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: 17 Nov 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

    • 15 Nov 2017 Planned number of patients changed from 25 to 30.
    • 01 Nov 2017 According to a bluebird bio media release, interim data from the study were presented at the Child Neurology Society (CNS) Annual Meeting.
    • 04 Oct 2017 Interim results from an initial cohort of 17 patients of this trial, published in the New England Journal of Medicine.
Restricted Access

Oops, it looks like you don’t have a valid subscription to this content. To gain full access to the content and functionality of the AdisInsight database try one of the following.

  • with a username/password associated to an account with a valid subscription
  • Contact your organization’s admin about adding this content to your AdisInsight subscription
  • Request a trial

If you are a subscriber to this content then contact us at AsktheExpert.AdisInsight@springer.com so we can help.

Back to top