Abstract：
:Recruiting sufficient patients within an acceptable time horizon is an issue for most clinical trials and is especially challenging in the field of rare diseases. It is therefore an attractive option to include historical data from previous (pilot) trials in the current study thus reducing the recruitment burden. In clinical trials with binary endpoint, the required sample size does not only depend on the type I error rate, the power, and the treatment group difference but additionally on the overall event rate. However, there is usually some uncertainty in the planning phase about the value of this nuisance parameter. We present methods for blinded sample size recalculation in the setting of two-arm superiority trials with historical control data where the overall rate is estimated mid-course and the sample size is recalculated accordingly. The operating characteristics of the method are investigated in terms of actual type I error rate, power, and expected sample size. Application is illustrated with a clinical trial example in patients with systemic sclerosis, a rare connective tissue disorder.
journal_name
Contemp Clin Trialsjournal_title
Contemporary clinical trialsauthors
Hees K,Kieser Mdoi
10.1016/j.cct.2017.07.013subject
Has Abstractpub_date
2017-12-01 00:00:00pages
2-7eissn
1551-7144issn
1559-2030pii
S1551-7144(17)30041-1journal_volume
63pub_type
杂志文章abstract：:This randomized, controlled trial tested the effectiveness of a nurse-run, telephone-based intervention to improve lipid control in patients with diabetes. Our patient population is predominantly low-income and Latino. Using our diabetes registry, we randomly assigned 381 patients to continue with their usual care and...
journal_title：Contemporary clinical trials
pub_type： 杂志文章
doi：10.1016/j.cct.2008.05.011
更新日期：2008-09-01 00:00:00
abstract：BACKGROUND:Risk factors for cardiovascular disease (CVD) not only increase the risk for clinical CVD events, but also are associated with a cascade of neurophysiologic and neuroanatomic changes that increase the risk of cognitive impairment and dementia. Although epidemiological studies have shown that exercise and die...
journal_title：Contemporary clinical trials
pub_type： 杂志文章
doi：10.1016/j.cct.2012.09.004
更新日期：2013-01-01 00:00:00
abstract：:The usual development cycles are too slow for the development of vaccines, diagnostics and treatments in pandemics such as the ongoing SARS-CoV-2 pandemic. Given the pressure in such a situation, there is a risk that findings of early clinical trials are overinterpreted despite their limitations in terms of size and d...
journal_title：Contemporary clinical trials
pub_type： 杂志文章
doi：10.1016/j.cct.2020.106213
更新日期：2020-12-01 00:00:00
abstract：:The combined use of cigarettes and alcohol is associated with an increased risk of morbidity and mortality. Yet, efficacious interventions that address both behaviors concurrently are lacking. Smoking cessation and alcohol modification not only garner health benefits, but there is also value in addressing alcohol use ...
journal_title：Contemporary clinical trials
pub_type： 杂志文章
doi：10.1016/j.cct.2020.106218
更新日期：2020-11-14 00:00:00
abstract：OBJECTIVE:To retrospectively analyze the effects of long-acting gonadotropin-releasing hormone agonist (GnRH-a) combined with transvaginal ultrasound-guided cyst aspiration on the pregnancy outcomes of in vitro fertilization-embryo transfer (IVF-ET) in the infertile patients with ovarian endometriosis. METHODS:The 134...
journal_title：Contemporary clinical trials
pub_type： 临床试验,杂志文章
doi：10.1016/j.cct.2012.07.009
更新日期：2012-11-01 00:00:00
abstract：INTRODUCTION:Cognitive impairment (CI) and cardiovascular disease (CVD) disproportionately affect women compared to men, and CVD increases risk of CI. Physical activity and cognitive training can improve cognition in older adults and may have additive or synergistic effects. However, no combined intervention has target...
journal_title：Contemporary clinical trials
pub_type： 杂志文章
doi：10.1016/j.cct.2020.106254
更新日期：2020-12-29 00:00:00
abstract：BACKGROUND:Atorvastatin and metformin are known energy restricting mimetic agents that act synergistically to produce molecular and metabolic changes in advanced prostate cancer (PCa). This trial seeks to determine whether these drugs favourably alter selected parameters in men with clinically-localized, aggressive PCa...
journal_title：Contemporary clinical trials
pub_type： 杂志文章,随机对照试验
doi：10.1016/j.cct.2016.06.014
更新日期：2016-09-01 00:00:00
abstract：:The fraction of patients who respond to treatment and the duration of response in the subset of responding patients are commonly evaluated in oncology trials of cytotoxic compounds. While formal, comparative analysis of the fraction of patients responding to treatment is straightforward in a randomised trial, analyses...
journal_title：Contemporary clinical trials
pub_type： 杂志文章
doi：10.1016/j.cct.2007.10.008
更新日期：2008-07-01 00:00:00
abstract：OBJECTIVES:To review current publications to examine safety of tai chi (TC). DESIGN:Cochrane Library, EBSCO host and MEDLINE/PubMed were searched for randomized controlled trials (RCTs) including TC as the core intervention and reporting adverse events (AEs). Data were extracted considering active vs. inactive control...
journal_title：Contemporary clinical trials
pub_type： 杂志文章,meta分析
doi：10.1016/j.cct.2019.06.004
更新日期：2019-07-01 00:00:00
abstract：:Missing data is common in clinical trials. Depending on the volume and nature of missing data, it may reduce statistical power for detecting treatment difference, introduce potential bias and invalidate conclusions. Non-responder imputation (NRI), where patients with missing information to determine endpoint status ar...
journal_title：Contemporary clinical trials
pub_type： 杂志文章
doi：10.1016/j.cct.2020.106226
更新日期：2020-11-22 00:00:00
abstract：:There is increasing evidence that exposure of developing brains in animals, including nonhuman primates, to commonly-utilized anesthetic agents may cause adverse effects on cognition and behavior. In this paper, we summarize our methodology for a population-based, propensity-matched study to evaluate possible anesthes...
journal_title：Contemporary clinical trials
pub_type： 杂志文章
doi：10.1016/j.cct.2014.12.020
更新日期：2015-03-01 00:00:00
abstract：BACKGROUND:Postmenopausal Latinas are a growing population group in the US who are at high risk for cardiovascular disease. Epidemiological studies have shown that excessive sitting is related to cardiovascular disease risk. Older women sit for prolonged periods and most individuals do not meet physical activity guidel...
journal_title：Contemporary clinical trials
pub_type： 杂志文章,随机对照试验
doi：10.1016/j.cct.2019.02.004
更新日期：2019-04-01 00:00:00
abstract：BACKGROUND:Formation of diet and physical activity habits begins during early childhood. However, many preschool-aged children in the United States do not achieve recommendations for a nutritious diet or active lifestyle. Two important spheres of influence, home and child care, could ensure that children receive consis...
journal_title：Contemporary clinical trials
pub_type： 杂志文章,随机对照试验
doi：10.1016/j.cct.2017.11.007
更新日期：2018-01-01 00:00:00
abstract：BACKGROUND:In the past 20-30 years, the prevalence of atopic diseases, particularly among children in the Western world, has increased. It has been suggested that Western lifestyle may have reduced the overall exposure to microbial stimulation early in life. The role of the gut flora in this connection has been studied...
journal_title：Contemporary clinical trials
pub_type： 杂志文章
doi：10.1016/j.cct.2006.02.009
更新日期：2006-08-01 00:00:00
abstract：:Assessing the probability that a Phase III clinical trial will demonstrate clinically relevant efficacy based on Phase II data is an important topic in clinical drug development. An accurate estimate of how likely a Phase III trial will succeed based on available data will inform the decision on whether to move an exp...
journal_title：Contemporary clinical trials
pub_type： 杂志文章
doi：10.1016/j.cct.2010.08.008
更新日期：2010-11-01 00:00:00
abstract：BACKGROUND:In a recent publication, the International Guidelines for Groin Hernia Management by the European Hernia Society (EHS) recognized the need to individualize and tailor the surgical approach for hernia repair. There may be different opportunities for optimization of the surgical technique for surgeons performi...
journal_title：Contemporary clinical trials
pub_type： 杂志文章,多中心研究
doi：10.1016/j.cct.2019.105853
更新日期：2019-11-01 00:00:00
abstract：:Although the Diabetes Prevention Program (DPP) developed a lifestyle weight loss intervention that has been demonstrated to prevent type 2 diabetes in high-risk individuals, it has yet to be widely adopted at the community level. The Healthy Living Partnership to Prevent Diabetes study (HELP PD) was designed to transl...
journal_title：Contemporary clinical trials
pub_type： 杂志文章,随机对照试验
doi：10.1016/j.cct.2009.09.002
更新日期：2010-01-01 00:00:00
abstract：BACKGROUND:Thousands of people die annually from prescription opioid overdoses; however there are few strategies to ensure patients receive medication risk information at the time of prescribing. OBJECTIVES:To compare the effectiveness of the Emergency Department (ED) Electronic Medication Complete Communication (EMC2...
journal_title：Contemporary clinical trials
pub_type： 杂志文章,随机对照试验
doi：10.1016/j.cct.2017.05.003
更新日期：2017-08-01 00:00:00
abstract：:Suicide remains the 10th leading cause of death among adults in the United States (U.S.). Annually, approximately 30 per 100,000 U.S. military Veterans commit suicide, compared to 14 per 100,000 U.S. civilians. Symptoms associated with suicidality can be treatment resistant and proven-effective pharmaceuticals may hav...
journal_title：Contemporary clinical trials
pub_type： 杂志文章,随机对照试验
doi：10.1016/j.cct.2016.02.002
更新日期：2016-03-01 00:00:00
abstract：:The ability to recruit qualified subjects who are willing to adhere to the study protocol in clinical trials is an essential component of translational research. Such tasks can be particularly challenging for chemoprevention studies when the targeted study population is healthy, at risk individuals who do not have sig...
journal_title：Contemporary clinical trials
pub_type： 杂志文章,随机对照试验
doi：10.1016/j.cct.2009.05.004
更新日期：2009-09-01 00:00:00
abstract：INTRODUCTION:When the Centers for Medicare and Medicaid Services announced coverage for low dose CT lung cancer screening, they also mandated that imaging centers offer smoking cessation services. We designed the Optimizing Lung Screening (OaSiS) trial to evaluate strategies to implement the Public Health Service Guide...
journal_title：Contemporary clinical trials
pub_type： 杂志文章
doi：10.1016/j.cct.2020.105963
更新日期：2020-04-01 00:00:00
abstract：PURPOSE:Black breast cancer survivors have high rates of obesity and low physical activity levels. Little is known about the acceptability and feasibility of interventions in this population. OBJECTIVE:A two-arm RCT was launched to assess the efficacy of a culturally targeted 12-week multimodal lifestyle intervention ...
journal_title：Contemporary clinical trials
pub_type： 杂志文章,随机对照试验
doi：10.1016/j.cct.2015.12.005
更新日期：2016-01-01 00:00:00
abstract：:The 2-in-1 adaptive design [Chen et al. 2018] allows seamless expansion of an ongoing Phase 2 trial into a Phase 3 trial to expedite a drug development program. An intermediate endpoint can be used for the adaptive decision. Under a mild assumption that is expected to generally hold in practice, both the Phase 2 trial...
journal_title：Contemporary clinical trials
pub_type： 杂志文章
doi：10.1016/j.cct.2020.106053
更新日期：2020-08-01 00:00:00
abstract：BACKGROUND:There are numerous potential sources of problems when performing complex clinical research trials. These issues are compounded when studies are multi-site and multiple personnel from different sites are responsible for varying actions from case report form design to primary data collection and data entry. M...
journal_title：Contemporary clinical trials
pub_type： 杂志文章
doi：10.1016/j.cct.2005.11.013
更新日期：2006-04-01 00:00:00
abstract：:Several randomized trials have found behavior change programs delivered via text messaging to be efficacious to improve preventive health behaviors such as physical activity and stopping smoking; however few have assessed its value in skin cancer prevention or early detection. The HealthyTexts study enrolled 678 parti...
journal_title：Contemporary clinical trials
pub_type： 杂志文章,随机对照试验
doi：10.1016/j.cct.2013.03.009
更新日期：2013-05-01 00:00:00
abstract：BACKGROUND:Unlike conventional time-to-event analysis of composite endpoints in clinical trials, the "win ratio" method allows for flexibility in prioritizing their components. Here, we compare the EVOLVE trial findings using the win ratio with those from time-to-event analysis. METHODS: EXPOSURE:Randomization to cin...
journal_title：Contemporary clinical trials
pub_type： 杂志文章
doi：10.1016/j.cct.2016.04.001
更新日期：2016-05-01 00:00:00
abstract：:Depression affects 20-30% of people living with HIV/AIDS (PLWHA) in the U.S. and predicts greater sexual risk behaviors, lower antiretroviral (ARV) medication adherence, and worse clinical outcomes. Yet little experimental evidence addresses the critical clinical question of whether depression treatment improves ARV a...
journal_title：Contemporary clinical trials
pub_type： 杂志文章,随机对照试验
doi：10.1016/j.cct.2012.04.002
更新日期：2012-07-01 00:00:00
abstract：:Breast cancer is the most common invasive cancer among women in developed countries. Obesity is a major risk factor for breast cancer recurrence and mortality in both pre- and postmenopausal women. Co-morbid medical conditions are common among breast cancer survivors. The Exercise and Nutrition to Enhance Recovery and...
journal_title：Contemporary clinical trials
pub_type： 杂志文章,随机对照试验
doi：10.1016/j.cct.2012.12.003
更新日期：2013-03-01 00:00:00
abstract：:The most relevant randomized controlled trials of interferon-alpha (IFN) for naive patients with chronic hepatitis C (CHC) published in a decade, just before appearance of pegylated IFN trials in 2000, were included in this paper. Its purpose is to review the relationship between sustained biochemical response in acti...
journal_title：Contemporary clinical trials
pub_type： 杂志文章,meta分析
doi：10.1016/j.cct.2004.12.009
更新日期：2005-04-01 00:00:00
abstract：:It is of vital importance to the success of a randomized clinical trial to maintain the balance of baseline characteristics (covariates) that could potentially confound the outcomes of the trial. Various randomization methods have been proposed to increase the likelihood of having balanced covariates at the end of a t...
journal_title：Contemporary clinical trials
pub_type： 杂志文章
doi：10.1016/j.cct.2011.03.004
更新日期：2011-07-01 00:00:00
