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: 29 Jul 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

    • 26 Jun 2017 Topline interim data (n=17) from this trial published in a bluebird bio Media Release.
    • 22 Feb 2017 According to a bluebird bio media release, the company expects to present full data from initial 17 patients treated in this trial by year end 2017.
    • 22 Feb 2017 According to a bluebird bio media release, this study has been reopened to expand enrollemtn by up to eight additional patients. The expansion of the study is intended to enable the first manufacture of Lenti-D in Europe and subsequent treatment of subjects in Europe, and to bolster the overall clinical data package for potential future regulatory filings in the United States and Europe.
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