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)

Active, no longer recruiting
Phase of Trial: Phase II/III

Latest Information Update: 19 Jun 2018

At a glance

  • Drugs Elivaldogene-tavalentivec (Primary) ; Busulfan; Cyclophosphamide
  • Indications Adrenoleucodystrophy
  • Focus Registrational; Therapeutic Use
  • Acronyms STARBEAM
  • Sponsors bluebird bio
  • Most Recent Events

    • 23 May 2018 According to a bluebird bio media release, based on the preliminary data from this study, the U.S. Food and Drug Administration has granted Breakthrough Therapy designation to Lenti-D for the treatment of patients with cerebral adrenoleukodystrophy (CALD).
    • 15 May 2018 Planned primary completion date changed from 1 Aug 2019 to 1 Jul 2020.
    • 15 May 2018 Status changed from recruiting to active, no longer recruiting.
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