Journal article
Designing clinical trials for dystonia
Neurotherapeutics, Vol.11(1), pp.117-127
01/2014
DOI: 10.1007/s13311-013-0221-6
PMCID: PMC3899487
PMID: 24282121
Abstract
With advances in the understanding of the pathophysiology of dystonia, novel therapeutics are being developed. Such therapies will require clinical investigation ranging from exploratory studies to examine safety, tolerability, dosage selection, and preliminary efficacy to confirmatory studies to evaluate efficacy definitively. As dystonia is a rare and complex disorder with clinical and etiological heterogeneity, clinical trials will require careful consideration of the trial design, including enrollment criteria, concomitant medication use, and outcome measures. Given the complexities of designing and implementing efficient clinical trials, it is important for clinicians and statisticians to collaborate closely throughout the clinical development process and that each has a basic understanding of both the clinical and statistical issues that must be addressed. To facilitate designing appropriate clinical trials in this field, we review important general clinical trial and regulatory principles, and discuss the critical components of trials with an emphasis on considerations specific to dystonia. Additionally, we discuss designs used in early exploratory, late exploratory, and confirmatory phases, including adaptive designs.
Details
- Title: Subtitle
- Designing clinical trials for dystonia
- Creators
- Wendy R Galpern - National Institute of Neurological Disorders and Stroke, National Institutes of Health, 6001 Executive Blvd., Rm 2225, Bethesda, MD, 20892, USA, galpernw@ninds.nih.govChristopher S CoffeyAlberto AlbaneseKen CheungCynthia L ComellaDixie J EcklundStanley FahnJoseph JankovicKarl KieburtzAnthony E LangMichael P McDermottJeremy M ShefnerJan K TellerJohn L P ThompsonSharon D YeattsH A Jinnah
- Resource Type
- Journal article
- Publication Details
- Neurotherapeutics, Vol.11(1), pp.117-127
- Publisher
- United States
- DOI
- 10.1007/s13311-013-0221-6
- PMID
- 24282121
- PMCID
- PMC3899487
- ISSN
- 1933-7213
- eISSN
- 1878-7479
- Grant note
- U13 NS079084 / NINDS NIH HHS U54 NS65701 / NINDS NIH HHS U54 TR001456 / NCATS NIH HHS U13NS079084 / NINDS NIH HHS U54 NS065701 / NINDS NIH HHS
- Language
- English
- Date published
- 01/2014
- Academic Unit
- Biostatistics
- Record Identifier
- 9983997303602771
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