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A Pilot Feasibility Study of Gene Transfer for X-Linked Severe Combined Immunodeficiency in Newly Diagnosed Infants Using a Self-Inactivating Lentiviral Vector to Transduce Autologous CD34+ Hematopoietic Cells

Trial Profile

A Pilot Feasibility Study of Gene Transfer for X-Linked Severe Combined Immunodeficiency in Newly Diagnosed Infants Using a Self-Inactivating Lentiviral Vector to Transduce Autologous CD34+ Hematopoietic Cells

Recruiting
Phase of Trial: Phase I/II

Latest Information Update: 20 Feb 2019

At a glance

  • Drugs MB 107 (Primary) ; Busulfan
  • Indications Immunodeficiency disorders
  • Focus Adverse reactions; Therapeutic Use
  • Acronyms LVXSCID-ND
  • Most Recent Events

    • 31 Aug 2018 Biomarkers information updated
    • 22 Aug 2018 New trial record
    • 13 Aug 2018 According to a Mustang Bio media release, this multicenters study is the first lentiviral gene therapy trial for infants with X-SCID.
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