Genetically engineered hematopoietic stem/progenitor cells (DrugBank: -)
1 diseaseID | Disease name (Link within this page) | Number of trials |
---|---|---|
285 | Fanconi anemia | 1 |
No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
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1 | NCT03157804 (ClinicalTrials.gov) | January 7, 2016 | 26/4/2017 | Lentiviral-mediated Gene Therapy of Fanconi Anemia Patients Subtype A | Clinical Trial Phase I / II to Evaluate the Safety and Efficacy of the Infusion of Autologous CD34 + Cells Transduced With a Lentiviral Vector Carrying the Gene FANCA in Patients With FA Subtype A (FANCOLEN-1) | Fanconi Anemia | Procedure: IV administration of Genetically Engineered Hematopoietic Stem/Progenitors Cells (HSPCs);Biological: Genetically Engineered Hematopoietic Stem/Progenitor Cells;Other: Laboratory Biomarker Analysis;Biological: Filgrastim;Drug: Plerixafor;Procedure: Bone Marrow Aspiration | Hospital Infantil Universitario Niño Jesús, Madrid, Spain | Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas (CIEMAT);Centro de Investigación en Red de Enfermedades Raras (CIBERER);Instituto de Investigación Sanitaria de la Fundación Jiménez Díaz;Hospital Vall d'Hebron;Universitat Autonoma de Barcelona | Active, not recruiting | 1 Year | 21 Years | All | 9 | Phase 1;Phase 2 | Spain |