Rethinking phase 2 trials in amyotrophic lateral sclerosis.

There is a long history in amyotrophic lateral sclerosis (ALS) of promoting therapies based on Phase 2 data, which then fail in Phase 3 trials. Experience suggests that studies of 6 months in duration are too short, especially with function-based outcome measures. Multiplicity poses a serious threat to data interpretation, and strategies to impute missing data may not be appropriate for ALS where progression is always expected.

Attrition and discontinuation in amyotrophic lateral sclerosis clinical trials: a meta-analysis.

Objectives: Attrition due to adverse events and disease progression impacts the integrity and generalizability of clinical trials. The aim of this study is to provide evidence-based estimates of attrition for clinical trials in amyotrophic lateral sclerosis (ALS), and identify study-related predictors, through a comprehensive systematic review and meta-analysis.

Methods: We systematically reviewed the literature to identify all randomized, placebo-controlled clinical trials in ALS and determined the number of patients who discontinued the study per randomized arm.

Feasibility of a new transmural care pathway for advance care planning for older persons: A qualitative study into community care registered nurses' perspectives.

Background: Transmural palliative care interventions aim to identify older persons with palliative care needs and timely provide advance care planning, symptom management, and coordination of care. Nurses can have an important role in these interventions; however, their expertise is currently underused. A new transmural care pathway with a central role for the community care registered nurse in advance care planning aims to contribute to the quality of palliative care for older persons.

Living systematic review and comprehensive network meta-analysis of ALS clinical trials: study protocol.

Introduction: Amyotrophic lateral sclerosis (ALS) is a fatal neurogenerative disease with no effective treatment to date. Despite numerous clinical trials, the majority of studies have been futile in their effort to significantly alter the course of the disease. However, these studies may still provide valuable information for identifying patient subgroups and generating new hypotheses for future research.

Amyotrophic lateral sclerosis established as a multistep process across phenotypes.

Background And Purpose: Given the accepted multistep process of disease causation in amyotrophic lateral sclerosis (ALS), the present study was undertaken to determine the number of steps required for disease onset across each of the ALS phenotypes.

Methods: Clinical and demographic data were prospectively accumulated using the Australian Motor Neurone Disease Registry (2005-2016), and age-specific incidence rates were calculated. Poisson regression was utilized to assess the relationship between log age-specific incidence and log age of onset, with McFadden's R used to assess the goodness of fit of the model.