Adaptive platform trials for rare disease populations

 

Synopsis

BANDICOOT is an international adaptive platform trial (APT) designed to identify effective therapies that improve health outcomes for critically ill children receiving a haematopoetic stem cell transplant (HCT). Building

on extensive engagement with patients, their parents, and clinicians, we will launch 3 trial domains focused on exercise, nutritional supplementation, and the gut microbiome. Broad stakeholder partnerships will ensure translation of evidence into health service delivery through policy reform and education of healthcare workers. Trial infrastructure developed in this project will also accelerate future testing of HCT interventions whilst building Australian capacity to conduct APTs into other complex health issues.

 

Speakers

Dr Hannah Walker is a paediatric oncologist at The Royal Children’s Hospital, specialising in bone marrow transplant and cellular therapies. She is currently undertaking her PhD through the University of Melbourne and Murdoch Children’s Research Institute. She is also the clinical lead in Melbourne for the first autologous gene therapy trial in children in Australia for RAG1 deficiency, made possible through the ReNEW collaboration and led by Leiden University Medical Centre. She is pursuing a career as a clinician scientist with a vision to improve the survivorship of patients undergoing treatment for cancer and cellular therapies.

Dr Deborah Meyran is a clinician-scientist and paediatric oncologist at The Royal Children’s Hospital, specialising in cellular therapies. She holds postdoctoral research positions at the Murdoch Children’s Research Institute and the Peter MacCallum Cancer Centre. A leader in paediatric cancer immunotherapy, she established Australia’s first immune profiling platform for paediatric tumours with the national Zero Childhood Cancer Program. Dr Meyran leads collaborative research to develop T-cell-based immunotherapies, including CAR T cells, for childhood and AYA cancers. She has secured over $6 million in competitive funding and holds two prestigious fellowships supporting her work. Dr Meyran is committed to translating cutting-edge science into personalised, patient-centred cancer care.

Associate Professor Rachel Conyers is a paediatric oncologist and clinician-scientist specialising in childhood leukaemia, lymphoma, and bone marrow transplantation. She is Clinical Lead of Bone Marrow Transplantation at The Royal Children’s Hospital Melbourne and Group Leader of Cancer Therapies at the Murdoch Children’s Research Institute. Internationally recognised for her work in pharmacogenomics and rare toxicities, she co-leads national programs including the Australian Cardio-Oncology Registry, MARVEL-Pic, and BANDICOOT—the first adaptive platform trial in paediatric stem cell transplantation. She has secured over $25M in research funding, published 110+ peer-reviewed papers, and contributed to over 20 international guidelines. A/Prof Conyers also holds an MBA and formal leadership training in health care strategy and administration.

Kaitlyn Taylor is the Project Manager for BANDICOOT and brings nearly 10 years of experience in the planning and delivery of investigator-initiated clinical trials at the Murdoch Children’s Research Institute. She has a strong track record managing complex paediatric studies across neurodevelopment and rare diseases. Kaitlyn has co-authored multiple peer-reviewed publications, including clinical trials investigating the use of medicinal cannabis for behavioural and neurological conditions in children and adolescents. Her work spans feasibility and efficacy studies, ethics and governance guidance, contributing to safe and evidence-based implementation of emerging therapies in paediatrics.

 

Comments are closed.

Previous post