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

Area of Science:

• Neuroscience
• Clinical Trials
• Evidence Synthesis

Background:

• Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disease with no current effective treatments.
• Numerous clinical trials have yielded limited success in altering disease progression.
• Synthesizing existing trial data can overcome individual study limitations and identify patient subgroups.

Purpose of the Study:

• To conduct a systematic review and network meta-analysis of clinical trials for amyotrophic lateral sclerosis (ALS).
• To refine the assessment of therapeutic efficacy in specific patient subgroups and evaluate intervention characteristics.
• To rank promising therapeutic areas and generate hypotheses for future ALS research.

Main Methods:

• Systematic review of pharmaceutical therapies, cell therapies, and supplements for ALS.
• Data collection from PubMed, Embase, and ClinicalTrials.gov (1999-2023).
• Meta-analysis of individual and aggregate patient data, presented in an open-source web application.

Main Results:

• The study aims to synthesize available evidence from ALS clinical trials.
• Network meta-analysis will assess efficacy in patient subgroups and rank interventions.
• A living, open-source web application will provide biannual updates on ALS trial data.

Conclusions:

• This synthesis will provide a comprehensive overview of the ALS clinical trial landscape.
• Findings will inform future research by identifying promising therapeutic strategies and patient subgroups.
• The living web application will serve as a dynamic resource for the ALS research community.