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Showing results for "clinical trials"

Research

The evolution of clinical trials for infant acute lymphoblastic leukemia

Despite initial improvements in survival of infants with ALL since establishment of the first pediatric cooperative group ALL trials, the poor outcome has...

Research

Outcome measures for artificial pancreas clinical trials: A consensus report

Research on and commercial development of the artificial pancreas (AP) continue to progress rapidly, and the AP promises to become a part of clinical care.

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Choosing primary endpoints for clinical trials of health care interventions

This narrative review describes the evolution, range and relative strengths and weaknesses of endpoints used in late phase trials

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Neonatal sepsis: a systematic review of core outcomes from randomised clinical trials

The lack of a consensus definition of neonatal sepsis and a core outcome set proves a substantial impediment to research that influences policy and practice relevant to key stakeholders, patients and parents.

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Exciting new clinical trials in cystic fibrosis: Infants need not apply

The 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 trials

A 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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Improving Outcome Reporting in Paediatric Airway Management in Clinical Trials (IMPACT): A Study Protocol for Core Outcomes and Clinical Endpoints

Although clinical trials are fundamental to advancing evidence-based practice, significant heterogeneity in outcome reporting poses a considerable challenge to the validity of systematic reviews. This inconsistency impedes the ability to compare, synthesise and interpret research findings effectively. In the field of paediatric airway management, this issue is particularly relevant because of the low incidence of critical events and the related high morbidity and mortality. The issue of inadequate and variable outcome reporting in clinical trials has been widely acknowledged, necessitating initiatives to enhance the quality of future research.

Research

Clinical Trials in the Brain Tumour Population: Challenges and Strategies for the Future

This review identifies challenges and barriers to successful development of drugs in neuro-oncology trials at the preclinical, clinical and translational stages that we believe has contributed to poor outcomes for patients over the last 30 years.

Research

IDH-mutant gliomas in children and adolescents - from biology to clinical trials

Gliomas account for nearly 30% of all primary central nervous system (CNS) tumors in children and adolescents and young adults (AYA), contributing to significant morbidity and mortality. The updated molecular classification of gliomas defines molecularly diverse subtypes with a spectrum of tumors associated with age-distinct incidence.