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From random care to randomized clinical trials of patients with invasive streptococcal infections—research prioritisation and areas of equipoise

Life-threatening invasive infections caused by Streptococcus pyogenes and Streptococcus dysgalactiae subsp. equisimilis (SDSE) are unpredictable, frequently fatal, and are increasing in incidence globally. In the absence of evidence from randomized controlled trials (RCTs), clinical management for these conditions varies. Understanding current management approaches and areas of clinical equipoise will inform planning of feasible high-impact RCTs.

“If you build it, they will come”: the convergence of funding, research and collaboration in paediatric brain cancer clinical trials

Each year, approximately 1000 children in Australia and New Zealand, aged 0–14 years, are diagnosed with cancer. Despite paediatric cancer accounting for less than 1% of all cancer cases, the impact on their families and communities is profound and disproportionate.

Walking the walk to include pregnant participants in non-obstetric clinical trials: Insights from the SNAP Trial

Despite several calls for greater inclusion of pregnant people in non-obstetric clinical trials, their systematic exclusion remains common practice. Excluding pregnant individuals from clinical trials may result in unintended consequences such as inadequate treatment of medical conditions in pregnancy, inappropriate dosing of medications, and investigational therapies being used off-label outside of the context of a clinical trial, risking adverse events in the absence of demonstrated efficacy.

Rett syndrome: Establishing a novel outcome measure for walking activity in an era of clinical trials for rare disorders

Rett syndrome is a pervasive neurological disorder with impaired gait as one criterion.

Clinical Associate Professor Mary Sharp

Honorary Research Associate

Longitudinal egg-specific regulatory T- and B-cell development: Insights from primary prevention clinical trials examining the timing of egg introduction

Egg allergy affects almost 1 in 10 Australian infants. Early egg introduction has been associated with a reduced risk in developing egg allergy; however, the immune mechanisms underlying this protection remain unclear.

International workshop: what is needed to ensure outcome measures for Rett syndrome are fit-for-purpose for clinical trials? June 7, 2023, Nashville, USA

The clinical, research and advocacy communities for Rett syndrome are striving to achieve clinical trial readiness, including having fit-for-purpose clinical outcome assessments. This study aimed to (1) describe psychometric properties of clinical outcome assessment for Rett syndrome and (2) identify what is needed to ensure that fit-for-purpose clinical outcome assessments are available for clinical trials.

The clinical utility of lung clearance index in early cystic fibrosis lung disease is not impacted by the number of multiple-breath washout trials

Clinical Associate Professor Deborah Lehmann

Honorary Emeritus Fellow

Development of the Consumer Involvement & Engagement Toolkit: a digital resource to build capacity for undertaking patient-centred clinical trials in Australia

This paper describes the novel approach to developing a toolkit to support meaningful consumer involvement in clinical trials in Australia to help guide others in considering the development of similar resources.The toolkit aims to support greater consumer involvement in shaping how clinical research is prioritised, designed and conducted. Type of program or service: A working group of researchers, research organisations and consumers was established to co-develop the Consumer Involvement and Engagement Toolkit (the 'Toolkit'), a digital resource to guide researchers and organisations regarding consumer involvement in clinical trials.