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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)

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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)

Status: Completed
Phase of Trial: Phase II/III

Latest Information Update: 19 Sep 2022

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At a glance

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

    • 16 Sep 2022 According to a bluebird bio media release, based on ALD-102 and ALD-104 trial results, the U.S. Food and Drug Administration (FDA) has granted Accelerated Approval of SKYSONA (elivaldogene autotemcel), also known as eli-cel, to slow the progression of neurologic dysfunction in boys 4-17 years of age with early, active cerebral adrenoleukodystrophy (CALD).
    • 09 Jun 2022 According to a bluebird bio media release, the FDA Cellular, Tissue, and Gene Therapies Advisory Committee (CTGTAC) endorsed unanimously elivaldogene autotemcel (eli-cel) for the treatment of early active cerebral adrenoleukodystrophy (CALD) in patients less than 18 years of age who do not have an available and willing human leukocyte antigen (HLA)-matched sibling hematopoietic stem cell (HSC) donor based on Biologics License Application (BLA) currently under priority review.
    • 09 May 2022 According to a bluebird bio media release, The full agenda will be determined by the FDA and included in briefing materials that will be posted by the FDA closer to the date of the advisory committee meeting.
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