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Single group multisite safety trial of sibling cord blood cell infusion to children with cerebral palsy: study
Kylie Crompton1,2,3, Iona Novak4, Michael Fahey5,6
1Neurodisability and Rehabilitation, Murdoch Children's Research Institute, Parkville, Victoria, Australia kylie.crompton@mcri.edu.au.
Insights
This study assesses the safety of sibling cord blood stem cell infusions for children with cerebral palsy (CP). It monitors adverse events and preliminary outcomes to guide future CP stem cell therapies.
Area of Science:
- Pediatric Neurology
- Regenerative Medicine
- Clinical Trials
Background:
- Cerebral palsy (CP) is a common childhood physical disability with no current cure.
- Stem cell therapy shows potential for improving brain injury and treating CP.
- Significant questions remain regarding optimal cell type, dosage, timing, and risks for CP stem cell therapy.
Purpose of the Study:
- To evaluate the safety and feasibility of single-dose intravenous infusion of human leucocyte antigen-matched sibling cord blood cells in children with CP.
- To explore preliminary efficacy signals in motor function, cognition, and quality of life.
- To establish a foundation for larger, randomized controlled trials in CP stem cell therapy.
Main Methods:
- A single-group study involving 12 children aged 1-16 years with CP, without immune suppression.
- Participants received a single intravenous infusion of sibling cord blood cells.
- Safety was assessed by monitoring adverse events and donor cell persistence. Efficacy was preliminarily evaluated through motor, cognitive, and quality of life assessments at baseline, 3, and 12 months.
Main Results:
- The study is designed to gather safety data and preliminary efficacy signals.
- Detailed results on adverse events, donor cell detection, and functional outcomes will be reported upon study completion.
- The findings will inform the design of future clinical trials for stem cell therapy in CP.
Conclusions:
- Human safety and efficacy trials are crucial for advancing stem cell therapy for cerebral palsy.
- This study aims to provide essential safety and preliminary efficacy data.
- The results will guide the development of evidence-based stem cell treatments for CP.
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 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.
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