Journal article

Single group multisite safety trial of sibling cord blood cell infusion to children with cerebral palsy: study protocol and rationale

Kylie Crompton, Iona Novak, Michael Fahey, Nadia Badawi, Euan Wallace, Katherine Lee, Francoise Mechinaud-Heloury, Paul B Colditz, Ngaire Elwood, Priya Edwards, Dinah Reddihough

BMJ Open | BMJ Journals | Published : 2020


INTRODUCTION: Cerebral palsy (CP) is the most common physical disability of childhood but has no cure. Stem cells have the potential to improve brain injury and are proposed as a therapy for CP. However, many questions remain unanswered about the most appropriate cell type, timing of infusions, dose required and associated risks. Therefore, human safety and efficacy trials are necessary to progress knowledge in the field. METHODS AND ANALYSIS: This is a single group study with sample size n=12 to investigate safety of single-dose intravenous 12/12 human leucocyte antigen-matched sibling cord blood cell infusion to children with CP aged 1-16 years without immune suppression. The study is simi..

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Funding Acknowledgements

This work is generously supported by Cell Care Australia, which aided in study design and is represented on the Trial Steering Committee but will not be involved in data analysis or interpretation; and the Cerebral Palsy Alliance Research Foundation, a not-for-profit organisation that aided in study design, is represented on the Trial Steering Committee and will be involved in data interpretation and dissemination. Neither organisation can withhold publication. The study is sponsored by Murdoch Children's Research Institute.