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Showing results for "clinical trials"
Research
Patient-advocate-led global coalition adapting fit-for-purpose outcomes measures to assure meaningful inclusion of DEEs in clinical trialsExisting clinical tools that measure non-seizure outcomes lack the range and granularity needed to capture skills in developmental and epileptic encephalopathy (DEE)-affected individuals who also fall in the severe to profound range of intellectual disability. This effectively excludes those with severe impairments from clinical trials, impeding the ability of sponsors to evaluate disease-modifying therapies.
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Infectious diseases clinician's variation in the management of pediatric Staphylococcus aureus bacteraemia and equipoise for clinical trialsLarge variation in antibiotic prescribing amongst clinicians is demonstrated and increased, corresponding with escalating case complexity and persisting MRSA bacteraemia
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A snapshot of consumer engagement in clinical trials in Australia: results of a national survey of clinical trial networks and research organisationsLittle is known about the extent, perceptions or experiences of consumers involved in clinical trials across Australia. The purpose of this National study was to better understand the activity and perceptions of clinical trial networks (CTNs), research co-ordinating centres and their consumers, around consumer involvement in clinical trials.
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Exciting new clinical trials in cystic fibrosis: Infants need not applyThe recent announcement of the negative results of the TIGER- 2 phase 3 study of denufosol tetrasodium
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Novel method to select meaningful outcomes for evaluation in clinical trialsA standardised framework for selecting outcomes for evaluation in trials has been proposed by the Core Outcome Measures in Effectiveness Trials working group. However, this method does not specify how to ensure that the outcomes that are selected are causally related to the disease and the health intervention being studied. Causal network diagrams may help researchers identify outcomes that are both clinically meaningful and likely to be causally dependent on the intervention, and endpoints that are, in turn, causally dependent on those outcomes.
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Longitudinal egg-specific regulatory T- and B-cell development: Insights from primary prevention clinical trials examining the timing of egg introductionEgg 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.
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Safety and Immunogenicity of MF59-Adjuvanted Cell Culture-Derived A/H5N1 Subunit Influenza Virus Vaccine: Dose-Finding Clinical Trials in Adults and the ElderlyIn adult and elderly participants, the full-dose aH5N1c vaccine formulation was well tolerated and met US and European licensure criteria for pandemic vaccines
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Walking the walk to include pregnant participants in non-obstetric clinical trials: Insights from the SNAP TrialDespite 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.
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“If you build it, they will come”: the convergence of funding, research and collaboration in paediatric brain cancer clinical trialsEach 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.
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International workshop: what is needed to ensure outcome measures for Rett syndrome are fit-for-purpose for clinical trials? June 7, 2023, Nashville, USAThe 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.