New and Updated Clinical Gene Therapy Trials
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A Gene Transfer Study Inducing Fetal Hemoglobin in Sickle Cell Disease (GRASP, BMT CTN 2001)
Apr 29, 2022 | 18:00 pmCondition: Sickle Cell DiseaseIntervention: Biological: Autologous CD34+ HSC cells transduced with the lentiviral vector containing a shRNA targeting BCL11aSponsors: David Williams; National Heart, Lung, and Blood Institute (NHLBI); California Institute for Regenerative Medicine (CIRM); bluebird bio; Blood and Marrow Transplant Clinical Trials NetworkNot yet recruiting
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Lentiviral Gene Therapy for p47 AR-CGD
Jan 26, 2022 | 18:00 pmCondition: P47-Phox, Deficiency ofIntervention: Drug: Lentiviral vector transduced CD34+ cellsSponsor: Great Ormond Street Hospital for Children NHS Foundation TrustNot yet recruiting
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T Cell Receptor Gene Therapy Targeting KK-LC-1 for Gastric, Breast, Cervical, Lung and Other KK-LC-1 Positive Epithelial Cancers
Sep 5, 2021 | 18:00 pmCondition: Kita-kyushu Lung Cancer Antigen 1, HumanInterventions: Drug: IL-2 (Aldesleukin); Drug: Cyclophosphamide; Biological: KK-LC-1 TCR; Drug: FludarabineSponsor: National Cancer Institute (NCI)Recruiting
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Clinical Study of UX701 AAV-Mediated Gene Transfer for the Treatment of Wilson Disease
May 13, 2021 | 18:00 pmCondition: Wilson DiseaseInterventions: Genetic: UX701; Other: Placebo; Drug: oral corticosteroids; Drug: Placebo for oral corticosteroidsSponsor: Ultragenyx Pharmaceutical IncRecruiting
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A Study of Gene Therapy for Classic Congenital Adrenal Hyperplasia (CAH)
Mar 5, 2021 | 18:00 pmCondition: Congenital Adrenal HyperplasiaIntervention: Biological: AAV BBP-631Sponsor: Adrenas Therapeutics IncRecruiting
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Pivotal 1 Study of RGX-314 Gene Therapy in Participants With nAMD
Jan 12, 2021 | 18:00 pmConditions: AMD; nAMD; Wet Age-related Macular Degeneration; wAMD; Wet AMD; CNVInterventions: Genetic: RGX-314; Biological: Ranibizumab (LUCENTIS®)Sponsor: Regenxbio Inc.Recruiting
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Study of Viral Transduction of Human Auditory Sensory Cells for the Development of Gene Therapy
Jun 25, 2019 | 18:00 pmCondition: Hearing Loss, SensorineuralIntervention: Other: Peroperative collect of inner ear cellsSponsors: Institut Pasteur; Assistance Publique - Hôpitaux de ParisRecruiting
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A Phase 1/2 Study of Intravenous Gene Transfer With an AAV9 Vector Expressing Human Beta-galactosidase in Type I and 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); Sio Gene TherapiesRecruiting
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Intrathecal Administration of scAAV9/JeT-GAN for the Treatment of Giant Axonal Neuropathy
Feb 13, 2015 | 18:00 pmConditions: Giant Axonal Neuropathy; Gene TransferIntervention: Genetic: scAAv9/JeT-GANSponsor: National Institute of Neurological Disorders and Stroke (NINDS)Recruiting
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Follow-Up Study of Subjects Previously Enrolled in Poxviral Vector Gene Transfer Studies
Mar 22, 2007 | 17:00 pmConditions: Prostate Cancer; Liver Cancer; Breast Cancer; Colon Cancer; Lung CancerIntervention: Sponsor: National Cancer Institute (NCI)Recruiting
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