Amyotrophic lateral sclerosis is a progressive and devastating neurodegenerative disease.
Despite decades of clinical trials, effective disease-modifying drugs remain scarce. To …

Abstract Background 20–60% of patients with initially locally advanced Renal Cell
Carcinoma (RCC) develop metastatic disease despite optimal surgical excision. Adjuvant …

Introduction Motor neuron disease (MND) is a rapidly fatal neurodegenerative disease.
Despite decades of research and clinical trials there remains no cure and only one globally …

Background For medical conditions with numerous interventions worthy of investigation,
there are many advantages of a multi-arm multi-stage (MAMS) platform trial approach …

Background Platform trials improve the efficiency of the drug development process through
flexible features such as adding and dropping arms as evidence emerges. The benefits and …

Platform trials evaluate multiple experimental treatments under a single master protocol,
where new treatment arms are added to the trial over time. Given the multiple treatment …

Global health pandemics, such as coronavirus disease 2019 (COVID-19), require efficient
and well-conducted trials to determine effective interventions, such as treatments and …

For the past few years, platform trials have experienced a significant increase, recently
amplified by the COVID-19 pandemic. The implementation of a platform trial is particularly …

Background Factorial designs and multi-arm multi-stage (MAMS) platform designs have
many advantages, but the practical advantages and disadvantages of combining the two …

Background: Multi-arm multi-stage trials are an efficient, adaptive approach for testing many
treatments simultaneously within one protocol. In settings where numbers of patients …