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BSH2020‐OR‐037 : Clinical outcomes following autologous haematopoietic stem cell transplantation with LentiGlobin gene therapy in the phase 3 Northstar‐2 and Northstar‐3 studies for transfusion‐dependent β‐thalassaemia

Researchers

Presenter

  • J. B. Porter

Principal Investigators

  • F. Locatelli

  • J. L. Kwiatkowski

  • A. A. Thompson

  • E. Yannaki

  • A. E. Kulozik

  • M. G. Sauer

  • I. Thuret

  • S. Hongeng

  • A. Lal

  • A. J. Thrasher

  • H. Elliot

  • G. Tao

  • W. Liu

  • R. A. Colvin

  • M. C. Walters

Medical Centers

  • Haematology Department, University College London Hospitals, London, UK

  • Department of Gene Therapy, IRCCS Bambino Gesù Children's Hospital, Rome, Italy

  • Department of Pediatrics, University of Pennsylvania Perelman School of Medicine

  • Division of Human Genetics, Children's Hospital of Philadelphia, Philadelphia, PA

  • Pediatrics/Hematology, Oncology & Stem Cell Transplant, Northwestern University Feinberg School of Medicine, Chicago, IL

  • Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, IL

  • Gene and Cell Therapy Center of the Hematology Dept /Hematopoietic Cell Transplantation Unit,Papanicolaou Hospital,Thessaloniki,Greece

  • Molecular Medicine Partnership Unit (MMPU), European Molecular Biology Laboratory

  • Department of Pediatric Oncology, Hematology and Immunology,, University of Heidelberg, Heidelberg, Germany

  • Dept. of Hematology/Oncology, Medizinische Hochschule Hannover, Hannover, Germany

  • Pediatric Hematology Department, Hopital de La Timone, Marseille, France

  • Mahidol University and Ramathibodi Hospital, Bangkok, Thailand,

  • UCSF Benioff Childrens Hospital Oakland, Oakland, CA

  • UCL Great Ormond Street Institute of Child Health, London, UK

  • bluebird bio, Inc., Cambridge, MA.

Locations

  • United Kingdom

  • Italy

  • United States

  • Greece

  • Germany

  • France

  • Thailand

Companies

  • N/A

Study Components

Therapeutic Area

  • Blood/haematological Diseases

Disease

  • Transfusion‐dependent β‐thalassaemia

  • Thrombocytopenia

  • Neutropenia

Biomarkers

  • Cluster of Differentiation 34

  • Ubiquitin A-52 Residue Ribosomal Protein Fusion Product 1

  • Tumor doubling time

Drug/Treatment

  • Defibrotide

  • Busulfan

Outcome

  • N/A

Study Design

  • Pharmacokinetics

Phase

  • III

Study Id's

  • NCT02906202

  • NCT03207009

Sponsors

  • N/A

Result

  • N/A

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