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Updated: Jun 8, 2025
A Protocol for Comprehensive Assessment of Bulbar Dysfunction in Amyotrophic Lateral Sclerosis ALS
Published on: February 21, 2011
Living systematic review and comprehensive network meta-analysis of ALS clinical trials: study protocol
Floris T Van Loon1, Georgios Seitidis2, Dimitris Mavridis3
1Department of Neurology, University Medical Center Utrecht, Utrecht, The Netherlands.
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. Additionally, synthesising evidence from these studies may help overcome the limitations of individual studies. Network meta-analysis may refine the assessment of efficacy in specific patient subgroups, evaluate intervention characteristics such as mode of administration or biological mechanisms of action, and rank order promising therapeutic areas of interest. Therefore, we aim to synthesise the available evidence from ALS clinical trials.
Methods And Analysis:
We will conduct a systematic review to identify all clinical trials that assessed disease-modifying pharmaceutical therapies, cell therapies, or supplements in patients with ALS. Outcomes of interest are clinical disease progression outcomes and survival. We will conduct this search in the period Q4 2024 in three databases: PubMed, Embase and ClinicalTrials.gov for studies from 1999 to 2023. Individual patient data and aggregate data will be collected and subsequentially synthesised in meta-analytical models. The final model will be presented as an open-source web application with biannual updates of the underlying data, thereby providing a 'living' overview of the ALS clinical trial landscape.
Ethics And Dissemination:
No ethics approvals are required. Findings will be presented at relevant conferences and submitted to peer-reviewed journals. Data will be stored anonymously in secure repositories.
Insights
This systematic review synthesizes evidence from amyotrophic lateral sclerosis (ALS) clinical trials to identify effective treatments. The findings will create a living overview of the ALS therapeutic landscape for future research.
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.
