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