publication . Article . 2015

Effect of sample size re-estimation in adaptive clinical trials for Alzheimer's disease and mild cognitive impairment

Wang, Guoqiao; Kennedy, Richard E.; Cutter, Gary R.; Schneider, Lon S.;
Open Access English
  • Published: 01 Jun 2015 Journal: Alzheimer's & Dementia : Translational Research & Clinical Interventions, volume 1, issue 1, pages 63-71 (eissn: 2352-8737, Copyright policy)
  • Publisher: Elsevier
Abstract
Abstract Introduction The sample size re-estimation (SSR) adaptive design allows interim analyses and resultant modifications of the ongoing trial to preserve or increase power. We investigated the applicability of SSR in Alzheimer's disease (AD) trials using a meta-database of clinical studies. Methods Based on six studies, we simulated clinical trials using Alzheimer's Disease Assessment Scale-cognitive subscale (ADAS-Cog) as primary outcome. A single SSR based on effect sizes or based on variances was conducted at 6 months and 12 months. Resultant power improvement and sample size adjustments were evaluated. Results SSR resulted in highly variable outcomes fo...
Subjects
Medical Subject Headings: food and beverages
free text keywords: Featured Article, Alzheimer's disease, Alzheimer's Disease Assessment Scale, Mild cognitive impairment, Sample size re-estimation, Adaptive design
Funded by
NIH| MOBILITY AMONG OLDER AFRICAN AMERICANS AND WHITES
Project
  • Funder: National Institutes of Health (NIH)
  • Project Code: 1R01AG015062-01A1
  • Funding stream: NATIONAL INSTITUTE ON AGING
,
NIH| Alzheimers Disease Neuroimaging Initiative
Project
  • Funder: National Institutes of Health (NIH)
  • Project Code: 1U01AG024904-01
  • Funding stream: NATIONAL INSTITUTE ON AGING
,
NIH| UAB Diabetes Research and Training Center
Project
  • Funder: National Institutes of Health (NIH)
  • Project Code: 5P60DK079626-04
  • Funding stream: NATIONAL INSTITUTE OF DIABETES AND DIGESTIVE AND KIDNEY DISEASES
,
NIH| Etiology of Geographic and Racial Differences in Stroke
Project
  • Funder: National Institutes of Health (NIH)
  • Project Code: 3U01NS041588-10S1
  • Funding stream: NATIONAL INSTITUTE OF NEUROLOGICAL DISORDERS AND STROKE
,
NIH| Longer-term Alzheimer Disease Studies to Improve Clinical Trials Methods Outcomes
Project
  • Funder: National Institutes of Health (NIH)
  • Project Code: 5R01AG037561-02
  • Funding stream: NATIONAL INSTITUTE ON AGING
24 references, page 1 of 2

Thies, W., Bleiler, L.. 2013 Alzheimer's disease facts and figures. Alzheimers Dement. 2013; 9: 208-245 [OpenAIRE] [PubMed]

Aisen, P., Andrieu, S., Sampaio, C., Carrillo, M., Khachaturian, Z., Dubois, B.. Report of the task force on designing clinical trials in early (predementia) AD. Neurology. 2011; 76: 280-286 [OpenAIRE] [PubMed]

Cummings, J., Gould, H., Zhong, K.. Advances in designs for Alzheimer’s disease clinical trials. Am J Neurodegener Dis. 2012; 1: 205 [OpenAIRE] [PubMed]

Chow, S.C., Chang, M.. Adaptive design methods in clinical trials—a review. Orphanet J Rare Dis. 2008; 3: 11 [OpenAIRE] [PubMed]

Kennedy, R.E., Cutter, G.R., Schneider, L.S.. Effect of APOE genotype status on targeted clinical trials outcomes and efficiency in dementia and mild cognitive impairment resulting from Alzheimer's disease. Alzheimers Dement. 2014; 10: 349-359 [OpenAIRE] [PubMed]

Wechsler, D.. 1987

Burton, A., Altman, D.G., Royston, P., Holder, R.L.. The design of simulation studies in medical statistics. Stat Med. 2006; 25: 4279-4292 [PubMed]

Schneider, L.S., Kennedy, R.E., Cutter, G.R.. Requiring an amyloid-β1-42 biomarker for prodromal Alzheimer's disease or mild cognitive impairment does not lead to more efficient clinical trials. Alzheimers Dement. 2010; 6: 367-377 [OpenAIRE] [PubMed]

Petersen, R.C., Thomas, R.G., Grundman, M., Bennett, D., Doody, R., Ferris, S.. Vitamin E and donepezil for the treatment of mild cognitive impairment. N Engl J Med. 2005; 352: 2379-2388 [OpenAIRE] [PubMed]

Doody, R., Ferris, S., Salloway, S., Sun, Y., Goldman, R., Watkins, W.. Donepezil treatment of patients with MCI A 48-week randomized, placebo-controlled trial. Neurology. 2009; 72: 1555-1561 [OpenAIRE] [PubMed]

Chang, M.. 2007

Lawrence Gould, A., Shih, W.J.. Sample size re-estimation without unblinding for normally distributed outcomes with unknown variance. Commun Stat Theory Methods. 1992; 21: 2833-2853

Dunn, O.J.. Multiple comparisons among means. J Am Stat Assoc. 1961; 56: 52-64

Schneider, L.S., Sano, M.. Current Alzheimer's disease clinical trials: methods and placebo outcomes. Alzheimers Dement. 2009; 5: 388-397 [OpenAIRE] [PubMed]

Wittes, J., Brittain, E.. The role of internal pilot studies in increasing the efficiency of clinical trials. Stat Med. 1990; 9: 65-72 [OpenAIRE] [PubMed]

24 references, page 1 of 2
Powered by OpenAIRE Research Graph
Any information missing or wrong?Report an Issue