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Erschienen in: Neurotherapeutics 2/2015

01.04.2015 | Review

Clinical Trial Designs in Amyotrophic Lateral Sclerosis: Does One Design Fit All?

verfasst von: Katharine A. Nicholson, Merit E. Cudkowicz, James D. Berry

Erschienen in: Neurotherapeutics | Ausgabe 2/2015

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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.
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Metadaten
Titel
Clinical Trial Designs in Amyotrophic Lateral Sclerosis: Does One Design Fit All?
verfasst von
Katharine A. Nicholson
Merit E. Cudkowicz
James D. Berry
Publikationsdatum
01.04.2015
Verlag
Springer US
Erschienen in
Neurotherapeutics / Ausgabe 2/2015
Print ISSN: 1933-7213
Elektronische ISSN: 1878-7479
DOI
https://doi.org/10.1007/s13311-015-0341-2

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