A mixed methodology study to evaluate the care of CALD patients
Research type
Research Study
Full title
A mixed methodology study to evaluate treatment patterns, healthcare resource use and outcomes for patients with cerebral adrenoleukodystrophy who either have received, or were deemed ineligible for, allogeneic haematopoietic stem cell transplantation in the United Kingdom
IRAS ID
260835
Contact name
Colin Graham Steward
Contact email
Sponsor organisation
bluebird bio, Inc
Clinicaltrials.gov Identifier
NA, NA
Duration of Study in the UK
0 years, 4 months, 14 days
Research summary
This is a UK, multi-centre, mixed methodology, non-interventional study designed to describe the clinical management and treatment pathways, healthcare resource use (HRU) and outcomes of 1. paediatric patients with cerebral adrenoleukodystrophy (CALD) treated with allo-HSCT in routine clinical practice in the UK and 2. paediatric patients with CALD deemed ineligible for allo-HSCT due to advanced disease at diagnosis. This study will be conducted in NHS hospitals in the UK. The study will also explore parent/carer burden, sources of support and demographics.
The study is based on retrospective chart review and a parents/carers cross-sectional survey. There will be no changes to patient management for the purposes of any part of the study and no additional tests, investigations or visits will be required.
The target population for this study is children diagnosed with cerebral adrenoleukodystrophy (CALD) and their parents/carers. It is expected to recruit 40 patients with CALD (15-20 patients with CALD who have received an allogeneic haematopoietic stem cell transplant (allo-HSCT) and 15-20 patients with CALD deemed ineligible for allo-HSCT because of advanced disease at diagnosis) and approx. 40 parents/carers for completion of questionnaires.
REC name
South Central - Berkshire Research Ethics Committee
REC reference
19/SC/0192
Date of REC Opinion
7 May 2019
REC opinion
Favourable Opinion