A Phase 2/3 Study of the Efficacy and Safety of Hematopoietic Stem Cells Transduced With Lenti-D Lentiviral Vector for the Treatment of Childhood Cerebral Adrenoleukodystrophy (CCALD)

Trial Profile

A Phase 2/3 Study of the Efficacy and Safety of Hematopoietic Stem Cells Transduced With Lenti-D Lentiviral Vector for the Treatment of Childhood Cerebral Adrenoleukodystrophy (CCALD)

Recruiting
Phase of Trial: Phase II/III

Latest Information Update: 11 Oct 2017

At a glance

  • Drugs Lenti-D ABCD1 gene therapy-bluebird bio (Primary) ; Busulfan; Cyclophosphamide; Filgrastim
  • Indications Adrenoleucodystrophy
  • Focus Registrational; Therapeutic Use
  • Acronyms STARBEAM
  • Sponsors bluebird bio
  • Most Recent Events

    • 04 Oct 2017 Interim results from an initial cohort of 17 patients of this trial, published in the New England Journal of Medicine.
    • 04 Oct 2017 Interim data published in the bluebird bio Media Release
    • 06 Sep 2017 According to a bluebird bio media release, interim data will be presented in a poster at the Child Neurology Society (CNS) 2017 and at the 13th International Congress of Inborn Errors of Metabolism (ICIEM).
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