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

ABSTRACT:

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 similar to a 3+3 design, where the first two groups of participants have severe CP, and the

SUBMITTER: Crompton K 

PROVIDER: S-EPMC7064081 | biostudies-literature | 2020 Mar

REPOSITORIES: biostudies-literature