Response adaptive randomisation in clinical trials: Current practice, gaps and future directions.

Adaptive designs (ADs) offer clinical trials flexibility to modify design aspects based on accumulating interim data. Response adaptive randomisation (RAR) adjusts treatment allocation according to interim results, favouring promising treatments. Despite scientific appeal, RAR adoption lags behind other ADs.

The Childhood Opportunity Index 2.0: Factor Structure in 9-10 Year Olds in the Adolescent Brain Cognitive Development Study.

The built physical and social environments are critical drivers of child neural and cognitive development. This study aimed to identify the factor structure and correlates of 29 environmental, education, and socioeconomic indicators of neighborhood resources as measured by the Child Opportunity Index 2.0 (COI 2.

Uncovering the state of knowledge about healthcare gentrification: a scoping review protocol.

Introduction: Healthcare gentrification is the process in which the distribution of healthcare resources within a neighbourhood affects residents' access to healthcare services. To understand the complexity of healthcare access and to consider the socio-structural dimensions affecting equity in access to care, we aim to explore how healthcare gentrification has been described in the scientific literature and to document the reported relations between gentrification and healthcare access.

Methods And Analysis: We will conduct a scoping review from data published from inception to September 2024 based on the methodology developed by Arksey and O'Malley (2005) and improved by Levac (2010).

A Tutorial on Value-Based Adaptive Designs: Could a Value-Based Sequential 2-Arm Design Have Created More Health Economic Value for the Big CACTUS Trial?

Objectives: Value-based trials aim to maximize the expected net benefit by balancing technology adoption decisions and clinical trial costs. Adaptive trials offer additional efficiency. This article provides guidance on determining whether a value-based sequential design is the best option for an adaptive 2-arm trial, illustrated through a case study.

Transparency and reproducibility in the Adolescent Brain Cognitive Development (ABCD) study.

Background: Transparency can build trust in the scientific process, but scientific findings can be undermined by poor and obscure data use and reporting practices. The purpose of this work is to report how data from the Adolescent Brain Cognitive Development (ABCD) Study has been used to date, and to provide practical recommendations on how to improve the transparency and reproducibility of findings.

Methods: Articles published from 2017 to 2023 that used ABCD Study data were reviewed using more than 30 data extraction items to gather information on data use practices.

Transparency and Reproducibility in the Adolescent Brain Cognitive Development (ABCD) Study.

Background: Transparency can build trust in the scientific process, but scientific findings can be undermined by poor and obscure data use and reporting practices. The purpose of this work is to report how data from the Adolescent Brain Cognitive Development (ABCD) Study has been used to date, and to provide practical recommendations on how to improve the transparency and reproducibility of findings.

Methods: Articles published from 2017 to 2023 that used ABCD Study data were reviewed using more than 30 data extraction items to gather information on data use practices.

Assessment of Safety and Biodistribution of AAVrh.10hCLN2 Following Intracisternal Administration in Nonhuman Primates for the Treatment of CLN2 Batten Disease.

CLN2 disease is a fatal, childhood autosomal recessive disorder caused by mutations in ceroid lipofuscinosis type 2 (CLN2) gene, encoding tripeptidyl peptidase 1 (TPP-1). Loss of TPP-1 activity leads to accumulation of storage material in lysosomes and resultant neuronal cell death with neurodegeneration. Genotype/phenotype comparisons suggest that the phenotype should be ameliorated with increase of TPP-1 levels to 5-10% of normal with wide central nervous system (CNS) distribution.

COVID-19 Risk Factors for Cancer Patients: A First Report with Comparator Data from COVID-19 Negative Cancer Patients.

Very few studies investigating COVID-19 in cancer patients have included cancer patients as controls. We aimed to identify factors associated with the risk of testing positive for SARS CoV2 infection in a cohort of cancer patients. We analyzed data from all cancer patients swabbed for COVID-19 between 1 March and 31 July 2020 at Guy's Cancer Centre.