Safety and efficacy of allogeneic umbilical cord blood therapy combined with erythropoietin in children with cerebral palsy: study protocol for a double-blind, randomized, placebo-controlled trial

Background and objectives: Cerebral palsy typically results from brain injury during prenatal, perinatal, or infant period. Established medical management involves rehabilitation therapy with comprehensive multidisciplinary approach to minimize various complications and to improve brain plasticity. Recently, direct brain tissue modifying therapy using stem cell therapy has been investigated as a new therapeutic modality. In this study, the combination therapy of allogeneic umbilical cord blood and/or erythropoietin is investigated to find out the efficacy and safety in children with cerebral palsy over empirical rehabilitation therapy. Design: A randomized, double blind, controlled trial. Methods: Patients with cerebral palsy who visited Rehabilitation medicine center in CHA Bundang Medical Center, Republic of Korea from October 2013 to October 2015 are included in this trial. A sample size of 120 patients is needed. Patients will be randomly assigned to three groups of cord blood treatment or a control group. Cord blood treatment group will receive either cord blood and erythropoietin, or cord blood and erythropoietin placebo, or placebo cord blood and erythropoietin in addition to conservative rehabilitation therapy. Outcome measures: The primary outcome of the study is the efficacy of combination therapy of allogeneic cord blood stem cell and erythropoietin and either monotherapy in terms of the change from baseline motor function assessments including Gross Motor Function Measure, Gross Motor Performance Measure, motor and mental subscales of Korean version Bayley Scales of Infant Development, 2nd edition. The secondary outcomes of the study include other functional assessment tools, imaging studies (fractional anisotropy of diffusion tensor imaging and positron emission tomography), electroencephalography, and evoked potentials. Discussion: The results of this trial will provide optimal evidence of cord blood transplantation with or without erythropoietin as a therapeutic modality in children with cerebral palsy. Ethics and dissemination: The study will be reviewed by IRB. The results of the trial will be published in a peer-reviewed journal and will be disseminated via various forms of media. Trial registration: This trial had been registered in the ClinicalTrials.gov (identifier: NCT0199114) on 17 November 2013.