New and Updated Clinical Gene Therapy Trials
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Phase IA Study of AAVrh.10hFXN Gene Therapy for the Cardiomyopathy of Friedreich's Ataxia
Mar 31, 2022 | 18:00 pmConditions: Friedreich Ataxia; Cardiomyopathies; Cardiac Hypertrophy; Myocardial FibrosisInterventions: Biological: AAVrh.10hFXN, serotype rh.10 adeno-associated virus (AAV) gene transfer vector expressing the cDNA coding for human FXN; Drug: PrednisoneSponsors: Weill Medical College of Cornell University; National Heart, Lung, and Blood Institute (NHLBI)Recruiting
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4D-710 in Adult Patients With Cystic Fibrosis
Feb 21, 2022 | 18:00 pmCondition: Cystic Fibrosis LungIntervention: Biological: 4D-710Sponsor: 4D Molecular TherapeuticsRecruiting
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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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A Gene Therapy Study of BMN 331 in Subjects With Hereditary Angioedema
Nov 16, 2021 | 18:00 pmConditions: Hereditary Angioedema; HAEInterventions: Genetic: Dose 1 of BMN 331; Genetic: Dose 2 of BMN 331; Genetic: Dose 3 of BMN 331; Genetic: Dose 4 of BMN 331; Genetic: Dose 5 of BMN 331Sponsor: BioMarin PharmaceuticalRecruiting
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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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GDNF Gene Therapy for Multiple System Atrophy
Dec 22, 2020 | 18:00 pmCondition: Multiple System AtrophyInterventions: Biological: AAV2-GDNF gene therapy; Procedure: Sham (Placebo) SurgerySponsor: Brain Neurotherapy Bio, Inc.Recruiting
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CORONA: A Study Using DeltaRex-G Gene Therapy for Symptomatic COVID-19
May 7, 2020 | 18:00 pmConditions: COVID-19; Cytokine Storm; Acute Respiratory Distress SyndromeIntervention: Drug: DeltaRex-GSponsor: Aveni FoundationNot yet recruiting
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Phase I/II Trial of Lentiviral Gene Transfer for SCID-X1 With Low Dose Targeted Busulfan Conditioning
Oct 17, 2017 | 18:00 pmConditions: Severe Combined Immunodeficiency, X Linked; Gene TherapyIntervention: Biological: autologous CD34+ cell transduced with G2SCID vectorSponsor: David WilliamsRecruiting
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A Gene Transfer Study for Hemophilia A
Dec 28, 2016 | 18:00 pmCondition: Hemophilia AIntervention: Genetic: SPK-8011Sponsor: Spark TherapeuticsRecruiting
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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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