Please use this identifier to cite or link to this item: https://dora.health.qld.gov.au/qldresearchjspui/handle/1/4601
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dc.contributor.authorEdwards, P.en
dc.contributor.authorReddihough, D.en
dc.contributor.authorCrompton, K.en
dc.contributor.authorNovak, I.en
dc.contributor.authorFahey, M.en
dc.contributor.authorBadawi, N.en
dc.contributor.authorWallace, E.en
dc.contributor.authorLee, K.en
dc.contributor.authorMechinaud-Heloury, F.en
dc.contributor.authorColditz, P. B.en
dc.contributor.authorElwood, N.en
dc.date.accessioned2022-11-07T23:54:28Z-
dc.date.available2022-11-07T23:54:28Z-
dc.date.issued2020en
dc.identifier.citation10, (3), 2020en
dc.identifier.otherRISen
dc.identifier.urihttp://dora.health.qld.gov.au/qldresearchjspui/handle/1/4601-
dc.description.abstractIntroduction 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 final six participants include children with all motor severities. Children will be monitored for adverse events and the duration that donor cells are detected. Assessments at baseline, 3 and 12 months will investigate safety and preliminary evidence of change in gross motor, fine motor, cognitive and quality of life outcomes. Ethics and dissemination Full approval was obtained from The Royal Children's Hospital Human Research Ethics Committee, and a clinical trial notification was accepted by Australia's Therapeutic Goods Administration. Participant guardian informed consent will be obtained before any study procedures. The main results of this study will be submitted for publication in a peer-reviewed journal. Trial registration number ACTRN12616000403437, NCT03087110.L6311496992020-03-19 <br />2020-03-26 <br />en
dc.language.isoenen
dc.relation.ispartofBMJ Openen
dc.titleSingle group multisite safety trial of sibling cord blood cell infusion to children with cerebral palsy: Study protocol and rationaleen
dc.typeArticleen
dc.identifier.doi10.1136/bmjopen-2019-034974en
dc.subject.keywordssibling cord blood cell infusionen
dc.subject.keywordsadolescentarticleen
dc.subject.keywordsBeery-Buktenica developmental test of visual-motor integrationen
dc.subject.keywordsbehavior assessmenten
dc.subject.keywordsbehavior rating inventory of executive functionen
dc.subject.keywordsblood transfusionen
dc.subject.keywordscerebral palsyen
dc.subject.keywordschilden
dc.subject.keywordsclinical articleen
dc.subject.keywordscord blood stem cell transplantationen
dc.subject.keywordsdisease associationen
dc.subject.keywordsHLA matchingen
dc.subject.keywordshumanen
dc.subject.keywordshuman cellen
dc.subject.keywordsimmunosuppressive treatmenten
dc.subject.keywordspatient monitoringen
dc.subject.keywordspatient safetyen
dc.subject.keywordsquality of lifeen
dc.subject.keywordsstrengths and difficulties questionnaireen
dc.subject.keywordsVineland Adaptive Behavior Scaleen
dc.subject.keywordsWechsler intelligence scale for childrenen
dc.relation.urlhttps://www.embase.com/search/results?subaction=viewrecord&id=L631149699&from=exporthttp://dx.doi.org/10.1136/bmjopen-2019-034974 |en
dc.identifier.risid2697en
item.grantfulltextnone-
item.openairetypeArticle-
item.fulltextNo Fulltext-
item.languageiso639-1en-
item.openairecristypehttp://purl.org/coar/resource_type/c_18cf-
item.cerifentitytypePublications-
Appears in Sites:Children's Health Queensland Publications
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