BOOSTB4: Views on stem cell therapy for Osteogenesis imperfecta v1
Research type
Research Study
Full title
Pre- and post-natal stem cell therapy for Osteogenesis imperfecta: A mixed-methods study to explore the views and attitudes of key stakeholders
IRAS ID
205688
Contact name
Vanshree Patel
Contact email
Sponsor organisation
Great Ormond Street Hospital NHS Foundation Trust
Duration of Study in the UK
1 years, 11 months, 27 days
Research summary
Osteogenesis Imperfecta (OI) is a genetic condition characterised by bones that break easily. Other medical problems include; loose joints, early hearing loss, brittle teeth, respiratory problems and easy bruising. OI is caused by a genetic mutation that affects the body’s production of collagen. In people affected with OI the collagen may be of poor quality or there may not be enough which makes the bones more susceptible to fractures. There is no cure for OI and current treatments are palliative and do not address the underlying bone brittleness and collagen defect. A new research study called Boost Brittle Bones Before Birth (BOOSTB4) is investigating the possibility of using stem cell therapy as treatment for severe OI. The treatment involves transplanting stem cells into the baby affected with OI during pregnancy or soon after birth. The stem cells will make healthy collagen that will support damaged bones and help with the development of new normal bone. The BOOSTB4 clinical trial aims to test whether the treatment is safe and effective. In this arm of the BOOSTB4 study we will evaluate the acceptability of stem cell treatment for OI from the patient and family perspective as well as from health professionals and patient advocates in the area. Focus groups and face-to-face or telephone interviews will be conducted and audio-recorded. The data will be transcribed and analysed thematically using codes to obtain an overall understanding of the views of respondents about stem cell therapy and the proposed BOOSTB4 trial. The qualitative data will be used to develop a questionnaire to gather views more widely amongst the stakeholder groups.
REC name
North of Scotland Research Ethics Committee 2
REC reference
16/NS/0084
Date of REC Opinion
1 Aug 2016
REC opinion
Further Information Favourable Opinion