Module232025

5/7/2025

• Paediatric trials require specific expertise and facilities, trained investigators, close patients engagement • PK studies: volume of blood withdrawn should be minimised • Certain investigations present difficulties: e.g., MRI in young children requires sedation/anaesthesia • Long travel/tiredness at time of examinations may impact assessment • Consideration of impact of interventions and hospital stays/visits on child psychology, QOL, school absence, time with friends • Global multi-centered CTs essential for rare paed diseases • Utilize principles of decentralize CTs including wearable devices, safe delivery of IPs at home, video/digital/local facility investigations • Reimbursement opportunities for families

Practical considerations : predictors of success

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• First in human study: healthy volunteers = adult patients  unless paediatric-specific disease and development -> phase I/II  Strict non-clinical data requirements

Phase 1/2

• Phase 2 : Single arm study vs multi-dose arms including comparator  Could be different in older (use of adult dose) or very young (not many patients) age cohorts  Sample size likely to be small, should be determined by study objectives

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