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 (in case of no expansion) and the Phase 3 trial (in case of expansion) can be tested at the full alpha level without inflating the overall Type I error of the study. Due to its flexibility and robustness, the design has quickly generated a lot of interest since its recent publication. We make three major extensions to the 2-in-1 design in this paper: 1) an increase of adaptive decisions from two to any number; 2) incorporation of the group sequential method for data monitoring; 3) expansion of the univariate design to multivariate. These extensions not only facilitate the application of 2-in-1 design in practice but also stimulate research interest in related statistical issues. Although we mainly use hypothetical trials in oncology and neuroscience for illustration, the application of the 2-in-1 design and its extensions is not limited to the two therapeutic areas.
journal_name
Contemp Clin Trialsjournal_title
Contemporary clinical trialsauthors
Chen C,Li W,Deng Qdoi
10.1016/j.cct.2020.106053subject
Has Abstractpub_date
2020-08-01 00:00:00pages
106053eissn
1551-7144issn
1559-2030pii
S1551-7144(20)30131-2journal_volume
95pub_type
杂志文章abstract：:Identifying effective strategies for treating obesity is a clinical challenge and a public health priority. The present study is an innovative test of the relative effectiveness of lottery-based financial incentives and environmental strategies on weight loss and maintenance. The Healthy Weigh study is evaluating the ...
journal_title：Contemporary clinical trials
pub_type： 杂志文章,随机对照试验
doi：10.1016/j.cct.2018.10.013
更新日期：2019-01-01 00:00:00
abstract：:Home-based telemedicine (HBT) is a validated method of evidence-based treatment delivery for posttraumatic stress disorder (PTSD), and justification for its use has centered on closing gaps related to provider availability and distance to treatment centers. However, another potential use of HBT may be to overcome barr...
journal_title：Contemporary clinical trials
pub_type： 杂志文章,随机对照试验
doi：10.1016/j.cct.2016.03.004
更新日期：2016-05-01 00:00:00
abstract：:The use of blinding strengthens the credibility of randomized controlled trials (RCTs) by minimizing bias. However, there is confusion surrounding the definition of blinding as well as the terms single, double, and triple blind. It has been suggested that these terms should be discontinued due to their broad misinterp...
journal_title：Contemporary clinical trials
pub_type： 杂志文章
doi：10.1016/j.cct.2010.11.004
更新日期：2011-03-01 00:00:00
abstract：BACKGROUND:Comparative effectiveness research (CER) evidence on childhood obesity provides the basis for effective screening and management strategies in pediatric primary care. The uses of health information technology including decision support tools in the electronic health records (EHRs), as well as remote and mobi...
journal_title：Contemporary clinical trials
pub_type： 杂志文章,多中心研究,随机对照试验
doi：10.1016/j.cct.2012.10.005
更新日期：2013-01-01 00:00:00
abstract：:Prostate cancer is a slow progressing cancer that affects millions of men in the US. Due to uncertainties in outcomes and treatment complications, it is important that patients engage in informed decision making to choose the "optimal treatment". Patient centered care that encompasses informed decision-making can impr...
journal_title：Contemporary clinical trials
pub_type： 杂志文章,随机对照试验
doi：10.1016/j.cct.2015.09.024
更新日期：2015-11-01 00:00:00
abstract：BACKGROUND:Chronic Obstructive Pulmonary Disease (COPD) frequently coexists with other diseases. Whereas COPD action plans are currently part of usual care, they are less suitable and potentially unsafe for use in the presence of comorbidities. This study evaluates whether an innovative treatment approach directed towa...
journal_title：Contemporary clinical trials
pub_type： 杂志文章,多中心研究,随机对照试验
doi：10.1016/j.cct.2013.06.003
更新日期：2013-09-01 00:00:00
abstract：:Hepatitis C virus (HCV) is a leading cause of chronic liver disease. The natural history of HCV infection is heterogeneous, and a person infected with HCV can clear the virus or progress to a chronic infection. The chronic infection can remain asymptomatic for decades before the development of liver cirrhosis and/or c...
journal_title：Contemporary clinical trials
pub_type： 杂志文章
doi：10.1016/j.cct.2008.03.006
更新日期：2008-09-01 00:00:00
abstract：BACKGROUND:Extant obesity efforts have had a limited impact among low-income underserved children, in part because existing programs are limited in terms of their short duration and low dosage, limited accessibility and sustainability; and failure to address barriers faced by diverse low-income families. METHODS:This ...
journal_title：Contemporary clinical trials
pub_type： 杂志文章,随机对照试验
doi：10.1016/j.cct.2018.12.010
更新日期：2019-02-01 00:00:00
abstract：:We developed a novel diagram to depict patient flow and outcomes in clinical trials. In contrast to flow diagrams such as the CONSORT chart, our diagram enables individual patient histories to be traced and depicts important patterns of treatment administration and outcomes, such as response and adverse events. Also, ...
journal_title：Contemporary clinical trials
pub_type： 杂志文章
doi：10.1016/j.cct.2009.01.010
更新日期：2009-05-01 00:00:00
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journal_title：Contemporary clinical trials
pub_type： 杂志文章
doi：10.1016/j.cct.2020.106238
更新日期：2020-12-05 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：:Dosing is potentially the most important decision that must be made when building or refining behavioral interventions. In this paper, we propose standardized terminology and reporting of dosing information, which would inform intervention development, refinement for dissemination, and systematic reviews of dose-respo...
journal_title：Contemporary clinical trials
pub_type： 杂志文章,评审
doi：10.1016/j.cct.2012.07.011
更新日期：2012-11-01 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：:Cancer related cognitive impairments (CRCI) are frequently reported by patients prior to, during and after medical treatment. Although this cognitive decline severely affects patients' quality of life, little is known about effective treatments. Exercise programs represent a promising supportive strategy in this field...
journal_title：Contemporary clinical trials
pub_type： 杂志文章,随机对照试验
doi：10.1016/j.cct.2016.05.007
更新日期：2016-07-01 00:00:00
abstract：INTRODUCTION:Breast cancer survivors face dual challenges: long term sequelae of treatment, and risk of recurrent disease. Obesity and a sedentary lifestyle complicate both challenges. The WISER Survivor trial assessed the effects of exercise and/or weight-loss on lymphedema, biomarkers of breast cancer recurrence, and...
journal_title：Contemporary clinical trials
pub_type： 杂志文章,随机对照试验
doi：10.1016/j.cct.2017.07.017
更新日期：2017-10-01 00:00:00
abstract：UNLABELLED:Latinos bear a disproportionate burden of the dual pandemic of obesity and diabetes. However, successful interventions addressing this disparity through primary care are lacking. To address this gap, the 5-year Vida Sana (Healthy Life) study tests a culturally adapted and technology-enhanced group-based Diab...
journal_title：Contemporary clinical trials
pub_type： 杂志文章,随机对照试验
doi：10.1016/j.cct.2016.03.003
更新日期：2016-05-01 00:00:00
abstract：:Knee osteoarthritis (OA) is a leading cause of late life pain and disability, and non-Hispanic black (NHB) adults experience greater OA-related pain and disability than non-Hispanic whites (NHWs). Recent evidence implicates psychosocial stress, cognitive-attentional processes, and altered central pain processing as co...
journal_title：Contemporary clinical trials
pub_type： 杂志文章
doi：10.1016/j.cct.2020.106159
更新日期：2020-11-01 00:00:00
abstract：BACKGROUND:Mammographic breast density is one of the strongest risk factors for breast cancer after age and family history. Mandatory breast density disclosure policies are increasing nationally without clear guidance on how to communicate density status to women. Coupling density disclosure with personalized risk coun...
journal_title：Contemporary clinical trials
pub_type： 杂志文章,多中心研究,随机对照试验
doi：10.1016/j.cct.2017.02.009
更新日期：2017-05-01 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：:Illustrated is the use of a patient enrichment adaptive design in a randomized phase II trial which allows the evaluation of treatment benefits by the biomarker expression level and makes interim adjustment according to the pre-specified rules. The design was applied to an actual phase II metastatic hepatocellular car...
journal_title：Contemporary clinical trials
pub_type： 杂志文章,随机对照试验
doi：10.1016/j.cct.2013.12.001
更新日期：2014-01-01 00:00:00
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journal_title：Contemporary clinical trials
pub_type： 杂志文章,随机对照试验
doi：10.1016/j.cct.2018.02.018
更新日期：2018-05-01 00:00:00
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journal_title：Contemporary clinical trials
pub_type： 杂志文章,随机对照试验
doi：10.1016/j.cct.2016.01.013
更新日期：2016-03-01 00:00:00
abstract：:Foundational elements of lifelong health are formed during the preschool years. Child care attendance has nearly doubled in the past 5 years making child care centers an ideal setting to establish healthy habits that prevent pediatric obesity. Despite the promising evidence of efficacy of child care-based obesity prev...
journal_title：Contemporary clinical trials
pub_type： 杂志文章,多中心研究,随机对照试验
doi：10.1016/j.cct.2019.105849
更新日期：2019-11-01 00:00:00
abstract：:In order to better characterize the safety profile of investigational new drugs (INDs) during clinical development, more interest and attention have been paid to ongoing safety monitoring and evaluation. The 2015 US FDA IND safety reporting draft guidance compels sponsors to periodically evaluate unblinded safety data...
journal_title：Contemporary clinical trials
pub_type： 杂志文章
doi：10.1016/j.cct.2019.06.022
更新日期：2019-08-01 00:00:00
abstract：INTRODUCTION:In promoting the health of chronically ill children, the hospital should help children cope both medically and psychosocially with their disease. The University Children's Hospital of Nancy proposes adapted physical activity for hospitalized children, but the practice during hospitalization is not well dev...
journal_title：Contemporary clinical trials
pub_type： 杂志文章,随机对照试验
doi：10.1016/j.cct.2009.12.003
更新日期：2010-03-01 00:00:00
abstract：:An instrumental variable (IV) estimator has been widely used to estimate causal effects among compliers in randomized trials with noncompliance. The estimator of complier average treatment effect can be expressed as a ratio of two unbiased estimators but the ratio estimator is not unbiased. The bias of IV estimator ca...
journal_title：Contemporary clinical trials
pub_type： 杂志文章
doi：10.1016/j.cct.2012.03.013
更新日期：2012-07-01 00:00:00
abstract：:While investigators designing clinical trials face the important issue of endpoint selection, an equally troublesome concern can be the a priori selection of the endpoint analysis. In this latter circumstance, there may be only one endpoint of interest in the clinical trial, but several competing endpoint analyses are...
journal_title：Contemporary clinical trials
pub_type： 杂志文章
doi：10.1016/j.cct.2006.05.010
更新日期：2007-01-01 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：:Growing Right Onto Wellness (GROW) is a randomized controlled trial that tests the efficacy of a family-centered, community-based, behavioral intervention to prevent childhood obesity among preschool-aged children. Focusing on parent-child pairs, GROW utilizes a multi-level framework, which accounts for macro (i.e., b...
journal_title：Contemporary clinical trials
pub_type： 杂志文章,随机对照试验
doi：10.1016/j.cct.2013.08.013
更新日期：2013-11-01 00:00:00
abstract：:The prevalence of obesity is high resulting from chronic imbalances between energy intake and expenditure. On the expenditure side, regular exercise is associated with health benefits, including enhanced brain function. The benefits of exercise are not immediate and require persistence to be realized. Brain regions as...
journal_title：Contemporary clinical trials
pub_type： 杂志文章
doi：10.1016/j.cct.2013.11.007
更新日期：2014-01-01 00:00:00
