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Gene Therapy for Leukocyte Adhesion Deficiency-I (LAD-I): A Phase I Clinical Trial to Evaluate the Safety and Efficacy of the Infusion of Autologous Hematopoietic Stem Cells Transduced with a Lentiviral Vector Encoding the ITGB2 Gene

Trial Profile

Gene Therapy for Leukocyte Adhesion Deficiency-I (LAD-I): A Phase I Clinical Trial to Evaluate the Safety and Efficacy of the Infusion of Autologous Hematopoietic Stem Cells Transduced with a Lentiviral Vector Encoding the ITGB2 Gene

Recruiting
Phase of Trial: Phase I

Latest Information Update: 02 Jan 2019

At a glance

  • Drugs RP L201 (Primary)
  • Indications Immunodeficiency disorders; Leucocyte disorders
  • Focus First in man; Registrational; Therapeutic Use
  • Sponsors Rocket Pharmaceuticals
  • Most Recent Events

    • 29 Dec 2018 Status changed from planning to recruiting.
    • 05 Dec 2018 New trial record
    • 19 Nov 2018 According to a Rocket Pharmaceuticals media release, the U.S. FDA has cleared the IND application submitted to initiate this trial. The company expects to commence this trial in 2019.
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