachytherapy, External beam radiotherapy; Procedure: Radical prostatectomySponsor: The Methodist Hospital SystemRecruiting
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Follow-Up Evaluation for Gene-Therapy-Related Delayed Adverse Events After Participation in Pediatric Oncology Branch Clinical Trials
Dec 12, 2014 | 18:00 pmConditions: Pediatric Cancers; Hematologic Malignancies; Solid TumorsIntervention: Sponsor: National Cancer Institute (NCI)Recruiting
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Lentiviral Gene Transfer for Treatment of Children Older Than Two Years of Age With X-Linked Severe Combined Immunodeficiency (XSCID)
Mar 1, 2011 | 18:00 pmCondition: X-Linked Severe Combined Immune Deficiency (XSCID)Interventions: Drug: Palifermin; Drug: Busulfan; Biological: Ex vivo culture and transduction of the patient's autologous CD34+ HSC with lentivirus vector VSV-G pseudotyped CL20- 4i-EF1a-hyc-OPT vectorSponsor: National Institute of Allergy and Infectious Diseases (NIAID)Recruiting
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