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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

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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

Status: Withdrawn prior to enrolment
Phase of Trial: Phase I

Latest Information Update: 03 Mar 2022

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At a glance

  • Drugs Marnetegragene autotemcel (Primary)
  • Indications Immunodeficiency disorders; Leucocyte disorders
  • Focus Adverse reactions; First in man; Therapeutic Use
  • Sponsors Rocket Pharmaceuticals

Most Recent Events

  • 23 Nov 2021 Status changed from recruiting to withdrawn prior to enrolment because patients were enrolled under global Phase 1/2 protocol RP-L201-0318.
  • 09 Mar 2021 According to a Rocket Pharmaceuticals media release, data from our LAD-I trial is anticipated in the second quarter 2021.
  • 05 Aug 2020 According to a Rocket Pharmaceuticals media release, trial data is expected in the Q4 2020.

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