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)
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
- 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.
Most Recent Events
Table of Contents
- At a glance
- Trial Overview
- Trial Details
- Trial Centres
- Trial History