ReviewNeurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics2015
Clinical Trial Designs in Amyotrophic Lateral Sclerosis: Does One Design Fit All?
Review in Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics, 2015. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 16 papers.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
16 citing papers in PubMed, 36 citations in OpenAlex.
- Rasagiline for amyotrophic lateral sclerosis: A randomized, controlled trial.Muscle & nerve · 2019Trial
- Sample enrichment for clinical trials to show delay of onset in huntington disease.Movement disorders : official journal of the Movement Disorder Society · 2019Trial
- Pridopidine Mediated Sigma-1 Receptor Activation and Therapeutic Implications in Neurodegenerative Diseases.Neurology international · 2026Review
- Next-Generation Phospholipid Nanocomplexes for Precision Neurotherapeutics: Harnessing Endogenous Blood-Brain Barrier Transport Mechanisms to Revolutionize the Treatment of Neurodegenerative Diseases.Recent advances in drug delivery and formulation · 2026Review
- A framework for translating tauopathy therapeutics: Drug discovery to clinical trials.Alzheimer's & dementia : the journal of the Alzheimer's Association · 2024Review
- At-home wearables and machine learning sensitively capture disease progression in amyotrophic lateral sclerosis.Nature communications · 2023Article
- A machine-learning based objective measure for ALS disease severity.NPJ digital medicine · 2022Article
- Improving clinical trial outcomes in amyotrophic lateral sclerosis.Nature reviews. Neurology · 2021Review
- Addressing heterogeneity in amyotrophic lateral sclerosis CLINICAL TRIALS.Muscle & nerve · 2020Review
- Tracking a Fast-Moving Disease: Longitudinal Markers, Monitoring, and Clinical Trial Endpoints in ALS.Frontiers in neurology · 2019Review
- Additional evidence for a therapeutic effect of dextromethorphan/quinidine on bulbar motor function in patients with amyotrophic lateral sclerosis: A quantitative speech analysis.British journal of clinical pharmacology · 2018Article
- Animal models of neurodegenerative diseases.Nature neuroscience · 2018Review
- Survival prediction in Amyotrophic lateral sclerosis based on MRI measures and clinical characteristics.BMC neurology · 2017Article
- Shortcomings in the Current Amyotrophic Lateral Sclerosis Trials and Potential Solutions for Improvement.Frontiers in neurology · 2017Review
- Fluid-Based Biomarkers for Amyotrophic Lateral Sclerosis.Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics · 2017Review
- The Spectrum of Motor Neuron Diseases: From Childhood Spinal Muscular Atrophy to Adult Amyotrophic Lateral Sclerosis.Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics · 2015Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
3 authors at 2 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
The last 2 decades have seen a surge in the number of amyotrophic lateral sclerosis (ALS) clinical trials with the hope of finding successful treatments. Clinical trialists aim to repurpose existing drugs and test novel compounds to target potential ALS disease pathophysiology. Recent technological advancements have led to the discovery of new causative genetic agents and modes of delivering potential therapy, calling for increasingly sophisticated trial design. The standard ALS clinical trial design may be modified depending on study needs: type of therapy; route of therapy delivery; phase of therapy development; applicable subpopulation; market availability of therapy; and utility of telemedicine. Novel biomarkers of diagnostic, predictive, prognostic, and pharmacodynamic value are undergoing development and validation for use in clinical trials. Design modifications build on the traditional clinical trial design and may be employed in either the learning or confirming trial phase. Novel designs aim to minimize patient risk, study duration, and sample size, while improving efficiency and promoting statistical power to herald an exciting era for clinical research in ALS.
Indexed as
Identifiers
What OpenQuestion holds
Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.